NCT06482437已完成2 期
A Phase 2a, Randomised, Double-Blind, Placebo-Controlled Study to Evaluate the Efficacy, Safety, and Tolerability of NMD670 Over 21 Days in Ambulatory Adult Patients With Type 1 and Type 2 Charcot-Marie-Tooth Disease
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
- 81
- 试验地点
- 19
- 主要终点
- Change from baseline to day 21 in 6-minute walk test total distance for NMD670 vs placebo
研究概览
简要总结
This Phase 2a study aims to evaluate the efficacy, safety and tolerability of NMD670 vs placebo administered twice a day (BID) for 21 days in ambulatory adult patients with Charcot-Marie-Tooth disease type 1 and type 2.
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
- Triple (Participant, Investigator, Outcomes Assessor)
入排标准
- 年龄范围
- 18 Years 至 70 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Male or female participants must be 18 to 70 years inclusive at the time of signing the ICF.
- •Diagnosis of CMT type 1 or 2 confirmed by genetic testing.
- •Body mass index between 18 and 35 kg/m2, inclusive, at screening, and with a minimum weight of 40 kg
- •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 and has given signed informed consent
排除标准
- •Participants with other significant disease that may interfere with the interpretation of study data (e.g., other neuromuscular diseases) and/or ability to complete the tests, in the opinion of the Investigator.
- •Participants with laboratory test result abnormalities at screening considered clinically significant by the Investigator.
- •Participants who have received treatment with another 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 therapy in the opinion of the Investigator.
- •Female participants who plan to become pregnant during the study or are currently pregnant or breastfeeding.
研究组 & 干预措施
NMD670
Experimental
干预措施: NMD670 (Drug)
Placebo
Placebo Comparator
干预措施: Placebo (Drug)
结局指标
主要结局
Change from baseline to day 21 in 6-minute walk test total distance for NMD670 vs placebo
时间窗: Baseline to day 21
次要结局
- Change from baseline to day 21 in CMT Functional Outcome Measure Total Score and Individual Items for NMD670 vs placebo(Baseline to day 21)
- Change from baseline to day 21 in 6-minute walk test fatigue index for NMD670 vs placebo(Baseline to day 21)
- Change from baseline to day 21 in jitter and blocking for NMD670 vs placebo(Baseline to day 21)
- Change from baseline to day 21 in the time to complete the 10MW/RT for NMD670 vs placebo(Baseline to day 21)
- Change from baseline to day 21 in Overall Neuropathy Limitation Scale total score and individual items for NMD670 vs placebo(Baseline to day 21)
- Change from baseline to day 21 in CMT Health Index total score and individual domains for NMD670 vs placebo(Baseline to day 21)
- Change from baseline to day 21 in SF-36 total score and individual domains for NMD670 vs placebo(Baseline to day 21)
- Incidence of clinically significant abnormalities on safety laboratory parameters(Over 21 days of dosing)
- Incidence of serious treatment emergent adverse events(Over 21 days of dosing)
- Incidence of clinically significant abnormalities on physical examinations(Over 21 days of dosing)
- Incidence of 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 vital signs abnormalities(Over 21 days of dosing)
- Incidence of clinically significant ECG abnormalities(Over 21 days of dosing)
- Incidence of clinically significant abnormalities on opthalmological examinations(From screening (day -28 to day -1) until follow up (day 28)])
- Proportion of participants with clinically meaningful change from baseline in CMT-FOM total score and individual items for NMD670 vs placebo(Baseline to day 21)
- Proportion of participants with clinically meaningful change from baseline in 10MW/RT for NMD670 vs placebo(Baseline to day 21)
- Proportion of participants with clinically meaningful change from baseline in ONLS total score for NMD670 vs placebo(Baseline to day 21)
- Proportion of participants with clinically meaningful change from baseline in CMT-HI total score and individual domains for NMD670 vs placebo(Baseline to day 21)
- Proportion of participants with clinically meaningful change from baseline in SF-36 total score and individual domains for NMD670 vs placebo(Baseline to day 21)
研究者
研究点 (19)
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相关资讯
NMD Pharma's Ignaseclant Shows Promise in Phase 2a Trial for Charcot-Marie-Tooth Disease- NMD Pharma's Phase 2a SYNAPSE-CMT study of ignaseclant demonstrated consistent improvements in muscle strength, motor performance, and patient-reported outcomes in 81 patients with Charcot-Marie-Tooth disease types 1 and 2.
- The first-in-class ClC-1 inhibitor showed robust improvements in handgrip strength and fine hand function, with benefits maintained through day 28 even after treatment discontinuation.
- Although the primary endpoint of 6-minute walk test was not met, the favorable safety profile and functional improvements support accelerated clinical development for this orphan disease with no approved therapies.7 months agoNMD Pharma's NMD670 Receives FDA Orphan Drug Designation for Charcot-Marie-Tooth Disease- NMD Pharma's NMD670, a novel oral small molecule, has been granted Orphan Drug Designation by the FDA for treating Charcot-Marie-Tooth disease (CMT).
- NMD670 inhibits the skeletal muscle-specific chloride ion channel ClC-1 and is currently in Phase 2 trials for CMT types 1 and 2, affecting over 3 million worldwide.
- The FDA's designation aims to incentivize the development of treatments for rare diseases, offering benefits like tax credits and market exclusivity.
- NMD670 has shown promise in preclinical studies and a Phase 2 trial (SYNAPSE-CMT) is underway, with data expected in 2H/2025 through 1H/2026.last yearNMD Pharma's NMD670 Enters Phase 2 Trial for Charcot-Marie-Tooth Disease- NMD Pharma initiates a Phase 2 clinical trial (SYNAPSE-CMT) to evaluate NMD670 in patients with Charcot-Marie-Tooth (CMT) disease types 1 and 2.
- The SYNAPSE-CMT trial is a randomized, double-blind, placebo-controlled study involving 80 adults, assessing NMD670's efficacy, safety, and tolerability over 21 days.
- NMD670, a novel small molecule inhibitor of the skeletal muscle-specific chloride ion channel ClC-1, aims to improve muscle function by enhancing muscle excitability.
- Previous Phase 1 data showed NMD670 to be generally safe and well-tolerated, supporting its further development for neuromuscular diseases.last yearNMD Pharma Initiates Phase 2 Trial of NMD670 for Charcot-Marie-Tooth Disease- NMD Pharma has commenced a Phase 2a clinical trial of NMD670 for Charcot-Marie-Tooth (CMT) disease types 1 and 2, following FDA IND clearance.
- The SYNAPSE-CMT trial (NCT06482437) will assess the efficacy, safety, and tolerability of NMD670 in 80 adult patients over 21 days.
- NMD670, a first-in-class small molecule inhibitor of the skeletal muscle ClC-1 channel, aims to improve neuromuscular transmission and muscle function.
- The study will evaluate changes in the 6-minute walk test, 10-meter walk/run test, and timed-up-and-go test, with sites in the US and Europe.last yearNMD Pharma Initiates Phase 2 Trial of NMD670 for Charcot-Marie-Tooth Disease- NMD Pharma has commenced a Phase 2a clinical trial of NMD670 for Charcot-Marie-Tooth (CMT) disease types 1 and 2, following FDA IND clearance.
- The SYNAPSE-CMT study is a randomized, double-blind, placebo-controlled trial evaluating NMD670's efficacy, safety, and tolerability over 21 days.
- The trial will assess changes in the 6-minute walk test, 10-meter walk/run test, and timed-up-and-go test in 80 adult patients across US and European sites.
- NMD670, a first-in-class small molecule inhibitor of the skeletal muscle-specific chloride ion channel (ClC-1), aims to improve neuromuscular transmission.last year