NCT03368742进行中（未招募）1 期
A Randomized, Controlled, Open-label, Single-ascending Dose, Phase I/II Study to Investigate the Safety and Tolerability, and Efficacy of Intravenous SGT-001 in Male Adolescents and Children With Duchenne Muscular Dystrophy
适应症
干预措施
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 入组人数
- 12
- 试验地点
- 2
- 主要终点
- Number of Participants with Treatment Emergent Adverse Events (TEAEs)
研究概览
简要总结
This is a controlled, open-label, single-ascending dose study to evaluate the safety and tolerability of SGT-001 in adolescents and children with Duchenne muscular dystrophy (DMD). Participants will receive a single intravenous (IV) infusion of SGT-001 and will be followed for approximately 5 years.
The protocol was amended to drop the control arm after 4 participants were dosed.
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 17 Years（Child）
- 性别
- Male
- 接受健康志愿者
- 否
入选标准
- •Established clinical diagnosis of DMD and documented dystrophin gene mutation predictive of DMD phenotype
- •Confirmed absence of dystrophin as determined by muscle biopsy (ambulatory participants)
- •Anti-AAV9 antibodies below protocol-specified thresholds
- •Stable cardiac and pulmonary function
- •Adolescents: non-ambulatory by protocol-specified criteria
- •Children: ambulatory by protocol-specified criteria
- •Stable daily dose (or equivalent) of oral corticosteroids ≥ 12 weeks
排除标准
- •Prior or ongoing medical condition or physical examination, ECG or laboratory findings that could adversely affect participant safety, compromise completion of treatment and follow-up, or impair assessment of study results
- •Abnormal liver function
- •Abnormal renal function
- •Clinically significant coagulation abnormalities
- •Impaired cardiovascular function based on cardiac MRI or ECHO
- •Impaired respiratory function based on FVC % predicted or need for daytime ventilatory support
- •Significant spinal deformity or presence of spinal rods
- •Body mass index ≥ 95th percentile for age
- •Exposure to another investigational drug within 3 months or 5 half-lives prior to screening
- •Exposure to drugs affecting dystrophin or utrophin expression within 6 months prior to screening
- •Additional inclusion/exclusion criteria may apply.
研究组 & 干预措施
Untreated Control
No Intervention
Untreated control group. After 1 year, treatment-eligible control participants will receive SGT-001 at the selected dose.
SGT-001 - Dose Level 1
Experimental
Single IV infusion of SGT-001 at starting dose
干预措施: SGT-001 (Genetic)
SGT-001 - Dose Level 2
Experimental
Single IV infusion of SGT-001 at next ascending dose
干预措施: SGT-001 (Genetic)
结局指标
主要结局
Number of Participants with Treatment Emergent Adverse Events (TEAEs)
时间窗: Up to 5 years
次要结局
- Number of Participants with Clinically Significant Abnormalities in Laboratory Parameters(Up to 5 years)
- Number of Participants with Clinically Significant Abnormalities in Vital Signs(Up to 5 years)
- Number of Participants with Clinically Significant Abnormalities in Physical Examinations(Up to 5 years)
- Number of Participants with Clinically Significant Abnormalities in Electrocardiogram (ECG)(Up to 5 years)
- Change from Baseline in Microdystrophin Protein Levels in Muscle Biopsies Using Western Blot (WB)(Baseline, 12 months)
- Change from Baseline in Microdystrophin Protein Levels in Muscle Biopsies Using Immunofluorescence (IF)(Baseline, 12 months)
- Change from Baseline in North Star Ambulatory Assessment (NSAA) score in Ambulatory Participants(Baseline, 12 months)
- Change from Baseline in 6-minute walk test (6MWT) Distance in Ambulatory Participants(Baseline, 12 months)
- Change from Baseline in Total Upper Limb Function, as Measured by the Total Performance of the Upper Limb (PUL) Functional Scale Score(Baseline, 12 months)
- Change from Baseline in Respiratory Function, as Measured by Forced Vital Capacity (FVC) % Predicted, Forced Expiratory Volume in 1 second (FEV1) % Predicted, and Peak Expiratory Flow (PEF) % Predicted(Baseline, 12 months)
- Change from Baseline in Ejection Fraction, As Measured by Echocardiography(Baseline,12 months)
- Change from Baseline in Left Ventricular End Systolic Volume, As Measured by Echocardiography(Baseline,12 months)
- Change from Baseline in Myocardial Peak Circumferential Strain (Ecc), As Measured by Echocardiography(Baseline,12 months)
- Change from Baseline in Quality of Life as Measured by the Paediatric Quality of Life Inventory (PedsQL) Duchenne muscular dystrophy (DMD) module and self-reported outcome measures as measured by the PODCI DMD module(Baseline, 12 months)
研究者
研究点 (2)
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