Study of Fordadistrogene Movaparvovec in Early Stage Duchenne Muscular Dystrophy

Brief Title

Study of Fordadistrogene Movaparvovec in Early Stage Duchenne Muscular Dystrophy

Official Title

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

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      (or withdrawn from the study prior to Week 52).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.

Study Phase

Phase 2

Study Type

Interventional

Primary Outcome

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

Secondary Outcome

 Distribution of mini-dystrophin expression in muscle

Condition

Muscular Dystrophy, Duchenne

Intervention

PF-06939926

Study Arms / Comparison Groups

 PF-06939926

Description:

Publications

* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.

Recruitment Information

Recruitment Status

Genetic

Estimated Enrollment

Start Date

August 5, 2022

Completion Date

June 22, 2028

Primary Completion Date

July 14, 2024

Eligibility Criteria

        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.

          -  Positive test performed by Pfizer for neutralizing antibodies to AAV9.

          -  Motor and cognitive function not adequate to participate in the study, as assessed by
             protocol-specified criteria.

          -  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.

Gender

Male

Ages

2 Years - 3 Years

Accepts Healthy Volunteers

No

Contacts

Pfizer CT.gov Call Center, 1-800-718-1021, [email protected]

Location Countries

Australia

Location Countries

Australia

Administrative Informations

NCT ID

NCT05429372

Organization ID

C3391008

Secondary IDs

2021-003379-33

Responsible Party

Sponsor

Study Sponsor

Pfizer

Study Sponsor

Pfizer CT.gov Call Center, Study Director, Pfizer

Verification Date

August 2022