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Clinical Trials List

Protocol NumberC3391003

NCT Number(ClinicalTrials.gov Identfier)NCT04281485

Active

2020-10-30 - 2039-12-04

Phase III

Recruiting3

ICD-10G71.0

Muscular dystrophy

ICD-9359.1

Hereditary progressive muscular dystrophy

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

Sponsor

Pfizer
Trial scale

Multi-Regional Multi-Center
Update

2026/02/01

Investigators and Locations

Principal Investigator Ting-Rong Hsu Division of Pediatrics

Taipei Veterans General Hospital

Co-Principal Investigator

李星原 Division of Pediatrics
Wei-Sheng Lin Division of Pediatrics

The Actual Total Number of Participants Enrolled

0 Recruiting

Principal Investigator WANG-TSO LEE Division of Pediatrics

National Taiwan University Hospital

Co-Principal Investigator

WEN-CHIN WENG Division of Pediatrics

The Actual Total Number of Participants Enrolled

0 Recruiting

Principal Investigator Yuh-Jyh Jong Division of Pediatrics

Kaohsiung Medical Univeristy Chung-Ho Memorial Hospital

Co-Principal Investigator

徐仲豪 Division of Pediatrics
Wen-Chen Liang Division of Pediatrics
施相宏 Division of Pediatrics
劉怡慶 Division of Pediatrics

The Actual Total Number of Participants Enrolled

0 Recruiting

Condition/Disease

Duchenne Muscular Dystrophy

Objectives

The study will assess the efficacy of PF-06939926 gene therapy on ambulatory function while also monitoring its safety. Approximately 99 boys with DMD will be enrolled and randomly assigned to one of two groups: approximately two thirds will be in Cohort 1 and receive gene therapy at the start of the study; approximately one third will be in Cohort 2 and receive placebo at the start of the study and receive gene therapy after one year, as long as it remains safe to do so. The treatment (PF-06939926 gene therapy or placebo) will be given as an intravenous infusion lasting up to 2 hours. The study includes boys who are at least 4 years old and less than 8 years old (including 7 year olds up until their 8th birthday). All boys will need to be on a daily dose of glucocorticoids (prednisone, prednisolone, or deflazacort) for at least 3 months prior to enrolling and to stay on daily glucocorticoids for the first 2 years of the study. 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 outcome of the study will be assessed at 52 weeks. All participants will be followed in the study for 5 years after treatment with gene therapy. The study medication, all medical tests associated with the study, and the visits to the study sites are free of charge. Participants will also be supported for travel costs associated with study visits.

Test Drug

PF-06939926

Active Ingredient

PF-06939926

Dosage Form

Sterile Solution for Infusion

Dosage

1E+14 vg/mL

Endpoints

Change from Baseline in North Star Ambulatory Assessment (NSAA) [ Time Frame: Week 52 ]
The NSAA is a 17-item test that measures gross motor function in children with Duchenne.

Inclution Criteria

Key inclusion criteria:

Confirmed diagnosis of Duchenne muscular dystrophy by prior genetic testing
Receiving a stable daily dose (at least 0.5 mg/kg/day prednisone or prednisolone, or at least 0.75 mg/kg/day deflazacort) for at least 3 months prior to Screening
Ambulatory, as assessed by protocol-specified criteria

Exclusion Criteria

Key exclusion criteria:

Positive test performed by Pfizer for neutralizing antibodies to AAV9
Any treatment designed to increase dystrophin expression within 6 months prior to screening (e.g., Translarna™, EXONDYS 51™, VYONDYS 53™)
Any prior treatment with gene therapy
Any non-healed injury that may impact functional testing (eg NSAA)
Abnormality in specified laboratory tests, including blood counts, liver and kidney function

The Estimated Number of Participants

Taiwan

12 participants
Global

99 participants