Clinical Trial

Study to Evaluate the Safety and Efficacy of PF-06939926 for the Treatment of Duchenne Muscular Dystrophy

Active, Not Recruiting Phase 3
View on ClinicalTrials.gov →
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.
Protocol Amendment History 77 amendments
This ClinicalTrials.gov record has been amended 77 times since 2020-02-20; most recent amendment 2026-07-21.
Status change: Recruiting → Active, Not Recruiting 2023-05-05
Status change: Active, Not Recruiting → Recruiting 2022-05-09
Status change: Recruiting → Active, Not Recruiting 2022-03-11
Status change: Active, Not Recruiting → Recruiting 2022-03-09
Status change: Recruiting → Active, Not Recruiting 2022-01-06
Status change: Not Yet Recruiting → Recruiting 2020-12-04
Trial Details
NCT Number NCT04281485
Lead Sponsor Pfizer
Conditions Duchenne Muscular Dystrophy
Enrollment 114 participants
Start Date 2020-11-05
Primary Completion 2024-05-15 (estimated)
Study Completion 2039-04-15 (estimated)
Updated on ClinicalTrials.gov 2026-07-22