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

Brief summary (registry, in English)

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.

What you commit to

• Study type: Interventional · Phase 3
• Participants enrolled: 114
• From 5 Nov 2020 to the primary completion date, 15 May 2024
• 52 sites in 15 countries
• Visits and tests: ask the trial team, the registry does not list them

Built from the registry fields of ClinicalTrials.gov. It describes the study, not your situation.

Who cannot take part

Copied verbatim from ClinicalTrials.gov and grouped by topic. Only the trial team decides who can take part.

Who the study is looking for

Age in the registry: 4 years to 7 years · Sex: Male · Does not accept healthy volunteers

Full criteria on ClinicalTrials.gov

Sites (52)

Sites in registry order. Pick your city on the disease page to sort them by distance.

Related publications

• Safety and efficacy of fordadistrogene movaparvovec in ambulatory participants with Duchenne muscular dystrophy (CIFFREO): a phase 3, double-blind, randomised, placebo-controlled study.Lancet Neurol · 2026 · PMID 41722591