A Study to Assess the Efficacy and Safety of Vamorolone in Boys With Duchenne Muscular Dystrophy (DMD)
Summary
The lay summary the sponsor registered on ClinicalTrials.gov.
Investigators
The overall officials registered on this study. Names that resolve to a findmyKOL profile link to it; the rest are shown as registered.
- Michela Guglieri, M.D.
John Walton Muscular Dystrophy Research Centre
Study Chair - Paula Clemens, M.D.
University of Pittsburgh
Study Chair
A profile link is shown only when the registered name resolves to a person record with a confident, namesake-checked match; ambiguous names stay unlinked.
Sites
33 registered facilities, with the per-site recruitment status where the registry publishes one. ClinicalTrials.gov does not publish per-site enrollment counts.
- Royal Children'S Hospital
Melbourne, Australia
- Sydney Children'S Hospital
Westmead, Australia
- Ghent University Hospital
Ghent, Belgium
- University Hospitals Leuven
Leuven, Belgium
- Alberta'S Children Hospital
Calgary, Alberta, Canada
- British Columbia Children'S Hospital
Vancouver, British Columbia, Canada
- Children'S Hospital of Eastern Ontario
Ottawa, Ontario, Canada
- Montreal Children'S Hospital
Montreal, Quebec, Canada
- University Hospital Brno
Brno, Czechia
- Charles University
Prague, Czechia
- Agia Sofia Children'S Hospital
Athens, Greece
- Schneider Children'S Medical Center
Petah Tikvah, Israel
- Leiden University Medical Center
Leiden, Netherlands
- Radboud University
Nijmegen, Netherlands
- Sant Joan de Deu Hospital - Barcelona, Spain
Barcelona, Spain
- Hospital Universitario Y Politécnico la Fe
Valencia, Spain
- Queen Silvia Children'S Hospital
Gothenburg, Sweden
- Birmingham Heartlands Hospital
Birmingham, United Kingdom
- Royal Hospital for Children
Glasgow, United Kingdom
- Leeds Teaching Hospital Trust
Leeds, United Kingdom
- Alder Hey Children'S Nhs Foundation Trust
Liverpool, United Kingdom
- Great Ormond Street Hospital
London, United Kingdom
- Newcastle Upon Tyne Hospitals Nhs Foundation Trust
Newcastle upon Tyne, United Kingdom
- University of California Davis
Davis, California, United States
- University of California Los Angeles
Los Angeles, California, United States
- Children'S Hospital Colorado
Aurora, Colorado, United States
- Nemours Children'S Hospital
Orlando, Florida, United States
- Ann & Robert H. Lurie Children'S Hospital
Chicago, Illinois, United States
- Gillette Children'S Speciality Health Care
Saint Paul, Minnesota, United States
- Duke Children'S Hospital
Durham, North Carolina, United States
- University of Texas Southwestern Medical Center
Dallas, Texas, United States
- Children'S Hospital of Virginia of Virginia Commonwealth University
Richmond, Virginia, United States
- Seattle Children'S Hospital
Seattle, Washington, United States
Site investigator names are published by ClinicalTrials.gov only while a study recruits, so completed studies list sites without them.
Drugs studied
The drug and biological interventions registered on this study, normalized to their RxNorm ingredient where possible.
Registration & key dates
Registry attributes as recorded on the ClinicalTrials.gov study record.
- Start
- 29 Jun 2018
- Primary completion
- 23 Feb 2021
- Completion
- 19 Aug 2021
- Last update posted
- 9 Mar 2023
- Registered enrollment
- 121
- Registry id
- NCT03439670
Source: ClinicalTrials.gov, via the CTTI AACT database, data as of 24 Aug 2026. Registry facts are shown as registered by the study sponsor; findmyKOL does not interpret them.