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(352) 733-0111

Pfizer Early Stage GT

  • Status
    Accepting Candidates
  • Age
    2 Years - 3 Years
  • Sexes
    Male
  • Healthy Volunteers
    No
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Objective

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

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The primary analysis will occur when all participants have completed visits through Week 52 (or withdrawn from the study prior to Week 52).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

Details

Full study 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
Protocol number OCR42752
ClinicalTrials.gov ID NCT05429372
Phase Phase 2

Eligibility

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.

Lead researcher

Participate in a study

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  1. Step
    1

    Contact the research team

    Call or email the research team listed within the specific clinical trial or study to let them know that you're interested. A member of the research team, such as the researcher or study coordinator, will be available to tell you more about the study and to answer any questions or concerns you may have.

    Primary contact

    Barry Byrne
  2. Step
    2

    Get screened to confirm eligibility

    You may be asked to take part in prescreening to make sure you are eligible for a study. The prescreening process ensures it is safe for you to participate. During the prescreening process, you will be asked some questions and you may also be asked to schedule tests or procedures to confirm your eligibility.

  3. Step
    3

    Provide your consent to participate

    If you are eligible and want to join the clinical trial or study, a member of the research team will ask for your consent to participate. To give consent, you will be asked to read and sign a consent form for the study. This consent form explains the study's purpose, procedures, risks, benefits and provides other important information, such as the study team's contact information.

  4. Step
    4

    Participate

    If you decide to participate in a clinical trial or study, the research team will keep you informed of the study requirements and what you will need to do to throughout the study. For some trials or studies, your health care provider may work with the research team to ensure there are no conflicts with other medications or treatments.