Status:
TERMINATED
Study of Fordadistrogene Movaparvovec in Early Stage Duchenne Muscular Dystrophy
Lead Sponsor:
Pfizer
Conditions:
Muscular Dystrophy, Duchenne
Eligibility:
MALE
2-3 years
Phase:
PHASE2
Brief Summary
The study will evaluate the safety and dystrophin expression following gene therapy in boys with Duchenne Muscular Dystrophy (DMD). It is a single-arm, non-randomized, open-label study
Detailed Description
The study will assess the safety and tolerability of fordadistrogene movaparvovec gene therapy. Approximately 10 participants will be enrolled in the study and receive a single IV infusion of PF-06939...
Eligibility Criteria
Inclusion
- Confirmed diagnosis of DMD by prior genetic testing.
Exclusion
- Any of the following genetic abnormalities in the dystrophin gene: a. Any mutation (exon deletion, exon duplication, insertion, or point mutation) affecting any exon between exon 9 and exon 13, inclusive; OR b. A deletion that affects both exon 29 and exon 30; OR c. A deletion that affects any exons between 56-71, inclusive.
- Positive test performed by Pfizer for neutralizing antibodies to AAV9.
- Any prior treatment with gene therapy.
- Any treatment designed to increase dystrophin expression within 6 months prior to screening (including, but not limited to, exon-skipping and nonsense read through).
- Previous or current treatment with oral glucocorticoids or other immunosuppressive agents for the indication of DMD.
- Abnormality in specified laboratory tests, including blood counts, liver and kidney function.
Key Trial Info
Start Date :
August 8 2022
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
October 3 2025
Estimated Enrollment :
10 Patients enrolled
Trial Details
Trial ID
NCT05429372
Start Date
August 8 2022
End Date
October 3 2025
Last Update
October 21 2025
Active Locations (13)
Enter a location and click search to find clinical trials sorted by distance.
1
UF Health Shands Hospital
Gainesville, Florida, United States, 32610
2
University of Florida
Gainesville, Florida, United States, 32610
3
The Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States, 19104
4
The Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States, 19146