A Phase 2, Randomized, Double-Blind, Placebo-Controlled Study of NIDO-361 in Patients With Spinal and Bulbar Muscular Atrophy (SBMA)
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 入组人数
- 54
- 试验地点
- 5
- 主要终点
- Number of Participants Discontinuing Study Drug and Number of Participants' Deaths
研究概览
简要总结
The purpose of this study is to evaluate the safety, tolerability, and efficacy of NIDO-361 in adult patients with Spinal and Bulbar Muscular Atrophy (SBMA).
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
- 18 Years 至 70 Years（Adult, Older Adult）
- 性别
- Male
- 接受健康志愿者
- 否
入选标准
- •Ambulatory male
- •Documented SBMA diagnosis confirmed by DNA genetic testing
- •Able to complete six-minute walk test (6MWT)
排除标准
- •Clinically significant cardiovascular, endocrine, hepatic, renal, pulmonary, gastrointestinal, neurologic, immunologic, malignant, metabolic, psychiatric, or other condition that, in the opinion of the Investigator, precludes the participant's safe participation in the study or would interfere with the study assessments
- •Inability to undergo MRI (mild sedation may be allowed)
研究组 & 干预措施
NIDO-361
Participants receive NIDO-361, 100 mg for at least 2 months and 200 mg for the remainder of the study.
干预措施: NIDO-361 (Drug)
Placebo
Participants receive matched dose placebo given orally once daily for 12 months.
干预措施: Placebo (Drug)
结局指标
主要结局
Number of Participants Discontinuing Study Drug and Number of Participants' Deaths
时间窗: Through study completion, an average of 1 year
Number of Participants With Adverse Events (AEs) or Serious Adverse Events (SAEs)
时间窗: Through study completion, an average of 1 year
Adverse events were coded with MedDRA Dictionary Version 26.1.
Analysis of Thigh and Total Lean Muscle Volume (LMV) [A+B+C]
时间窗: Baseline, Day 180, Days 360-374 (Last Visit)
Analysis of thigh included adductors, hamstrings, and quadriceps and total (whole-body) included muscles in the rotator cuff, arm, torso, and leg muscle regions (38 muscles in total). Muscles were categorized according to their fat content as follows: A-muscles were defined as muscle fat fraction (MFF) \< 50% and muscle fat infiltration (MFI) \< 10%. A-muscles have a low or slightly elevated fat content to a level that can be present in individuals without a neuromuscular disease. B-muscles were defined as MFF \< 50% and MFI ≥ 10%. B-muscles have a muscle fat content at a level where disease involvement is likely. C-muscles were defined as MFF ≥ 50%. C-muscles have a muscle fat content where more than 50% of the muscle tissue has been replaced by fat and most of the functional capacity is likely lost.
Total Number of Mild, Moderate, and Severe Adverse Events (AEs) Across All Participants
时间窗: Through study completion, an average of 1 year
Adverse events were coded with MedDRA Dictionary Version 26.1.
次要结局
- Change From Baseline to Day 180 and Day 360 in Modified-SBMAFRS (m-SBMAFRS)(Baseline, Day 180, Day 360)
- Change From Baseline to Day 180 and Day 360 in Two-Minute Walk Test (2MWT)(Baseline, Day 180, Day 360)
- Change From Baseline to Day 180 and Day 360 in 6-Minute Walk Test (6MWT)(Baseline, Day 180, Day 360)
- Change From Baseline to Day 180 and Day 360 in Timed Up and Go (TUG) Test(Baseline, Day 180, Day 360)
- Change From Baseline to Treatment (Days 331 to 360) in Actigraphy-Derived Measurement: Non-Sedentary Behavior(Baseline, Treatment (Days 331 to 360))
- Change From Baseline to Treatment (Days 331 to 360) in Actigraphy-Derived Measurement: 95th Percentile of 6-Minute Physical Activity Windows(Baseline, Treatment (Days 331 to 360))
- Change From Baseline to Treatment (Days 331 to 360) in Acitgraphy-Derived Measurement: Maximum Walking Bout Duration(Baseline, Treatment (Days 331 to 360))
- Change From Baseline to Treatment (Days 331 to 360) in Actigraphy-Derived Measurement: Peak 95th Percentile Cadence(Baseline, Treatment (Days 331 to 360))
- Change From Baseline to Treatment (Days 331 to 360) in Actigraphy Derived Measurement: Average Breathing Rate(Baseline, Treatment (Days 331 to 360))
- Change From Baseline to Day 180 and Day 360 in Grip Strength as Measured by Handheld Dynamometer (HHD)(Baseline, Day 180, Day 360)