Study to Evaluate the Safety and Efficacy of PF-06939926 for the Treatment of Duchenne Muscular Dystrophy

NCTID	NCT04281485 (View at clinicaltrials.gov)
Description	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. The one third of participants who are randomized to the placebo arm will have an opportunity for treatment with gene therapy at the beginning of the second year. (Show More)
Development Status	Inactive
Indication	Duchenne Muscular Dystrophy (DMD)
Disease Ontology Term	DOID:11723
Compound Name	PF-06939926
Compound Alias	Fordadistrogene movaparvovec
Compound Description	AAV9.hCK.Hopti-Dys3978.spA
Sponsor	Pfizer
Funder Type	Industry
Recruitment Status	Active not recruiting
Enrollment Count	114 (ACTUAL)
Results Posted	View Results

Target Gene/Variant	Mini-dystrophin
Therapy Type	Gene transfer
Therapy Route	In-vivo
Mechanism of Action	Functional gene replacement
Route of Administration	Intravenous
Drug Product Type	Viral vector
Delivery System	Viral transduction
Vector Type	AAV9
Dose 1	Phase 1: 1E14 vg/kg (n=3)
Dose 2	Phase 1: 3E14 vg/kg (n=19)
Dose 3	Phase 3: 2E14 vg/kg

Eligible Age	4 Years - 7 Years
Standard Ages	Child
Sexes Eligible for Study	MALE
Eligibility Criteria	Key inclusion criteria: 1. Confirmed diagnosis of Duchenne muscular dystrophy by prior genetic testing 2. 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 3. Ambulatory, as assessed by protocol-specified criteria Key exclusion criteria: 1. Positive test performed by Pfizer for neutralizing antibodies to AAV9 2. Any treatment designed to increase dystrophin expression within 6 months prior to screening (e.g., Translarna™, EXONDYS 51™, VYONDYS 53™) 3. Any prior treatment with gene therapy 4. Any non-healed injury that may impact functional testing (eg NSAA) 5. Abnormality in specified laboratory tests, including blood counts, liver and kidney function 6. Any of the following genetic abnormalities in the dystrophin gene: 1. Any mutation (exon deletion, exon duplication, insertion, or point mutation) affecting any exon between exon 9 and exon 13, inclusive; OR 2. A deletion that affects both exon 29 and exon 30;OR 3. A deletion that affects any exons between 56-71, inclusive. View Inclusion and Exclusion Criteria at ClinicalTrials.gov

No.of Trial Sites	53
Locations	United States,Japan,United Kingdom,Switzerland,Russia,Spain,Canada,South Korea,Belgium,Taiwan,Italy,Israel,Australia,France,Germany

Has US IND	True
FDA Designations	Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation
Recent Updates	Phase 3 study did not meet its primary efficacy endpoint, discontinuation was announced Q3 2024