A Randomized, Double Blind, Placebo-Controlled, Study to Assess the Efficacy, Safety, and Tolerability of RO7239361 in Ambulatory Boys With Duchenne Muscular Dystrophy
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 入组人数
- 166
- 试验地点
- 44
- 主要终点
- Change From Baseline in the North Star Ambulatory Assessment (NSAA) Total Score at Week 48
研究概览
简要总结
This is a multi-center, randomized, double-blind, placebo-controlled study to assess the efficacy, safety and tolerability of two different weekly doses of RO7239361 in ambulatory boys with Duchenne Muscular Dystrophy (DMD).
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
入排标准
- 年龄范围
- 6 Years 至 11 Years（Child）
- 性别
- Male
- 接受健康志愿者
- 否
入选标准
- •Diagnosed with DMD by confirmed medical history and genetic testing
- •Able to walk without assistance
- •Minimum North Star Ambulatory Assessment score of 15 at screening
- •Able to walk up 4 stairs in 8 seconds or less
- •Weigh at least 15 kg (33 lbs)
- •Taking corticosteroids for DMD
排除标准
- •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
- •Concomitant or previous participation at any time in a gene therapy study
- •Other protocol defined Inclusion/Exclusion Criteria could apply.
研究组 & 干预措施
RO7239361 High Dose
Participants received high dose RO7239361 SC on specified days of the 48-week DB period. Following the DB period participants received high dose RO7239361 on specified days for up to 192 weeks during the open-label period followed by 24 weeks of follow-up.
干预措施: RO7239361 (Drug)
Placebo
Participants received matching placebo solution subcutaneously (SC) on specified days of the 48-week double-blind (DB) period. Following the DB period participants received low dose or high dose RO7239361 on specified days for up to 192 weeks during the open-label period followed by 24 weeks of follow-up.
干预措施: Placebo for RO7239361 (Drug)
RO7239361 Low Dose
Participants received low dose RO7239361 SC on specified days of the 48-week DB period. Following the DB period participants received low dose RO7239361 on specified days for up to 192 weeks during the open-label period followed by 24 weeks of follow-up.
干预措施: RO7239361 (Drug)
结局指标
主要结局
Change From Baseline in the North Star Ambulatory Assessment (NSAA) Total Score at Week 48
时间窗: Baseline, Week 48
The NSAA is a functional scale specifically designed for ambulant boys with Duchenne muscular dystrophy (DMD) that can provide information about motor function. The NSAA is a 17-item test of standing, ability to transition from lying to sitting, sitting to standing, and other mobility assessments. Each of the 17 items is evaluated on an ordinal scale of 0-2: 0 = unable to achieve independently, 1 = modified method but achieves goal independent of physical assistance from another, or 2 = normal with no obvious modification of activity. Total score range is 0 to 34. Higher scores reflect better performance. A positive change from baseline indicates an improvement. Based on the mixed-effect model of repeated measures (MMRM).
Baseline for the North Star Ambulatory Assessment (NSAA) Total Score
时间窗: Baseline
The NSAA is a functional scale specifically designed for ambulant boys with Duchenne muscular dystrophy (DMD) that can provide information about motor function. The NSAA is a 17-item test of standing, ability to transition from lying to sitting, sitting to standing, and other mobility assessments. Each of the 17 items is evaluated on an ordinal scale of 0-2: 0 = unable to achieve independently, 1 = modified method but achieves goal independent of physical assistance from another, or 2 = normal with no obvious modification of activity. Total score range is 0 to 34. Higher scores reflect better performance.
次要结局
- Baseline Time for 4 Stair Climb(Baseline)
- Change From Baseline at Week 48 in Stand From Supine Velocity(Baseline, Week 48)
- Change From Baseline at Week 48 in 4 Stair Climb Velocity (4SCV)(Baseline, Week 48)
- Baseline for the Pediatric Outcome Data Collection Instrument (PODCI) Transfer and Basic Mobility Subscale(Baseline)
- Baseline for the 6 Minute Walk Distance (6MWD)(Baseline)
- Percentage of Participants for Each Clinical Global Impression of Change (CGI-C) Assessment Status at Week 48(Baseline, Week 48)
- Baseline for the Time to Stand From Supine(Baseline)
- Change From Baseline at Week 48 in 10 M Walk/Run Velocity(Baseline, Week 48)
- Change From Baseline at Week 48 in Proximal Lower Extremity Flexor Strength(Baseline, Week 48)
- Change From Baseline at Week 48 in 6 Minute Walk Distance (6MWD)(Baseline, Week 48)
- Number of Participants With Adverse Events (AEs)(During DB period (48 weeks) and Whole study (up to approximately 34 months))
- Number of Participants With AEs Leading to Discontinuation(During DB period (48 weeks) and Whole study (up to approximately 34 months))
- Baseline Time for 10 Meter Walk/Run(Baseline)
- Change From Baseline at Week 48 in 95th Percentile Stride Velocity(Baseline, Week 48)
- Change From Baseline at Week 48 in Pediatric Outcome Data Collection Instrument (PODCI) Transfer and Basic Mobility Subscale(Baseline, Week 48)