Phase 4 Study to Evaluate the Safety and Effectiveness of ELEVIDYS in Patients With Duchenne Muscular Dystrophy Treated in a Post-Marketing Setting (ENHANCE)
试验速览
- 阶段
- 4 期
- 状态
- 尚未招募
- 入组人数
- 20
- 试验地点
- 1
- 主要终点
- Cohort 1: Number of Participants with ALI
研究概览
简要总结
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.
研究设计
- 研究类型
- Interventional
- 分配方式
- Non Randomized
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 4 Years 至 —（Child, Adult, Older Adult）
- 性别
- Male
- 接受健康志愿者
- 否
入选标准
- •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.
排除标准
- •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
- •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
- •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.
研究组 & 干预措施
Cohort 1: ELEVIDYS
Participants will receive ELEVIDYS in a commercial setting on Day 1. Participants will also receive sirolimus, glucocorticoids and antibiotics orally.
干预措施: ELEVIDYS (Drug)
Cohort 1: ELEVIDYS
Participants will receive ELEVIDYS in a commercial setting on Day 1. Participants will also receive sirolimus, glucocorticoids and antibiotics orally.
干预措施: Sirolimus (Drug)
Cohort 1: ELEVIDYS
Participants will receive ELEVIDYS in a commercial setting on Day 1. Participants will also receive sirolimus, glucocorticoids and antibiotics orally.
干预措施: Antibiotics (Drug)
Cohort 2: ELEVIDYS
Participants who have previously received ELEVIDYS in a commercial setting after prophylactic treatment with sirolimus and corticosteroids will participate in one study visit during which a muscle biopsy will be performed. No study treatment will be administered in this cohort.
Cohort 1: ELEVIDYS
Participants will receive ELEVIDYS in a commercial setting on Day 1. Participants will also receive sirolimus, glucocorticoids and antibiotics orally.
干预措施: Glucocorticoids (Drug)
结局指标
主要结局
Cohort 1: Number of Participants with ALI
时间窗: 12 weeks
次要结局
- Cohort 1: Number of Participants with Treatment-emergent Adverse Events (TEAEs), Adverse Events of Special Interest (AESIs) and Serious Adverse Events (SAEs)(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(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(Day 1 up to Week 24)
- Cohort 1: Number of Participants with Severe ALI(Day 1 up to Week 24)
- Cohort 1: Number of Participants with ALI(Day 1 up to Week 24)
- Cohort 1: Duration of ALI(Day 1 up to Week 24)
- Cohort 1: Amount of Steroid Use(Day 1 up to Week 24)
- Cohort 1: Duration of Steroid Use(Day 1 up to Week 24)
- Cohort 1: Quantity of ELEVIDYS Dystrophin Protein Expression as Measured by Western Blot(Week 12)