lead_sponsor: Rhythm Pharmaceuticals, Inc.

summary: To demonstrate statistically significant and clinically meaningful effects of setmelanotide on percent body weight change in participants with LEPR deficiency obesity due to rare bi-allelic or loss-of function mutations at the end of 1 year of treatment.

ReferenceCitation: Clement K, van den Akker E, Argente J, Bahm A, Chung WK, Connors H, De Waele K, Farooqi IS, Gonneau-Lejeune J, Gordon G, Kohlsdorf K, Poitou C, Puder L, Swain J, Stewart M, Yuan G, Wabitsch M, Kuhnen P; Setmelanotide POMC and LEPR Phase 3 Trial Investigators. Efficacy and safety of setmelanotide, an MC4R agonist, in individuals with severe obesity due to LEPR or POMC deficiency: single-arm, open-label, multicentre, phase 3 trials. Lancet Diabetes Endocrinol. 2020 Dec;8(12):960-970. doi: 10.1016/S2213-8587(20)30364-8. Epub 2020 Oct 30.

location_facility: University of Cambridge Metabolic Research Laboratories

location_facility: Institute of Cardiometabolism and Nutrition / Pitié-Salpêtrière Hospital

title: An Open Label, 1-Year Trial, Including a Double-Blind Placebo-Controlled Withdrawal Period, of Setmelanotide (RM-493), a Melanocortin 4 Receptor (MC4R) Agonist, in Leptin Receptor (LEPR) Deficiency Obesity Due to Bi-Allelic Loss-of-Function LEPR Genetic Mutation

location_country:
Germany
Réunion
United Kingdom
Netherlands
Canada
France


title: Performance of a New REsting Pressure Index During Invasive Angiography Compared To Adenosine Hyperemic FFR

acronym: PREDICT

summary: To test the feasibility and diagnostic accuracy of a new automated pressure derived resting index (Pd/Pamin), using FFR as gold standard, in de novo coronary lesions in which invasive physiological evaluation is warranted.

study_start_date_type: Actual

status: Unknown status

study_completion_date_type: Anticipated

study_type: Observational

time_perspective: Prospective

enrollment_type: Anticipated

ReferenceType: background

ReferenceCitation: Levine GN, Bates ER, Blankenship JC, Bailey SR, Bittl JA, Cercek B, Chambers CE, Ellis SG, Guyton RA, Hollenberg SM, Khot UN, Lange RA, Mauri L, Mehran R, Moussa ID, Mukherjee D, Nallamothu BK, Ting HH; American College of Cardiology Foundation; American Heart Association Task Force on Practice Guidelines; Society for Cardiovascular Angiography and Interventions. 2011 ACCF/AHA/SCAI Guideline for Percutaneous Coronary Intervention. A report of the American College of Cardiology Foundation/American Heart Association Task Force on Practice Guidelines and the Society for Cardiovascular Angiography and Interventions. J Am Coll Cardiol. 2011 Dec 6;58(24):e44-122. doi: 10.1016/j.jacc.2011.08.007. Epub 2011 Nov 7. No abstract available.

ReferenceCitation: Task Force on Myocardial Revascularization of the European Society of Cardiology (ESC) and the European Association for Cardio-Thoracic Surgery (EACTS); European Association for Percutaneous Cardiovascular Interventions (EAPCI); Wijns W, Kolh P, Danchin N, Di Mario C, Falk V, Folliguet T, Garg S, Huber K, James S, Knuuti J, Lopez-Sendon J, Marco J, Menicanti L, Ostojic M, Piepoli MF, Pirlet C, Pomar JL, Reifart N, Ribichini FL, Schalij MJ, Sergeant P, Serruys PW, Silber S, Sousa Uva M, Taggart D. Guidelines on myocardial revascularization. Eur Heart J. 2010 Oct;31(20):2501-55. doi: 10.1093/eurheartj/ehq277. Epub 2010 Aug 29. No abstract available.

ReferenceCitation: Mamas MA, Horner S, Welch E, Ashworth A, Millington S, Fraser D, Fath-Ordoubadi F, Neyses L, El-Omar M. Resting Pd/Pa measured with intracoronary pressure wire strongly predicts fractional flow reserve. J Invasive Cardiol. 2010 Jun;22(6):260-5.

ReferenceCitation: Sen S, Escaned J, Malik IS, Mikhail GW, Foale RA, Mila R, Tarkin J, Petraco R, Broyd C, Jabbour R, Sethi A, Baker CS, Bellamy M, Al-Bustami M, Hackett D, Khan M, Lefroy D, Parker KH, Hughes AD, Francis DP, Di Mario C, Mayet J, Davies JE. Development and validation of a new adenosine-independent index of stenosis severity from coronary wave-intensity analysis: results of the ADVISE (ADenosine Vasodilator Independent Stenosis Evaluation) study. J Am Coll Cardiol. 2012 Apr 10;59(15):1392-402. doi: 10.1016/j.jacc.2011.11.003. Epub 2011 Dec 7.

ReferenceCitation: Ganz P, Abben R, Friedman PL, Garnic JD, Barry WH, Levin DC. Usefulness of transstenotic coronary pressure gradient measurements during diagnostic catheterization. Am J Cardiol. 1985 Apr 1;55(8):910-4. doi: 10.1016/0002-9149(85)90716-7.

ReferenceCitation: Gruntzig AR, Senning A, Siegenthaler WE. Nonoperative dilatation of coronary-artery stenosis: percutaneous transluminal coronary angioplasty. N Engl J Med. 1979 Jul 12;301(2):61-8. doi: 10.1056/NEJM197907123010201.

ReferenceCitation: Berry C, van 't Veer M, Witt N, Kala P, Bocek O, Pyxaras SA, McClure JD, Fearon WF, Barbato E, Tonino PA, De Bruyne B, Pijls NH, Oldroyd KG. VERIFY (VERification of Instantaneous Wave-Free Ratio and Fractional Flow Reserve for the Assessment of Coronary Artery Stenosis Severity in EverydaY Practice): a multicenter study in consecutive patients. J Am Coll Cardiol. 2013 Apr 2;61(13):1421-7. doi: 10.1016/j.jacc.2012.09.065. Epub 2013 Feb 6.

ReferenceCitation: Ganz P, Harrington DP, Gaspar J, Barry WH. Phasic pressure gradients across coronary and renal artery stenoses in humans. Am Heart J. 1983 Dec;106(6):1399-406. doi: 10.1016/0002-8703(83)90052-2.

ipd_sharing: No

lead_sponsor: Centro Hospitalar Lisboa Ocidental

collaborators: Cardiovascular Research Foundation, New York

location_country:
United States
Italy
Portugal
France

location_facility: Institute of Cardiology, Catholic University of the Sacred Heart

location_facility: Hospital Prof. Doutor Fernando da Fonseca

location_facility: Department of Cardiology, University Hospital

location_facility: Centro Hospitalar de Lisboa Ocidental - Hospital de Santa Cruz

location_facility: Centro Hospitalar de Lisboa Central (CHLC) - Hospital de Santa Marta

lead_sponsor_type: academique

status_simplified: Unknown


title: A Randomised, Placebo Controlled, Double Blind, Multicentre Proof of Concept Study to Assess the Safety and Efficacy of Two Doses of VAD044 in Patients With Hereditary Hemorrhagic Telangiectasia (HHT)

summary: The purpose of this Phase 1b proof of concept study, randomised, placebo controlled, double blind, multicentre study is to asssess safety and efficacy of 2 doses of VAD044 in adult HHT patients

status: Recruiting

study_type: Interventional

design_allocation: Randomized

primary_purpose: Treatment

lead_sponsor: Vaderis Therapeutics AG

location_country:
Italy
Netherlands
Belgium
United States
Spain
France

location_facility: Washington University School of Medicine

intervention_type: Drug

lead_sponsor_type: industriel

status_simplified: Ongoing


type: EudraCT Number

title: A Phase I, Open-label, Multi-center Study of PIT565 in Patients With Relapsed and/or Refractory B-cell Malignancies

summary: This is an open-label, multicenter, phase I study, which primary objective is to characterize the safety and tolerability of PIT565 and to identify maximal tolerated doses (MTDs) and/or recommended doses (RDs), schedule and route of administration in relapsed and/or refractory B-cell Non-Hodgkin lymphoma (R/R B-NHL) and relapsed and/or refractory B-cell acute lymphoblastic leukemia (R/R B-ALL).

design_allocation: Non-Randomized

lead_sponsor: Novartis Pharmaceuticals

location_country:
France
Italy
Singapore
Japan
Belgium
United States
Spain
Israel

location_facility:
Novartis Investigative Site
Memorial Sloan Kettering Cancer Ctr .
University Of Miami .

intervention_type: Biological


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.

status: Completed

study_completion_date_type: Actual

primary_purpose: Basic Science

enrollment_type: Actual

ReferenceCitation: Cadoudal T, Buleon M, Sengenes C, Diene G, Desneulin F, Molinas C, Eddiry S, Conte-Auriol F, Daviaud D, Martin PG, Bouloumie 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.

oa_host_type: publisher

evidence: open (via free pdf)

host_type: publisher

license_normalized: no license

version: publishedVersion

repositories_concat: closed

unpaywall_oa_status: bronze

publisher_dissemination: Springer

ReferenceType: derived

ReferenceCitation: Beauloye V, Diene G, Kuppens R, Zech F, Winandy C, Molinas C, Faye S, Kieffer I, Beckers D, Nergardh 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.

oa_colors: green gold

oa_host_type: publisher;repository

evidence: oa journal (via doaj)

license: cc-by

license_normalized: cc-by

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

host_type: repository

repository_institution: PubMed Central - Europe PMC

repository_normalized: PubMed Central

repository_institution: Erasmus University Rotterdam - EUR Research Repository

evidence: oa repository (via pmcid lookup)

repositories_concat: PubMed Central;pure.eur.nl;repub.eur.nl

unpaywall_oa_status: gold

publisher_dissemination: Springer-Nature

lead_sponsor: University Hospital, Toulouse

collaborators: European Society for Paediatric Endocrinology

location_country:
Germany
France
United Kingdom
Netherlands
Belgium
Sweden

location_facility: Department of Endocrinology / University Children's Hospital

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

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

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

intervention_type: Other

status_simplified: Completed


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:
United Kingdom
Algeria
Spain
Ukraine
Russian Federation
France

location_facility: Centre hospitalo-universitaire de Beni Messous

location_facility: Complejo Hospitalario Universitario de Santiago

location_facility: Lviv State Oncological Regional Treatment and Diagnostic Center


lead_sponsor: Cellaion SA

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

location_facility: Hospital Universitari Vall d'Hebron de Barcelona

location_facility: Instytut - Pomnik Centrum Zdrowia Dziecka

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

source: euctr

location_country: Spain Poland Belgium France


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

collaborators: Great Ormond Street Hospital for Children NHS Foundation Trust

location_country:
United States
United Kingdom
Netherlands
France

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

ReferenceType: result

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.

lead_sponsor: Catholic University of the Sacred Heart

location_country:
United States
Italy
Japan
France

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

location_facility: Indiana Univerisity Health Medical Center

location_facility: Teikyo University Mizonokuchi Hospital, Departement of Gastroenterology

intervention_type: Device


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.

status: Active, not recruiting

time_perspective: Retrospective

lead_sponsor: Zimmer Biomet

location_country:
Ireland
Germany
Netherlands
United States
Spain
France

location_facility: Midwestern Regional Orthopaedic Hospital


acronym: Neptuno

lead_sponsor: PharmaMar

summary: Treatment of patients hospitalised for management of moderate COVID-19 infection

location_facility: Sotiria Hospital General Hospital of Chest Diseases of Athens "Sotiria" 3rd Department of Internal Medicine of University of Athens

location_facility: Instituto de Investigación Sanitaria Valdecilla (IDIVAL)

location_facility: Hospital Universitario de Jerez de la Frontera

location_facility: Centre Hospitalier Regional et Universitaire de Tours (CHRU Tours) - Hopital Bretonneau

location_facility: Institutul National De Boli Infectioase "Prof. Dr. Matei Bals"

location_facility: Evangelismos Hospital General Hospital of Athens Evangelismos, Intensive Care Unit

location_facility: Spitalul Clinic de Boli Infectioase si Tropicale Dr. Victor Babes - Bucharest

location_facility: Hospital General Universitario de Alicante

location_facility: Hospital Universitario Virgen de las Nieves (HUVN)

location_facility: Spitalul Judetean de Urgenta 'Sf. Ioan cel Nou' Suceava, Sectia de Boli Infectioase

location_facility: Hospital de Emergencias Enfermera Isabel Zendal

location_facility: Hospital Universitari Germans Trias i Pujol

location_facility: Universidad Autonoma de Nuevo Leon - Hospital Universitario "Dr. Jose Eleuterio Gonzalez"

location_facility: Spitalul Clinic De Boli Infectioase "Sfanta Parascheva" IASI, Sectia Boli Infectioase III

status: Terminated

title: A Phase 3, Multicentre, Randomised, Controlled Trial to Determine the Efficacy and Safety of Two Dose Levels of Plitidepsin Versus Control in Adult Patient Requiring Hospitalisation for Management of Moderate COVID-19 Infection

location_country: Colombia Romania Brazil Mexico Spain Greece Bulgaria France


title: A Prospective, Open, Non-comparative, Post-market Clinical Follow-up Investigation to Confirm the Safety and Performance of Avance Solo and Avance Solo Adapt NPWT Systems in Low to Moderate Exuding Chronic Wounds

acronym: ASOLO-CW

summary: The primary objective of this post market clinical follow-up (PMCF) investigation is to confirm the safety and performance of Avance®Solo and Avance®Solo Adapt NPWT Systems in low to moderate exuding chronic wounds when used in accordance with the Instructions for Use, for up to 28 days.

lead_sponsor: Molnlycke Health Care AB

sponsor_collaborators:
Molnlycke Health Care AB
Syntactx

location_country: Germany Ireland Italy Portugal Denmark Belgium France

location_facility: Klinik fur Dermatologie, Venerologie und Allergologie Campus Benjamin Franklin

location_facility: Unidade de Cuidados Continuados António Francisco Guimarães

location_facility: Trofa Saúde Hospital Central Hospital da Trofa

location_facility: A.O.U. Citta della Salute e della Scienza di Torino - A.O.U. Molinette San Giovanni Battista


lead_sponsor: BioMarin Pharmaceutical

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.

location_facility: Great Ormond Street Hospital for Children

location_facility: Institute of Genetic Medicine International Centre for Life

location_facility: Policlinico Universitario Agostino Gemelli

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.

location_country:
Italy
United Kingdom
Netherlands
Belgium
France


title: A Phase 1/2, Open-Label, Multiple Ascending Dose Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of INZ-701 Followed by an Open-Label Long-Term Extension Period in Adults With ENPP1 Deficiency

summary: The purpose of this study is to assess the safety, tolerability, pharmacokinetics (PK), and pharmacodynamics (PD) of multiple ascending doses of INZ-701, an ectonucleotide pyrophosphatase/phosphodiesterase 1 (ENPP1) enzyme replacement therapy, for the treatment of ENPP1 Deficiency. The goal of the study is to identify a dose regimen for further clinical development in the treatment of ENPP1 Deficiency.

lead_sponsor: Inozyme Pharma

location_country:
Germany
United Kingdom
United States
Canada
France

location_facility: Necker University Hospital-Sick Children

location_facility: University of Hamburg (Universitatklinikum Hamburg-Eppendorf)


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:
Germany
Italy
Portugal
United Kingdom
Australia
France

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


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: Germany Italy India Netherlands Belgium France

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: Germany Poland Hungary Romania Czechia France


acronym: INSIGHT

lead_sponsor: Janssen Research & Development, LLC

summary: The purpose of this study is to assess changes in intrahepatic hepatitis B surface antigen (HBsAg) between baseline and on-treatment liver biopsy in response to JNJ-3989-based combination treatment.

location_facility:
Hopital Beaujon
Johns Hopkins University
University Medical Center
New Zealand Clinical Research
ID Clinic
Kings College Hospital
Irccs Ospedale Maggiore Di Milano
UZ Antwerpen
Toronto General Hospital
Grahame Hayton Unit

title: A Phase 2 Randomized, Open-label, Parallel-group, Multicenter Study to Assess Intrahepatic and Peripheral Changes of Immunologic and Virologic Markers in Response to Combination Regimens Containing JNJ-73763989 and Nucleos(t)Ide Analog With or Without JNJ-56136379 in Patients With Chronic Hepatitis B Virus Infection

type: Other Identifier

ipd_sharing_description:
The data sharing policy of the Janssen Pharmaceutical Companies of Johnson & Johnson is available at www.janssen.com/clinical-trials/transparency.

As noted on this site, requests for access to the study data can be submitted through Yale Open Data Access (YODA) Project site at yoda.yale.edu

location_country:
Germany
Italy
Poland
New Zealand
United Kingdom
Belgium
United States
Canada
France

ipd_sharing: Yes


title: Myeloproliferative Neoplasms (MPN) and COVID-19

acronym: MPN-COVID

summary: An increased risk of both venous and arterial thromboembolism was noted in reports from SARS-CoV-2-infected patients in China and has been confirmed in autopsy findings from patients who experienced sudden death. Myeloproliferative Neoplasms (MPNs), which encompass polycythemia vera, essential thrombocythemia and primary myelofibrosis, are thrombophilic disorders with a natural propensity to thrombosis that is fuelled by the intrinsic activation of inflammatory cytokines. It therefore follows that an underlying diagnosis of MPN may increase the risk of worse clinical outcomes and death during periods of active Covid-19 disease. This ambispective, observational study aims to elucidate the key factors which affect the clinical course of patients with MPN who develop Covid-19 disease.

time_perspective: Other

ipd_sharing_description: Each participant can visualize its own data on eCRF after the completion patient's electronic data form.

lead_sponsor: Fondazione per la Ricerca Ospedale Maggiore

location_country:
Germany
Italy
Poland
United Kingdom
United States
Spain
Croatia
Bulgaria
France

location_facility: A.O.U. Città della Salute e della Scienza di Torino

location_facility: ICO L'Hospitalet - Hospital Moisès Broggi

location_facility: A.S.O. SS. Antonio e Biagio e C.Arrigo di Alessandria

location_facility: University Clinic for Hematology, Oncology, Hemostaseology and Palliative Care, Johannes Wesling Medical Center

location_facility: ICO Girona Hospital Josep Trueta Servei d'hematologia

location_facility: University Medicine Greifswald - Hematology, Oncology, Stem Cell Transplantation and Palliative Care

location_facility: Fondazione IRCCS Cà Granda Ospedale Maggiore Policlinico

location_facility: Policlinico Universitario Fondazione Agostino Gemelli

location_facility: FEA Hematología Hospital Universitario de Móstoles

location_facility: Guy's and St. Thomas' NHS Foundation Trust.

location_facility: Azienda Ospedaliero Universitaria Maggiore della Carità

location_facility: New York-Presbyterian/Weill Cornell Medical Center

location_facility: Servicio de Hematología Hospital General Universitario de Albacete

location_facility: Hospital Universitario Príncipe de Asturias

location_facility: National Specialised Hospital for Active Treatment of Hematological Diseases

location_facility: University Hospital Halle, Department of Hematology/Oncology

location_facility: Institut Català d' Oncologia - Hospital Germans Trias i Pujol

location_facility: Ospedale Policlinico "G.B. Rossi" Borgo Roma

location_facility: University hospital Dubrava-School of Medicine University of Zagreb


lead_sponsor: Sumitomo Pharma America, Inc.

summary: This is an open-label, multi-center, multi-national, non-randomized rollover study designed to allow continued access to napabucasin for patients who have participated in a Boston Biomedical-sponsored study and are being treated with napabucasin (monotherapy or combination) and who are deriving continued clinical benefit in the parent study at the time of closure.

location_facility: Winship Cancer Institute, Emory University

title: A Rollover Study to Provide Continued Access to Napabucasin for Patients Enrolled in Boston Biomedical-sponsored Napabucasin Protocols

location_country:
United States
Japan
Taiwan
France


title: Observational Study, for Quality Assessment, of Sarcoma as a Model to Improve Diagnosis and Clinical Care of Rare Tumors Through a European and Latin American Multidisciplinary NETWORK

acronym: (SELNET)

summary:
Post-authorisation, multicentric, observational, retrospective and prospective study to assess quality of care of sarcoma patients in expert and non-expert centers by analysing correlation of quality items and outcomes such as relapse free survival, overall survival, percentage of amputation, etc.

Expert pathology peer review will be performed to detect differences between expert and non-expert centers as well as differences in treatment and patient prognosis.

Tumor samples of 4 types of sarcoma would also be included in translational research to detect biomarkers and produce preclinical models.

ipd_sharing_description: Data could be shared receiving an application to the Study Main contact indicating reason and objectives.

lead_sponsor: Grupo Espanol de Investigacion en Sarcomas

location_country:
Italy
Argentina
Brazil
Costa Rica
Mexico
Spain
Peru
France

location_facility: Instituo Nacional de enfermedades Neoplásicas


title: A Phase II, Randomized, Double-Blind, Placebo-Controlled, Parallel Group, Efficacy and Safety Study of NV1FGF in Patients With Severe Peripheral Artery Occlusive Disease.

acronym: TALISMAN 201

summary: This study is a double-blind study to evaluate the efficacy and safety of NV1FGF, a pCOR plasmid constructed by inserting the gene coding for the FGF compared to placebo in patients with severe Peripheral Arterial Occlusive Disease, Fontaine's stage IV. The efficacy was assessed by the complete healing of at least one ulcer in the treated limb, 25 week post-baseline and secondary by the rate of amputation and death.

ReferenceCitation: Nikol S, Baumgartner I, Van Belle E, Diehm C, Visona A, Capogrossi MC, Ferreira-Maldent N, Gallino A, Wyatt MG, Wijesinghe LD, Fusari M, Stephan D, Emmerich J, Pompilio G, Vermassen F, Pham E, Grek V, Coleman M, Meyer F; TALISMAN 201 investigators. Therapeutic angiogenesis with intramuscular NV1FGF improves amputation-free survival in patients with critical limb ischemia. Mol Ther. 2008 May;16(5):972-8. doi: 10.1038/mt.2008.33. Epub 2008 Apr 1.

ReferenceCitation: Niebuhr A, Henry T, Goldman J, Baumgartner I, van Belle E, Gerss J, Hirsch AT, Nikol S. Long-term safety of intramuscular gene transfer of non-viral FGF1 for peripheral artery disease. Gene Ther. 2012 Mar;19(3):264-70. doi: 10.1038/gt.2011.85. Epub 2011 Jun 30.

lead_sponsor: Sanofi

location_country:
Germany
Italy
United Kingdom
Switzerland
Belgium
France

location_facility:
Hôpital Européen Georges Pompidou
Ghent University Hospital
St George's Hospital
Universitätsklinik
Klinikum Karlsbad-Langensteinbach
Istituto Dermopatico dell'Immacolata


intervention_type: Genetic

lead_sponsor: bluebird bio

summary: The purpose of this study is to evaluate the efficacy and safety of Lenti-D Drug Product (also known as elivaldogene autotemcel or Skysona, hereafter referred to as eli-cel) after myeloablative conditioning with busulfan and fludarabine in participants with CALD. A participant's blood stem cells will be collected and modified (transduced) using the Lenti-D lentiviral vector encoding human adrenoleukodystrophy protein. After modification (transduction) with the Lenti-D lentiviral vector, the cells will be transplanted back into the participant following myeloablative conditioning. Enrollment and treatment in Study ALD-104 have been completed and further enrollment in this study is not expected, although participants follow-up remains ongoing.

location_facility: Boston Children's Hospital/Massachusetts General Hospital

title: A Phase 3 Study of Lenti-D Drug Product After Myeloablative Conditioning Using Busulfan and Fludarabine in Subjects ≤17 Years of Age With Cerebral Adrenoleukodystrophy (CALD)

ipd_sharing_description: Bluebird bio is committed to transparency and appropriately de-identified patient-level datasets and supporting documents may be shared following attainment of applicable marketing approvals associated with this study and consistent with criteria established by bluebird bio and/or industry best practices to maintain the privacy of study participants. For enquiries, please contact us at datasharing@bluebirdbio.com.

location_country:
Germany
Italy
United Kingdom
Netherlands
United States
France


title: Evaluation of the Xpert® Bladder Cancer Monitor Assay Compared to Cystoscopy for the Follow-up of Patients With History of Low or Intermediate Risk Non-muscle-invasive Bladder Cancer (NMIBC): an Observational Prospective International Multicenter Study

acronym: ANTICIPATE X

summary: Bladder cancer is the 5th most common cancer in Europe, with more than 151,000 new cases diagnosed in 2012 (4% of the total). Bladder cancer has the highest recurrence rate of any malignancy, often as high as 70% within 5 years of successful treatment. This high recurrence rate requires diligent and accurate monitoring as a means for early diagnosis and treatment. Considering the burden associated to repeated invasive cystoscopies, there is a need for robust but accurate tests for surveillance. In that prospect, urinary molecular tests have been developed although none were deemed adequate in the European clinical guidelines to replace cystoscopies. The Xpert Bladder Cancer Monitor Assay is a qualitative in vitro diagnostic test designed to monitor for the recurrence of bladder cancer in patients previously diagnosed with this cancer. The test provides a fast and accurate result, is non-invasive and easy to perform. The aim of this study is to assess the non-inferiority of the Xpert Bladder Cancer Monitor assay in detecting recurrences in comparison to cystoscopy in the follow-up of patients with low or intermediate risk non-muscle-invasive bladder cancer (NMIBC).

lead_sponsor: Cepheid

collaborators:
Axonal-Biostatem
Stève Consultants

sponsor_collaborators:
Cepheid
Axonal-Biostatem
Stève Consultants

location_country:
Germany
Italy
United Kingdom
Sweden
Netherlands
Austria
Spain
Czechia
France

location_facility: University Clinic for Urology and Andrology

location_facility: Humanitas University - Gradenigo Hospital of Turin

location_facility: CHU Toulouse / Institut Universitaire du Cancer Toulouse Oncopole

intervention_type: Diagnostic Test


title: Study of CNGB1 Retinitis Pigmentosa and Allied Hereditary Disorders

summary: Mutations in the rod-expressed gene, cyclic nucleotide-gated channel beta subunit (CNGB1) and associated inborn errors in metabolism are causes of retinal disease that causes progressive loss of vision. Retinitis pigmentosa (RP) is a major cause of untreatable blindness associated with CNGB1 (CNGB1-RP). RP involves the death of photoreceptor cells that can be caused by mutations in a number of different genes. Treatment by gene therapy could prevent blindness in cases of inherited retinal dystrophies including RP. In the future RP due to mutations in CNGB1 may be treatable by gene therapy since this form of photoreceptor degeneration involves a slow loss of rod photoreceptor cells. This provides a wide window of opportunity for the identification of patients and initiation of treatment. Our efforts are directed toward developing gene therapy as a treatment. To this end, our objective is to better understand the disease process of CNGB1-RP and other allied inherited disorders so that we can develop clinical tests to measure the outcomes of treatment.

lead_sponsor: Columbia University

collaborators: Moorfields Eye Hospital NHS Foundation Trust

collaborators: Ludwig-Maximilians - University of Munich

location_country:
United States
United Kingdom
Germany
France

location_facility: Klinikum der Universität München University Eye Hospital, Ludwig-Maximilians-University (LMU) Munich

location_facility: Institut de la Vision/Centre de maladies rares du Centre Hospitalier National Ophtalmologique des Quinze-Vingts


title: JuggerStitch™ for Meniscal Repair Post Market Clinical Follow-up Study

summary: This study is a post-market follow-up study. The data collected from this study will serve the purpose of confirming the safety and performance of the JuggerStitch Device used for meniscal repair according to the product labelling (Instruction For Use).

location_country:
United States
Japan
Germany
France

location_facility: Ramsay General de Sante - Hôpital Privé Jean Mermoz - Centre Orthopédique Santy

location_facility: Tokyo Medical and Dental University Hospital of Medicine

location_facility: Medizinische Fakultät der Otto-von-Guericke-Universität Magdeburg


title: The Comprehensive Aneurysm Management Trial

acronym: CAM

summary:
The uncertainty regarding the management of Unruptured Intracranial Aneurysms (UIAs) has not progressed in the last 30 years. The fundamental ethical basis for this study is that physicians should only offer a risky preventive treatment when it has been shown to be beneficial. Before that, such treatment should be offered as an RCT. The CAM trial offers a comprehensive framework, so that all patients confronted with the clinical dilemma can be offered participation.

The prinicpal questions to be addressed are :

do patients with UIAs, considered for curative treatments, have a better long-term clinical outcome with active treatment or conservative management?
when patients are considered ineligible for conservative management, and surgical and endovascular management are both judged reasonable, do patients with UIAs have a better long-term clinical outcome with surgical or endovascular management? The primary hypothesis for patients allocated to at least 2 options, one of which is conservative management is: the 10 year combined neurological morbidity and mortality (mRS>2) will be reduced from 24% to 16% (beta 80%; alpha 0.048; sample size 961 patients (836 plus 15% losses to FU and cross-overs) with active treatment.

This study is designed as a pragmatic, comprehensive way to address the unruptured aneurysm clinical dilemma, combining large simple RCTs whenever patients are judged eligible for more than one management option, or otherwise a registry of each option. All patients with one or more UIAs will be eligible for participation in either a registry or one of the trials. Patients will be followed for 10 years according to a standard of car follow-up schedule.

The primary outcome is survival without neurological dependency (mRS<3) at 10 years.

The secondary outcomes are:

the incidence of SAH during follow-up and related morbidity and mortality;
the morbidity and mortality related to endovascular or surgical treatment of the UIA at one year;
overall mortality at 1, 5 and 10 years;
overall morbidity (mRS>2) at 1, 5 and 10 years;
length of hospitalization;
discharge to location other than home

lead_sponsor: Centre hospitalier de l'Université de Montréal (CHUM)

location_country: Italy Canada France

location_facility: Centre Hospitalier Régional Universitaire de Tours


title: The CorNeat EverPatch - a First-In-Man Clinical Study for Demonstrating the Safety of a Synthetic Tissue Substitute for Concealment of Artificial Implants and Glaucoma Tube Shunts

summary:
Study Name: The CorNeat EverPatch - a First-In-Man Clinical Study for demonstrating the Safety of a Synthetic Tissue Substitute for concealment of artificial implants and glaucoma tube shunts

Objective: The objective of this clinical trial is to demonstrate the Safety of the Corneat Everpatch for concealment of artificial implants and glaucoma tube shunts

The study will consist 10 subjects requiring concealment of a glaucoma shunt or other ophthalmic implant. Eligible subjects who signs an ICF will be enrolled to the study. Subjects will be implanted with the Corneart EverPatch as part of a glaucoma shunt surgery or during a corrective surgery to repair a breached conjunctiva over an implanted device. Subjects will be monitored for a period of 12 months post-op during which follow up visits will occur at 1 week, 1, 2, 3, 6, 9 & 12 months following surgery including clinical examination of the operated eye using slit-lamp biomicroscopy and imaging using OCT or UBM (will be performed only at the 6 & 12 months follow up visits).

lead_sponsor: CorNeat Vision Ltd.

location_country: Kenya Canada France

location_facility: University Health Network - Toronto Western Hospital

location_facility: Hospital Foundation Adolphe De Rothschild


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: Finland Italy Belgium Greece France

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.

lead_sponsor: Rennes University Hospital

location_country:
Germany
United Kingdom
Australia
Belgium
Luxembourg
France

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


acronym: RESTORE

lead_sponsor: GenSight Biologics

summary: The goal of this clinical trial is to assess the long-term safety and efficacy of GS010, a gene therapy, and assess the quality of life in subjects with LHON due to the G11778A ND4 mitochondrial mutation and who were treated in the Rescue or Reverse studies.

ReferenceCitation: Newman NJ, Yu-Wai-Man P, Carelli V, Biousse V, Moster ML, Vignal-Clermont C, Sergott RC, Klopstock T, Sadun AA, Girmens JF, La Morgia C, DeBusk AA, Jurkute N, Priglinger C, Karanjia R, Josse C, Salzmann J, Montestruc F, Roux M, Taiel M, Sahel JA. Intravitreal Gene Therapy vs. Natural History in Patients With Leber Hereditary Optic Neuropathy Carrying the m.11778G>A ND4 Mutation: Systematic Review and Indirect Comparison. Front Neurol. 2021 May 24;12:662838. doi: 10.3389/fneur.2021.662838. eCollection 2021.

repository_institution: Le Centre pour la Communication Scientifique Directe - HAL - Université de Nantes

repository_normalized: HAL

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

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

repository_institution: University College London - UCL Discovery

repository_normalized: UCL Discovery

repository_institution: California Digital Library - eScholarship

repositories:
HAL
PubMed Central
UCL Discovery
escholarship.org

publisher_dissemination: Frontiers

location_facility: LMU Klinikum der Universität München / Friedrich-Baur-Institut

title: Long-term Follow-up of ND4 LHON Subjects Treated With GS010 Ocular Gene Therapy in the RESCUE or REVERSE Phase III Clinical Trials (RESTORE)

location_country:
Germany
Italy
United Kingdom
United States
France


title: International Multicentre Observational Trial of Perioperative Ventilatory Management in Cardiac Surgery

acronym: VENICE

summary: The purpose of this study is to evaluate prospectively ventilatory practices in the perioperative cardiac surgery period.

study_start_date_type: Anticipated

ReferenceCitation: Futier E, Constantin JM, Paugam-Burtz C, Pascal J, Eurin M, Neuschwander A, Marret E, Beaussier M, Gutton C, Lefrant JY, Allaouchiche B, Verzilli D, Leone M, De Jong A, Bazin JE, Pereira B, Jaber S; IMPROVE Study Group. A trial of intraoperative low-tidal-volume ventilation in abdominal surgery. N Engl J Med. 2013 Aug 1;369(5):428-37. doi: 10.1056/NEJMoa1301082.

evidence: open (via free article)

publisher_dissemination: Massachusetts Medical Society

ReferenceCitation: Lellouche F, Delorme M, Bussieres J, Ouattara A. Perioperative ventilatory strategies in cardiac surgery. Best Pract Res Clin Anaesthesiol. 2015 Sep;29(3):381-95. doi: 10.1016/j.bpa.2015.08.006. Epub 2015 Sep 4.

ReferenceCitation: Guldner A, Kiss T, Serpa Neto A, Hemmes SN, Canet J, Spieth PM, Rocco PR, Schultz MJ, Pelosi P, Gama de Abreu M. Intraoperative protective mechanical ventilation for prevention of postoperative pulmonary complications: a comprehensive review of the role of tidal volume, positive end-expiratory pressure, and lung recruitment maneuvers. Anesthesiology. 2015 Sep;123(3):692-713. doi: 10.1097/ALN.0000000000000754.

lead_sponsor: University Hospital, Caen

location_country: Germany Italy Poland Norway Japan Brazil Netherlands Belgium Czechia France

location_facility: Klinik für Anästhesiologie und Intensivmedizin

location_facility: Osaka city University Graduate school of Medecine

location_facility: Universidade Federal de São Paulo (UNIFESP)

location_facility: CUB Erasm, Université libre de Bruxelles


title: A Randomized, Double-blind, Placebo-controlled, Multinational, Multicenter Study With Open-label Treatment Extension to Assess the Effect of MIN-102 (IMP) on the Progression of Adrenomyeloneuropathy in Male Patients With X-linked Adrenoleukodystrophy

acronym: Advance

summary: This is a Phase II/III, randomized, double-blind, placebo-controlled, multicenter, two parallel-group study in male patients with the AMN phenotype of X-linked adrenoleukodystrophy (X-ALD) to assess the efficacy and safety of MIN-102 treatment. Study sites will consist of specialist referral centers experienced in the management of adrenoleukodystrophy (ALD).

lead_sponsor: Minoryx Therapeutics, S.L.

location_country:
Germany
Italy
Hungary
United Kingdom
Netherlands
United States
Spain
France

location_facility: National hospital for Neurology and Neurosurgery Charles Dent Metabolic Unit)

location_facility: Institute of Genomic Medicine and Rare Disorders

location_facility: Universitat Leipzig Klinik and Poliklinik für Neurologie


summary: This is a single-arm, multi-site, single-dose, Phase 3 study in approximately 18 participants less than or equal to (<=) 50 years of age with transfusion-dependent β-thalassemia (TDT), who have a β0/β0, β0/IVS-I-110, or IVS-I-110/IVS-I-110 genotype. The study will evaluate the efficacy and safety of autologous hematopoietic stem cell transplantation (HSCT) using LentiGlobin BB305 Drug Product.

location_facility: General Hospital of Thessaloniki 'G.Papanikolaou'

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

location_facility: UCSF Benioff Children's Hospital Oakland

title: A Phase 3 Single Arm Study Evaluating the Efficacy and Safety of Gene Therapy in Subjects With Transfusion-dependent β-Thalassemia by Transplantation of Autologous CD34+ Stem Cells Transduced Ex Vivo With a Lentiviral βA-T87Q-Globin Vector in Subjects ≤50 Years of Age

location_country:
Germany
Italy
United Kingdom
United States
Greece
France


lead_sponsor: H. Lundbeck A/S

summary: Evaluation of the long-term safety and tolerability of vortioxetine in child and adolescent patients with a Diagnostic and Statistical Manual of Mental Disorders, 5th edition (DSM-5™) diagnosis of MDD

location_facility: Medicorehabilitation Research Center "Phoenix"

location_facility: Rostov State Medical University of the Minzdravsotsrazvitiya of Russia

location_facility: Universita degli Studi di Messina - Facolta di Medicina e Chirurgia

location_facility: Clinical Center of Vojvodina - Clinic of Psychiatry

location_facility: Samodzielny Publiczny Zaklad Opieki Zdrowotnej (SPZOZ) - Centrum Neuropsychiatrii "NEUROMED"

location_facility: SPECTRUM Centrum Neurologii i Psychiatrii

location_facility: Rheinhessen-Fachklinik Mainz, Kinder und Jugendpsychiatri

location_facility: Prywatne Gabinety Lekarskie ¿Promedicus¿ Anna Agnieszka Tomczak

location_facility: Unidad de Salud Mental Infanto-Juvenil (USMI-J) Edificio de Consultas Externas. Hospital MarAtimo

location_facility: Clinic of Neurology and Psychiatry for Children and Adolescents

location_facility: Lipetsk Regional Psychoneurological Hospital

location_facility: Przychodnia Syntonia Poradnia Zdrowia Psychicznego

location_facility: Bekes Megyei Kozponti Korhaz Pandy Kalman Tagkorhaza

location_facility: Guz "Saratov Regional Psychiatric Hospital St. Sofii"

location_facility: Hospital General Universitario Gregorio Maranon

location_facility: Specialized Clinical Psychiatric Hospital 1 of the Ministry of Health

location_facility: Centrum Badan Klinicznych PI-House Sp. z o.o.

location_facility: State Budgetary Healthcare Institution of Sverdlovsk Region

location_facility: Nizhny Novgorod Region State Institution Of Healthcare

location_facility: Scientific Institute Fondazione Stella Maris

location_facility: Filip Rybakowski Specjalistyczna Praktyka Lekarska

location_facility: Marienthal Center of Psychiatry and Psychology

title: Long-term, Open-label, Flexible-dose, Continuation Extension Study With Vortioxetine in Child and Adolescent Patients With Major Depressive Disorder (MDD) From 7 to 17 Years of Age

location_country:
Germany
Italy
Poland
Hungary
United Kingdom
Latvia
Spain
South Africa
Bulgaria
Estonia
Serbia
Russian Federation
France


title: Paediatric Hepatic International Tumour Trial

acronym: PHITT

summary:
The PHITT trial is an over-arching study for patients with Hepatoblastoma (HB) and Hepatocellular Carcinoma (HCC). This trial will use a risk-adapted approach to the treatment of children diagnosed with HB.

Children with HCC will be included as a separate cohort.

lead_sponsor: University of Birmingham

collaborators: Cliniques universitaires Saint-Luc- Université Catholique de Louvain

collaborators: Princess Maxima Center for Pediatric Oncology

collaborators: Gothia Forum - Center for Clinical Trial

collaborators: Bambino Gesù Hospital and Research Institute

collaborators: Newcastle University Centre for Cancer, Newcastle

sponsor_collaborators: Ludwig-Maximilians - University of Munich

location_country:
Germany
Ireland
Poland
Israel
Norway
United Kingdom
Netherlands
Austria
Belgium
Switzerland
Spain
Czechia
France

location_facility: Noah's Ark Children's Hospital for Wales

location_facility: Royal Belfast Hospital for Sick Children


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: Germany France Denmark Belgium Sweden

location_facility: Swedish Orphan Biovitrum Investigational Site

location_facility: Swedish Orphan Biovitrum Investigational site


title: A Randomised, Double-blind, Parallel Group, Equivalence, Multicentre Phase III Trial to Compare the Efficacy, Safety and Pharmacokinetics of HD201 to Herceptin® in Patients With HER2+ Early Breast Cancer

acronym: TROIKA

summary: In the TROIKA study, the proposed biosimilar HD201 will be compared to its reference product Herceptin®. The aim of the study is to demonstrate equivalence of HD201 and Herceptin® in terms of efficacy, safety and pharmacokinetics.

ReferenceCitation: Pivot X, Georgievich MA, Shamrai V, Dzagnidze G, Soo Hoo HF, Kaewkangsadan V, Petrelli F, Villanueva C, Nikolaevich LO, Hii J, Kim J, Pradhan S, Jaison L, Feyaerts P, Kaufman L, Derde MP, Bonamy GMC, Deforce F, Cox DG. Efficacy of HD201 vs Referent Trastuzumab in Patients With ERBB2-Positive Breast Cancer Treated in the Neoadjuvant Setting: A Multicenter Phase 3 Randomized Clinical Trial. JAMA Oncol. 2022 May 1;8(5):698-705. doi: 10.1001/jamaoncol.2021.8171. Erratum In: JAMA Oncol. 2022 May 1;8(5):784.

oa_colors: green hybrid

license: cc-by-nc-nd

license_normalized: cc-by-nc-nd

repositories_concat: PubMed Central

unpaywall_oa_status: hybrid

publisher_dissemination: American Medical Association

lead_sponsor: Prestige Biopharma Limited

location_country:
Belarus
Italy
Poland
Hungary
Estonia
Spain
Thailand
Malaysia
Ukraine
Bulgaria
Georgia
Russian Federation
France

location_facility: GI "V. T. Zaycev Institute General and Urgent Surgery of NAMS of Ukraine "


title: Randomized Study of IN.PACT 014 Paclitaxel-Coated Percutaneous Transluminal Angioplasty Balloon Catheter vs. Standard Percutaneous Transluminal Angioplasty for the Treatment of Chronic Total Occlusions in the Infrapopliteal Arteries

summary: To assess the safety and efficacy of the paclitaxel drug-eluting balloon IN.PACT 014 versus conventional percutaneous transluminal angioplasty (PTA) for the treatment of patients with chronic total occlusions in the infrapopliteal arteries.

ReferenceCitation: Liistro F, Weinberg I, Almonacid Popma A, Shishehbor MH, Deckers S, Micari A. Paclitaxel-coated balloons versus percutaneous transluminal angioplasty for infrapopliteal chronic total occlusions: the IN.PACT BTK randomised trial. EuroIntervention. 2022 Apr 1;17(17):e1445-e1454. doi: 10.4244/EIJ-D-21-00444.

oa_host_type: repository

unpaywall_oa_status: green

publisher_dissemination: Europa Digital & Publishing

lead_sponsor: Medtronic Endovascular

location_country: Italy Switzerland Belgium Greece France

location_facility: Hopital Guillaume et Rene Laennec - Centre Hospitalier Universitaire


title: A Phase I, Open-label, Multi-center Dose Escalation Study of FAZ053 as Single Agent and in Combination With PDR001 in Adult Patients With Advanced Malignancies

summary:
The purpose of this "first-in-human" study of FAZ053 is to characterize the safety, tolerability, pharmacokinetics (PK), pharmacodynamics (PD) and antitumor activity of FAZ053 administered Intravenously (i.v.)as a single agent or in combination with PDR001 in adult patients with advanced solid tumors.

By blocking the interaction between Programmed Death Ligand-1 (PD-L1) and its receptors, Programmed Death-1 (PD-1) and B7.1, FAZ053 inhibits the PD-L1 immune checkpoint, resulting in activation of an antitumor immune response by activating effector T-cells and inhibiting regulatory T-cells.

This study has been designed as a Phase I, open-label, multi-center study with a dose escalation part of FAZ053 as single agent and in combination with PDR001, followed by a dose expansion part of FAZ053 as single agent.

FAZ053 will initially be dosed every three weeks. A less frequent dosing regimen such as every 6 weeks may be evaluated in parallel.

A patient may continue treatment with FAZ053 single agent or in combination with PDR001 until the patient experiences unacceptable toxicity, confirmed disease progression per immune related Response Criteria and/or treatment is discontinued at the discretion of the investigator or the patient.

lead_sponsor: Novartis Pharmaceuticals

location_country:
France
Italy
Singapore
Japan
United States
Spain
Taiwan
Canada
Israel

location_facility: Memorial Sloan Kettering Cancer Center .


summary: This is a single-arm, multi-site, single-dose, Phase 3 study in 23 participants less than or equal to (<=) 50 years of age with transfusion-dependent β-thalassemia (TDT), also known as β-thalassemia major, who do not have a β0 mutation at both alleles of the hemoglobin β (HBB) gene. The study will evaluate the efficacy and safety of autologous hematopoietic stem cell transplantation (HSCT) using LentiGlobin BB305 Drug Product.

ReferenceCitation: Locatelli F, Thompson AA, Kwiatkowski JL, Porter JB, Thrasher AJ, Hongeng S, Sauer MG, Thuret I, Lal A, Algeri M, Schneiderman J, Olson TS, Carpenter B, Amrolia PJ, Anurathapan U, Schambach A, Chabannon C, Schmidt M, Labik I, Elliot H, Guo R, Asmal M, Colvin RA, Walters MC. Betibeglogene Autotemcel Gene Therapy for Non-beta0/beta0 Genotype beta-Thalassemia. N Engl J Med. 2022 Feb 3;386(5):415-427. doi: 10.1056/NEJMoa2113206. Epub 2021 Dec 11.

oa_colors: green other

repositories_concat: UCL Discovery

repositories_institution: University College London - UCL Discovery

ReferenceCitation: Hamed EM, Meabed MH, Aly UF, Hussein RRS. Recent Progress in Gene Therapy and Other Targeted Therapeutic Approaches for Beta Thalassemia. Curr Drug Targets. 2019;20(16):1603-1623. doi: 10.2174/1389450120666190726155733.

oa_host_type: closed

publisher_dissemination: Bentham Science Publishers Ltd.

title: A Phase 3 Single Arm Study Evaluating the Efficacy and Safety of Gene Therapy in Subjects With Transfusion-dependent β-Thalassemia, Who do Not Have a β0/β0 Genotype, by Transplantation of Autologous CD34+ Stem Cells Transduced Ex Vivo With a Lentiviral βA-T87Q-Globin Vector in Subjects ≤50 Years of Age

location_country:
Germany
Italy
United Kingdom
United States
Thailand
France


title: Safety and Efficacy Study Comparing a New hMG Formulation (hMG-IBSA) to a Reference Product (Menopur®) in Patients Undergoing Ovarian Stimulation for in Vitro Fertilisation (IVF)

summary: The purpose of the non-inferiority study is to evaluate the clinical efficacy and the safety of two different subcutaneous hMG preparations when administered to patients undergoing controlled ovarian stimulation for IVF.

lead_sponsor: IBSA Institut Biochimique SA

location_country:
Hungary
United Kingdom
Denmark
Switzerland
France

location_facility: Groupe Hospitalier Cochin - Saint Vincent de Paul

location_facility: First Dept. Obstetric and Gynaecology, Semmelweiss University


title: Short to Mid Term Follow-up of Patient Implanted With the HINTEGRA® Total Ankle Prosthesis

summary:
The Hintegra® Total Ankle Prosthesis is a non constrained, three-component prosthesis that consists of a flat tibial component, an anatomically-shaped talar component, and a high-density polyethylene inlay. The Hintegra ankle was specifically developed as an attempt to address the needs of minimal bone resection, extended bone support, proper ligament balancing, and minimal contact stressed within and around the prosthesis.

The objective of this study is to evaluate the safety and efficacy of the HINTEGRA® Total Ankle prosthesis at short and mid term follow up.

lead_sponsor: Integra LifeSciences Services

location_country:
Germany
Austria
Korea, Republic of
Spain
Canada
France

location_facility:
AukammKlinik
CHU d'Amiens - Hopital Nord
St. Michael's Hospital
Hopital San Rafael
Severance Hospital - Yonsei University
Medical University of Innsbruck


title: A Phase I/II Pharmacokinetic Study of Intravenous and Oral Forodesine in Children With Relapsed or Refractory T-cell or B-cell Precursor Acute Lymphoblastic Leukaemia or T-cell Non-Hodgkin's Lymphoma.

summary: The purpose of this study is to evaluate the pharmacokinetics, pharmacodynamics and safety of different doses of intravenous and oral Forodesine in children with relapsed or refractory T-cell or B-cell precursor Acute Lymphoblastic Leukaemia or T-cell Non-Hodgkin's Lymphoma. Preliminary efficacy will also be assessed.

lead_sponsor: Mundipharma Research Limited

collaborators: Innovative Therapies For Children with Cancer Consortium

location_country:
Germany
Italy
United Kingdom
Austria
Czech Republic
France

location_facility:
Charite Universitymedicine
Dr Giovanna Gioriani
Prof Gerard Michel
Sally Kinsey


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.

location_country: France Italy Poland Belgium Sweden


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: Norway Denmark Sweden Belgium Spain France


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.

lead_sponsor: University of Milan

sponsor_collaborators:
University of Milan
Baxter Healthcare Corporation
Pr Giampiero CAMPANELLI

location_country:
Germany
United Kingdom
Denmark
Belgium
Spain
France

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 KU Leuven

location_country: Germany France Australia Belgium Spain Israel

location_facility: Hopital Avicenne and Paris 13 University


title: EnSite™ HD Grid Catheter Mapping System for Advanced High Density Three-Dimensional Mapping in Non-Paroxysmal Atrial Fibrillation and Atrial Tachycardia

summary: In this clinical investigation, the safety, feasibility and performance of the novel EnSite™ HD Grid Catheter mapping system for advanced high-density three-dimensional mapping will be studied in patients undergoing catheter ablation procedures for the treatment of non-paroxysmal atrial fibrillation (AF) or left atrial tachycardia (AT).

lead_sponsor: Abbott Medical Devices

location_country:
Germany
Italy
Hong Kong
Australia
France

location_facility: Herzzentrum Dresden GmbH Universitatsklinik


title: Testing the Usefulness of Lantus When Initiated Prematurely In Patients With Type 2 Diabetes

summary:
Primary objective:

The primary objective of this trial is:

To evaluate the efficacy of initiating Lantus in combination with oral antidiabetics drugs compared to oral antidiabetic treatment optimised by enhancing hygienic and dietary measures in type 2 diabetics whose blood glucose control is acceptable but not optimal on maximum oral treatment, based on the number of patients achieving a HbA1c value < 7% at the end of treatment.

Secondary objectives:

The secondary objectives of this trial are to compare between the two treatment groups:

The variation in HbA1c between baseline and end of trial.
The frequency of episodes of symptomatic hypoglycaemia (diurnal and nocturnal), severe hypoglycaemia (diurnal and nocturnal) and asymptomatic hypoglycaemia.
Mean blood glucose levels at different times of the day.
The variation in weight and lipid in each group between baseline and end of trial.
The incidence of adverse events.

location_country:
Slovenia
Romania
Croatia
Czech Republic
Russian Federation
France


title: A Multi-centre Study to Define Novel Individualised Dosing Regimens to Maximise Antibiotic Effectiveness for Treatment of Pneumonia in Intensive Care Units

acronym: PNEUDOS

summary:
Pneumonia is the most common infection in intensive care unit (ICU) patients and occurs in 10% of all ICU admissions. Unfortunately, ICU patient outcomes remain poor with a high mortality rate associated with pneumonia despite recent therapeutic advances.

Previous studies of antibiotics used in ICU patients, which includes ceftriaxone, meropenem and piperacillin/tazobactam, have quantified major differences in pharmacokinetics (PK) between ICU and non-ICU patients, with ICU patients displaying a unique spectrum of plasma concentration-time profiles. These PK differences can lead to suboptimal antibiotic concentrations in blood, which have been associated with a reduced likelihood of clinical cure for pneumonia. Furthermore, highlighting the importance of optimised dosing for pneumonia is that multi-drug resistant (MDR) pathogens emerge during antibiotic therapy in approximately half of the ICU patients, frequently emerging from the lung.

Previous work has highlighted how infection site concentrations determine patient outcome. For pneumonia, the infection site is best described as the epithelial lining fluid (ELF) in the lung.

Although optimal antibiotic therapy should be considered a priority for ICU patients with pneumonia to improve the persisting poor outcomes, the dosing regimens that can achieve therapeutic concentrations at the infection site (i.e., ELF) in ICU patients with pneumonia remain unknown.

The PNEUDOS study aims to address this significant knowledge gap by defining novel individualised dosing regimens that can maximise antibiotic efficacy by achieving therapeutic concentrations in the blood and ELF of ICU patients with pneumonia. These dosing regimens can then be validated in future clinical trials.

ReferenceCitation: Abdul-Aziz MH, Alffenaar JC, Bassetti M, Bracht H, Dimopoulos G, Marriott D, Neely MN, Paiva JA, Pea F, Sjovall F, Timsit JF, Udy AA, Wicha SG, Zeitlinger M, De Waele JJ, Roberts JA; Infection Section of European Society of Intensive Care Medicine (ESICM); Pharmacokinetic/pharmacodynamic and Critically Ill Patient Study Groups of European Society of Clinical Microbiology and Infectious Diseases (ESCMID); Infectious Diseases Group of International Association of Therapeutic Drug Monitoring and Clinical Toxicology (IATDMCT); Infections in the ICU and Sepsis Working Group of International Society of Antimicrobial Chemotherapy (ISAC). Antimicrobial therapeutic drug monitoring in critically ill adult patients: a Position Paper. Intensive Care Med. 2020 Jun;46(6):1127-1153. doi: 10.1007/s00134-020-06050-1. Epub 2020 May 7.

lead_sponsor: The University of Queensland

collaborators: Centre Hospitalier Universitaire de Nīmes

location_country:
Hong Kong
Australia
Belgium
Malaysia
France

location_facility: Centre Hospitalier Universitaire de Nimes


title: Phase 1/2a Open Label, Multicenter Clinical Trial of a Novel Small Molecule EBNA1 Inhibitor, VK 2019, in Patients With Epstein Barr Virus Positive Nasopharyngeal Cancer, With Pharmacokinetic and Pharmacodynamic Correlative Studies

summary: VK-2019-001 is a 1/2a trial of the oral EBNA-1 targeting agent VK-2019 in patients with EBV-positive recurrent or metastatic NPC to determine the Maximum Tolerated Dose (MTD) and Recommended Phase 2 Dose (RP2D), as well as to evaluate the PK profile of VK-2019.

ReferenceCitation: Young LS. Epstein-Barr virus at 50-future perspectives. Chin J Cancer. 2014 Nov;33(11):527-8. doi: 10.5732/cjc.014.10208.

ReferenceCitation: Young LS, Dawson CW. Epstein-Barr virus and nasopharyngeal carcinoma. Chin J Cancer. 2014 Dec;33(12):581-90. doi: 10.5732/cjc.014.10197. Epub 2014 Nov 21.

ReferenceCitation: Tsao SW, Tsang CM, Lo KW. Epstein-Barr virus infection and nasopharyngeal carcinoma. Philos Trans R Soc Lond B Biol Sci. 2017 Oct 19;372(1732):20160270. doi: 10.1098/rstb.2016.0270.

lead_sponsor: Cullinan Oncology Inc.

sponsor_collaborators:
Cullinan Oncology Inc.
National Cancer Institute (NCI)

location_country:
Singapore
Hong Kong
United States
China
France

location_facility: Hong Kong University - Queen Mary Hospital

location_facility: The University of Texas - MD Anderson Cancer Center

location_facility: Stanford University, School of Medicine, Stanford Cancer Institute


title: A Randomized, Double-Masked, Sham-Controlled Clinical Trial to Evaluate the Efficacy of a Single Intravitreal Injection of GS010 in Subjects Affected for 6 Months or Less by LHON Due to the G11778A Mutation in the Mitochondrial ND4 Gene

acronym: RESCUE

summary: The goal of this clinical trial is to assess the effectiveness of GS010, a gene therapy, in improving the visual outcome in participants with Leber Hereditary Optic Neuropathy (LHON) due to the G11778A ND4 mitochondrial mutation when vision loss is present for six months or less.

repositories_institution: California Digital Library - eScholarship

repositories_institution: Le Centre pour la Communication Scientifique Directe - HAL - Université de Nantes

repositories_institution: Le Centre pour la Communication Scientifique Directe - HAL - memSIC

ReferenceCitation: Newman NJ, Yu-Wai-Man P, Carelli V, Moster ML, Biousse V, Vignal-Clermont C, Sergott RC, Klopstock T, Sadun AA, Barboni P, DeBusk AA, Girmens JF, Rudolph G, Karanjia R, Taiel M, Blouin L, Smits G, Katz B, Sahel JA; LHON Study Group. Efficacy and Safety of Intravitreal Gene Therapy for Leber Hereditary Optic Neuropathy Treated within 6 Months of Disease Onset. Ophthalmology. 2021 May;128(5):649-660. doi: 10.1016/j.ophtha.2020.12.012. Epub 2021 Jan 12.

evidence: open (via crossref license)

endpoint_id: fhisxppmrqpajgzqkvft

repository_institution: Alma Mater Studiorum Università di Bologna - Archivio istituzionale della ricerca - Alma Mater Studiorum Università di Bologna

repository_institution: DZNE - DZNE Pub

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

repository_institution: University of Cambridge - Apollo

publisher_dissemination: Elsevier

lead_sponsor: GenSight Biologics

location_country:
Germany
Italy
United Kingdom
United States
France

location_facility: Department of Ophthalmology, Emory University School of Medicine

location_facility: Department of Neurology, University of Munich, Friedrich-Baur-Institute

location_facility: Neuro Ophthalmologic Associates, Wills Eye Hospital, Thomas Jefferson University

location_facility: Doheny Eye Center, University of California, Los Angeles

location_facility: IRCCS Istituto delle Scienze Neurologiche di Bologna, UOC Clinica Neurologica, Ospedale Bellaria

location_facility: Centre National Hospitalier d'Ophtalmologie des Quinze-VingtCentre National Hospitalier d'Ophtalmologie des Quinze-Vingt

location_facility: Moorfields Eye Hospital NHS Foundation Trust


lead_sponsor: Genzyme, a Sanofi Company

summary:
Primary Objective:

To assess the long-term safety of GZ/SAR402671 in adult male participants with Fabry disease who previously completed study ACT13739 (NCT02489344).

Secondary Objective:

To assess the long-term effect of GZ/SAR402671 on pharmacodynamic and exploratory efficacy endpoints in adult male participants with Fabry disease who previously completed study ACT13739.

location_facility:
Investigational Site Number 840003
Investigational Site Number 826002
Investigational Site Number 616001
Investigational Site Number 643002
Investigational Site Number 250001
Investigational Site Number 840002
Investigational Site Number 840001

title: An Open-label, Multicenter, Multinational Extension Study of the Long-term Safety, Pharmacodynamics, and Exploratory Efficacy of GZ/SAR402671 in Adult Male Patients Diagnosed With Fabry Disease

location_country:
Poland
United Kingdom
United States
Russian Federation
France


lead_sponsor: Abivax S.A.

summary: This study is a placebo-controlled study aimed at assessing the safety of ABX464 administered at 50 mg and 150 mg o.d. versus placebo in HIV infected patients who are treated with darunavir + ritonavir (DRV/RTV) or darunavir + cobicistat (DRV/COBI).

ReferenceCitation: Rutsaert S, Steens JM, Gineste P, Cole B, Kint S, Barrett PN, Tazi J, Scherrer D, Ehrlich HJ, Vandekerckhove L. Safety, tolerability and impact on viral reservoirs of the addition to antiretroviral therapy of ABX464, an investigational antiviral drug, in individuals living with HIV-1: a Phase IIa randomised controlled study. J Virus Erad. 2019 Jan 1;5(1):10-22.

location_facility: Hospital Universitari Germans Trias i Pujo

location_facility: CHU de Montpellier - Hôpital Gui de Chauliac

title: A Multi-center, Randomized, Double-blind, Placebo-controlled Phase IIa Trial to Compare the Safety of ABX464 Given at a Fixed Dose to Placebo in Fully Controlled HIV Infected Patients Treated With Boosted Protease Inhibitor Treatment (Darunavir/Ritonavir or Darunavir/Cobicistat).

location_country: Spain Belgium France


title: A Randomised Phase IIb Trial of Bevacizumab Added to Temozolomide ± Irinotecan for Children With Refractory/Relapsed Neuroblastoma - BEACON-Neuroblastoma Trial

acronym: BEACON

summary:
The purpose of this study is to investigate whether Bevacizumab (an anti-VEGF monoclonal antibody) added to a backbone chemotherapy regimen (Temozolomide, Irinotecan-Temozolomide or Topotecan-Temozolomide) demonstrates activity in children with relapsed or refractory neuroblastoma. Also, to investigate whether the addition of Irinotecan or Topotecan to Temozolomide increases the activity of chemotherapy.The primary objective of the study is the best response (Complete Response or Partial Response) while trial treatment, within 18 or 24 weeks depending on the arm of the trial the participant is randomised to. Secondary endpoints are assessing the side effects, the length of time before progression (Progression Free Survival) and overall survival (OS).

This trial will address two important questions:

does targeting blood vessel development using bevacizumab, (a monoclonal antibody against the Vascular Endothelial Growth Factor (VEGF)), add to the effect on a tumour when used with existing chemotherapy, compared to the effect of the existing chemotherapy alone (temozolomide)? NOTE- This question has been completed.
does the addition of a second chemotherapy drug (irinotecan or topotecan) increase the effect on a tumour compared to the effect of one alone (temozolomide) NOTE - This question has been completed.
does the addition of dinutuximab beta added to a backbone chemotherapy (temozolomide or temozolomide + topotecan) increase the effect of backbone alone.

Patients aged 1-21 years of age with relapsed or refractory high-risk neuroblastoma are randomised to one of two treatment arms: temozolomide-topotecan (TTo) or dinutuximab beta-temozolomide-topotecan (dBTTo). Temozolomide (T), irinotecan-temozolomide (IT), bevacizumab-T (BT), BIT (bevacizumab-IT), bevacizumab-temozolomide-topotecan (BTTo) and dinutuximab beta-temozolomide (dBT) are now closed to recruitment.

lead_sponsor: University of Birmingham

sponsor_collaborators:
University of Birmingham
Cancer Research UK
Roche Pharma AG
Imagine for Margo
EUSA Pharma, Inc.

location_country:
Ireland
Italy
United Kingdom
Denmark
Netherlands
Austria
Belgium
Switzerland
Spain
France

location_facility: St Anna Children's Hospital and CCRI/Studies and Statistics

location_facility: The Royal Marsden NHS Foundation Trust and Institute of Cancer Research


summary:
This was a multi-center, randomized, double blind (investigator and subject), placebo controlled Phase II study to determine the efficacy and safety of treatment with ribociclib versus placebo in subjects with progressive relapsed, refractory incurable teratoma. Eligible subjects were randomized in a 2:1 ratio to ribociclib or placebo.

After discontinuation of study treatment, patients were followed up for safety, disease progression and overall survival.

location_facility: USC Kenneth Norris Comprehensive Cancer Center Oncology Dept

location_facility: Memorial Sloan Kettering Oncology Department.

title: A Randomized, Blinded, Placebo-controlled, Phase II Trial of LEE011 in Patients With Relapsed, Refractory, Incurable Teratoma With Recent Progression

ipd_sharing_description:
Novartis is committed to sharing with qualified external researchers, access to patient-level data and supporting clinical documents from eligible studies. These requests are reviewed and approved by an independent review panel on the basis of scientific merit. All data provided is anonymized to respect the privacy of patients who have participated in the trial in line with applicable laws and regulations.

This trial data availability is according to the criteria and process described on www.clinicalstudydatarequest.com

location_country:
United States
Spain
Netherlands
France


title: Open-Label, Non Randomized Phase 2 Study With Safety Run-In Evaluating Efficacy and Safety of PQR309 in Patients With Relapsed or Refractory Lymphoma

summary: The main goal of this study is to determine the Maximum Tolerated Dose (MTD) and the Recommended Phase II Dose (RP2D) as well as preliminary antitumor activity of bimiralisib (PQR309) administered orally, as once daily capsules continuously and on intermittent schedule in patients with relapsed or refractory lymphomas.

lead_sponsor: PIQUR Therapeutics AG

collaborators: Weill Medical College of Cornell University

collaborators: Institute for Oncology and Radiology Serbia, Belgrade

collaborators: University Clinical Centre of Republic of Srpska

location_country:
France
United Kingdom
United States
Bosnia and Herzegovina
Serbia
Israel

location_facility: University Clinical Center Republic of Srpska

location_facility: Institute for Oncology and radiology of Serbia


title: A Phase 2 Study to Evaluate the Safety, Pharmacodynamics, Pharmacokinetics, and Exploratory Efficacy of GZ/SAR402671 in Enzyme Replacement Therapy (ERT) Treatment-naïve Adult Male Patients Diagnosed With Fabry Disease

summary:
Primary Objective:

To assess the safety, pharmacokinetics (PK), pharmacodynamics (PD), and exploratory efficacy of GZ/SAR402671 in enzyme replacement therapy treatment-naïve adult male participants diagnosed with Fabry disease.

location_facility:
Investigational Site Number 840003
Investigational Site Number 826002
Investigational Site Number 826003
Investigational Site Number 616001
Investigational Site Number 643002
Investigational Site Number 250001
Investigational Site Number 840002
Investigational Site Number 840001


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.

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

repositories_concat: PubMed Central;dash.harvard.edu

publisher_dissemination: American Diabetes Association

lead_sponsor: Jaeb Center for Health Research

location_country:
United States
Israel
Italy
France

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


title: A Prospective, Randomized, Intra-patient, Comparative, Open, Multi-centre Study to Evaluate the Efficacy of a Single-Use NPWT System on the Prevention of Incision Healing Complications in Patients Undergoing Reduction Mammoplasty

summary: The purpose of this study is to see if the use of a single-use negative pressure wound therapy system (NPWT) will have an effect on the reduction of post-surgical incision healing complications following breast reduction surgery and to assess the medium-term aesthetic appearance and quality of the resultant scar, compared with standard of care dressings.

primary_purpose: Prevention

lead_sponsor: Smith & Nephew, Inc.

location_country:
United States
South Africa
Netherlands
France

location_facility: Northwestern University Feinberg School of Medicine


title: CardioRoot Post-Marketing Surveillance Registry

acronym: CardioRoot

summary: Post-marketing surveillance registry of the CardioRoot graft.

ReferenceCitation: Tasca G, Lindner J, Barandon L, Santavy P, Antona C, Burkert J, Gamba A. Aortic root surgery with the CARDIOROOT vascular graft: results of a prospective multicenter post-market surveillance study. J Cardiothorac Surg. 2019 May 21;14(1):94. doi: 10.1186/s13019-019-0914-y.

repository_institution: Universita Degli Studi Di Milano - Archivio Istituzionale della Ricerca

repositories_concat: PubMed Central;air.unimi.it

lead_sponsor: Maquet Cardiovascular

location_country: Czechia Italy France

location_facility: Centre Hospitalier Universitaire (CHU) de Bordeaux


title: Evaluation of the Safety and Effectiveness of Sepraspray™ in Limiting the Formation of Postoperative Adhesions to Serosal Tissue (Abdominal Surgery)

summary: This study will examine the safety and efficacy of Sepraspray in the following model of abdominal surgery: total proctocolectomy and pelvic pouch with diverting ileostomy via laparotomy to treat ulcerative colitis or familial polyposis. Adhesion formation will be evaluated laparoscopically at ileostomy take down. NOTE: A decision was made to terminate this study in June 2008 due to low enrollment.

location_country:
Germany
United Kingdom
Denmark
Czech Republic
France


title: An Eight-week, Double-blind, Placebo-controlled Study to Evaluate the Efficacy, Safety, and Tolerability of Saredutant 100 mg Once Daily in Combination With Escitalopram 10 mg Once Daily in Patients With Major Depressive Disorder

summary: The main purpose of this study is to evaluate the efficacy of saredutant 100mg once daily in combination with escitalopram 10mg once daily compared to saredutant placebo in combination with escitalopram 10mg once daily in patients with major depressive disorder. This study also includes a double-placebo group (saredutant placebo in combination with escitalopram placebo).

location_country:
Finland
Argentina
Sweden
Mexico
United States
France


title: A Randomized Phase III Multicenter Trial of Neoadjuvant Docetaxel (Taxotere®) Plus Cisplatin and 5-Fluorouracil (TPF) Versus Neoadjuvant Cisplatin Plus 5-Fluorouracil Followed by Concomitant Chemoradiotherapy to Improve the Overall Survival and Progression Free Survival in Patients With Locally Advanced Squamous Cell Carcinoma of the Head and Neck

summary:
1.To compare overall survival after treatment with the test tri-therapy (TPF: docetaxel plus cisplatin and 5FU) or the control treatment (PF: cisplatin plus 5-FU) followed by chemoradiotherapy in patients with locally advanced SCCHN.
2.The main secondary endpoint is progression free survival (PFS). The other secondary endpoints are to evaluate and compare improvement of local symptoms; time-to-treatment failure; quality of life; clinical complete response rate (CR and CR/PR); toxicity and to evaluate the relationship of tumor markers and response to therapy.

ReferenceCitation: Posner MR, Hershock DM, Blajman CR, Mickiewicz E, Winquist E, Gorbounova V, Tjulandin S, Shin DM, Cullen K, Ervin TJ, Murphy BA, Raez LE, Cohen RB, Spaulding M, Tishler RB, Roth B, Viroglio Rdel C, Venkatesan V, Romanov I, Agarwala S, Harter KW, Dugan M, Cmelak A, Markoe AM, Read PW, Steinbrenner L, Colevas AD, Norris CM Jr, Haddad RI; TAX 324 Study Group. Cisplatin and fluorouracil alone or with docetaxel in head and neck cancer. N Engl J Med. 2007 Oct 25;357(17):1705-15. doi: 10.1056/NEJMoa070956.

ReferenceCitation: Lorch JH, Goloubeva O, Haddad RI, Cullen K, Sarlis N, Tishler R, Tan M, Fasciano J, Sammartino DE, Posner MR; TAX 324 Study Group. Induction chemotherapy with cisplatin and fluorouracil alone or in combination with docetaxel in locally advanced squamous-cell cancer of the head and neck: long-term results of the TAX 324 randomised phase 3 trial. Lancet Oncol. 2011 Feb;12(2):153-9. doi: 10.1016/S1470-2045(10)70279-5. Epub 2011 Jan 11.

location_country:
Portugal
Argentina
United States
Canada
Russian Federation
France


title: Dose-Ranging Pharmacodynamic Assessment of Platelet Aggregation Inhibition With Clopidogrel in Children of Blalock-Taussig Shunt Age Categories (Neonates and Infants/Toddlers)

summary: PICOLO is a double blind placebo controlled phase II dose ranging, dose escalating study in patients of Blalock-Taussig age categories (neonates and infants/toddlers), to determine the dose providing inhibition of platelet aggregation similar to adults.

ReferenceCitation: Li JS, Yow E, Berezny KY, Bokesch PM, Takahashi M, Graham TP Jr, Sanders SP, Sidi D, Bonnet D, Ewert P, Jennings LK, Michelson AD; PICOLO Investigators. Dosing of clopidogrel for platelet inhibition in infants and young children: primary results of the Platelet Inhibition in Children On cLOpidogrel (PICOLO) trial. Circulation. 2008 Jan 29;117(4):553-9. doi: 10.1161/CIRCULATIONAHA.107.715821. Epub 2008 Jan 14.

ReferenceCitation: Pasquali SK, Yow E, Jennings LK, Li JS. Platelet activity associated with concomitant use of clopidogrel and proton pump inhibitors in children with cardiovascular disease. Congenit Heart Dis. 2010 Nov-Dec;5(6):552-5. doi: 10.1111/j.1747-0803.2010.00461.x.

sponsor_collaborators:
Sanofi
Bristol-Myers Squibb

location_country:
Germany
Italy
Belgium
United States
Canada
France


title: An Open-Label, Multicenter, Multinational, Study of the Safety, Efficacy, Pharmacokinetics, and Pharmacodynamics of rhGAA Treatment in Patients Greater Than 6 Months and Less Than or Equal to 36 Months Old With Infantile-Onset GSD-II

summary: Glycogen Storage Disease Type II ("GSD-II"; also known as Pompe disease) is caused by a deficiency of a critical enzyme in the body called acid alpha-glucosidase (GAA). Normally, GAA is used by the body's cells to break down glycogen (a stored form of sugar) within specialized structures called lysosomes. In patients with GSD-II, an excessive amount of glycogen accumulates and is stored in various tissues, especially heart and skeletal muscle, which prevents their normal function. This study is being conducted to evaluate the safety and effectiveness of recombinant human acid alpha-glucosidase (rhGAA) as a potential enzyme replacement therapy for GSD-II. Patients diagnosed with infantile-onset GSD-II who are greater than 6 months old, but less than or equal to 36 months old will be studied.

ReferenceCitation: Kishnani PS, Goldenberg PC, DeArmey SL, Heller J, Benjamin D, Young S, Bali D, Smith SA, Li JS, Mandel H, Koeberl D, Rosenberg A, Chen YT. Cross-reactive immunologic material status affects treatment outcomes in Pompe disease infants. Mol Genet Metab. 2010 Jan;99(1):26-33. doi: 10.1016/j.ymgme.2009.08.003.

location_country:
United States
Israel
United Kingdom
France

location_facility: University of Florida College of Medicine


title: Long-term Follow-up of a Multicentre, Randomized, Double-blind, Placebo-controlled Trial (AN004T) Assessing the Efficacy and Tolerability of 2 Dosing Regimens of AllerT in Adult Subjects Allergic to Birch Pollen

summary: Study aiming to evaluate the efficacy of a 2-month pre-seasonal treatment with an AllerT 50 µg or 100 µg maintenance dose administered in previous study AN004T in reducing symptoms of allergic rhinoconjunctivitis during the 2nd following birch pollen season.

lead_sponsor: Anergis

location_country: Lithuania Poland Denmark Sweden Switzerland Latvia France

location_facility: Antakalnio affiliation of the Vilnius City Allergy Center

location_facility: Centre Hospitalier Universitaire Vaudois

location_facility: Center of examination and treatment of allergic diseases

location_facility: Viktorija Vevere private practice of Allergology

location_facility: Silesian Piasts University of Medicine in Wrocław

location_facility: SPZOZ Uniwersytecki Szpital Kliniczny - Allergology


title: Natural History and Outcomes in X-Linked Hypohidrotic Ectodermal Dysplasia

summary: The proposed natural history study will enroll male and female patients, ages 36 months and younger, who have a diagnosis of XLHED based on genetic testing and who have not received an investigational study drug. The study protocol will include collection of all relevant medical history and documentation of clinical outcomes using age-appropriate, minimally invasive technologies. Data will be collected both retrospectively, back to pregnancy assessments that may be available, and prospectively through age 5 yrs.

lead_sponsor: Edimer Pharmaceuticals

location_facility: Washington University School of Medicine

location_facility: Azienda Ospedaliera-Polo Universitario "Luigi Sacco"


title: Repetitive Transcranial Magnetic Stimulation of the Precentral-gyrus in the Relief of Fibromyalgia Pain: an International Multicenter Controlled Adaptative Trial

acronym: TMSFFI

summary: There are currently several points concerning rTMS for pain relief that have a huge impact in the practical application of rTMS in clinical practice, but which have not been approached by previous studies. Also, an even more important issue is the frequency of the sessions in each treatment protocol. The idea of performing stimulation in chronic pain patients on a consecutive daily basis for long-term treatments seem unrealistic and potentially costly. However, it has been reported in animals, healthy subjects and FM patients that the analgesic effects of rTMS can outlast the stimulation session for several minutes and even days. This has led to the proposal that pain treatment with rTMS could be performed in consecutive daily induction sessions, followed by spaced (weekly, fortnightly and even monthly) maintenance sessions. This approach is similar to what is performed routinely in patients with major depression treated by rTMS to the dorsolateral prefrontal cortex, and has successfully been tested in FM patients in a single- center study. Should this approach prove effective in larger prospective multicentric studies, rTMS could enter the armamentarium of non-invasive, non- pharmacological, low-adverse event therapeutic options to relieve fibromyalgia- related pain and associate symptoms. Hence, our aim is to conduct a multicentric international clinical trial on the influence of rTMS in the control of pain in a consecutive adaptive trial design where conventional rTMS (10Hz) and patterned rTMS (theta-burst) will be studied. Stimulations will be performed in a clinical practice-friendly approach, where daily maintenance sessions will be followed by weekly, and fortnightly stimulation sessions. There have been very scarce adaptive trials in the pain and non-invasive stimulation fields. This methodological approach allows for the use of lower number of patients in consecutive trials and, among other qualities, it allows for the imputation of data from the first trial into the second one, thus decreasing the duration of the studies and the number of participants.

lead_sponsor: University of Sao Paulo

collaborators: Fundação de Amparo à Pesquisa do Estado de São Paulo

location_country: Japan Brazil France

location_facility: Department of Neuromodulation Osaka University

location_facility: Centre d'Evaluation et de Traitement de la Douleur Hôpital Ambroise Paré

location_facility: Hospital das Clínicas da Faculdade de Medicina da Universidade de São Paulo (HC/FMUSP)

location_facility: Ambulatório de Fisioterapia do Hospital das Clínicas da Faculdade de Medicina da Universidade de São Paulo


acronym: WINGS

lead_sponsor: Phoenicis Therapeutics

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.

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.

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

version: submittedVersion

publisher_dissemination: Informa

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

location_country:
United States
Spain
France


title: A Long-Term Study to Assess the Ongoing Safety and Efficacy of Olipudase Alfa in Patients With Acid Sphingomyelinase Deficiency

summary:
The primary objective of this study is to obtain data regarding the safety of olipudase alfa in patients with acid sphingomyelinase deficiency (ASMD) who are exposed to long term treatment with olipudase alfa.

The secondary objectives of this study are to obtain data regarding the efficacy of olipudase alfa and to characterize olipudase alfa pharmacodynamics (PD) and pharmacokinetics (PK) following long-term administration.

ReferenceCitation: Thurberg BL, Diaz GA, Lachmann RH, Schiano T, Wasserstein MP, Ji AJ, Zaher A, Peterschmitt MJ. Long-term efficacy of olipudase alfa in adults with acid sphingomyelinase deficiency (ASMD): Further clearance of hepatic sphingomyelin is associated with additional improvements in pro- and anti-atherogenic lipid profiles after 42 months of treatment. Mol Genet Metab. 2020 Sep-Oct;131(1-2):245-252. doi: 10.1016/j.ymgme.2020.06.010. Epub 2020 Jun 24.

ReferenceCitation: Wasserstein MP, Diaz GA, Lachmann RH, Jouvin MH, Nandy I, Ji AJ, Puga AC. Olipudase alfa for treatment of acid sphingomyelinase deficiency (ASMD): safety and efficacy in adults treated for 30 months. J Inherit Metab Dis. 2018 Sep;41(5):829-838. doi: 10.1007/s10545-017-0123-6. Epub 2018 Jan 5.

publisher_dissemination: Wiley

ipd_sharing_description: Qualified researchers may request access to patient 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. Patient 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://vivli.org

location_country:
Germany
Italy
United Kingdom
Brazil
Belgium
United States
France

location_facility:
Investigational Site Number 826002
Investigational Site Number 276002
Investigational Site Number 826001
Investigational Site Number 250002
Investigational Site Number 380002
Investigational Site Number 076001
Investigational Site Number 380001
Investigational Site Number 056001
Investigational Site Number 840001


acronym: ASPIRO

lead_sponsor: Astellas Gene Therapies

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 10 years

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

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

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

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

ipd_sharing_description: Access to anonymized individual participant level data will not be provided for this trial as it meets one or more of the exceptions described on www.clinicalstudydatarequest.com under "Sponsor Specific Details for Astellas."

location_country:
United States
Canada
Germany
France


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.

lead_sponsor: Eli Lilly and Company

location_country:
United States
Spain
Australia
France

location_facility: University of Texas MD Anderson Cancer Center

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 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.

sponsor_collaborators:
Daiichi Sankyo, Inc.
Plexxikon

location_country:
United States
Germany
France

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