title: Cochlear-Implanted Recipient Observational Study

acronym: IROS

summary:
The purpose of this study is to collect patient related benefit data following treatment for permanent hearing loss with a hearing implant from the company Cochlear over a period of 2 years post treatment.

Assessment of benefits is based on standard questionnaires of hearing ability and quality of life in general.

ReferenceCitation: Maes IH, Joore MA, Cima RF, Vlaeyen JW, Anteunis LJ. Assessment of health state in patients with tinnitus: a comparison of the EQ-5D and HUI mark III. Ear Hear. 2011 Jul-Aug;32(4):428-35. doi: 10.1097/AUD.0b013e3181fdf09f.

ReferenceCitation: Noble W, Tyler R, Dunn C, Bhullar N. Unilateral and bilateral cochlear implants and the implant-plus-hearing-aid profile: comparing self-assessed and measured abilities. Int J Audiol. 2008 Aug;47(8):505-14. doi: 10.1080/14992020802070770.

ReferenceCitation: Mauch H, Kaur J, Irwin C, Wyss J. Design, implementation, and management of an international medical device registry. Trials. 2021 Nov 25;22(1):845. doi: 10.1186/s13063-021-05821-5.

family: Mauch

full_name: Herbert Mauch

given: Herbert

family: Kaur

full_name: Jasmin Kaur

given: Jasmin

family: Irwin

full_name: Colin Irwin

given: Colin

family: Wyss

full_name: Josie Wyss

given: Josie

journal_name: Trials

lead_sponsor: Cochlear

location_country:
Austria
Brazil
South Africa
Sweden
Netherlands
Spain
France
Poland
Hungary
Argentina
Belgium
Portugal
Turkey
Germany
Slovenia
Colombia

location_facility: Uniwersytecki Szpital Kliniczny w Bialymstoku

location_facility: Wojewodzki Szpital Dzieciecy w Bydgoszczy

location_facility: Samodzielny Publiczny Szpital Kliniczny nr 4 w Lublinie

location_facility: Instituto Nacional de Otologia Garcia Gomez

location_facility: Durban Cochlear Implant Pogramme, Parklands Hospital

location_facility: Universitätsklinik für Hals-, Nasen- und Ohrenheilkunde

location_facility: Hospital Clinico Universitario de Salamanca

location_facility: Hospital Universitario Clinica San Rafael

location_facility: Samodzielny Publiczny Szpital Kliniczny nr 1 w Szczecinie

location_facility: Instytut Centrum Zdrowia Matki Polki w Lodzi

location_facility: Centro de Investigaciones Otoaudiologicas (CIOA)

location_facility: Hospital Clinico Universitario de Valencia

location_facility: Johannesburg Cochlear Implant Center, Wits Donald Gordon Medical Centre

location_facility: Hospital General Universitario Gregorio Maranon

location_facility: Uniwersytecki Szpital Kliniczny nr 1 w Lodzi

location_facility: Hospital Clinico Universitario Lozano Blesa

location_facility: Samodzielny Publiczny Szpital Kliniczny nr 2 w Poznaniu

location_facility: Implantes Cocleares Equipo Multicentrico (ICEM)


type: EudraCT Number

type: Other Identifier

title: A PHASE 4, RANDOMIZED, DOUBLE-BLIND, PLACEBO-CONTROLLED STUDY OF CONTINUED ENZALUTAMIDE TREATMENT BEYOND PROGRESSION IN PATIENTS WITH CHEMOTHERAPY-NAÏVE METASTATIC CASTRATION-RESISTANT PROSTATE CANCER

acronym: PLATO

summary: The purpose of this study is to determine if continued treatment with Enzalutamide is effective in patients with metastatic prostate cancer.

study_start_date_type: Actual

status: Active, not recruiting

study_completion_date_type: Anticipated

study_type: Interventional

design_allocation: Randomized

primary_purpose: Treatment

enrollment_type: Actual

ReferenceType: derived

ReferenceCitation: Attard G, Borre M, Gurney H, Loriot Y, Andresen-Daniil C, Kalleda R, Pham T, Taplin ME; PLATO collaborators. Abiraterone Alone or in Combination With Enzalutamide in Metastatic Castration-Resistant Prostate Cancer With Rising Prostate-Specific Antigen During Enzalutamide Treatment. J Clin Oncol. 2018 Sep 1;36(25):2639-2646. doi: 10.1200/JCO.2018.77.9827. Epub 2018 Jul 20.

name: Gerhardt Attard, The Institute of Cancer Research and the Royal Marsden National Health Service Foundation Trust, London, United Kingdom; Michael Borre, Aarhus University Hospital, Aarhus, Denmark; Howard Gurney, Macquarie University, Sydney, New South Wales, Australia; Yohann Loriot, Gustave Roussy, Institut National de la Santé et de la Recherche Médicale U981, University of Paris Saclay, Villejuif, France; Corina Andresen-Daniil, Ranjith Kalleda, and Trinh Pham, Pfizer, New York, NY; and Mary-Ellen...

family: Attard

full_name: Gerhardt Attard

given: Gerhardt

sequence: first

family: Borre

full_name: Michael Borre

given: Michael

sequence: additional

family: Gurney

full_name: Howard Gurney

given: Howard

family: Loriot

full_name: Yohann Loriot

given: Yohann

family: Andresen-Daniil

full_name: Corina Andresen-Daniil

given: Corina

family: Kalleda

full_name: Ranjith Kalleda

given: Ranjith

family: Pham

full_name: Trinh Pham

given: Trinh

family: Taplin

full_name: Mary-Ellen Taplin

given: Mary-Ellen

bso_classification: Medical research

datasource: user

genre: journal-article

genre_raw: journal-article

journal_name: Journal of Clinical Oncology

oa_colors: green hybrid

oa_host_type: publisher;repository

evidence: open (via page says license)

host_type: publisher

license: cc-by-nc-nd

license_normalized: cc-by-nc-nd

version: publishedVersion

evidence: oa repository (via OAI-PMH doi match)

host_type: repository

repository_institution: PubMed Central - Europe PMC

repository_normalized: PubMed Central

repository_institution: Elsevier - Scopus

repository_normalized: Pure (Denmark)

evidence: oa repository (via OAI-PMH title and first author match)

repository_institution: University College London - UCL Discovery

repository_normalized: UCL Discovery

evidence: oa repository (via pmcid lookup)

license_normalized: no license

evidence: oa repository (semantic scholar lookup)

version: submittedVersion

repositories:
PubMed Central
Pure (Denmark)
UCL Discovery
pdfs.semanticscholar.org

publisher: American Society of Clinical Oncology (ASCO)

ipd_sharing: Yes

ipd_sharing_description: Pfizer will provide access to individual de-identified participant data and related study documents (e.g. protocol, Statistical Analysis Plan (SAP), Clinical Study Report (CSR)) upon request from qualified researchers, and subject to certain criteria, conditions, and exceptions. Further details on Pfizer's data sharing criteria and process for requesting access can be found at: https://www.pfizer.com/science/clinical_trials/trial_data_and_results/data_requests.

lead_sponsor: Pfizer

location_country:
Italy
Sweden
Finland
France
Slovakia
Belgium
Australia
Spain
Denmark
United States
United Kingdom

location_facility: Helsingin yliopistollinen keskussairaala, Meilahden sairaala

location_facility: Azienda Ospedaliera S. Camillo Forlanini, UOC per il governo clinico in Oncologia Medica

location_facility: U.O. Oncologia Medica, IRCCS Istituto Scientifico Romagnolo per lo Studio e la Cura

location_facility: SS Medicina Nucleare, A.O.U. San Luigi Gonzaga

location_facility: Uroexam, spol. s r.o., Urologicka ambulancia

location_facility: North Shore Radiology and Nuclear Medicine

location_facility: GAMMALAB, spol.s r.o., Oddelenie nuklearnej mediciny

location_facility: Port Macquarie Base Hospital,North Coast Cancer Institute

location_facility: Farmacia Ospedaliera, A.O.U. San Luigi Gonzaga

location_facility: Farmacia, Azienda Ospedaliera S. Camillo Forlanini

location_facility: Hospital Universitario Madrid Sanchinarro

location_facility: Fakultna nemocnica s poliklinikou F.D. Roosevelta

location_facility: SCDU Radiodiagnostica, A.O.U. San Luigi Gonzaga

location_facility: Institut nuklearnej a molekularnej mediciny

location_facility: Institut Gustave Roussy - Service d'Urologie

location_facility: UOC Radiologia Piasta, Azienda Ospedaliera S. Camillo Forlanini

location_facility: Epic Pharmacy Port Macquarie base hospital

location_facility: ALTAHIA, Xarxa Assistencial Universitaria de Manresa

location_facility: Concord Cancer Centre, Medical Oncology Department

location_facility: University College London Hospitals NHS Foundation Trust

location_facility: Servizio di Radiologia, Azienda Ospedaliera "Istituti Ospitalieri" di Cremona

location_facility: SCDU Oncologia Medica II Pad, A.O.U. San Luigi Gonzaga

location_facility: Tenpharm Pty Ltd trading as EPIC Pharmacy Tennyson

location_facility: Apoteket AB Kliniska Provningar Molnlycke

location_facility: U.O di Oncologia-Presidio Ospedaliero di Cremona-Istituti Ospitalieri di Cremona

location_facility: CO Badalona-Instituto Germans Trias i Pujol

location_facility: Farmacia, Azienda Ospedaliera "Istituti Ospitalieri" di Cremona

location_facility: Medicina Nucleare, Azienda Ospedaliera "Istituti Ospitalieri" di Cremona

intervention_type: Drug

lead_sponsor_type: industriel

status_simplified: Ongoing


status: Completed

lead_sponsor: Ultragenyx Pharmaceutical Inc

location_country:
Italy
France
Israel
Australia
Spain
United States
United Kingdom

location_facility: Service de Neurologie Pédiatrique et des Maladies Métaboliques - INSERM U1141 Hôpital Robert Debré - APHP

location_facility: Children's Hospital Colorado - University of Colorado, Denver, School of Medicine

location_facility: Unita' Operativa Neurologia Pediatrica e Malattie Muscolari Istituto "Giannina Gaslini"

location_facility: Columbia University - Department of Neurology

location_facility: University of Texas Neurometabolic Clinic

study_completion_date_type: Actual

source: euctr

title: A Randomized, Double-blind, Placebo-controlled, Parallel-group, Study to Assess the Safety and Efficacy of UX007 in Subjects With Glucose Transporter Type 1 Deficiency Syndrome

summary: The primary objectives of the study are to evaluate the efficacy of UX007 compared to placebo as measured by the reduction from randomization to Week 8 in frequency of seizures and to evaluate the safety of UX007 via adverse event (AE) rates, laboratory values, and electrocardiogram (ECG).

status_simplified: Completed


title: An Open-Label, Multicenter Study To Evaluate The Efficacy And Safety Of Sofosbuvir/Ledipasvir Fixed-Dose Combination ± Ribavirin For 12 or 24 Weeks In Chronic Genotype 1 HCV Infected Subjects Who Participated In A Prior Gilead-Sponsored HCV Treatment Study

summary: The primary objectives of this study are to evaluate the efficacy, safety, and tolerability of ledipasvir/sofosbuvir (LDV/SOF) fixed-dose combination (FDC) with or without ribavirin (RBV) in participants with chronic genotype 1 hepatitis C virus (HCV) infection who have participated in a prior Gilead-sponsored HCV treatment study, and who did not achieve sustained virologic response (SVR24), defined as HCV RNA < lower limit of quantification (LLOQ) 24 weeks after last dose of study drug (SVR24).

design_allocation: Non-Randomized

ReferenceType: background

ReferenceCitation: Lawitz E, Pockros PJ, Yang JC, Pang PS, Zhu Y, Svarovskaia E, et al. Ledipasvir/sofosbuvir regimens for the retreatment of patients who failed sofosbuvir-based regimens [Abstract 10868]. Presented at: The 25th Conference of the Asian Pacific Association for the Study of Liver (APASL); 2016 February 20-24; Tokyo, Japan.

ReferenceCitation: Lawitz E, Flamm S, Yang JC, Pang PS, Zhu Y, Svarovskaia E, et al. Retreatment of patients who failed 8 or 12 weeks of ledipasvir/sofosbuvir-based regimens with ledipasvir/sofosbuvir for 24 weeks [Abstract 1627]. Presented at: The 50th Annual Congress of the European Association for the Study of Liver: The International Liver Congress (EASL); 2015 April 22-26; Vienna, Austria

ReferenceType: result

ReferenceCitation: Wyles D, Pockros P, Morelli G, Younes Z, Svarovskaia E, Yang JC, Pang PS, Zhu Y, McHutchison JG, Flamm S, Lawitz E. Ledipasvir-sofosbuvir plus ribavirin for patients with genotype 1 hepatitis C virus previously treated in clinical trials of sofosbuvir regimens. Hepatology. 2015 Jun;61(6):1793-7. doi: 10.1002/hep.27814. Epub 2015 Apr 27.

family: Wyles

full_name: David Wyles

given: David

family: Pockros

full_name: Paul Pockros

given: Paul

name: University of Florida Health; Gainesville FL

family: Morelli

full_name: Giuseppe Morelli

given: Giuseppe

family: Younes

full_name: Ziad Younes

given: Ziad

family: Svarovskaia

full_name: Evguenia Svarovskaia

given: Evguenia

family: Yang

full_name: Jenny C. Yang

given: Jenny C.

family: Pang

full_name: Phillip S. Pang

given: Phillip S.

family: Zhu

full_name: Yanni Zhu

given: Yanni

family: McHutchison

full_name: John G. McHutchison

given: John G.

name: Northwestern University Feinberg School of Medicine; Chicago IL

family: Flamm

full_name: Steven Flamm

given: Steven

name: Texas Liver Institute and the University of Texas Health Science Center; San Antonio TX

family: Lawitz

full_name: Eric Lawitz

given: Eric

journal_name: Hepatology

publisher: Wiley

publisher_dissemination: Wiley

publisher_group: Wiley

publisher_normalized: Wiley

ipd_sharing_description: Qualified external researchers may request IPD for this study after study completion. For more information, please visit our website at http://www.gilead.com/research/disclosure-and-transparency.

lead_sponsor: Gilead Sciences

location_country:
France
Puerto Rico
Australia
Spain
United States


ipd_sharing_description: Qualified researchers may request access to individual patient level data through the clinical study data request platform (www.vivli.org). Further details on Roche's criteria for eligible studies are available here (https://vivli.org/ourmember/roche/). For further details on Roche's Global Policy on the Sharing of Clinical Information and how to request access to related clinical study documents, see here (https://www.roche.com/research_and_development/who_we_are_how_we_work/clinical_trials/our_commitment_to_data_sharing.htm).

lead_sponsor: Hoffmann-La Roche

location_country:
Italy
United States
Netherlands
France
Israel
Germany
United Kingdom
Canada

location_facility: Johannes Wesling Academic Medical Center

title: A Phase 2, Prospective Study Of PRM-151 In Subjects With Primary Myelofibrosis (PMF), Post-Polycythemia Vera MF (Post-PV MF), Or Post-Essential Thrombocythemia MF (Post-ET MF)

summary:
PRM-151 is an investigational drug that is being developed for possible use in the treatment of myelofibrosis (MF), a disease in which the bone marrow, which is the organ in the body that makes blood cells, is replaced by fibrosis, or excess scar tissue.

The purpose of this study is to gather information on whether PRM-151 has an effect on the MF disease, whether it is safe in patients with MF, and how well it is tolerated.


title: Natural History of Progression of Atrophy Secondary to Stargardt Disease: Retrospective, and Prospective Longitudinal Observational Study Incl. Ancillary SMART Study- Scotopic Microperimetric Assessment of Rod Function in Stargardt Disease

acronym: ProgSTAR

summary:
Stargardt disease is currently an incurable and untreatable macular dystrophy that causes severe visual loss in children and young adults, thereby causing enormous morbidity with economic, psychological, emotional, and social implications. There are no FDA approved therapeutic treatments for this disease. Therefore, the objective of this study is to collect natural history data from a large population of children and adults in order to evaluate possible efficacy measures for planned clinical trials.

Participants will be recruited from each Investigator's own patient population as the study requires the availability of both multiyear retrospective data, as well as ongoing prospectively collected data. A concurrent ancillary study (SMART study) is also being conducted with a subset of the prospective study patients during their regular ProgSTAR study visits to expand the collection of retinal images to include microperimetry measurements gathered under scotopic (low light) conditions.

study_type: Observational

time_perspective: Other

ReferenceCitation: Schönbach EM, Ibrahim MA, Strauss RW, Birch DG, Cideciyan AV, Hahn GA, Ho A, Kong X, Nasser F, Sunness JS, Zrenner E, Sadda SR, West SK, Scholl HPN; Progression of Stargardt Disease Study Group. Fixation Location and Stability Using the MP-1 Microperimeter in Stargardt Disease: ProgStar Report No. 3. Ophthalmol Retina. 2017 Jan - Feb;1(1):68-76. doi: 10.1016/j.oret.2016.08.009. Epub 2016 Oct 31.

ReferenceCitation: Kong X, West SK, Strauss RW, Munoz B, Cideciyan AV, Michaelides M, Ho A, Ahmed M, Schönbach EM, Cheetham JK, Ervin AM, Scholl HPN; ProgStar study group. Progression of Visual Acuity and Fundus Autofluorescence in Recent-Onset Stargardt Disease: ProgStar Study Report #4. Ophthalmol Retina. 2017 Nov - Dec;1(6):514-523. doi: 10.1016/j.oret.2017.02.008. Epub 2017 Apr 28.

ReferenceCitation: Strauss RW, Muñoz B, Ho A, Jha A, Michaelides M, Mohand-Said S, Cideciyan AV, Birch D, Hariri AH, Nittala MG, Sadda S, Scholl HPN; ProgStar Study Group. Incidence of Atrophic Lesions in Stargardt Disease in the Progression of Atrophy Secondary to Stargardt Disease (ProgStar) Study: Report No. 5. JAMA Ophthalmol. 2017 Jul 1;135(7):687-695. doi: 10.1001/jamaophthalmol.2017.1121.

ReferenceCitation: Schönbach EM, Wolfson Y, Strauss RW, Ibrahim MA, Kong X, Muñoz B, Birch DG, Cideciyan AV, Hahn GA, Nittala M, Sunness JS, Sadda SR, West SK, Scholl HPN; ProgStar Study Group. Macular Sensitivity Measured With Microperimetry in Stargardt Disease in the Progression of Atrophy Secondary to Stargardt Disease (ProgStar) Study: Report No. 7. JAMA Ophthalmol. 2017 Jul 1;135(7):696-703. doi: 10.1001/jamaophthalmol.2017.1162.

ReferenceCitation: Strauss RW, Ho A, Muñoz B, Cideciyan AV, Sahel JA, Sunness JS, Birch DG, Bernstein PS, Michaelides M, Traboulsi EI, Zrenner E, Sadda S, Ervin AM, West S, Scholl HP; Progression of Stargardt Disease Study Group. The Natural History of the Progression of Atrophy Secondary to Stargardt Disease (ProgStar) Studies: Design and Baseline Characteristics: ProgStar Report No. 1. Ophthalmology. 2016 Apr;123(4):817-28. doi: 10.1016/j.ophtha.2015.12.009. Epub 2016 Jan 16.

family: Strauss

full_name: Rupert W. Strauss

given: Rupert W.

family: Ho

full_name: Alex Ho

given: Alex

family: Muñoz

full_name: Beatriz Muñoz

given: Beatriz

family: Cideciyan

full_name: Artur V. Cideciyan

given: Artur V.

family: Sahel

full_name: José-Alain Sahel

given: José-Alain

family: Sunness

full_name: Janet S. Sunness

given: Janet S.

family: Birch

full_name: David G. Birch

given: David G.

family: Bernstein

full_name: Paul S. Bernstein

given: Paul S.

family: Michaelides

full_name: Michel Michaelides

given: Michel

family: Traboulsi

full_name: Elias I. Traboulsi

given: Elias I.

family: Zrenner

full_name: Eberhart Zrenner

given: Eberhart

family: Sadda

full_name: SriniVas Sadda

given: SriniVas

family: Ervin

full_name: Ann-Margret Ervin

given: Ann-Margret

family: West

full_name: Sheila West

given: Sheila

family: Scholl

full_name: Hendrik P.N. Scholl

given: Hendrik P.N.

journal_name: Ophthalmology

publisher: Elsevier BV

publisher_dissemination: Elsevier

publisher_group: Elsevier

publisher_normalized: Elsevier

ReferenceCitation: Kong X, Strauss RW, Michaelides M, Cideciyan AV, Sahel JA, Muñoz B, West S, Scholl HP; ProgStar Study Group. Visual Acuity Loss and Associated Risk Factors in the Retrospective Progression of Stargardt Disease Study (ProgStar Report No. 2). Ophthalmology. 2016 Sep;123(9):1887-97. doi: 10.1016/j.ophtha.2016.05.027. Epub 2016 Jul 2.

family: Kong

full_name: Xiangrong Kong

given: Xiangrong

repositories_institution: University College London - UCL Discovery

ReferenceCitation: Kong X, Strauss RW, Cideciyan AV, Michaelides M, Sahel JA, Munoz B, Ahmed M, Ervin AM, West SK, Cheetham JK, Scholl HPN; ProgStar Study Group. Visual Acuity Change over 12 Months in the Prospective Progression of Atrophy Secondary to Stargardt Disease (ProgStar) Study: ProgStar Report Number 6. Ophthalmology. 2017 Nov;124(11):1640-1651. doi: 10.1016/j.ophtha.2017.04.026. Epub 2017 May 23.

ReferenceCitation: Strauss RW, Muñoz B, Ho A, Jha A, Michaelides M, Cideciyan AV, Audo I, Birch DG, Hariri AH, Nittala MG, Sadda S, West S, Scholl HPN; ProgStar Study Group. Progression of Stargardt Disease as Determined by Fundus Autofluorescence in the Retrospective Progression of Stargardt Disease Study (ProgStar Report No. 9). JAMA Ophthalmol. 2017 Nov 1;135(11):1232-1241. doi: 10.1001/jamaophthalmol.2017.4152.

name: Wilmer Eye Institute, Johns Hopkins University, Baltimore, Maryland2Moorfields Eye Hospital National Health Service Foundation Trust, London, England3University College London Institute of Ophthalmology, University College London, London, England4Department of Ophthalmology, Medical University Graz and Johannes Kepler University Linz, Linz, Austria5Department of Ophthalmology, University of Basel, Basel, Switzerland

name: Doheny Eye Institute, David Geffen School of Medicine at University of California–Los Angeles

name: Scheie Eye Institute, Perelman School of Medicine, University of Pennsylvania, Philadelphia

name: Sorbonne Universités, University Pierre et Marie Curie Université de Paris 06, Paris, France9Institut National de la Santé et de la Recherche Médicale, Centre Hospitalier National d’Ophtalmologie des Quinze-Vingts, Paris, France10Centre National de la Recherche Scientifique, Institut de la Vision, Paris, France11Centre Hospitalier National d’Ophtalmologie des Quinze-Vingts, Paris, France

name: Retina Foundation of the Southwest, Dallas, Texas

name: Wilmer Eye Institute, Johns Hopkins University, Baltimore, Maryland5Department of Ophthalmology, University of Basel, Basel, Switzerland

ReferenceCitation: Kong X, Fujinami K, Strauss RW, Munoz B, West SK, Cideciyan AV, Michaelides M, Ahmed M, Ervin AM, Schönbach E, Cheetham JK, Scholl HPN; ProgStar Study Group. Visual Acuity Change Over 24 Months and Its Association With Foveal Phenotype and Genotype in Individuals With Stargardt Disease: ProgStar Study Report No. 10. JAMA Ophthalmol. 2018 Aug 1;136(8):920-928. doi: 10.1001/jamaophthalmol.2018.2198.

name: School of Public Health and Health Sciences, University of Massachusetts-Amherst, Amherst

name: Bloomberg School of Public Health, Johns Hopkins University, Baltimore, Maryland

name: Laboratory of Visual Physiology, Division for Vision Research, National Institute of Sensory Organs, National Hospital Organization, Tokyo Medical Center, Tokyo, Japan

name: Department of Ophthalmology, Keio University, School of Medicine, Tokyo, Japan

name: Moorfields Eye Hospital, London, United Kingdom

name: UCL Institute of Ophthalmology, University College London, London, United Kingdom

name: Department of Ophthalmology, Johannes Kepler University (Clinic) Linz, Linz, Austria

name: Department of Ophthalmology, Medical University Graz, Graz, Austria

name: Foundation Fighting Blindness, Clinical Research Institute, Columbia, Maryland

name: Institute of Molecular and Clinical Ophthalmology Basel, Basel, Switzerland

location_country:
Germany
United States
United Kingdom
France

location_facility: Scheie Eye Institute, University of Pennsylvania

location_facility: Center for Ophthalmic Research, University of Teubingen


title: Assessment of the Efficacy and Safety of a New Medical Device in the Local Treatment of Diabetic Foot Ulcers: a Prospective, Randomized, Controlled, Double-blind, European Multicentre Clinical Trial

acronym: DISCOVERY

summary: Evaluation of the efficacy and safety of a device with LU3103209 compound versus the same device, free of LU3103209 compound, in the local management of DFUs.

status: Unknown status

enrollment_type: Anticipated

ipd_sharing: No

lead_sponsor: Laboratoires URGO

location_country:
Italy
France
Czechia
Poland
Spain
United Kingdom

location_facility:
Pr PETIT
Pr LAZARO
Dr KORZON
Pr EDMONDS
Pr PIAGGESI
Pr JIRKOVSKA

intervention_type: Device

status_simplified: Unknown


title: Study of Early Endocrine Profile in Infants With Prader-Willi Syndrome (PWS) in Order to Unravel the Switch From Early Feeding Difficulties to Obesity and Hyperphagia.

summary:
The present project aims to determine the underlying mechanisms for the switch from failure to thrive to excessive weight gain and hyperphagia with impaired satiety in PWS. The primary objective is to describe the evolution of circulating hormones involved in feeding and appetite regulation during the 4 first years of life. The secondary objective is to make this blood bank available for other research projects and particularly the investigation of hormones involved in hypogonadism.

Over the last ten years, the age at diagnosis in PWS has fallen significantly and the majority of cases is now diagnosed during the 1st trimester of life giving the possibility to collect precise clinical data and serum samples at early stages. The investigators of the project are involved in the care of patients with PWS and have a devoted clinic and an organized network in their country through clinical networks or patient associations.

primary_purpose: Basic Science

ReferenceCitation: Cadoudal T, Buléon M, Sengenès C, Diene G, Desneulin F, Molinas C, Eddiry S, Conte-Auriol F, Daviaud D, Martin PG, Bouloumié A, Salles JP, Tauber M, Valet P. Impairment of adipose tissue in Prader-Willi syndrome rescued by growth hormone treatment. Int J Obes (Lond). 2014 Sep;38(9):1234-40. doi: 10.1038/ijo.2014.3. Epub 2014 Jan 10.

family: Cadoudal

full_name: T Cadoudal

given: T

family: Buléon

full_name: M Buléon

given: M

family: Sengenès

full_name: C Sengenès

given: C

family: Diene

full_name: G Diene

given: G

family: Desneulin

full_name: F Desneulin

given: F

family: Molinas

full_name: C Molinas

family: Eddiry

full_name: S Eddiry

given: S

family: Conte-Auriol

full_name: F Conte-Auriol

family: Daviaud

full_name: D Daviaud

given: D

family: Martin

full_name: P G P Martin

given: P G P

family: Bouloumié

full_name: A Bouloumié

given: A

family: Salles

full_name: J-P Salles

given: J-P

family: Tauber

full_name: M Tauber

family: Valet

full_name: P Valet

given: P

journal_name: International Journal of Obesity

oa_host_type: publisher

evidence: open (via free pdf)

publisher: Springer Science and Business Media LLC

publisher_dissemination: Springer

publisher_group: Springer

publisher_normalized: Springer

title: Impairment of adipose tissue in Prader–Willi syndrome rescued by growth hormone treatment

ReferenceCitation: Beauloye V, Diene G, Kuppens R, Zech F, Winandy C, Molinas C, Faye S, Kieffer I, Beckers D, Nergårdh R, Hauffa B, Derycke C, Delhanty P, Hokken-Koelega A, Tauber M. High unacylated ghrelin levels support the concept of anorexia in infants with prader-willi syndrome. Orphanet J Rare Dis. 2016 May 4;11(1):56. doi: 10.1186/s13023-016-0440-0.

family: Beauloye

full_name: Veronique Beauloye

given: Veronique

full_name: Gwenaelle Diene

given: Gwenaelle

family: Kuppens

full_name: Renske Kuppens

given: Renske

family: Zech

full_name: Francis Zech

given: Francis

family: Winandy

full_name: Coralie Winandy

given: Coralie

location_country:
Sweden
Netherlands
France
Belgium
Germany
United Kingdom

location_facility: Unité d'Endocrinologie Pédiatrique / Université Catholique de Louvain

location_facility: Department of Endocrinology / University Children's Hospital

location_facility: Metabolic & Molecular Imaging Group / MRC Clinical Sciences Centre / Imperial College London / Hammersmith Hospital

location_facility: Department of Pediatrics / Division of Endocrinology / Erasmus University Medical Center / Sophia Children's Hospital Rotterdam


title: Phase 2/3 Study of Masitinib in Combination With Gemcitabine Versus Gemcitabine Alone in Advanced/Metastatic Epithelial Ovarian Cancer Patients in Second Line Being Refractory to First Line Platinum Treatment or in Third Line

summary: The objective is to evaluate the efficacy and safety of masitinib in combination with gemcitabine in refractory ovarian cancer patients.

lead_sponsor: AB Science

location_country:
France
Russian Federation
Algeria
Spain
United Kingdom
Ukraine

location_facility: Complejo Hospitalario Universitario de Santiago

location_facility: Centre hospitalo-universitaire de Beni Messous

location_facility: Lviv State Oncological Regional Treatment and Diagnostic Center


title: Prospective, Open Label, Multicenter, Efficacy and Safety Study of Several Infusions of HepaStem in Urea Cycle Disorders Paediatric Patients

summary: The aim of the study is to assess the efficacy of HepaStem treatment in paediatric patients suffering from urea cycle disorders.

lead_sponsor: Promethera Biosciences

location_country: Spain Belgium France Poland

location_facility: Hospital Universitari Vall d'Hebron de Barcelona

location_facility: Instytut - Pomnik Centrum Zdrowia Dziecka

intervention_type: Biological


title: A Comparison of Glycosade® and Uncooked Cornstarch (UCCS) for the Dietary Management of Hepatic Glycogen Storage Diseases (GSD)

acronym: Glyde

summary: To compare efficacy of Glycosade® with uncooked corn starch (UCCS for the dietary management of hepatic glycogen storage diseases (GSD).

ipd_sharing: Undecided

lead_sponsor: Vitaflo International, Ltd

location_country:
United States
United Kingdom
France
Netherlands

location_facility: Great Ormond Street Hospital for Children

location_facility: National Hospital for Neurology and Neurosurgery

intervention_type: Dietary Supplement


title: EUS-guided Fine Needle Tissue Acquisition Using a Newly Developed Nitinol Ultra Flex 19 Gauge Needle for Transduodenal Lesions: a Multicenter Prospective Feasibility Study

acronym: NIFLEX

summary: This study evaluates the feasibility, safety and accuracy of a 19 gauge (19G) needle in nitinol in the performance of endoscopic ultrasound (EUS) guided transduodenal biopsy for the acquisition of samples for histologic analysis. Patients with lesions that can be approached only from the duodenum will be prospectively enrolled.

primary_purpose: Diagnostic

ReferenceCitation: Varadarajulu S, Bang JY, Hebert-Magee S. Assessment of the technical performance of the flexible 19-gauge EUS-FNA needle. Gastrointest Endosc. 2012 Aug;76(2):336-43. doi: 10.1016/j.gie.2012.04.455.

family: Varadarajulu

full_name: Shyam Varadarajulu

given: Shyam

family: Bang

full_name: Ji Young Bang

given: Ji Young

family: Hebert-Magee

full_name: Shantel Hebert-Magee

given: Shantel

journal_name: Gastrointestinal Endoscopy

lead_sponsor: Catholic University of the Sacred Heart

location_country:
Italy
United States
France
Japan

location_facility: Teikyo University Mizonokuchi Hospital, Departement of Gastroenterology

location_facility: Digestive Endoscopy Unit, Universita' Cattolica del Sacro Cuore

location_facility: Indiana Univerisity Health Medical Center

lead_sponsor_type: academique


title: A Multicentre Observational Study to Evaluate Clinical Outcomes of the G7 Acetabular System

summary: The primary purpose of this study is to evaluate the clinical and radiographic performance of the G7 Acetabular Cup System.

time_perspective: Retrospective

lead_sponsor: Zimmer Biomet

location_country:
United States
Netherlands
France
Ireland
Spain
Germany

location_facility: Midwestern Regional Orthopaedic Hospital


title: An Exploratory Phase 2 a Study to Assess Safety, Tolerability, Pharmacodynamic and Pharmacokinetics of VAS203 in Patients With Moderate and Severe Traumatic Brain Injury

acronym: NOSTRA

summary:
In the exploratory multi-center Phase 2 a study safety, tolerability, pharmacodynamics and pharmacokinetics of the Nitric Oxide Synthase inhibitor VAS203 is assessed in patients with moderate and severe traumatic brain injury. Traumatic brain injury patients (32 males) receive 15, 20 and 30 mg/kg VAS203, respectively, by continuous infusion in three cohorts (Cohort 1 open; Cohorts 2 and 3 double blind, randomised placebo-controlled). End of Study for all patients will be Day 14; adverse events and concomitant medications will be documented throughout the study.

Objectives are to assess safety and tolerability of VAS203, to evaluate concentrations of metabolites of VAS203 in plasma and microdialysate and to assess pharmacodynamic effects of VAS203 on surrogate parameters. Safety parameter will include vital signs (blood pressure heart rate, respiration rate, oxygen saturation and blood gases), fluid balance, ECG, laboratory examinations (clinical chemistry, liver function, haematology/coagulation, urinalysis, renal parameters) and adverse events. Concentration of VAS203 will be determined in plasma and microdialysate. Pharmacodynamic parameters will include intracranial pressure (ICP), biochemical parameters in microdialysate (nitrite/nitrate, arginine, citrulline, pyruvate, lactate, glucose), Partial Oxygen Pressure in brain parenchyma and Therapy Intensity Level (TIL).

ReferenceCitation: Stover JF, Belli A, Boret H, Bulters D, Sahuquillo J, Schmutzhard E, Zavala E, Ungerstedt U, Schinzel R, Tegtmeier F; NOSTRA Investigators. Nitric oxide synthase inhibition with the antipterin VAS203 improves outcome in moderate and severe traumatic brain injury: a placebo-controlled randomized Phase IIa trial (NOSTRA). J Neurotrauma. 2014 Oct 1;31(19):1599-606. doi: 10.1089/neu.2014.3344. Epub 2014 Jul 28.

name: University Hospital Zuerich, Zuerich, Switzerland.

family: Stover

full_name: John F. Stover

given: John F.

name: University Hospital Southampton, Southampton, United Kingdom.

family: Belli

full_name: Antonio Belli

given: Antonio

family: Boret

full_name: Henry Boret

given: Henry

family: Bulters

full_name: Diederik Bulters

given: Diederik

name: Vall d'Hebron University Hospital, Universitat Autònoma de Barcelona, Barcelona, Spain.

family: Sahuquillo

full_name: Juan Sahuquillo

given: Juan

family: Schmutzhard

full_name: Erich Schmutzhard

given: Erich

name: Hospital Clinic, University of Barcelona, IDIBAPS, Barcelona, Spain.

family: Zavala

full_name: Elisabeth Zavala

given: Elisabeth

name: Karolinska Institutet, Stockholm, Sweden.

family: Ungerstedt

full_name: Urban Ungerstedt

given: Urban

family: Schinzel

full_name: Reinhard Schinzel

given: Reinhard

family: Tegtmeier

full_name: Frank Tegtmeier

given: Frank

journal_name: Journal of Neurotrauma

publisher: Mary Ann Liebert Inc

publisher_dissemination: Mary Ann Liebert Inc

publisher_group: Mary Ann Liebert Inc

publisher_normalized: Mary Ann Liebert Inc

title: Nitric Oxide Synthase Inhibition with the Antipterin VAS203 Improves Outcome in Moderate and Severe Traumatic Brain Injury: A Placebo-Controlled Randomized Phase IIa Trial (NOSTRA)

lead_sponsor: Vasopharm GmbH

location_country:
Austria
Switzerland
France
Spain
United Kingdom

location_facility: Vall d'Hebron University Hospital Department of Neurosurgery

location_facility: Southampton University Hospital Division of Clinical Neurosciences

location_facility: Medical University Innsbruck Department of Neurology

location_facility: Hospital Clinic University of Barcelona Surgical Intensive Care Unit

location_facility: University Hospital Zuerich Surgical Intensive Care


status: Terminated

lead_sponsor: BioMarin Pharmaceutical

location_country:
Italy
Netherlands
France
Belgium
United Kingdom

location_facility: Policlinico Universitario Agostino Gemelli

location_facility: Institute of Genetic Medicine International Centre for Life

title: A Phase I/II, Open-label, Dose Escalating With 48 Week Treatment Study to Assess the Safety and Tolerability, Pharmacokinetics, Pharmacodynamics and Efficacy of BMN053 (Previously Known as PRO053) in Subjects With Duchenne Muscular Dystrophy.

summary: The purpose of the study is to see whether BMN053 is safe and effective to use as medication for Duchenne muscular dystrophy (DMD) patients with a mutation around location 53 in the DNA for the dystrophin protein.


title: A Phase IIb, Open-label Study to Assess the Efficacy, Safety, Pharmacodynamics and Pharmacokinetics of Multiple Subcutaneous Doses of PRO045 in Subjects With Duchenne Muscular Dystrophy

summary: The purpose of the study is to see whether PRO045 is safe and effective to use as medication for Duchenne Muscular Dystrophy (DMD) patients with a mutation around location 45 in the DNA for the dystrophin protein.


title: Clinical Evaluation of Therapy™ Cool Flex Ablation Catheter for the Treatment of Paroxysmal Atrial Fibrillation

acronym: FACT-AF

summary:
The purpose of this study is to observe the performance of Therapy™ Cool Flex™ catheter by documenting the procedural parameters, major complications and recurrence of AF after ablation using Cool Flex ablation catheter for the treatment of paroxysmal atrial fibrillation.

This study is an observational clinical study which is not based on any specific endpoints.

primary_purpose: Other

lead_sponsor: Abbott Medical Devices

location_country:
Italy
France
Australia
Portugal
Germany
United Kingdom

location_facility:
Hospital Santa Cruz
Universitat Leipzig Herzzentrum
Ospendale dell'Angelo
Heart Hospital
Royal Adelaide Hospital
Hopital du Haut Leveque


title: A Multi-Center, Single Arm, Prospective Study of the WallFlex™ Biliary Partially-covered Stent for the Palliative Treatment of Malignant Bile Duct Obstruction

summary: This is a a prospective study of the WallFlex™ Biliary Partially-covered Stent designed to collect data to support regulatory clearance by the FDA in the United States and to determine the functionality of the WallFlex™ Biliary Partially Covered Stent as a Palliative treatment for malignant bile duct obstruction.

lead_sponsor: Boston Scientific Corporation

location_country: Italy India Netherlands France Belgium Germany

location_facility: EVK Krankenhaus der Universitat Dusseldorf

location_facility: Academisch Medisch Centrum Universiteit van Amsterdam


summary: Study compares the efficacy and safety of FLUTIFORM® with Seretide® in the treatment of mild to moderate persistent asthma in pediatric subjects.

lead_sponsor: Mundipharma Research Limited

location_country: France Czechia Poland Romania Hungary Germany


title: An Open-Label Extension Study to Evaluate the Long-Term Safety and Efficacy of Reslizumab (3.0 mg/kg) as Treatment for Patients With Eosinophilic Asthma Who Completed a Prior Teva-Sponsored Study in Eosinophilic Asthma

summary: The primary objective of the study is to evaluate the long-term safety of reslizumab at a dosage of 3.0 mg/kg every 4 weeks for approximately 24 months in pediatric and adult patients with eosinophilic asthma as assessed by adverse events, physical examination findings, vital sign measurements, and concomitant medication usage throughout the study (every 4 weeks), clinical laboratory test results, and measurement of antidrug antibodies.

lead_sponsor: Teva Branded Pharmaceutical Products R&D, Inc.

location_country:
Peru
Malaysia
Greece
Romania
Belgium
Australia
Russian Federation
Denmark
Czech Republic
Taiwan
Ukraine
France
Poland
Thailand
Slovakia
Argentina
Canada
Korea, Republic of
Colombia
South Africa
Chile
Sweden
Netherlands
Philippines
Hungary
Germany
United States
Brazil
Mexico
Israel
New Zealand

location_facility:
Teva Investigational Site 120
Teva Investigational Site 226
Teva Investigational Site 555
Teva Investigational Site 361
Teva Investigational Site 560
Teva Investigational Site 404
Teva Investigational Site 422
Teva Investigational Site 284
Teva Investigational Site 740
Teva Investigational Site 588
Teva Investigational Site 31
Teva Investigational Site 128
Teva Investigational Site 741
Teva Investigational Site 620
Teva Investigational Site 103
Teva Investigational Site 160
Teva Investigational Site 205
Teva Investigational Site 17
Teva Investigational Site 624
Teva Investigational Site 700
Teva Investigational Site 743
Teva Investigational Site 64
Teva Investigational Site 643
Teva Investigational Site 631
Teva Investigational Site 625
Teva Investigational Site 633
Teva Investigational Site 41
Teva Investigational Site 203
Teva Investigational Site 220
Teva Investigational Site 622
Teva Investigational Site 1
Teva Investigational Site 21
Teva Investigational Site 705
Teva Investigational Site 702
Teva Investigational Site 552
Teva Investigational Site 557
Teva Investigational Site 262
Teva Investigational Site 521
Teva Investigational Site 523
Teva Investigational Site 540
Teva Investigational Site 142
Teva Investigational Site 505
Teva Investigational Site 433
Teva Investigational Site 554
Teva Investigational Site 74
Teva Investigational Site 723
Teva Investigational Site 341
Teva Investigational Site 645
Teva Investigational Site 559
Teva Investigational Site 227
Teva Investigational Site 742
Teva Investigational Site 509
Teva Investigational Site 144
Teva Investigational Site 20
Teva Investigational Site 264
Teva Investigational Site 542
Teva Investigational Site 55
Teva Investigational Site 582
Teva Investigational Site 745
Teva Investigational Site 520
Teva Investigational Site 604
Teva Investigational Site 682
Teva Investigational Site 223
Teva Investigational Site 632
Teva Investigational Site 33
Teva Investigational Site 150
Teva Investigational Site 59
Teva Investigational Site 727
Teva Investigational Site 522
Teva Investigational Site 421
Teva Investigational Site 642
Teva Investigational Site 280
Teva Investigational Site 372
Teva Investigational Site 370
Teva Investigational Site 402
Teva Investigational Site 400
Teva Investigational Site 34
Teva Investigational Site 127
Teva Investigational Site 209
Teva Investigational Site 143
Teva Investigational Site 543
Teva Investigational Site 63
Teva Investigational Site 601
Teva Investigational Site 46
Teva Investigational Site 221
Teva Investigational Site 207
Teva Investigational Site 73
Teva Investigational Site 286
Teva Investigational Site 260
Teva Investigational Site 53
Teva Investigational Site 182
Teva Investigational Site 426
Teva Investigational Site 587
Teva Investigational Site 181
Teva Investigational Site 125
Teva Investigational Site 104
Teva Investigational Site 704
Teva Investigational Site 602
Teva Investigational Site 18
Teva Investigational Site 701
Teva Investigational Site 425
Teva Investigational Site 45
Teva Investigational Site 428
Teva Investigational Site 15
Teva Investigational Site 9
Teva Investigational Site 629
Teva Investigational Site 583
Teva Investigational Site 12
Teva Investigational Site 720
Teva Investigational Site 681
Teva Investigational Site 512
Teva Investigational Site 2
Teva Investigational Site 49
Teva Investigational Site 32
Teva Investigational Site 367
Teva Investigational Site 126
Teva Investigational Site 185
Teva Investigational Site 52
Teva Investigational Site 589
Teva Investigational Site 782
Teva Investigational Site 541
Teva Investigational Site 121
Teva Investigational Site 369
Teva Investigational Site 683
Teva Investigational Site 641
Teva Investigational Site 147
Teva Investigational Site 123
Teva Investigational Site 724
Teva Investigational Site 14
Teva Investigational Site 513
Teva Investigational Site 621
Teva Investigational Site 180
Teva Investigational Site 25
Teva Investigational Site 66
Teva Investigational Site 300
Teva Investigational Site 222
Teva Investigational Site 504
Teva Investigational Site 27
Teva Investigational Site 584
Teva Investigational Site 685
Teva Investigational Site 225
Teva Investigational Site 204
Teva Investigational Site 58
Teva Investigational Site 586
Teva Investigational Site 763
Teva Investigational Site 261
Teva Investigational Site 43
Teva Investigational Site 24
Teva Investigational Site 585
Teva Investigational Site 47
Teva Investigational Site 6
Teva Investigational Site 420
Teva Investigational Site 524
Teva Investigational Site 342
Teva Investigational Site 545
Teva Investigational Site 285
Teva Investigational Site 140
Teva Investigational Site 432
Teva Investigational Site 166
Teva Investigational Site 40
Teva Investigational Site 511
Teva Investigational Site 11
Teva Investigational Site 562
Teva Investigational Site 229
Teva Investigational Site 287
Teva Investigational Site 164
Teva Investigational Site 507
Teva Investigational Site 561
Teva Investigational Site 283
Teva Investigational Site 19
Teva Investigational Site 780
Teva Investigational Site 600
Teva Investigational Site 28
Teva Investigational Site 680
Teva Investigational Site 8
Teva Investigational Site 549
Teva Investigational Site 401
Teva Investigational Site 30
Teva Investigational Site 122
Teva Investigational Site 630
Teva Investigational Site 405
Teva Investigational Site 105
Teva Investigational Site 403
Teva Investigational Site 371
Teva Investigational Site 5
Teva Investigational Site 200
Teva Investigational Site 563
Teva Investigational Site 50
Teva Investigational Site 544
Teva Investigational Site 26
Teva Investigational Site 366
Teva Investigational Site 161
Teva Investigational Site 281
Teva Investigational Site 623
Teva Investigational Site 202
Teva Investigational Site 380
Teva Investigational Site 407
Teva Investigational Site 721
Teva Investigational Site 603
Teva Investigational Site 263
Teva Investigational Site 423
Teva Investigational Site 3
Teva Investigational Site 460
Teva Investigational Site 4
Teva Investigational Site 146
Teva Investigational Site 363
Teva Investigational Site 686
Teva Investigational Site 784
Teva Investigational Site 16
Teva Investigational Site 626
Teva Investigational Site 558
Teva Investigational Site 580
Teva Investigational Site 162
Teva Investigational Site 101
Teva Investigational Site 761
Teva Investigational Site 500
Teva Investigational Site 44
Teva Investigational Site 69
Teva Investigational Site 546


title: A Prospective, Multicenter Evaluation of the CD HORIZON® Fenestrated Screw Spinal System With Fenestrated Screw Cement When Used in the Treatment of Spinal Conditions in Subjects With Compromised Bone Quality

acronym: FNS

summary: The primary objective of this post-market study is to demonstrate that Oswestry disability index (ODI) score improved significantly at 12 months post-operatively as compared to baseline for each indication (degenerative spinal disease and deformity) in subjects with compromised bone quality, who will receive a surgical procedure requiring posterior stabilization and/or immobilization of one or more spinal segments using CD HORIZON® Fenestrated Screw Spinal System with Fenestrated Screw Cement.

lead_sponsor: Medtronic Spinal and Biologics

location_country: Italy Finland France Greece Belgium

location_facility: IRCCS Istituto Clinico Humanitas di Milano


title: Effect of Intense Sport Practice in Athletes With Non-ischemic Scar

acronym: EISCAR

summary:
The prognostic relevance of isolated non-ischemic LGE (i.e. with no underlying "labelled" cardiomyopathy) is unclear, and current guidelines to not state on the clearance of athlete with this type of findings as regards to competitive or intense sport practice.

The principal objective of the study is to evaluate during a five-years follow up, the clinical outcome of athletes with this kind of findings. The secondary objective is the determination of prognostic factors. The management and follow-up of the athletes will be let at the appraisal of each center.

status: Recruiting

time_perspective: Prospective

lead_sponsor: Rennes University Hospital

location_country:
Luxembourg
France
Belgium
Australia
Germany
United Kingdom

location_facility:
Saarland University
St. George's University of London
Hospital Center of Luxembourg
University Hospital Gasthuisberg
CHU de Rennes
Baker IDI Heart and Diabetes Institute

intervention_type: Other


ReferenceCitation: Schweighardt B, Tompkins T, Lau K, Jesaitis L, Qi Y, Musson DG, Farmer P, Haller C, Shaywitz AJ, Yang K, O'Neill CA. Immunogenicity of Elosulfase Alfa, an Enzyme Replacement Therapy in Patients With Morquio A Syndrome: Results From MOR-004, a Phase III Trial. Clin Ther. 2015 May 1;37(5):1012-1021.e6. doi: 10.1016/j.clinthera.2014.11.005. Epub 2014 Dec 6.

family: Schweighardt

full_name: Becky Schweighardt

given: Becky

family: Tompkins

full_name: Troy Tompkins

given: Troy

family: Lau

full_name: Kelly Lau

given: Kelly

family: Jesaitis

full_name: Lynne Jesaitis

given: Lynne

family: Qi

full_name: Yulan Qi

given: Yulan

family: Musson

full_name: Donald G. Musson

given: Donald G.

family: Farmer

full_name: Pamela Farmer

given: Pamela

family: Haller

full_name: Christine Haller

given: Christine

family: Shaywitz

full_name: Adam J. Shaywitz

given: Adam J.

family: Yang

full_name: Ke Yang

given: Ke

family: O’Neill

full_name: Charles A. O’Neill

given: Charles A.

journal_name: Clinical Therapeutics

location_country:
Italy
Brazil
United States
Qatar
Saudi Arabia
Netherlands
Germany
France
Japan
Argentina
Portugal
Denmark
Canada
United Kingdom
Korea, Republic of
Taiwan
Colombia

title: A Phase 3, Randomized, Double-Blind, Placebo-Controlled, Multinational Clinical Study to Evaluate the Efficacy and Safety of 2.0 mg/kg/Week and 2.0 mg/kg/Every Other Week BMN 110 in Patients With Mucopolysaccharidosis IVA (Morquio A Syndrome)

summary:
This Phase 3 study will evaluate the efficacy and safety of 2.0 mg/kg/week BMN 110 and 2.0 mg/kg/every other week BMN 110 in patients with mucopolysaccharidosis IVA (Morquio A Syndrome).

There is currently no standard accepted treatment for MPS IVA other than supportive care. Enzyme replacement therapy (ERT) may be a potential new treatment option for MPS IVA patients. BMN 110 is administered to MPS IVA patients by IV infusion, allowing cellular uptake by the mannose-6-phosphate receptor and transportation to the lysosomes.

This enzyme uptake into the lysosomes is hypothesized to promote increased catabolism of keratan sulfate (KS) in tissue macrophages, hyaline cartilage, other connective tissues, and heart valve, and reduce the progressive accumulation of KS which is responsible for the clinical manifestations of the disorders.


title: A Multicenter Longitudinal Study for Disease Profiling of Asthma and Chronic Obstructive Pulmonary Disease

summary: The purpose of this study is to characterize the clinical, physiologic, and molecular profiles of healthy participants, participants with mild, moderate, and severe asthma; and participants with moderate to severe Chronic Obstructive Pulmonary Disease (COPD).

ReferenceCitation: Cass SP, Yang Y, Xiao J, McGrath JJC, Fantauzzi MF, Thayaparan D, Wang F, Liang Z, Long F, Stevenson CS, Chen R, Stampfli MR. Current smoking status is associated with reduced sputum immunoglobulin M and G expression in COPD. Eur Respir J. 2021 Feb 4;57(2). pii: 1902338. doi: 10.1183/13993003.02338-2019. Print 2021 Feb.

family: Cass

full_name: Steven P. Cass

given: Steven P.

full_name: Yuqiong Yang

given: Yuqiong

family: Xiao

full_name: Jing Xiao

given: Jing

family: McGrath

full_name: Joshua J.C. McGrath

given: Joshua J.C.

family: Fantauzzi

full_name: Matthew F. Fantauzzi

given: Matthew F.

family: Thayaparan

full_name: Danya Thayaparan

given: Danya

family: Wang

full_name: Fengyan Wang

given: Fengyan

family: Liang

full_name: Zhenyu Liang

given: Zhenyu

family: Long

full_name: Fei Long

given: Fei

family: Stevenson

full_name: Christopher S. Stevenson

given: Christopher S.

family: Chen

full_name: Rongchang Chen

given: Rongchang

family: Stampfli

full_name: Martin R. Stampfli

given: Martin R.

journal_name: European Respiratory Journal

publisher: European Respiratory Society (ERS)

publisher_dissemination: European Respiratory Society

publisher_group: European Respiratory Society

publisher_normalized: European Respiratory Society

ReferenceCitation: Loza MJ, Djukanovic R, Chung KF, Horowitz D, Ma K, Branigan P, Barnathan ES, Susulic VS, Silkoff PE, Sterk PJ, Baribaud F; ADEPT (Airways Disease Endotyping for Personalized Therapeutics) and U-BIOPRED (Unbiased Biomarkers for the Prediction of Respiratory Disease Outcome Consortium) investigators. Validated and longitudinally stable asthma phenotypes based on cluster analysis of the ADEPT study. Respir Res. 2016 Dec 15;17(1):165.

lead_sponsor: Janssen Research & Development, LLC

location_country:
Netherlands
Germany
France
Romania
Denmark
United States
United Kingdom
Canada


title: Open-label, Multicentre, Multiple-dose Trial to Evaluate Pharmacokinetics, Efficacy and Safety of Once Daily Dosing Compared to Twice Daily Dosing of Orfadin in Patients Diagnosed With Hereditary Tyrosinemia Type 1

summary: The purpose of this study is to look at the steady-state serum concentrations of nitisinone when switching from twice daily and once daily dosing.

lead_sponsor: Swedish Orphan Biovitrum

location_country: Sweden France Belgium Denmark Germany

location_facility: Swedish Orphan Biovitrum Investigational Site

location_facility: Swedish Orphan Biovitrum Investigational site


ReferenceCitation: Diaz GA, Jones SA, Scarpa M, Mengel KE, Giugliani R, Guffon N, Batsu I, Fraser PA, Li J, Zhang Q, Ortemann-Renon C. One-year results of a clinical trial of olipudase alfa enzyme replacement therapy in pediatric patients with acid sphingomyelinase deficiency. Genet Med. 2021 Aug;23(8):1543-1550. doi: 10.1038/s41436-021-01156-3. Epub 2021 Apr 19.

family: Diaz

full_name: George A. Diaz

given: George A.

family: Jones

full_name: Simon A. Jones

given: Simon A.

family: Scarpa

full_name: Maurizio Scarpa

given: Maurizio

family: Mengel

full_name: Karl Eugen Mengel

given: Karl Eugen

family: Giugliani

full_name: Roberto Giugliani

given: Roberto

family: Guffon

full_name: Nathalie Guffon

given: Nathalie

family: Batsu

full_name: Isabela Batsu

given: Isabela

family: Fraser

full_name: Patricia A. Fraser

given: Patricia A.

family: Li

full_name: Jing Li

family: Zhang

full_name: Qi Zhang

given: Qi

family: Ortemann-Renon

full_name: Catherine Ortemann-Renon

given: Catherine

journal_name: Genetics in Medicine

evidence: open (via crossref license)

license: cc-by

license_normalized: cc-by

publisher_dissemination: Springer-Nature

publisher_group: Springer-Nature

ipd_sharing_description: Qualified researchers may request access to participant level data and related study documents including the clinical study report, study protocol with any amendments, blank case report form, statistical analysis plan, and dataset specifications. Participant level data will be anonymized and study documents will be redacted to protect the privacy of trial participants. Further details on Sanofi's data sharing criteria, eligible studies, and process for requesting access can be found at: https://www.clinicalstudydatarequest.com /

lead_sponsor: Genzyme, a Sanofi Company

location_country:
Italy
Brazil
Germany
France
United States
United Kingdom

acronym: ASCEND-Peds

location_facility:
Investigational Site Number 276001
Investigational Site Number 840001
Investigational Site Number 380001
Investigational Site Number 250002
Investigational Site Number 076001
Investigational Site Number 826001

title: A Phase 1/2, Multi-Center, Open-Label, Ascending Dose Study to Evaluate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Exploratory Efficacy of Olipudase Alfa in Pediatric Patients Aged <18 Years With Acid Sphingomyelinase Deficiency

summary:
Primary Objective:

To evaluate the safety and tolerability of olipudase alfa administered intravenously in pediatric participants every 2 weeks for 64 weeks.

Secondary Objective:

To characterize the pharmacokinetic profile and evaluate the pharmacodynamics and exploratory efficacy of olipudase alfa administered intravenously in pediatric participants every 2 weeks for 64 weeks.


title: A Multicenter, Randomized, Double-blind, Placebo-controlled, 4-arm, 26 Week Parallel-Group Study to Evaluate Efficacy, Safety and Tolerability of 2 Oral Doses and 2 Regimes of Tideglusib vs Placebo in Mild-to-Moderate AD Patients

acronym: ARGO

summary:
The main purpose of this study is to evaluate the cognitive changes after administration of tideglusib versus placebo at two oral doses and two treatment regimes for 26 weeks in patients with mild to moderate Alzheimer's disease.

After the 26 week core treatment period, the patients may continue in the study under blinded conditions for an optional extension period up to a maximum of 39 additional weeks (total study duration up to 65 weeks), until the last patient in the study has completed the 26 week of treatment.

lead_sponsor: Noscira SA

location_country:
Finland
France
Belgium
Spain
Germany
United Kingdom


title: An Open-label, Multicenter, Randomized Phase 2 Study Evaluating the Safety and Efficacy of Cisplatin and Pemetrexed With or Without Cixutumumab as First-Line Therapy in Patients With Advanced Nonsquamous Non-Small Cell Lung Carcinoma

summary: The primary purpose of this study is to evaluate the hypothesis that cixutumumab given in combination with cisplatin and pemetrexed is superior to cisplatin and pemetrexed as first-line therapy for patients with advanced nonsquamous non-small cell lung carcinoma (NSCLC).

ipd_sharing_description: Anonymized individual patient level data will be provided in a secure access environment upon approval of a research proposal and a signed data sharing agreement.

lead_sponsor: Eli Lilly and Company

location_country:
Italy
United States
Brazil
Netherlands
Germany
France
Israel
Belgium
Argentina
Spain
Turkey
Canada

location_facility: For additional information regarding investigative sites for this trial, contact 1-877-CTLILLY (1-877-285-4559, 1-317-615-4559) Mon - Fri from 9 AM to 5 PM Eastern Time (UTC/GMT - 5 hours, EST), or speak with your personal physician.


title: A Phase Ib Study Investigating the Combination of Everolimus With Trastuzumab and Vinorelbine in Patients With HER2-overexpressing Metastatic Breast Cancer

summary: This study will look at different dose levels and regimens of everolimus combined with weekly trastuzumab and vinorelbine therapy in patients with HER-2 overexpressing metastatic breast cancer.

ReferenceCitation: Jerusalem G, Fasolo A, Dieras V, Cardoso F, Bergh J, Vittori L, Zhang Y, Massacesi C, Sahmoud T, Gianni L. Phase I trial of oral mTOR inhibitor everolimus in combination with trastuzumab and vinorelbine in pre-treated patients with HER2-overexpressing metastatic breast cancer. Breast Cancer Res Treat. 2011 Jan;125(2):447-55. doi: 10.1007/s10549-010-1260-x. Epub 2010 Nov 25.

family: Jerusalem

full_name: Guy Jerusalem

given: Guy

family: Fasolo

full_name: Angelica Fasolo

given: Angelica

family: Dieras

full_name: Veronique Dieras

given: Veronique

family: Cardoso

full_name: Fatima Cardoso

given: Fatima

family: Bergh

full_name: Jonas Bergh

given: Jonas

family: Vittori

full_name: Luc Vittori

given: Luc

full_name: Yufen Zhang

given: Yufen

family: Massacesi

full_name: Cristian Massacesi

given: Cristian

family: Sahmoud

full_name: Tarek Sahmoud

given: Tarek

family: Gianni

full_name: Luca Gianni

given: Luca

journal_name: Breast Cancer Research and Treatment

oa_host_type: repository

repository_institution: Le Centre pour la Communication Scientifique Directe - HAL - Inria

repository_normalized: HAL

lead_sponsor: Novartis Pharmaceuticals

location_country: Italy Sweden France Poland Belgium


title: An International 7-Week, Double-Blind, Placebo-Controlled, 2-Parallel Group Study to Assess the Efficacy of Dianicline 40 mg BID as an Aid to Smoking Cessation in Cigarette Smokers

acronym: EURODIAN

summary: The primary objective is to demonstrate the efficacy of dianicline as an aid to smoking cessation in cigarette smokers. The main secondary objectives are: to assess the craving for cigarettes, nicotine withdrawal symptoms and the safety of dianicline.

lead_sponsor: Sanofi

location_country: Sweden France Norway Belgium Spain Denmark


title: Prospective, Controlled, Randomized Study to Evaluate Pain and Further Disabling Complications in Patients Undergoing Lichtenstein Technique for Primary Inguinal Hernia Repair by Fixing the Mesh With Fibrin Sealant Versus Sutures

summary: The purpose of the study is to evaluate mid and long term postoperative pain and further disabling complications in open inguinal hernia repair by Lichtenstein technique after mesh fixation with fibrin sealant (FS), compared to mesh fixation with sutures.

ReferenceCitation: Campanelli G, Pascual MH, Hoeferlin A, Rosenberg J, Champault G, Kingsnorth A, Miserez M. Randomized, controlled, blinded trial of Tisseel/Tissucol for mesh fixation in patients undergoing Lichtenstein technique for primary inguinal hernia repair: results of the TIMELI trial. Ann Surg. 2012 Apr;255(4):650-7. doi: 10.1097/SLA.0b013e31824b32bf.

family: Campanelli

full_name: Giampiero Campanelli

given: Giampiero

family: Pascual

full_name: Manuel Hidalgo Pascual

given: Manuel Hidalgo

family: Hoeferlin

full_name: Andreas Hoeferlin

given: Andreas

family: Rosenberg

full_name: Jacob Rosenberg

given: Jacob

family: Champault

full_name: Gérard Champault

given: Gérard

family: Kingsnorth

full_name: Andrew Kingsnorth

given: Andrew

family: Miserez

full_name: Marc Miserez

given: Marc

journal_name: Annals of Surgery

publisher: Ovid Technologies (Wolters Kluwer Health)

publisher_dissemination: Wolters Kluwer Health

publisher_group: Wolters Kluwer Health

publisher_normalized: Wolters Kluwer Health

title: Randomized, Controlled, Blinded Trial of Tisseel/Tissucol for Mesh Fixation in Patients Undergoing Lichtenstein Technique for Primary Inguinal Hernia Repair

lead_sponsor: University of Milan

location_country:
France
Belgium
Spain
Denmark
Germany
United Kingdom

location_facility: Copenhagen university hospital, Gentofte

intervention_type: Procedure


title: A Prospective Multicentre Phase II Trial of Zoledronic Acid in Patients With Myelofibrosis With Myeloid Metaplasia (MMM)

summary: In this trial, the question is addressed if zoledronic acid (Zometa, Novartis Pharma)could be of clinical benefit for patients with myelofibrosis and myeloid metaplasia (MMM).

lead_sponsor: Universitaire Ziekenhuizen Leuven

location_country: France Israel Belgium Australia Spain Germany

location_facility: Hopital Avicenne and Paris 13 University


title: Randomized, Multicenter, Double-blind, Placebo-controlled, Two-arm Parallel Group Trial of Rimonabant 20-mg od, for Inhibition of Atherosclerosis Progression Assessed by Carotid Artery Intima-media Thickness (CIMT), in Overweight Patients With Additional Risk Factors

acronym: AUDITOR

summary:
Objectives:

Primary: To evaluate the effect of rimonabant 20-mg once daily in comparison with placebo, on the quantitative progression of atherosclerosis as assessed by carotid artery intima-media thickness (CIMT)
Secondary: To evaluate the safety and tolerability of the above rimonabant regimen in the study population of atherosclerosis patients.

ReferenceCitation: O'Leary DH, Reuwer AQ, Nissen SE, Després JP, Deanfield JE, Brown MW, Zhou R, Zabbatino SM, Job B, Kastelein JJ, Visseren FL; AUDITOR investigators. Effect of rimonabant on carotid intima-media thickness (CIMT) progression in patients with abdominal obesity and metabolic syndrome: the AUDITOR Trial. Heart. 2011 Jul;97(14):1143-50. doi: 10.1136/hrt.2011.223446. Epub 2011 May 24.

family: O'Leary

full_name: D. H. O'Leary

given: D. H.

family: Reuwer

full_name: A. Q. Reuwer

given: A. Q.

family: Nissen

full_name: S. E. Nissen

given: S. E.

family: Despres

full_name: J.-P. Despres

family: Deanfield

full_name: J. E. Deanfield

given: J. E.

family: Brown

full_name: M. W. Brown

given: M. W.

family: Zhou

full_name: R. Zhou

given: R.

family: Zabbatino

full_name: S. M. Zabbatino

given: S. M.

family: Job

full_name: B. Job

given: B.

family: Kastelein

full_name: J. J. P. Kastelein

given: J. J. P.

family: Visseren

full_name: F. L. J. Visseren

given: F. L. J.

journal_name: Heart

repository_institution: The University of Manchester - Research Explorer

repository_institution: DANS - Data Archiving and Networked Services - NARCIS - National Academic Research and Collaborations Information System

publisher: BMJ

publisher_dissemination: British Medical Journal

publisher_group: British Medical Journal

publisher_normalized: British Medical Journal

location_country:
Netherlands
France
Spain
United States
United Kingdom
Canada


title: Study of GSK2302024A Antigen-Specific Cancer Immunotherapeutic Combined With Standard Neoadjuvant Treatment in Patients With WT1-positive Primary Invasive Breast Cancer

acronym: INDUCT

summary: The purpose of this study is to evaluate the safety, immunogenicity and clinical activity of a new WT1 anti-cancer immunotherapy in patients with WT1-positive Stage II or III breast cancer. The treatment will be given before surgery in combination with standard therapy.

ReferenceCitation: Higgins M, Curigliano G, Dieras V, Kuemmel S, Kunz G, Fasching PA, Campone M, Bachelot T, Krivorotko P, Chan S, Ferro A, Schwartzberg L, Gillet M, De Sousa Alves PM, Wascotte V, Lehmann FF, Goss P. Safety and immunogenicity of neoadjuvant treatment using WT1-immunotherapeutic in combination with standard therapy in patients with WT1-positive Stage II/III breast cancer: a randomized Phase I study. Breast Cancer Res Treat. 2017 Apr;162(3):479-488. doi: 10.1007/s10549-017-4130-y. Epub 2017 Feb 7.

family: Higgins

full_name: M. Higgins

given: M.

family: Curigliano

full_name: G. Curigliano

given: G.

family: Dieras

full_name: V. Dieras

given: V.

family: Kuemmel

full_name: S. Kuemmel

given: S.

family: Kunz

full_name: G. Kunz

family: Fasching

full_name: P. A. Fasching

given: P. A.

family: Campone

full_name: M. Campone

family: Bachelot

full_name: T. Bachelot

given: T.

family: Krivorotko

full_name: P. Krivorotko

given: P.

family: Chan

full_name: S. Chan

family: Ferro

full_name: A. Ferro

given: A.

family: Schwartzberg

full_name: L. Schwartzberg

given: L.

family: Gillet

full_name: M. Gillet

family: De Sousa Alves

full_name: P. M. De Sousa Alves

given: P. M.

family: Wascotte

full_name: V. Wascotte

family: Lehmann

full_name: F. F. Lehmann

given: F. F.

family: Goss

full_name: P. Goss

ipd_sharing_description: IPD for this study is available via the Clinical Study Data Request site (click on the link provided below).

location_country:
Italy
United States
France
Belgium
Russian Federation
Germany
United Kingdom


title: A Randomized, Open-Label Study to Compare the Efficacy and Safety of AT1001 and Enzyme Replacement Therapy (ERT) in Patients With Fabry Disease and AT1001-Responsive GLA Mutations, Who Were Previously Treated With ERT

summary: Study to compare the efficacy and safety of migalastat and enzyme replacement therapy (ERT) in male and female participants with Fabry disease who are currently receiving ERT and who have an alpha galactosidase-A (α Gal-A) mutation that is amenable to migalastat, based on the clinical trial human embryonic kidney cell (HEK) assay.

ReferenceCitation: Feldt-Rasmussen U, Hughes D, Sunder-Plassmann G, Shankar S, Nedd K, Olivotto I, Ortiz D, Ohashi T, Hamazaki T, Skuban N, Yu J, Barth JA, Nicholls K. Long-term efficacy and safety of migalastat treatment in Fabry disease: 30-month results from the open-label extension of the randomized, phase 3 ATTRACT study. Mol Genet Metab. 2020 Sep - Oct;131(1-2):219-228. doi: 10.1016/j.ymgme.2020.07.007. Epub 2020 Aug 15.

family: Feldt-Rasmussen

full_name: Ulla Feldt-Rasmussen

given: Ulla

family: Hughes

full_name: Derralynn Hughes

given: Derralynn

family: Sunder-Plassmann

full_name: Gere Sunder-Plassmann

given: Gere

family: Shankar

full_name: Suma Shankar

given: Suma

family: Nedd

full_name: Khan Nedd

given: Khan

family: Olivotto

full_name: Iacopo Olivotto

given: Iacopo

family: Ortiz

full_name: Damara Ortiz

given: Damara

family: Ohashi

full_name: Toya Ohashi

given: Toya

family: Hamazaki

full_name: Takashi Hamazaki

given: Takashi

family: Skuban

full_name: Nina Skuban

given: Nina

family: Yu

full_name: Julie Yu

given: Julie

family: Barth

full_name: Jay A. Barth

given: Jay A.

family: Nicholls

full_name: Kathleen Nicholls

given: Kathleen

journal_name: Molecular Genetics and Metabolism

ReferenceCitation: Bichet DG, Aerts JM, Auray-Blais C, Maruyama H, Mehta AB, Skuban N, Krusinska E, Schiffmann R. Assessment of plasma lyso-Gb(3) for clinical monitoring of treatment response in migalastat-treated patients with Fabry disease. Genet Med. 2021 Jan;23(1):192-201. doi: 10.1038/s41436-020-00968-z. Epub 2020 Sep 30. Erratum in: Genet Med. 2020 Nov 20;:.

family: Bichet

full_name: Daniel G. Bichet

given: Daniel G.

family: Aerts

full_name: Johannes M. Aerts

given: Johannes M.

family: Auray-Blais

full_name: Christiane Auray-Blais

given: Christiane

repositories_institution: DANS - Data Archiving and Networked Services - NARCIS - National Academic Research and Collaborations Information System

title: Assessment of plasma lyso-Gb3 for clinical monitoring of treatment response in migalastat-treated patients with Fabry disease

ReferenceCitation: Haninger-Vacariu N, El-Hadi S, Pauler U, Foretnik M, Kain R, Prohászka Z, Schmidt A, Skuban N, Barth JA, Sunder-Plassmann G. Pregnancy Outcome after Exposure to Migalastat for Fabry Disease: A Clinical Report. Case Rep Obstet Gynecol. 2019 Dec 21;2019:1030259. doi: 10.1155/2019/1030259. eCollection 2019.

name: Division of Nephrology and Dialysis, Department of Medicine III, Medical University of Vienna, Vienna, Austria

name: Department of Medicine I, University Hospital St. Pölten, Lower Austria, Austria

name: Department of Pathology, Medical University of Vienna, Vienna, Austria

name: Semmelweis University, Research Laboratory, Third Department of Internal Medicine, Budapest, Hungary

name: Amicus Therapeutics, Inc., Cranbury, NJ, USA

journal_name: Case Reports in Obstetrics and Gynecology

repositories_institution:
Hindawi - Hindawi
PubMed Central - Europe PMC

ReferenceCitation: Narita I, Ohashi T, Sakai N, Hamazaki T, Skuban N, Castelli JP, Lagast H, Barth JA. Efficacy and safety of migalastat in a Japanese population: a subgroup analysis of the ATTRACT study. Clin Exp Nephrol. 2020 Feb;24(2):157-166. doi: 10.1007/s10157-019-01810-w. Epub 2019 Dec 30.

ReferenceCitation: Hughes DA, Nicholls K, Shankar SP, Sunder-Plassmann G, Koeller D, Nedd K, Vockley G, Hamazaki T, Lachmann R, Ohashi T, Olivotto I, Sakai N, Deegan P, Dimmock D, Eyskens F, Germain DP, Goker-Alpan O, Hachulla E, Jovanovic A, Lourenco CM, Narita I, Thomas M, Wilcox WR, Bichet DG, Schiffmann R, Ludington E, Viereck C, Kirk J, Yu J, Johnson F, Boudes P, Benjamin ER, Lockhart DJ, Barlow C, Skuban N, Castelli JP, Barth J, Feldt-Rasmussen U. Oral pharmacological chaperone migalastat compared with enzyme replacement therapy in Fabry disease: 18-month results from the randomised phase III ATTRACT study. J Med Genet. 2017 Apr;54(4):288-296. doi: 10.1136/jmedgenet-2016-104178. Epub 2016 Nov 10. Erratum in: J Med Genet. 2018 Apr 16;:.

repository_normalized: University of Melbourne - Minerva Access

repository_institution: University of Oxford - Oxford Academic Notes & Queries

repository_institution: University of Copenhagen - Research at the University of Copenhagen

repository_institution: University of Florence - Florence Research

repository_institution: University of California - eScholarship University of California

repository_normalized: California Digital Library - eScholarship

ReferenceCitation: Benjamin ER, Della Valle MC, Wu X, Katz E, Pruthi F, Bond S, Bronfin B, Williams H, Yu J, Bichet DG, Germain DP, Giugliani R, Hughes D, Schiffmann R, Wilcox WR, Desnick RJ, Kirk J, Barth J, Barlow C, Valenzano KJ, Castelli J, Lockhart DJ. The validation of pharmacogenetics for the identification of Fabry patients to be treated with migalastat. Genet Med. 2017 Apr;19(4):430-438. doi: 10.1038/gim.2016.122. Epub 2016 Sep 22.

location_country:
Italy
Austria
Brazil
France
Japan
Australia
Belgium
Denmark
United States
United Kingdom


title: Assessment of the Efficacy of a Sensorimotor Approach After Maximum Three Sessions in Subjects With Symptomatic Temporomandibular Dysfunction (TMD) With Hypertonia: A Prospective, Multicenter Case Study.

summary:
The study aims to demonstrate the efficacy of the sensorimotor approach developed by A. Piron (taught to the patient in three sessions) regarding three parameters related to TMD: facial pain, functional incapacities and hypertonia.

The subjects in the study are 18 to 77 years old and present the following conditions: a TMD disorder caused or aggravated by hypertonia and responsible for cervico-maxillo-facial pain and/or a functional incapacity in the last 30 days.

lead_sponsor: Osteovox

location_country: Italy Belgium France

location_facility:
ANTONIO BIANCO Ortodontista e Osteopata
Cabinet d'Ostéopathie PIRON
Cabinet de groupe LHR
Cabinet de Logopédie Liesens-Denis
Osteopata MARCO SBARBARO
Cabinet d'Ostéopathie Garcion

intervention_type: Behavioral


title: Pilot Study 3 of Outpatient Control-to-Range: Safety and Efficacy With Day-and-Night In-Home Use

summary:
The purpose of this study is to see if an automated insulin management system ("study system") can safely be used at home to manage blood sugar. The study system includes (1) a CGM that measures glucose levels, (2) a computer program on a smartphone that determines how much insulin is needed and allows the study participant user to control the whole system, and (3) an insulin pump that delivers the insulin. The CGM will be from Dexcom. The pump will be from Roche. The CGM and pump are similar to the devices that are currently available for people to purchase and use. However, the smartphone device, the CGM sensor type used with it, and the overall study system can only be used for research at this time.

The study will be completed by about 24 individuals at 6 centers in the United States and Europe. This study has several phases and will take about 11-14 weeks to complete depending on whether the study participant is a CGM user or not.

At selected sites (based on subject eligibility and availability), approximately 10-20 subjects who exhibit safe and competent use of the system at home will be given the option to continue home use of the system in Day-and-Night Closed-Loop mode for up to 5 months.

ReferenceCitation: Anderson SM, Raghinaru D, Pinsker JE, Boscari F, Renard E, Buckingham BA, Nimri R, Doyle FJ 3rd, Brown SA, Keith-Hynes P, Breton MD, Chernavvsky D, Bevier WC, Bradley PK, Bruttomesso D, Del Favero S, Calore R, Cobelli C, Avogaro A, Farret A, Place J, Ly TT, Shanmugham S, Phillip M, Dassau E, Dasanayake IS, Kollman C, Lum JW, Beck RW, Kovatchev B; Control to Range Study Group. Multinational Home Use of Closed-Loop Control Is Safe and Effective. Diabetes Care. 2016 Jul;39(7):1143-50. doi: 10.2337/dc15-2468. Epub 2016 Apr 13.

family: Anderson

full_name: Stacey M. Anderson

given: Stacey M.

family: Raghinaru

full_name: Dan Raghinaru

given: Dan

family: Pinsker

full_name: Jordan E. Pinsker

given: Jordan E.

family: Boscari

full_name: Federico Boscari

given: Federico

family: Renard

full_name: Eric Renard

given: Eric

family: Buckingham

full_name: Bruce A. Buckingham

given: Bruce A.

family: Nimri

full_name: Revital Nimri

given: Revital

family: Doyle

full_name: Francis J. Doyle

given: Francis J.

suffix: III

family: Brown

full_name: Sue A. Brown

given: Sue A.

family: Keith-Hynes

full_name: Patrick Keith-Hynes

given: Patrick

family: Breton

full_name: Marc D. Breton

given: Marc D.

family: Chernavvsky

full_name: Daniel Chernavvsky

given: Daniel

family: Bevier

full_name: Wendy C. Bevier

given: Wendy C.

family: Bradley

full_name: Paige K. Bradley

given: Paige K.

family: Bruttomesso

full_name: Daniela Bruttomesso

given: Daniela

family: Del Favero

full_name: Simone Del Favero

given: Simone

family: Calore

full_name: Roberta Calore

given: Roberta

family: Cobelli

full_name: Claudio Cobelli

given: Claudio

family: Avogaro

full_name: Angelo Avogaro

given: Angelo

family: Farret

full_name: Anne Farret

given: Anne

family: Place

full_name: Jerome Place

given: Jerome

family: Ly

full_name: Trang T. Ly

given: Trang T.

family: Shanmugham

full_name: Satya Shanmugham

given: Satya

repository_institution: Harvard University - Digital Access to Scholarship at Harvard (DASH)

repository_institution: University of Padua - Padua Research Archive

location_facility: National Center for Childhood Diabetes- Schneider Children's Medical Center


title: A Phase 2 Multicenter, Randomized, Double Blind, Placebo Controlled Study Of The Safety And Efficacy Of Pf-03049423 In Subjects With Ischemic Stroke

summary: The purpose of this study is to evaluate the safety and tolerability of PF-03049423 following multiple dose administration to subjects with ischemic stroke. The study will also evaluate the efficacy of PF-03049423, relative to placebo, in subjects with ischemic stroke following 90 days of therapy. The study will also explore the relationship between PF-03049423 concentration and blood pressure.

ReferenceCitation: Di Cesare F, Mancuso J, Woodward P, Bednar MM, Loudon PT; A9541004 Stroke Study Group. Phosphodiesterase-5 Inhibitor PF-03049423 Effect on Stroke Recovery: A Double-Blind, Placebo-Controlled Randomized Clinical Trial. J Stroke Cerebrovasc Dis. 2016 Mar;25(3):642-9. doi: 10.1016/j.jstrokecerebrovasdis.2015.11.026. Epub 2015 Dec 28.

family: Di Cesare

full_name: Franco Di Cesare

given: Franco

family: Mancuso

full_name: Jessica Mancuso

given: Jessica

family: Woodward

full_name: Phil Woodward

given: Phil

family: Bednar

full_name: Martin M. Bednar

given: Martin M.

family: Loudon

full_name: Peter T. Loudon

given: Peter T.

journal_name: Journal of Stroke and Cerebrovascular Diseases

lead_sponsor: Pfizer

location_country:
India
Germany
France
Bulgaria
Korea, Republic of
Hungary
United States
Czech Republic
Canada
Taiwan

location_facility: Universitetska mnogoprofilna bolnitsa za aktivno lechenie Aleksandrovska, Klinika po Nevrologia

location_facility: Inha University Hospital, Department of Neurology

location_facility: Samsung Medical Center, Department of Neurology

location_facility: Seoul National University Bundang Hospital, Department of Neurology

location_facility: Klinikum Rechts der Isar, Neurologische Klinik

location_facility: Petz Aladar Megyei Oktato Korhaz, Neurologiai Osztaly

location_facility: Oklahoma University Health Sciences Center

location_facility: Hallym University Sacred Heart Hospital, Department of Neurology

location_facility: UMBAL Tsaritsa Yoanna, Klinika po nevrologia

location_facility: Medical Research & Health Education Foundation, Inc.

location_facility: Dr. Kennessey Albert Korhaz-Rendelointezet, Neurologiai Osztaly

location_facility: Penn State Hershey Rehabilitation Hospital

location_facility: Asan Medical Center, Department of Neurology

location_facility: The Methodist Hospital Neurological Institute

location_facility: Orszagos Idegtudomanyi Intezet, Stroke-ambulancia

location_facility: Chang Gung Medical Foundation-Linkou Branch

location_facility: Massachusetts General Hospital/Department of Neurology

location_facility: MBAL "Tokuda Bolnitsa", Otdelenie po nevrologiya

location_facility: Semmelweis Egyetem AOK / Neurologiai Klinika

location_facility: UNC Department of Neurology Stroke Division

location_facility: Penn State Milton South Hershey Medical Center / Penn State College of Medicine

location_facility: Severance Hospital, Yonsei University College of Medicine, Department of Neurology

location_facility: Investigational Drug Services at OU Medical Center

location_facility: Honvedkorhaz-Allami Egeszsegugyi Kozpont, Ideggyogyaszati Osztaly

location_facility: Jose Canedo, M.D., West Georgia Neurology

location_facility: Chang Gung Medical Foundation-Kaohsiung Chang Gung Memorial Hospital

location_facility: MBALNP "Sveti Naum" EAD, Klinika za Intenzivno Lechenie na Nervni Bolesti

location_facility: Universitaetsklinikum Essen, Neurologische Klinik

location_facility: Chonnam National University Hospital, Department of Neurology

location_facility: Fovarosi Onkormanyzat Peterfy Sandor utcai Korhaz-Rendelointezet es Baleseti Kozpont/Neurologia

location_facility: Vtora Mnogoprofilna Bolnitsa za Aktivno Lechenie, Otdelenie po Nevrologia

location_facility: Voennomeditsinska Akademia - MBAL- Pleven, Otdelenie po Nervni bolesti


title: A Phase 3, Multicenter, Randomized, Double-blind, Placebo-Controlled Study to Evaluate the Efficacy and Safety of Subcutaneous LY2127399 in Patients With Systemic Lupus Erythematosus (SLE)

summary: The purpose of this SLE study is to evaluate the efficacy, safety and tolerability of two different doses of LY2127399 administered in addition to standard of care therapy in participants with active SLE.

ReferenceCitation: Hannon CW, McCourt C, Lima HC, Chen S, Bennett C. Interventions for cutaneous disease in systemic lupus erythematosus. Cochrane Database Syst Rev. 2021 Mar 9;3:CD007478. doi: 10.1002/14651858.CD007478.pub2.

name: Dermatologist, Masters of Public Health Program; Harvard School of Public Health; Boston Massachusetts USA

family: Hannon

full_name: Cora W Hannon

given: Cora W

name: Department of Dermatology; Royal Victoria Hospital; Belfast UK

family: McCourt

full_name: Collette McCourt

given: Collette

name: Department of Dermatology, Clinical Unit for Research Trials and Outcomes in Skin (CURTIS); Massachusetts General Hospital; Boston Massachusetts USA

family: Lima

full_name: Hermenio C Lima

given: Hermenio C

name: Emory University Hospital; Emory Healthcare; Atlanta Georgia USA

family: Chen

full_name: Suephy Chen

given: Suephy

name: Office of Research and Innovation; Royal College of Surgeons in Ireland Coláiste Ríoga na Máinleá in Éirinn; Dublin Ireland

family: Bennett

full_name: Cathy Bennett

given: Cathy

journal_name: Cochrane Database of Systematic Reviews

ReferenceCitation: Li Y, Higgs RE, Hoffman RW, Dow ER, Liu X, Petri M, Wallace DJ, Dörner T, Eastwood BJ, Miller BB, Liu Y. A Bayesian gene network reveals insight into the JAK-STAT pathway in systemic lupus erythematosus. PLoS One. 2019 Dec 2;14(12):e0225651. doi: 10.1371/journal.pone.0225651. eCollection 2019.

family: Li

full_name: Yupeng Li

given: Yupeng

family: Higgs

full_name: Richard E. Higgs

given: Richard E.

family: Hoffman

full_name: Robert W. Hoffman

given: Robert W.

family: Dow

full_name: Ernst R. Dow

given: Ernst R.

family: Liu

full_name: Xiong Liu

given: Xiong

family: Petri

full_name: Michelle Petri

given: Michelle

family: Wallace

full_name: Daniel J. Wallace

given: Daniel J.

family: Dörner

full_name: Thomas Dörner

given: Thomas

family: Eastwood

full_name: Brian J. Eastwood

given: Brian J.

family: Miller

full_name: Bradley B. Miller

given: Bradley B.

full_name: Yushi Liu

given: Yushi

currency_apc_doaj: USD

journal_name: PLOS ONE

oa_colors: green gold

evidence: oa journal (via doaj)

ReferenceCitation: Kalunian KC, Urowitz MB, Isenberg D, Merrill JT, Petri M, Furie RA, Morgan-Cox MA, Taha R, Watts S, Silk M, Linnik MD. Clinical trial parameters that influence outcomes in lupus trials that use the systemic lupus erythematosus responder index. Rheumatology (Oxford). 2018 Jan 1;57(1):125-133. doi: 10.1093/rheumatology/kex368.

ReferenceCitation: Rovin BH, Dooley MA, Radhakrishnan J, Ginzler EM, Forrester TD, Anderson PW. The impact of tabalumab on the kidney in systemic lupus erythematosus: results from two phase 3 randomized, clinical trials. Lupus. 2016 Dec;25(14):1597-1601. Epub 2016 May 24.

ReferenceCitation: Hoffman RW, Merrill JT, Alarcón-Riquelme MM, Petri M, Dow ER, Nantz E, Nisenbaum LK, Schroeder KM, Komocsar WJ, Perumal NB, Linnik MD, Airey DC, Liu Y, Rocha GV, Higgs RE. Gene Expression and Pharmacodynamic Changes in 1,760 Systemic Lupus Erythematosus Patients From Two Phase III Trials of BAFF Blockade With Tabalumab. Arthritis Rheumatol. 2017 Mar;69(3):643-654. doi: 10.1002/art.39950.

name: Eli Lilly and Company; Indianapolis Indiana

name: Oklahoma Medical Research Foundation; Oklahoma City

name: Oklahoma Medical Research Foundation, Oklahoma City, and Pfizer-Universidad de Granada-Junta de Andalucía; Granada Spain

name: Johns Hopkins School of Medicine; Baltimore Maryland

ReferenceCitation: Merrill JT, van Vollenhoven RF, Buyon JP, Furie RA, Stohl W, Morgan-Cox M, Dickson C, Anderson PW, Lee C, Berclaz PY, Dörner T. Efficacy and safety of subcutaneous tabalumab, a monoclonal antibody to B-cell activating factor, in patients with systemic lupus erythematosus: results from ILLUMINATE-2, a 52-week, phase III, multicentre, randomised, double-blind, placebo-controlled study. Ann Rheum Dis. 2016 Feb;75(2):332-40. doi: 10.1136/annrheumdis-2015-207654. Epub 2015 Aug 20.

repositories_institution: University of Oxford - Oxford Academic Notes & Queries

location_country:
Malaysia
Romania
Australia
Russian Federation
Taiwan
Ecuador
India
France
Canada
South Africa
Hungary
United States
United Kingdom
Brazil
Serbia
Mexico
Israel
Tunisia
New Zealand
Spain
Latvia

location_facility: For additional information regarding investigative sites for this trial, contact 1-877-CTLILLY (1-877-285-4559, 1-317-615-4559) Mon - Fri from 9 AM to 5 PM Eastern Time (UTC/GMT - 5 hours, EST), or speak with your personal physician.


ReferenceCitation: Kjelgaard-Petersen CF, Platt A, Braddock M, Jenkins MA, Musa K, Graham E, Gantzel T, Slynn G, Weinblatt ME, Karsdal MA, Thudium CS, Bay-Jensen AC. Translational Biomarkers and Ex Vivo Models of Joint Tissues as a Tool for Drug Development in Rheumatoid Arthritis. Arthritis Rheumatol. 2018 Sep;70(9):1419-1428. doi: 10.1002/art.40527. Epub 2018 Jul 24.

name: Technical University of Denmark; Kongens Lyngby; Denmark

family: Kjelgaard-Petersen

full_name: Cecilie F. Kjelgaard-Petersen

given: Cecilie F.

family: Platt

full_name: Adam Platt

given: Adam

family: Braddock

full_name: Martin Braddock

given: Martin

family: Jenkins

full_name: Martin A. Jenkins

given: Martin A.

family: Musa

full_name: Kishwar Musa

given: Kishwar

family: Graham

full_name: Emma Graham

given: Emma

name: Gentofte University Hospital; Gentofte Denmark

family: Gantzel

full_name: Thorbjørn Gantzel

given: Thorbjørn

family: Slynn

full_name: Gillian Slynn

given: Gillian

name: Brigham and Women's Hospital; Boston Massachusetts

family: Weinblatt

full_name: Michael E. Weinblatt

given: Michael E.

family: Karsdal

full_name: Morten A. Karsdal

given: Morten A.

family: Thudium

full_name: Christian S. Thudium

given: Christian S.

family: Bay-Jensen

full_name: Anne-C. Bay-Jensen

given: Anne-C.

journal_name: Arthritis & Rheumatology

license: cc-by-nc

license_normalized: cc-by-nc

repository_institution: Technical University of Denmark, DTU - Technical University of Denmark, DTU Orbit

repositories:
PubMed Central
Pure (Denmark)

lead_sponsor: AstraZeneca

location_country:
Brazil
Chile
Peru
India
Mexico
France
Bulgaria
Poland
Slovakia
Hungary
Belgium
Argentina
Australia
Estonia
United States
United Kingdom
Ukraine

acronym: OSKIRA - 1

title: (OSKIRA-1): A Phase III, Multi-centre, Randomised, Double-Blind, Placebo-Controlled, Parallel Group Study of Two Dosing Regimens of Fostamatinib Disodium in Rheumatoid Arthritis Patients With an Inadequate Response to Methotrexate

summary: The purpose of the study is to evaluate the effectiveness of two dosing regimens of fostamatinib compared to placebo, in patients with rheumatoid arthritis (RA) who are taking methotrexate but not responding. The study will last for 1 year.


lead_sponsor: Debiopharm International SA

location_country:
Italy
Germany
France
Poland
Romania
Hungary
Belgium
Australia
Puerto Rico
Spain
Turkey
United States
United Kingdom
Taiwan

title: A Multicenter, Randomized, Double-blind, Placebo-controlled, Parallel-group Phase II Study on Efficacy and Safety of DEB025 Combined With Peg-IFN Alfa-2a and Ribavirin in Chronic Hepatitis C Genotype 1 Relapsers and Non-responders to Previous Peg-IFN Alfa-2 Plus Ribavirin Treatment

summary: The study is to investigate whether participants with hepatitis C virus (HCV) genotype 1 who have a history of non-response/relapse to peginterferon alfa-2a (PEG) and ribavirin (RBV) may benefit from treatment with triple therapy alisporivir (ALV; DEB025) with PEG and RBV versus placebo with PEG and RBV.


ReferenceCitation: Miah KM, Hyde SC, Gill DR. Emerging gene therapies for cystic fibrosis. Expert Rev Respir Med. 2019 Aug;13(8):709-725. doi: 10.1080/17476348.2019.1634547. Epub 2019 Jun 27. Review.

name: Gene Medicine Group, Nuffield Division of Clinical Laboratory Science, Radcliffe Department of Medicine, University of Oxford, Oxford, UK

family: Miah

full_name: Kamran M. Miah

given: Kamran M.

family: Hyde

full_name: Stephen C. Hyde

given: Stephen C.

family: Gill

full_name: Deborah R. Gill

given: Deborah R.

journal_name: Expert Review of Respiratory Medicine

repository_institution: University of Oxford - Oxford University Research Archive (ORA)

publisher: Informa UK Limited

publisher_dissemination: Informa

publisher_group: Informa

publisher_normalized: Informa

lead_sponsor: Phoenicis Therapeutics

location_country:
United States
France
Spain

acronym: WINGS

location_facility: Stanford University School of Medicine, LPCH

location_facility: Journey Clinic, Center for Pediatric Blood and Marrow Transplantation

title: A First in Human, Double-blind, Randomized, Intra-subject Placebo-controlled, Multiple Dose Study of QR-313 Evaluating Safety, Proof of Mechanism, Preliminary Efficacy and Systemic Exposure in Subjects With DDEB or RDEB Due to Mutation(s) in Exon 73 of the COL7A1 Gene

summary: A double-blind, randomized, intra-subject placebo-controlled, multicenter, multiple dose study, evaluating safety, proof of mechanism, preliminary efficacy and systemic exposure in subjects with confirmed DDEB or RDEB diagnosis with one or more pathogenic mutations in exon 73 in the COL7A1 gene.


title: An Open-Label Safety Study of Retinal Gene Therapy for Choroideremia With Bilateral, Sequential Administration of Adeno-Associated Viral Vector (AAV2) Encoding Rab Escort Protein 1 (REP1)

acronym: GEMINI

summary: The objective of the study is to evaluate the safety of bilateral, sequential sub-retinal administration of a single dose of BIIB111 in adult male participants with Choroideremia (CHM).

ReferenceCitation: Davis JL. The Blunt End: Surgical Challenges of Gene Therapy for Inherited Retinal Diseases. Am J Ophthalmol. 2018 Dec;196:xxv-xxix. doi: 10.1016/j.ajo.2018.08.038. Epub 2018 Sep 5. Review.

family: Davis

full_name: Janet L. Davis

given: Janet L.

journal_name: American Journal of Ophthalmology

lead_sponsor: NightstaRx Ltd, a Biogen Company

location_country:
Germany
United States
France


title: A Phase 1 Study of AG-270 in the Treatment of Subjects With Advanced Solid Tumors or Lymphoma With Homozygous Deletion of MTAP

summary: This study will evaluate the safety, pharmacokinetics, pharmacodynamics, and clinical activity of AG-270 in participants with advanced solid tumors or lymphoma with homozygous MTAP deletion.

ReferenceCitation: Konteatis Z, Travins J, Gross S, Marjon K, Barnett A, Mandley E, Nicolay B, Nagaraja R, Chen Y, Sun Y, Liu Z, Yu J, Ye Z, Jiang F, Wei W, Fang C, Gao Y, Kalev P, Hyer ML, DeLaBarre B, Jin L, Padyana AK, Dang L, Murtie J, Biller SA, Sui Z, Marks KM. Discovery of AG-270, a First-in-Class Oral MAT2A Inhibitor for the Treatment of Tumors with Homozygous MTAP Deletion. J Med Chem. 2021 Apr 22;64(8):4430-4449. doi: 10.1021/acs.jmedchem.0c01895. Epub 2021 Apr 8.

name: Agios Pharmaceuticals, Inc., 88 Sidney Street, Cambridge, Massachusetts 02139, United States

family: Konteatis

full_name: Zenon Konteatis

given: Zenon

family: Travins

full_name: Jeremy Travins

given: Jeremy

family: Gross

full_name: Stefan Gross

given: Stefan

family: Marjon

full_name: Katya Marjon

given: Katya

family: Barnett

full_name: Amelia Barnett

given: Amelia

family: Mandley

full_name: Everton Mandley

given: Everton

family: Nicolay

full_name: Brandon Nicolay

given: Brandon

family: Nagaraja

full_name: Raj Nagaraja

given: Raj

family: Chen

full_name: Yue Chen

given: Yue

family: Sun

full_name: Yabo Sun

given: Yabo

family: Liu

full_name: Zhixiao Liu

given: Zhixiao

family: Yu

full_name: Jie Yu

given: Jie

family: Ye

full_name: Zhixiong Ye

given: Zhixiong

family: Jiang

full_name: Fan Jiang

given: Fan

family: Wei

full_name: Wentao Wei

given: Wentao

family: Fang

full_name: Cheng Fang

given: Cheng

family: Gao

full_name: Yi Gao

given: Yi

family: Kalev

full_name: Peter Kalev

given: Peter

family: Hyer

full_name: Marc L. Hyer

given: Marc L.

family: DeLaBarre

full_name: Byron DeLaBarre

given: Byron

family: Jin

full_name: Lei Jin

given: Lei

family: Padyana

full_name: Anil K. Padyana

given: Anil K.

family: Dang

full_name: Lenny Dang

given: Lenny

ipd_sharing_description:
Qualified scientific and medical researchers can request access to anonymized patient-level and study-level clinical trial data.

Access can be requested for all interventional clinical studies:

used for Marketing Authorization (MA) of medicines and new indications approved after 1 January 2014 in the European Economic Area (EEA) or the United States (US).
where Servier is the Marketing Authorization Holder (MAH). The date of the first MA of the new medicine (or the new indication) in one of the EEA Member States will be considered for this scope.

In addition, access can be requested for all interventional clinical studies in patients:

sponsored by Servier
with a first patient enrolled as of 1 January 2004 onwards
for New Chemical Entity or New Biological Entity (new pharmaceutical form excluded) for which development has been terminated before any Marketing authorization (MA) approval.

lead_sponsor: Institut de Recherches Internationales Servier

location_facility:
Nashville
Barcelona
New York
Villejuif Cedex
Boston
New Haven


title: ASPIRO: A Phase 1/2/3, Randomized, Open-Label, Ascending-Dose, Delayed-Treatment Concurrent Control Clinical Study to Evaluate the Safety and Efficacy of AT132, an AAV8-Delivered Gene Therapy in X-Linked Myotubular Myopathy (XLMTM) Patients

acronym: ASPIRO

summary: This is a multinational, open-label, ascending-dose, delayed-treatment concurrent control clinical study to evaluate the safety and efficacy of AT132 in subjects with X-Linked Myotubular Myopathy aged less than 5 years old. Subjects will receive a single dose of AT132 and will be followed for safety and efficacy for 5 years

lead_sponsor: Audentes Therapeutics

location_country:
Germany
United States
Canada
France

location_facility: Kinderklinik und Kinderpoliklinik im Dr. Von Haunerschen Kinderspital Klinikum der Universitat Munchen

location_facility: National Institute of Neurological Disorders and Stroke/NIH Porter

location_facility: Ann & Robert H Lurie Children's Hospital of Chicago

intervention_type: Genetic


title: A Phase 1 Study of LY3009120 in Patients With Advanced or Metastatic Cancer

summary: The main purpose of this study is to see how safe the investigational drug known as LY3009120 is and whether it will work to help people with advanced cancer or cancer that has spread to other parts of the body.

location_country:
United States
Australia
France
Spain

location_facility: University of Texas MD Anderson Cancer Center


title: A Phase 1b Open Label, Dose Escalation Study to Assess Safety, Pharmacokinetics, Pharmacodynamics, and Antitumor Activity of PLX3397 in Combination With Vemurafenib in V600-mutated BRAF Unresectable or Metastatic Melanoma

summary: The purpose of this research study is to test the safety of an investigational new drug called PLX3397 when used in combination with Vemurafenib (Zelboraf™) at different dose levels. Vemurafenib has been approved by the United States Food and Drug Administration (FDA)/European Medicines Agency (EMA) for the treatment of a specific category of unresectable or metastatic melanoma.

ipd_sharing_description: De-identified individual participant data (IPD) and applicable supporting clinical trial documents may be available upon request at https://vivli.org/. In cases where clinical trial data and supporting documents are provided pursuant to our company policies and procedures, Daiichi Sankyo will continue to protect the privacy of our clinical trial participants. Details on data sharing criteria and the procedure for requesting access can be found at this web address: https://vivli.org/ourmember/daiichi-sankyo/

lead_sponsor: Daiichi Sankyo, Inc.

location_facility:
Institute Gustave Roussy
UCLA
University of Colorado, Denver
University Hospital Essen
Vanderbilt University
Seattle Cancer Care Alliance


title: pCLE For the Diagnosis Of Cancer in Unknown Bile Duct Stricture

acronym: FOCUS

summary: This study will collect data from patients undergoing an ERCP procedure with probe-based Confocal Laser Endomicroscopy for suspicion of bile duct cancer. the objective is to evaluate the diagnostic performance of pCLE for the diagnosis of indeterminate biliary stricture when associated with other diagnostic information (standard ERCP and tissue sampling).

ReferenceCitation: Slivka A, Gan I, Jamidar P, Costamagna G, Cesaro P, Giovannini M, Caillol F, Kahaleh M. Validation of the diagnostic accuracy of probe-based confocal laser endomicroscopy for the characterization of indeterminate biliary strictures: results of a prospective multicenter international study. Gastrointest Endosc. 2015 Feb;81(2):282-90. doi: 10.1016/j.gie.2014.10.009.

family: Slivka

full_name: Adam Slivka

given: Adam

family: Gan

full_name: Ian Gan

given: Ian

family: Jamidar

full_name: Priya Jamidar

given: Priya

family: Costamagna

full_name: Guido Costamagna

given: Guido

family: Cesaro

full_name: Paola Cesaro

given: Paola

family: Giovannini

full_name: Marc Giovannini

given: Marc

family: Caillol

full_name: Fabrice Caillol

given: Fabrice

family: Kahaleh

full_name: Michel Kahaleh

given: Michel

lead_sponsor: Mauna Kea Technologies

location_country:
Italy
United States
France

location_facility: Policlinico Univertitario Agostino Gemelli

location_facility: New York presbyterian Weill Cornell Medical center


title: A Phase Ib, Open-label, Multi-center, Dose-escalation and Expansion Study of an Orally Administered Combination of BEZ235 Plus MEK162 in Adult Patients With Selected Advanced Solid Tumors

summary:
This is an open label, dose finding, phase Ib clinical trial to determine the maximum tolerated dose (MTD) and/or RP2D of the orally administered PI3K/mTOR inhibitor BEZ235 in combination with the MEK1/2 inhibitor MEK162. This combination will be explored in patients with EGFR mutant NSCLC which has progressed on EGFR inhibitors and triple negative breast cancer, as well as pancreatic cancer, colorectal cancer, malignant melanoma, NSCLC, and other advanced solid tumors with KRAS, NRAS, and/or BRAF mutations. Dose escalation will be guided by a Bayesian logistic regression model with overdose control. At MTD or RP2D, two expansion arms will be opened in order to further assess safety and preliminary anti-tumor activity of the combination of BEZ235 and MEK162.

Study drugs will be administered orally on a continuous schedule, MEK162 bid and BEZ235 qd, a treatment cycle is defined as 28 days.

location_country:
United States
France
Australia
Spain
Canada

location_facility: University of Texas/MD Anderson Cancer Center MD Anderson PSC

location_facility: Massachusetts General Hospital Mass General 2