Extension of the CBYM338B2203 Phase IIb/III Study to Evaluate the Long-term Efficacy, Safety and Tolerability of Intravenous BYM338 in Patients With Sporadic Inclusion Body Myositis
试验速览
- 阶段
- 3 期
- 状态
- 已完成
- 入组人数
- 211
- 试验地点
- 1
- 主要终点
- Number of Participants With Adverse Events (AEs), Serious Adverse Events (SAEs) and Deaths.
研究概览
简要总结
This extension study will provide data to further evaluate the efficacy, safety, and tolerability of three doses of BYM338 and to assess the long-term effects of BYM338 in patients with sporadic inclusion body myositis. The extension study was planned to consist of a Screening epoch (to assess patient eligibility), followed by a Treatment Period 1 epoch (double-blind and placebo-controlled), and a Treatment Period 2 epoch (open-label). A Post-treatment Follow-up (FUP) epoch was also planned for patients who discontinued prematurely. Patients who complete the core study and qualify for this extension study entered Treatment Period 1 and continued on the study drug to which they were randomized in the core study (either to one of the three bimagrumab doses (1 mg/kg, 3 mg/kg, and 10mg/kg) or placebo) during Treatment Period 1. Thus, Treatment Period 1 was double-blind and placebo-controlled. Participants were to continue in Treatment Period 1 until the dose with the best benefit-risk profile was determined from the core study data and selected (duration of Treatment Period 1 was estimated to be between 6 and 8 months). Once the dose with the best benefit-risk profile was selected, all participants (including those who were receiving placebo) were planned to enter Treatment Period 2 and switch to open-label treatment with bimagrumab at the selected dose. The core study has been completed but since the core study did not meet the primary end point (no bimagrumab dose was identified based on the core study efficacy results) the extension study was terminated as per protocol/sponsor's decision; therefore, no patients had entered Treatment Period 2. Instead, all patients were to return for the End of Treatment Period 1 (EOT1) visit at their next scheduled visit. As per protocol, all patients who discontinued study medication during Treatment Period 1 for any reason, including due to the study having been stopped as per protocol/sponsor's decision, were to have entered and complete the 6-month FUP after their EOT1 visit.
Due to the nature of the design of the core and extension studies and termination of study medication in the extension study, the treatment duration for individual patients varied considerably. Consequently, the number of patients contributing data to the efficacy analyses at Week 104 and later timepoints was decreased.
研究设计
- 研究类型
- Interventional
- 分配方式
- Non 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
- 36 Years 至 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Patients who completed the core study
- •Written informed consent must be obtained before any extension study assessment is performed.
- •Able to communicate well with the investigator.
- •Willing to participate for the entire duration of the extension study with commitment to follow study requirements and procedures.
排除标准
- •Women who are pregnant
- •Women of child-bearing potential unless they are using highly effective methods of contraception during dosing and for 6 months after the last BYM338 dose.
- •Current use of prohibited treatments
- •History of severe hypersensitivity reaction in the core study
- •History of adverse event(s) (including those from the core study) prior to the start of study drug in the extension study that, in the judgment of the investigator, taking into account the subject's overall status, prevent the subject from entering the extension study
- •Clinically significant abnormal liver function tests
- •Any medical condition or laboratory finding which, in the opinion of the investigator may interfere with participation in the study, might confound the results of the study, or pose an additional safety risk in administering BYM338
研究组 & 干预措施
BYM338/bimagrumab 10 mg/kg
Participants received BYM338 10 mg/kg administered via intravenous infusion every 4 weeks for up to a maximum of 8 months after which they entered a 6-month, treatment-free follow-up period.
干预措施: Bimagrumab (Drug)
BYM338/bimagrumab 3 mg/kg
Participants received BYM338 3 mg/kg administered via intravenous infusion every 4 weeks for up to a maximum of 8 months after which they entered a 6-month, treatment-free follow-up period.
干预措施: Bimagrumab (Drug)
BYM338/bimagrumab 1 mg/kg
Participants received BYM338 1 mg/kg administered via intravenous infusion every 4 weeks for up to a maximum of 8 months after which they entered a 6-month, treatment-free follow-up period.
干预措施: Bimagrumab (Drug)
Placebo
Participants received placebo administered via intravenous infusion every 4 weeks for up to a maximum of 8 months after which they entered a 6-month, treatment-free follow-up period.
干预措施: Placebo (Drug)
结局指标
主要结局
Number of Participants With Adverse Events (AEs), Serious Adverse Events (SAEs) and Deaths.
时间窗: to end of study (up to 14 months, including the 6-month treatment-free follow-up period)
Safety monitoring was conducted throughout the study. AEs starting on or after the day of first administration of extension study drug until last administration of study drug + 56 days are considered. SAEs starting on or after the day of first administration of extension study drug are considered. Deaths which occurred on or after the day of first administration of extension study drug are considered.
Change From Core Study Baseline in 6 Minute Walking Distance Test (6MWD)
时间窗: Core study baseline, weeks 52, 78, 104, and >=117
The 6MWD test measures the distance (in meters) that a participant can walk in a 6 minute time frame. A positive change from baseline indicates improvement. The efficacy analysis and time points were based on windowed visits relative to the first dose of the double-blind treatment in the core study.
次要结局
- Change From Core Study Baseline in Quadriceps Quantitative Muscle Testing (QMT) on the Right Side(Core study baseline, week 52, week 78, week 104 and >=week 117)
- Change From Core Study Baseline in Sporadic Inclusion Body Myositis (sIBM) Functional Assessment (sIFA) Score(Core study baseline, week 52, week 78, week 104, and >=week 117)
- Estimated Annual Number of Falls Per Participant Within Treatment Group(Core baseline to end of extension double-blind treatment (up to a maximum of 32 months))
- Change From Core Study Baseline in Short Physical Performance Battery (SPPB) Score(Core study baseline, week 52, week 78, week 104 and >=week 117)
- Change in Muscles of the Thigh(up to 1 year, up to 2 years)
- Number of Patients With Anti-BYM338 Antibodies(end of double-blind treatment (up to 8 months))