Two-Part Study for Dose Determination of Vesleteplirsen (SRP-5051) (Part A), Then Dose Expansion (Part B) in Participants With Duchenne Muscular Dystrophy Amenable to Exon 51-Skipping Treatment
Stopped early · Phase 2
Conditions studied: Duchenne Muscular Dystrophy
In brief
This study will be comprised of 2 parts: 1) Part A (Multiple Ascending Dose \[MAD\]) will be conducted to evaluate the safety and tolerability of vesleteplirsen at MAD levels to determine the maximum tolerated dose (MTD), and 2) Part B will be conducted to further evaluate the vesleteplirsen doses selected in Part A. Participants enrolling in Part B will be those who completed Part A or Study 5051-102 (NCT03675126) and meet applicable eligibility criteria for Part B, as well as additional participants who meet applicable eligibility criteria for enrollment at the beginning of Part B.
Key facts
- Study ID
- NCT04004065
- Run by
- Sarepta Therapeutics, Inc.
- People needed
- 62
- Starts
- 2019-06-26
- Expected to finish
- 2025-02-07
- Last updated by the study team
- 2025-03-10
Who can join
Age: 7 and older, up to 21. Sex: male. Healthy volunteers: not accepted.
Where it is running
- University of California Davis Health — Sacramento, California, United States
- Connecticut Children's — Farmington, Connecticut, United States
- Northwest Florida Clinical Research Group, LLC — Gulf Breeze, Florida, United States
- Rare Disease Research, LLC — Atlanta, Georgia, United States
- University of Iowa Hospitals and Clinics — Iowa City, Iowa, United States
- University of Kansas Medical Center Research Inst. — Kansas City, Kansas, United States
- University of Massachusetts — Worcester, Massachusetts, United States
- UPMC Children's Hospital of Pittsburgh — Pittsburgh, Pennsylvania, United States
- Austin Neuromuscular Center — Austin, Texas, United States
- Children's Health Ambulatory Pavilion — Dallas, Texas, United States
- Seattle Children's — Seattle, Washington, United States
- Universitair Ziekenhuis Gent — Ghent, Belgium
- UZ Leuven — Leuven, Belgium
- Children's Hospital - London Health Sciences Centre (LHSC) — London, Ontario, Canada
- University of Essen - Children's Hospital — Essen, Germany
- Klinikum der Universität München — Munich, Germany
- Fondazione Policlinico Universitario A Gemelli — Rome, Italy
- A.O.U. Citta della Salute e della Scienza di Torino - SS Malattie Neuromuscolari, Department of Neurosciences — Torino, Italy
- Leiden University Medical Center — Leiden, Netherlands
- Hospital Sant Joan de Déu. U.B. — Barcelona, Spain
- Hospital Universitari I Politecnic La Fe de Valencia — Valencia, Spain
- Alder Hey Children's NHS Foundation Trust — Liverpool, Lancashire, United Kingdom
- Royal Hospital for Children (Glasgow) — Glasgow, United Kingdom
- Great Ormond Street Hospital for Children NHS Foundation Trust — London, United Kingdom
- Oxford University Hospial NHS Foundation Trust, John Radcliffe Hospital — Oxford, United Kingdom
Full record on ClinicalTrials.gov
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