Study of Fordadistrogene Movaparvovec in Early Stage Duchenne Muscular Dystrophy

Phase 2

Active, not recruiting

• Conditions: Muscular Dystrophy, Duchenne
• Interventions: Genetic: PF-06939926

• Registration Number: NCT05429372
• Lead Sponsor: Pfizer

Brief Summary

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

Detailed Description

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The primary analysis will occur when all participants have completed visits through Week 52 (or withdrawn from the study prior to Week 52).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.

Basic Information
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Recruitment & Eligibility
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Study & Design
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Trial Locations
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Related News

Study Timeline

• Study First Submitted: 2021-10-08
• Study First Submitted QC: 2022-06-20
• Study First Posted: 2022-06-23
• Study Start: 2022-08-08
• Status Verified: 2024-04
• Last Update Submitted: 2024-04-03
• Last Update Posted: 2024-04-04
• Primary Completion: 2024-12-27
• Study Completion: 2029-01-03

Recruitment & Eligibility

• Status: ACTIVE_NOT_RECRUITING
• Sex: Male
• Target Recruitment: 10

Inclusion Criteria

• Confirmed diagnosis of DMD by prior genetic testing.

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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.

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Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
PF-06939926	PF-06939926	-

Primary Outcome Measures

Name	Time	Method
Number of participants with abnormal and clinically relevant changes in neurological examinations	Through Week 52
Incidence and severity of Treatment-Emergent Adverse Events and Serious Adverse Events	Through Week 52
Number of participants with abnormal hematology test results	Through Week 52	Blood samples will be collected from subjects for the analysis of hematology
Number of participants with abnormal and clinically relevant changes on electrocardiogram (ECG)	Through Week 52
Number of participants with abnormal and clinically relevant changes on echocardiogram	Through Week 52
Number of participants with abnormal biochemistry test results	Through Week 52	Blood samples will be collected from subjects for the analysis of biochemistry
Number of participants with abnormal urine analysis	Through Week 52	Urine samples will be collected from subjects for the analysis of urine
Number of participants with abnormal and clinically relevant changes in body weight	Through Week 52
Number of participants with abnormal and clinically relevant changes on cardiac troponin I	Through Week 52
Number of participants with abnormal and clinically relevant changes in vital signs	Through Week 52

Secondary Outcome Measures

Name	Time	Method
Distribution of mini-dystrophin expression in muscle	At Week 9, Week 52 and Year 5 (if available)	Mini-dystrophin distribution from a muscle biopsy will be assessed by immunofluorescence
Number of participants with abnormal urine analysis	Through 5 years	Urine samples will be collected from subjects for the analysis of urine
Number of participants with abnormal and clinically relevant changes in neurological examinations	Through 5 years
Number of participants with abnormal and clinically relevant changes in body weight	Through 5 years
Level of mini-dystrophin expression in muscle	At Week 9, Week 52 and Year 5 (if available)	Mini-dystrophin expression level from a muscle biopsy will be assessed by liquid chromatography mass spectrometry
Number of participants with abnormal biochemistry test results	Through 5 years	Blood samples will be collected from subjects for the analysis of biochemistry
Number of participants with abnormal and clinically relevant changes on electrocardiogram (ECG)	Through 5 years
Number of participants with abnormal and clinically relevant changes on echocardiogram	Through 5 years
Incidence and severity of Treatment-Emergent Adverse Events and Serious Adverse Events	Through 5 years
Number of participants with abnormal and clinically relevant changes in vital signs	Through 5 years
Number of participants with abnormal hematology test results	Through 5 years	Blood samples will be collected from subjects for the analysis of hematology
Number of participants with abnormal and clinically relevant changes on cardiac troponin I	Through 5 years

Trial Locations

Locations (12)

UF Health Shands Hospital; 🇺🇸 Gainesville, Florida, United States

The Children's Hospital of Philadelphia; 🇺🇸 Philadelphia, Pennsylvania, United States

The Children's Hospital at Westmead; 🇦🇺 Westmead, New South Wales, Australia

The Royal Children's Hospital Melbourne; 🇦🇺 Parkville, Victoria, Australia

Perth Children's Hospital; 🇦🇺 Nedlands, Western Australia, Australia

University of Florida; 🇺🇸 Gainesville, Florida, United States

CTSI Clinical Research Center; 🇺🇸 Salt Lake City, Utah, United States

University of Utah Imaging and Neurosciences Center; 🇺🇸 Salt Lake City, Utah, United States

University of Utah Hospital & Clinics Investigational Drug Services; 🇺🇸 Salt Lake City, Utah, United States

Primary Children's Hospital; 🇺🇸 Salt Lake City, Utah, United States

University of Utah Hospital; 🇺🇸 Salt Lake City, Utah, United States

University of Utah Clinical Neurosciences Center; 🇺🇸 Salt Lake City, Utah, United States