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

Официальное название

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

Обзор

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.

Вмешательства

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

Первичные конечные точки

• Cohort 1: Number of Participants with ALI [Срок оценки: 12 weeks]

Вторичные конечные точки (9)

• Cohort 1: Number of Participants with Treatment-emergent Adverse Events (TEAEs), Adverse Events of Special Interest (AESIs) and Serious Adverse Events (SAEs) [Срок оценки: 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 [Срок оценки: 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 [Срок оценки: Day 1 up to Week 24]
• Cohort 1: Number of Participants with Severe ALI [Срок оценки: Day 1 up to Week 24]
• Cohort 1: Number of Participants with ALI [Срок оценки: Day 1 up to Week 24]
• Cohort 1: Duration of ALI [Срок оценки: Day 1 up to Week 24]
• Cohort 1: Amount of Steroid Use [Срок оценки: Day 1 up to Week 24]
• Cohort 1: Duration of Steroid Use [Срок оценки: Day 1 up to Week 24]
• Cohort 1: Quantity of ELEVIDYS Dystrophin Protein Expression as Measured by Western Blot [Срок оценки: Week 12]

Критерии участия

Критерии включения

• 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.

Критерии исключения

• 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.

Критерии приведены из реестра в оригинале (на английском). Окончательную оценку соответствия проводит исследовательский центр.

Здоровые добровольцы: Нет

Дизайн исследования

Распределение

Нерандомизированное

Модель

Одна группа

Маскирование

Открытое

Основная цель

Лечение

Центры проведения

Список центров уточняется — проверьте первичный протокол.

Идентификаторы

NCT: NCT07542314 · SRP-9001-402

Первоисточники (государственные реестры)

• ClinicalTrials.gov (NIH, США)

Открыть это исследование на ClinicalTrials.gov ↗