Study of Fordadistrogene Movaparvovec in Early Stage Duchenne Muscular Dystrophy
Stopped early · Phase 2
Conditions studied: Muscular Dystrophy, Duchenne
In brief
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
Key facts
- Study ID
- NCT05429372
- Run by
- Pfizer
- People needed
- 10
- Starts
- 2022-08-08
- Expected to finish
- 2025-10-03
- Last updated by the study team
- 2025-10-21
Who can join
Age: 2 and older, up to 3. Sex: male. Healthy volunteers: not accepted.
You may qualify if…
- Confirmed diagnosis of DMD by prior genetic testing.
You may not qualify if…
- 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.
Where it is running
- UF Health Shands Hospital — Gainesville, Florida, United States
- University of Florida — Gainesville, Florida, United States
- The Children's Hospital of Philadelphia — Philadelphia, Pennsylvania, United States
- The Children's Hospital of Philadelphia — Philadelphia, Pennsylvania, United States
- CTSI Clinical Research Center — Salt Lake City, Utah, United States
- University of Utah Imaging and Neurosciences Center — Salt Lake City, Utah, United States
- University of Utah Hospital & Clinics Investigational Drug Services — Salt Lake City, Utah, United States
- Primary Children's Hospital — Salt Lake City, Utah, United States
- University of Utah Clinical Neurosciences Center — Salt Lake City, Utah, United States
- University of Utah Hospital — Salt Lake City, Utah, United States
- The Children's Hospital at Westmead — Westmead, New South Wales, Australia
- The Royal Children's Hospital Melbourne — Parkville, Victoria, Australia
- Perth Children's Hospital — Nedlands, Western Australia, Australia
Full record on ClinicalTrials.gov
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