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NCT04281485 · ClinicalTrials.gov registry record · Phase 3
Study to Evaluate the Safety and Efficacy of PF-06939926 for the Treatment of Duchenne Muscular Dystrophy
A Phase 3 study of Duchenne Muscular Dystrophy, sponsored by Pfizer.
- Active
- Registry status
- Phase 3
- Development phase
- 114
- Enrollment target
- 20
- Study locations
NCT04281485: Active Phase 3 study of Duchenne Muscular Dystrophy, sponsored by Pfizer.
NCT04281485 is a Phase 3 study of Duchenne Muscular Dystrophy that is active but no longer recruiting, run by Pfizer. The registered enrollment target is 114 participants, below the 410-participant average among 48 other Duchenne Muscular Dystrophy trials with a reported enrollment target (72% lower). The trial reports 20 study locations across 12 states. According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT04281485, a Phase 3 study of Duchenne Muscular Dystrophy, is active but no longer recruiting, sponsored by Pfizer.
- ACTIVE NOT RECRUITING
- Registry status
- Phase 3
- Development phase
- 114 participants
- Enrollment target
- 20
- Study locations
Study Summary
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.
Primary Outcome
The NSAA was a 17-item test that graded performance of various functional skills using the following scale: 0 (unable to achieve independently), 1 (modified method but achieves goal independent of physical assistance from another), and 2 ("normal"- no obvious modification of activity). Total score was calculated as the sum of all 17 individual item responses and ranged from 0 (worst) to 34 (fully independent function) with higher scores indicating better function. Baseline NSAA total score is de
Conditions Studied
Interventions
- OTHER Placebo
- GENETIC PF-06939926
Study Locations (20)
Kansas
- KU Clinical Research Center - Clinical and Translational Science Unit (CTSU) - Fairway - Fairway
- KU Clinical Research Center - Clinical and Translational Science Unit (CTSU) - Rainbow - Kansas City
- University of Kansas Hospital - Investigational Pharmacy - Kansas City
- University of Kansas Hospital - Pediatric and Pediatric ICU - Operating Room - Kansas City
- University of Kansas Medical Center - Kansas City
- Pediatric Cardiology - Prairie Village
Utah
- Primary Childrens Hospital - Salt Lake City
- University of Utah Clinical Neurosciences Center - Salt Lake City
- University of Utah Hospital - Salt Lake City
Arkansas
- Arkansas Children's Hospital - Little Rock
- Arkansas Children's - Little Rock
California
- UCLA Medical Center - Los Angeles
Florida
- University of Florida - Gainesville
Iowa
- University of Iowa Hospitals and Clinics - Iowa City
North Carolina
- Lenox Baker Children's Hospital - Durham
Pennsylvania
- The Children's Hospital of Philadelphia - Philadelphia
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 114 participants |
| Start Date | 2020-11-05 |
| Est. Completion | 2039-04-15 |
| Phase | Phase 3 |
What the registry record for NCT04281485 still lists
NCT04281485 is an interventional study that assigns participants to a tested intervention. The registered 114 participants enrollment target is mid-sized for trials with a published cap, below the 410-participant average among 48 other Duchenne Muscular Dystrophy trials with a reported enrollment target (72% lower).
The record links to 1 condition, with Duchenne Muscular Dystrophy appearing as the primary indexed condition, and to 2 interventions - of which Placebo is the first listed.
NCT04281485 names 20 study sites across 12 states, led by Kansas, Utah, Arkansas.
Frequently Asked Questions
What is clinical trial NCT04281485 about?
NCT04281485 is a clinical study titled "Study to Evaluate the Safety and Efficacy of PF-06939926 for the Treatment of Duchenne Muscular Dystrophy". 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 trea...
What is the current status of trial NCT04281485?
This trial is currently active not recruiting. It is a Phase 3 study. The enrollment target is 114 participants. The study started on 2020-11-05. Estimated completion is 2039-04-15.
What conditions does trial NCT04281485 study?
This clinical trial studies the following conditions: Duchenne Muscular Dystrophy.
What interventions are being tested in trial NCT04281485?
The interventions under investigation include: Placebo (OTHER), PF-06939926 (GENETIC).
Who is sponsoring clinical trial NCT04281485?
This trial is sponsored by Pfizer, which has 1,845 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT04281485 being conducted?
This trial has 20 study locations across Arkansas, California, Florida, Iowa, Kansas. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
Learn More About Clinical Trials
Similar trials for Duchenne Muscular Dystrophy
Matched on the same primary condition, ranked to surface studies in the same phase first, then by recruiting status, no relevance scoring or editorial curation.
Where NCT04281485's enrollment target sits among peer trials
114 13th of 48 higher than 36 of 48 other Duchenne Muscular Dystrophy trials
participants (enrollment target), bucketed by value
Each bar is a band; taller bars hold more other Duchenne Muscular Dystrophy trials. The dashed line + filled bar mark this entry. Hover or tap any bar for its full count and share, and where it sits relative to this entry.
Source ClinicalTrials.gov registry export · 2026-08-08
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