Study of Fordadistrogene Movaparvovec in Early Stage Duchenne Muscular Dystrophy

NCT05429372 | Terminated Phase 2 DMC FDA Drug

• 2 other identifiers: C33910082021-003379-33
• Study Type: interventional
• Target: 10
• Locations: 2 countries
• Sites: 13

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

Conditions

Muscular Dystrophy, Duchenne

Keywords

Early Stage Duchenne Muscular Dystrophy; DMD; gene therapy; fordadistrogene movaparvovec

Outcome Measures

Primary Outcomes (10)

• 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 Outcomes (12)

• Distribution of mini-dystrophin expression in muscle

  At Week 9, Week 52 and Year 5 (if available)

• Level of mini-dystrophin expression in muscle

  At Week 9, Week 52 and Year 5 (if available)

• Incidence and severity of Treatment-Emergent Adverse Events and Serious Adverse Events

  Through 5 years

• Number of participants with abnormal hematology test results

  Through 5 years

• Number of participants with abnormal biochemistry test results

  Through 5 years

Study Arms (1)

PF-06939926

EXPERIMENTAL

Genetic: PF-06939926

Interventions

PF-06939926 GENETIC

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

Also known as: Fordadistrogene Movaparvovec

PF-06939926

Eligibility Criteria

• Age: 2 Years - 3 Years
• Gender Eligibility Details: Male participants age ≥2 to \<4 years, at Screening (Visit 1)
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• Confirmed diagnosis of DMD by prior genetic testing.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Pfizer: lead

Study Sites (13)

UF Health Shands Hospital

Gainesville, Florida, 32610, United States

Location

University of Florida

Gainesville, Florida, 32610, United States

Location

The Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, 19104, United States

Location

The Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, 19146, United States

Location

CTSI Clinical Research Center

Salt Lake City, Utah, 84108, United States

Location

University of Utah Imaging and Neurosciences Center

Salt Lake City, Utah, 84108, United States

Location

University of Utah Hospital & Clinics Investigational Drug Services

Salt Lake City, Utah, 84112, United States

Location

Primary Children's Hospital

Salt Lake City, Utah, 84113, United States

Location

University of Utah Clinical Neurosciences Center

Salt Lake City, Utah, 84132, United States

Location

University of Utah Hospital

Salt Lake City, Utah, 84132, United States

Location

The Children's Hospital at Westmead

Westmead, New South Wales, 2145, Australia

Location

The Royal Children's Hospital Melbourne

Parkville, Victoria, 3052, Australia

Location

Perth Children's Hospital

Nedlands, Western Australia, 6009, Australia

Location

Related Links

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

MeSH Terms

Conditions

Muscular Dystrophy, Duchenne

Condition Hierarchy (Ancestors)

Muscular Dystrophies; Muscular Disorders, Atrophic; Muscular Diseases; Musculoskeletal Diseases; Neuromuscular Diseases; Nervous System Diseases; Genetic Diseases, X-Linked; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Officials

• Pfizer CT.gov Call Center

  Pfizer

  STUDY DIRECTOR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: October 8, 2021
• First Posted: June 23, 2022
• Study Start: August 8, 2022
• Primary Completion: October 3, 2025
• Study Completion: October 3, 2025
• Last Updated: October 21, 2025

Record last verified: 2025-10

Data Sharing

• IPD Sharing: Will share

Locations

US (10); AU (3)