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NCT04281485

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

Study 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.

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Interventions

PF-06939926GENETIC
PF-06939926 will be administered as a single IV infusion at Year 1 for Cohort 1.
PlaceboOTHER
Placebo will be administered as a single IV infusion at Year 1 for Cohort 2.
PlaceboOTHER
Placebo will be administered as a single IV infusion at Year 2 for Cohort 1.
PF-06939926GENETIC
PF-06939926 will be administered as a single IV infusion at Year 2 for Cohort 2

Study Locations

FacilityCityStateCountry
Arkansas Children's HospitalLittle RockArkansasUnited States
Arkansas Children'sLittle RockArkansasUnited States
UCLA Medical CenterLos AngelesCaliforniaUnited States
University of FloridaGainesvilleFloridaUnited States
University of Iowa Hospitals and ClinicsIowa CityIowaUnited States
KU Clinical Research Center - Clinical and Translational Science Unit (CTSU) - FairwayFairwayKansasUnited States
KU Clinical Research Center - Clinical and Translational Science Unit (CTSU) - RainbowKansas CityKansasUnited States
University of Kansas Hospital - Investigational PharmacyKansas CityKansasUnited States
University of Kansas Hospital - Pediatric and Pediatric ICU - Operating RoomKansas CityKansasUnited States
University of Kansas Medical CenterKansas CityKansasUnited States
Pediatric CardiologyPrairie VillageKansasUnited States
Lenox Baker Children's HospitalDurhamNorth CarolinaUnited States
The Children's Hospital of PhiladelphiaPhiladelphiaPennsylvaniaUnited States
Primary Childrens HospitalSalt Lake CityUtahUnited States
University of Utah Clinical Neurosciences CenterSalt Lake CityUtahUnited States
University of Utah HospitalSalt Lake CityUtahUnited States
Seattle Children'sSeattleWashingtonUnited States
The Children's Hospital at WestmeadWestmeadNew South WalesAustralia
The Royal Children's Hospital MelbourneParkvilleVictoriaAustralia
Perth Children's HospitalNedlandsWestern AustraliaAustralia
UZ GentGhentBelgium
UZ leuvenLeuvenBelgium
Alberta Children's HospitalCalgaryAlbertaCanada
Children's Hospital - London Health Sciences CentreLondonOntarioCanada
Childrens Hospital of Eastern OntarioOttawaOntarioCanada
The Hospital For Sick ChildrenTorontoOntarioCanada
CHU de Nantes- Hotel DieuNantesFrance
Hopital NeckerParisFrance
Charité - Universitätsmedizin BerlinBerlinGermany
Universitatsklinikum EssenEssenGermany
Hadassah University Medical Center, Ein KeremJerusalemIsrael
Schneider Children's Medical Center of IsraelPetach TikvahIsrael
Fondazione Policlinico Universitario Agostino Gemelli IRCCSRomaItaly
IRCCS Ospedale Pediatrico Bambino GesùRomeItaly
Nagoya City University HospitalNagoyaAichi-kenJapan
Hyogo College of Medicine College HospitalNishinomiyaHyōgoJapan
National Center of Neurology and PsychiatryTokyoJapan
Saint Petersburg State Paediatric Medical UniversitySaint PetersburgRussia
State Autonomous Healthcare Institution of Sverdlovsk Region Children's City Clinical Hospital No 9YekaterinburgRussia
Pusan National University Yangsan HospitalYangsanGyeongsangnam-doSouth Korea
Seoul National University HospitalSeoulSouth Korea
Samsung Medical CenterSeoulSouth Korea
Hospital Sant Joan de DéuEsplugues de LlobregatBarcelonaSpain
Hospital Universitario Vall d'HebronBarcelonaSpain
Hospital Universitari i Politecnic La Fe de ValenciaValenciaSpain
Inselspital, University Children's Hospital BerneBernSwitzerland
Universitaets-Kinderspital ZuerichZurichSwitzerland
Kaohsiung Medical University Chung-Ho Memorial HospitalKaohsiung CityTaiwan
National Taiwan University HospitalTaipeiTaiwan
Taipei Veterans General HospitalTaipeiTaiwan
The Newcastle upon Tyne Hospitals NHS Foundation Trust, Royal Victoria InfirmaryNewcastle upon TyneEnglandUnited Kingdom
Alder Hey Children's NHS Foundation TrustLiverpoolMerseysideUnited Kingdom
Great Ormond Street Institute of Child HealthLondonUnited Kingdom

Official Trial Information

View on ClinicalTrials.gov

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