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Gene Therapy

Gene Therapy (PF-06939926) for Duchenne Muscular Dystrophy

Phase 2
Waitlist Available
Research Sponsored by Pfizer
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Confirmed diagnosis of DMD by prior genetic testing
Be younger than 18 years old
Must not have
Any of the following genetic abnormalities in the dystrophin gene: a. Any mutation affecting any exon between exon 9 and exon 13, inclusive; b. A deletion that affects both exon 29 and exon 30; c. A deletion that affects any exons between 56-71, inclusive
Previous or current treatment with oral glucocorticoids or other immunosuppressive agents for the indication of DMD
Timeline
Screening 3 weeks
Treatment Varies
Follow Up at week 9, week 52 and year 5 (if available)
Awards & highlights
No Placebo-Only Group

Summary

This trialwill study gene therapy to help boys with Duchenne Muscular Dystrophy (DMD) with safety and muscle strength. No randomization, open to all.

Who is the study for?
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.
What is being tested?
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.
What are the potential side effects?
Potential side effects may include immune reactions to the therapy vector AAV9, changes in liver enzymes indicating liver stress, possible allergic responses, or other unforeseen issues related to gene therapy.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
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I have a confirmed genetic diagnosis of Duchenne Muscular Dystrophy.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:
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My genetic test shows a specific mutation in the dystrophin gene.
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I have taken steroids or other immune-weakening drugs for Duchenne Muscular Dystrophy.
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I have tested positive for antibodies against AAV9.
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I have never had gene therapy before.

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~at week 9, week 52 and year 5 (if available)
This trial's timeline: 3 weeks for screening, Varies for treatment, and at week 9, week 52 and year 5 (if available) for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.
Secondary study objectives
Distribution of mini-dystrophin expression in muscle
Level of mini-dystrophin expression in muscle
Number of participants with abnormal biochemistry test results
+2 more

Awards & Highlights

No Placebo-Only Group
All patients enrolled in this study will receive some form of active treatment.

Trial Design

1Treatment groups
Experimental Treatment
Group I: PF-06939926Experimental Treatment1 Intervention

Find a Location

Who is running the clinical trial?

PfizerLead Sponsor
4,669 Previous Clinical Trials
17,861,914 Total Patients Enrolled
Pfizer CT.gov Call CenterStudy DirectorPfizer
3,553 Previous Clinical Trials
14,902,916 Total Patients Enrolled

Media Library

PF-06939926 (Gene Therapy) Clinical Trial Eligibility Overview. Trial Name: NCT05429372 — Phase 2
Duchenne Muscular Dystrophy Research Study Groups: PF-06939926
Duchenne Muscular Dystrophy Clinical Trial 2023: PF-06939926 Highlights & Side Effects. Trial Name: NCT05429372 — Phase 2
PF-06939926 (Gene Therapy) 2023 Treatment Timeline for Medical Study. Trial Name: NCT05429372 — Phase 2
~0 spots leftby Dec 2024
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