Gene Therapy (PF-06939926) for Duchenne Muscular Dystrophy

Palo Alto (17 mi)

Age: < 18

Sex: Male

Travel: May be covered

Time Reimbursement: Varies

Trial Phase: Phase 2

Waitlist Available

Sponsor: Pfizer

No Placebo Group

Prior Safety Data

Trial Summary

What is the purpose of this trial?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 Criteria

This trial is for boys with a confirmed diagnosis of Duchenne Muscular Dystrophy (DMD) who haven't had gene therapy before. They must have normal blood counts and liver/kidney function, no specific genetic mutations in the dystrophin gene, and no recent treatments aimed at increasing dystrophin expression.

Inclusion Criteria

I have a confirmed genetic diagnosis of Duchenne Muscular Dystrophy.

Exclusion Criteria

My genetic test shows a specific mutation in the dystrophin gene.

I have taken steroids or other immune-weakening drugs for Duchenne Muscular Dystrophy.

I have tested positive for antibodies against AAV9.

I have never had gene therapy before.

Treatment Details

The study tests Fordadistrogene Movaparvovec (PF-06939926), a gene therapy for DMD. It's an open-label study where all participants receive the treatment to assess safety and how well it promotes dystrophin production.

1Treatment groups

Experimental Treatment

Group I: PF-06939926Experimental Treatment1 Intervention