NCT05794139已完成2 期
A Phase 2, Randomised, Double-blind, Placebo-controlled, 2-way Crossover Study to Evaluate the Efficacy, Safety, and Tolerability of NMD670 in Ambulatory Adults With Type 3 Spinal Muscular Atrophy
适应症
干预措施
相关药物
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 入组人数
- 52
- 试验地点
- 49
- 主要终点
- Change from baseline in 6 minute walk test (6MWT) total distance versus placebo
研究概览
简要总结
The purpose of this study is to evaluate the efficacy, safety, tolerability and pharmacokinetics of NMD670 in the treatment of ambulatory adults with spinal muscular atrophy type 3
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Crossover
- 主要目的
- Treatment
- 盲法
- Triple (Participant, Investigator, Outcomes Assessor)
入排标准
- 年龄范围
- 18 Years 至 75 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Participants with a clinical diagnosis of Type 3 SMA.
- •Participants who are ambulatory, defined as being able to walk at least 50 metres without walking aids at screening during the 6-minute walk test.
- •Participant with genetic confirmation of diagnosis (e.g., homozygous deletion or compound heterozygous deletion and mutation of survival of motor neuron 1 gene [SMN1])
- •Participant with 3 to 5 copies of survival of motor neuron 2 gene [SMN2].
- •Participant has a body mass index (BMI) within the range 19-35 kg/m2 (inclusive).
- •Participant is male or female.
- •Contraceptive use by men and women must be consistent with local regulations regarding the methods of contraception for those participating in clinical studies.
- •Participant is capable of giving signed informed consent which includes compliance with the requirements and restrictions listed in the informed consent form (ICF) and in the protocol.
排除标准
- •Participants with prior surgery or fixed deformity (scoliosis, contractures) which would restrict ability to perform study-related tasks.
- •Participants with other significant disease that may interfere with the interpretation of study data (e.g., other neuromuscular or muscular diseases).
- •Participants with other significant clinical and/or laboratory safety findings that may interfere with the conduction or interpretation of the study
- •Participants received treatment with an investigational medical product (IMP) within 30 days (or 5 half-lives of the medication, whichever is longer) prior to Day
- •Participants with history of poor compliance with relevant SMA therapy.
研究组 & 干预措施
Cohort 2
Experimental
Placebo followed by experimental drug
干预措施: Placebo (Drug)
Cohort 1
Experimental
Experimental drug followed by placebo
干预措施: NMD670 (Drug)
Cohort 2
Experimental
Placebo followed by experimental drug
干预措施: NMD670 (Drug)
Cohort 1
Experimental
Experimental drug followed by placebo
干预措施: Placebo (Drug)
结局指标
主要结局
Change from baseline in 6 minute walk test (6MWT) total distance versus placebo
时间窗: Baseline to day 21
Distance walked (meters)
次要结局
- Incidence of treatment emergent adverse events(Over 21 days of dosing)
- Change from baseline in Revised Hammersmith Scale (RHS) versus placebo(Baseline to day 21)
- Incidence of clinically significant abnormalities on safety laboratory parameters(Over 21 days of dosing)
- Incidence of clinically significant vital signs abnormalities(Over 21 days of dosing)
- Incidence of clinically significant ECG abnormalities(Over 21 days of dosing)
- Change from baseline in muscle strength versus placebo(Baseline to day 21)
- Change from baseline in 6 minute walk test (6MWT) fatigue index versus placebo(Baseline to day 21)
- Change from baseline in jitter versus placebo(Baseline to day 21)
- Change from baseline in blocking versus placebo(Baseline to day 21)
- Incidence of serious treatment emergent adverse events(Over 21 days of dosing)
- Incidence of Suicidal Ideation or Suicidal Behavior(Over 21 days of dosing)
- Incidence of clinically significant abnormalities on opthalmological examinations(Over 21 days of dosing)
- Incidence of clinically significant abnormalities on physical examinations(Over 21 days of dosing)
研究者
研究点 (49)
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