title: Antiplatelet Removal and HemocompatIbility EventS With the HeartMate 3 Pump IDE Study

summary: Prospective, randomized, double-blinded, placebo-controlled clinical investigation of advanced heart failure patients treated with the HM3 with two different antithrombotic regimens: vitamin K antagonist with aspirin versus vitamin K antagonist with placebo

primary_purpose: Supportive Care

ReferenceCitation: Mehra MR, Crandall DL, Gustafsson F, Jorde UP, Katz JN, Netuka I, Uriel N, Connors JM, Sood P, Heatley G, Pagani FD. Aspirin and left ventricular assist devices: rationale and design for the international randomized, placebo-controlled, non-inferiority ARIES HM3 trial. Eur J Heart Fail. 2021 Jul;23(7):1226-1237. doi: 10.1002/ejhf.2275. Epub 2021 Jul 1. Review.

name: Department of Medicine Brigham and Women's Hospital and Harvard Medical School  Boston MA USA

family: Mehra

full_name: Mandeep R. Mehra

given: Mandeep R.

name: Clinical Affairs Heart Failure, Abbott  Chicago IL USA

family: Crandall

full_name: Daniel L. Crandall

given: Daniel L.

name: Cardiology, Rigshospitalet  Copenhagen Denmark

family: Gustafsson

full_name: Finn Gustafsson

given: Finn

name: Einstein Center for Heart and Vascular Care, Montefiore Medical Center  Bronx NY USA

family: Jorde

full_name: Ulrich P. Jorde

given: Ulrich P.

name: Division of Cardiology Duke University  Durham NC USA

family: Katz

full_name: Jason N. Katz

given: Jason N.

name: Department of Cardiovascular Surgery, IKEM  Prague Czech Republic

family: Netuka

full_name: Ivan Netuka

given: Ivan

name: Heart Failure, Heart Transplant & Mechanical Circulatory Support, Columbia University Medical Center  New York NY USA

family: Uriel

full_name: Nir Uriel

given: Nir

name: Division of Hematology Brigham and Women's Hospital  Boston MA USA

family: Connors

full_name: Jean M. Connors

given: Jean M.

family: Sood

full_name: Poornima Sood

given: Poornima

name: Global Biometrics, Abbott  Chicago IL USA

family: Heatley

full_name: Gerald Heatley

given: Gerald

name: Department of Cardiac Surgery University of Michigan  Ann Arbor MI USA

family: Pagani

full_name: Francis D. Pagani

given: Francis D.

journal_name: European Journal of Heart Failure

oa_colors: green hybrid

evidence: open (via crossref license)

license: cc-by-nc

license_normalized: cc-by-nc

publisher: Wiley

publisher_dissemination: Wiley

publisher_group: Wiley

publisher_normalized: Wiley

title: Aspirin and left ventricular assist devices: rationale and design for the international randomized, placebo‐controlled, non‐inferiority ARIES HM3 trial

ipd_sharing_description: To be determined

lead_sponsor: Abbott Medical Devices

location_country:
Austria
France
Czechia
Australia
Kazakhstan
United States

location_facility: National Research Center for Cardiac Surgery

location_facility: California Pacific Medical Center - Van Ness Campus

location_facility: University of Minnesota Medical Center Fairview

location_facility: Montefiore Medical Center - Moses Division

location_facility: New York-Presbyterian/Columbia University Medical Center


intervention_type: Drug

status: Completed

study_start_date_type: Actual

study_type: Interventional

ipd_sharing_description: Data from this trial may be requested by qualified researchers six months after product approval in the US and/or Europe, or global development is discontinued, and 18 months after trial completion. Investigators may request access to anonymized individual patient-level data and redacted trial documents which may include: analysis-ready datasets, study protocol, annotated case report form, statistical analysis plan, dataset specifications, and clinical study report. Prior to use of the data, proposals need to be approved by an independent review panel at www.Vivli.org and a signed data sharing agreement will need to be executed. All documents are available in English only, for a prespecified time, typically 12 months, on a password protected portal. This plan may change if the risk of re-identifying trial participants is determined to be too high after the trial is completed; in this case and to protect participants, individual patient-level data would not be made available.

lead_sponsor: UCB Biopharma SRL

enrollment_type: Actual

primary_purpose: Treatment

location_country:
Netherlands
Germany
France
Belgium
Spain
Denmark
United States
United Kingdom

ipd_sharing: Yes

study_completion_date_type: Actual

type: EudraCT Number

source: euctr

title: An Open-Label Extension Study to Investigate the Long-Term Safety, Tolerability, and Efficacy of Rozanolixizumab in Subjects With Chronic Inflammatory Demyelinating Polyradiculoneuropathy (CIDP)

summary: The purpose of the study is to assess long-term safety and tolerability of weekly doses of rozanolixizumab in subjects with chronic inflammatory demyelinating polyradiculoneuropathy (CIDP).

lead_sponsor_type: industriel

status_simplified: Completed


intervention_type: Biological

status: Enrolling by invitation

lead_sponsor: Novartis Gene Therapies

enrollment_type: Anticipated

primary_purpose: Prevention

location_country:
Italy
United States
France
Japan
Belgium
Australia
Canada
United Kingdom
Taiwan

acronym: not applicable

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

location_facility: Spectrum Health Hospitals Helen DeVos Children's Hospital

location_facility: Children's Hospital of Eastern Ontario Research Institute

location_facility: Washington Unviersity School of Medicine in Saint Louis

location_facility: Fondazione Policlinico Universitario Agostino Gemelli

location_facility: John Hopkins Hospital - David M. Rubenstein Child Health Building

location_facility: Great Ormond Street Hospital for Children NHS Foundation Trust

location_facility: Centre de Référence des Maladies Neuromusculaires

location_facility: Children's Hospital of The King's Daughters

location_facility: Tokyo Women's Medical University Hospital

location_facility: Children's Health Specialty Center Dallas Campus

location_facility: The Newcastle Upon Tyne Hospitals NHS Foundation Trust

study_completion_date_type: Anticipated

type: Other Identifier

title: A Long-term Follow-up Study of Patients in the Clinical Trials for Spinal Muscular Atrophy Receiving AVXS-101

summary: This is a long-term follow-up safety and efficacy study of participants in clinical trials for spinal muscular atrophy (SMA) who were treated with onasemnogene abeparvovec-xioi. Participants will roll over from their respective previous (parent) study into this long-term study for continuous monitoring of safety as well as monitoring of continued efficacy and durability of response to onasemnogene abeparvovec-xioi treatment.

status_simplified: Unknown


title: A Phase 2, Multicenter, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Safety, Tolerability, and Efficacy of Zilucoplan in Subjects With Immune-Mediated Necrotizing Myopathy

summary: The purpose of the study is to evaluate the safety and efficacy of zilucoplan in patients with Immune-Mediated Necrotizing Myopathy (IMNM). Subjects will be randomized in a 1:1 ratio to receive daily SC doses of 0.3 mg/kg zilucoplan or matching placebo for 8 weeks.

status: Terminated

design_allocation: Randomized

lead_sponsor: Ra Pharmaceuticals

location_country:
United States
United Kingdom
France
Netherlands

location_facility: The University of Texas Health Science Center at Houston

location_facility: Salford Royal NHS Barnes Clinical Research Facility

location_facility: University of Florida Health Jacksonville

location_facility: Washington University School of Medicine in Saint Louis

location_facility: University College London Hospitals NHS Foundation Trust

intervention_type: Other


title: AURORAX-0087A: Glycosaminoglycan Scores for Surveillance of Recurrence in Leibovich Points ≥5 Non-metastatic Clear Cell Renal Cell Carcinoma

summary: AUR87A is an observational prospective multicenter diagnostics test cohort study for detection of renal cell carcinoma recurrence as determined by the reference standard, which is imaging using computed tomography (CT) of the chest and abdomen at defined intervals after primary surgery.

status: Recruiting

study_type: Observational

time_perspective: Prospective

ipd_sharing: Undecided

ipd_sharing_description: Unidentified clinical and biochemical data will become available to the research community along with the publication of a scientific article related to the present investigation.

lead_sponsor: Elypta

location_country:
Italy
Sweden
Spain
Finland
France
Portugal
Denmark
United States
United Kingdom
Canada

location_facility:
Helsinki University Central Hospital
Hôpital Henri Mondor
Western General Hospital
Frimley Park Hospital
Memorial Sloan Kettering Cancer Center
Hospital Universitario Cabueñes
San Raffaele Hospital
Prostate Cancer Centre
Emory University School of Medicine
Addenbrooke's Hospital
Guys & St Thomas Hospital
Royal Berkshire Hospital
MD Anderson Cancer Center
Royal Free Hospital
Careggi University Hospital
Hospital da Luz Coimbra
Aarhus University Hospital
Sahlgrenska University Hospital
Istituto Nazionale Tumori Regina Elena
Odense University Hospital
AOU Integrata Verona
Salford Royal NHS Foundation Trust
AOU San Orsola Malpighi
Zealand University Hospital
Norfolk & Norwich University Hospital
AOU San Luigi Gonzaga

intervention_type: Diagnostic Test

status_simplified: Ongoing


title: A Phase 2, Randomized, Double-Blind, Placebo-Controlled, 48-Week, Parallel-Group Study of the Efficacy and Safety of Losmapimod in Treating Subjects With Facioscapulohumeral Muscular Dystrophy (FSHD)

acronym: FSHD

summary: This is a study to evaluate the safety and efficacy of Losmapimod in treating patients with Facioscapulohumeral Muscular Dystrophy (FSHD) over 48 weeks.

ReferenceType: background

ReferenceCitation: Barbour AM, Sarov-Blat L, Cai G, Fossler MJ, Sprecher DL, Graggaber J, McGeoch AT, Maison J, Cheriyan J. Safety, tolerability, pharmacokinetics and pharmacodynamics of losmapimod following a single intravenous or oral dose in healthy volunteers. Br J Clin Pharmacol. 2013 Jul;76(1):99-106. doi: 10.1111/bcp.12063.

ReferenceCitation: Cheriyan J, Webb AJ, Sarov-Blat L, Elkhawad M, Wallace SM, Mäki-Petäjä KM, Collier DJ, Morgan J, Fang Z, Willette RN, Lepore JJ, Cockcroft JR, Sprecher DL, Wilkinson IB. Inhibition of p38 mitogen-activated protein kinase improves nitric oxide-mediated vasodilatation and reduces inflammation in hypercholesterolemia. Circulation. 2011 Feb 8;123(5):515-23. doi: 10.1161/CIRCULATIONAHA.110.971986. Epub 2011 Jan 24.

ReferenceCitation: de Greef JC, Lemmers RJ, van Engelen BG, Sacconi S, Venance SL, Frants RR, Tawil R, van der Maarel SM. Common epigenetic changes of D4Z4 in contraction-dependent and contraction-independent FSHD. Hum Mutat. 2009 Oct;30(10):1449-59. doi: 10.1002/humu.21091.

ReferenceCitation: Han JJ, Kurillo G, Abresch RT, de Bie E, Nicorici A, Bajcsy R. Reachable workspace in facioscapulohumeral muscular dystrophy (FSHD) by Kinect. Muscle Nerve. 2015 Feb;51(2):168-75. doi: 10.1002/mus.24287. Epub 2014 Nov 19.

ReferenceCitation: Jagannathan S, Shadle SC, Resnick R, Snider L, Tawil RN, van der Maarel SM, Bradley RK, Tapscott SJ. Model systems of DUX4 expression recapitulate the transcriptional profile of FSHD cells. Hum Mol Genet. 2016 Oct 15;25(20):4419-4431. doi: 10.1093/hmg/ddw271.

ReferenceCitation: Statland JM, Tawil R. Risk of functional impairment in Facioscapulohumeral muscular dystrophy. Muscle Nerve. 2014 Apr;49(4):520-7. doi: 10.1002/mus.23949. Epub 2014 Feb 10.

ReferenceCitation: Wang LH, Friedman SD, Shaw D, Snider L, Wong CJ, Budech CB, Poliachik SL, Gove NE, Lewis LM, Campbell AE, Lemmers RJFL, Maarel SM, Tapscott SJ, Tawil RN. MRI-informed muscle biopsies correlate MRI with pathology and DUX4 target gene expression in FSHD. Hum Mol Genet. 2019 Feb 1;28(3):476-486. doi: 10.1093/hmg/ddy364.

ipd_sharing: No

lead_sponsor: Fulcrum Therapeutics

location_country:
United States
Canada
France
Spain

location_facility: University of California Los Angeles (UCLA)

location_facility: University of Massachusetts Memorial Medical Center

location_facility: Montreal Neurological Institute and Hospital

location_facility: Washington University School of Medicine


title: An Open-label, First-in-human, Single Agent, Dose-escalation and Expansion Study for the Evaluation of Safety, Pharmacokinetics, Pharmacodynamics and Anti-tumor Activity of SAR442085 in Patients With Relapsed or Refractory Multiple Myeloma (RRMM)

summary:
Primary Objectives:

Dose Escalation Part A: To determine the maximum tolerated dose (MTD) of SAR442085 administered as a single agent in patients with relapsed or refractory multiple myeloma (RRMM), and determine the recommended Phase 2 dose (RP2D) for the subsequent Expansion Part B
Dose Expansion Part B: To assess the antitumor activity of single agent of SAR442085 at the RP2D in patients with RRMM

Secondary Objectives:

To characterize the safety profile of SAR442085
To characterize the pharmacokinetics (PK) profile of SAR442085 when administered as a single agent
To evaluate the potential immunogenicity of SAR442085
To assess preliminary evidence of antitumor activity in the Dose Escalation Part A

design_allocation: Non-Randomized

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://www.clinicalstudydatarequest.com/

lead_sponsor: Sanofi

location_country:
France
Czechia
Greece
Spain
United States
Taiwan

location_facility:
Investigational Site Number 2500001
Investigational Site Number 7240001
Investigational Site Number 2030001
Investigational Site Number 8400003
Investigational Site Number 1580002
Investigational Site Number 2030002
Investigational Site Number 8400006
Investigational Site Number 8400005
Investigational Site Number 8400004
Investigational Site Number 8400001
Investigational Site Number 3000001
Investigational Site Number 8400002
Investigational Site Number 7240002
Investigational Site Number 2030003


title: Randomized Controlled Clinical Study to Assess the Benefit of Non-invasive PGT-A, by the Analysis of Spent Blastocyst Media, as a Tool for Embryo Prioritization in Infertile Patients Undergoing Assisted Reproduction.

summary:
Chromosomal aneuploidies are linked with spontaneous miscarriages and abnormal offspring in human pregnancies. In addition, some types of aneuploidy are reported to prevent implantation. Thus, there is a need to identify the embryos with highest implantation potential on in vitro fertilization (IVF) programs.

Since embryo morphology and kinetics have a weak association with embryo ploidy, trophectoderm biopsy plus Next-Generation Sequencing (NGS) is becoming a very popular approach to determine the embryo chromosomal status. This technique is called Preimplantation Genetic Testing for Aneuploidy (PGT-A). Although shown to be efficient, it is invasive for the embryo, requires specific technical skills and it remains expensive. Therefore, the development of a non-invasive, rapid and cheaper method for assessing embryo ploidy status would represent a progress in the field of IVF.

The non-invasive approach has been explored by some groups that analyzed the Spent Blastocyst Medium (SBM) where the embryo was incubated up to the time of transfer or freezing. In daily routine, this media is discarded after finishing the culture of the embryo. Importantly, though, this media reportedly contains traces of embryonic cell-free DNA (cfDNA) that can represent the genetic load of the embryo.

On the basis of that, the hypothesis of this study is that embryo prioritization according to the analysis of the embryonic cfDNA in the SBM could improve ongoing pregnancy rate in 10 percentual points compared to standard blastocyst transfer based on morphology.

primary_purpose: Diagnostic

lead_sponsor: Igenomix

location_country:
Italy
Brazil
France
Argentina
Spain
Turkey
United States

location_facility: Nilo Frantz - Centro de Reprodução Humana

location_facility: Società Italiana Studi di Medicina della Riproduzione (S.I.S.M.e.R.)

location_facility: Fertility Associates of California (FSAC)

location_facility: Crecer: Centro de Reproducción y Genética Humana

location_facility: South Florida Institute for Reproductive Medicine (IVFMD)


title: Long-term Extension Safety and Efficacy Study of SAR442168 in Participants With Relapsing Multiple Sclerosis

summary:
Primary Objective:

To determine the long-term safety and tolerability of SAR442168 in RMS participants

Secondary Objective:

To evaluate efficacy of SAR442168 on disease activity, assessed by clinical and imaging methods

status: Active, not recruiting

location_country:
Netherlands
France
Czechia
Russian Federation
Spain
Estonia
United States
Canada
Ukraine

location_facility:
Investigational Site Number 8400003
Investigational Site Number 1240001
Investigational Site Number 8400008
Investigational Site Number 8040003
Investigational Site Number 2030002
Investigational Site Number 8400009
Investigational Site Number 2030004
Investigational Site Number 8400001
Investigational Site Number 7240003
Investigational Site Number 2030007
Investigational Site Number 2030003
Investigational Site Number 5280001
Investigational Site Number 2030001
Investigational Site Number 6430007
Investigational Site Number 2030005
Investigational Site Number 6430003
Investigational Site Number 6430001
Investigational Site Number 8040007
Investigational Site Number 6430006
Investigational Site Number 2500004
Investigational Site Number 8040002
Investigational Site Number 8400006
Investigational Site Number 7240004
Investigational Site Number 7240005
Investigational Site Number 8040009
Investigational Site Number 8040005
Investigational Site Number 8400002
Investigational Site Number 2030006
Investigational Site Number 1240003
Investigational Site Number 7240001
Investigational Site Number 6430005
Investigational Site Number 2330001
Investigational Site Number 8040006
Investigational Site Number 8400005
Investigational Site Number 8040001
Investigational Site Number 7240002
Investigational Site Number 8400007


title: Establishing Biomarkers and Clinical Endpoints in Myotonic Dystrophy Type 1 (END-DM1)

summary: Building on previous work of the Myotonic Dystrophy Clinical Research Network (DMCRN), the present study seeks to overcome insufficient data on natural history; lack of reliable biomarkers; and incomplete characterization and limited biological understanding of the phenotypic heterogeneity of Myotonic Dystrophy 1 by examining strategies to improve the reliability by making further refinements in our sample collection and analysis procedures by developing strategies for managing patient heterogeneity going forward.

time_perspective: Other

ipd_sharing_description: Aggregated and deidentified data will be shared with qualified investigators upon majority approval of the DMCRN investigators.

lead_sponsor: Virginia Commonwealth University

location_country:
Italy
United States
Netherlands
France
Germany
United Kingdom

location_facility: Neuromuscular Reference Center Institute of Myology

lead_sponsor_type: academique


title: Treatment of Cardiovascular Disease With Low Dose Rivaroxaban in Advanced Chronic Kidney Disease

acronym: TRACK

summary:
The TRACK trial is an investigator-initiated, multicentre, prospective, randomised, double-blind, placebo-controlled trial. TRACK is a global trial and will be conducted in renal units that provide comprehensive CKD care. Approximately 2000 participants will be recruited.

The TRACK trial will assess a strategy of administering low dose rivaroxaban to reduce the risk of major adverse cardiac event (MACE) in people with Chronic Kidney Disease (CKD) stages 4 or 5 or dialysis-dependent End Stage Kidney Disease (ESKD), and elevated cardiovascular (CV) risk, whilst maintaining an acceptable bleeding risk.

ipd_sharing_description:
Trial data will be disseminated in the form of a publication to a relevant clinical journal and presentation at appropriate scientific conferences.

Individual participant data that underlie the results reported, after de-identification (text, tables, figures, and appendices), may be shared with Investigators whose proposed use of the data has been approved by an independent review committee ("learned intermediary") identified for this purpose.

lead_sponsor: The George Institute

location_country: India France Malaysia Singapore Australia Taiwan

location_facility: Kaohsiung Medical University Chung-Ho Memorial Hospital

location_facility: Kasturba Medical College and Hospital, Manipal

location_facility: Centre Hospitalier Régional Universitaire de Nancy

location_facility: KG Hospital, K.Govindaswamy Naidu Medical Trust

location_facility: Nizam's Institute of Medical Sciences, Hyderabad

location_facility: Chang Gung Memorial Hospital, Linkou Medical Center

location_facility: Institute of Post-Graduate Medical Education and Research

location_facility: All India Institute Of Medical Sciences, Raipur

location_facility: Postgraduate Institute of Medical Education and Research, Chandigarh


title: A Phase 1, Open-label, Dose-escalation and Expansion Study of MEDI1191 Administered Intratumorally as Monotherapy and in Combination With Durvalumab in Subjects With Advanced Solid Tumors

summary: To evaluate MEDI1191 administered intratumorally in sequential and concurrent combination with intravenous durvalumab in patients with solid tumors.

ipd_sharing_description:
Qualified researchers can request access to anonymized individual patient-level data from AstraZeneca group of companies sponsored clinical trials via the request portal. All request will be evaluated as per the AZ disclosure commitment:

https://astrazenecagrouptrials.pharmacm.com/ST/Submission/Disclosure.

Yes, indicates that AZ are accepting requests for IPD, but this does not mean all requests will be shared.

lead_sponsor: MedImmune LLC

location_country:
Spain
United States
France
Netherlands


title: Evaluation of an eXtended and proacTive Dosing regimEn in Treatment-Naïve Patients With Wet Age-related Macular Degeneration (wAMD)

acronym: XTEND

summary: In this observational study researchers want to learn more about changes in visual acuity (clarity of vision) with proactive flexible treatments over time in patients suffering from wet age-related macular degeneration (wAMD) after decision to treat with Aflibercept (Eylea) was made. Wet AMD is an eye disease that progressively destroys the macula, the central portion of the retina, impairing central vision.

ipd_sharing_description: Availability of this study's data will later be determined according to Bayer's commitment to the EFPIA/PhRMA "Principles for responsible clinical trial data sharing". This pertains to scope, timepoint and process of data access. As such, Bayer commits to sharing upon request from qualified researchers patient-level clinical trial data, study-level clinical trial data, and protocols from clinical trials in patients for medicines and indications approved in the US and EU as necessary for conducting legitimate research. This applies to data on new medicines and indications that have been approved by the EU and US regulatory agencies on or after January 01, 2014. Interested researchers can use www.clinicalstudydatarequest.com to request access to anonymized patient-level data and supporting documents from clinical studies to conduct research. Information on the Bayer criteria for listing studies and other relevant information is provided in the Study sponsors section of the portal.

lead_sponsor: Bayer

location_country:
Italy
Sweden
Switzerland
Spain
France
Thailand
Norway
Ireland
Belgium
Argentina
Australia
China
Denmark
Canada
United Kingdom
Korea, Republic of
Colombia


type: Other Grant/Funding Number

title: An Innovative Approach Towards Understanding and Arresting Type 1 Diabetes (INNODIA)

acronym: INNODIA

summary:
INNODIA is a global consortium linking 26 academic institutions, 4 industrial partners, a small to medium enterprise (SME), and 2 patient organisations, bringing their knowledge and experience together with one common goal: "To fight type 1 diabetes". (www.innodia.eu).

The project, approved in November 2015 and launched in January 2016, runs under the framework of the Innovative Medicines Initiative - Joint Undertaking (https://www.imi.europa.eu/projects-results/project-factsheets/innodia) with a dedicated governance structure ensuring close interaction, communication and adherence to the objectives and deliverables of the consortium.

The overall aim of INNODIA is to advance in a decisive way how to predict, stage, evaluate and prevent the onset and progression of type 1 diabetes (T1D). For this, INNODIA has established a comprehensive and interdisciplinary network of clinical and basic scientists, who are leading experts in the field of T1D research in Europe, with complementary expertise from the areas of immunology, Beta-cell biology, biomarker research and T1D therapy, joining forces in a coordinated fashion with industry partners and two foundations, as well as with all major stakeholders in the process, including regulatory bodies and patients with T1D and their families.

One of the objectives of INNODIA is to develop a new European clinical research network with standardized protocol based on repeated measures of C-peptide (including home measurements) and comprehensive collection of appropriate biological samples for 'omics', immune, viral and microbiome studies in new onset T1D patients and high-risk auto-antibody positive subjects. A protocol for the harmonization of sample collections in newly diagnosed type 1 diabetic patients and first degree relatives of patients with type 1 diabetes was developed following extensive preliminary work involving partners from across all specialities. Core laboratories with experience in their respective field were set up for analysis of auto-antibodies, fresh immune cells, handling of frozen immune cells, C-peptide measures. A series of standard operating procedures for sample collections and analysis were agreed. Sample tracking between clinical centres and central laboratories was included into a purposely designed electronic case report form (eCRF) into which all clinical and laboratory data collected are captured.

lead_sponsor: University of Cambridge

location_country:
Italy
Austria
Luxembourg
Sweden
United Kingdom
Finland
France
Poland
Norway
Belgium
Denmark
Germany
Slovenia

location_facility: Institut National de la Sante et de la Recherche Medicale (INSERM)

location_facility: Children's Hospital Auf der Bult, Hannover Medical School


title: Observational Study Evaluating Effectiveness and Safety of Real-World Treatment With Damoctocog Alfa Pegol in Previously Treated Patients With Hemophilia A

acronym: HEM-POWR

summary: The aim of the HEM-POWR study is to understand better how Damoctocog alfa pegol (Jivi) is used to treat people with Hemophilia A in day-to-day life, how well the treatment is tolerated and how satisfied patients and physicians are with the treatment.

ReferenceType: derived

ReferenceCitation: Sanabria M, Álvarez Román MT, Castaman G, Janbain M, Matsushita T, Meijer K, Oldenburg J, Friedl S, Reding MT. Design of the HEM-POWR study: a prospective, observational study of real-world treatment with damoctocog alfa pegol in patients with haemophilia A. BMJ Open. 2021 Sep 2;11(9):e044997. doi: 10.1136/bmjopen-2020-044997.

ipd_sharing_description:
Availability of this study's data will be determined according to Bayer's commitment to the EFPIA/PhRMA "Principles for responsible clinical trial data sharing". This pertains to scope, timepoint and process of data access.

As such, Bayer commits to sharing upon request from qualified researchers patient-level clinical trial data, study-level clinical trial data, and protocols from clinical trials in patients for medicines and indications approved in the US and EU as necessary for conducting legitimate research. This applies to data on new medicines and indications that have been approved by the EU and US regulatory agencies on or after January 01, 2014.

Interested researchers can use www.clinicalstudydatarequest.com to request access to anonymized patient-level data and supporting documents from clinical studies to conduct research. Information on the Bayer criteria for listing studies and other relevant information is provided in the Study sponsors section of the portal.

location_country:
Luxembourg
Finland
Greece
Belgium
Russian Federation
Denmark
Taiwan
France
Japan
Kuwait
Norway
Canada
Slovenia
Colombia
Sweden
United Arab Emirates
Saudi Arabia
Switzerland
Netherlands
Portugal
China
United States
Germany
Italy
Austria
Brazil
Mexico
Spain

location_facility: East Carolina University - Brody School of Medicine

location_facility: The Center for Comprehensive Care and Diagnosis of Inherited Blood Disorders

location_facility: South Alabama Medical Science Foundation

location_facility: University of Colorado Hemophilia and Thrombosis Center

location_facility: Orthopaedic Hospital DBA Orthopaedic Institute for Children


title: Feasibility of the Multiple Sclerosis Performance Test for Assessment of Functional Performance Measures in Patients With Multiple Sclerosis

summary: The primary objective is to evaluate the feasibility of the Multiple Sclerosis Performance Test (MSPT) in a clinical care setting when used by participants with Multiple Sclerosis (MS).

ipd_sharing_description: In accordance with Biogen's Clinical Trial Transparency and Data Sharing Policy on http://clinicalresearch.biogen.com/

lead_sponsor: Biogen

location_country:
Switzerland
United States
France
Czechia


lead_sponsor: Merck Sharp & Dohme Corp.

location_country:
Brazil
Peru
Guatemala
Mexico
Netherlands
Finland
France
Malaysia
Hungary
Belgium
Australia
Russian Federation
Denmark
United States

location_facility: C.H.R.U. de Lille. Hopital Jeanne de Flandres ( Site 3304)

location_facility: University of Vermont Medical Center ( Site 3013)

location_facility: Erasmus University Medical Center ( Site 4225)

location_facility: Royal Childrens Hospital Melbourne ( Site 3801)

location_facility: Children City Clinical Hospital 13 n.a N.F.Filatov ( Site 4285)

location_facility: The Children's Hospital of Philadelphia ( Site 3021)

location_facility: Lucille Packard Children's Hospital ( Site 3008)

location_facility: Women and Children Hospital Kuala Lumpur (Hospital Tunku Azizah) ( Site 3875)

location_facility: Instituto da Crianca do Hospital das Clinicas-FMUSP ( Site 3825)

location_facility: Centenario Hospital Miguel Hidalgo-Pediatrics Department ( Site 4152)

location_facility: UZ Leuven - Campus Gasthuisberg ( Site 3202)

location_facility: The Children s Hospital at Westmead ( Site 3805)

location_facility: Wilhelmina Kinderziekenhuis ( Site 4228)

location_facility: NMRC Obstetrics Gynecology and Perinatology n.a. V.I. Kulakov ( Site 4287)

location_facility: Instituto Nacional Cardiovascular Incor ( Site 3928)

location_facility: McGovern Medical School at UT Health/ Memorial Hermann ( Site 3014)

location_facility: University Medical Center Groningen ( Site 4227)

location_facility: Radboud University Medical Center ( Site 4226)

location_facility: Debreceni Egyetem Klinikai Kozpont ( Site 4200)

location_facility: Queensland Children s Hospital ( Site 3806)

location_facility: Universitaire Ziekenhuis Antwerpen - UZA ( Site 3200)

location_facility: St.Petersburg State Pediatric Medical University ( Site 4281)

location_facility: Pediatric Hematology Oncology and Immunology Centre n.a. D.Rogachev. ( Site 4275)

location_facility: Hospital General de Zona No. 1 ( Site 4153)

location_facility: Variety Children's Hospital D.B.A. Nicklaus Children's Hospital ( Site 3019)

location_facility: Children's Hospital of Pittsburgh UPMC ( Site 3017)

location_facility: Scientific-Research Clinical Pediatric Institution n.a. Veltischev ( Site 4276)

location_facility: Unidad de Cirugía Cardiovascular ( Site 4126)

location_facility: Scientific Research Institute Complex Problems Cardiovascular Disease ( Site 4288)

location_facility: Children City Clinical Hospital #9 n.a. G.N.Speransky ( Site 4290)

location_facility: University Malaya Medical Centre. ( Site 3876)

title: A Phase 4 Double-blinded, Randomized, Active Comparator-controlled Clinical Trial to Study the Efficacy, Safety, and Pharmacokinetics of Sugammadex (MK-8616) for Reversal of Neuromuscular Blockade in Pediatric Participants Aged Birth to <2 Years

summary: This study will evaluate the efficacy, safety, and pharmacokinetics (PK) of sugammadex (MK-8616) for reversal of both moderate and deep neuromuscular blockade (NMB) in pediatric participants aged birth to <2 years. The primary hypothesis of this study is that sugammadex is superior to neostigmine in reversing moderate NMB as measured by time to neuromuscular recovery.


lead_sponsor: Loxo Oncology, Inc.

location_country:
Italy
United States
Germany
France
Japan
Australia
Spain
Denmark
Canada
Korea, Republic of

location_facility: The Children's Hospital for Cancer and Blood Disorders

location_facility: Fondazione IRCCS Istituto Nazionale dei Tumori

location_facility: Seattle Children's Hospital Research Foundation

location_facility: University of Texas Southwestern Medical Center at Dallas

title: A Phase 1/2 Study of the Oral RET Inhibitor LOXO 292 in Pediatric Patients With Advanced RET-Altered Solid or Primary Central Nervous System Tumors

summary: This is an open-label, multi-center Phase 1/2 study of oral LOXO-292 in pediatric participants with an activating rearranged during transfection (RET) alteration and an advanced solid or primary CNS tumor.


ReferenceCitation: Seftel MD. Hyper-CVAD: a regimen for all seasons. Lancet Haematol. 2020 Jul;7(7):e501-e502. doi: 10.1016/S2352-3026(20)30179-4.

family: Seftel

full_name: Matthew D Seftel

given: Matthew D

sequence: first

bso_classification: Medical research

datasource: user

genre: journal-article

genre_raw: journal-article

journal_name: The Lancet Haematology

publisher: Elsevier BV

publisher_dissemination: Elsevier

publisher_group: Elsevier

publisher_normalized: Elsevier

title: Hyper-CVAD: a regimen for all seasons

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 expert 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 is currently available according to the process described on www.clinicalstudydatarequest.com.

lead_sponsor: Novartis Pharmaceuticals

location_country:
Italy
United States
Sweden
Netherlands
Germany
Finland
France
Norway
Belgium
Spain
Denmark
Canada
United Kingdom

acronym: CASSIOPEIA

location_facility: James Whitcomb Riley Hospital for Children

location_facility: Medical University of South Carolina Medical Univ of SC

location_facility: Dana Farber Cancer Institute Dept.of DFCI

location_facility: Monroe Carell Jr Childrens Hospital at Vanderbilt

location_facility: University of Utah Clinical Trials Office

location_facility: Univ Hospital - Cleveland/Rainbow Babies and Children's Hosp Pediatric Nephrology

location_facility: Children's Healthcare of Atlanta Emory University IRB

location_facility: Columbia University Medical Center Oncology

location_facility: Texas Children's Cancer and Hematology Center

location_facility: Johns Hopkins Oncology Center Cancer Research Building

location_facility: University of Wisconsin Hospital and Clinics Pharmacy/Drug Shipping Address

location_facility: Ann and Robert H Lurie Childrens Hospital of Chicago

location_facility: Prinses Maxima Centrum voor Kinderoncologie

location_facility: The Childrens Hospital of Philadelphia Div Gastroint., Hepat. & Nutr.

location_facility: University of Texas Southwestern Medical Center

location_facility: Cincinnati Children s Hospital Medical Center

location_facility: Childrens Hospital Los Angeles SC CTL019

title: A Phase II Trial of Tisagenlecleucel in First-line High-risk (HR) Pediatric and Young Adult Patients With B-cell Acute Lymphoblastic Leukemia (B-ALL) Who Are Minimal Residual Disease (MRD) Positive at the End of Consolidation (EOC) Therapy

summary: This is a single arm, open-label, multi-center, phase II study to determine the efficacy and safety of tisagenlecleucel in de novo HR pediatric and young adult B-ALL patients who received first-line treatment and are EOC MRD positive. The study will have the following sequential phases: screening, pre-treatment, treatment & follow-up, and survival. After tisagenlecleucel infusion, patient will have assessments performed more frequently in the first month and then at Day 29, then every 3 months for the first year, every 6 months for the second year, then yearly until the end of the study. Efficacy and safety will be assessed at study visits and as clinically indicated throughout the study. The study is expected to end in approximately 8 years after first patient first treatment (FPFT). A post-study long term follow-up for lentiviral vector safety will continue under a separate protocol per health authority guidelines.


title: A Phase 1b, Multicenter Study to Determine the Dose, Safety, Efficacy and Pharmacokinetics of TRK-950 When Used in Combinations With Selected Anti-Cancer Treatment Regimens in Patients With Selected Advanced Solid Tumors

summary: The main purpose of this study is to establish the safety and the recommended dose of TRK-950 in combination with FOLFIRI, Gemcitabine / Cisplatin, Gemcitabine / Carboplatin, Ramucirumab / Paclitaxel, PD1 inhibitors (Nivolumab or Pembrolizumab), and Imiquimod Cream, Bevacizumab, Gemcitabine / Carboplatin / Bevacizumab, Topotecan, and Pegylated liposomal doxorubicin (PLD) for selected advanced solid tumors.

lead_sponsor: Toray Industries, Inc

location_country:
United States
France

location_facility:
Centre Léon Bérard
Atlantic Health System
HonorHealth Research Institute
HOAG Memorial Hospital Presbyterian
USC Norris Comprehensive Cancer Center
Ochsner Clinic Foundation
Medical College of Wisconsin


title: EUS-guided Biliary Drainage of First Intent With the Lumen Apposing Metal Stent vs. ERCP in the Management of Malignant Distal Biliary Obstruction: a Randomized Controlled Trial

summary: Endoscopic ultrasound guided biliary drainage (EUS-BD) is an evolving field that has grown in popularity in the management of malignant biliary obstruction. Although Endoscopic retrograde cholangio-pancreatography (ERCP) with stent insertion has been the mainstay therapy throughout several decades, the transpapillary approach through tumor tissue is associated with significant risk for adverse events such as post-ERCP pancreatitis and stent dysfunction from tumor tissue overgrowth and ingrowth. EUS-BD, through the creation of a choledochoduodenostomy with a stent, has the potential advantage of avoiding the papilla and its associated complications while potentially improving stent patency with lower risks for tumor tissue ingrowth and/or overgrowth.

ReferenceCitation: Boulay BR, Lo SK. Endoscopic Ultrasound-Guided Biliary Drainage. Gastrointest Endosc Clin N Am. 2018 Apr;28(2):171-185. doi: 10.1016/j.giec.2017.11.005. Epub 2018 Feb 3. Review.

ReferenceCitation: Khashab MA, Messallam AA, Penas I, Nakai Y, Modayil RJ, De la Serna C, Hara K, El Zein M, Stavropoulos SN, Perez-Miranda M, Kumbhari V, Ngamruengphong S, Dhir VK, Park DH. International multicenter comparative trial of transluminal EUS-guided biliary drainage via hepatogastrostomy vs. choledochoduodenostomy approaches. Endosc Int Open. 2016 Feb;4(2):E175-81. doi: 10.1055/s-0041-109083. Epub 2016 Jan 15.

ReferenceCitation: Khashab MA, Van der Merwe S, Kunda R, El Zein MH, Teoh AY, Marson FP, Fabbri C, Tarantino I, Varadarajulu S, Modayil RJ, Stavropoulos SN, Peñas I, Ngamruengphong S, Kumbhari V, Romagnuolo J, Shah R, Kalloo AN, Perez-Miranda M, Artifon EL. Prospective international multicenter study on endoscopic ultrasound-guided biliary drainage for patients with malignant distal biliary obstruction after failed endoscopic retrograde cholangiopancreatography. Endosc Int Open. 2016 Apr;4(4):E487-96. doi: 10.1055/s-0042-102648. Epub 2016 Mar 30.

ReferenceCitation: Almadi MA, Barkun A, Martel M. Plastic vs. Self-Expandable Metal Stents for Palliation in Malignant Biliary Obstruction: A Series of Meta-Analyses. Am J Gastroenterol. 2017 Feb;112(2):260-273. doi: 10.1038/ajg.2016.512. Epub 2016 Nov 15. Review.

ReferenceCitation: Almadi MA, Barkun JS, Barkun AN. Stenting in Malignant Biliary Obstruction. Gastrointest Endosc Clin N Am. 2015 Oct;25(4):691-711. doi: 10.1016/j.giec.2015.06.002. Epub 2015 Aug 28. Review.

ReferenceCitation: American Society for Gastrointestinal Endoscopy (ASGE) Standards of Practice Committee, Anderson MA, Appalaneni V, Ben-Menachem T, Decker GA, Early DS, Evans JA, Fanelli RD, Fisher DA, Fisher LR, Fukami N, Hwang JH, Ikenberry SO, Jain R, Jue TL, Khan K, Krinsky ML, Malpas PM, Maple JT, Sharaf RN, Shergill AK, Dominitz JA, Cash BD. The role of endoscopy in the evaluation and treatment of patients with biliary neoplasia. Gastrointest Endosc. 2013 Feb;77(2):167-74. doi: 10.1016/j.gie.2012.09.029. Epub 2012 Dec 7.

ReferenceCitation: Bang JY, Navaneethan U, Hasan M, Hawes R, Varadarajulu S. Stent placement by EUS or ERCP for primary biliary decompression in pancreatic cancer: a randomized trial (with videos). Gastrointest Endosc. 2018 Jul;88(1):9-17. doi: 10.1016/j.gie.2018.03.012. Epub 2018 Mar 21.

ReferenceCitation: Lee TH, Choi JH, Park do H, Song TJ, Kim DU, Paik WH, Hwangbo Y, Lee SS, Seo DW, Lee SK, Kim MH. Similar Efficacies of Endoscopic Ultrasound-guided Transmural and Percutaneous Drainage for Malignant Distal Biliary Obstruction. Clin Gastroenterol Hepatol. 2016 Jul;14(7):1011-1019.e3. doi: 10.1016/j.cgh.2015.12.032. Epub 2015 Dec 31.

ReferenceCitation: Paik WH, Lee TH, Park DH, Choi JH, Kim SO, Jang S, Kim DU, Shim JH, Song TJ, Lee SS, Seo DW, Lee SK, Kim MH. EUS-Guided Biliary Drainage Versus ERCP for the Primary Palliation of Malignant Biliary Obstruction: A Multicenter Randomized Clinical Trial. Am J Gastroenterol. 2018 Jul;113(7):987-997. doi: 10.1038/s41395-018-0122-8. Epub 2018 Jul 2. Erratum in: Am J Gastroenterol. 2018 Oct;113(10):1566.

ReferenceCitation: Park JK, Woo YS, Noh DH, Yang JI, Bae SY, Yun HS, Lee JK, Lee KT, Lee KH. Efficacy of EUS-guided and ERCP-guided biliary drainage for malignant biliary obstruction: prospective randomized controlled study. Gastrointest Endosc. 2018 Aug;88(2):277-282. doi: 10.1016/j.gie.2018.03.015. Epub 2018 Mar 30.

ReferenceCitation: Kunda R, Pérez-Miranda M, Will U, Ullrich S, Brenke D, Dollhopf M, Meier M, Larghi A. EUS-guided choledochoduodenostomy for malignant distal biliary obstruction using a lumen-apposing fully covered metal stent after failed ERCP. Surg Endosc. 2016 Nov;30(11):5002-5008. Epub 2016 Mar 11.

ReferenceCitation: Anderloni A, Fugazza A, Troncone E, Auriemma F, Carrara S, Semeraro R, Maselli R, Di Leo M, D'Amico F, Sethi A, Repici A. Single-stage EUS-guided choledochoduodenostomy using a lumen-apposing metal stent for malignant distal biliary obstruction. Gastrointest Endosc. 2019 Jan;89(1):69-76. doi: 10.1016/j.gie.2018.08.047. Epub 2018 Sep 4.

ReferenceCitation: Cotton PB, Eisen GM, Aabakken L, Baron TH, Hutter MM, Jacobson BC, Mergener K, Nemcek A Jr, Petersen BT, Petrini JL, Pike IM, Rabeneck L, Romagnuolo J, Vargo JJ. A lexicon for endoscopic adverse events: report of an ASGE workshop. Gastrointest Endosc. 2010 Mar;71(3):446-54. doi: 10.1016/j.gie.2009.10.027.

ReferenceCitation: Siegel RL, Miller KD, Jemal A. Cancer statistics, 2018. CA Cancer J Clin. 2018 Jan;68(1):7-30. doi: 10.3322/caac.21442. Epub 2018 Jan 4.

ReferenceCitation: Liao WC, Chien KL, Lin YL, Wu MS, Lin JT, Wang HP, Tu YK. Adjuvant treatments for resected pancreatic adenocarcinoma: a systematic review and network meta-analysis. Lancet Oncol. 2013 Oct;14(11):1095-1103. doi: 10.1016/S1470-2045(13)70388-7. Epub 2013 Sep 12. Review.

ReferenceCitation: Freeman ML, Nelson DB, Sherman S, Haber GB, Herman ME, Dorsher PJ, Moore JP, Fennerty MB, Ryan ME, Shaw MJ, Lande JD, Pheley AM. Complications of endoscopic biliary sphincterotomy. N Engl J Med. 1996 Sep 26;335(13):909-18.

ReferenceCitation: Kochar B, Akshintala VS, Afghani E, Elmunzer BJ, Kim KJ, Lennon AM, Khashab MA, Kalloo AN, Singh VK. Incidence, severity, and mortality of post-ERCP pancreatitis: a systematic review by using randomized, controlled trials. Gastrointest Endosc. 2015 Jan;81(1):143-149.e9. doi: 10.1016/j.gie.2014.06.045. Epub 2014 Aug 1. Review.

ReferenceCitation: Almadi MA, Barkun A, Martel M. Self-expandable metal stents versus plastic stents for malignant biliary obstruction. Gastrointest Endosc. 2016 Apr;83(4):852-3. doi: 10.1016/j.gie.2015.10.035.

ReferenceCitation: Gardner TB, Spangler CC, Byanova KL, Ripple GH, Rockacy MJ, Levenick JM, Smith KD, Colacchio TA, Barth RJ, Zaki BI, Tsapakos MJ, Gordon SR. Cost-effectiveness and clinical efficacy of biliary stents in patients undergoing neoadjuvant therapy for pancreatic adenocarcinoma in a randomized controlled trial. Gastrointest Endosc. 2016 Sep;84(3):460-6. doi: 10.1016/j.gie.2016.02.047. Epub 2016 Mar 10.

ReferenceCitation: Barkun AN, Adam V, Martel M, AlNaamani K, Moses PL. Partially covered self-expandable metal stents versus polyethylene stents for malignant biliary obstruction: a cost-effectiveness analysis. Can J Gastroenterol Hepatol. 2015 Oct;29(7):377-83. Epub 2015 Jun 30.

ReferenceCitation: Yachimski PS, Ross A. The Future of Endoscopic Retrograde Cholangiopancreatography. Gastroenterology. 2017 Aug;153(2):338-344. doi: 10.1053/j.gastro.2017.06.015. Epub 2017 Jun 21.

ReferenceCitation: Giovannini M, Moutardier V, Pesenti C, Bories E, Lelong B, Delpero JR. Endoscopic ultrasound-guided bilioduodenal anastomosis: a new technique for biliary drainage. Endoscopy. 2001 Oct;33(10):898-900.

ReferenceCitation: Sharaiha RZ, Khan MA, Kamal F, Tyberg A, Tombazzi CR, Ali B, Tombazzi C, Kahaleh M. Efficacy and safety of EUS-guided biliary drainage in comparison with percutaneous biliary drainage when ERCP fails: a systematic review and meta-analysis. Gastrointest Endosc. 2017 May;85(5):904-914. doi: 10.1016/j.gie.2016.12.023. Epub 2017 Jan 4. Review.

ReferenceCitation: Binmoeller KF, Shah JN. Endoscopic ultrasound-guided gastroenterostomy using novel tools designed for transluminal therapy: a porcine study. Endoscopy. 2012 May;44(5):499-503. doi: 10.1055/s-0032-1309382. Epub 2012 Apr 24.

ReferenceCitation: Chen YI, Kunda R, Storm AC, Aridi HD, Thompson CC, Nieto J, James T, Irani S, Bukhari M, Gutierrez OB, Agarwal A, Fayad L, Moran R, Alammar N, Sanaei O, Canto MI, Singh VK, Baron TH, Khashab MA. EUS-guided gastroenterostomy: a multicenter study comparing the direct and balloon-assisted techniques. Gastrointest Endosc. 2018 May;87(5):1215-1221. doi: 10.1016/j.gie.2017.07.030. Epub 2017 Jul 24.

ReferenceCitation: Kamarajah SK, Burns WR, Frankel TL, Cho CS, Nathan H. Validation of the American Joint Commission on Cancer (AJCC) 8th Edition Staging System for Patients with Pancreatic Adenocarcinoma: A Surveillance, Epidemiology and End Results (SEER) Analysis. Ann Surg Oncol. 2017 Jul;24(7):2023-2030. doi: 10.1245/s10434-017-5810-x. Epub 2017 Feb 17.

ReferenceCitation: Chen YI, Callichurn K, Chatterjee A, Desilets E, Fergal D, Forbes N, Gan I, Kenshil S, Khashab MA, Kunda R, Lam E, May G, Mohamed R, Mosko J, Paquin SC, Sahai A, Sandha G, Teshima C, Barkun A, Barkun J, Bessissow A, Candido K, Martel M, Miller C, Waschke K, Zogopoulos G, Wong C; ELEMENT trial and for the Canadian Endoscopic Research Collaborative (CERC). ELEMENT TRIAL: study protocol for a randomized controlled trial on endoscopic ultrasound-guided biliary drainage of first intent with a lumen-apposing metal stent vs. endoscopic retrograde cholangio-pancreatography in the management of malignant distal biliary obstruction. Trials. 2019 Dec 9;20(1):696. doi: 10.1186/s13063-019-3918-y.

family: Chen

full_name: Yen-I Chen

given: Yen-I

name: for the ELEMENT trial and for the Canadian Endoscopic Research Collaborative (CERC)

family: Callichurn

full_name: Kashi Callichurn

given: Kashi

sequence: additional

family: Chatterjee

full_name: Avijit Chatterjee

given: Avijit

family: Desilets

full_name: Etienne Desilets

given: Etienne

family: Fergal

full_name: Donnellan Fergal

given: Donnellan

family: Forbes

full_name: Nauzer Forbes

given: Nauzer

family: Gan

full_name: Ian Gan

given: Ian

family: Kenshil

full_name: Sana Kenshil

given: Sana

family: Khashab

full_name: Mouen A. Khashab

given: Mouen A.

family: Kunda

full_name: Rastislav Kunda

given: Rastislav

family: Lam

full_name: Eric Lam

given: Eric

family: May

full_name: Gary May

given: Gary

family: Mohamed

full_name: Rachid Mohamed

given: Rachid

family: Mosko

full_name: Jeff Mosko

given: Jeff

family: Paquin

full_name: Sarto C. Paquin

given: Sarto C.

family: Sahai

full_name: Anand Sahai

given: Anand

family: Sandha

repositories_institution:
PubMed Central - Europe PMC
University of Calgary - PRISM
University of Ottawa - uO Research

lead_sponsor: McGill University Health Centre/Research Institute of the McGill University Health Centre

location_facility: Centre Hospitalier Universite de Montreal


title: Rostock International Parkinson's Disease Study: an International, Multicenter, Epidemiological Observational Study

acronym: ROPAD

summary: Rostock International Parkinson's Disease Study - An International, multicentre, epidemiological observational study aiming at identification of 1,500 LRRK2-positive patients, the identification of 1,500 non-LRRK2 PD patients and the establishment of a candidate biomarker in the LRRK2-positive cohort.

ReferenceType: result

ReferenceCitation: Baumann CR. Epidemiology, diagnosis and differential diagnosis in Parkinson's disease tremor. Parkinsonism Relat Disord. 2012 Jan;18 Suppl 1:S90-2. doi: 10.1016/S1353-8020(11)70029-3. Review.

family: Baumann

full_name: Christian R. Baumann

given: Christian R.

journal_name: Parkinsonism & Related Disorders

ipd_sharing_description: The plan will be defined at a later stages

lead_sponsor: CENTOGENE GmbH Rostock

location_country:
Italy
Brazil
United States
Spain
Germany
France
Albania
Greece
Israel
Norway
Belgium
Portugal
Turkey
Canada
United Kingdom

location_facility: Royal Preston Hospital, Department of Neurology

location_facility: Michigan Institute for Neurological Disorders

location_facility: Neurologisches Fachkrankenhaus für Bewegungsstörungen / Parkinson

location_facility: Hospital Clinico Universitario Santiago de Compostela

location_facility: Universidade Federal de São Paulo, UNIFESP

location_facility: Universitätsklinikum Marburg Klinik und Poliklinik für Neurologie Parkinson Netzwerk Allianz Marburg (PANAMA)

location_facility: Hospital General Unversitario Gregorio Marañón

location_facility: Biocruces Bizkaia Health Research Institute

location_facility: Parkinson's Disease and Movement Disorder Center of Baco Raton

location_facility: Samson Assuta Ashdod University Hospital

location_facility: Hospital Universitário Walter Cândido/ Universidade Federal do Ceará

location_facility: Centro Hospitalar Universitário de São João

location_facility: Vero Beach Neurology & Research Institute

location_facility: Università di Pavia, Dipartimento di Medicina Molecolare

location_facility: Asklepios Fachklinikum Stadtroda Klinik für Neurologie, Schmerztherapie und Schlafmedizin

location_facility: Duke University, Department of Neurology

location_facility: Instituto Israelita de Ensino e Pesquisa Albert Einstein

location_facility: Universitätsklinikum Schleswig-Holstein, Klinik für Neurologie

location_facility: Newcastle University, Clinical Ageing Research Unit

location_facility: University Hospital Marqués de Valdecilla

location_facility: AOUP Secondo Policlinico Federico II Napoli

location_facility: HM CINAC | Integrated Neuroscience Center

location_facility: Stony Brook University School of Medicine

location_facility: Toronto Western Hospital, University Health Network, University of Toronto

location_facility: Hospital das Clínicas da Universidade Federal de Minas Gerais

location_facility: University of Oxford, Nuffield Department of Clinical Neurosciences

location_facility: Universitätsklinikum Carl Gustav Carus die Dresdner

location_facility: Hospital Universitario Pedro Ernesto (HUPE)

location_facility: Faculdade de Medicina de Ribeirão Preto - USP

location_facility: Tel Aviv Sourasky Medical Center - TASMC

location_facility: Department of Neurology, Centre Hospitalier Universitaire (CHU) de Leige

location_facility: Indiana University, Department of Neurology

location_facility: Hospital Universitario Fundación Alcorcón


ipd_sharing_description: AbbVie is committed to responsible data sharing regarding the clinical trials we sponsor. This includes access to anonymized, individual and trial-level data (analysis data sets), as well as other information (e.g., protocols and clinical study reports), as long as the trials are not part of an ongoing or planned regulatory submission. This includes requests for clinical trial data for unlicensed products and indications.

lead_sponsor: AbbVie

location_country:
Austria
United States
Sweden
Hong Kong
Mexico
Spain
France
Poland
Greece
Ireland
Australia
Belgium
New Zealand
Portugal
Russian Federation
Denmark
Canada
United Kingdom
Turkey
Taiwan

location_facility: University Hospital Southampton NHS Foundation Trust /ID# 225161

location_facility: UCLA Santa Monica Hematology Oncology /ID# 210551

location_facility: Swedish Cancer Institute - Edmonds /ID# 213120

location_facility: Skane University Hospital Lund /ID# 224747

location_facility: Dana-Farber Cancer Institute /ID# 215360

location_facility: China Medical University Hospital /ID# 224945

location_facility: Federal State Budgetary Scientific Institution N.N. Blokhin /ID# 225194

location_facility: Dokuz Eylul University Medical Faculty /ID# 225044

location_facility: Wellington Regional Hospital /ID# 225593

location_facility: The Clatterbridge Cancer Centre NHS Foundation Trust /ID# 218736

location_facility: Blackpool Teaching Hospitals NHS Foundation Trust /ID# 225163

location_facility: Hospital Universitario Fundacion Jimenez Diaz /ID# 225476

location_facility: CHUQ- Hôpital de l'Enfant-Jesus /ID# 224616

location_facility: Hospital Universitario Dr. Jose Eleuterio Gonzalez /ID# 227017

location_facility: Ondokuz mayis University Facul /ID# 225042

location_facility: Duplicate_Gabrail Cancer Center Research /ID# 212061

location_facility: Royal Prince Alfred Hospital /ID# 239557

location_facility: Centro de Investigacion Clínica Chapultepec SA de CV /ID# 227018

location_facility: Istanbul University Istanbul Medical Faculty /ID# 225045

location_facility: General Hospital of Thessaloniki George Papanikolaou /ID# 224088

location_facility: University Hospital Plymouth NHS Trust /ID# 218731

location_facility: Medizinische Universitaet Graz /ID# 223817

location_facility: SP ZOZ Zespol Szpitali Miejskich w Chorzowie /ID# 225474

location_facility: Regional Oncological Dispensary /ID# 225195

location_facility: General Hospital of Athens Laiko /ID# 224087

location_facility: King's College Hospital NHS Foundation Trusts /ID# 218735

location_facility: The Royal Wolverhampton NHS Trust /ID# 225164

location_facility: University of Michigan Comprehensive Cancer Center Michigan Medicine /ID# 231132

location_facility: University of Arizona Cancer Center - Tucson /ID# 210548

location_facility: Dartmouth-Hitchcock Medical Center /ID# 210555

location_facility: National Taiwan University Hospital /ID# 224946

location_facility: Instytut Hematologii i Transfuzjologii /ID# 225478

location_facility: Vehbi Koc vakfi Amerikan Hasta /ID# 225046

location_facility: University Hospitals Bristol NHS Foundation Trust /ID# 225162

title: An Extension Study of Venetoclax for Subjects Who Have Completed a Prior Venetoclax Clinical Trial

summary: The purpose of this extension study is to provide venetoclax and obtain long-term safety data for subjects who continue to tolerate and derive benefit from receiving venetoclax in ongoing studies.


title: REtroperitoneal SArcoma Registry (RESAR): Prospective Collection of Primary Retroperitoneal Sarcoma Patient's Data, Radiological and Pathological Material for the TransAtlantic Retroperitoneal Sarcoma Working Group

acronym: RESAR

summary:
Surgery is currently the only potentially curative treatment modality for localized retroperitoneal sarcoma (RPS). Available studies regarding oncologic outcomes are mainly retrospective in nature, and RPS are recognized as a rare disease. Therefore, prospective analysis of high quality data is a top priority.

Primary Objectives of this study are:

to prospectively collect standardized clinical data and radiological and pathological material from primary RPS patients treated with surgery at reference centers.
patient outcome will be evaluated in terms of overall survival (OS), disease-free survival (DFS), crude cumulative incidence (CCI) of local recurrence (LR) and distant metastasis (DM).

Secondary Objectives:

to estimate the efficacy and safety of surgical treatment, including extended surgical approach to primary RPS;
to prospectively evaluate the impact of multimodality therapy, including radiation therapy and chemotherapy;
to identify clinical, radiological and pathological characteristics that may influence the oncological outcome or may be used as predictors of LR/DM/OS. These may be important biomarkers of disease;
to utilize collected pathological material for research collaborations.

ReferenceCitation: Gronchi A, Strauss DC, Miceli R, Bonvalot S, Swallow CJ, Hohenberger P, Van Coevorden F, Rutkowski P, Callegaro D, Hayes AJ, Honoré C, Fairweather M, Cannell A, Jakob J, Haas RL, Szacht M, Fiore M, Casali PG, Pollock RE, Raut CP. Variability in Patterns of Recurrence After Resection of Primary Retroperitoneal Sarcoma (RPS): A Report on 1007 Patients From the Multi-institutional Collaborative RPS Working Group. Ann Surg. 2016 May;263(5):1002-9. doi: 10.1097/SLA.0000000000001447.

ReferenceCitation: Trans-Atlantic RPS Working Group. Management of primary retroperitoneal sarcoma (RPS) in the adult: a consensus approach from the Trans-Atlantic RPS Working Group. Ann Surg Oncol. 2015 Jan;22(1):256-63. doi: 10.1245/s10434-014-3965-2. Epub 2014 Oct 15.

ReferenceCitation: MacNeill AJ, Gronchi A, Miceli R, Bonvalot S, Swallow CJ, Hohenberger P, Van Coevorden F, Rutkowski P, Callegaro D, Hayes AJ, Honoré C, Fairweather M, Cannell A, Jakob J, Haas RL, Szacht M, Fiore M, Casali PG, Pollock RE, Barretta F, Raut CP, Strauss DC. Postoperative Morbidity After Radical Resection of Primary Retroperitoneal Sarcoma: A Report From the Transatlantic RPS Working Group. Ann Surg. 2018 May;267(5):959-964. doi: 10.1097/SLA.0000000000002250.

lead_sponsor: Fondazione IRCCS Istituto Nazionale dei Tumori, Milano

location_country:
Italy
United States
United Kingdom
Germany
France
Poland
Australia
Spain
Canada
Slovenia
Korea, Republic of

location_facility: Brigham and Women's Hospital/Dana-Farber Cancer Institute

location_facility: Maisonneuve-Rosemont Hospital / University of Montreal

location_facility: Hospital Clinico Universitario "Virgen de la Arrixaca"

location_facility: University Hospital Birmingham (Queen Elizabeth)

location_facility: Maria Sklodowska-Curie Memorial Cancer Center and Institute of Oncology

location_facility: University Medical Center and Medical Faculty Mannheim, University of Heidelberg


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

lead_sponsor: Eli Lilly and Company

location_country:
Italy
Austria
United States
Mexico
Germany
France
Czechia
Japan
Hungary
Argentina
Puerto Rico
Spain
Canada

acronym: ADmIRe

location_facility: Instituto de Neumonología y Dermatología

location_facility: The Guenther Dermatology Research Centre

location_facility: Kyoto Prefectural University of Medicine

location_facility: Piedmont Plastic Surgery and Dermatology

location_facility: Tien Q. Nguyen, MD inc. DBA First OC Dermatology

location_facility: Dokkyo Medical University Saitama Medical Center

location_facility: Centro de Investigaciones Metabólicas (CINME)

title: A Multicenter, Randomized, Double-Blind, Placebo-Controlled, Phase 2 Study to Evaluate the Efficacy and Safety of LY3375880 in Adult Subjects With Moderate-to-Severe Atopic Dermatitis: The ADmIRe Study

summary: The reason for this study is to see if the study drug LY3375880 is safe and effective in adults with moderate-to-severe atopic dermatitis (AD).


title: A Phase 3, 22-week, Multi-center, Randomized Withdrawal Study of TD-9855 in Treating Symptomatic Neurogenic Orthostatic Hypotension in Subjects With Primary Autonomic Failure

acronym: REDWOOD

summary: A Phase 3, 22-week, Multi-center, Randomized Withdrawal Study of ampreloxetine in Treating Symptomatic Neurogenic Orthostatic Hypotension in Subjects with Primary Autonomic Failure

ipd_sharing_description: Theravance Biopharma, Inc. will not be sharing individual de-identified participant data or other relevant study documents.

lead_sponsor: Theravance Biopharma

location_country:
Italy
Austria
United Kingdom
Spain
Germany
France
Bulgaria
Poland
Israel
Hungary
Argentina
Australia
New Zealand
Portugal
Estonia
Denmark
United States
Russian Federation
Canada
Ukraine

location_facility: University of Cincinnati Medical Center (UCGNI)

location_facility: Universita di Bologna-Clinica Neurologica - Dipt di Scienze Neurologiche Ospedale Bellaria

location_facility: Saint Petersburg State Budgetary Institution of Healthcare City Hospital #40 of Kurortnyi Region

location_facility: Azienda Ospedaliera Universitaria Policlinico - Vittorio Emanuele (Presidio Gaspare Rodolico)

location_facility: Medizinische Universitat Innsbruck, Abteilung fur Neurologie

location_facility: FSBI of Science Institute of Human Brain named after N .P. Bekhtereva of Russian Academy of Sciences

location_facility: The National Hospital for Neurology & Neurosurgery

location_facility: Universita degli studi Gabriele D' Annunzio Chieti

location_facility: Communal Noncommercial Enterprise of Lviv Regional Council Lviv Regional Clinical Hospital

location_facility: Communal Noncommercial Enterprise City Policlinic #9 of Kharkiv City Council

location_facility: Krakowska Akademia Neurologii Sp. Zo.o. Centrum Neurologii Klinicznej

location_facility: Hospital da Senhora da Oliveira Guimarães

location_facility: Concord Hospital, Neurosciences Department

location_facility: FSBI National Medical Research Centre of psychiatry and neurology named after V.M. Bekhterev of the MOH of the Russian Federation

location_facility: Ineba - Instituto de Neurociencias de Buenos Aires

location_facility: University of Kansas Medical Center Research Institute, Inc.

location_facility: Colorado Springs Neurological Associates, PC

location_facility: The Royal Melbourne Hospital Neurology Department

location_facility: AOU San Giovanni di Dio e Ruggi d'Aragona

location_facility: Azienda Ospedaliero-Universitaria Pisana- Ospedale S. Chiara, U.O. di Neurologia - Neurofisiopatologia

location_facility: Charite Universitaetsmedizin Berlin - Campus Benjamin Franklin

location_facility: Specjalistyczna Praktyka Lekarska, Prof. Grzegorz Opala

location_facility: Fondazione IRCCS CA Granda Ospedale Maggiore Policlinico

location_facility: City Neurological Center Sibneiromed, LLC

location_facility: Perron Institute for Neurological and Translational Science

location_facility: Wake Forest University Baptist Health Sciences

location_facility: Montreal Neurological Institute & Hospital

location_facility: Complejo Médico de la Policía Federal Argentina Churruca Visca

location_facility: Fondazione Policlinico Universitario Agostino Gemelli IRCCS / Istituto di Neurologia - Ambulatorio Disturbi del Movimento

location_facility: FSBI Federal Sibirian Scientific and Clinical Center of Federal Medico-Biological Agency

location_facility: Clinical Research Centre, William Harvey Heart Centre

location_facility: Communal Institution Acad. O.I. Yuschenko VRPsH Vinnytsia M.I. Pyrogov NMU Ch of ND with the Course of Neurosurgery

location_facility: Ohio State University - Wexner Medical Center

location_facility: MHATNP Sv. Naum EAD Clinic of Neurological Diseases for Locomotor Disorders

location_facility: Parkinson's Disease and Movement Disorders Center

location_facility: State Budgetary Institution of Healthcare of Novosibirsk region City Clinical Hospital #34

location_facility: Clinical Trials Centre, Level 3 Monash Health Translational Precinct Building Monash Medical Centre

location_facility: Royal Devon and Exeter Hospital NHS Trust

location_facility: Charite - Campus Virchow-Klinikum, Klinik fur Neurologie

location_facility: University of Calgary Teaching Research and Wellness Building

location_facility: Fondazione PTV - Policlinico Tor Vergata I U.0.C. Neurologia

location_facility: Universitatsklinikum Tulln Abteilung fur Neurologie

location_facility: University of Texas Southwestern Medical Center


title: A Randomized, Double-Blind, Active-Controlled Phase 3 Study Evaluating the Efficacy and Safety of ABP 959 Compared With Eculizumab in Adult Subjects With Paroxysmal Nocturnal Hemoglobinuria (PNH)

acronym: DAHLIA

summary: This is a randomized, double-blind, active-controlled phase 3 study of ABP 959 in participants with paroxysmal nocturnal hemoglobinuria.

ipd_sharing_description: De-identified individual patient data for variables necessary to address the specific research question in an approved data sharing request

lead_sponsor: Amgen

location_country:
Italy
Sweden
United Kingdom
Netherlands
Spain
Finland
France
Czechia
Norway
Ireland
Portugal
Turkey
United States
Slovenia

location_facility: Istituto Scientifico Romagnolo per lo Studio e la Cura dei Tumori

location_facility: Ege Universitesi Hastanesi - Sağlık Uygulama ve Araştırma Merkezi

location_facility: Instituto Português de Oncologia do Porto Francisco Gentil

location_facility: Azienda Ospedaliera San Gerardo di Monza

location_facility: King's College Hospital NHS Foundation Trust

location_facility: Fondazione Policlinico Universitario Agostino Gemelli

location_facility: Azienda Ospedaliera S. Croce e Carle Cuneo

location_facility: The Oncology Institute of Hope and Innovation

location_facility: Karolinska Universitetssjukhuset - Huddinge

location_facility: Children's Healthcare of Atlanta at Egleston

location_facility: Oslo University Hospital - Rikshospitalet


title: French African Pediatric Oncology Registry

acronym: RFAOP

summary:
The ultimate aim of this registry is to collect precise information concerning the children coming to oncology units working with the French African Oncology Group. This data will help to plan and provide correct pediatric oncology treatment and care for this population.

Collecting the data will give much needed information on numbers, stage, treatment and outcome. The register will give data for local and national health authorities in planning pediatric cancer programs.

ipd_sharing_description: Units have acess to their own data and can use it for reporting.

lead_sponsor: French Africa Pediatric Oncology Group

location_country:
Mauritania
Côte D'Ivoire
Congo, The Democratic Republic of the
Gabon
Mali
Cameroon
Togo
France
Madagascar
Niger
Senegal
Burkina Faso

location_facility: Cliniques Universitaires de Lubumbashi (CUL)

location_facility: Centre Hospitalier Universitaire Pédiatrique Charles De Gaulle

location_facility: Hôpital Aristide Le Dantec, Avenue Pasteur, BP 3001

location_facility: l'Institut de Cancérologie de Libreville

location_facility: Centre Mère et Enfant CME-FCB (Fondation Chantal Biya)

location_facility: HJRA, Hôpital universitaire Joseph Ravoahangy Andrianavalona

location_facility: CNLC, Centre National de Lutte contre le Cancer


title: A Phase 1 Study Evaluating the Safety, Tolerability, Pharmacokinetics, and Efficacy of Prostate Specific Membrane Antigen Half-life Extended Bispecific T-cell Engager Acapatamab in Subjects With Metastatic Castration-resistant Prostate Cancer

summary: A phase 1 study evaluating the safety, tolerability, pharmacokinetics, and efficacy of prostate specific membrane antigen half-life extended bispecific T-cell engager acapatamab in subjects with metastatic castration-resistant prostate cancer, and to determine the maximum tolerated dose (MTD) or recommended phase 2 dose (RP2D).

location_country:
Austria
United States
Netherlands
France
Japan
Singapore
Australia
Belgium
Canada
Taiwan

location_facility: Universite Catholique de Louvain Cliniques Universitaires Saint Luc

location_facility: Linkou Chang Gung Memorial Hospital of Chang Gung Medical Foundation

location_facility: Krankenhaus der Barmherzigen Brueder Wien

location_facility: Universitaetsklinikum Allgemeines Krankenhaus Wien

location_facility: University of Iowa Hospitals and Clinics

location_facility: University of Texas Southwestern Medical Center


title: A Phase 1, Open-Label Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Preliminary Efficacy of HMPL-689 in Patients With Relapsed or Refractory Lymphoma

summary: An open-label, dose escalation and expansion clinical trial to evaluate the safety, tolerability and PK of HMPL-689 in patients with relapsed or refractory lymphomas

lead_sponsor: Hutchison Medipharma Limited

location_country:
Italy
Finland
France
Poland
Spain
Denmark
United States

location_facility: ICO Badalona - Hospital Universitari Germans Trias i Pujol

location_facility: Baylor Scott and White Research Institute

location_facility: Azienda Ospedaliera San Camillo Forlanini

location_facility: Uniwersytecki Szpital Kliniczny im. Jana Mikulicza Radeckiego

location_facility: University of Texas MD Anderson Cancer Center

location_facility: Winship Cancer Institute of Emory University

location_facility: Mays Cancer Center, UT Health San Antonio, MD Anderson Cancer Center


title: A Multicenter Study to Evaluate Long-term Safety and Tolerability of Open Label Sacubitril/Valsartan in Pediatric Patients With Heart Failure Due to Systemic Left Ventricle Systolic Dysfunction Who Have Completed Study CLCZ696B2319

summary: The purpose of this study is to evaluate long-term safety and tolerability data in eligible CLCZ696B2319 (PANORAMA-HF) patients receiving open-label sacubitril/valsartan.

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:
Finland
Russian Federation
Taiwan
India
Croatia
France
Japan
Poland
Thailand
Argentina
Canada
Korea, Republic of
South Africa
Switzerland
Bulgaria
Singapore
Hungary
Portugal
United States
Germany
Italy
Austria
Lebanon
Czechia
Israel
Spain
Turkey


ipd_sharing_description: De-identified individual participant data from this particular study will not be shared as there is a reasonable likelihood that individual patients could be re-identified (due to the limited number of study participants).

lead_sponsor: Shire

location_country:
Brazil
United States
Netherlands
Germany
France
Japan
Israel
Belgium
Spain
Canada
United Kingdom

acronym: Embolden

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

location_facility: University of Iowa Stead Family Children's Hospital

location_facility: Birmingham Children's Hospital NHS Foundation Trust

location_facility: Cincinnati Children's Hospital Medical Center

location_facility: New York University Langone Medical Center

location_facility: Los Angeles Biomedical Research Institute at Harbor-UCLA

type: Registry Identifier

title: A Global, Multicenter, Open-label, Matched Historical Control Study of Intrathecal SHP611 in Subjects With Late Infantile Metachromatic Leukodystrophy

summary: The purpose of this study is to evaluate the safety and efficacy of intrathecal (IT) administration of SHP611 on gross motor function, using the Gross Motor Function Classification in Metachromatic Leukodystrophy (GMFC-MLD) compared with matched historical control data in children with metachromatic leukodystrophy (MLD).


lead_sponsor: Kite, A Gilead Company

location_country:
Canada
United States
Australia
France

location_facility: H. Lee Moffitt Cancer Center and Research Institute

location_facility: The University of Texas MD Anderson Cancer Center

title: A Phase 2 Multicenter Study Evaluating the Efficacy and Safety of Axicabtagene Ciloleucel as First-Line Therapy in Subjects With High-Risk Large B-Cell Lymphoma (ZUMA-12)

summary: The primary objective of this study is to estimate the efficacy of axicabtagene ciloleucel in participants with high-risk large B-cell lymphoma.


summary: The purpose of the Micra Atrial TRacking Using A Ventricular AccELerometer 2 (MARVEL 2) study is to demonstrate safe and effective operation of the MARVEL 2 features for providing AV synchronous pacing in patients with normal sinus node function and AV block

ReferenceCitation: Garweg C, Khelae SK, Chan JYS, Chinitz L, Ritter P, Johansen JB, Sagi V, Epstein LM, Piccini JP, Pascual M, Mont L, Willems R, Splett V, Stromberg K, Sheldon T, Kristiansen N, Steinwender C. Behavior of AV synchrony pacing mode in a leadless pacemaker during variable AV conduction and arrhythmias. J Cardiovasc Electrophysiol. 2021 Jul;32(7):1947-1957. doi: 10.1111/jce.15061. Epub 2021 May 20.

name: Department of Cardiovascular Sciences University Hospitals Leuven, University of Leuven  Leuven Belgium

family: Garweg

full_name: Christophe Garweg

given: Christophe

name: Department of Electrophysiology Institut Jantung Negara  Kuala Lumpur Malaysia

family: Khelae

full_name: Surinder Kaur Khelae

given: Surinder Kaur

name: Department of Medicine and Therapeutics, Prince of Wales Hospital Chinese University of Hong Kong  Shatin Hong Kong

family: Chan

full_name: Joseph Yat Sun Chan

given: Joseph Yat Sun

name: Leon H. Charney Division of Cardiology NYU Langone Medical Center  New York New York USA

family: Chinitz

full_name: Larry Chinitz

given: Larry

name: Department of Electrophysiology and Cardiac Stimulation Hôpital Haut‐ Lévêque–CHU de Bordeaux  Pessac France

family: Ritter

full_name: Philippe Ritter

given: Philippe

name: Department of Cardiology Odense University Hospital  Odense Denmark

family: Johansen

full_name: Jens Brock Johansen

given: Jens Brock

name: Baptist Heart Specialists Baptist Medical Center  Jacksonville Florida USA

family: Sagi

full_name: Venkata Sagi

given: Venkata

name: Department of Electrophysiology North Shore University Hospital  Manhasset New York USA

family: Epstein

full_name: Laurence M. Epstein

given: Laurence M.

name: Division of Cardiology Duke University Medical Center and Duke Clinical Research Institute  Durham North Carolina USA

family: Piccini

full_name: Jonathan P. Piccini

given: Jonathan P.

name: Miami Cardiac & Vascular Institute Baptist Hospital  Miami Florida USA

family: Pascual

full_name: Mario Pascual

given: Mario

name: Institut Clinic Cardiovascular (ICCV), Hospital Clínic Universitat de Barcelona  Barcelona Catalonia Spain

family: Mont

full_name: Lluis Mont

given: Lluis

family: Willems

full_name: Rik Willems

given: Rik

name: Medtronic, Inc.  Minneapolis Minnesota USA

family: Splett

full_name: Vincent Splett

given: Vincent

family: Stromberg

full_name: Kurt Stromberg

given: Kurt

family: Sheldon

full_name: Todd Sheldon

given: Todd

name: Medtronic Bakken Research Center  Maastricht The Netherlands

family: Kristiansen

full_name: Nina Kristiansen

given: Nina

name: Department of Cardiology, Kepler University Hospital, Medical Faculty Johannes Kepler University  Linz Austria

name: Department of Cardiology Paracelsus Medical University Salzburg  Salzburg Austria

family: Steinwender

full_name: Clemens Steinwender

given: Clemens

journal_name: Journal of Cardiovascular Electrophysiology

oa_host_type: publisher;repository

host_type: publisher

license: cc-by

license_normalized: cc-by

version: publishedVersion

evidence: oa repository (via pmcid lookup)

host_type: repository

license_normalized: no license

repository_normalized: PubMed Central

ReferenceCitation: Garweg C, Khelae SK, Steinwender C, Chan JYS, Ritter P, Johansen JB, Sagi V, Epstein LM, Piccini JP, Pascual M, Mont L, Willems R, Sheldon T, Splett V, Stromberg K, Wood N, Chinitz L. Predictors of atrial mechanical sensing and atrioventricular synchrony with a leadless ventricular pacemaker: Results from the MARVEL 2 Study. Heart Rhythm. 2020 Dec;17(12):2037-2045. doi: 10.1016/j.hrthm.2020.07.024. Epub 2020 Jul 24.

repository_institution: University of Southern Denmark - University of Southern Denmark Research Portal

ReferenceCitation: Steinwender C, Khelae SK, Garweg C, Chan JYS, Ritter P, Johansen JB, Sagi V, Epstein LM, Piccini JP, Pascual M, Mont L, Sheldon T, Splett V, Stromberg K, Wood N, Chinitz L. Atrioventricular Synchronous Pacing Using a Leadless Ventricular Pacemaker: Results From the MARVEL 2 Study. JACC Clin Electrophysiol. 2020 Jan;6(1):94-106. doi: 10.1016/j.jacep.2019.10.017. Epub 2019 Nov 11.

lead_sponsor: Medtronic Cardiac Rhythm and Heart Failure

location_country:
Austria
Hong Kong
France
Malaysia
Belgium
Spain
Denmark
United States

location_facility: Hospital Universitari Clínic de Barcelona

location_facility: North Shore University Hospital- Northwell

location_facility: Kepler Universitätsklinikum Med Campus III


title: A Phase 1/2 Study to Evaluate the Safety and Efficacy of a Single Dose of Autologous CRISPR-Cas9 Modified CD34+ Human Hematopoietic Stem and Progenitor Cells (CTX001) in Subjects With Severe Sickle Cell Disease

summary: This is a single-arm, open-label, multi-site, single-dose Phase 1/2 study in subjects with severe sickle cell disease (SCD). The study will evaluate the safety and efficacy of autologous CRISPR-Cas9 Modified CD34+ Human Hematopoietic Stem and Progenitor Cells (hHSPCs) using CTX001.

ReferenceCitation: Brusson M, Miccio A. Genome editing approaches to β-hemoglobinopathies. Prog Mol Biol Transl Sci. 2021;182:153-183. doi: 10.1016/bs.pmbts.2021.01.025. Epub 2021 Mar 1.

family: Brusson

full_name: Mégane Brusson

given: Mégane

family: Miccio

full_name: Annarita Miccio

given: Annarita

bso_classification: Biology (fond.)

genre: book-chapter

genre_raw: book-chapter

journal_name: Curing Genetic Diseases Through Genome Reprogramming

publisher: Elsevier

ReferenceCitation: Frangoul H, Altshuler D, Cappellini MD, Chen YS, Domm J, Eustace BK, Foell J, de la Fuente J, Grupp S, Handgretinger R, Ho TW, Kattamis A, Kernytsky A, Lekstrom-Himes J, Li AM, Locatelli F, Mapara MY, de Montalembert M, Rondelli D, Sharma A, Sheth S, Soni S, Steinberg MH, Wall D, Yen A, Corbacioglu S. CRISPR-Cas9 Gene Editing for Sickle Cell Disease and β-Thalassemia. N Engl J Med. 2021 Jan 21;384(3):252-260. doi: 10.1056/NEJMoa2031054. Epub 2020 Dec 5.

name: From the Sarah Cannon Center for Blood Cancer at the Children’s Hospital at TriStar Centennial, Nashville (H.F., J.D.), and St. Jude Children’s Research Hospital, Memphis (A.S.) — both in Tennessee; Vertex Pharmaceuticals (D.A., B.K.E., J.L.-H., A.Y.) and Boston University School of Medicine (M.H.S.), Boston, and CRISPR Therapeutics, Cambridge (Y.-S.C., T.W.H., A. Kernytsky, S. Soni) — both in Massachusetts; the University of Milan, Milan (M.D.C.), and Ospedale Pediatrico Bambino Gesù Rome, Sapienza,...

family: Frangoul

full_name: Haydar Frangoul

given: Haydar

family: Altshuler

full_name: David Altshuler

given: David

family: Cappellini

full_name: M. Domenica Cappellini

given: M. Domenica

family: Chen

full_name: Yi-Shan Chen

given: Yi-Shan

family: Domm

full_name: Jennifer Domm

given: Jennifer

family: Eustace

full_name: Brenda K. Eustace

given: Brenda K.

family: Foell

full_name: Juergen Foell

given: Juergen

family: de la Fuente

full_name: Josu de la Fuente

given: Josu

family: Grupp

full_name: Stephan Grupp

given: Stephan

family: Handgretinger

full_name: Rupert Handgretinger

given: Rupert

family: Ho

full_name: Tony W. Ho

given: Tony W.

family: Kattamis

full_name: Antonis Kattamis

given: Antonis

family: Kernytsky

full_name: Andrew Kernytsky

given: Andrew

family: Lekstrom-Himes

full_name: Julie Lekstrom-Himes

given: Julie

family: Li

full_name: Amanda M. Li

given: Amanda M.

family: Locatelli

full_name: Franco Locatelli

given: Franco

family: Mapara

full_name: Markus Y. Mapara

given: Markus Y.

family: de Montalembert

full_name: Mariane de Montalembert

given: Mariane

family: Rondelli

full_name: Damiano Rondelli

given: Damiano

ReferenceCitation: Modarai SR, Kanda S, Bloh K, Opdenaker LM, Kmiec EB. Precise and error-prone CRISPR-directed gene editing activity in human CD34+ cells varies widely among patient samples. Gene Ther. 2021 Feb;28(1-2):105-113. doi: 10.1038/s41434-020-00192-z. Epub 2020 Sep 1.

location_country:
Italy
United States
Germany
France
Belgium
Canada
United Kingdom

location_facility: Lucille Packard Children's Hospital of Stanford University

location_facility: Columbia University Medical Center (21+ years)

location_facility: Columbia University Medical Center (≤21 years)

location_facility: Methodist Children's Hospital/Texas Transplant Institute

location_facility: Imperial College Healthcare NHS Trust, Hammersmith Hospital

location_facility: University of Illinois at Chicago Hospitals and Health Systems

location_facility: Hopital Universitaire des Enfants Reine Fabiola (HUDERF)

location_facility: Regensburg University Hospital, Clinic and Polyclinic for Paediatric and Adolescent Medicine, Paediatric Haemotology, Oncology and Stem Cell Transplantation

location_facility: The Children's Hospital at TriStar Centennial Medical Center/ Sarah Cannon Center for Blood Cancers

location_facility: Dipartimento di Onco-Ematologia e Terapia Cellulare e Genica Ospedale Pediatrico Bambino Gesu - IRCCS

location_facility: Royal London and St Bartholomew's Hospital, Pathology and Pharmacy Building

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


title: A Phase 1/2 Study of Oral LOXO-305 in Patients With Previously Treated Chronic Lymphocytic Leukemia/Small Lymphocytic Lymphoma (CLL/SLL) or Non-Hodgkin Lymphoma (NHL)

summary: This is an open-label, multi-center Phase 1/2 study of oral LOXO-305 in patients with CLL/SLL and NHL who have failed or are intolerant to standard of care.

ReferenceCitation: Kipps TJ. Mining the Microenvironment for Therapeutic Targets in Chronic Lymphocytic Leukemia. Cancer J. 2021 Jul-Aug 01;27(4):306-313. doi: 10.1097/PPO.0000000000000536. Review.

family: Kipps

full_name: Thomas J. Kipps

given: Thomas J.

journal_name: The Cancer Journal

publisher: Ovid Technologies (Wolters Kluwer Health)

publisher_dissemination: Wolters Kluwer Health

publisher_group: Wolters Kluwer Health

publisher_normalized: Wolters Kluwer Health

ReferenceCitation: Mato AR, Shah NN, Jurczak W, Cheah CY, Pagel JM, Woyach JA, Fakhri B, Eyre TA, Lamanna N, Patel MR, Alencar A, Lech-Maranda E, Wierda WG, Coombs CC, Gerson JN, Ghia P, Le Gouill S, Lewis DJ, Sundaram S, Cohen JB, Flinn IW, Tam CS, Barve MA, Kuss B, Taylor J, Abdel-Wahab O, Schuster SJ, Palomba ML, Lewis KL, Roeker LE, Davids MS, Tan XN, Fenske TS, Wallin J, Tsai DE, Ku NC, Zhu E, Chen J, Yin M, Nair B, Ebata K, Marella N, Brown JR, Wang M. Pirtobrutinib in relapsed or refractory B-cell malignancies (BRUIN): a phase 1/2 study. Lancet. 2021 Mar 6;397(10277):892-901. doi: 10.1016/S0140-6736(21)00224-5.

family: Mato

full_name: Anthony R Mato

given: Anthony R

family: Shah

full_name: Nirav N Shah

given: Nirav N

family: Jurczak

full_name: Wojciech Jurczak

given: Wojciech

family: Cheah

full_name: Chan Y Cheah

given: Chan Y

family: Pagel

full_name: John M Pagel

given: John M

family: Woyach

full_name: Jennifer A Woyach

given: Jennifer A

family: Fakhri

full_name: Bita Fakhri

given: Bita

family: Eyre

full_name: Toby A Eyre

given: Toby A

family: Lamanna

full_name: Nicole Lamanna

given: Nicole

family: Patel

full_name: Manish R Patel

given: Manish R

family: Alencar

full_name: Alvaro Alencar

given: Alvaro

family: Lech-Maranda

full_name: Ewa Lech-Maranda

given: Ewa

family: Wierda

full_name: William G Wierda

given: William G

family: Coombs

full_name: Catherine C Coombs

given: Catherine C

family: Gerson

full_name: James N Gerson

given: James N

family: Ghia

full_name: Paolo Ghia

given: Paolo

family: Le Gouill

full_name: Steven Le Gouill

given: Steven

family: Lewis

full_name: David John Lewis

given: David John

family: Sundaram

full_name: Suchitra Sundaram

given: Suchitra

family: Cohen

full_name: Jonathon B Cohen

given: Jonathon B

location_country:
Italy
Sweden
Switzerland
France
Japan
Poland
Australia
United States
United Kingdom
Korea, Republic of

location_facility: University of North Carolina at Chapel Hill

location_facility: Policlinico S. Orsola Malpighi - Universita di Bologna

location_facility: Smilow Cancer Hospital at Yale-New Haven

location_facility: Ospedale Regionale di Bellinzona e Valli - Bellinzona

location_facility: University of Texas MD Anderson Cancer Center

location_facility: Kyoto Furitsu Medical University Hospital

location_facility: Centre Hospitalier Universitaire de Nantes - L' Hopital l'hôtel-Dieu

location_facility: University of California San Francisco, Medical Center at Paranassus


title: Peanut Oral Immunotherapy Study of Early Intervention for Desensitization (POSEIDON)

acronym: POSEIDON

summary: The purpose of this study is to determine the efficacy and safety of AR101 in peanut-allergic children aged 1 to < 4 years.

lead_sponsor: Aimmune Therapeutics, Inc.

location_country:
Germany
United States
United Kingdom
France

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

location_facility: Sean N. Parker Center for Allergy & Asthma Reseach, LPCH at El Camino Hospital

location_facility: University Hospital Southampton Foundation NHS Trust Southampton General Hospital

location_facility: Allergy & Asthma Medical Group and Research Center

location_facility: Royal Manchester Children's Hospital Central Manchester University Hospitals

location_facility: University of Michigan Division of Allergy and Clinical Immunology

location_facility: Snowy Owl, First Floor, Evelina Childrens Hospital

location_facility: UNC-CH School of Medicine, Pediatric Allergy, Immunology & Rheumatology, Food Allergy

location_facility: Children's Center for Advanced Pediatrics Clinical Research Lab


title: A Phase 2, Open-Label, Multi-center Study of AL101 in Patients With Adenoid Cystic Carcinoma (ACC) Bearing Activating Notch Mutations

acronym: ACCURACY

summary: This is a Phase 2, non comparative, open label, multicenter study of AL101 in patients with recurrent or metastatic ACC who harbor NOTCH 1,2,3,4 activating mutations.

lead_sponsor: Ayala Pharmaceuticals, Inc,

location_country:
Netherlands
France
Israel
United States
United Kingdom
Canada

location_facility: Hamilton Health Sciences Juravinski Cancer Centre

location_facility: H. Lee Moffitt Cancer Center & Research Institute

location_facility: University of Maryland School of Medicine


title: Multi-center, Double-blind, Parallel-group, Randomized, Placebo-controlled, Three Doses, 40-week Extension to Studies ID-078A301 and ID-078A302 to Assess the Long Term Safety and Tolerability of ACT-541468 in Adult and Elderly Subjects With Insomnia Disorder

summary: Study to assess the long term safety and tolerability of ACT-541468 in adult and elderly subjects suffering from difficulties to sleep

lead_sponsor: Idorsia Pharmaceuticals Ltd.

location_country:
United States
Sweden
Switzerland
Germany
Finland
France
Bulgaria
Poland
Hungary
Belgium
Spain
Denmark
Canada
Korea, Republic of

location_facility: Sömnutredningsmottagningen, smärtcentrum Akademiska sjukhuset

location_facility: Acibadem City Clinic Tokuda Hospital EAD

location_facility: Central Insitute of Mental Health Sleep laboratory Medical Faculty Mannheim/Heidelberg University

location_facility: CRIUSMQ- CIUSSSCN, CETS (clinique du sommeil)

location_facility: Oregon Center for Clinical Investigations,Inc

location_facility: Clinical Trial Center North GmbH & Co. KG

location_facility: Centro Médico Teknon - Medicina del Sueño

location_facility: Göteborgs Universitet, Centrum för sömn och vakenhetsstörningar

location_facility: Hospital MAZ - Neurophisiology and Sleep Department

location_facility: Magyar Honvédség Egészségügyi Központ, Neurológiai Osztály

location_facility: Ośrodek Medycyny Study Nurseu Instytutu Psychiatrii i Neurologii (Sleep Disorders Center)

location_facility: Zentrum für Schlafmedizin Zürcher Oberland, Zürcher RehaZentrum Wald

location_facility: Hospital UZ Leuven_ Pneumology Department

location_facility: St Hedwig-Krankenhaus, Klinik für Schlaf- und Chronomedizin

location_facility: ZMS Zentrum für medizinische Studien GmbH

location_facility: Hospital Universitario Araba - Unidad Funcional de Trastornos del Sueño

location_facility: St. Francis Sleep Allergy and Lung Institute

location_facility: Clinical Research Group of St. Petersburgh

location_facility: Fleming Island Center for Clinical Research

location_facility: 4F Neuroimaging analysis laboratory, 56 Dalseong-ro, Jung-gu

location_facility: EMC Instytut Medyczny SA, Przychodnia przy Łowieckiej

location_facility: Klinikum Rechts der lsar TU München Dept. of Psychiatry and Psychotherapy

location_facility: 2F Psychiatry Outpatient, 93, Jungbu-daero, Paldal-gu

location_facility: Universitetssjukhuset Örebro Neurokliniken, Sömnenheten

location_facility: Klinik und Poliklinik für Psychiatrie, Psychosomatik und Psychotherapie der Universität am Bezirksklinikum Regensburg

location_facility: Klinik imd Poliklinik fur Neurochirurgie

location_facility: Hospital Universitari Vall d'Hebron - Neurophisiology Deparment - Sleep Unit

location_facility: Queensway Sleep Lab Sleep Clinic (MedSleep)

location_facility: Zentrum für Integrative Psychiatrie (ZiP) Universität zu Lübeck

location_facility: Jacksonville Center for Clinical Research

location_facility: Pulmonary Associates of the Southeast/WCR

location_facility: Sleep Disorders Centers of the Mid-Atlantic

location_facility: Charité - Universitätsmedizin Berlin - Campus Benjamin Franklin Kompetenzzentrum Schlafmedizin

location_facility: Artemis Institute For Clinical Research - Riverside

location_facility: Sleep Disorders Center of the Mid-Atlantic

location_facility: SOMNIBENE Institut für Medizinische Forschung und Schlafmedizin Schwerin GmbH