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(352) 733-0111

9001-303 (ENVISION)

  • Status
    Accepting Candidates
  • Age
    N/A - N/A
  • Sexes
    Male
  • Healthy Volunteers
    No
I'm interested
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Objective

The study will evaluate the safety and efficacy of delandistrogene moxeparvovec gene transfer therapy in non-ambulatory and ambulatory males with DMD. This is a randomized, double-blind, placebo-controlled 2-part study. Participants will be in the study for approximately 128 weeks. All participants will have the opportunity to receive intravenous (IV) delandistrogene moxeparvovec in either Part 1 or Part 2.

Details

Full study title A Phase 3, Multinational, Randomized, Double-Blind, Placebo-Controlled Systemic Gene Transfer Therapy Study to Evaluate the Safety and Efficacy of SRP-9001 in Non-Ambulatory and Ambulatory Subjects With Duchenne Muscular Dystrophy (ENVISION)
Protocol number OCR41934
ClinicalTrials.gov ID NCT05881408
Phase Phase 3

Eligibility

Inclusion Criteria:

  • Definitive diagnosis of DMD based on documented clinical findings and prior genetic testing.

  • Cohort 1 only: Non-ambulatory per protocol specified criteria.

  • Cohort 2 only: Ambulatory per protocol specified criteria and ≥8 to

Lead researcher

  • Carmen Leon Astudillo, MD
    Pediatric Neuromuscular Rehabilitation Specialist, Pediatric Pulmonologist (Child Lung Specialist)
    Carmen Leon Astudillo

Participate in a study

Here are some general steps to consider when participating in a research study:

  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

    Carmen Leon Astudillo
  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.