ELEVIDYS safety in Duchenne patients treated after market approval

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

What this trial studies

This phase 4, interventional trial evaluates acute liver injury rates when ELEVIDYS is used with sirolimus as a prophylactic immunosuppression in Duchenne patients. Cohort 1 includes eligible, ambulatory males aged 4 and older who are candidates for commercial ELEVIDYS, while Cohort 2 includes males who have previously received ELEVIDYS after sirolimus and corticosteroids. The study also considers glucocorticoids and antibiotics as part of the regimen and requires appropriate birth control and consent processes. The lead sponsor is Sarepta Therapeutics, with the Baylor College of Medicine as a participating site.

Why it matters

Who can join: the full rules

Key Inclusion Criteria:

1. Cohort 1 only: Is male at birth, ambulatory, and ≥ 4 years of age at the time of dosing.
2. Cohort 1 only: Is eligible for commercial ELEVIDYS.
3. Cohort 2 only: Is male at birth and has previously received ELEVIDYS in a commercial setting after pre-treatment with sirolimus and corticosteroids.
4. Cohort 1 only: Participants who are sexually active must agree to use, for the entire duration of the study, a condom and the female sexual partner must also use a medically acceptable form of birth control (eg, oral contraceptive).
5. Has (a) parent(s) or legal guardian(s) who is (are) able to understand and comply with the study visit schedule and all other protocol requirements, or is ≥ 18 years of age and personally able to understand and comply with the protocol requirements.
6. Either has a parent or legal guardian who is willing to provide informed consent, or is ≥ 18 years of age and able to provide informed consent independently.

Key Exclusion Criteria:

1. Cohort 1 only: Contraindicated to receive ELEVIDYS per the United States Package Insert (USPI).
2. Cohort 1 only: Has serological evidence of current, chronic, or active human immunodeficiency virus, hepatitis C, or hepatitis B infection.
3. Has a medical condition or confounding circumstances (eg, prior traumatic limitation for mobility or significant behavioral comorbidity) that, in the opinion of the Investigator, might compromise:

   1. The participant's ability to comply with the protocol-required procedures, and/or
   2. The participant's well-being or safety, and/or
   3. The clinical interpretability of the data collected from the participant
4. Cohort 1 only: Has a symptomatic infection (eg, upper respiratory tract infection, pneumonia, pyelonephritis, meningitis) within 4 weeks prior to Day 1.
5. Cohort 1 only: Has received a live virus vaccine within 4 weeks or inactive vaccine within 2 weeks of the Day 1 visit or expects to receive a vaccination during the first 3 months after Day 1.
6. Cohort 1 only: Any confounding factors that would prevent the use of oral sirolimus including a known hypersensitivity to sirolimus or any of its excipients.
7. Cohort 1 only: Any wounds or recent injuries that, in the opinion of the Investigator, would be at risk of dehiscence or impaired healing in the setting of sirolimus administration.

Other inclusion/exclusion criteria may apply, per protocol.

Where it's running

• Baylor College of Medicine
  Houston, Texas, United States Recruiting

Study contacts

• Study coordinator: Sarepta Therapeutics Inc. For Clinical Trial Information, Select Option 4,
• Email: SareptAlly@sarepta.com
• Phone: 1-888-SAREPTA (1-888-727-3782)

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How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.