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Not yet recruiting NCT07542314

Study to Evaluate the Safety and Effectiveness of ELEVIDYS in Participants With Duchenne Muscular Dystrophy Treated in a Post-Marketing Setting

Phase IV Interventional Duchenne Muscular Dystrophy

For patients and families

In plain language

An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.

What is being studied
The protocol lists: ELEVIDYS, Sirolimus, Glucocorticoids, Antibiotics.
Who it may be relevant to
Registry conditions: Duchenne Muscular Dystrophy. Basic parameters: from 4 years · Male.
What needs checking
Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
Where it takes place
Center list to be confirmed — check the primary protocol.
Next step
Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →

Unsure about the terms? Read our patient guide →

Official title

Phase 4 Study to Evaluate the Safety and Effectiveness of ELEVIDYS in Patients With Duchenne Muscular Dystrophy Treated in a Post-Marketing Setting (ENHANCE)

Overview

The primary objective of this study is to evaluate acute liver injury (ALI) rates associated with ELEVIDYS with the addition of sirolimus as an adjunct prophylactic immunosuppression agent.

Interventions

  • Drug ELEVIDYS
    Administered via an intravenous infusion.
  • Drug Sirolimus
    Administered orally.
  • Drug Glucocorticoids
    Administered orally.
  • Drug Antibiotics
    Administered orally.

Primary outcome measures

  • Cohort 1: Number of Participants with ALI [Time frame: 12 weeks]
Secondary outcome measures (9)
  • Cohort 1: Number of Participants with Treatment-emergent Adverse Events (TEAEs), Adverse Events of Special Interest (AESIs) and Serious Adverse Events (SAEs) [Time frame: Day 1 up to Week 24]
  • Cohort 1: Number of Participants with Infections, Edema, Wound-healing Complications, Hyperlipidemia, Angioedema, and Interstitial Lung Disease/Non-infectious Pneumonitis [Time frame: Day 1 up to Week 24]
  • Cohort 1: Number of Participants with Hepatic Adverse Events, Hepatic Biomarkers, and Laboratory Assessments Indicative of Either Acute Hepatocellular Injury or Acute Liver Dysfunction [Time frame: Day 1 up to Week 24]
  • Cohort 1: Number of Participants with Severe ALI [Time frame: Day 1 up to Week 24]
  • Cohort 1: Number of Participants with ALI [Time frame: Day 1 up to Week 24]
  • Cohort 1: Duration of ALI [Time frame: Day 1 up to Week 24]
  • Cohort 1: Amount of Steroid Use [Time frame: Day 1 up to Week 24]
  • Cohort 1: Duration of Steroid Use [Time frame: Day 1 up to Week 24]
  • Cohort 1: Quantity of ELEVIDYS Dystrophin Protein Expression as Measured by Western Blot [Time frame: Week 12]

Eligibility criteria

Inclusion criteria

  • Cohort 1 only: Is male at birth, ambulatory, and ≥ 4 years of age at the time of dosing.
  • Cohort 1 only: Is eligible for commercial ELEVIDYS.
  • Cohort 2 only: Is male at birth and has previously received ELEVIDYS in a commercial setting after pre-treatment with sirolimus and corticosteroids.
  • 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).
  • 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.
  • 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.

Exclusion criteria

  • Cohort 1 only: Contraindicated to receive ELEVIDYS per the United States Package Insert (USPI).
  • Cohort 1 only: Has serological evidence of current, chronic, or active human immunodeficiency virus, hepatitis C, or hepatitis B infection.
  • 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:
  • The participant's ability to comply with the protocol-required procedures, and/or
  • The participant's well-being or safety, and/or
  • The clinical interpretability of the data collected from the participant
  • Cohort 1 only: Has a symptomatic infection (eg, upper respiratory tract infection, pneumonia, pyelonephritis, meningitis) within 4 weeks prior to Day 1.
  • 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.
  • 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.
  • 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.

Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.

Healthy volunteers: No

Study design

Allocation
Non-randomized
Model
Single group
Masking
Open label
Primary purpose
Treatment

Study locations

Center list to be confirmed — check the primary protocol.

Identifiers

NCT: NCT07542314 · SRP-9001-402

Primary sources (government registries)

View this study on ClinicalTrials.gov ↗