A Phase 1, Randomized, Placebo-Controlled, Double-blind, Single and Multiple Ascending Dose Study to Evaluate the Safety, Tolerability, and PK of EDG-5506 in Adult Healthy Volunteers and Adults With Becker Muscular Dystrophy
试验速览
- 阶段
- 1 期
- 状态
- 已完成
- 入组人数
- 127
- 试验地点
- 1
- 主要终点
- Incidence, frequency, severity and dose-relationship of adverse events
研究概览
简要总结
EDG-5506 is an investigational product intended to protect and improve function of dystrophic muscle fibers. This Phase 1 study of EDG-5506 will assess the safety, tolerability, and pharmacokinetics (PK) and of EDG-5506 in adult healthy volunteers and in adults with Becker muscular dystrophy (BMD).
详细描述
Enrolled participants in this study will receive a single oral dose or multiple oral doses of EDG-5506 or a placebo. Blood and urine samples will be collected to measure how EDG-5506 is processed by the body and how the body responds when exposed to EDG-5506. Participants in the single ascending dose part of the study will remain in the clinic for 7 days with a 42-day follow-up period. Participants in the multiple ascending dose part of the study will remain in the clinic for 16 days with a 13-day follow-up period. Safety, tolerability, and pharmacokinetics of EDG-5506 will be assessed in healthy volunteers prior to enrolling participants with Becker muscular dystrophy.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Sequential
- 主要目的
- Treatment
- 盲法
- Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
入排标准
- 年龄范围
- 18 Years 至 55 Years（Adult）
- 性别
- All
- 接受健康志愿者
- 是
入选标准
- •For all potential participants (Healthy volunteers and BMD): Male or for HV: female. For all: adults aged 18 to 55 years at time of consent.
- •For HVs: Good general health, with no significant medical history, no clinically significant abnormalities on physical exam
- •For BMD: Diagnosis of BMD based on documentation of mutation(s) in the dystrophin gene and BMD phenotype
- •For BMD: Ability to ambulate
- •For all: Weight greater than or equal to 50 kg and BMI less than 33 kg/m2
- •For HV: Females must be of non-childbearing potential.
- •For all: Males with female partners must use a medically accepted contraceptive regimen from first dose through 90 days after the last dose
- •For all: Non-smoker and must not have used any tobacco products within 3 months prior to the Screening visit.
- •For all: Able and willing to attend the necessary visits at the study center.
排除标准
- •For all: History of, or physical exam findings indicating clinically significant endocrine, neurological, gastrointestinal, cardiovascular, hematological, hepatic, immunological, renal, respiratory, or genitourinary abnormalities or diseases that, in the opinion of the Investigator, would render the subject being unsuitable for the study.
- •For all: Unable to refrain from strenuous exercise for 3 days prior to check-in and during study.
- •For all: Participation in any other investigational drug study within 30 days or 5 half-lives (whichever is longer) of dosing in the present study.
研究组 & 干预措施
Healthy Volunteer: Single Ascending Dose
Single oral ascending dose in healthy volunteers
Interventions:
Drug: EDG-5506 Drug: Placebo
干预措施: EDG-5506 (Drug)
Healthy Volunteer: Single Ascending Dose
Single oral ascending dose in healthy volunteers
Interventions:
Drug: EDG-5506 Drug: Placebo
干预措施: Placebo (Drug)
Healthy Volunteer: Multiple Ascending Dose
Multiple oral ascending doses in healthy volunteers
Interventions:
Drug: EDG-5506 Drug: Placebo
干预措施: EDG-5506 (Drug)
Healthy Volunteer: Multiple Ascending Dose
Multiple oral ascending doses in healthy volunteers
Interventions:
Drug: EDG-5506 Drug: Placebo
干预措施: Placebo (Drug)
Becker Muscular Dystrophy: Multiple Ascending Dose
Multiple oral ascending doses in adults with Becker muscular dystrophy
Interventions:
Drug: EDG-5506 Drug: Placebo
干预措施: EDG-5506 (Drug)
Becker Muscular Dystrophy: Multiple Ascending Dose
Multiple oral ascending doses in adults with Becker muscular dystrophy
Interventions:
Drug: EDG-5506 Drug: Placebo
干预措施: Placebo (Drug)
结局指标
主要结局
Incidence, frequency, severity and dose-relationship of adverse events
时间窗: Up to 42 days of monitoring
Incidence of abnormal laboratory test results (clinical chemistry, hematology, urinalysis, coagulation)
时间窗: Up to 42 days of monitoring
Incidence of treatment-emergent clinically abnormal electrocardiogram (ECG)
时间窗: Up to 42 days of monitoring
Incidence of abnormal vital signs
时间窗: Up to 42 days of monitoring
Incidence of abnormal physical exam findings
时间窗: Up to 42 days of monitoring
次要结局
- Time of maximum concentration (Tmax)(Up to 42 days of testing)
- Plasma maximum measured drug concentration (Cmax)(Up to 42 days of testing)
- Area under the concentration-time curve (AUC)(Up to 42 days of testing)
- Plasma half-life (T½)(Up to 42 days of testing)
- Renal clearance (CLR)(Up to 42 days of testing)
- Drug excreted unchanged in urine (Amt0-24)(Up to 42 days of testing)
- Fraction excreted in urine (Fe)(Up to 42 days of testing)