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NCT05429372

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

Study 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

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Interventions

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

Study Locations

FacilityCityStateCountry
UF Health Shands HospitalGainesvilleFloridaUnited States
University of FloridaGainesvilleFloridaUnited States
The Children's Hospital of PhiladelphiaPhiladelphiaPennsylvaniaUnited States
The Children's Hospital of PhiladelphiaPhiladelphiaPennsylvaniaUnited States
CTSI Clinical Research CenterSalt Lake CityUtahUnited States
University of Utah Imaging and Neurosciences CenterSalt Lake CityUtahUnited States
University of Utah Hospital & Clinics Investigational Drug ServicesSalt Lake CityUtahUnited States
Primary Children's HospitalSalt Lake CityUtahUnited States
University of Utah Clinical Neurosciences CenterSalt Lake CityUtahUnited States
University of Utah HospitalSalt Lake CityUtahUnited States
The Children's Hospital at WestmeadWestmeadNew South WalesAustralia
The Royal Children's Hospital MelbourneParkvilleVictoriaAustralia
Perth Children's HospitalNedlandsWestern AustraliaAustralia

Official Trial Information

View on ClinicalTrials.gov

Data sourced from ClinicalTrials.gov. Last updated: April 14, 2026