title: A Double-Blind, Placebo-Controlled, Multi-Center Study With an Open-Label Extension to Evaluate the Efficacy and Safety of SRP-4045 and SRP-4053 in Patients With Duchenne Muscular Dystrophy

acronym: ESSENCE

summary: The main objective of this study is to evaluate the efficacy of SRP-4045 and SRP-4053 compared to placebo in participants with DMD with out-of-frame deletion mutations amenable to skipping exon 45 and exon 53, respectively.

ReferenceCitation: Wagner KR, Kuntz NL, Koenig E, East L, Upadhyay S, Han B, Shieh PB. Safety, tolerability, and pharmacokinetics of casimersen in patients with Duchenne muscular dystrophy amenable to exon 45 skipping: A randomized, double-blind, placebo-controlled, dose-titration trial. Muscle Nerve. 2021 Sep;64(3):285-292. doi: 10.1002/mus.27347. Epub 2021 Jun 29.

name: Center for Genetic Muscle Disorders Kennedy Krieger Institute  Baltimore Maryland USA

name: Departments of Neurology and Neuroscience Johns Hopkins School of Medicine  Baltimore Maryland USA

family: Wagner

full_name: Kathryn R. Wagner

given: Kathryn R.

name: Ann & Robert H. Lurie Children's Hospital  Chicago Illinois USA

family: Kuntz

full_name: Nancy L. Kuntz

given: Nancy L.

name: Clinical Development, Sarepta Therapeutics, Inc  Cambridge Massachusetts USA

family: Koenig

full_name: Erica Koenig

given: Erica

name: Clinical Pharmacology, Sarepta Therapeutics, Inc  Cambridge Massachusetts USA

family: East

full_name: Lilly East

given: Lilly

name: Pharmacovigilance, Sarepta Therapeutics, Inc  Cambridge Massachusetts USA

family: Upadhyay

full_name: Sameer Upadhyay

given: Sameer

name: Biostatistics, Sarepta Therapeutics, Inc  Cambridge Massachusetts USA

family: Han

full_name: Baoguang Han

given: Baoguang

name: Department of Neurology University of California, Los Angeles  California USA

family: Shieh

full_name: Perry B. Shieh

given: Perry B.

journal_name: Muscle & Nerve

publisher: Wiley

publisher_dissemination: Wiley

publisher_group: Wiley

publisher_normalized: Wiley

title: Safety, tolerability, and pharmacokinetics of casimersen in patients with D uchenne muscular dystrophy amenable to exon 45 skipping: A randomized, double‐blind, placebo‐controlled, dose‐titration trial

lead_sponsor: Sarepta Therapeutics, Inc.

location_country:
Italy
United States
Sweden
Serbia
Spain
Germany
France
Bulgaria
Czechia
Greece
Israel
Poland
Hungary
Belgium
Australia
Ireland
Russian Federation
Denmark
Canada
United Kingdom

location_facility: Cincinnati Children's Hospital Medical Center (CCHMC)

location_facility: Clinic for Neurology and Psychiatry for Children and Youth

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

location_facility: University Multiprofile Hospital for Active Treatment Aleksandrovska EAD

location_facility: Fondazione IRCCS Istituto Neurologico Carlo Besta

location_facility: Rady Children's Hospital San Diego/ UCSD

location_facility: Semmelweis Egyetem Genomikai Medicina és Ritka Betegsegek Intezete

location_facility: Children's and Women's Health Centre of British Columbia

location_facility: Ippokratio General Hospital of Thessaloniki

location_facility: Samodzielny Publiczny Centralny Szpital Kliniczny

location_facility: Children's Hospital of Pittsburgh of UPMC

location_facility: Hospital Universitari i Politecnic La Fe de Valencia

location_facility: Rigshospitalet Copenhagen University Hospital

location_facility: Federal state budget educational institution of higher education "Russian national research medical university n.a. N.I. Pirogov" of Ministry of healthcare of Russian Federation

location_facility: Schneider Children's Medical Center of Israel

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

location_facility: Stanford University School of Medicine/Medical Center

location_facility: Reference Centre for Neuromuscular Diseases

location_facility: University of Rochester Clinical Research Center

location_facility: Drottning Silvias Barn Och Ungdomssjukhus

location_facility: Center for Integrative Rare Disease Research (CIRDR)

location_facility: Azienda Ospedaliero-Universitaria di Ferrara - Arcispedale Sant' Anna


title: A Retrospective Observational Multicenter Study on the Management and Outcome of Patients With Systemic AL Amyloidosis in Europe

summary: This is a retrospective, observational, multicenter study to collect Real-World Evidence (RWE) data on systemic AL-AMY patients in Europe. Data from paper/electronic medical records and/or electronic databases from key reference centers in Europe will be used. Data will either be entered by the site staff in the electronic Case Report Form (eCRF) or, where feasible, transferred directly, always in accordance to local regulations.

study_start_date_type: Actual

status: Recruiting

study_completion_date_type: Anticipated

study_type: Observational

time_perspective: Retrospective

enrollment_type: Anticipated

ipd_sharing: Undecided

lead_sponsor: European Myeloma Network

location_country:
Italy
Austria
Netherlands
Spain
France
Czechia
Greece
Portugal
Germany
United Kingdom

location_facility: Service de Néphrologie, Hémodialyse Et Transplantation Rénale, Hôpital Jean Bernard, Chu Poitiers

location_facility: Centro Per Lo Studio E La Cura Delle Amiloidosi Sistemiche Padiglione Forlanini

location_facility: University Medical Center Utrecht, Umc Utrecht, Department of Hematology

location_facility: Alexandra Hospital, University of Athens School of Medicine, Department of Clinical Therapeutics

location_facility: Centre Hospitalier Universitaire de Limoges, Department of Clinical Hematology and Cell Therapy

location_facility: Universität Heidelberg, Department of Internal Medicine V: Hematology, Oncology and Rheumatology

location_facility: Allgemeines Krankenhaus Der Stadt Wien (General Hopsital), Universitätsklinik Für Innere Medizin I Klinische Abteilung Für Onkologie (Clinic of Internal Medicine I), Medizinische Universität Wien

location_facility: Fundação Champalimaud, Hematology Research, Imunology - Medical School, Nova University

location_facility: University Hospital Ostrava, Clinic of Hematooncology

location_facility: Centro Hospitalar E Universitário São João

location_facility: Servicio de Hematología, Hospital Clínic de Barcelona

location_facility: University College London Medical School, Uk Royal Free Hospital

lead_sponsor_type: industriel

status_simplified: Ongoing


type: EudraCT Number

type: Other Identifier

title: A 104-Week, Multicenter, Single-Arm, Long-Term, Phase 3 Extension Trial Investigating the Safety and Efficacy of Glepaglutide in Adult Patients With Short Bowel Syndrome (SBS) Completing the EASE SBS 2 Trial

acronym: EASE SBS 3

summary: This trial is an extension trial to EASE SBS 2. The study looks at whether glepaglutide is a safe treatment for participants with Short Bowel Syndrome (SBS), as well as how well effectiveness is maintained during long term treatment. Participants in this trial will receive glepaglutide as once-weekly injections under the skin (subcutaneous, s.c.) for approximately 2 years.

status: Enrolling by invitation

study_type: Interventional

primary_purpose: Treatment

ipd_sharing: No

lead_sponsor: Zealand Pharma

location_country:
United States
Belgium
France
Poland

location_facility: Wojewodzki Specjalistyczny Szpital im. M. Pirogowa w Lodzi

location_facility: Szpital Skawina sp. z o.o. im. Stanley Dudricka

intervention_type: Drug

status_simplified: Unknown


title: A Prospective, Open, Non-comparative, Post-market Clinical Follow-up Investigation to Confirm Safety and Performance of Avance Solo NPWT System in Low to Moderate Exuding Acute (Traumatic Wounds and Flaps and Grafts), as Well as Subacute (e.g., Dehisced Wounds) Wounds

acronym: ASOLO-SW

summary: The primary objective of this post market clinical follow-up (PMCF) investigation is to confirm safety and performance of Avance® Solo NPWT System in low to moderate exuding acute (traumatic wounds and flaps and grafts), as well as subacute (e.g., dehisced wounds) wounds when used in accordance with the Instructions for Use, for up to 28 days.

design_allocation: Non-Randomized

lead_sponsor: Molnlycke Health Care AB

location_country: Italy Germany Belgium France

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

intervention_type: Device


design_allocation: Randomized

intervention_type: Biological

status: Active, not recruiting

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

lead_sponsor: Novartis Pharmaceuticals

enrollment_type: Actual

location_country:
Italy
Guatemala
Switzerland
United Kingdom
Germany
France
Japan
Poland
Israel
Romania
Hungary
Belgium
Russian Federation
Egypt
Spain
Estonia
United States
Latvia
Colombia

ipd_sharing: Yes

source: euctr

title: A Randomized, Double-blind, Placebo- and Active Controlled Multicenter Trial to Demonstrate Efficacy of Subcutaneous Secukinumab Compared to Placebo and Etanercept (in a Single-blinded Arm) After Twelve Weeks of Treatment, and to Assess the Safety, Tolerability, and Long-term Efficacy in Subjects From 6 to Less Than 18 Years of Age With Severe Chronic Plaque Psoriasis

summary: This was a multicenter, randomized, double-blind, placebo- and active-controlled (etanercept in single blinded arm) study in pediatric subjects aged 6 years to less than 18 years with severe chronic plaque psoriasis. Approximately 160 subjects aged 6 years to <18 years were enrolled, of which at least 30 were 6 years to <12 years old. Subjects were enrolled at approximately 70 study sites worldwide.


title: Setting a Benchmark for Resource Utilization and Quality of Care in Patients Undergoing Transcatheter Aortic Valve Implantation in Europe - The BENCHMARK Registry

acronym: BENCHMARK

summary:
Non-interventional, multi-center, international registry in patients with severe aortic stenosis (sAS) undergoing transcatheter aortic valve implantation (TAVI). The BENCHMARK Registry aims to implement standardized, reproducible and scalable quality improvement measures, which will help to reduce resource requirements, intensive care unit bed occupancy and overall length of hospitalization with uncompromised patient safety.

The registry will consist of the following phases:

Retrospective phase (purpose: capture the status quo of each site prior to any Quality of Care improvement measures): The TAVI of patients being eligible for retrospective documentation must have been performed prior to the site's enrollment into the BENCHMARK prospective phase (follow-up: 30 days, 12 months).
Education phase: Each center will undergo an online BENCHMARK education. The contents of the education will be: Reflection on treatment pathways with the TAVI team, education on 8 defined Quality of Care improvement measures, followed by a center self-assessment (to self-assess the current alignment of hospital performance with the BENCHMARK Quality od Care measures).
Implementation phase: After having self-assessed the current alignment of hospital performance with the following BENCHMARK Quality of care measures, each center will start to introduce the tailored Quality of Care improvement measures to their hospital routine within a time frame of 2 months.
Prospective phase (purpose: to document the effects of defined quality of care improvement measure and to make sure patient safety is as least as good as in the retrospective phase. (8 months of recruitment, Follow-up: 30 days, 12 months)

time_perspective: Prospective

ReferenceType: derived

ReferenceCitation: McCalmont G, Durand E, Lauck S, Muir DF, Spence MS, Vasa-Nicotera M, Wood D, Saia F, Chatel N, Lüske CM, Kurucova J, Bramlage P, Frank D. Setting a benchmark for resource utilization and quality of care in patients undergoing transcatheter aortic valve implantation in Europe-Rationale and design of the international BENCHMARK registry. Clin Cardiol. 2021 Oct;44(10):1344-1353. doi: 10.1002/clc.23711. Epub 2021 Sep 9.

name: Cardiology Department James Cook University Hospital  Middlesbrough UK

family: McCalmont

full_name: Gemma McCalmont

given: Gemma

sequence: first

name: Department of Cardiology, FHU CARNAVAL Normandie University, UNIROUEN  Rouen France

family: Durand

full_name: Eric Durand

given: Eric

sequence: additional

name: Center for Heart Valve Innovation, St Paul's Hospital University of Vancouver  Vancouver British Columbia Canada

family: Lauck

full_name: Sandra Lauck

given: Sandra

family: Muir

full_name: Douglas F. Muir

given: Douglas F.

name: Cardiology Department, Royal Victoria Hospital  Belfast UK

family: Spence

full_name: Mark S. Spence

given: Mark S.

name: Cardiology Department Frankfurt University  Frankfurt Germany

family: Vasa‐Nicotera

full_name: Mariuca Vasa‐Nicotera

given: Mariuca

family: Wood

full_name: David Wood

given: David

name: Department of Cardiology University of Bologna  Bologna Italy

family: Saia

full_name: Francesco Saia

given: Francesco

family: Chatel

full_name: Nicolas Chatel

given: Nicolas

name: Institute for Pharmacology and Preventive Medicine  Cloppenburg Germany

family: Lüske

full_name: Claudia M. Lüske

given: Claudia M.

name: Edwards Lifesciences  Prague Czech Republic

family: Kurucova

full_name: Jana Kurucova

given: Jana

family: Bramlage

full_name: Peter Bramlage

given: Peter

name: Department of Internal Medicine III (Cardiology, Angiology and Intensive Care Medicine) University Clinical Center Schleswig‐Holstein (UKSH)  Kiel Germany

name: German Center for Cardiovascular Research, partner site Hamburg/Kiel/Lübeck  Kiel Germany

family: Frank

full_name: Derk Frank

given: Derk

bso_classification: Medical research

datasource: user

genre: journal-article

genre_raw: journal-article

journal_name: Clinical Cardiology

oa_colors: green gold

oa_host_type: publisher;repository

evidence: oa journal (via doaj)

host_type: publisher

license: cc-by

license_normalized: cc-by

version: publishedVersion

evidence: oa repository (via pmcid lookup)

host_type: repository

license_normalized: no license

repository_normalized: PubMed Central

title: Setting a benchmark for resource utilization and quality of care in patients undergoing transcatheter aortic valve implantation in Europe—Rationale and design of the international BENCHMARK registry

ipd_sharing_description: no individual participant data (IPD) will be shared

lead_sponsor: Institut für Pharmakologie und Präventive Medizin

location_country: Italy Austria France Czechia Romania Spain Germany

location_facility: Centre Hospitalier Universitaire de Besancon

location_facility: Azienda Ospedaliera Ordine Mauriziano di Torino

location_facility: Centre Hospitalier Universitaire de Montpellier

location_facility: Institutul de Urgenta pentru Boli Cardiolvasculaire

intervention_type: Other


title: A First-in-human, Two-part Clinical Study to Assess the Safety, Tolerability and Activity of IV Doses of ICT01 as Monotherapy and in Combination With a Checkpoint Inhibitor, in Patients With Advanced-stage, Relapsed/Refractory Cancer

acronym: EVICTION

summary: Part 1 will be a dose escalation study of IV ICT01 (a monoclonal antibody targeting BTN3A) as monotherapy in patients with advanced solid or hematologic tumors, followed by a cohort examining the combination of ICT01 plus pembrolizumab (Keytruda). Part 2 will be a cohort expansion into 2-3 solid tumor indications and one hematologic malignancy for ICT01 monotherapy, and 1-2 solid tumor indication receiving ICT01 plus pembrolizumab.

lead_sponsor: ImCheck Therapeutics

location_country:
United States
France
Belgium
Spain
Germany
United Kingdom

location_facility: University Carl Gustav Carus Clinical Trial Unit


title: A Phase 1/2 Trial of a Novel Therapeutic Vaccine (EO2401) in Combination With Immune Check Point Blockade, for Treatment of Patients With Locally Advanced or Metastatic Adrenocortical Carcinoma, or Malignant Pheochromocytoma/Paraganglioma

acronym: Spencer

summary: This is a multicenter, Phase 1/2, First-In-Human study to assess the safety, tolerability, immunogenicity, and preliminary efficacy of EO2401 in Metastatic Adrenocortical Carcinoma, or Malignant Pheochromocytoma/Paraganglioma.

lead_sponsor: Enterome

location_country:
Italy
Sweden
Netherlands
Germany
France
Spain
Denmark
United States

location_facility:
Centre Léon Bérard
MD Anderson Cancer Center
Máxima Medisch Centrum
Rigshospitalet
Universitätsklinikum Würzburg
Lmu Klinikum
Karolinska University Hospital
Hospital Universitari Vall d'Hebron
Azienda Ospedaliera Spedali Civili
Institut Gustave Roussy


title: Phase 3, Single-arm, Open-label, Multidose, Titration, PK, PD, and Safety Study of Etelcalcetide in Children and Adolescents ≥ 2 to < 18 Years of Age With Secondary HPT and Chronic Kidney Disease Receiving Maintenance Hemodialysis

summary: Assess the efficacy, safety, pharmacokinetics (PK) and pharmacodynamics (PD) of etelcalcetide in the treatment of secondary hyperparathyroidism (SHPT) in pediatric subjects between ≥ 2 to < 18 years of age, with chronic kidney disease (CKD) on hemodialysis

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
Spain
France
Czechia
Greece
Lithuania
Poland
Hungary
Belgium
Portugal
Germany
United Kingdom

location_facility: Childrens Hospital, Affiliate of Vilnius University Hospital Santaros Klinikos

location_facility: Hospices Civils de Lyon Hopital Femme Mere Enfant

location_facility: Ippokrateio General Hospital of Thessaloniki

location_facility: Uniwersytecki Szpital Dzieciecy w Krakowie

location_facility: Szegedi Tudomanyegyetem Szent-Gyorgyi Albert Klinikai Kozpont Altalanos Orvostudomanyi Kar

location_facility: Centro Hospitalar Universitario do Porto - Hospital de Santo Antonio

location_facility: Great Ormond Street Hospital for Children

location_facility: Zentrum für Kinder und Jugendmedizin des Universitätsklinikums Heidelberg

location_facility: General Children Hospital Panagioti and Aglaias Kyriakou


title: A Multicenter Phase II Clinical Trial of Lurbinectedin (PM01183) in Selected Advanced Solid Tumors

summary: Multicenter, open-label, exploratory, phase II clinical trial to evaluate the efficacy and safety of PM01183 in previously treated patients with advanced solid tumors

status: Completed

study_completion_date_type: Actual

ReferenceCitation: Fernández-Teruel C, Fudio S, Lubomirov R. Integrated exposure-response analysis of efficacy and safety of lurbinectedin to support the dose regimen in small-cell lung cancer. Cancer Chemother Pharmacol. 2021 Nov 5. doi: 10.1007/s00280-021-04366-3. [Epub ahead of print]

family: Fernández-Teruel

full_name: Carlos Fernández-Teruel

given: Carlos

family: Fudio

full_name: Salvador Fudio

given: Salvador

family: Lubomirov

full_name: Rubin Lubomirov

given: Rubin

journal_name: Cancer Chemotherapy and Pharmacology

oa_host_type: publisher

evidence: open (via crossref license)

publisher: Springer Science and Business Media LLC

publisher_dissemination: Springer-Nature

publisher_group: Springer-Nature

publisher_normalized: Springer

title: Integrated exposure–response analysis of efficacy and safety of lurbinectedin to support the dose regimen in small-cell lung cancer

ReferenceCitation: Subbiah V, Paz-Ares L, Besse B, Moreno V, Peters S, Sala MA, López-Vilariño JA, Fernández C, Kahatt C, Alfaro V, Siguero M, Zeaiter A, Zaman K, López R, Ponce S, Boni V, Arrondeau J, Delord JP, Martínez M, Wannesson L, Antón A, Valdivia J, Awada A, Kristeleit R, Olmedo ME, Rubio MJ, Sarantopoulos J, Chawla SP, Mosquera-Martinez J, D' Arcangelo M, Santoro A, Villalobos VM, Sands J, Trigo J. Antitumor activity of lurbinectedin in second-line small cell lung cancer patients who are candidates for re-challenge with the first-line treatment. Lung Cancer. 2020 Dec;150:90-96. doi: 10.1016/j.lungcan.2020.10.003. Epub 2020 Oct 10.

family: Subbiah

full_name: Vivek Subbiah

given: Vivek

family: Paz-Ares

full_name: Luis Paz-Ares

given: Luis

family: Besse

full_name: Benjamin Besse

given: Benjamin

family: Moreno

full_name: Victor Moreno

given: Victor

family: Peters

full_name: Solange Peters

given: Solange

family: Sala

full_name: María Angeles Sala

given: María Angeles

family: López-Vilariño

full_name: José Antonio López-Vilariño

given: José Antonio

family: Fernández

full_name: Cristian Fernández

given: Cristian

family: Kahatt

full_name: Carmen Kahatt

given: Carmen

family: Alfaro

full_name: Vicente Alfaro

given: Vicente

family: Siguero

full_name: Mariano Siguero

given: Mariano

family: Zeaiter

full_name: Ali Zeaiter

given: Ali

family: Zaman

full_name: Khalil Zaman

given: Khalil

family: López

full_name: Rafael López

given: Rafael

family: Ponce

full_name: Santiago Ponce

given: Santiago

family: Boni

full_name: Valentina Boni

given: Valentina

family: Arrondeau

repository_institution: Université Libre de Bruxelles - Dépôt institutionnel de l'Université libre de Bruxelles

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

ReferenceCitation: Trigo J, Subbiah V, Besse B, Moreno V, López R, Sala MA, Peters S, Ponce S, Fernández C, Alfaro V, Gómez J, Kahatt C, Zeaiter A, Zaman K, Boni V, Arrondeau J, Martínez M, Delord JP, Awada A, Kristeleit R, Olmedo ME, Wannesson L, Valdivia J, Rubio MJ, Anton A, Sarantopoulos J, Chawla SP, Mosquera-Martinez J, D'Arcangelo M, Santoro A, Villalobos VM, Sands J, Paz-Ares L. Lurbinectedin as second-line treatment for patients with small-cell lung cancer: a single-arm, open-label, phase 2 basket trial. Lancet Oncol. 2020 May;21(5):645-654. doi: 10.1016/S1470-2045(20)30068-1. Epub 2020 Mar 27. Erratum in: Lancet Oncol. 2020 Dec;21(12):e553.

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

location_facility: Complejo Hospitalario de Especialidades Virgen de la Victoria

location_facility: Hospital Universitario Virgen de las Nieves

location_facility: Istituto Oncologico della Svizzera Italiana (IOSI)

location_facility: Complexo Hospitalario Universitario de Santiago

location_facility: Institute for Drug Development, Cancer Therapy & Research Center at University of Texas Health Science Center

location_facility: Hospital Universitari i Polotècnic la Fe

location_facility: Hospital Universitario Madrid Sanchinarro

location_facility: ASST Monza - Ospedale San Gerardo di Monza Struttura Complessa di Oncologia Medica

location_facility: AUSL Romagna - Ospedale Santa Maria delle Croci

location_facility: The University of Texas MD Anderson Cancer Center

location_facility: Complexo Hospitalario Universitario A Coruña

location_facility: Charité Universitätsmedizin Berlin - Campus Benjamin Franklin - Comprehensive Cancer Center

location_facility: Hopsital Universitario Donostia - Donostia Unibertsitate Ospitalea

location_facility: Centre Hospitalier Universitaire Vaudois

location_facility: Hospital Universitario Central de Asturias

location_facility: Complejo Hospitalario Regional Reina Sofía

location_facility: Hospital Universitario Puerta de Hierro Majadahonda

location_facility: Hospital Universitario Fundación Jiménez Díaz


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: Genzyme, a Sanofi Company

location_country:
Italy
Austria
Netherlands
France
Poland
Belgium
Russian Federation
Portugal
Turkey
United Kingdom

acronym: LemKids

location_facility:
Investigational Site Number :0560001
Investigational Site Number :5280001
Investigational Site Number :2500001
Investigational Site Number :7920001
Investigational Site Number :6430004
Investigational Site Number :6200001
Investigational Site Number :8260002
Investigational Site Number :7920002
Investigational Site Number :6160002
Investigational Site Number :6430005
Investigational Site Number :6430001
Investigational Site Number :6160001
Investigational Site Number :7920003
Investigational Site Number :6160003
Investigational Site Number :3800004
Investigational Site Number :7920004
Investigational Site Number :6430002
Investigational Site Number :2500002
Investigational Site Number :0400001
Investigational Site Number :3800001
Investigational Site Number :3800005

title: A Multi-center, Open-label, Single-arm, Before and After Switch Study to Evaluate the Efficacy, Safety and Tolerability of Alemtuzumab in Paediatric Patients With Relapsing Remitting Multiple Sclerosis (RRMS) With Disease Activity on Prior Disease Modifying Therapy (DMT)

summary:
Primary Objective:

To evaluate the efficacy, safety, and tolerability of alemtuzumab intravenously (IV) in pediatric patients from 10 to <18 years of age with Relapsing Remitting Multiple Sclerosis (RRMS) who have disease activity on prior Disease Modifying Therapy (DMT).

Secondary Objective:

To assess the pharmacokinetics (PK), pharmacodynamics (PD), anti-drug antibody (ADA) formation, and potential effects of alemtuzumab on other multiple sclerosis (MS) disease characteristics such as cognition and quality of life (QoL).


title: A Phase 3, Multicenter, Long Term, Extension Study of the Safety and Efficacy of AVP-786 (Deuterated [d6] Dextromethorphan Hydrobromide [d6-DM]/Quinidine Sulfate [Q]) for the Treatment of Agitation in Patients With Dementia of the Alzheimer's Type

summary: This is an extension study of the Phase 3 Studies 15-AVP-786-301, 15-AVP-786-302, and 17-AVP-786-305, which also allows participants from the Phase 2 Study 12-AVR-131 to be included.

lead_sponsor: Avanir Pharmaceuticals

location_country:
Italy
South Africa
France
Czechia
Bulgaria
Poland
Hungary
Spain
United States
Canada

location_facility: MidAmerica Neuroscience Research Foundation

location_facility: Psychiatry and Alzheimer's Care of Rochester, PLLC

location_facility: Dr. Kenessey Albert Kórház-Rendelőintézet

location_facility: Neurology Research Institute Palm Beach, LLC #2

location_facility: Neurology and Neuroscience Associates, Inc.

location_facility: Lytle and Weiss, PLLC dba Clinical Trials of the Rockies

location_facility: Clinical Neuroscience Research Associates, Inc. dba The Memory Clinic

location_facility: Clinical Research Associates of South Florida #2

location_facility: The Samuel & Alexia Bratton Memory Clinic

location_facility: Complejo Asistencial de Zamora Hospital Virgen de la Concha

location_facility: Columbus Research & Wellness Institute, INC

location_facility: Centre Hospitalier Universitaire Dijon Bourgogn

location_facility: Alexian Brothers Neurosciences Institute

location_facility: Cleveland Clinic Lou Ruvo Center for Brain Health at Lakewood Hospital

location_facility: Texas Medical Research Associates, L.L.C.

location_facility: Centre Hospitalier Universitaire de Saint-Étienne - Hôpital Nord

location_facility: University Texas Southwestern Medical Center

location_facility: Eastside Comprehensive Medical Center, LLC

location_facility: Debreceni Egyetem Kenezy Gyula Egyetemi Korhaz

location_facility: Wielospecjalistyczna Poradnia Lekarska Synapsis

location_facility: Research Center for Clinical Studies, Inc.

location_facility: Nathan S. Kline Institute for Psychiatric Research #2

location_facility: Centre Hospitalier Universitaire Toulouse - Casselardit Ancely

location_facility: New Foundation Medical Group Clinical Trials

location_facility: Medical Research & Health Education Foundation, Inc.

location_facility: Neurology Research Institute Palm Beach, LLC #1

location_facility: Premier Clinical Research Institute, Inc. #2

location_facility: Alzheimer's Research and Treatment Center #2

location_facility: Collaborative Neuroscience Network, LLC.

location_facility: University Multiprofile Hospital for Active Treatment Alexandrovska

location_facility: The Birches at Newton / Family Medical Associates

location_facility: Alliance for Wellness, Inc dba Alliance for Research

location_facility: Pharmaceuticals Research Associates, Inc.

location_facility: Fondazione Santa Lucia - Istituto di Ricovero e Cura a Carattere Scientifico

location_facility: Alzheimer's Research and Treatment Center #1

location_facility: Veteran Affairs Medical Center, Salem Virginia

location_facility: Rhode Island Mood & Memory Research Institute

location_facility: Josephson Wallack Munshower Neurology, PC

location_facility: Clinical Research Associates of South Florida #1

location_facility: Houston Methodist Neurological Institute

location_facility: Miami Dade Medical Research Institute, LLC

location_facility: ANI Neurology PLLC dba Alzheimer's Memory Center

location_facility: Alzheimer's Research and Treatment Center #3

location_facility: Michigan State University Department of Neurology

location_facility: Pszichiatriai es Pszichiatriai Rehabilitacios Osztaly

location_facility: Flora Baker Center for Alzheimer's Disease and Related Disorders

location_facility: Premier Clinical Research Institute, Inc. #1

location_facility: Nathan S. Kline Institute for Psychiatric Research #1


title: Phase I Study of Intratumoral CV8102 in Patients With Advanced Melanoma, Squamous Cell Carcinoma of the Skin, Squamous Cell Carcinoma of the Head and Neck, or Adenoid Cystic Carcinoma

summary:
This study evaluates intratumoral administration of CV8102 in patients with advanced melanoma, squamous cell carcinoma of the skin, squamous cell carcinoma of the head and neck, or adenoid cystic carcinoma.

Patients will receive CV8102 as single agent or in combination with SoC anti-PD-1 therapy.

lead_sponsor: CureVac AG

location_country:
Austria
France
Russian Federation
Spain
Germany

location_facility: Hospital Universitario Virgen de la Victoria

location_facility: Universitätsklinikum Schleswig-Holstein, Klinik für Dermatologie, Allergologie und Venerologie

location_facility: Universitätsklinikum Münster, Klinik für Hautkrankheiten, ZiD- Zentrum für innovative Dermatologie

location_facility: Saint-Petersburg State University, Clinic of advanced medical technologies n. a. Nicolay I. Pirogov.

location_facility: Center for Personalized Oncology, I.M. Sechenov First Moscow State Medical University of the Ministry of Health of the Russian Federation

location_facility: Nationales Centrum für Tumorerkrankungen (NCT) Heidelberg

location_facility: Hospital Universitario Marqus de Valdecilla Santander

location_facility: FSBI "National Medical Research Oncology Center n.a. N.N. Petrov

location_facility: Universitätsklinik für Dermatologie der Paracelsus medizinischen Privatuniversität Salzburg

location_facility: FSBI "National Medical Research Center of Oncology n.a. N.N. Blokhin" of the Ministry of Healthcare of the Russian Federation

location_facility: Universitäts-Hautklinik, Abtl. Dermatologische Onkologie

location_facility: Medizinische Klinik III, Universitätsklinikum Bonn, Hämatologie, Immunonkologie und Rheumatologie

location_facility: Universitätsklinikum Erlangen,Hautklinik, Internistisches Zentrum (INZ)

location_facility: Hospital Duran i Reynals - Institut Catala dOncologia ICO


title: A Randomized, Double-blind, Placebo-controlled, Dose-ranging, Study to Evaluate the Efficacy, Safety and Tolerability of Single Doses of BCX7353 as an Acute Attack Treatment in Subjects With Hereditary Angioedema

summary: This 3-part study will evaluate the efficacy and safety of an oral kallikrein inhibitor, BCX7353, in the treatment angioedema attacks in subjects with Type I or II hereditary angioedema (HAE). In each study part, subjects will treat 3 attacks with BCX7353 (2 attacks) or placebo (1 attack), in a randomly allocated order. In Part 1, the dose of 750mg will be assessed relative to placebo in up to 36 patients. If this is shown to be effective, then a further 12 patients will be enrolled at a 500mg dose (Part 1), followed by a further 12 (if efficacy still shown) at a dose of 250mg (Part 3) to determine the minimum effective dose of BCX7353 compared to placebo for treating HAE attacks. Efficacy will be determined by subject diary entries completed at pre-defined times post-dose.

lead_sponsor: BioCryst Pharmaceuticals

location_country:
Italy
Austria
Switzerland
North Macedonia
France
Poland
Israel
Romania
Hungary
Denmark
Germany
United Kingdom

location_facility:
Study center
Study Center

status_simplified: Completed


status: Terminated

lead_sponsor: Hoffmann-La Roche

location_country:
Italy
United States
Serbia
France
Czechia
Greece
Poland
Romania
Hungary
Russian Federation
Spain
Germany
United Kingdom
Korea, Republic of

location_facility: Jasz-Nagykun-Szolnok Megyei Hetenyi Geza Korhaz-Rend.Int.

location_facility: A.O.U.I. VERONA-OSPEDALE BORGO TRENTO; ONCOLODIA MEDICA-d.O.

location_facility: Penn State Milton S. Hershey Medical Center

location_facility: Arkhangelsk Regional Clinical Oncology Dispensary

location_facility: California Cancer Associates for Research & Excellence, Inc.

location_facility: Irccs Istituto Nazionale Dei Tumori (Int);S.C. Medicina Oncologica 2

location_facility: Filial #1 Regional Oncology Dispensary of Nizhniy Novgorod

location_facility: Clin Hospital Center - Kragujevac; Pulmonary Diseases

location_facility: Dr Constantin Opris Emergency County Hospital Baia Mare

location_facility: Chao Family Comprehensive Cancer Center; UC Irvine Medical Center

location_facility: IRST Istituto Scientifico Romagnolo Per Lo Studio E Cura Dei Tumori, Sede Meldola; Oncologia Medica

location_facility: Azienda Ospedaliera Istituti Ospitalieri

location_facility: Sibiu Emergency Clinical County Hospital

location_facility: Azienda Ospedaliera Città della Salute e della Scienza di Torino

location_facility: Carolina Oncology Specialists, PA - Hickory

location_facility: Seoul National University Bundang Hospital

location_facility: Hospital Universitari Germans Trias i Pujol; Servicio de Oncologia

location_facility: Kursk Regional Clinical Oncology Dispensary

location_facility: Fundacion Investigacion Hospital La Fe de Valencia

location_facility: Clinical Hospital Center Bezanijska kosa; Clinic for Oncology

location_facility: The Catholic University of Korea St. Vincent's Hospital

location_facility: Szabolcs-Szatmar-Bereg Megyei; Korhazak es Egyetemi Oktatokorhaz

location_facility: Irccs Centro Di Riferimento Oncologico (CRO)

location_facility: Eastern Connecticut Hematology and Oncology Associates; (ECHO)

location_facility: Teo Health SA - Saint Constantin Hospital

location_facility: Institutul Regional de Oncologie Iasi; Clinica de Hematologie

location_facility: Chonnam National University Hwasun Hospital

location_facility: Georgios Papanikolaou General Hosp. of Thessaloniki

location_facility: MultiCare Regional Cancer Center - Auburn

location_facility: University of Pecs, I st Dept of Internal Medicine

title: A Phase III, Open-Label, Randomized Study of Atezolizumab (Anti-PD-L1 Antibody) Compared With Gemcitabine+Cisplatin or Carboplatin for PD-L1-Selected, Chemotherapy Naive Patients With Stage IV Squamous Non-Small Cell Lung Cancer

summary: This randomized, open-label study was designed to evaluate and compare the safety and efficacy of atezolizumab with gemcitabine + cisplatin or carboplatin in PD-L1 selected participants with chemotherapy-naive, Stage IV squamous NSCLC. The study was closed due to low patient enrollment and the Sponsor's decision to include patients with squamous NSCLC into the GO29431 study, NCT02409342. Therefore the planned objectives of this study are no longer applicable and formal analyses of efficacy or safety have not been performed.


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 BIOSCIENCES, Inc.

location_country:
Romania
Australia
Russian Federation
Taiwan
France
Japan
Poland
Slovakia
Korea, Republic of
Bulgaria
Hungary
China
Portugal
Germany
United States
Italy
Brazil
Mexico
Czechia
Israel
Spain

acronym: VALUE

title: An Open-label, Multicenter Extension Study to Evaluate the Long-term Safety and Efficacy of Lacosamide as Adjunctive Therapy for Uncontrolled Primary Generalized Tonic-Clonic Seizures in Subjects With Idiopathic Generalized Epilepsy

summary: Assessment of long-term safety and efficacy of oral lacosamide (LCM) as an adjunctive therapy for uncontrolled primary generalized tonic-clonic seizures (PGTCS) in subjects >= 4 years of age with idiopathic generalized epilepsy (IGE). This study will enroll subjects from the LCM SP0982 [NCT02408523] study.


ReferenceCitation: Capdevila J, Wirth LJ, Ernst T, Ponce Aix S, Lin CC, Ramlau R, Butler MO, Delord JP, Gelderblom H, Ascierto PA, Fasolo A, Führer D, Hütter-Krönke ML, Forde PM, Wrona A, Santoro A, Sadow PM, Szpakowski S, Wu H, Bostel G, Faris J, Cameron S, Varga A, Taylor M. PD-1 Blockade in Anaplastic Thyroid Carcinoma. J Clin Oncol. 2020 Aug 10;38(23):2620-2627. doi: 10.1200/JCO.19.02727. Epub 2020 May 4.

name: Vall d’Hebron University Hospital, Vall d’Hebron Institute of Oncology, Universitat Autònoma de Barcelona, Barcelona, Spain

family: Capdevila

full_name: Jaume Capdevila

given: Jaume

name: Massachusetts General Hospital and Harvard Medical School, Boston, MA

family: Wirth

full_name: Lori J. Wirth

given: Lori J.

name: Abteilung Hämatologie/Onkologie, Klinik für Innere Medizin II, Universitätsklinikum Jena, Jena, Germany

family: Ernst

full_name: Thomas Ernst

given: Thomas

name: University Hospital 12 de Octubre, Madrid, Spain

family: Ponce Aix

full_name: Santiago Ponce Aix

name: National Taiwan University Hospital, Taipei, Taiwan

family: Lin

full_name: Chia-Chi Lin

given: Chia-Chi

name: Poznań University of Medical Sciences, Poznań, Poland

family: Ramlau

full_name: Rodryg Ramlau

given: Rodryg

name: Princess Margaret Cancer Centre, University Health Network, Toronto, Ontario, Canada

family: Butler

full_name: Marcus O. Butler

given: Marcus O.

family: Delord

full_name: Jean-Pierre Delord

given: Jean-Pierre

name: Leiden University Medical Center, Leiden, the Netherlands

family: Gelderblom

full_name: Hans Gelderblom

given: Hans

name: Istituto Nazionale Tumori IRCCS Fondazione G. Pascale, Naples, Italy

family: Ascierto

full_name: Paolo A. Ascierto

given: Paolo A.

family: Fasolo

full_name: Angelica Fasolo

given: Angelica

name: University Hospital Essen, Essen, Germany

family: Führer

full_name: Dagmar Führer

given: Dagmar

family: Hütter-Krönke

full_name: Marie Luise Hütter-Krönke

given: Marie Luise

name: Sidney Kimmel Comprehensive Cancer Center, Johns Hopkins University School of Medicine, Baltimore, MD

family: Forde

full_name: Patrick M. Forde

given: Patrick M.

name: Uniwersyteckie Centrum Kliniczne, Gdansk, Poland

family: Wrona

full_name: Anna Wrona

given: Anna

name: IRCCS Humanitas Clinical and Research Center–Humanitas University, Rozzano, Italy

family: Santoro

full_name: Armando Santoro

given: Armando

family: Sadow

full_name: Peter M. Sadow

given: Peter M.

name: Novartis Institutes for BioMedical Research, Cambridge, MA

family: Szpakowski

full_name: Sebastian Szpakowski

given: Sebastian

name: Novartis Pharmaceuticals, East Hanover, NJ

family: Wu

full_name: Hongqian Wu

given: Hongqian

name: Novartis Institutes for BioMedical Research, Basel, Switzerland

family: Bostel

full_name: Geraldine Bostel

given: Geraldine

family: Faris

full_name: Jason Faris

given: Jason

family: Cameron

full_name: Scott Cameron

given: Scott

name: Gustave Roussy Cancer Campus, Paris, France

family: Varga

full_name: Andreea Varga

given: Andreea

name: Oregon Health & Science University, Portland, OR

family: Taylor

full_name: Matthew Taylor

given: Matthew

publisher: American Society of Clinical Oncology (ASCO)

ReferenceCitation: Naing A, Gainor JF, Gelderblom H, Forde PM, Butler MO, Lin CC, Sharma S, Ochoa de Olza M, Varga A, Taylor M, Schellens JHM, Wu H, Sun H, Silva AP, Faris J, Mataraza J, Cameron S, Bauer TM. A first-in-human phase 1 dose escalation study of spartalizumab (PDR001), an anti-PD-1 antibody, in patients with advanced solid tumors. J Immunother Cancer. 2020 Mar;8(1). pii: e000530. doi: 10.1136/jitc-2020-000530.

title: A first-in-human phase 1 dose escalation study of spartalizumab (PDR001), an anti–PD-1 antibody, in patients with advanced solid tumors

location_country:
Italy
United States
Lebanon
Netherlands
France
Poland
Thailand
Norway
Hungary
Spain
Turkey
Germany
Canada
Taiwan

location_facility: The Sidney Kimmel Cancer Center at Johns Hopkins Hospital Johns Hopkins

location_facility: Oregon Health and Science University SC-10

location_facility: Huntsman Cancer Institute Univ. of Utah HCI

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

title: Open Label Multicenter Phase I/II Study of the Safety and Efficacy of PDR001 Administered to Patients With Advanced Malignancies

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

By blocking the interaction between PD-1 and its ligands, PD-L1 and PD-L2, PDR001 inhibits the PD-1 immune checkpoint, resulting in activation of an antitumor immune response by activating effector T-cells and inhibiting regulatory T-cells.


title: A Phase 3, Multi-center, Randomized, Double-Blind, Placebo Controlled Study of the Efficacy and Safety of SD-101 Cream in Patients With Epidermolysis Bullosa

summary: The aim was to assess the efficacy and safety of SD-101-6.0 cream versus Placebo (SD-101-0.0) cream in the treatment of skin lesions in participants with Epidermolysis Bullosa. Funding Source - United States Food and Drug Administration (FDA) Office of Orphan Products Development (OOPD).

ReferenceCitation: Murrell DF, Paller AS, Bodemer C, Browning J, Nikolic M, Barth JA, Lagast H, Krusinska E, Reha A; ESSENCE Study Group. Wound closure in epidermolysis bullosa: data from the vehicle arm of the phase 3 ESSENCE Study. Orphanet J Rare Dis. 2020 Jul 21;15(1):190. doi: 10.1186/s13023-020-01435-3.

family: Murrell

full_name: Dedee F. Murrell

given: Dedee F.

family: Paller

full_name: Amy S. Paller

given: Amy S.

family: Bodemer

full_name: Christine Bodemer

given: Christine

family: Browning

full_name: John Browning

given: John

family: Nikolic

full_name: Milos Nikolic

given: Milos

family: Barth

full_name: Jay A. Barth

given: Jay A.

family: Lagast

full_name: Hjalmar Lagast

given: Hjalmar

family: Krusinska

full_name: Eva Krusinska

given: Eva

family: Reha

full_name: Allen Reha

given: Allen

currency_apc_doaj: GBP

journal_name: Orphanet Journal of Rare Diseases

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

repository_institution: PubMed Central - Europe PMC

ReferenceCitation: Paller AS, Browning J, Nikolic M, Bodemer C, Murrell DF, Lenon W, Krusinska E, Reha A, Lagast H, Barth JA; ESSENCE Study Group. Efficacy and tolerability of the investigational topical cream SD-101 (6% allantoin) in patients with epidermolysis bullosa: a phase 3, randomized, double-blind, vehicle-controlled trial (ESSENCE study). Orphanet J Rare Dis. 2020 Jun 23;15(1):158. doi: 10.1186/s13023-020-01419-3.

family: Lenon

full_name: Willistine Lenon

location_country:
Italy
Austria
Serbia
Netherlands
Germany
France
Poland
Lithuania
Israel
Australia
Spain
United States
United Kingdom


title: A Twelve-Week, Double-Blind, Placebo-Controlled, Randomized, Parallel-Group, Multicenter Study of the Safety and Efficacy of JZP-110 [(R)-2-amino-3-phenylpropylcarbamate Hydrochloride] in the Treatment of Excessive Sleepiness in Subjects With Obstructive Sleep Apnea (OSA)

acronym: OSA

summary: This trial is a 12 week, randomized, double-blind, placebo controlled, multicenter, 5-arm parallel group study of safety and efficacy of JZP-110 in the treatment of excessive sleepiness in adult subjects with OSA.

ReferenceCitation: Rosenberg R, Thorpy MJ, Dauvilliers Y, Schweitzer PK, Zammit G, Gotfried M, Bujanover S, Scheckner B, Malhotra A. Incidence and duration of common early-onset adverse events in randomized controlled trials of solriamfetol for treatment of excessive daytime sleepiness in obstructive sleep apnea and narcolepsy. J Clin Sleep Med. 2021 Jul 20. doi: 10.5664/jcsm.9550. [Epub ahead of print]

family: Rosenberg

full_name: Russell Rosenberg

given: Russell

family: Thorpy

full_name: Michael J. Thorpy

given: Michael J.

name: Sleep Unit, Department of Neurology, Gui-de-Chauliac Hospital, CHU Montpellier, INSERM, France

family: Dauvilliers

full_name: Yves Dauvilliers

given: Yves

name: Sleep Medicine and Research Center, St. Luke’s Hospital, Chesterfield, MO

family: Schweitzer

full_name: Paula K. Schweitzer

given: Paula K.

name: Clinilabs Drug Development Corporation, New York, NY

name: Icahn School of Medicine at Mount Sinai, New York, NY

family: Zammit

full_name: Gary Zammit

given: Gary

family: Gotfried

full_name: Mark Gotfried

given: Mark

family: Bujanover

full_name: Shay Bujanover

given: Shay

family: Scheckner

full_name: Brian Scheckner

given: Brian

family: Malhotra

full_name: Atul Malhotra

given: Atul

journal_name: Journal of Clinical Sleep Medicine

publisher: American Academy of Sleep Medicine (AASM)

ReferenceCitation: Schweitzer PK, Mayer G, Rosenberg R, Malhotra A, Zammit GK, Gotfried M, Chandler P, Baladi M, Strohl KP. Randomized Controlled Trial of Solriamfetol for Excessive Daytime Sleepiness in OSA: An Analysis of Subgroups Adherent or Nonadherent to OSA Treatment. Chest. 2021 Jul;160(1):307-318. doi: 10.1016/j.chest.2021.02.033. Epub 2021 Feb 22.

family: Mayer

full_name: Geert Mayer

given: Geert

full_name: Gary K. Zammit

given: Gary K.

family: Chandler

full_name: Patricia Chandler

given: Patricia

family: Baladi

full_name: Michelle Baladi

given: Michelle

family: Strohl

full_name: Kingman P. Strohl

given: Kingman P.

journal_name: Chest

oa_colors: green hybrid

license: cc-by-nc-nd

license_normalized: cc-by-nc-nd

ReferenceCitation: Weaver TE, Menno DM, Bron M, Crosby RD, Morris S, Mathias SD. Determination of thresholds for minimally important difference and clinically important response on the functional outcomes of sleep questionnaire short version in adults with narcolepsy or obstructive sleep apnea. Sleep Breath. 2021 Sep;25(3):1707-1715. doi: 10.1007/s11325-020-02270-3. Epub 2021 Jan 4.

ReferenceCitation: Rosenberg R, Baladi M, Bron M. Clinically relevant effects of solriamfetol on excessive daytime sleepiness: a posthoc analysis of the magnitude of change in clinical trials in adults with narcolepsy or obstructive sleep apnea. J Clin Sleep Med. 2021 Apr 1;17(4):711-717. doi: 10.5664/jcsm.9006.

name: NeuroTrials Research, Inc., Atlanta, Georgia

name: Atlanta School of Sleep Medicine, Atlanta, Georgia

name: Jazz Pharmaceuticals, Palo Alto, California

ReferenceCitation: Weaver TE, Drake CL, Benes H, Stern T, Maynard J, Thein SG, Andry JM Sr, Hudson JD, Chen D, Carter LP, Bron M, Lee L, Black J, Bogan RK. Effects of Solriamfetol on Quality-of-Life Measures from a 12-Week Phase 3 Randomized Controlled Trial. Ann Am Thorac Soc. 2020 Aug;17(8):998-1007. doi: 10.1513/AnnalsATS.202002-136OC.

name: University of Illinois at Chicago College of Nursing, Chicago, Illinois

name: Henry Ford Hospital Sleep Disorders and Research Center, Detroit, Michigan

name: Somni Bene Institut für Medizinische Forschung und Schlafmedizin Schwerin GmbH, Schwerin, Germany

name: Medical Center, University of Rostock, Rostock, Germany

name: Advanced Respiratory and Sleep Medicine, PLLC, Huntersville, North Carolina

name: CTI Clinical Research Center, Cincinnati, Ohio

name: Pacific Research Network, Evolution Research Group, LLC, San Diego, California

name: Sleep Therapy & Research Center, San Antonio, Texas

name: FutureSearch Trials of Neurology LP, Austin, Texas

name: University of Arkansas for Medical Sciences, Little Rock, Arkansas

name: Stanford Center for Sleep Sciences and Medicine, Palo Alto, California

name: SleepMed, Inc., Columbia, South Carolina; and

name: University of South Carolina School of Medicine, Columbia, South Carolina

ReferenceCitation: Schweitzer PK, Rosenberg R, Zammit GK, Gotfried M, Chen D, Carter LP, Wang H, Lu Y, Black J, Malhotra A, Strohl KP; TONES 3 Study Investigators. Solriamfetol for Excessive Sleepiness in Obstructive Sleep Apnea (TONES 3). A Randomized Controlled Trial. Am J Respir Crit Care Med. 2019 Jun 1;199(11):1421-1431. doi: 10.1164/rccm.201806-1100OC.

name: Sleep Medicine and Research Center, St. Luke’s Hospital, Chesterfield, Missouri

name: Clinilabs Drug Development Corporation, New York, New York

name: Icahn School of Medicine at Mount Sinai, New York, New York

name: Pulmonary Associates, PA, Phoenix, Arizona

name: University of California, San Diego, San Diego, California; and

name: Case Western Reserve University, Cleveland, Ohio

journal_name: American Journal of Respiratory and Critical Care Medicine

repository_institution: University of California - eScholarship University of California

repository_normalized: California Digital Library - eScholarship

location_country:
United States
Netherlands
France
Germany
Canada

location_facility: Hickory Research Center, ARSM Research, LLC

location_facility: Somni bene GmbH Institut für Medizinische Forschung und Schlafmedizin Schwerin GmbH

location_facility: Northwestern University Feinberg School of Medicine

location_facility: Henry Ford Hospital Sleep Disorders & Research Center

location_facility: Clinical Research Group of St. Petersburg

location_facility: So Cal Institute For Respiratory Diseases, Inc.

location_facility: Southwest Cleveland Sleep Research Center

location_facility: Mercy St. Anne & Mercy St. Charles Sleep Disorders Center

location_facility: Stanford University Center for Narcolepsy

location_facility: Universitätsklinikum Münster Department für Neurologie

location_facility: Sleep Medicine & Research Center, St. Luke's Hospital

location_facility: medbo Bezirksklinikum Regensburg Schlafmedizinisches Zentrum

location_facility: Ohio Sleep Medicine & Neuroscience Institute


title: Efficacy and Safety of CD5024 1% in Acne Vulgaris

summary: Exploratory, multi-center, randomised, investigator-blinded, vehicle controlled study using intra-individual comparison involving subjects with acne vulgaris on face to evaluate the efficacy of CD5024 1% cream over a 6-week treatment period compared to its vehicle.

primary_purpose: Other

lead_sponsor: Galderma R&D

location_country: Germany Canada France

location_facility:
Galderma Investigational site
Galderma investigational site
Galderma Investigational Site (# 8060)


title: A Long-term Follow-up Study for Cardiac Safety in the Patients With HER2 Positive Early or Locally Advanced Breast Cancer Who Have Completed the SB3-G31-BC

summary: A Long-term Follow-up Study for Cardiac Safety in the Patients with HER2 Positive Early or Locally Advanced Breast Cancer Who Have Completed the SB3-G31-BC

ReferenceCitation: Pivot X, Pegram M, Cortes J, Lüftner D, Lyman GH, Curigliano G, Bondarenko I, Yoon YC, Kim Y, Kim C. Three-year follow-up from a phase 3 study of SB3 (a trastuzumab biosimilar) versus reference trastuzumab in the neoadjuvant setting for human epidermal growth factor receptor 2-positive breast cancer. Eur J Cancer. 2019 Oct;120:1-9. doi: 10.1016/j.ejca.2019.07.015. Epub 2019 Aug 21.

family: Pivot

full_name: Xavier Pivot

given: Xavier

family: Pegram

full_name: Mark Pegram

family: Cortes

full_name: Javier Cortes

given: Javier

family: Lüftner

full_name: Diana Lüftner

given: Diana

family: Lyman

full_name: Gary H. Lyman

given: Gary H.

family: Curigliano

full_name: Giuseppe Curigliano

given: Giuseppe

family: Bondarenko

full_name: Igor Bondarenko

given: Igor

family: Yoon

full_name: Ye Chan Yoon

given: Ye Chan

family: Kim

full_name: Younsoo Kim

given: Younsoo

full_name: Chul Kim

given: Chul

journal_name: European Journal of Cancer

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

publisher: Elsevier BV

publisher_dissemination: Elsevier

publisher_group: Elsevier

publisher_normalized: Elsevier

title: Three-year follow-up from a phase 3 study of SB3 (a trastuzumab biosimilar) versus reference trastuzumab in the neoadjuvant setting for human epidermal growth factor receptor 2–positive breast cancer

lead_sponsor: Samsung Bioepis Co., Ltd.

location_country:
France
Czechia
Bulgaria
Poland
Romania
Russian Federation
Ukraine

location_facility: Spitalul Clinic Filantropia, Compartimentul Oncologie Ginecologica

location_facility: Spitalul Municipal Ploiesti, Sectia Oncologie Medicala

location_facility: Lviv State Oncological Regional Treatment and Diagnostic Center

location_facility: SHBI of Yaroslavl Region "Regional Clinical Oncology Hospital"

location_facility: Complex Oncological Center - Vratsa, EOOD

location_facility: Centre Hospitalier de Belfort-Montbeliard

location_facility: Bialostockie Centrum Onkologii im.M.Sklodowskiej-Curie w Bialymstoku

location_facility: BHI of Omsk Region "Clinical Oncology Dispensary"

location_facility: Uzhgorod Central City Clinical Hospital City Oncological Center

location_facility: SC Centrul Medical Unirea SRL-Policlinica Baneasa, Specialitatea Oncologie Medicala

location_facility: S.I. Russian Oncological Research Center n.a. N.N. Blokhin

location_facility: SBI of Ryazan region "Regional Clinical Oncological Dispensary"

location_facility: SBHI "Saint-Petersburg Scientific and Practical Center of Specialized Methods of Medical Help (oncological)

location_facility: SBHI "Leningrad Regional Oncology Dispensary"

location_facility: Samodzielny Publiczny Zakład Opieki Zdrowotnej

location_facility: SBHI of Moscow City "Moscow City Oncology Hospital №62" of Moscow Healthcare Department

location_facility: Communal Institution Dnipropetrovsk City Multifield Clinical Hospital No.4 of Dnipropetrovsk Regional Council

location_facility: Regional Communal Institution Sumy Regional Clinical Oncological Dispensary

location_facility: Centrum Onkologii im. Prof. Franciszka Lukaszczyka w Bydgoszczy

location_facility: Centrum Onkologii-Instytut im. M. Sklodowskiej Curie

location_facility: Institutul Oncologic "Prof. Dr. Ion Chiricuta" Cluj-Napoca

location_facility: FSI "Scientific and Research Institution of Oncology n.a. N.N.Petrov" of Ministry of Healthcare and SD of RF

location_facility: Spitalul Clinic Judetean de Urgenta Cluj Napoca

location_facility: Communal Institution of Kherson Regional Council Kherson Regional Oncological Dispensary

location_facility: Saint-Petersburg SBHI "City Clinical Oncology Dispensary"

location_facility: Uniwersyteckie Centrum Kliniczne, Klinika Onkologii I Radioterapii

location_facility: Wielkopolskie Centrum Onkologii, im Marii Sklodowskiej-Curie

location_facility: Non-state Healthcare Institution "Roadway Clinical Hospital of OJSC Russian Railways"

location_facility: Spitalul Judetean de Urgenta "Dr. Constantin Opris" Baia Mare

location_facility: Communal Institution Zaporizhzhia Regional Clinical Oncological Dispensary of Zaporizhzhia Regional Council

location_facility: Federal State Budgetary Institution "Federal Medical Research Center n.a. P.A Gertsen" of Ministry of healthcare of RF/3

location_facility: Communal Institution Cherkasy Regional Oncological Dispensary of Cherkasy Regional Council

location_facility: Spitalul Clinic Municipal de Urgenta Timisoara

location_facility: Vinnytsia Regional Clinical Oncological Dispensary

location_facility: Samodzielny Publiczny ZOZ Opolskiego Centrum Onkologii w Opolu im. T. Koszarowskiego

location_facility: Communal Non-commercial Enterprise Regional Center of Oncology


title: Effects of Oral Nutrition Supplements in Children With Disease Associated Underweight

acronym: MIntS

summary:
The purpose of this study is to test whether the provision of ONS to malnourished children across Europe, in addition to standard counselling, will improve weight gain and other outcomes.

The hypothesis to be tested is:

• Supplementation with ONS in malnourished paediatric patients will lead to increased WFH gain, as well as less malnutrition related complications (e.g. infections).

Primary outcome • The change in BMI z-score over the intervention period of 3 months

Secondary outcomes

• The change in WFH-weight for height (z-scores) over the intervention period of 3 months

primary_purpose: Supportive Care

lead_sponsor: Dr. Michael Chourdakis

location_country:
Croatia
France
Greece
Poland
Norway
Belgium
Russian Federation
Turkey
Iran, Islamic Republic of

location_facility: The Children's Memorial Health Institute

location_facility: Hospital Jeanne de Flandre, Pediatrics Department, CHRU

location_facility: Russian Medical Academy of Postgraduate Education

location_facility: 3rd Paediatric Dept, Hippokration Hospital, AUTH

location_facility: Department of Pediatrics, Women and Children's Division, Oslo University Hospital

location_facility: 1st Paediatric Dept, Hippokration Hospital, AUTH

location_facility: Aristotle University of Thessaloniki, School of Medicine

location_facility: Tehran University of Medical Sciences Tehrān, Children Medical Center

intervention_type: Dietary Supplement


title: Safety and Tolerability of Single and Repeated Doses of ODM-203: An Open-label, Non-randomised, Uncontrolled, Dose Escalation, Multicentre, First-in-Human Study in Subjects With Advanced Solid Tumours

summary: The purpose of this first-in-human study is to evaluate the safety and tolerability of escalating doses of ODM-203 in subjects with advanced solid tumours and to determine the maximum tolerated dose and dose limiting toxicities.

ReferenceCitation: Bono P, Massard C, Peltola KJ, Azaro A, Italiano A, Kristeleit RS, Curigliano G, Lassen U, Arkenau HT, Hakulinen P, Garratt C, Ikonen T, Mustonen MVJ, Rodon JA. Phase I/IIa, open-label, multicentre study to evaluate the optimal dosing and safety of ODM-203 in patients with advanced or metastatic solid tumours. ESMO Open. 2020 Dec;5(6):e001081. doi: 10.1136/esmoopen-2020-001081.

family: Bono

full_name: Petri Bono

given: Petri

family: Massard

full_name: Christophe Massard

given: Christophe

family: Peltola

full_name: Katriina J. Peltola

given: Katriina J.

family: Azaro

full_name: Analía Azaro

given: Analía

family: Italiano

full_name: Antoine Italiano

given: Antoine

family: Kristeleit

full_name: Rebecca S. Kristeleit

given: Rebecca S.

family: Lassen

full_name: Ulrik Lassen

given: Ulrik

family: Arkenau

full_name: Hendrik-Tobias Arkenau

given: Hendrik-Tobias

family: Hakulinen

full_name: Pasi Hakulinen

given: Pasi

family: Garratt

full_name: Chris Garratt

given: Chris

family: Ikonen

full_name: Tarja Ikonen

given: Tarja

family: Mustonen

full_name: Mika V.J. Mustonen

given: Mika V.J.

family: Rodon

full_name: Jordi A. Rodon

given: Jordi A.

currency_apc_doaj: EUR

journal_name: ESMO Open

license: cc-by-nc

license_normalized: cc-by-nc

repository_institution: University of Helsinki - Helda

repository_normalized: HELDA - Digital Repository of the University of Helsinki

repository_institution: University College London - UCL Discovery

repository_normalized: UCL Discovery

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

location_country:
Italy
Finland
France
Spain
Denmark
United Kingdom

location_facility: Helsinki University Central Hospital, Department of Oncology


title: Study to Improve Outcomes in Aortic Stenosis. International, Multi-center, Prospective, Observation and Interventional Cohort Registry

acronym: IMPULSE

summary: The purpose of this study is to narrow the gap between patients being diagnosed with severe aortic stenosis and those being appropriately monitored and treated. For this purpose a prospective survey of current practice (3 months) will be conducted followed by a six month period of intervention during which a variety of quality improvements measures will be implemented. There will then be a 3 month follow-up period during which the legacy of this initiative will be monitored during which no intervention will be carried out.

ReferenceCitation: Steeds RP, Lutz M, Thambyrajah J, Serra A, Schulz E, Maly J, Aiello M, Rudolph TK, Lloyd G, Bortone AS, Hauptmann KE, Clerici A, Delle-Karth G, Rieber J, Indolfi C, Mancone M, Belle L, Lauten A, Arnold M, Bouma BJ, Deutsch C, Kurucova J, Thoenes M, Bramlage P, Frey N, Messika-Zeitoun D. Facilitated Data Relay and Effects on Treatment of Severe Aortic Stenosis in Europe. J Am Heart Assoc. 2019 Oct;8(19):e013160. doi: 10.1161/JAHA.119.013160. Epub 2019 Sep 24.

name: Queen Elizabeth Hospital & Institute of Cardiovascular Sciences University of Birmingham UK

family: Steeds

full_name: Richard P. Steeds

given: Richard P.

name: Department of Cardiology and Angiology University of Kiel Germany

family: Lutz

full_name: Matthias Lutz

given: Matthias

family: Thambyrajah

full_name: Jeetendra Thambyrajah

given: Jeetendra

name: Interventional Cardiology Unit, Hospital de la Santa Creu i Sant Pau Barcelona Spain

family: Serra

full_name: Antonio Serra

given: Antonio

name: Cardiology Department I University Clinic Mainz Germany

family: Schulz

full_name: Eberhard Schulz

given: Eberhard

name: Department of Cardiovascular Surgery Institute for Clinical and Experimental Medicine Prague Czech Republic

name: Department of Cardiovascular Surgery Second Faculty of Medicine Charles University Prague Czech Republic

family: Maly

full_name: Jiri Maly

given: Jiri

name: Department of Cardiothoracic Surgery Foundation IRCCS Policlinico S.Matteo Pavia Italy

family: Aiello

full_name: Marco Aiello

given: Marco

name: Department of Cardiology University of Cologne Heart Center Cologne Germany

family: Rudolph

full_name: Tanja K. Rudolph

given: Tanja K.

family: Lloyd

full_name: Guy Lloyd

given: Guy

family: Bortone

full_name: Alessandro Santo Bortone

given: Alessandro Santo

name: Krankenhaus der Barmherzigen Brüder Trier Germany

family: Hauptmann

full_name: Karl Eugen Hauptmann

given: Karl Eugen

family: Clerici

full_name: Alberto Clerici

given: Alberto

name: 4th Medical Department Hietzing Hospital Vienna Austria

family: Delle‐Karth

full_name: Georg Delle‐Karth

given: Georg

name: Herzkatheterlabor Nymphenburg and Department of Cardiology University of Munich Germany

family: Rieber

full_name: Johannes Rieber

given: Johannes

name: Division of Cardiology and URT CNR of IFC University Magna Graecia Catanzaro Italy

family: Indolfi

full_name: Ciro Indolfi

given: Ciro

family: Mancone

full_name: Massimo Mancone

given: Massimo

family: Belle

full_name: Loic Belle

given: Loic

name: German Centre for Cardiovascular Research (DZHK) University Heart Center & Charité Berlin Germany

family: Lauten

full_name: Alexander Lauten

given: Alexander

name: Department of Cardiology University Hospital Erlangen Germany

family: Arnold

full_name: Martin Arnold

given: Martin

family: Bouma

full_name: Berto J. Bouma

given: Berto J.

name: Institute for Pharmacology and Preventive Medicine Cloppenburg Germany

family: Deutsch

full_name: Cornelia Deutsch

given: Cornelia

family: Kurucova

full_name: Jana Kurucova

given: Jana

family: Thoenes

full_name: Martin Thoenes

name: University of Ottawa Heart Institute Ottawa Canada

journal_name: Journal of the American Heart Association

publisher: Ovid Technologies (Wolters Kluwer Health)

ReferenceCitation: Frey N, Steeds RP, Rudolph TK, Thambyrajah J, Serra A, Schulz E, Maly J, Aiello M, Lloyd G, Bortone AS, Hauptmann KE, Clerici A, Delle Karth G, Rieber J, Indorfi C, Mancone M, Belle L, Lauten A, Arnold M, Bouma BJ, Lutz M, Pohlmann C, Kurucova J, Thoenes M, Bramlage P, Messika-Zeitoun D; IMPULSE registry group. Symptoms, disease severity and treatment of adults with a new diagnosis of severe aortic stenosis. Heart. 2019 Nov;105(22):1709-1716. doi: 10.1136/heartjnl-2019-314940. Epub 2019 Jul 13.

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

location_facility: Universitätsmedizin der Johannes Gutenberg-Universität Mainz

location_facility: Università degli Studi di Bari Aldo Moro

location_facility: IKEM - Institute for Clinical and Experimental Medicine

location_facility: UKSK Campus Kiel, Klinik für Kardiologie und Angiologie


title: The Eurocyst Initiative: Building a Reference Center Network Across EUROpe to Establish a Large-scale Longitudinal Observational Cohort of Autosomal Dominant polyCYSTic Kidney Disease (ADPKD) Patients

summary: EuroCYST initiative aims to build a large, well-characterized cohort of Autosomal Dominant polyCYSTic Kidney Disease (ADPKD) subjects who are followed in a longitudinal observational cohort study has the potential to identify progression factors and biomarkers, and to assess disease stage specific mortality, morbidity and health care costs.

status: Unknown status

lead_sponsor: Andreas L. Serra

location_country:
Italy
Switzerland
Netherlands
United Kingdom
France
Belgium
Spain
Turkey
Germany
Czech Republic

location_facility: University of Cambridge, Cambridge Cancer Center

location_facility: University of Sheffield Medical School Academic Unit of Nephrology Department of Infection and Immunity

location_facility: Centre hospitalier universitaire de Brest

location_facility: Istituto di Ricerche Farmacologiche "Mario Negri"


title: A Phase I, Open Label, Multicenter, Dose-escalation Study of HDM201 in Adult Patients With Advanced Solid and Hematological Tumors Characterized by Wild-type TP53

summary: To determine and evaluate a safe and tolerated dose of HDM201 in adult patients with selected advanced tumors characterized by wild-type TP53.

location_country:
United States
Netherlands
France
Japan
Singapore
Spain
Germany
Taiwan

location_facility:
Memorial Sloan Kettering Onc. Dep
Novartis Investigative Site
Dana Farber Cancer Institute SC-6


lead_sponsor: Italfarmaco

location_country:
Italy
Serbia
Netherlands
France
Poland
Romania
Hungary
Spain
Czech Republic

acronym: POC

location_facility: Institutul de Endocrinologie C.I.Parhon, Sectia clinica de endocrinologie VI

location_facility: Klinika Endokrynologii Centrum Medycznego Kształcenia Podyplomowego

location_facility: University Children's Hospital of Cracow

location_facility: Complejo Hospitalario Universitario de Santiago de Compostela

location_facility: Institutul de Endocrinologie C.I.Parhon, Sectia clinica de endocrinologie II

title: A Randomized, Multicenter, Phase II Study to Investigate Efficacy and Safety of ITF2984 in Acromegalic Patients

summary: The purpose of this study is to investigate in acromegalic patients the effect of different doses of ITF2984 on GH and IGF-1 concentrations and to investigate safety and tolerability of three different doses of ITF2984.


title: Patient Registry of the European Friedreich's Ataxia Consortium for Translational Studies (EFACTS)

acronym: EFACTS

summary:
This is a multi-centre, multi-national, prospective, observational study of Friedreich's Ataxia (FRDA) with a control group to:

obtain natural history data on individuals affected by FRDA
relate clinical assessments and results from proteomic analyses
expedite identification and recruitment of participants for clinical trials
develop and validate sensitive and reliable outcome measures for detecting onset and change over the natural course of FRDA which may also be potential outcome measures for use in future clinical trials and clinical care
plan for future research studies

ReferenceType: background

ReferenceCitation: Reetz K, Dogan I, Costa AS, Dafotakis M, Fedosov K, Giunti P, Parkinson MH, Sweeney MG, Mariotti C, Panzeri M, Nanetti L, Arpa J, Sanz-Gallego I, Durr A, Charles P, Boesch S, Nachbauer W, Klopstock T, Karin I, Depondt C, vom Hagen JM, Schöls L, Giordano IA, Klockgether T, Bürk K, Pandolfo M, Schulz JB. Biological and clinical characteristics of the European Friedreich's Ataxia Consortium for Translational Studies (EFACTS) cohort: a cross-sectional analysis of baseline data. Lancet Neurol. 2015 Feb;14(2):174-82. doi: 10.1016/S1474-4422(14)70321-7. Epub 2015 Jan 5.

ReferenceCitation: Reetz K, Dogan I, Hilgers RD, Giunti P, Parkinson MH, Mariotti C, Nanetti L, Durr A, Ewenczyk C, Boesch S, Nachbauer W, Klopstock T, Stendel C, Rodríguez de Rivera Garrido FJ, Rummey C, Schöls L, Hayer SN, Klockgether T, Giordano I, Didszun C, Rai M, Pandolfo M, Schulz JB; EFACTS study group. Progression characteristics of the European Friedreich's Ataxia Consortium for Translational Studies (EFACTS): a 4-year cohort study. Lancet Neurol. 2021 May;20(5):362-372. doi: 10.1016/S1474-4422(21)00027-2. Epub 2021 Mar 23.

family: Reetz

full_name: Kathrin Reetz

given: Kathrin

family: Dogan

full_name: Imis Dogan

given: Imis

family: Hilgers

full_name: Ralf-Dieter Hilgers

given: Ralf-Dieter

family: Giunti

full_name: Paola Giunti

given: Paola

family: Parkinson

full_name: Michael H Parkinson

given: Michael H

family: Mariotti

full_name: Caterina Mariotti

given: Caterina

family: Nanetti

full_name: Lorenzo Nanetti

given: Lorenzo

family: Durr

full_name: Alexandra Durr

given: Alexandra

family: Ewenczyk

full_name: Claire Ewenczyk

given: Claire

family: Boesch

full_name: Sylvia Boesch

given: Sylvia

family: Nachbauer

full_name: Wolfgang Nachbauer

given: Wolfgang

family: Klopstock

full_name: Thomas Klopstock

given: Thomas

family: Stendel

full_name: Claudia Stendel

given: Claudia

family: Rodríguez de Rivera Garrido

full_name: Francisco Javier Rodríguez de Rivera Garrido

given: Francisco Javier

family: Rummey

full_name: Christian Rummey

given: Christian

family: Schöls

full_name: Ludger Schöls

given: Ludger

family: Hayer

full_name: Stefanie N Hayer

given: Stefanie N

family: Klockgether

full_name: Thomas Klockgether

family: Giordano

full_name: Ilaria Giordano

given: Ilaria

family: Didszun

full_name: Claire Didszun

family: Rai

full_name: Myriam Rai

given: Myriam

family: Pandolfo

full_name: Massimo Pandolfo

family: Schulz

full_name: Jörg B Schulz

given: Jörg B

family: Labrum

repositories_institution: University College London - UCL Discovery

ReferenceCitation: Indelicato E, Nachbauer W, Eigentler A, Amprosi M, Matteucci Gothe R, Giunti P, Mariotti C, Arpa J, Durr A, Klopstock T, Schöls L, Giordano I, Bürk K, Pandolfo M, Didszdun C, Schulz JB, Boesch S; EFACTS (European Friedreich's Ataxia Consortium for Translational Studies). Onset features and time to diagnosis in Friedreich's Ataxia. Orphanet J Rare Dis. 2020 Aug 3;15(1):198. doi: 10.1186/s13023-020-01475-9.

name: on behalf of the EFACTS (European Friedreich’s Ataxia Consortium for Translational Studies)

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

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

repositories:
HAL
PubMed Central
Research Square
UCL Discovery
dipot.ulb.ac.be

repositories_institution: Université Libre de Bruxelles - Dépôt institutionnel de l'Université libre de Bruxelles

title: Onset features and time to diagnosis in Friedreich’s Ataxia

ReferenceCitation: Bonnechère B, Jansen B, Haack I, Omelina L, Feipel V, Van Sint Jan S, Pandolfo M. Automated functional upper limb evaluation of patients with Friedreich ataxia using serious games rehabilitation exercises. J Neuroeng Rehabil. 2018 Oct 4;15(1):87. doi: 10.1186/s12984-018-0430-7.

journal_name: Journal of NeuroEngineering and Rehabilitation

ReferenceCitation: Reetz K, Dogan I, Hohenfeld C, Didszun C, Giunti P, Mariotti C, Durr A, Boesch S, Klopstock T, Rodríguez de Rivera Garrido FJ, Schöls L, Giordano I, Bürk K, Pandolfo M, Schulz JB; EFACTS Study Group. Nonataxia symptoms in Friedreich Ataxia: Report from the Registry of the European Friedreich's Ataxia Consortium for Translational Studies (EFACTS). Neurology. 2018 Sep 4;91(10):e917-e930. doi: 10.1212/WNL.0000000000006121. Epub 2018 Aug 10.

repository_institution: Ludwid Maxmilian's Universitat Munchen - Open access LMU

title: Nonataxia symptoms in Friedreich Ataxia

ReferenceCitation: Tanguy Melac A, Mariotti C, Filipovic Pierucci A, Giunti P, Arpa J, Boesch S, Klopstock T, Müller Vom Hagen J, Klockgether T, Bürk K, Schulz JB, Reetz K, Pandolfo M, Durr A, Tezenas du Montcel S; EFACTS group. Friedreich and dominant ataxias: quantitative differences in cerebellar dysfunction measurements. J Neurol Neurosurg Psychiatry. 2018 Jun;89(6):559-565. doi: 10.1136/jnnp-2017-316964. Epub 2017 Dec 26.

journal_name: Journal of Neurology, Neurosurgery & Psychiatry

ReferenceCitation: Lad M, Parkinson MH, Rai M, Pandolfo M, Bogdanova-Mihaylova P, Walsh RA, Murphy S, Emmanuel A, Panicker J, Giunti P. Urinary, bowel and sexual symptoms in a cohort of patients with Friedreich's ataxia. Orphanet J Rare Dis. 2017 Sep 26;12(1):158. doi: 10.1186/s13023-017-0709-y.

repository_institution: CERN European Organization for Nuclear Research - Zenodo

repositories:
PubMed Central
UCL Discovery
Zenodo
dipot.ulb.ac.be

title: Urinary, bowel and sexual symptoms in a cohort of patients with Friedreich’s ataxia

ReferenceCitation: Reetz K, Dogan I, Hilgers RD, Giunti P, Mariotti C, Durr A, Boesch S, Klopstock T, de Rivera FJR, Schöls L, Klockgether T, Bürk K, Rai M, Pandolfo M, Schulz JB; EFACTS Study Group. Progression characteristics of the European Friedreich's Ataxia Consortium for Translational Studies (EFACTS): a 2 year cohort study. Lancet Neurol. 2016 Dec;15(13):1346-1354. doi: 10.1016/S1474-4422(16)30287-3. Erratum in: Lancet Neurol. 2017 Dec;16(12 ):954.

title: Progression characteristics of the European Friedreich’s Ataxia Consortium for Translational Studies (EFACTS): a 2 year cohort study

location_country:
Italy
Austria
France
Belgium
Spain
Germany
United Kingdom

location_facility: Medical University Innsbruck, Department of Neurology

location_facility: Fondazione IRCCS Istituto Neurologico Carlo Besta

location_facility: University Hospital Bonn, Dept. of Neurology

location_facility: Hospital Universitario La Paz, Servicio de Neurologia

location_facility: Hôptal de Hautepierre, Department of Neurology

location_facility: University Hospital Aachen, Dept. of Neurology

location_facility: University College of London, Institute of Neurology

location_facility: University of Tübingen, Dept. of Neurodegenerative Diseases, Hertie-Institute for Clinical Brain Research

location_facility: University of Munich, Dept. of Neurology

location_facility: Philipps University Marburg, Dept. of Neurology


title: A Phase I, Open-label, Multi-arm, Multi-centre, Multi-dose, Dose Escalation Study of LTX-315 as Monotherapy or in Combination With Either Ipilimumab or Pembrolizumab in Patients With Transdermally Accessible Tumours

summary: The study will assess the safety, tolerability, PK and efficacy of different intra-tumoral dosing regimens of LTX-315; a lytic-peptide that induces long-term anti-cancer immune responses, as monotherapy or in combination with ipilimumab or pembrolizumab.

ReferenceCitation: Jebsen NL, Apelseth TO, Haugland HK, Rekdal Ø, Patel H, Gjertsen BT, Jøssang DE. Enhanced T-lymphocyte infiltration in a desmoid tumor of the thoracic wall in a young woman treated with intratumoral injections of the oncolytic peptide LTX-315: a case report. J Med Case Rep. 2019 Jun 10;13(1):177. doi: 10.1186/s13256-019-2088-6.

family: Jebsen

full_name: Nina Louise Jebsen

given: Nina Louise

family: Apelseth

full_name: Torunn Oveland Apelseth

given: Torunn Oveland

family: Haugland

full_name: Hans Kristian Haugland

given: Hans Kristian

family: Rekdal

full_name: Øystein Rekdal

given: Øystein

family: Patel

full_name: Hamina Patel

given: Hamina

family: Gjertsen

full_name: Bjørn Tore Gjertsen

given: Bjørn Tore

family: Jøssang

full_name: Dag Eirik Jøssang

given: Dag Eirik

journal_name: Journal of Medical Case Reports

repository_institution: University of Oslo - Duo Research Archive

lead_sponsor: Lytix Biopharma AS

location_country:
Italy
France
Norway
Belgium
United Kingdom

location_facility: Oslo University Hospital Radiumhospitalet

location_facility: Cliniques Universitaires St-Luc, Service d'oncologie médicale


title: A Multicenter, Randomized, Open-Label Study to Assess the Impact of Natalizumab Versus Fingolimod on Central Nervous System Tissue Damage and Recovery in Active Relapsing-Remitting Multiple Sclerosis Subjects

acronym: REVEAL

summary: The primary objective of this study is to assess the effect of natalizumab compared to fingolimod on the evolution of new on-treatment T1-gadolinium-enhancing (Gd+) lesions to persistent black holes (PBH) over 52 weeks. The secondary objectives of this study in this study population are to assess the effect of natalizumab compared to fingolimod on: magnetic resonance imaging (MRI) measures of central nervous system (CNS) tissue destruction as measured by the number of new T1-Gd+ lesions; various other MRI measures of disease activity; No Evidence of Disease Activity (NEDA); Relapse on treatment over 52 weeks; The change in information processing speed as measured by the Symbol Digit Modalities Test (SDMT).

ReferenceCitation: Butzkueven H, Licata S, Jeffery D, Arnold DL, Filippi M, Geurts JJ, Santra S, Campbell N, Ho PR; REVEAL Investigators. Natalizumab versus fingolimod for patients with active relapsing-remitting multiple sclerosis: results from REVEAL, a prospective, randomised head-to-head study. BMJ Open. 2020 Oct 20;10(10):e038861. doi: 10.1136/bmjopen-2020-038861.

family: Butzkueven

full_name: Helmut Butzkueven

given: Helmut

family: Licata

full_name: Stephanie Licata

given: Stephanie

family: Jeffery

full_name: Douglas Jeffery

given: Douglas

family: Arnold

full_name: Douglas L Arnold

given: Douglas L

family: Filippi

full_name: Massimo Filippi

given: Massimo

family: Geurts

full_name: Jeroen JG Geurts

given: Jeroen JG

family: Santra

full_name: Sourav Santra

given: Sourav

family: Campbell

full_name: Nolan Campbell

given: Nolan

family: Ho

full_name: Pei-Ran Ho

given: Pei-Ran

journal_name: BMJ Open

repository_normalized: University of Melbourne - Minerva Access

publisher: BMJ

publisher_dissemination: British Medical Journal

publisher_group: British Medical Journal

publisher_normalized: British Medical Journal

lead_sponsor: Biogen

location_country:
Italy
United States
Sweden
France
Czechia
Australia
Spain
Germany
United Kingdom


title: BIO monitorinG in Patients With Preserved Left ventricUlar Function AfteR Diagnosed Myocardial Infarction

acronym: BIO-GUARD-MI

summary: The BIO|GUARD-MI study investigates whether continuous arrhythmia monitoring and the consequent treatment after detected arrhythmias in patients after myocardial infarction with preserved cardiac function, but other risk factors, decreases the risk of major adverse cardiac events.

primary_purpose: Diagnostic

ReferenceCitation: Jons C, Sogaard P, Behrens S, Schrader J, Mrosk S, Bloch Thomsen PE. The clinical effect of arrhythmia monitoring after myocardial infarction (BIO-GUARD|MI):study protocol for a randomized controlled trial. Trials. 2019 Sep 11;20(1):563. doi: 10.1186/s13063-019-3644-5.

family: Jons

full_name: Christian Jons

given: Christian

family: Sogaard

full_name: Peter Sogaard

given: Peter

family: Behrens

full_name: Steffen Behrens

given: Steffen

family: Schrader

full_name: Jürgen Schrader

given: Jürgen

family: Mrosk

full_name: Sascha Mrosk

given: Sascha

family: Bloch Thomsen

full_name: Poul Erik Bloch Thomsen

given: Poul Erik

journal_name: Trials

lead_sponsor: Biotronik SE & Co. KG

location_country:
Austria
Netherlands
Germany
France
Czechia
Poland
Slovakia
Hungary
Australia
Belgium
Spain
Denmark
United States
Latvia

location_facility: Institute for Clinical and Experimental Medicine (IKEM)

location_facility: Charité Universitätsklinikum - Campus Benjamin Franklin

location_facility: Onze Lieve Vrouwe Gasthuis Amsterdam (OLVG)

location_facility: Universitätsklinikum Schleswig-Holstein (UKSH) - Campus Lübeck

location_facility: Sjaellands Universitets Hospital, Roskilde

location_facility: Hungarian Defence Forces Military Hospital

location_facility: Schwarzwald-Baar Klinikum Villingen-Schwenningen GmbH

location_facility: Pauls Stradins Clinical University Hospital

location_facility: Herz- und Gefäß- Klinik GmbH Bad Neustadt

location_facility: Hôpital Gabriel Montpied, Clermont Ferrand

location_facility: Klinika i Katedra Chorób Wewn. i Kardiologii

location_facility: East-Slovak Cardiology Institute (VUSCH)


summary: This study will evaluate the safety and efficacy of a carboxymethylcellulose based eye drop formulation compared with carboxymethylcellulose based preservative-free lubricant eye drops (OPTIVE™) in subjects with dry eye disease.

lead_sponsor: Allergan

location_country:
Italy
France
Belgium
Australia
Russian Federation
Spain
Germany
United Kingdom


title: A Multi-national, Multi-center, Prospective, Randomized, Double Blinded, Placebo-controlled Trial to Evaluate the Efficacy of HyperBox Cyclical Topical Wound Oxygen Therapy (TWO2) in the Treatment of Chronic Diabetic Foot Ulcers

summary: The purpose of this study is to evaluate the efficacy, safety and economical benefits of Cyclical Pressure Topical Wound Oxygen (TWO2) Therapy in the treatment of chronic diabetic foot ulcers. Subjects will wear a standardized off-loading device and use advanced moist wound therapy (AMWT) dressings. Following a 2 week run-in period with the standardized care and after meeting all the eligibility criteria, half the subjects will use the TWO2 device, while the other half will use a sham device.

ReferenceCitation: Frykberg RG, Franks PJ, Edmonds M, Brantley JN, Téot L, Wild T, Garoufalis MG, Lee AM, Thompson JA, Reach G, Dove CR, Lachgar K, Grotemeyer D, Renton SC; TWO2 Study Group. A Multinational, Multicenter, Randomized, Double-Blinded, Placebo-Controlled Trial to Evaluate the Efficacy of Cyclical Topical Wound Oxygen (TWO2) Therapy in the Treatment of Chronic Diabetic Foot Ulcers: The TWO2 Study. Diabetes Care. 2020 Mar;43(3):616-624. doi: 10.2337/dc19-0476. Epub 2019 Oct 16.

family: Frykberg

full_name: Robert G. Frykberg

given: Robert G.

family: Franks

full_name: Peter J. Franks

given: Peter J.

family: Edmonds

full_name: Michael Edmonds

given: Michael

family: Brantley

full_name: Jonathan N. Brantley

given: Jonathan N.

family: Téot

full_name: Luc Téot

given: Luc

family: Wild

full_name: Thomas Wild

given: Thomas

family: Garoufalis

full_name: Matthew G. Garoufalis

given: Matthew G.

family: Lee

full_name: Aliza M. Lee

given: Aliza M.

family: Thompson

full_name: Janette A. Thompson

given: Janette A.

family: Reach

full_name: Gérard Reach

given: Gérard

family: Dove

full_name: Cyaandi R. Dove

given: Cyaandi R.

family: Lachgar

full_name: Karim Lachgar

given: Karim

family: Grotemeyer

full_name: Dirk Grotemeyer

given: Dirk

family: Renton

full_name: Sophie C. Renton

given: Sophie C.

journal_name: Diabetes Care

evidence: open (via free pdf)

publisher: American Diabetes Association

publisher_dissemination: American Diabetes Association

publisher_group: American Diabetes Association

publisher_normalized: American Diabetes Association

lead_sponsor: AOTI Ltd.

location_country:
Luxembourg
United States
France
Germany
United Kingdom

location_facility:
Städtisches Klinikum Dessau
Montpellier University Hospital
Hunter Holmes McGuire VA Medical Center
Salem VA Medical Center
Kirchberg Hospital
King's College Hospital
Washington DC VA Medical Center
The Research Center Inc.
Advanced Foot & Ankle Center
Advanced Research Institute of Miami
Avicenne Hospital
Edward Hines, Jr. VA Hospital
Hospital Simone Veil
Northwick Park Hospital
NorthBay Center for Wounds
Carl T. Hayden VA Medical Center


title: Phase II Study of the BioVentrix PliCath HF™ System for the Treatment of Ischemic Cardiomyopathy

acronym: CONFIGURE-HF

summary: The purpose of this prospective, single-armed, multi-center clinical trial is to further establish the safety and feasibility of using the BioVentrix PliCath HF System for the treatment of left ventricular dysfunction in appropriate cohorts of humans suffering from heart failure.

ReferenceType: result

ReferenceCitation: Wechsler AS, Sadowski J, Kapelak B, Bartus K, Kalinauskas G, Rucinskas K, Samalavicius R, Annest L. Durability of epicardial ventricular restoration without ventriculotomy. Eur J Cardiothorac Surg. 2013 Sep;44(3):e189-92; discussion e192. doi: 10.1093/ejcts/ezt292. Epub 2013 Jun 5.

family: Wechsler

full_name: Andrew S. Wechsler

given: Andrew S.

family: Sadowski

full_name: Jerzy Sadowski

given: Jerzy

family: Kapelak

full_name: Boguslaw Kapelak

given: Boguslaw

family: Bartus

full_name: Krzysztof Bartus

given: Krzysztof

family: Kalinauskas

full_name: Ginteras Kalinauskas

given: Ginteras

family: Rucinskas

full_name: Kestutis Rucinskas

given: Kestutis

family: Samalavicius

full_name: Robertas Samalavicius

given: Robertas

family: Annest

full_name: Lon Annest

given: Lon

journal_name: European Journal of Cardio-Thoracic Surgery

publisher: Oxford University Press (OUP)

publisher_dissemination: Oxford University Press

publisher_group: Oxford University Press

publisher_normalized: Oxford University Press

title: Durability of epicardial ventricular restoration without ventriculotomy†

lead_sponsor: BioVentrix

location_country:
Italy
Austria
United Kingdom
Spain
France
Czechia
Greece
Lithuania
Poland
Portugal
Latvia

location_facility: Hospital Clinic and University of Barcelona

location_facility: Hospital Pitie Sal Petirere Institute of Cardiology


title: A Randomized, Open-label, 2-arm Parallel-group, Multicenter, 26-week Study Assessing the Safety and Efficacy of H0E901-U300 Versus Lantus in Older Patients With Type 2 Diabetes Inadequately Controlled on Antidiabetic Regimens Either Including no Insulin, or With Basal Insulin as Their Only Insulin

acronym: SENIOR

summary:
Primary Objective:

To demonstrate the non-inferiority of H0E901-U300 to Lantus, in change of glycated hemoglobin A1c (HbA1c).

Secondary Objectives:

To demonstrate the superiority of H0E901-U300 in comparison with Lantus in:

Percentage of participants with at least one severe and/or confirmed (by plasma glucose ≤70mg/dL [3.9mmol/L]) hypoglycemia event from 22:00 to 08:59 next morning
Percentage of participants with at least one nocturnal (from 00:00-05:59) severe and/or confirmed (≤70mg/dL [3.9mmol/L]) hypoglycemia event
Percentage of participants with at least one severe and/or confirmed (by plasma glucose ≤70mg/dL [3.9mmol/L]) hypoglycemia event occurring at any time of day
HbA1c change

ReferenceCitation: Ritzel R, Harris SB, Baron H, Florez H, Roussel R, Espinasse M, Muehlen-Bartmer I, Zhang N, Bertolini M, Brulle-Wohlhueter C, Munshi M, Bolli GB. A Randomized Controlled Trial Comparing Efficacy and Safety of Insulin Glargine 300 Units/mL Versus 100 Units/mL in Older People With Type 2 Diabetes: Results From the SENIOR Study. Diabetes Care. 2018 Aug;41(8):1672-1680. doi: 10.2337/dc18-0168. Epub 2018 Jun 12. Erratum in: Diabetes Care. 2019 Aug;42(8):1604.

family: Ritzel

full_name: Robert Ritzel

given: Robert

family: Harris

full_name: Stewart B. Harris

given: Stewart B.

family: Baron

full_name: Helen Baron

given: Helen

family: Florez

full_name: Hermes Florez

given: Hermes

family: Roussel

full_name: Ronan Roussel

given: Ronan

family: Espinasse

full_name: Melanie Espinasse

given: Melanie

family: Muehlen-Bartmer

full_name: Isabel Muehlen-Bartmer

given: Isabel

family: Zhang

full_name: Nianxian Zhang

given: Nianxian

family: Bertolini

full_name: Monica Bertolini

given: Monica

family: Brulle-Wohlhueter

full_name: Claire Brulle-Wohlhueter

given: Claire

family: Munshi

full_name: Medha Munshi

given: Medha

family: Bolli

full_name: Geremia B. Bolli

given: Geremia B.

lead_sponsor: Sanofi

location_country:
Italy
United States
Peru
Sweden
Mexico
Germany
France
Japan
Poland
Romania
Hungary
Argentina
Australia
Spain
Canada
United Kingdom
Korea, Republic of
Colombia

location_facility:
Investigational Site Number 124007
Investigational Site Number 840061
Investigational Site Number 250005
Investigational Site Number 840053
Investigational Site Number 840016
Investigational Site Number 840063
Investigational Site Number 642004
Investigational Site Number 616002
Investigational Site Number 840031
Investigational Site Number 410004
Investigational Site Number 604004
Investigational Site Number 032006
Investigational Site Number 724001
Investigational Site Number 604002
Investigational Site Number 840038
Investigational Site Number 840058
Investigational Site Number 840011
Investigational Site Number 752004
Investigational Site Number 840056
Investigational Site Number 380005
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Investigational Site Number 348007
Investigational Site Number 380003
Investigational Site Number 410001
Investigational Site Number 840047
Investigational Site Number 392012
Investigational Site Number 032002
Investigational Site Number 840057
Investigational Site Number 392004
Investigational Site Number 392013
Investigational Site Number 840060
Investigational Site Number 642002
Investigational Site Number 124003
Investigational Site Number 036002
Investigational Site Number 840028
Investigational Site Number 392003
Investigational Site Number 724007
Investigational Site Number 840007
Investigational Site Number 276003
Investigational Site Number 032004
Investigational Site Number 724002
Investigational Site Number 348009
Investigational Site Number 840045
Investigational Site Number 840042
Investigational Site Number 642006
Investigational Site Number 124008
Investigational Site Number 840005
Investigational Site Number 840014
Investigational Site Number 840009
Investigational Site Number 124009
Investigational Site Number 124004
Investigational Site Number 484003
Investigational Site Number 826005
Investigational Site Number 840020
Investigational Site Number 840023
Investigational Site Number 826003
Investigational Site Number 840059
Investigational Site Number 840040
Investigational Site Number 840001
Investigational Site Number 392008
Investigational Site Number 124001
Investigational Site Number 124002
Investigational Site Number 410003
Investigational Site Number 840036
Investigational Site Number 840018
Investigational Site Number 840010
Investigational Site Number 724003
Investigational Site Number 036004
Investigational Site Number 840072
Investigational Site Number 840034
Investigational Site Number 170004
Investigational Site Number 724005
Investigational Site Number 826002
Investigational Site Number 348003
Investigational Site Number 840013
Investigational Site Number 604003
Investigational Site Number 826004
Investigational Site Number 348005
Investigational Site Number 840019
Investigational Site Number 840048
Investigational Site Number 276002
Investigational Site Number 410005
Investigational Site Number 484001
Investigational Site Number 724008
Investigational Site Number 840035
Investigational Site Number 840041
Investigational Site Number 840046
Investigational Site Number 380001
Investigational Site Number 604005
Investigational Site Number 642003
Investigational Site Number 840037
Investigational Site Number 032001
Investigational Site Number 380004
Investigational Site Number 840021
Investigational Site Number 840050
Investigational Site Number 840064
Investigational Site Number 840015
Investigational Site Number 752003
Investigational Site Number 276004
Investigational Site Number 840070
Investigational Site Number 840054
Investigational Site Number 036003
Investigational Site Number 124005
Investigational Site Number 170003
Investigational Site Number 604001
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Investigational Site Number 724006
Investigational Site Number 752002
Investigational Site Number 752001
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Investigational Site Number 840032
Investigational Site Number 840002
Investigational Site Number 032003
Investigational Site Number 380002
Investigational Site Number 840024
Investigational Site Number 124006
Investigational Site Number 348004
Investigational Site Number 840022
Investigational Site Number 840055
Investigational Site Number 826006
Investigational Site Number 604006
Investigational Site Number 276001
Investigational Site Number 840027
Investigational Site Number 250004
Investigational Site Number 826001
Investigational Site Number 840004
Investigational Site Number 840030
Investigational Site Number 036001
Investigational Site Number 840026
Investigational Site Number 616005
Investigational Site Number 840003
Investigational Site Number 484002
Investigational Site Number 616003
Investigational Site Number 250006
Investigational Site Number 642005
Investigational Site Number 642007
Investigational Site Number 840067
Investigational Site Number 840017
Investigational Site Number 484004
Investigational Site Number 348001
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Investigational Site Number 348008
Investigational Site Number 392002
Investigational Site Number 840006
Investigational Site Number 410002
Investigational Site Number 616001
Investigational Site Number 724004
Investigational Site Number 170007
Investigational Site Number 170005
Investigational Site Number 276005
Investigational Site Number 840065
Investigational Site Number 840062
Investigational Site Number 642001
Investigational Site Number 840025
Investigational Site Number 840069
Investigational Site Number 170001
Investigational Site Number 348002
Investigational Site Number 840012
Investigational Site Number 124010
Investigational Site Number 604007
Investigational Site Number 616004
Investigational Site Number 392010


primary_purpose: Prevention

location_country:
Italy
United States
Germany
France
Hungary
Belgium
Canada
United Kingdom

title: OPuS-2: A Multicenter, Randomized, Double-blind, Placebo-controlled, Parallel Group Study to Evaluate the Efficacy and Safety of Two Dose Levels of BCX4161 for 12 Weeks as an Oral Prophylaxis Treatment for Attacks of Hereditary Angioedema

summary: This study will evaluate the safety and efficacy of an oral treatment, BCX4161, in preventing acute attacks in subjects with hereditary angioedema (HAE). Eligible subjects will be randomized to receive one of two doses of BCX4161 or placebo for 12 weeks. The study will compare the number of acute attacks in each treatment group, as well as a number of other clinical outcomes, and the safety and tolerability of each dose of BCX4161 compared to placebo.