Status:
ACTIVE_NOT_RECRUITING
Study to Evaluate the Safety and Efficacy of PF-06939926 for the Treatment of Duchenne Muscular Dystrophy
Lead Sponsor:
Pfizer
Conditions:
Duchenne Muscular Dystrophy
Eligibility:
MALE
4-7 years
Phase:
PHASE3
Brief 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. T...
Detailed Description
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 ...
Eligibility Criteria
Inclusion
- 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
- 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
- Any of the following genetic abnormalities in the dystrophin gene:
- Any mutation (exon deletion, exon duplication, insertion, or point mutation) affecting any exon between exon 9 and exon 13, inclusive; OR
- A deletion that affects both exon 29 and exon 30;OR
- A deletion that affects any exons between 56-71, inclusive.
Exclusion
Key Trial Info
Start Date :
November 5 2020
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
April 15 2039
Estimated Enrollment :
114 Patients enrolled
Trial Details
Trial ID
NCT04281485
Start Date
November 5 2020
End Date
April 15 2039
Last Update
January 2 2026
Active Locations (53)
Enter a location and click search to find clinical trials sorted by distance.
1
Arkansas Children's Hospital
Little Rock, Arkansas, United States, 72202
2
Arkansas Children's
Little Rock, Arkansas, United States, 72202
3
UCLA Medical Center
Los Angeles, California, United States, 90095
4
University of Florida
Gainesville, Florida, United States, 32610