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Study of Fordadistrogene Movaparvovec in Early Stage Duchenne Muscular Dystrophy

TerminatedPhase 2

Sponsor: Pfizer

Conditions: Muscular Dystrophy, Duchenne

Interventions: PF-06939926

Countries: United States, Australia

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

Eligibility overview

Sex: MALE

Age: 2 Years to 3 Years

Healthy volunteers: No

Study type: INTERVENTIONAL

Eligibility criteria

Inclusion Criteria:

* Confirmed diagnosis of DMD by prior genetic testing.

Exclusion Criteria:

* 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.

Locations (13)

Gainesville, Florida, United States
Gainesville, Florida, United States
Philadelphia, Pennsylvania, United States
Philadelphia, Pennsylvania, United States
Salt Lake City, Utah, United States
Salt Lake City, Utah, United States
Salt Lake City, Utah, United States
Salt Lake City, Utah, United States
Salt Lake City, Utah, United States
Salt Lake City, Utah, United States
Westmead, New South Wales, Australia
Parkville, Victoria, Australia
Nedlands, Western Australia, Australia