A Study to Evaluate the Safety and Tolerability of PF-06939926 Gene Therapy in Duchenne Muscular Dystrophy

PHASE1TerminatedINTERVENTIONAL
Enrollment

23

Participants

Timeline

Start Date

January 23, 2018

Primary Completion Date

March 28, 2022

Study Completion Date

July 28, 2025

Conditions
Duchenne Muscular Dystrophy
Interventions
GENETIC

PF-06939926

"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."

Trial Locations (22)

27705

Duke Neurology, Durham

Duke University Medical Center, Lenox Baker Children's Hospital, Durham

27710

Biospecimen Repository & Processing Core - BPRC, Durham

Duke Cardiovascular Magnetic Resonance Center, Durham

Duke Children's Hospital & Health Center, Durham

Duke University Hospital Investigational Drug Services (IDS) Pharmacy, Durham

84108

CCTS Clinical Research Center, Salt Lake City

University of Utah Imaging and Neurosciences Center, Salt Lake City

84112

University of Utah Hospital & Clinics Investigational Drug Services, Salt Lake City

University of Utah Hospital, Salt Lake City

84113

Primary Children's Hospital, Salt Lake City

84132

University of Utah Clinical Neurosciences Center, Salt Lake City

90095

MRI Research Center, Los Angeles

Reed Neurological Research Center, Los Angeles

Ronald Reagan UCLA Medical Center (Investigational Drug Section), Los Angeles

Ronald Reagan UCLA Medical Center - Interventional Radiology, Los Angeles

Ronald Reagan UCLA Medical Center Drug Information Center, Los Angeles

UCLA (David Geffen School of Medicine), Los Angeles

UCLA Children's Heart Center, Los Angeles

UCLA Mattel Children's Hospital, Los Angeles

UCLA Medical Center, Los Angeles

UCLA Outpatient Surgery Center, Los Angeles

Sponsors

Lead Sponsor

All Listed Sponsors
lead

Pfizer

INDUSTRY

NCT03362502 - A Study to Evaluate the Safety and Tolerability of PF-06939926 Gene Therapy in Duchenne Muscular Dystrophy | Biotech Hunter | Biotech Hunter