acronym: NETTER-P

lead_sponsor: Advanced Accelerator Applications

summary: This is a multicenter, open-label, single-arm study to evaluate the safety and dosimetry of Lutathera in adolescent patients 12 to <18 years old with somatostatin receptor positive GEP-NETs and PPGLs. The study will enroll at least 8 patients in the GEP-NET cohort and as many adolescents with PPGL as possible in the exploratory PPGL cohort.

location_facility: Maria Sklodowska-Curie National Research Institute of Oncology

status: Active, not recruiting

title: A Multicenter Open-label Study to Evaluate Safety and Dosimetry of Lutathera in Adolescent Patients With Somatostatin Receptor Positive Gastroenteropancreatic Neuroendocrine (GEP-NET) Tumors, Pheochromocytoma and Paragangliomas (PPGL)

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:
Poland
United Kingdom
United States
Spain
France


study_start_date_type: Actual

intervention_type: Drug

lead_sponsor: BioMarin Pharmaceutical

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

location_facility: Great Ormond Street Hospital for Children

location_facility: Institute of Genetic Medicine International Centre for Life

location_facility: Policlinico Universitario Agostino Gemelli

status: Terminated

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

primary_purpose: Treatment

source: euctr

enrollment_type: Actual

design_allocation: Non-Randomized

study_completion_date_type: Actual

location_country:
Italy
United Kingdom
Netherlands
Belgium
France

study_type: Interventional

lead_sponsor_type: industriel

status_simplified: Unknown


title: SUrgical Versus PERcutaneous AXillary Artery International Registry A Physician-initiated, International, Multicentric, Retrospective, Observational, Voluntary Registry - SUPER AXA International Registry

summary: The purpose of the Study is to compare the outcomes of the surgical and the percutaneous approach to the upper extremity access (axillary or brachial artery) during endovascular procedures on the aortic valve, the aorta, and its side branches.

status: Completed

study_type: Observational

time_perspective: Retrospective

ReferenceType: background

ReferenceCitation: Mirza AK, Oderich GS, Sandri GA, Tenorio ER, Davila VJ, Karkkainen JM, Hofer J, Cha S. Outcomes of upper extremity access during fenestrated-branched endovascular aortic repair. J Vasc Surg. 2019 Mar;69(3):635-643. doi: 10.1016/j.jvs.2018.05.214. Epub 2018 Oct 24.

ReferenceCitation: Branzan D, Steiner S, Haensig M, Scheinert D, Schmidt A. Percutaneous Axillary Artery Access for Endovascular Treatment of Complex Thoraco-abdominal Aortic Aneurysms. Eur J Vasc Endovasc Surg. 2019 Sep;58(3):344-349. doi: 10.1016/j.ejvs.2019.01.011. Epub 2019 Jul 30.

ReferenceCitation: Malgor RD, Marques de Marino P, Verhoeven E, Katsargyris A. A systematic review of outcomes of upper extremity access for fenestrated and branched endovascular aortic repair. J Vasc Surg. 2020 May;71(5):1763-1770.e2. doi: 10.1016/j.jvs.2019.09.028. Epub 2019 Nov 15.

ReferenceCitation: Deuschl F, Schofer N, Seiffert M, Hakmi S, Mizote I, Schaefer A, Schirmer J, Reichenspurner H, Blankenberg S, Conradi L, Schafer U. Direct percutaneous transaxillary implantation of a novel self-expandable transcatheter heart valve for aortic stenosis. Catheter Cardiovasc Interv. 2017 Dec 1;90(7):1167-1174. doi: 10.1002/ccd.26986. Epub 2017 Mar 15.

ReferenceCitation: Schafer U, Deuschl F, Schofer N, Frerker C, Schmidt T, Kuck KH, Kreidel F, Schirmer J, Mizote I, Reichenspurner H, Blankenberg S, Treede H, Conradi L. Safety and efficacy of the percutaneous transaxillary access for transcatheter aortic valve implantation using various transcatheter heart valves in 100 consecutive patients. Int J Cardiol. 2017 Apr 1;232:247-254. doi: 10.1016/j.ijcard.2017.01.010. Epub 2017 Jan 7.

ReferenceCitation: Frohlich GM, Baxter PD, Malkin CJ, Scott DJ, Moat NE, Hildick-Smith D, Cunningham D, MacCarthy PA, Trivedi U, de Belder MA, Ludman PF, Blackman DJ; National Institute for Cardiovascular Outcomes Research. Comparative survival after transapical, direct aortic, and subclavian transcatheter aortic valve implantation (data from the UK TAVI registry). Am J Cardiol. 2015 Nov 15;116(10):1555-9. doi: 10.1016/j.amjcard.2015.08.035. Epub 2015 Sep 3.

ReferenceCitation: Arnett DM, Lee JC, Harms MA, Kearney KE, Ramos M, Smith BM, Anderson EC, Tayal R, McCabe JM. Caliber and fitness of the axillary artery as a conduit for large-bore cardiovascular procedures. Catheter Cardiovasc Interv. 2018 Jan 1;91(1):150-156. doi: 10.1002/ccd.27416. Epub 2017 Nov 11.

ReferenceCitation: Kappetein AP, Head SJ, Genereux P, Piazza N, van Mieghem NM, Blackstone EH, Brott TG, Cohen DJ, Cutlip DE, van Es GA, Hahn RT, Kirtane AJ, Krucoff MW, Kodali S, Mack MJ, Mehran R, Rodes-Cabau J, Vranckx P, Webb JG, Windecker S, Serruys PW, Leon MB; Valve Academic Research Consortium (VARC)-2. Updated standardized endpoint definitions for transcatheter aortic valve implantation: the Valve Academic Research Consortium-2 consensus document (VARC-2). Eur J Cardiothorac Surg. 2012 Nov;42(5):S45-60. doi: 10.1093/ejcts/ezs533. Epub 2012 Oct 1.

ReferenceCitation: Bertoglio L, Grandi A, Melloni A, Kahlberg A, Melissano G, Chiesa R. Percutaneous AXillary Artery (PAXA) Access at the First Segment During Fenestrated and Branched Endovascular Aortic Procedures. Eur J Vasc Endovasc Surg. 2020 Jun;59(6):929-938. doi: 10.1016/j.ejvs.2020.01.027. Epub 2020 Feb 20.

ReferenceCitation: Schaefer A, Schirmer J, Schofer N, Schneeberger Y, Deuschl F, Blankenberg S, Reichenspurner H, Conradi L, Schafer U. Transaxillary transcatheter aortic valve implantation utilizing a novel vascular closure device with resorbable collagen material: a feasibility study. Clin Res Cardiol. 2019 Jul;108(7):779-786. doi: 10.1007/s00392-018-1407-z. Epub 2018 Dec 17.

ReferenceCitation: De Palma R, Ruck A, Settergren M, Saleh N. Percutaneous axillary arteriotomy closure during transcatheter aortic valve replacement using the MANTA device. Catheter Cardiovasc Interv. 2018 Nov 1;92(5):998-1001. doi: 10.1002/ccd.27383. Epub 2017 Oct 25.

ReferenceType: result

ReferenceCitation: Puippe GD, Kobe A, Rancic Z, Pfiffner R, Lachat M, Pfammatter T. Safety of percutaneous axillary artery access with a suture-mediated closing device for parallel endograft aortic procedures - a retrospective pilot study. Vasa. 2018 Jun;47(4):311-317. doi: 10.1024/0301-1526/a000702. Epub 2018 Mar 27.

oa_host_type: closed

repositories_concat: closed

publisher_dissemination: Hogrefe Publishing Group

ipd_sharing: Undecided

lead_sponsor: IRCCS San Raffaele

location_country:
Germany
Italy
United Kingdom
Sweden
Switzerland
Austria
Belgium
United States
Spain
France

location_facility: University Heart and Vascular Center Hamburg

location_facility: Hospital General Universitario Gregorio Marañón

location_facility: Ascension St John Hospital and Medical Center

location_facility: Service de Chirurgie Thoracique et Cardiovasculaire

location_facility: Nuffield Dept. of Clinical Neurosciences, University of Oxford

intervention_type: Other

status_simplified: Completed


title: A Postmarketing, Multicenter, Longitudinal, Prospective, Pharmacokinetic, Phase 1B Study in Pregnant Women With Chronic Inflammatory Diseases Treated With Cimzia (Certolizumab Pegol)

acronym: CHERISH

summary: The purpose of the study is to assess systemic certolizumab pegol (CZP) exposure, the formation of anti-CZP antibodies and safety of CZP across the course of pregnancy in study participants with chronic inflammatory diseases.

primary_purpose: Basic Science

ipd_sharing: No

ipd_sharing_description: Due to the small sample size in this trial, IPD cannot be adequately anonymized i.e., there is a reasonable likelihood that individual participants could be re-identified. For this reason, data from this trial cannot be shared.

lead_sponsor: UCB Biopharma SRL

location_country:
Germany
Netherlands
Switzerland
United States
Spain
France


title: User Evaluation New Ambient Experience Content

summary:
The study aims to investigate the effect of Philips Ambient Experience with specially designed paediatric content on the experience of pediatric patients undergoing an MRI examination, their caregiver, and clinical staff, on workflow and on clinical outcome. The designed paediatric content exists of personal selection of a character, video projection on the wall and/or a screen visible when in the bore, audio, and room ambience lighting.

The study will be conducted in collaboration with six partner hospitals within the EU. At each hospital, 50 pediatric patients between 6 and 12 years old will be included in the study.

status: Recruiting

study_completion_date_type: Anticipated

design_allocation: Randomized

primary_purpose: Supportive Care

enrollment_type: Anticipated

lead_sponsor: Philips Electronics Nederland B.V. acting through Philips CTO organization

location_country: Germany Poland Denmark Belgium Spain France

location_facility: Polish Mother's Hospital / Institute of Polish Mother's Health Center

intervention_type: Behavioral

status_simplified: Ongoing


title: Multinational, Observational, Retrospective Study to Evaluate Coagulation Changes and the Influence of Antithrombin III Treatment in Patients With Severe COVID-19 Infection

summary: Multicentre, multinational, non-interventional, observational, retrospective, patient record study to evaluate changes in coagulation parameters in patients with severe COVID-19 infection receiving/not treatment with antithrombin (AT) III

lead_sponsor: Octapharma

location_country: Germany Sweden Austria France

location_facility:
ECMO Centre Karolinska
Octapharma Research Site


title: Persistence Of Major Molecular Remission In Chronic Myeloid Leukemia After A Second Stop Of Tki Treatment In Patients Who Failed An Initial Stop Attempt: A Multicenter Prospective Trial

summary: This study will enroll CML patients who have failed a first TKI stopping attempt. After failure and at least a year of TKI treatment, patients will proceed to dasatinib treatment for another 2 years. If MR4 or better is re-achieved and maintained for at least one year, patients will be eligible for a second stop. After verification of MR4, TKI treatment will be stopped and patients followed in the same manner as after first stop. If MMR is lost (BCR-ABL >0.1% (IS)), TKI treatment will once again be restarted.

ipd_sharing_description: Study results will be published as soon as possible. Steering committee will decide on sharing IPD. This is still pending.

lead_sponsor: Amsterdam UMC, location VUmc

sponsor_collaborators:
Amsterdam UMC, location VUmc
Uppsala University Hospital
Skane University Hospital
Helsinki University Central Hospital
Odense University Hospital
Henri Mondor University Hospital
St. Olavs Hospital
Helse Stavanger HF
Aarhus University Hospital
University Hospital, Bonn

location_country: Finland Germany Norway Denmark Sweden Netherlands France

location_facility: Uppsala University Hospital (Akademiska)

location_facility: St Olavs Hospital-Trondheim University Hospital


title: The Effect of Low Protein, Extensively Hydrolyzed Infant Formula on Allergy Prevention in At-risk Infants up to 1 Year of Age: a Randomized, Double-blind, Controlled Intervention Study and the Long-term Effect on Allergy Prevention of Early Nutrition Given in the First 120 Days of Life in At-risk Infants Until the Child is 6 Years of Age

summary: A multi-center, prospective, randomized, controlled, doubleblind intervention clinical Trial is performed to proof the efficiency of hydrolyzed infant formula showing a risk reduction of developing an allergy.

primary_purpose: Prevention

lead_sponsor: HiPP GmbH & Co. Vertrieb KG

location_country: Germany Finland Italy Poland Portugal Spain Czechia Bulgaria Serbia France

location_facility: Second specialized obstetric and gynecological hospital for active treatment Sheinovo EAD

location_facility: Klinik für Kinder- und Jugendmedizin Marienhospital gGmbH Wesel

location_facility: Clinical Hospital Center "Dr Dragisa Misovic-Dedinje"

location_facility: Complejo Hospitalario Universitario de Santiago

location_facility: Klinik für Kinder- und Jugendmedizin der Ruhruniversität Bochum im St. Josef Hospital

location_facility: Studienambulanz der pädiatrische Pneumologie und AllergologieKlinik für Kinder- und Jugendmedizin (KKJM)


title: Incidence of Dyssynchronous Spontaneous Breathing Effort, Breath-stacking and Reverse Triggering in Early ARDS

acronym: BEARDS

summary:
Patients sedated under mechanical ventilation with acute hypoxemic respiratory failure with a PaO2/FiO2 equal or less than 200mmHg (Acute Respiratory Distress Syndrome, ARDS and non-ARDS) will be included in the study early in the course of the disease (first week of mechanical ventilation). At enrollment, data on the clinical condition of the patient will be recorded together with ventilation settings: ventilation mode, the fraction of inspired oxygen (FiO2), PEEP, tidal volume, set pressure, respiratory rate, time of the respiratory cycle, recent blood gas parameters.

Airway pressure, flow, and esophageal pressure (or alternatively electrical activity of the diaphragm, Eadi) will be recorded 3 times a day for 7 days:

Period 1 (morning): duration 20-30 minutes
Period 2 (afternoon): duration 20-30 minutes
Period 3 (evening / night): duration 20-30 minutes

Registration will be ended at extubation, death or at eight days from the first recording.

Monitoring of vital parameters (hemodynamic and respiratory) will be continuous throughout the duration of the study, as per normal clinical practice. All drugs used during the day of the measurements will be recorded. The patient will then be followed until discharge from the ICU and after 60 days of discharge to evaluate mortality.

As an ancillary study, in a subgroup of patients continuous simplified measurement of respiratory recordings together with hourly clinical data on sedation and extended simplified polysomnography recordings will be performed within the first 7 days from inclusion.

The analysis of the recorded waveforms will be performed in a single center by a centralized system that will quantify dyssynchrony and its intensity, calculate pressure time product, collect clinical and physiological data and outcome, and investigate possible correlations.

status: Unknown status

time_perspective: Prospective

ReferenceType: derived

ReferenceCitation: Bianchi I, Grassi A, Pham T, Telias I, Teggia Droghi M, Vieira F, Jonkman A, Brochard L, Bellani G. Reliability of plateau pressure during patient-triggered assisted ventilation. Analysis of a multicentre database. J Crit Care. 2022 Apr;68:96-103. doi: 10.1016/j.jcrc.2021.12.002. Epub 2021 Dec 21. Erratum In: J Crit Care. 2022 Apr 1;:154030.

publisher_dissemination: Elsevier

ReferenceCitation: Pham T, Montanya J, Telias I, Piraino T, Magrans R, Coudroy R, Damiani LF, Mellado Artigas R, Madorno M, Blanch L, Brochard L; BEARDS study investigators. Automated detection and quantification of reverse triggering effort under mechanical ventilation. Crit Care. 2021 Feb 15;25(1):60. doi: 10.1186/s13054-020-03387-3.

oa_colors: green gold

oa_host_type: publisher;repository

evidence: oa journal (via doaj)

host_type: publisher

license: cc-by

license_normalized: cc-by

version: publishedVersion

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

host_type: repository

repository_institution: University of Ferrara - Institutional Research Information System University of Ferrara

repository_institution: Universitat Autònoma de Barcelona - Dipòsit Digital de Documents de la UAB

evidence: oa repository (via pmcid lookup)

license_normalized: no license

repository_normalized: PubMed Central

unpaywall_oa_status: gold

publisher_dissemination: Springer-Nature

ipd_sharing_description: We plan to share physiologic tracings with no participant identification.

lead_sponsor: Unity Health Toronto

location_country: Germany Italy Netherlands China Spain Thailand Greece Taiwan Canada France

location_facility: Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico

location_facility: Azienda Ospedaliero - Universitaria OORR Ospedali Riuniti di Foggia

location_facility: Beijing Tiantan Hospital, Capital Medical University

location_facility: Centre Hospitalier Universitaire - CHU Angers

location_facility: National Cheng-Kung University and Hospital


title: Use of the VisuMax Femtosecond Laser Lenticule Removal Procedure for the Correction of Hyperopia With or Without Astigmatism

summary: The correction of farsightedness using ReLEx SMILE for hyperopia is focus of this investigation. The objective is to evaluate the safety and effectiveness.

lead_sponsor: Carl Zeiss Meditec AG

location_country:
Germany
India
United Kingdom
China
Czechia
France

location_facility: Medical Center Nethradhama Superspeciality Eye Hospital

location_facility: University Medical Center Universitätsklinikum Gießen Marburg

intervention_type: Procedure


title: ULTRA Study: a Feasibility, Prospective, Multicenter, Single-arm Trial of the May Health Device in Transvaginal Ablation of Ovarian Tissue Under ULTRAsound Visualisation in Women With Polycystic Ovary Syndrome (PCOS)

summary: The objective of this study is to provide preliminary evidence for the safety and effectiveness of the May Health Kit in transvaginal ablation of ovarian tissue under ultrasound visualization in women with infertility due to polycystic ovary syndrome.

primary_purpose: Device Feasibility

lead_sponsor: May Health

location_country:
United Kingdom
Belgium
France

location_facility:
CU Saint-Luc
Royal Hallamshire Hospital
CHU Saint-Pierre
Royal Derby Hospital
Liverpool Women Hospital
Bicetre Hospital

intervention_type: Device


intervention_type: Biological

lead_sponsor: RHEACELL GmbH & Co. KG

summary: The aim of this clinical trial is to investigate the efficacy (by monitoring overall improvement of EB symptoms) and safety (by monitoring adverse events) of three doses of allo-APZ2-EB administered intravenously to patients with recessive dystrophic epidermolysis bullosa (RDEB).

ReferenceCitation: Kiritsi D, Dieter K, Niebergall-Roth E, Fluhr S, Daniele C, Esterlechner J, Sadeghi S, Ballikaya S, Erdinger L, Schauer F, Gewert S, Laimer M, Bauer JW, Hovnanian A, Zambruno G, El Hachem M, Bourrat E, Papanikolaou M, Petrof G, Kitzmuller S, Ebens CL, Frank MH, Frank NY, Ganss C, Martinez AE, McGrath JA, Tolar J, Kluth MA. Clinical trial of ABCB5+ mesenchymal stem cells for recessive dystrophic epidermolysis bullosa. JCI Insight. 2021 Nov 22;6(22):e151922. doi: 10.1172/jci.insight.151922.

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

repository_institution: Edith Cowan University - Research Online

repositories_concat: PubMed Central;ro.ecu.edu.au

publisher_dissemination: American Society for Clinical Investigation

ReferenceCitation: Kerstan A, Niebergall-Roth E, Esterlechner J, Schroder HM, Gasser M, Waaga-Gasser AM, Goebeler M, Rak K, Schrufer P, Endres S, Hagenbusch P, Kraft K, Dieter K, Ballikaya S, Stemler N, Sadeghi S, Tappenbeck N, Murphy GF, Orgill DP, Frank NY, Ganss C, Scharffetter-Kochanek K, Frank MH, Kluth MA. Ex vivo-expanded highly pure ABCB5+ mesenchymal stromal cells as Good Manufacturing Practice-compliant autologous advanced therapy medicinal product for clinical use: process validation and first in-human data. Cytotherapy. 2021 Feb;23(2):165-175. doi: 10.1016/j.jcyt.2020.08.012. Epub 2020 Oct 1.

oa_colors: green hybrid

evidence: open (via crossref license)

license: cc-by-nc-nd

license_normalized: cc-by-nc-nd

version: acceptedVersion

unpaywall_oa_status: hybrid

sponsor_collaborators: Granzer Regulatory Consulting & Services

location_facility: University of Minnesota, Masonic Cancer Center and Medical Center

location_facility: King's College London; St John's Institute of Dermatology;

location_facility: EB-Haus Austria; Salzburger Landeskliniken (SALK); Paracelsus Medizinische Privatuniversität Salzburg (PMU)

location_facility: Hôpital Saint-Louis; Département de dermatologie

location_facility: Department of Dermatology, Medical Center-University of Freiburg

location_facility: Great Ormond Street Hospital; Dermatology Department

title: An Interventional, Multicenter, Single Arm, Phase I/IIa Clinical Trial to Investigate the Efficacy and Safety of Allo-APZ2-EB on Epidermolysis Bullosa (EB)

location_country:
Germany
United Kingdom
Austria
United States
France


lead_sponsor: Institut de Recherches Internationales Servier

summary: The purpose of this study is to determine the safety profile, the maximum tolerated dose (MTD) and the associated dose-limiting toxicities (DLTs) of S 81694 in combination with paclitaxel in metastatic breast cancer (mBC) patients, and to investigate the antitumour activity of the combination in metastatic triple negative breast cancer (mTNBC) patients.

location_facility: Erasmus MC Section Clinical Pharmacology

location_facility: UZ Leuven Campus Gasthuisberg Dept. of General Medical

location_facility: Institut Jules Bordet Clinique Oncologie Médicale

location_facility: Institut de Cancérologie de l'Ouest site Saint Herblain

title: Phase I/II Trial of S 81694 Administered Intravenously in Combination With Paclitaxel to Evaluate the Safety, Pharmacokinetic and Efficacy in Metastatic Breast Cancer

type: EudraCT Number

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

Access can be requested for all interventional clinical studies:

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

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

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

location_country: Japan Netherlands Belgium France

ipd_sharing: Yes


lead_sponsor: Chiesi Farmaceutici S.p.A.

summary: The main objectives of the study are to evaluate safety and efficacy of repeated treatment with recombinant human alfa-mannosidase of patients with alfa-mannosidosis aged less than 6 years

sponsor_collaborators:
Chiesi Farmaceutici S.p.A.
Cromsource

title: A 24-month Multicenter, Open-label Phase II Trial Investigating the Safety and Efficacy of Repeated Velmanase Alfa (Recombinant Human Alpha-mannosidase) Treatment in Pediatric Patients Below 6 Years of Age With Alpha-Mannosidosis

location_country: Germany Italy Denmark Austria France


title: A Phase I, Multi-center, Open-label, Study of LXS196, an Oral Protein Kinase C Inhibitor, in Patients With Metastatic Uveal Melanoma

summary: This study was to characterize the safety, tolerability, pharmacokinetics (PK), pharmacodynamics (PD) and preliminary anti-tumor activity of LXS196 as a single agent and in combination with HDM201 in patients with metastatic uveal melanoma.

lead_sponsor: Novartis Pharmaceuticals

location_country:
Norway
Australia
Netherlands
United States
Spain
France

location_facility:
Novartis Investigative Site
Columbia University Medical Center


title: An Open-label, Non-randomized, Multicenter Phase I Dose Escalation Study to Characterize Safety, Tolerability, Pharmacokinetics and Maximum Tolerated Dose of BAY 1238097 in Subjects With Advanced Malignancies

summary:
This is the first study where BAY1238097 is given to humans. Impact of the study is to evaluate if patients with advanced cancer show clinical benefit under the treatment with BET(Bromodomain and extraterminal domain family ) inhibitor.Patients with solid tumors (all comers) and lymphoma will receive the study drug treatment in an escalation scheme (no placebo group) to determine the safety, tolerability and maximum tolerated dose (MTD) of BAY1238097. the relative bioavailability of Liquid Service Formulation and tablets will be determined

After MTD is defined, patients with solid tumors (all comer, hepato cellular carcinoma, lung cancer, NUT(nuclear protein in testis)-midline carcinoma), melanoma and lymphoma will be enrolled A separate escalation scheme will be applied to patients with leucemias, and at the maximal tolerated dose, patients with AML amd multiple myeloma will be enrolled.

the study will also assess the pharmacokinetics, biomarker status, pharmacodynamic parameters of BAY1238097 and tumor response to the treatment.

BAY1238097 will be given twice weekly as oral application. Treatment will be stopped if the tumor continues to grow, if side effects occur, wich the patient cannot tolerate or if the patient decides to withdraw from the treatment.

ReferenceCitation: Postel-Vinay S, Herbschleb K, Massard C, Woodcock V, Soria JC, Walter AO, Ewerton F, Poelman M, Benson N, Ocker M, Wilkinson G, Middleton M. First-in-human phase I study of the bromodomain and extraterminal motif inhibitor BAY 1238097: emerging pharmacokinetic/pharmacodynamic relationship and early termination due to unexpected toxicity. Eur J Cancer. 2019 Mar;109:103-110. doi: 10.1016/j.ejca.2018.12.020. Epub 2019 Jan 31.

oa_colors: green other

evidence: open (via crossref license, author manuscript)

license: publisher-specific-oa

license_normalized: publisher-specific

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

version: submittedVersion

lead_sponsor: Bayer

location_country:
Germany
Finland
United Kingdom
Switzerland
United States
France


acronym: ASCEND-Peds

lead_sponsor: Genzyme, a Sanofi Company

summary:
Primary Objective:

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

Secondary Objective:

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

ReferenceCitation: Diaz GA, Jones SA, Scarpa M, Mengel KE, Giugliani R, Guffon N, Batsu I, Fraser PA, Li J, Zhang Q, Ortemann-Renon C. One-year results of a clinical trial of olipudase alfa enzyme replacement therapy in pediatric patients with acid sphingomyelinase deficiency. Genet Med. 2021 Aug;23(8):1543-1550. doi: 10.1038/s41436-021-01156-3. Epub 2021 Apr 19. Erratum In: Genet Med. 2022 Oct;24(10):2209.

license: cc-by-nc-sa

license_normalized: cc-by-nc-sa

repository_institution: Red Federada de Repositorios Institucionales de Publicaciones Científicas - LA Referencia

repositories_concat: PubMed Central;www.lume.ufrgs.br

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

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

type: Other Identifier

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
United States
France


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

acronym: ARGO

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

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

lead_sponsor: Noscira SA

sponsor_collaborators:
Noscira SA
ICON Clinical Research

location_country:
Finland
Germany
United Kingdom
Belgium
Spain
France


lead_sponsor: Rhythm Pharmaceuticals, Inc.

summary: To demonstrate statistically significant and clinically meaningful effects of setmelanotide on percent body weight change in participants with pro-opiomelanocortin (POMC) deficiency or proprotein convertase subtilisin/kexin type 1 (PCSK1) deficiency obesity due to rare biallelic 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: Institute of Cardiometabolism and Nutrition / Hopital de la Pitié-Salpêtrière

location_facility: University of Cambridge Metabolic Research Laboratories

location_facility: Charité Campus Virchow-Klinikum / Institute for Experimental Pediatric Endocrinology

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 Early Onset POMC Deficiency Obesity Due to Bi-Allelic Loss-of-Function POMC or PCSK1 Genetic Mutation

location_country:
Germany
United Kingdom
Belgium
United States
Spain
Canada
France


title: Molecular Reclassification to Find Clinically Useful Biomarkers for Systemic Autoimmune Diseases: Cross Sectional Cohort

acronym: PRECISESADS

summary: Connective tissue diseases (CTD) or systemic autoimmune diseases (SADs) as they are known today are a group of chronic inflammatory conditions with autoimmune aetiology with few treatment options and difficult diagnosis.Brest team contribute to perform a new classification of the following systemic autoimmune diseases in a European Union's Seventh Framework Programme. The aim of this research is to reclassify the individuals affected by SADs into molecular clusters instead of clinical entities through the determination of molecular profiles using several "Omics" techniques.

lead_sponsor: Fundación Pública Andaluza Progreso y Salud

collaborators: Fondazione IRCCS Ca' Granda, Ospedale Maggiore Policlinico

collaborators: August Pi Sunyer Biomedical Research Institute

collaborators: The Cyprus Foundation for Muscular Dystrophy Research

collaborators: Institut de Recherches Internationales Servier

collaborators: Institut d'Investigació Biomèdica de Bellvitge

location_country: Germany Italy Portugal Hungary Switzerland Austria Belgium Spain France

location_facility: Fondazione IRCCS Ca Granda Ospedale Maggiore Policlinico (IRCCS)

location_facility: Hospital Clinic I Provicia- Institut d'Investigacions Biomèdiques August Pi i Sunyer (IDIBAPS)

location_facility: UZ Leuven - KU Leuven, Department of Rheumatology (KU LEUVEN)

location_facility: Hospital Universitario San Cecilio Servicio Andaluz de Salud

location_facility: Hospital Universitario Marqués de Valdecilla, Servicio Cántabro de Salud

location_facility: Université catholique de Louvain - Cliniques Universitaires Saint-Luc

location_facility: Hospital Regional de Málaga Servicio Andaluz de Salud

location_facility: Deutsches Rheuma-Forschungszentrum Berlin (DRFZ)

location_facility: Hospital Universitario Reina Sofía Andaluz de Salud


lead_sponsor: Grifols Therapeutics LLC

summary: Approximately 60 subjects will be enrolled in order to have approximately 20 adult subjects and 20 pediatric subjects treated with subcutaneously administered Immune Globulin Subcutaneous (Human), 20% Caprylate/Chromatography Purified (IGSC 20%) who complete the entire study. This study will include 3 study stages: Screening/Previous Regimen Phase, IGSC 20% Treatment Stage 1 (13 IGSC 20% weekly doses), and IGSC 20% Treatment Stage 2 (39 IGSC 20% weekly doses). A total of 52 doses of IGSC 20% will be administered with a final follow-up visit 1 week after the last dose at Week 53. Subjects/caregivers will be trained on self-administration of IGSC 20% by the clinical site personnel.

location_facility: Children's Hospital at Municipal Hospital St. Georg

location_facility: The Children's Memorial Health Institute

title: A Multi-Center, Open-Label, Single-Arm Trial to Evaluate Efficacy, Pharmacokinetics, and Safety and Tolerability of IGSC 20% in Subjects With Primary Immunodeficiency

location_country:
Germany
Poland
Hungary
United Kingdom
Australia
Sweden
Spain
Czechia
France


title: Pipeline Flex With SHield Technology Embolization - An International MulticEnter ObservationaL Post Market StuDy of Treated Intra Cranial Aneurysms (SHIELD)

acronym: SHIELD

summary: This is a prospective, single-arm, multi-center post-market observational study assessing the performance of the Pipeline™ Flex Embolization Device with Shield Technology™ in subjects undergoing treatment for intracranial aneurysms in a large real-world, post-market setting.

ReferenceCitation: Nelson PK, Lylyk P, Szikora I, Wetzel SG, Wanke I, Fiorella D. The pipeline embolization device for the intracranial treatment of aneurysms trial. AJNR Am J Neuroradiol. 2011 Jan;32(1):34-40. doi: 10.3174/ajnr.A2421. Epub 2010 Dec 9.

ReferenceCitation: Becske T, Kallmes DF, Saatci I, McDougall CG, Szikora I, Lanzino G, Moran CJ, Woo HH, Lopes DK, Berez AL, Cher DJ, Siddiqui AH, Levy EI, Albuquerque FC, Fiorella DJ, Berentei Z, Marosfoi M, Cekirge SH, Nelson PK. Pipeline for uncoilable or failed aneurysms: results from a multicenter clinical trial. Radiology. 2013 Jun;267(3):858-68. doi: 10.1148/radiol.13120099. Epub 2013 Feb 15.

ReferenceCitation: Kallmes DF, Hanel R, Lopes D, Boccardi E, Bonafe A, Cekirge S, Fiorella D, Jabbour P, Levy E, McDougall C, Siddiqui A, Szikora I, Woo H, Albuquerque F, Bozorgchami H, Dashti SR, Delgado Almandoz JE, Kelly ME, Turner R 4th, Woodward BK, Brinjikji W, Lanzino G, Lylyk P. International retrospective study of the pipeline embolization device: a multicenter aneurysm treatment study. AJNR Am J Neuroradiol. 2015 Jan;36(1):108-15. doi: 10.3174/ajnr.A4111. Epub 2014 Oct 29. Erratum In: AJNR Am J Neuroradiol. 2015 May;36(5):E39-40.

ReferenceCitation: Chitale R, Gonzalez LF, Randazzo C, Dumont AS, Tjoumakaris S, Rosenwasser R, Chalouhi N, Gordon D, Jabbour P. Single center experience with pipeline stent: feasibility, technique, and complications. Neurosurgery. 2012 Sep;71(3):679-91; discussion 691. doi: 10.1227/NEU.0b013e318260fe86.

ReferenceCitation: Saatci I, Yavuz K, Ozer C, Geyik S, Cekirge HS. Treatment of intracranial aneurysms using the pipeline flow-diverter embolization device: a single-center experience with long-term follow-up results. AJNR Am J Neuroradiol. 2012 Sep;33(8):1436-46. doi: 10.3174/ajnr.A3246. Epub 2012 Jul 19.

ReferenceCitation: Kan P, Siddiqui AH, Veznedaroglu E, Liebman KM, Binning MJ, Dumont TM, Ogilvy CS, Gaughen JR Jr, Mocco J, Velat GJ, Ringer AJ, Welch BG, Horowitz MB, Snyder KV, Hopkins LN, Levy EI. Early postmarket results after treatment of intracranial aneurysms with the pipeline embolization device: a U.S. multicenter experience. Neurosurgery. 2012 Dec;71(6):1080-7; discussion 1087-8. doi: 10.1227/NEU.0b013e31827060d9.

ReferenceCitation: Yu SC, Kwok CK, Cheng PW, Chan KY, Lau SS, Lui WM, Leung KM, Lee R, Cheng HK, Cheung YL, Chan CM, Wong GK, Hui JW, Wong YC, Tan CB, Poon WL, Pang KY, Wong AK, Fung KH. Intracranial aneurysms: midterm outcome of pipeline embolization device--a prospective study in 143 patients with 178 aneurysms. Radiology. 2012 Dec;265(3):893-901. doi: 10.1148/radiol.12120422. Epub 2012 Sep 20.

ReferenceCitation: McAuliffe W, Wycoco V, Rice H, Phatouros C, Singh TJ, Wenderoth J. Immediate and midterm results following treatment of unruptured intracranial aneurysms with the pipeline embolization device. AJNR Am J Neuroradiol. 2012 Jan;33(1):164-70. doi: 10.3174/ajnr.A2727. Epub 2011 Oct 6.

ReferenceCitation: Skukalek SL, Winkler AM, Kang J, Dion JE, Cawley CM, Webb A, Dannenbaum MJ, Schuette AJ, Asbury B, Tong FC. Effect of antiplatelet therapy and platelet function testing on hemorrhagic and thrombotic complications in patients with cerebral aneurysms treated with the pipeline embolization device: a review and meta-analysis. J Neurointerv Surg. 2016 Jan;8(1):58-65. doi: 10.1136/neurintsurg-2014-011145. Epub 2014 Nov 10.

ReferenceCitation: Rice H, Martinez Galdamez M, Holtmannspotter M, Spelle L, Lagios K, Ruggiero M, Vega P, Sonwalkar H, Chapot R, Lamin S. Periprocedural to 1-year safety and efficacy outcomes with the Pipeline Embolization Device with Shield technology for intracranial aneurysms: a prospective, post-market, multi-center study. J Neurointerv Surg. 2020 Nov;12(11):1107-1112. doi: 10.1136/neurintsurg-2020-015943. Epub 2020 Jun 1.

evidence: open (via page says license)

license: cc-by-nc

license_normalized: cc-by-nc

repositories_concat: PubMed Central

publisher_dissemination: British Medical Journal

ipd_sharing_description: No individual participant data will be available

lead_sponsor: Medtronic Neurovascular Clinical Affairs

location_country:
Finland
Germany
Italy
Hungary
Israel
Denmark
Australia
United Kingdom
Spain
Greece
France

location_facility: Hospital Universitario Central de Asturias


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

acronym: AUDITOR

summary:
Objectives:

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

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

lead_sponsor: Sanofi

location_country:
United Kingdom
Netherlands
United States
Spain
Canada
France


title: Randomized, Double-Masked, Sham-Controlled Clinical Trial to Evaluate the Efficacy of a Single Intravitreal Injection of GS010 in Subjects Affected for More Than 6 Months and To 12 Months by LHON Due to the G11778A Mutation in the ND4 Gene

acronym: REVERSE

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 more than six months and up to one year.

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

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

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: Centre National Hospitalier d'Ophtalmologie des Quinze-Vingt

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: Moorfields Eye Hospital NHS Foundation Trust


title: A Prospective Observational, Long Term Follow up Study of Patients Treated With Imlifidase Prior to Kidney Transplantation

summary:
The rationale for the current protocol is to collect data from extended follow up in subjects that have received a kidney transplant following imlifidase dosing to provide a better understanding regarding the long-term outcome for these subjects. Data of parameters such as patient and graft survival, comorbidity, treatment of graft rejection episodes and quality of life as well as anti-drug antibody levels will be collected.

This prospective, observational follow up study of subjects who have received imlifidase prior to kidney transplantation will provide important data to future prescribers and patients of the potential long-term benefits of imlifidase mediated transplantation.

lead_sponsor: Hansa Biopharma AB

location_country:
United States
Sweden
France

location_facility:
Karolinska University Hospital
Cedars-Sinai Medical Center
Uppsala University Hospital
New York University School of Medicine
Necker Hospital
The Johns Hopkins Hospital


title: A Multicentre Open-label Phase IIa Study With Escalating Dose of MS1819- SD, to Investigate the Efficacy and Safety of a Yarrowia Lipolytica Lipase Preparation for the Compensation of Exocrine Pancreatic Insufficiency Caused by Chronic Pancreatitis and/or Distal Pancreatectomy

summary:
This is a Phase IIa study sponsored by AzurRx SAS and Syneos Health is a local representative sponsor and involves testing of a new medication for the compensation of exocrine pancreatic insufficiency (EPI) caused by chronic pacreatitis (CP) and/or distal pancreatectomy. The new medication is called MS1819 Spray Dried (MS1819-SD) which is a lipase produced by the LIP2 gene of Yarrowia lipolytica using recombinant DNA technology.

The primary purpose of this study is to investigate the safety of escalating doses of study drug MS1819-SD in people with chronic pancreatitis. This enzyme has demonstrated an appropriate profile to compensate the pancreatic lipase (enzyme) deficiency that is common with CP patients. The deficiency in this enzyme can be responsible of greasy diarrhea, fecal urge and weight loss.

The design of the study is open-label, meaning that all eligible participants will receive the study drug MS1819-SD. The MS1819-SD dose will increase throughout the study during dose escalation visits in each treatment period; study includes a total of four treatment periods.

The total duration of the MS1819-SD treatment phase is of 48-60 days, The total duration of patient participation in the study is of 74-93 days.

Approximately twelve patients will be enrolled in this study.

lead_sponsor: AzurRx SAS

location_country:
Australia
New Zealand
France

location_facility: Mackay Institute of Research and Innovation


title: Long Term Follow-up of Integra® Cadence™ Total Ankle System in Primary Ankle Joint Replacement

summary: This study will evaluate the long term performance and safety data for the Cadence™ Total Ankle System (CTAS) when used for primary arthroplasty in patients with primary arthritis (e.g. degenerative disease), secondary arthritis (e.g. post-traumatic, avascular necrosis, if minimally 2/3 of the talus is preserved), and systemic arthritis of the ankle (e.g. rheumatoid arthritis, hemochromatosis)

lead_sponsor: Smith & Nephew, Inc.

sponsor_collaborators:
Smith & Nephew, Inc.
Smith & Nephew Orthopaedics AG

location_country:
United Kingdom
Belgium
Spain
Canada
France

location_facility:
Universitair Ziekenhuis Gent
North Cumbria University Hospitals
St. Michael's Hospital
CHRU Tours
Foot and Ankle Institute
Clinica Nostra Senhora del Remei


acronym: PALL

summary: This study aims to evaluate the safety and feasibility of UCART19 to induce molecular remission in pediatric patients with relapsed or refractory CD19-positive B-cell acute lymphoblastic leukemia (B-ALL).

ReferenceCitation: Benjamin R, Graham C, Yallop D, Jozwik A, Mirci-Danicar OC, Lucchini G, Pinner D, Jain N, Kantarjian H, Boissel N, Maus MV, Frigault MJ, Baruchel A, Mohty M, Gianella-Borradori A, Binlich F, Balandraud S, Vitry F, Thomas E, Philippe A, Fouliard S, Dupouy S, Marchiq I, Almena-Carrasco M, Ferry N, Arnould S, Konto C, Veys P, Qasim W; UCART19 Group. Genome-edited, donor-derived allogeneic anti-CD19 chimeric antigen receptor T cells in paediatric and adult B-cell acute lymphoblastic leukaemia: results of two phase 1 studies. Lancet. 2020 Dec 12;396(10266):1885-1894. doi: 10.1016/S0140-6736(20)32334-5.

sponsor_collaborators: Institut de Recherches Internationales Servier

location_facility: University of Texas Southwestern Medical Center

title: A Phase 1, Open Label, Non-comparative Study to Evaluate the Safety and the Ability of UCART19 to Induce Molecular Remission in Paediatric Patients With Relapsed/Refractory B-cell Acute Lymphoblastic Leukaemia

location_country:
United States
Spain
United Kingdom
France


summary: The purpose of this study was to determine the maximum tolerated dose (MTD) / recommended phase 2 dose (RP2D) of nazartinib (EGF816) in combination with capmatinib (INC280) and to estimate the preliminary anti-tumor activity of nazartinib in combination with capmatinib in participants with advanced non-small cell lung cancer (NSCLC) with documented EGFR mutation.

ReferenceCitation: Jia Y, Juarez J, Li J, Manuia M, Niederst MJ, Tompkins C, Timple N, Vaillancourt MT, Pferdekamper AC, Lockerman EL, Li C, Anderson J, Costa C, Liao D, Murphy E, DiDonato M, Bursulaya B, Lelais G, Barretina J, McNeill M, Epple R, Marsilje TH, Pathan N, Engelman JA, Michellys PY, McNamara P, Harris J, Bender S, Kasibhatla S. EGF816 Exerts Anticancer Effects in Non-Small Cell Lung Cancer by Irreversibly and Selectively Targeting Primary and Acquired Activating Mutations in the EGF Receptor. Cancer Res. 2016 Mar 15;76(6):1591-602. doi: 10.1158/0008-5472.CAN-15-2581. Epub 2016 Jan 29.

evidence: open (via free pdf)

evidence: oa repository (via page says license)

unpaywall_oa_status: bronze

publisher_dissemination: American Association for Cancer Research

location_facility: Massachusetts General Hospital Mass General

title: A Phase Ib/II, Multicenter, Open-label Study of EGF816 in Combination With INC280 in Adult Patients With EGFR Mutated Non-small Cell Lung Cancer.

location_country:
Germany
Italy
Norway
Singapore
Australia
United States
Korea, Republic of
Spain
Taiwan
Canada
France


title: A Phase II, Multicenter, Open-label Study of EGF816 in Combination With Nivolumab in Adult Patients With EGFR Mutated Non-small Cell Lung Cancer and of INC280 in Combination With Nivolumab in Adult Patients With cMet Positive Non-small Cell Lung Cancer

summary: To determine the efficacy and safety of nivolumab in combination with EGF816 and of nivolumab in combination with INC280 in previously treated NSCLC patients

location_country:
Germany
Italy
Singapore
Australia
Switzerland
United States
Spain
France

location_facility: University of Texas MD Anderson Cancer Center Thoractic Head/Neck Med.Onc(2)


title: Testing the Effect of Intrauterine Slow Release Insemination (SRI) on the Pregnancy Rate in Women Designated for Standard Intrauterine Insemination (IUI)

acronym: EVIE

summary:
Overall Study Design and Plan Description

The study will be conducted on women with fertility difficulties who are designated for intrauterine insemination treatment (IUI). The research sample size is 137 treatment cycles per group (total of 274 treatment cycles). The women will be randomly divided into two groups. Some will undergo the standard bolus IUI treatment and others will be treated with the EVIE Slow Release Insemination method (SRI). Women who fail to conceive in the first treatment will next receive the alternative treatment. This means that a woman treated with the IUI method who does not become pregnant will then be treated with SRI, and vice versa (crossover method). The study will include women treated with Clomiphene Citrate or with other Gonadotropin-based treatment. Every couple treated will undergo at least 2 insemination cycles (unless pregnancy has already been achieved).
Before beginning the hormone treatment, each couple will have the trial procedure explained to them. This explanation will include all the detailed information and instruction about the IUI and the Slow Release Insemination methods. A Patient Information Sheet will be available to them.
Preparation of the spermatozoa for intra uterine insemination will be identical to the method performed currently before IUI.

Approximately two weeks after the insemination treatment, a blood sample will be taken from the woman to check for Beta hCG levels to indicate pregnancy.

Hypothesis:

SRI leads to higher pregnancy rates compared to standard IUI

Primary endpoint:

• Pregnancy rate Effect of controlled Intra-uterine slow release insemination on the pregnancy rate of women designated for intra uterine insemination in comparison to the accepted IUI method.

ReferenceCitation: Muharib NS, Abdel Gadir A, Shaw RW. Slow release intrauterine insemination versus the bolus technique in the treatment of women with cervical mucus hostility. Hum Reprod. 1992 Feb;7(2):227-9. doi: 10.1093/oxfordjournals.humrep.a137622.

lead_sponsor: Medical University of Vienna

sponsor_collaborators:
Medical University of Vienna
Reproductive Sciences Ltd.

location_country:
Germany
United Kingdom
Austria
France

location_facility: Klinikum der Johann Wolfgang-Goethe-Universität Klinik für Frauenheilkunde und Geburtshilfe

location_facility: Dept. Obstetrics and Gynaecology, Medical University of Vienna

location_facility: Kinderwunschzentrum der Goldenes Kreuz Privatklinik

location_facility: LMU, Klinikum der Universität München Klinik und Poliklinik für Frauenheilkunde und Geburtshilfe

location_facility: Frauenärztliche Gemeinschaftspraxis Dr. Maarten R. van Santen; KriegsstraBe 216

location_facility: Centre for Reproductive and Genetic Health - Eastman Dental Hospital

location_facility: Dept. Obstetrics and Gynaekology, Medical Faculty of Aachen

lead_sponsor_type: academique


title: A Phase 2a, Randomized, Double-Blind, Placebo-Controlled, Parallel-Group Study to Evaluate the Efficacy and Safety of AMG 714 in Adult Patients With Type II Refractory Celiac Disease, an In Situ Small Bowel T Cell Lymphoma

summary: Protocol CELIM-RCD-002 is designed to evaluate the efficacy and safety of AMG 714 for the treatment of adult patients with type II refractory celiac disease (RCD-II), an in-situ small bowel T-cell lymphoma.

ReferenceCitation: Cellier C, Bouma G, van Gils T, Khater S, Malamut G, Crespo L, Collin P, Green PHR, Crowe SE, Tsuji W, Butz E, Cerf-Bensussan N, Macintyre E, Parnes JR, Leon F, Hermine O, Mulder CJ; RCD-II Study Group Investigators. Safety and efficacy of AMG 714 in patients with type 2 refractory coeliac disease: a phase 2a, randomised, double-blind, placebo-controlled, parallel-group study. Lancet Gastroenterol Hepatol. 2019 Dec;4(12):960-970. doi: 10.1016/S2468-1253(19)30265-1. Epub 2019 Sep 4.

evidence: open (author manuscript)

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

repositories_concat: HAL

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

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

ipd_sharing_description: Publication in peer-reviewed journals

lead_sponsor: Amgen

location_country:
Finland
Netherlands
United States
Spain
France


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

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

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

lead_sponsor: Osteovox

location_country: Italy Belgium France

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


title: CONTRAST (Can cONTrast Injection Better Approximate FFR compAred to Pure reSTing Physiology?)

acronym: CONTRAST

summary: The purpose of this study is to determine the diagnostic performances of iodine contrast medium and resting conditions to predict fractional flow reserve (FFR). Reference FFR will be measured using standard adenosine. We hypothesize that contrast FFR will offer superior diagnostic agreement compared to resting conditions.

ReferenceCitation: De Bruyne B, Pijls NH, Barbato E, Bartunek J, Bech JW, Wijns W, Heyndrickx GR. Intracoronary and intravenous adenosine 5'-triphosphate, adenosine, papaverine, and contrast medium to assess fractional flow reserve in humans. Circulation. 2003 Apr 15;107(14):1877-83. doi: 10.1161/01.CIR.0000061950.24940.88. Epub 2003 Mar 31.

ReferenceCitation: Johnson NP, Jeremias A, Zimmermann FM, Adjedj J, Witt N, Hennigan B, Koo BK, Maehara A, Matsumura M, Barbato E, Esposito G, Trimarco B, Rioufol G, Park SJ, Yang HM, Baptista SB, Chrysant GS, Leone AM, Berry C, De Bruyne B, Gould KL, Kirkeeide RL, Oldroyd KG, Pijls NHJ, Fearon WF. Continuum of Vasodilator Stress From Rest to Contrast Medium to Adenosine Hyperemia for Fractional Flow Reserve Assessment. JACC Cardiovasc Interv. 2016 Apr 25;9(8):757-767. doi: 10.1016/j.jcin.2015.12.273.

repository_institution: Eindhoven University of Technology - Eindhoven University of Technology Research Portal

repository_institution: Hospital Prof. Dr. Fernando Fonseca - The Institutional Repository of Hospital Prof. Dr. Fernando Fonseca

repository_institution: The University of Glasgow - Enlighten: Publications

ReferenceCitation: Johnson NP, Matsuo H, Nakayama M, Eftekhari A, Kakuta T, Tanaka N, Christiansen EH, Kirkeeide RL, Gould KL. Combined Pressure and Flow Measurements to Guide Treatment of Coronary Stenoses. JACC Cardiovasc Interv. 2021 Sep 13;14(17):1904-1913. doi: 10.1016/j.jcin.2021.07.041.

repository_institution: Elsevier - Scopus

repository_normalized: Pure (Denmark)

repositories_concat: Pure (Denmark)

ReferenceCitation: Johnson NP, Collet C. Can FFR After Stenting Help Reduce Target Vessel Failure? JACC Cardiovasc Interv. 2021 Sep 13;14(17):1901-1903. doi: 10.1016/j.jcin.2021.08.001. No abstract available.

oa_host_type: publisher

ReferenceCitation: Gould KL, Johnson NP, Roby AE, Nguyen T, Kirkeeide R, Haynie M, Lai D, Zhu H, Patel MB, Smalling R, Arain S, Balan P, Nguyen T, Estrera A, Sdringola S, Madjid M, Nascimbene A, Loyalka P, Kar B, Gregoric I, Safi H, McPherson D. Regional, Artery-Specific Thresholds of Quantitative Myocardial Perfusion by PET Associated with Reduced Myocardial Infarction and Death After Revascularization in Stable Coronary Artery Disease. J Nucl Med. 2019 Mar;60(3):410-417. doi: 10.2967/jnumed.118.211953. Epub 2018 Aug 16.

repository_institution: PubMed Central - Europe PMC

publisher_dissemination: Society of Nuclear Medicine

ReferenceCitation: Johnson NP, Kirkeeide RL, Gould KL. Same Lesion, Different Artery, Different FFR!? JACC Cardiovasc Imaging. 2019 Apr;12(4):718-721. doi: 10.1016/j.jcmg.2017.11.029. Epub 2018 Jan 17. No abstract available.

ReferenceCitation: Kobayashi Y, Johnson NP, Zimmermann FM, Witt N, Berry C, Jeremias A, Koo BK, Esposito G, Rioufol G, Park SJ, Nishi T, Choi DH, Oldroyd KG, Barbato E, Pijls NHJ, De Bruyne B, Fearon WF; CONTRAST Study Investigators. Agreement of the Resting Distal to Aortic Coronary Pressure With the Instantaneous Wave-Free Ratio. J Am Coll Cardiol. 2017 Oct 24;70(17):2105-2113. doi: 10.1016/j.jacc.2017.08.049.

oa_host_type: repository

ReferenceCitation: Nishi T, Johnson NP, De Bruyne B, Berry C, Gould KL, Jeremias A, Oldroyd KG, Kobayashi Y, Choi DH, Pijls NHJ, Fearon WF; CONTRAST Study Investigators. Influence of Contrast Media Dose and Osmolality on the Diagnostic Performance of Contrast Fractional Flow Reserve. Circ Cardiovasc Interv. 2017 Oct;10(10):e004985. doi: 10.1161/CIRCINTERVENTIONS.117.004985.

ReferenceCitation: Matsumura M, Johnson NP, Fearon WF, Mintz GS, Stone GW, Oldroyd KG, De Bruyne B, Pijls NHJ, Maehara A, Jeremias A. Accuracy of Fractional Flow Reserve Measurements in Clinical Practice: Observations From a Core Laboratory Analysis. JACC Cardiovasc Interv. 2017 Jul 24;10(14):1392-1401. doi: 10.1016/j.jcin.2017.03.031.

ReferenceCitation: Gargiulo G, Stabile E, Ferrone M, Barbato E, Zimmermann FM, Adjedj J, Hennigan B, Matsumura M, Johnson NP, Fearon WF, Jeremias A, Trimarco B, Esposito G; CONTRST Study Investigators. Diabetes does not impact the diagnostic performance of contrast-based fractional flow reserve: insights from the CONTRAST study. Cardiovasc Diabetol. 2017 Jan 13;16(1):7. doi: 10.1186/s12933-016-0494-2.

ReferenceCitation: Johnson NP, Gould KL, Di Carli MF, Taqueti VR. Invasive FFR and Noninvasive CFR in the Evaluation of Ischemia: What Is the Future? J Am Coll Cardiol. 2016 Jun 14;67(23):2772-2788. doi: 10.1016/j.jacc.2016.03.584.

ReferenceCitation: Gould KL, Johnson NP. Coronary Blood Flow After Acute MI: Alternative Truths. JACC Cardiovasc Interv. 2016 Mar 28;9(6):614-7. doi: 10.1016/j.jcin.2016.02.009. No abstract available.

ReferenceCitation: Gould KL, Johnson NP. Myocardial Bridges: Lessons in Clinical Coronary Pathophysiology. JACC Cardiovasc Imaging. 2015 Jun;8(6):705-9. doi: 10.1016/j.jcmg.2015.02.013. No abstract available.

ReferenceCitation: Gould KL, Johnson NP, Kaul S, Kirkeeide RL, Mintz GS, Rentrop KP, Sdringola S, Virmani R, Narula J. Patient selection for elective revascularization to reduce myocardial infarction and mortality: new lessons from randomized trials, coronary physiology, and statistics. Circ Cardiovasc Imaging. 2015 May;8(5):e003099. doi: 10.1161/CIRCIMAGING.114.003099. No abstract available.

lead_sponsor: The University of Texas Health Science Center, Houston

location_country:
Italy
Portugal
United Kingdom
Sweden
Netherlands
Belgium
United States
Korea, Republic of
France

location_facility: Seoul National University College of Medicine


title: A PHASE I TRIAL OF BUTYRATE AND GANCICLOVIR IN EBV-ASSOCIATED MALIGNANCIES

summary:
RATIONALE: The Epstein Barr virus can cause cancer and lymphoproliferative disorders. Ganciclovir is an antiviral drug that acts against the Epstein Barr virus. Arginine butyrate may make virus cells more sensitive to ganciclovir. Combining ganciclovir and arginine butyrate may kill more Epstein Barr virus cells and tumor cells.

PURPOSE: Phase I trial to study the effectiveness of arginine butyrate plus ganciclovir in treating patients who have cancer or lymphoproliferative disorders that are associated with the Epstein Barr virus.

ReferenceCitation: Faller DV, Mentzer SJ, Perrine SP. Induction of the Epstein-Barr virus thymidine kinase gene with concomitant nucleoside antivirals as a therapeutic strategy for Epstein-Barr virus-associated malignancies. Curr Opin Oncol. 2001 Sep;13(5):360-7. doi: 10.1097/00001622-200109000-00008.

ReferenceCitation: Mentzer SJ, Perrine SP, Faller DV. Epstein--Barr virus post-transplant lymphoproliferative disease and virus-specific therapy: pharmacological re-activation of viral target genes with arginine butyrate. Transpl Infect Dis. 2001 Sep;3(3):177-85. doi: 10.1034/j.1399-3062.2001.003003177.x.

ReferenceCitation: Perrine SP, Hermine O, Small T, Suarez F, O'Reilly R, Boulad F, Fingeroth J, Askin M, Levy A, Mentzer SJ, Di Nicola M, Gianni AM, Klein C, Horwitz S, Faller DV. A phase 1/2 trial of arginine butyrate and ganciclovir in patients with Epstein-Barr virus-associated lymphoid malignancies. Blood. 2007 Mar 15;109(6):2571-8. doi: 10.1182/blood-2006-01-024703. Epub 2006 Nov 21.

ReferenceCitation: Mentzer SJ, Fingeroth J, Reilly JJ, Perrine SP, Faller DV. Arginine butyrate-induced susceptibility to ganciclovir in an Epstein-Barr-virus-associated lymphoma. Blood Cells Mol Dis. 1998 Jun;24(2):114-23. doi: 10.1006/bcmd.1998.0178.

lead_sponsor: Boston Medical Center

location_country:
United States
Italy
Germany
France

location_facility: Cancer Research Center at Boston Medical Center

location_facility: Istituto Nazionale per lo Studio e la Cura dei Tumori

location_facility: Methodist Cancer Center at Methodist Hospital


title: A Phase Ib/II Study of LEE011 in Combination With Fulvestrant and BYL719 or BKM120 in the Treatment of Postmenopausal Women With Hormone Receptor Positive, HER2 Negative Locally Recurrent or Advanced Metastatic Breast Cancer

summary:
The purpose of this trial is to explore the clinical utility of the three investigational agents in HR+, HER2- breast cancer. LEE011 (CDK4/6 inhibitor), BKM120 (PI3K-pan class I-inhibitor) and BYL719 (PI3K-alpha specific class I inhibitor) in combination with fulvestrant.

This is a multi-center, open-label Phase Ib/II study. The Phase Ib portion of the study is a dose escalation to estimate the MTD and/or RP2D for three regimens: LEE011 with fulvestrant; LEE011 and BKM120 with fulvestrant; LEE011and BYL719 with fulvestrant.

The Phase II portion of the study was planned to be a randomized study to assess the anti-tumor activity as well as safety and tolerability of LEE011 with fulvestrant to LEE011 and BKM120 with fulvestrant, and LEE011 and BYL719 with fulvestrant in patients with ER+/HER2- locally advanced or metastatic breast cancer.

Approximately 216 adult women with ER+/HER2- locally advanced or metastatic breast cancer were planned to be enrolled.

ReferenceCitation: Tolaney SM, Im YH, Calvo E, Lu YS, Hamilton E, Forero-Torres A, Bachelot T, Maur M, Fasolo A, Tiedt R, Nardi L, Stammberger U, Abdelhady AM, Ruan S, Lee SC. Phase Ib Study of Ribociclib plus Fulvestrant and Ribociclib plus Fulvestrant plus PI3K Inhibitor (Alpelisib or Buparlisib) for HR+ Advanced Breast Cancer. Clin Cancer Res. 2021 Jan 15;27(2):418-428. doi: 10.1158/1078-0432.CCR-20-0645. Epub 2020 Sep 4.

repository_institution: University of Illinois at Chicago - INDIGO

lead_sponsor: Novartis Pharmaceuticals

location_country:
Italy
United Kingdom
Singapore
United States
Korea, Republic of
Spain
Taiwan
France

location_facility: Sarah Cannon Research Institute Onc Dept

location_facility: University of Alabama at Birmingham/ Kirklin Clinic Dept Onc


title: A Phase I Open-label, Dose Escalation Study to Investigate The Safety, Pharmacokinetics, Pharmacodynamics and Clinical Activity of GSK2879552 Given Orally in Subjects With Relapsed/Refractory Small Cell Lung Carcinoma

summary: GSK2879552 is a potent, selective, mechanism-based inactivator of Lysine Specific Demethylase 1 (LSD1)/ CoRepressor for Element-1-Silencing Transcription factor (CoREST) activity. This is a phase I, open-label, multi-center, non-randomized, 2-part first time in human (FTIH) study for GSK2879552. Part 1 is a dose escalation phase to determine the recommended phase 2 dose (RP2D) for GSK2879552 based on the safety, tolerability, pharmacokinetics (PK) and pharmacodynamics (PD) profiles observed after oral administration of GSK2879552. Any dose level(s) may be expanded up to 12 subjects in order to collect additional data on PK and PD.The safety and PK/PD data will be reviewed prior to the dose decision, and the dose escalation will be guided by the Neuenschwander -continuous reassessment method (N-CRM). Built-in safety constraints are in place to prevent exposing subjects to undue risk of toxicity. Once RP2D is identified, an expansion cohort (Part 2) of up to 30 subjects will be enrolled to further evaluate the clinical activity and tolerability of GSK2879552 in subjects with relapsed/refractory SCLC.

ReferenceCitation: Bauer TM, Besse B, Martinez-Marti A, Trigo JM, Moreno V, Garrido P, Ferron-Brady G, Wu Y, Park J, Collingwood T, Kruger RG, Mohammad HP, Ballas MS, Dhar A, Govindan R. Phase I, Open-Label, Dose-Escalation Study of the Safety, Pharmacokinetics, Pharmacodynamics, and Efficacy of GSK2879552 in Relapsed/Refractory SCLC. J Thorac Oncol. 2019 Oct;14(10):1828-1838. doi: 10.1016/j.jtho.2019.06.021. Epub 2019 Jun 28.

ipd_sharing_description: IPD for this study will be made available via the Clinical Study Data Request site.

lead_sponsor: GlaxoSmithKline

location_country:
United States
Spain
France


acronym: GEMINI

lead_sponsor: Biogen

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

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

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

location_country:
United States
Germany
France


title: An Open-Label, Multi-Center, Dose-Escalation, Phase I Study With an Expansion Phase, to Evaluate Safety, Pharmacokinetics and Therapeutic Activity of RO6895882, an Immunocytokine, Consisting of a Variant of Interleukin-2 (IL-2v) Targeting Carcinoembryonic Antigen (CEA) Administered Intravenously, in Patients With Advanced and/or Metastatic Solid Tumors

summary: This open-label, multi-center, dose-escalation study will evaluate the safety, pharmacokinetics, and therapeutic activity of RO6895882 in participants with Carcinoembryonic Antigen (CEA)-positive solid tumors who have progressed on the standard of care therapy. The study will be conducted in 3 parts. Part 1 will be a single ascending dose study in single participant cohort at low RO6895882 dose (less than or equal to [</=] 6 milligrams [mg]). Part 2 will be a dose-escalation study of RO6895882 monotherapy given every week (qw), every 2 weeks (q2w), and possibly every 3 weeks (q3w). Part 3 will be an expansion phase of the qw, q2w, and possibly q3w at maximum tolerated dose (MTD) (as determined in Part 2). Part 3 will only be conducted if the risk/benefit assessment, as evaluated by the Sponsor and the investigators, is in favor of the participants. Participants will be treated until disease progression, unacceptable toxicity or withdrawal from treatment for other reasons or death for a maximum duration of 24 months.

lead_sponsor: Hoffmann-La Roche

location_country:
Finland
United Kingdom
Denmark
Netherlands
Switzerland
United States
Spain
France

location_facility: Helsinki University Central Hospital; Dept of Oncology

location_facility: Oxford University Hospitals NHS Trust - Churchill Hospital

location_facility: Institut Gustave Roussy; Departement Oncologie Medicale

location_facility: Clinica Universitaria de Navarra; Servicio de oncología

location_facility: Hospital Univ Vall d'Hebron; Servicio de Oncologia

location_facility: VU MEDISCH CENTRUM; Dept. of Medical Oncology


title: An Analysis of Historical Data on Hematological Remission and Survival Among Adult Patients With Relapsed / Refractory B-Precursor Acute Lymphoblastic Leukemia

summary: A retrospective analysis of historical data looking at hematological remission and survival in adult relapsed / refractory B-precursor acute lymphoblastic leukemia patients.

ReferenceCitation: Barlev A, Lin VW, Katz A, Hu K, Cong Z, Barber B. Estimating Long-Term Survival of Adults with Philadelphia Chromosome-Negative Relapsed/Refractory B-Precursor Acute Lymphoblastic Leukemia Treated with Blinatumomab Using Historical Data. Adv Ther. 2017 Jan;34(1):148-155. doi: 10.1007/s12325-016-0447-x. Epub 2016 Nov 21.

ReferenceCitation: Gokbuget N, Dombret H, Ribera JM, Fielding AK, Advani A, Bassan R, Chia V, Doubek M, Giebel S, Hoelzer D, Ifrah N, Katz A, Kelsh M, Martinelli G, Morgades M, O'Brien S, Rowe JM, Stieglmaier J, Wadleigh M, Kantarjian H. International reference analysis of outcomes in adults with B-precursor Ph-negative relapsed/refractory acute lymphoblastic leukemia. Haematologica. 2016 Dec;101(12):1524-1533. doi: 10.3324/haematol.2016.144311. Epub 2016 Sep 1.

endpoint_id: fhisxppmrqpajgzqkvft

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

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

repository_institution: University of California - eScholarship University of California

repositories_institution: University College London - UCL Discovery

publisher_dissemination: Ferrata Storti Foundation (Haematologica)

location_country:
Germany
Italy
Poland
United Kingdom
United States
Spain
Czechia
France


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

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

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

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

lead_sponsor: Pfizer

location_country:
Australia
United States
Spain
Canada
France

location_facility: Massachusetts General Hospital Mass General 2

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


title: A Phase 1b Multiple Ascending Dose Study to Evaluate the Safety, Pharmacokinetics and Pharmacodynamics of BMS-833923 (XL139) in Combination With Carboplatin and Etoposide Followed by BMS-833923 Alone in Subjects With Extensive-Stage Small Cell Lung Cancer

summary: The purpose of this study is to determine the maximum tolerated dose (MTD) of BMS-833923 administered in combination with carboplatin and etoposide followed by BMS-833923 alone in subjects with extensive-stage Small Cell Lung Cancer (SCLC).

lead_sponsor: Bristol-Myers Squibb

sponsor_collaborators:
Bristol-Myers Squibb
Exelixis

location_country:
Ireland
Australia
United States
Canada
France

location_facility:
Karmanos Cancer Institute
Local Institution


title: A Randomized, Phase II, Placebo-controlled Study of Ipatasertib (GDC-0068), an Inhibitor to Akt, in Combination With Fluoropyrimidine Plus Oxaliplatin in Patients With Locally Advanced or Metastatic Gastric or Gastroesophageal Junction Adenocarcinoma

summary: This multicenter, randomized, double-blind, placebo-controlled study will evaluate the efficacy of ipatasertib in combination with oxaliplatin, 5-fluorouracil, and leucovorin (modified FOLFOX6 [mFOLFOX6]) chemotherapy in participants with advanced or metastatic gastric or gastroesophageal junction (GEJ) cancer. Participants will be randomized to receive either ipatasertib or placebo orally daily on Days 1 to 7 of each 14-day cycle in combination with mFOLFOX6 on Day 1 of each cycle.

ReferenceCitation: Bang YJ, Kang YK, Ng M, Chung HC, Wainberg ZA, Gendreau S, Chan WY, Xu N, Maslyar D, Meng R, Chau I, Ajani JA. A phase II, randomised study of mFOLFOX6 with or without the Akt inhibitor ipatasertib in patients with locally advanced or metastatic gastric or gastroesophageal junction cancer. Eur J Cancer. 2019 Feb;108:17-24. doi: 10.1016/j.ejca.2018.11.017. Epub 2018 Dec 25.

lead_sponsor: Genentech, Inc.

location_country:
Germany
Italy
United Kingdom
Hong Kong
Singapore
United States
Korea, Republic of
Spain
Malaysia
Taiwan
France

location_facility: Mount Vernon Hospital; Centre For Cancer Treatment

location_facility: National Cancer Centre; Medical Oncology

location_facility: Univ of Calif, Los Angeles; Hematology/Oncology

location_facility: University of Texas M.D. Anderson Cancer Center

location_facility: Hospital Universiti Sains Malaysia [Neurology]

location_facility: Yonsei Uni College of Medicine, Severance Hospital; Internal Medicine Dept.

location_facility: IRCCS Istituto Nazionale Per La Ricerca Sul Cancro (IST); Oncologia Medica A

location_facility: Medizinische Hochschule Hannover; Zentrum Innere Medizin; Abt. Hämatologie u. Onkologie

location_facility: Chang Gung Medical Foundation - Linkou; Division of Hematology- Oncology

location_facility: Seoul National University Bundang Hospital

location_facility: University Malaya Medical Centre; Clinical Oncology Unit,

location_facility: Gachon Medical School Gil Medical Centre; Internal Medicine

location_facility: Queen Mary Hospital; Dept. of Clinical Oncology

location_facility: HOSPITAL DE MADRID NORTE SANCHINARRO- CENTRO INTEGRAL ONCOLOGICO CLARA CAMPAL; Servicio de Oncologia

location_facility: Universitatsklinik Heidelberg; Universitätshautklinik und Nationales Centrum für Tumorerkrankungen

location_facility: Seoul National University Hosp; Dept Internal Med Hem Onc

location_facility: National Taiwan Uni Hospital; Dept of Oncology

location_facility: IRCCS Istituto Oncologico Veneto (IOV); Oncologia Medica Seconda

location_facility: Hospital Clinico Universitario de Valencia

location_facility: Azienda Ospedaliero Universitaria Pisana; Uff. Sperim. Clin. U.O. Farmaceutica

location_facility: Charité-Universitätsm. Berlin; Med. Klinik mit Schwerpunkt Hämatologie, Onkologie und Tumorimmunolo.


title: A Prospective Randomized Clinical Trial on 90Yttrium Trans-arterial Radio-Embolization (TheraSphere®) vs. Standard of Care (Sorafenib) for the Treatment of Advanced Hepatocellular Carcinoma (HCC) With Portal Vein Thrombosis (PVT)

acronym: YES-P

summary: This is a two-arm, open-label, prospective, multi-center, randomized, active-controlled clinical trial to assess efficacy and safety of TheraSphere in comparison to standard of care therapy (sorafenib) in the treatment of participants with inoperable liver cancer and blockage of the portal vein.

lead_sponsor: Boston Scientific Corporation

sponsor_collaborators:
Boston Scientific Corporation
Biocompatibles UK Ltd

location_country:
Italy
United Kingdom
Belgium
United States
Spain
France

location_facility: University Hospitals Birmingham NHS Foundation Trust

location_facility: Azienda Ospedaliero-Universitaria di Bologna

location_facility: S.C Radiologia Interventistica, A.O.Ospedale Niguarda Ca Granda

location_facility: Hospital Universitario Puerta de Hierro, Gastroenterology & Hepatology Dept

location_facility: Azienda Ospedaliera Ospedali Riuniti "Villa Sofia-Cervello"

location_facility: Fondazione Istituto Nazionale Tumori di Milano

location_facility: Hospital Clínic Barcelona. IDIBAPS. CIBEREHD. Liver Unit

location_facility: UO Radiologia, Ospedali Riuniti di Bergamo

location_facility: Northwestern Medical Faculty Foundation, Div of Hematology/Oncology

location_facility: Hospital Universitari i Politecnic la Fe


summary:
Study of the safety and efficacy of AEB071 and EVEROLIMUS in patients with CD79-mutant or ABC subtype Diffuse Large B-Cell Lymphoma.

The trial did not progress into Phase II due to the suboptimal tolerability of the combination treatment of sotrastaurin and everolimus in the Phase Ib part of the study. There were no serious safety concerns associated with this combination.

location_facility: Washington University School of Medicine Dept of Oncology.

location_facility: Sarah Cannon Research Institute Dept of Onc

location_facility: Memorial Sloan Kettering Cancer Center Onc. Dept.

title: An Open-Label, Single-arm, Phase Ib/II Study of AEB071 (a Protein Kinase C Inhibitor) and Everolimus (mTOR Inhibitor) in Patients With CD79-mutant or ABC Subtype Diffuse Large B-Cell Lymphoma

location_country:
Germany
Italy
Hong Kong
Netherlands
United States
Korea, Republic of
Taiwan
France


lead_sponsor: Edimer Pharmaceuticals

summary: This Phase 2 first-in-neonate EDI200 study will enroll treatment-naïve, XLHED-affected male newborns in the first two weeks of life. All subjects will meet entry criteria including documentation of an Ectodysplasin (EDA) mutation associated with XLHED. Following Baseline evaluations, EDI200 dosing will be initiated between day-of-life 2 and 14, with each study subject receiving 2 doses/week for a total of 5 doses. The study will enroll subjects in two cohorts with subjects in cohort 1 dosed at 3 mg/kg/dose, associated with partial efficacy, and cohort 2 dosed at 10 mg/kg/dose where enhanced efficacy was demonstrated in the most relevant preclinical model. Given the challenge of identifying families where the subject is yet to be born, it is expected that cohort size and time for recruitment will be variable.

location_facility: Washington University School of Medicine

location_facility: Azienda Ospedaliera-Polo Universitario "Luigi Sacco"

title: A Phase 2 Open-label, Dose-escalation Study to Evaluate the Safety, Pharmacokinetics, Immunogenicity and Pharmacodynamics/Efficacy of EDI200, an EDA-A1 Replacement Protein, Administered to Male Infants With X-Linked Hypohidrotic Ectodermal Dysplasia (XLHED)

location_country:
Germany
Italy
United Kingdom
United States
France


title: A Phase 2, Open-Label, Multiple Dose Level, 12-Week Study to Evaluate the Safety, Tolerability, and Pharmacodynamics of AT1001 in Female Patients With Fabry Disease

summary: Study to evaluate the safety, tolerability, and pharmacodynamics of migalastat hydrochloride (HCl) (migalastat) in participants with Fabry disease.

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

repositories:
PubMed Central
UCL Discovery

repositories_concat: PubMed Central;UCL Discovery

ReferenceCitation: Giugliani R, Waldek S, Germain DP, Nicholls K, Bichet DG, Simosky JK, Bragat AC, Castelli JP, Benjamin ER, Boudes PF. A Phase 2 study of migalastat hydrochloride in females with Fabry disease: selection of population, safety and pharmacodynamic effects. Mol Genet Metab. 2013 May;109(1):86-92. doi: 10.1016/j.ymgme.2013.01.009. Epub 2013 Jan 26.

lead_sponsor: Amicus Therapeutics

location_country:
United Kingdom
Australia
Brazil
United States
Canada
France


title: A Phase 1 Study to Assess the Safety, Tolerability, Pharmacokinetics, and Biological Activity of SAR405838 in Patients With Advanced Cancer

summary:
Primary Objectives:

To determine safety and the maximum tolerated dose (MTD) of SAR405838 through the characterization of dose-limiting toxicities (DLTs).
To assess biological activities in patients with dedifferentiated liposarcoma during MTD cohort expansion.

Secondary Objectives:

Pharmacokinetic (PK) profile of SAR405838.
Biomarkers in association with SAR405838.
Anti-tumor activity in response to SAR405838.
Food effect on SAR405838 PK.
Compliance with SAR405838 treatment.
Cytochrome P450 3A4/5 (CYP3A4/5) activity.

ReferenceCitation: de Jonge M, de Weger VA, Dickson MA, Langenberg M, Le Cesne A, Wagner AJ, Hsu K, Zheng W, Mace S, Tuffal G, Thomas K, Schellens JH. A phase I study of SAR405838, a novel human double minute 2 (HDM2) antagonist, in patients with solid tumours. Eur J Cancer. 2017 May;76:144-151. doi: 10.1016/j.ejca.2017.02.005. Epub 2017 Mar 17.

lead_sponsor: Sanofi

location_country:
United States
Netherlands
France

location_facility:
Investigational Site Number 528002
Investigational Site Number 840101
Investigational Site Number 528003
Investigational Site Number 250001
Investigational Site Number 528001
Investigational Site Number 840002
Investigational Site Number 840001


title: CALM- 2 - Controlling and Lowering Blood Pressure With the MobiusHD™

summary: The objective of this study is to evaluate the safety and effectiveness of the MobiusHD System in a prospective, randomized, double-blind, sham-controlled multi-center pivotal study.

ReferenceCitation: Calhoun DA, Schiffrin EL, Flack JM. Resistant Hypertension: An Update. Am J Hypertens. 2019 Jan 1;32(1):1-3. doi: 10.1093/ajh/hpy156. No abstract available.

evidence: open (via free article)

publisher_dissemination: Oxford University Press

lead_sponsor: Vascular Dynamics, Inc.

location_country:
United States
France

location_facility: The Carl & Edyth Lindner Center for Research & Education at The Christ Hospital


title: A Multi-centre, Randomised, Double-blind, Parallel Group, Placebo Controlled Trial to Evaluate the Efficacy and Safety of Activated Recombinant Factor VII (rFVIIa/NovoSeven®/ Niastase®) in the Treatment of Bleeding in Severely Injured Trauma Subjects

summary: This trial is conducted in Africa, Asia, Europe, Oceania and North America. The aim of this trial is to evaluate the efficacy of activated recombinant human factor VII given in conjunction with standard therapy in the treatment of massive bleeding in subjects with severe blunt and/or penetrating trauma injury.

ReferenceCitation: Rossaint R, Christensen MC, Choong PI, Boffard KD, Riou B, Rizoli S, Kluger Y, Lefering R, Morris S; NovoSeven(R) Trauma Study Group. Cost-Effectiveness of Recombinant Activated Factor VII as Adjunctive Therapy for Bleeding Control in Severely Injured Trauma Patients in Germany. Eur J Trauma Emerg Surg. 2007 Oct;33(5):528-38. doi: 10.1007/s00068-007-6210-x. Epub 2007 Jul 20.

ReferenceCitation: Boffard KD, Riou B, Warren B, Choong PI, Rizoli S, Rossaint R, Axelsen M, Kluger Y; NovoSeven Trauma Study Group. Recombinant factor VIIa as adjunctive therapy for bleeding control in severely injured trauma patients: two parallel randomized, placebo-controlled, double-blind clinical trials. J Trauma. 2005 Jul;59(1):8-15; discussion 15-8. doi: 10.1097/01.ta.0000171453.37949.b7.

ReferenceCitation: Rizoli SB, Boffard KD, Riou B, Warren B, Iau P, Kluger Y, Rossaint R, Tillinger M; NovoSeven Trauma Study Group. Recombinant activated factor VII as an adjunctive therapy for bleeding control in severe trauma patients with coagulopathy: subgroup analysis from two randomized trials. Crit Care. 2006;10(6):R178. doi: 10.1186/cc5133.

ReferenceCitation: Morris S, Ridley S, Munro V, Christensen MC. Cost effectiveness of recombinant activated factor VII for the control of bleeding in patients with severe blunt trauma injuries in the United Kingdom. Anaesthesia. 2007 Jan;62(1):43-52. doi: 10.1111/j.1365-2044.2006.04896.x.

ReferenceCitation: Champion HR, Fingerhut A, Escobar MA, Weiskopf RB. The role of data and safety monitoring in acute trauma resuscitation research. J Am Coll Surg. 2007 Jan;204(1):73-83. doi: 10.1016/j.jamcollsurg.2006.10.024.

ReferenceCitation: Klitgaard T, Tabanera y Palacios R, Boffard KD, Iau PT, Warren B, Rizoli S, Rossaint R, Kluger Y, Riou B; NovoSeven Trauma Study Group. Pharmacokinetics of recombinant activated factor VII in trauma patients with severe bleeding. Crit Care. 2006;10(4):R104. doi: 10.1186/cc4977.

ReferenceCitation: Kluger Y, Riou B, Rossaint R, Rizoli SB, Boffard KD, Choong PI, Warren B, Tillinger M. Safety of rFVIIa in hemodynamically unstable polytrauma patients with traumatic brain injury: post hoc analysis of 30 patients from a prospective, randomized, placebo-controlled, double-blind clinical trial. Crit Care. 2007;11(4):R85. doi: 10.1186/cc6092.

ReferenceCitation: Hsia CC, Chin-Yee IH, McAlister VC. Use of recombinant activated factor VII in patients without hemophilia: a meta-analysis of randomized control trials. Ann Surg. 2008 Jul;248(1):61-8. doi: 10.1097/SLA.0b013e318176c4ec.

ReferenceCitation: Levy JH, Fingerhut A, Brott T, Langbakke IH, Erhardtsen E, Porte RJ. Recombinant factor VIIa in patients with coagulopathy secondary to anticoagulant therapy, cirrhosis, or severe traumatic injury: review of safety profile. Transfusion. 2006 Jun;46(6):919-33. doi: 10.1111/j.1537-2995.2006.00824.x.

lead_sponsor: Novo Nordisk A/S

location_country:
Germany
Israel
Singapore
United Kingdom
Australia
Austria
South Africa
Canada
France


type: Other Grant/Funding Number

title: Canadian Study on the Endovascular Treatment of Unruptured Intracranial Aneurysms Versus Surgical Treatment. A Randomized Comparison of Clinical and Angiographic Results of Intracranial Aneurysms

acronym: CURES

summary:
Purpose:

Phase 1: (Pilot Phase)

To compare the treatment efficacy of surgical clipping and endovascular coiling for unruptured intracranial aneurysms.

To obtain better estimates of morbidity and mortality related to a surgical or endovascular treatment strategy at one year within the context of an RCT.

To show that an RCT comparing the morbidity and mortality of a surgical management strategy to an endovascular management strategy is feasible.

Phase 2:

To compare the results of surgical and endovascular management strategies, in terms of:

Overall mortality and morbidity at 1 and 5 years.
The clinical efficacy and safety of a surgical or endovascular management strategy at 1 and 5 years

Hypotheses: Phase 1 Hypotheses:

Surgical clipping of intradural, saccular, unruptured intracranial aneurysms is superior to endovascular management in terms of a lesser number of patients experiencing treatment failure.
An RCT comparing the clinical outcomes of a surgical versus endovascular management strategy is feasible.

Phase 1 Primary End-points:

• Treatment failure, hereby defined as having occurred when either: the intended initial modality (surgical or endovascular) fails to occlude the aneurysm, a "major" (saccular) angiographic aneurysm recurrence is found, or an intracranial hemorrhagic event occurs during the 1-year follow-up period.

Phase 1 Secondary End-points:

Overall morbidity and mortality at one year.
Occurrence of morbidity (mRS >2) or mortality following treatment.
Occurrence of failure of aneurysm occlusion using the initial intended treatment modality.
Occurrence of a "major" (saccular) angiographic aneurysm recurrence.
Occurrence of an intracranial hemorrhage following treatment.
Peri-treatment hospitalization lasting more than 5 days
Discharge following treatment to a location other than home

Treatment:

Trial feasibility, or the capacity for patient recruitment, would require enrollment of at least 8 patients per actively recruiting center per year.

Phase 2 Hypotheses:

It may be too early to explicitly define the primary hypothesis of Phase 2, however, the intent of Phase 2 can be expressed as:

One management strategy is superior to the other in terms of clinical outcome at five years.
One management strategy is superior to the other in terms of clinical efficacy at five years.

ReferenceCitation: Naggara O, Darsaut T, Trystram D, Tselikas L, Raymond J. Unruptured intracranial aneurysms: why we must not perpetuate the impasse for another 25 years. Lancet Neurol. 2014 Jun;13(6):537-8. doi: 10.1016/S1474-4422(14)70091-2. No abstract available.

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

collaborators: Centre de Recherche du Centre Hospitalier de l'Université de Montréal

location_country: Canada Belgium France

location_facility: Centre hospitalier de l'Université de Montréal - CHUM

location_facility: Centre hospitalier universitaire de Liège


title: An Open-label, Single Arm, Phase II Trial to Investigate the Safety and Efficacy of Sym004 in Patients With Recurrent and/or Metastatic Squamous Cell Carcinoma of the Head and Neck (SCCHN) Who Have Failed Anti-EGFR Monoclonal Antibody-based Therapy

summary:
The trial is designed as a multi-center, open label Phase 2 trial that investigates the efficacy and safety of Sym004 in subjects with squamous cell cancer of the head and neck (SCCHN). Subjects included must have responded to previous anti-epidermal growth factor receptor (anti-EGFR) monoclonal antibody-based therapy and subsequently become resistant to that therapy. It is believed that Sym004 has the potential to induce tumor responses and provide a superior treatment option to subjects with advanced SCCHN.

Symphogen was the sponsor for planning/conducting and reporting results for this trial.

ReferenceCitation: Machiels JP, Specenier P, Krauss J, Dietz A, Kaminsky MC, Lalami Y, Henke M, Keilholz U, Knecht R, Skartved NJ, Horak ID, Pamperin P, Braun S, Gauler TC. A proof of concept trial of the anti-EGFR antibody mixture Sym004 in patients with squamous cell carcinoma of the head and neck. Cancer Chemother Pharmacol. 2015 Jul;76(1):13-20. doi: 10.1007/s00280-015-2761-4. Epub 2015 May 8.

lead_sponsor: Symphogen A/S

location_country: Germany Belgium France

location_facility: 4907; University Hospital Heidelberg; Nationales Centrum für Tumorerkrankungen (NCT)

location_facility: 4906; University Medical Center Hamburg Eppendorf; Department of Otorhinolaryngology and Head and Neck Surgery

location_facility: 3203; Antwerp University Hospital; Department of Medical Oncology

location_facility: 4905; Charité Campus Benjamin Franklin; Hematology, Oncology and Transfusion Medicine

location_facility: 3202; Jules Bordet Institute; Clinique d'Oncologie Médicale

location_facility: 3303; Centre Alexis Vautrin; Département d'Oncologie Médicale

location_facility: 3201; Cliniques Universitaires St-Luc; Centre du Cancer


summary: This study is conducted in 3 stages. Stage 1 is an open-label, dose-escalation assessment of the safety of AGN-150998 administered as a single intravitreal injection to patients with advanced exudative Age-related Macular Degeneration (AMD). Stage 2 and Stage 3 are randomized, double-masked, comparisons of the safety and treatment effects on retinal edema and best-corrected visual acuity (BCVA) of AGN-150998 and ranibizumab in treatment-naive patients with exudative AMD. Study medication is administered as needed in Stage 2 and with a fixed-dosing schedule in Stage 3. The study objectives are (1) to identify the highest tolerated dose of AGN-150998, (2) to assess the safety and duration of treatment effects on retinal edema and BCVA, and (3) to characterize the systemic pharmacokinetic profile of AGN-150998.

ReferenceCitation: Callanan D, Kunimoto D, Maturi RK, Patel SS, Staurenghi G, Wolf S, Cheetham JK, Hohman TC, Kim K, Lopez FJ, Schneider S. Double-Masked, Randomized, Phase 2 Evaluation of Abicipar Pegol (an Anti-VEGF DARPin Therapeutic) in Neovascular Age-Related Macular Degeneration. J Ocul Pharmacol Ther. 2018 Dec;34(10):700-709. doi: 10.1089/jop.2018.0062. Epub 2018 Nov 9.

lead_sponsor: Allergan

location_country:
Germany
Italy
Israel
Australia
Switzerland
Austria
United States
France


title: A Re-Survey Study of Patients With MPS VI (Maroteaux-Lamy Syndrome) Who Previously Participated in ASB-00-02

summary: There is limited information on the long-term effects of treating patients with MPS VI with Naglazyme® and limited data on the natural history of treated and untreated MPS VI patients. The Re-survey Study ASB-00-03 will assist in understanding the effects of long-term Naglazyme treatment and the natural history of the MPS VI patient population.

ReferenceCitation: Giugliani R, Lampe C, Guffon N, Ketteridge D, Leao-Teles E, Wraith JE, Jones SA, Piscia-Nichols C, Lin P, Quartel A, Harmatz P. Natural history and galsulfase treatment in mucopolysaccharidosis VI (MPS VI, Maroteaux-Lamy syndrome)--10-year follow-up of patients who previously participated in an MPS VI Survey Study. Am J Med Genet A. 2014 Aug;164A(8):1953-64. doi: 10.1002/ajmg.a.36584. Epub 2014 Apr 24.

evidence: open (via page says Open Access)

license: unspecified-oa

license_normalized: unspecified-oa

repositories_concat: PubMed Central;escholarship.org

repositories_institution: University of California - eScholarship University of California

publisher_dissemination: Wiley

lead_sponsor: BioMarin Pharmaceutical

location_country:
Germany
Portugal
United Kingdom
Australia
Brazil
United States
France

location_facility: Manchester Academic Health Sciences Centre


lead_sponsor: Novartis Pharmaceuticals

summary: This trial will assess the safety, efficacy and tolerability of ACZ885 in patients aged 4 years and younger with cryopyrin associated periodic syndromes (CAPS)

ReferenceCitation: Brogan PA, Hofer M, Kuemmerle-Deschner JB, Kone-Paut I, Roesler J, Kallinich T, Horneff G, Calvo Penades I, Sevilla-Perez B, Goffin L, Lauwerys BR, Lachmann HJ, Uziel Y, Wei X, Laxer RM. Rapid and Sustained Long-Term Efficacy and Safety of Canakinumab in Patients With Cryopyrin-Associated Periodic Syndrome Ages Five Years and Younger. Arthritis Rheumatol. 2019 Nov;71(11):1955-1963. doi: 10.1002/art.41004. Epub 2019 Sep 9.

repository_institution: Université de Lausanne - SERVAL

title: A One-year Open-label, Multicenter Trial to Assess Efficacy, Safety and Tolerability of Canakinumab (ACZ885) and the Efficacy and Safety of Childhood Vaccinations in Patients Aged 4 Years or Younger With Cryopyrin Associated Periodic Syndromes (CAPS)

location_country:
Germany
United Kingdom
Switzerland
Belgium
Spain
Canada
France


title: The Effectiveness of Single or Repeat Zoledronate Infusion Versus Oral Alendronate in Consolidating the Bone Accrual Achieved With Denosumab: a Study Organised by the European Calcified Tissue Society

acronym: EURODEC

summary: A 24-month prospective, open-label, randomized, multicenter, multinational, non-inferiority pragmatic clinical trial evaluating zoledronate single or double infusion versus oral alendronate following denosumab

lead_sponsor: 424 General Military Hospital

location_country: Germany Italy Greece France

location_facility: Fondazione IRCCS Cà Granda-Ospedale Maggiore Policlinico

location_facility: Department of Medicine III and Center for Healthy Aging, Technische Universität Dresden

location_facility: First Department of Propaedeutic and Internal Medicine, Medical School, National and Kapodistrian University of Athens

location_facility: Department of Medicine, Surgery and Neurosciences, University of Siena

location_facility: University-Hospital S. Maria della Misericordia

location_facility: 251 Hellenic Airforce and VA General Hospital


type: Registry Identifier

title: Standard Dosed Alectinib Versus Therapeutic Drug Monitoring Guided Alectinib Dosing

acronym: ADAPT ALEC

summary: The ADAPT ALEC randomized controlled trial (RCT) is performed in patients with Anaplastic Lymphoma Kinase (ALK) positive non-small cell lung cancer (NSCLC). The RCT will compare the use of Therapeutic Drug Monitoring (TDM) and dose increases if alectinib 35 ng/Ml (arm A) with standard of care (arm B).

ipd_sharing_description: Individual participant data that underlie the results will only be shared upon reasonable request from the Principal Investigator at the UMCG, who will discuss the request with the ADAPT ALEC study team.

lead_sponsor: University Medical Center Groningen

collaborators: Academisch Medisch Centrum - Universiteit van Amsterdam (AMC-UvA)

location_country: Netherlands France

location_facility:
Amsterdam University Medical Center
Erasmus Medical Center
Gustave Roussy
University Medical Center Groningen
Leiden University Medical Center
Maastricht University Medical Center +
Radboud University Medical Center
The Netherlands Cancer Institute