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NCT03362502

A PHASE 1B MULTICENTER, OPEN-LABEL, SINGLE ASCENDING DOSE STUDY TO EVALUATE THE SAFETY AND TOLERABILITY OF PF-06939926 IN AMBULATORY AND NON-AMBULATORY SUBJECTS WITH DUCHENNE MUSCULAR DYSTROPHY

Study Summary

This is a first-in-human/first-in-patient, multi-center, open-label, non-randomized, ascending dose, safety and tolerability study of a single intravenous infusion of PF-06939926 in ambulatory and non-ambulatory subjects with Duchenne muscular dystrophy (DMD). Other objectives include measurement of dystrophin expression and distribution, and assessments of muscle strength, quality, and function. A total of approximately 22 subjects will receive PF-06939926, and these will include both ambulatory and non-ambulatory subjects. Up to 13 subjects may be included in a cohort that includes the concomitant medication, sirolimus. In order to mitigate unanticipated risks to subject safety, enrollment will be staggered within and between two planned dose-levels and will include a formal review by an external data monitoring committee (E-DMC) prior to dose progression.

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Interventions

PF-06939926GENETIC
Recombinant adeno-associated virus, serotype 9 (AAV9) carrying a truncated human dystrophin gene (mini-dystrophin) under the control of a muscle-specific promoter. Subjects will receive a single intravenous infusion of one of 2 dose levels.

Study Locations

FacilityCityStateCountry
MRI Research CenterLos AngelesCaliforniaUnited States
Reed Neurological Research CenterLos AngelesCaliforniaUnited States
Ronald Reagan UCLA Medical Center (Investigational Drug Section)Los AngelesCaliforniaUnited States
Ronald Reagan UCLA Medical Center - Interventional RadiologyLos AngelesCaliforniaUnited States
Ronald Reagan UCLA Medical Center Drug Information CenterLos AngelesCaliforniaUnited States
UCLA (David Geffen School of Medicine)Los AngelesCaliforniaUnited States
UCLA Children's Heart CenterLos AngelesCaliforniaUnited States
UCLA Mattel Children's HospitalLos AngelesCaliforniaUnited States
UCLA Medical CenterLos AngelesCaliforniaUnited States
UCLA Outpatient Surgery CenterLos AngelesCaliforniaUnited States
Duke NeurologyDurhamNorth CarolinaUnited States
Duke University Medical Center, Lenox Baker Children's HospitalDurhamNorth CarolinaUnited States
Biospecimen Repository & Processing Core - BPRCDurhamNorth CarolinaUnited States
Duke Cardiovascular Magnetic Resonance CenterDurhamNorth CarolinaUnited States
Duke Children's Hospital & Health CenterDurhamNorth CarolinaUnited States
Duke University Hospital Investigational Drug Services (IDS) PharmacyDurhamNorth CarolinaUnited States
CCTS 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
University of Utah HospitalSalt Lake CityUtahUnited States
Primary Children's HospitalSalt Lake CityUtahUnited States
University of Utah Clinical Neurosciences CenterSalt Lake CityUtahUnited States

Official Trial Information

View on ClinicalTrials.gov

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