Study of Fordadistrogene Movaparvovec in Early Stage Duchenne Muscular Dystrophy

A PHASE 2, MULTICENTER, SINGLE-ARM STUDY TO EVALUATE THE SAFETY AND DYSTROPHIN EXPRESSION AFTER FORDADISTROGENE MOVAPARVOVEC (PF-06939926) ADMINISTRATION IN MALE PARTICIPANTS WITH EARLY STAGE DUCHENNE MUSCULAR DYSTROPHY

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

Study Overview

• Status: Terminated
• Conditions: Muscular Dystrophy, Duchenne
• Intervention / Treatment: Genetic: PF-06939926

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The primary analysis will occur when all participants have completed visits through Week 52 (or withdrawn from the study prior to Week 52).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

• Study Type: Interventional
• Enrollment (Actual): 10
• Phase: Phase 2

Contacts and Locations

Study Locations

• Australia
  • New South Wales
    • Westmead, New South Wales, Australia, 2145
      • The Children's Hospital at Westmead
  • Victoria
    • Parkville, Victoria, Australia, 3052
      • The Royal Children's Hospital Melbourne
  • Western Australia
    • Nedlands, Western Australia, Australia, 6009
      • Perth Children's Hospital
• United States
  • Florida
    • Gainesville, Florida, United States, 32610
      • University of Florida
    • Gainesville, Florida, United States, 32610
      • UF Health Shands Hospital
  • Pennsylvania
    • Philadelphia, Pennsylvania, United States, 19104
      • The Children's Hospital of Philadelphia
    • Philadelphia, Pennsylvania, United States, 19146
      • The Children's Hospital of Philadelphia
  • Utah
    • Salt Lake City, Utah, United States, 84113
      • Primary Children's Hospital
    • Salt Lake City, Utah, United States, 84132
      • University of Utah Hospital
    • Salt Lake City, Utah, United States, 84108
      • University of Utah Imaging and Neurosciences Center
    • Salt Lake City, Utah, United States, 84112
      • University of Utah Hospital & Clinics Investigational Drug Services
    • Salt Lake City, Utah, United States, 84132
      • University of Utah Clinical Neurosciences Center
    • Salt Lake City, Utah, United States, 84108
      • CTSI Clinical Research Center

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 2 years to 3 years (Child)
• Accepts Healthy Volunteers: No

Description

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.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: PF-06939926	Genetic: PF-06939926; All participants will receive a single dose of PF-06939926 on Day 1.; Other Names: Fordadistrogene Movaparvovec

What is the study measuring?

Primary Outcome Measures

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

Secondary Outcome Measures

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

Collaborators and Investigators

• Sponsor: Pfizer
• Investigators: Study Director: Pfizer CT.gov Call Center, Pfizer

Publications and helpful links

Helpful Links

• To obtain contact information for a study center near you, click here.

Study record dates

Study Major Dates

• Study Start (Actual): August 8, 2022
• Primary Completion (Actual): October 3, 2025
• Study Completion (Actual): October 3, 2025

Study Registration Dates

• First Submitted: October 8, 2021
• First Submitted That Met QC Criteria: June 20, 2022
• First Posted (Actual): June 23, 2022

Study Record Updates

• Last Update Posted (Estimated): October 21, 2025
• Last Update Submitted That Met QC Criteria: October 18, 2025
• Last Verified: October 1, 2025

More Information

Terms related to this study

• Keywords: gene therapy; DMD; fordadistrogene movaparvovec; Early Stage Duchenne Muscular Dystrophy
• Additional Relevant MeSH Terms: Musculoskeletal Diseases; Nervous System Diseases; Muscular Diseases; Neuromuscular Diseases; Genetic Diseases, Inborn; Genetic Diseases, X-Linked; Muscular Disorders, Atrophic; Muscular Dystrophies; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Muscular Dystrophy, Duchenne
• Other Study ID Numbers: C3391008; 2021-003379-33 (EudraCT Number)

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: YES
• IPD Plan Description: Pfizer will provide access to individual de-identified participant data and related study documents (e.g. protocol, Statistical Analysis Plan (SAP), Clinical Study Report (CSR)) upon request from qualified researchers, and subject to certain criteria, conditions, and exceptions. Further details on Pfizer's data sharing criteria and process for requesting access can be found at: https://www.pfizer.com/science/clinical_trials/trial_data_and_results/data_requests.

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No