Study of Fordadistrogene Movaparvovec in Early Stage Duchenne Muscular Dystrophy

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

TERMINATED

Clinical Phase

PHASE2

Total Enrollment

10 participants

Study Classification

INTERVENTIONAL

Study Start Date

2022-08-08

Study Completion Date

2025-10-03

Brief Summary

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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

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Detailed Description

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The study will assess the safety and tolerability of fordadistrogene movaparvovec gene therapy. Approximately 10 participants will be enrolled in the study and receive a single IV infusion of PF-06939926; there is no placebo arm. The study includes boys who are at least 2 years old and less than 4 years old (including 3 year olds up until their 4th birthday). All boys will need to be negative for neutralizing antibodies against AAV9, as measured by the test done for the study as part of screening.

The primary analysis will occur when all participants have completed visits through Week 52 (or withdrawn from the study prior to Week 52). All participants will be followed in the study for 5 years after treatment with gene therapy.

Conditions

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Muscular Dystrophy, Duchenne

Study Design

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Allocation Method

NA

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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PF-06939926

Group Type EXPERIMENTAL

PF-06939926

Intervention Type GENETIC

All participants will receive a single dose of PF-06939926 on Day 1.

Interventions

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PF-06939926

All participants will receive a single dose of PF-06939926 on Day 1.

Intervention Type GENETIC

Other Intervention Names

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Fordadistrogene Movaparvovec

Eligibility Criteria

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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.

Minimum Eligible Age

2 Years

Maximum Eligible Age

3 Years

Eligible Sex

MALE

Accepts Healthy Volunteers

No

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Pfizer CT.gov Call Center

Role: STUDY_DIRECTOR

Pfizer

Locations

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UF Health Shands Hospital

Gainesville, Florida, United States

Site Status

University of Florida

Gainesville, Florida, United States

Site Status

The Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, United States

Site Status