Study to Evaluate the Safety and Efficacy of PF-06939926 for the Treatment of Duchenne Muscular Dystrophy (NCT04281485)
A PHASE 3, MULTICENTER, RANDOMIZED, DOUBLE-BLIND, PLACEBO CONTROLLED STUDY TO EVALUATE THE SAFETY AND EFFICACY OF PF 06939926 FOR THE TREATMENT OF DUCHENNE MUSCULAR DYSTROPHY
🩺 Plain-English Summary (8th-Grade Level)
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.
Who can join: Review the eligibility criteria below.
Location: Arkansas Children's Hospital — Little Rock, Arkansas
Age range: 4 Years to 7 Years
🧪 Interventions in This Study
- PF-06939926 (GENETIC)
- Placebo (OTHER)
- Placebo (OTHER)
- PF-06939926 (GENETIC)
📋 "Do I Qualify?" — 1-Minute Self Screener
Instant ChecklistFull eligibility criteria are available on the official NIH protocol. View the criteria ↗
📍 All Participating Trial Locations (53 Sites)
Showing the first 25 of 53 sites.
- Arkansas Children's Hospital — Little Rock, Arkansas
- Arkansas Children's — Little Rock, Arkansas
- UCLA Medical Center — Los Angeles, California
- University of Florida — Gainesville, Florida
- University of Iowa Hospitals and Clinics — Iowa City, Iowa
- KU Clinical Research Center - Clinical and Translational Science Unit (CTSU) - Fairway — Fairway, Kansas
- KU Clinical Research Center - Clinical and Translational Science Unit (CTSU) - Rainbow — Kansas City, Kansas
- University of Kansas Hospital - Investigational Pharmacy — Kansas City, Kansas
- University of Kansas Hospital - Pediatric and Pediatric ICU - Operating Room — Kansas City, Kansas
- University of Kansas Medical Center — Kansas City, Kansas
- Pediatric Cardiology — Prairie Village, Kansas
- Lenox Baker Children's Hospital — Durham, North Carolina
- The Children's Hospital of Philadelphia — Philadelphia, Pennsylvania
- Primary Childrens Hospital — Salt Lake City, Utah
- University of Utah Clinical Neurosciences Center — Salt Lake City, Utah
- University of Utah Hospital — Salt Lake City, Utah
- Seattle Children's — Seattle, Washington
- The Children's Hospital at Westmead — Westmead, New South Wales
- The Royal Children's Hospital Melbourne — Parkville, Victoria
- Perth Children's Hospital — Nedlands, Western Australia
- UZ Gent — Ghent,
- UZ leuven — Leuven,
- Alberta Children's Hospital — Calgary, Alberta
- Children's Hospital - London Health Sciences Centre — London, Ontario
- Childrens Hospital of Eastern Ontario — Ottawa, Ontario
💰 Cost, Insurance & Patient Rights
- 100% Free Items: The investigational treatment and protocol-required procedures are supplied at zero cost by the sponsor.
- Routine Patient Care: Under federal law (ACA § 2709) and many state statutes, routine care costs are covered during participation.
- Voluntary Participation: You may withdraw at any time without affecting your standard medical care.
More studies in Duchenne Muscular Dystrophy
- Characterization of Clinical Skeletal and Cardiac Impairment in Carriers of DMD and BMD — Columbus, Ohio
- Microdystrophin Gene Transfer Study in Adolescents and Children With DMD — Los Angeles, California
- NS-050/NCNP-03 in Boys With DMD (Meteor50) — Los Angeles, California
- A Study to Assess the Efficacy and Safety of Satralizumab in Duchenne Muscular Dystrophy (DMD) — Little Rock, Arkansas
- Pediatric Radio Frequency Coils Generic — Cincinnati, Ohio
Explore by condition: All Duchenne Muscular Dystrophy clinical trials
💡 How to Participate
- Check the checklist above to see if you may qualify.
- Review the official NIH record below to find the coordinating site.
- Ask about the visit schedule, what is covered at no cost, and any travel support.
- Confirm with your own doctor before making a decision.