At a glance
ClinicalIndex Comparison RecordStandardized by ClinicalIndex from the ClinicalTrials.gov record · verify against the source.
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
In Brief
A Phase 3 clinical trial evaluating PF-06939926 and Placebo for Duchenne Muscular Dystrophy. Active but no longer recruiting, targeting 114 participants across 53 sites in 15 countries.
Detailed 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.
Study Details
Timeline
Interventions
PF-06939926 will be administered as a single IV infusion at Year 1 for Cohort 1.
Placebo will be administered as a single IV infusion at Year 1 for Cohort 2.
Placebo will be administered as a single IV infusion at Year 2 for Cohort 1.
PF-06939926 will be administered as a single IV infusion at Year 2 for Cohort 2