NCT05429372

Study of Fordadistrogene Movaparvovec in Early Stage Duchenne Muscular Dystrophy

A PHASE 2, MULTICENTER, SINGLE-ARM STUDY TO EVALUATE THE SAFETY AND DYSTROPHIN EXPRESSION AFTER FORDADISTROGENE MOVAPARVOVEC (PF-06939926) ADMINISTRATION IN MALE PARTICIPANTS WITH EARLY STAGE DUCHENNE MUSCULAR DYSTROPHY

• Status: Terminated
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 10 (actual)
• Sponsor: Pfizer · Industry
• Sex: Male
• Age: 2 Years – 3 Years
• Healthy volunteers: Not accepted

Summary

The study will evaluate the safety and dystrophin expression following gene therapy in boys with Duchenne Muscular Dystrophy (DMD). It is a single-arm, non-randomized, open-label study

Detailed description

The study will assess the safety and tolerability of fordadistrogene movaparvovec gene therapy. Approximately 10 participants will be enrolled in the study and receive a single IV infusion of PF-06939926; there is no placebo arm. The study includes boys who are at least 2 years old and less than 4 years old (including 3 year olds up until their 4th birthday). All boys will need to be negative for neutralizing antibodies against AAV9, as measured by the test done for the study as part of screening. The primary analysis will occur when all participants have completed visits through Week 52 (or withdrawn from the study prior to Week 52). All participants will be followed in the study for 5 years after treatment with gene therapy.

Conditions

• Muscular Dystrophy, Duchenne

Interventions

Type	Name	Description
GENETIC	PF-06939926	All participants will receive a single dose of PF-06939926 on Day 1.

Timeline

• Start date: 2022-08-08
• Primary completion: 2025-10-03
• Completion: 2025-10-03
• First posted: 2022-06-23
• Last updated: 2025-10-21

Locations

13 sites across 2 countries: United States, Australia

Regulatory

• FDA-regulated drug study