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

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.

Timeline

Start

2020-11-05

Primary completion

2024-05-15

Completion

2039-04-15

Outcome

Missed primary endpoint

paper The study did not meet its primary efficacy endpoint. PMID 41722591 ↗

Publications

• Muntoni F, Nascimento A, Shin J, Guglieri M, Stettner GM, Veerapandiyan A, Gallo S, Shi H, Gundapaneni B, Neelakantan S, Lobello K, Shen Q, Levy DI, Mercuri E; CIFFREO Study Group. 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 Mar;25(3):245-255. doi: 10.1016/S1474-4422(26)00036-0.

Drugs

Indications