Study to Evaluate the Safety and Efficacy of PF-06939926 for the Treatment of Duchenne Muscular Dystrophy
Running, not enrolling · Phase 3 · Has a placebo group
Conditions studied: Duchenne Muscular Dystrophy
In brief
The study will evaluate the safety and efficacy of gene therapy in boys with DMD. It is a randomized, double-blind, placebo-controlled study with two thirds of participants assigned to gene therapy. The one third of participants who are randomized to the placebo arm will have an opportunity for treatment with gene therapy at the beginning of the second year.
Key facts
- Study ID
- NCT04281485
- Run by
- Pfizer
- People needed
- 114
- Starts
- 2020-11-05
- Expected to finish
- 2039-04-15
- Last updated by the study team
- 2026-07-22
Who can join
Age: 4 and older, up to 7. Sex: male. Healthy volunteers: not accepted.
Where it is running
- Arkansas Children's — Little Rock, Arkansas, United States
- UCLA Medical Center — Los Angeles, California, United States
- University of Florida — Gainesville, Florida, United States
- University of Iowa Hospitals and Clinics — Iowa City, Iowa, United States
- KU Clinical Research Center - Clinical and Translational Science Unit (CTSU) - Fairway — Fairway, Kansas, United States
- KU Clinical Research Center - Clinical and Translational Science Unit (CTSU) - Rainbow — Kansas City, Kansas, United States
- University of Kansas Hospital - Investigational Pharmacy — Kansas City, Kansas, United States
- University of Kansas Hospital - Pediatric and Pediatric ICU - Operating Room — Kansas City, Kansas, United States
- University of Kansas Medical Center — Kansas City, Kansas, United States
- Pediatric Cardiology — Prairie Village, Kansas, United States
- Lenox Baker Children's Hospital — Durham, North Carolina, United States
- The Children's Hospital of Philadelphia — Philadelphia, Pennsylvania, United States
- Primary Childrens Hospital — Salt Lake City, Utah, United States
- University of Utah Clinical Neurosciences Center — Salt Lake City, Utah, United States
- University of Utah Hospital — Salt Lake City, Utah, United States
- Seattle Children's — Seattle, Washington, United States
- The Children's Hospital at Westmead — Westmead, New South Wales, Australia
- The Royal Children's Hospital Melbourne — Parkville, Victoria, Australia
- Perth Children's Hospital — Nedlands, Western Australia, Australia
- UZ Gent — Ghent, Belgium
- UZ leuven — Leuven, Belgium
- Alberta Children's Hospital — Calgary, Alberta, Canada
- Children's Hospital - London Health Sciences Centre — London, Ontario, Canada
- Childrens Hospital of Eastern Ontario — Ottawa, Ontario, Canada
- Arkansas Children's Hospital — Little Rock, Arkansas, United States
Full record on ClinicalTrials.gov
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