Norfolk, VANCT04626674Now EnrollingIRB Ready

Duchenne Muscular Dystrophy Clinical Trial in Norfolk, VA

Access cutting-edge duchenne muscular dystrophy treatment through this clinical trial at a research site in Norfolk. Study-provided care at no cost to qualified participants.

Sponsored by Sarepta Therapeutics, Inc.

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Expert Care in Norfolk

Access duchenne muscular dystrophy specialists at no cost

IRB Approved

This study follows strict safety protocols and ethical guidelines

No-Cost Care

All study-related duchenne muscular dystrophy treatment provided free

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Why Participate?

  • No-Cost Study Care

  • Local to Norfolk

    Convenient for VA residents

  • Cutting-Edge Treatment

    Access to innovative therapies

  • Expert Medical Care

    Close monitoring by specialists

  • Possible Compensation*

    For time and travel

*Compensation varies by study. Confirm details with coordinator.

Simple Process

  1. 1Submit this form
  2. 2Phone screening
  3. 3Visit Norfolk site if eligible
  4. 4Begin participation

About This Duchenne Muscular Dystrophy Study in Norfolk

Cohort 8 (non-ambulatory participants) is currently enrolling new participants. Enrollment for Cohorts 1 through 7 has been completed. This is an open-label gene transfer therapy study evaluating the safety of and expression from delandistrogene moxeparvovec in participants with Duchenne Muscular Dystrophy (DMD). The maximum participant duration for this study is 156 weeks.

Sponsor: Sarepta Therapeutics, Inc.

Who Can Participate

Inclusion Criteria

For Cohorts 1-8: Has a definitive diagnosis of DMD based on documented clinical findings and prior genetic testing.
Cohort 8: Non-ambulatory per protocol-specified criteria at the time of Screening, has a performance upper limb (PUL) entry item score ≥3 at the Screening visit and has a total PUL score of ≥20 and ≤40 at the time of Screening.
Cohorts 1, 2, 3, 5, 7 and 8 only: Stable dose equivalent of oral glucocorticoids for at least 12 weeks before screening and the dose is expected to remain constant (except for modifications to accommodate changes in weight) throughout the first year of the study.
Cohort 1: Is ambulatory, and ≥4 to \<8 years of age at the time of Screening.
Cohort 2: Is ambulatory, and ≥8 to \<18 years of age at the time of Screening.
Cohort 3: Non-ambulatory per protocol specified criteria at the time of Screening.
Cohort 4: Is ambulatory and ≥3 to \<4 years of age at the time of Screening.
Cohort 5a: Is ambulatory and ≥4 to \<9 years of age with time to rise from the floor ≤7 seconds at the screening visit.
Cohort 5b: Non-ambulatory per protocol specified criteria at the time of Screening.
Cohort 6: Is ambulatory, and ≥2 to \<3 years of age at the time of Screening.
Cohort 7: Non-ambulatory per protocol-specified criteria at the time of Screening.
Cohorts 4 and 6: Do not yet require use of chronic steroids for treatment of their DMD, in the opinion of the Investigator, and are not receiving steroids at the time of Screening.
Genetic mutation inclusion criteria vary by cohort. All Cohorts:
Ability to cooperate with motor assessment testing.
rAAVrh74 antibody titers are not elevated as per protocol-specified requirements.

Exclusion Criteria

Cohort 8: Any confounding factors that would prevent the use of oral sirolimus including a known hypersensitivity to sirolimus or any of its excipients.
Has a concomitant illness, autoimmune disease, chronic drug treatment, and/or cognitive delay/impairment that in the opinion of the Investigator creates unnecessary risks for gene transfer.
Exposure to gene therapy, investigational medication, or any treatment designed to increase dystrophin expression within protocol-specified time limits.
Abnormality in protocol-specified diagnostic evaluations or laboratory tests. Note: Other inclusion/exclusion criteria apply.

Not sure if you qualify? Submit your interest and a study coordinator will help determine your eligibility.

Frequently Asked Questions

Q:Is this study available in Norfolk?

Yes, this clinical trial (NCT04626674) has an active research site in Norfolk, VA that is currently enrolling participants.

Q:Is it safe to participate?

Clinical trials follow strict safety guidelines and ethical standards. This study has been reviewed and approved, and participants are closely monitored by medical professionals. You can withdraw at any time.

Q:Will I be compensated?

Many clinical trials offer compensation for your time and travel expenses. Specific compensation details will be discussed during the screening process. All study-related medical care is provided at no cost.

Q:Can I leave the trial if I change my mind?

Absolutely. Participation is completely voluntary. You have the right to withdraw from the study at any time, for any reason, without penalty.

Still have questions? Our study coordinators are here to help.

Duchenne Muscular Dystrophy Treatment Options in Norfolk, VA

If you're searching for duchenne muscular dystrophy treatment options in Norfolk, VA, this clinical trial (NCT04626674) may be an excellent opportunity. Clinical trials provide access to cutting-edge treatments that aren't yet available to the general public, often at no cost to participants.

Our Norfolk research site is actively enrolling participants for this clinical trial. You'll receive care from experienced duchenne muscular dystrophy specialists who are at the forefront of medical research. All study-related care, including examinations, treatments, and monitoring, is provided at no cost to qualified participants.

Looking for more options? Browse all duchenne muscular dystrophy clinical trials near you to find additional studies recruiting in your area.

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