A Randomized, Double-blind, Placebo-controlled, Multicenter, Parallel Group, Dose-finding, Pivotal, Phase 2b/3 Study to Evaluate the Efficacy, Safety, and Tolerability of Intravenous BYM338 at 52 Weeks on Physical Function, Muscle Strength, and Mobility and Additional Long Term Safety up to 2 Years in Patients With Sporadic Inclusion Body Myositis
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 入组人数
- 251
- 试验地点
- 1
- 主要终点
- Change From Baseline in 6 Minute Walking Distance (6MWD) Test at Week 52
研究概览
简要总结
This study evaluated the efficacy, safety and tolerability of multiple doses of bimagrumab/BYM338 vs placebo, when administered intravenously (i.v.), on physical function, muscle strength, and mobility in patients with sporadic inclusion body myositis (sIBM).
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
- Double (Participant, Investigator)
入排标准
- 年龄范围
- 36 Years 至 85 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Diagnosed with sporadic inclusion body myositis;
- •Must be able to walk (assistive aids allowed, including intermittent use of wheelchair);
排除标准
- •Must not have other conditions that significantly limit ability to move around;
- •Must not be using corticosteroids. Must not have used systemic corticosteroid (at daily dose >=10mg prednisone) for the past 3 months;
- •Must meet cardiovascular requirements;
- •Must not be pregnant or nursing;
- •Must not have a chronic active infection (e.g., HIV, hepatitis B or C, tuberculosis, etc.);
研究组 & 干预措施
BYM338/bimagrumab 10 mg/kg
Participants received study medication with BYM338 at 10 mg/kg from Day 1 to Week 52 and up to Week 104, administered by intravenous (i.v.) infusion every 4 weeks.
干预措施: BYM338/bimagrumab (Drug)
BYM338/bimagrumab 3 mg/kg
Participants received study medication with BYM338 at 3 mg/kg from Day 1 to Week 52 and up to Week 104, administered by i.v. infusion every 4 weeks.
干预措施: BYM338/bimagrumab (Drug)
BYM338/bimagrumab 1 mg/kg
Participants received study medication with BYM338 at 1 mg/kg from Day 1 to Week 52 and up to Week 104, administered by i.v. infusion every 4 weeks.
干预措施: BYM338/bimagrumab (Drug)
Placebo
Participants received matching placebo to BYM338 from Day 1 to Week 52 and up to Week 104, administered by i.v. infusion every 4 weeks.
干预措施: Placebo (Drug)
结局指标
主要结局
Change From Baseline in 6 Minute Walking Distance (6MWD) Test at Week 52
时间窗: Baseline, Week 52
The 6MWD test measured the distance (in meters) that a participant walked in a 6 minute timeframe. A positive change from baseline indicates improvement.
次要结局
- Change From Baseline in Quadriceps Quantitative Muscle Testing (QMT) on the Right Side at Week 52(Baseline, Week 52)
- Estimated Within Treatment Group Lean Body Mass (LBM) Ratio at Week 52(Baseline, Week 52)
- Change From Baseline in Sporadic Inclusion Body Myositis (sIBM) Functional Assessment (sIFA) Score at Week 52(Baseline, Week 52)
- Estimated Annual Number of Falls Per Patient Within Treatment Group(Week 52)
- Change From Baseline in Short Physical Performance Battery (SPPB) Score at Week 52(Baseline, Week 52)