NCT02515669终止1 期
A Multi-Site, Randomized, Placebo-Controlled, Double-Blind, Multiple Ascending Subcutaneous Dose Study to Evaluate the Safety, Tolerability, and Pharmacokinetics of RO7239361 (BMS-986089) in Ambulatory Boys With Duchenne Muscular Dystrophy
适应症
干预措施
相关药物
试验速览
- 阶段
- 1 期
- 状态
- 终止
- 入组人数
- 43
- 试验地点
- 12
- 主要终点
- Safety Summary for the 24 Week Double-Blind Phase
研究概览
简要总结
The purpose of this study is to determine the safety and tolerability of RO7239361 in boys with Duchenne Muscular Dystrophy with any genetic mutation.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 主要目的
- Basic Science
- 盲法
- Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
入排标准
- 年龄范围
- 5 Years 至 10 Years（Child）
- 性别
- Male
- 接受健康志愿者
- 否
入选标准
- •Diagnosed with DMD
- •Able to walk without assistance
- •Able to walk up 4 stairs in 8 seconds or less
- •Weigh at least 15 kg
- •Taking corticosteroids for DMD
排除标准
- •Ejection fraction < 55% on echocardiogram, based on central read
- •Any behavior or mental issue that will affect the ability to complete the required study procedures
- •Previously or currently taking medications like androgens or human growth hormone
- •Use of a ventilator during the day
- •Unable to have blood samples collected or receive an injection under the skin
- •Treatment with exon skipping therapies 6 months prior to study start
- •Treatment with ataluren or any investigational drug currently or within 5 half-lives prior to study start
研究组 & 干预措施
RO7239361
Active Comparator
RO7239361 subcutaneous injections on specified days
干预措施: RO7239361 (Drug)
Placebo
Placebo Comparator
Placebo subcutaneous injections on specified days
干预措施: Placebo (Drug)
结局指标
主要结局
Safety Summary for the 24 Week Double-Blind Phase
时间窗: Baseline to Week 24
Percentage of participants with fatalities, adverse event (AEs) and serious adverse events (SAEs) up to Week 24. Participants in Panel 1 received 4 mg RO7239361; participants in Panel 2 received either 12.5 mg (weight between 15 and 45 kg) or 20 mg (weight \>45 kg) RO7239361, and participants in Panel 3 and the Expansion Panel received either 35 mg (weight between 15 and 45 kg) or 50 mg (weight \>45 kg) RO7239361.
Safety Summary up to Week 72
时间窗: Baseline to Week 72
Percentage of participants with fatalities, adverse event (AEs) and serious adverse events (SAEs) up to Week 72. Participants in Panel 1 received 4 mg RO7239361; participants in Panel 2 received either 12.5 mg (weight between 15 and 45 kg) or 20 mg (weight \>45 kg) RO7239361, and participants in Panel 3 and the Expansion Panel received either 35 mg (weight between 15 and 45 kg) or 50 mg (weight \>45 kg) RO7239361.
次要结局
- Time of Maximum Observed Serum Concentrations (Tmax) of RO7239361 at Steady State for 50 mg QW Dose.(Day 1: predose, 3, 6, 72 and 96 h postdose; Days 8, 15 and 22: predose; Day 29: predose and 96 h postdose; Weeks 12 and 24: predose)
- Area Under the Concentration-Time Curve From Time Zero to Time of Next Dosing (AUCtau) of RO7239361 at Steady State for 50 mg QW Dose.(Day 1: predose, 3, 6, 72 and 96 h postdose; Days 8, 15 and 22: predose; Day 29: predose and 96 h postdose; Weeks 12 and 24: predose)
- RO7239361 Trough Concentrations(Day 1: predose, 3, 6, 72 and 96 h postdose; Days 8, 15 and 22: predose; Day 29: predose and 96 h postdose; Weeks 12 and 24: predose)
- Maximum Observed Serum Concentrations (Cmax) of RO7239361 at Steady State for 4 mg QW, 12.5 mg QW and 35 mg QW Doses.(Day 1: predose, 3, 6, 72 and 96 hours (h) postdose; Days 8, 15 and 22: predose; Day 29: predose and 96 h postdose; Weeks 12 and 24: predose)
- Maximum Observed Serum Concentrations (Cmax) of RO7239361 at Steady State for 50 mg QW Dose.(Day 1: predose, 3, 6, 72 and 96 h postdose; Days 8, 15 and 22: predose; Day 29: predose and 96 h postdose; Weeks 12 and 24: predose)
- Time of Maximum Observed Serum Concentrations (Tmax) of RO7239361 at Steady State for 4 mg QW, 12.5 mg QW and 35 mg QW Doses.(Day 1: predose, 3, 6, 72 and 96 h postdose; Days 8, 15 and 22: predose; Day 29: predose and 96 h postdose; Weeks 12 and 24: predose)
- Area Under the Concentration-Time Curve From Time Zero to Time of Next Dosing (AUCtau) of RO7239361 at Steady State for 4 mg QW, 12.5 mg QW and 35 mg QW Doses.(Day 1: predose, 3, 6, 72 and 96 h postdose; Days 8, 15 and 22: predose; Day 29: predose and 96 h postdose; Weeks 12 and 24: predose)
- Percentage of Participants With Positive Anti-RO7239361 Antibodies (ADA) Assessment up to Week 72(Day 8 through Week 72, baseline and on-study information represented in table.)
- Percentage of Participants With Positive Anti-RO7239361 Antibodies (ADA) Assessment, Double-Blind Phase(Day 8 through Week 24, baseline and on-study information represented in table.)
- Serum Concentration of Free Myostatin in the Double-Blind Phase(Baseline through Week 24)
- Serum Concentration of Drug-Myostatin Complex in the Double-Blind Phase(Baseline through Week 24)
- Fold Change From Baseline in Contractile Versus Non-contractile Content for Muscles in the Right Thigh in the Double-Blind Phase(Baseline through Week 24)
- Percent Inhibition of Free Myostatin in the Double-Blind Phase(Baseline through Week 24)
- Change From Baseline in Thigh Muscle Maximal Cross Sectional Area (CSAmax) in the Double-Blind Phase(Baseline through Week 24)
- Percentage of Participants With Positive Anti-RO7239361 Antibodies (ADA) Assessment, Whole Study(Day 8 through Week 228, baseline and on-study information represented in table.)
- Serum Concentration of Free Myostatin, Whole Study(Baseline through Week 252)
- Percent Inhibition of Free Myostatin, Whole Study(Baseline through Week 252)
- Serum Concentration of Drug-Myostatin Complex, Whole Study(Baseline through Week 252)
研究者
研究点 (12)
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