Safety and Efficacy of NMD670 in Adult Patients With Type 1 and Type 2 Charcot-Marie-Tooth Disease
NCT Number: NCT06482437
Acronym: SYNAPSE-CMT
Status: NOT_YET_RECRUITING
Study Type: INTERVENTIONAL
Phases: PHASE2
Conditions: Charcot-Marie-Tooth Disease
Interventions: DRUG: NMD670|DRUG: Placebo
Sponsor: NMD Pharma A/S
Sex: ALL
Age: ADULT, OLDER_ADULT
Enrollment: 80
Study Design: Allocation: RANDOMIZED|Intervention Model: PARALLEL|Masking: TRIPLE (PARTICIPANT, INVESTIGATOR, OUTCOMES_ASSESSOR)|Primary Purpose: TREATMENT
Start Date: 2024-09-25
Completion Date: 2026-03-16

Summary:
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.

Primary Outcome Measures: Change from baseline to day 21 in 6-minute walk test total distance for NMD670 vs placebo, Baseline to day 21|Change from baseline to day 21 in the time to complete 10 meter walk/run test for NMD670 vs placebo, Baseline to day 21|Change from baseline to day 21 in the time to complete timed-up-and-go for NMD670 vs placebo, Baseline to day 21
Secondary Outcome Measures: Change from baseline to day 21 in CMT Functional Outcome Measure Total Score and Individual Items for NMD670 vs placebo, CMT Functional Outcome Meausre is a 12-item scale. Scale goes from 0-100 and a higher score indicates worse symptomatology, 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 Overall Neuropathy Limitation Scale total score and individual items for NMD670 vs placebo, The Overall Neuropathy Limitation Scale consists of an arm and a leg scale. Scale goes from 0-12 and a higher score indicates worse symptomatology, Baseline to day 21|Change from baseline to day 21 in CMT Health Index total score and individual domains for NMD670 vs placebo, The CMT Health Index has 18 domains. Scale goes from 0-100 and a higher score indicates worse symptomatology, Baseline to day 21|Change from baseline to day 21 in SF-36 total score and individual domains for NMD670 vs placebo, The SF-36 has 8 domains. Scale goes from 0-100 and a lower score indicates worse symptomatology, Baseline to day 21|Incidence of treatment emergent adverse events, Summarised per treatment, Over 21 days of dosing|Incidence of serious treatment emergent adverse events, Summarised per treatment, Over 21 days of dosing|Incidence of clinically significant abnormalities on physical examinations, Summarised per treatment, Over 21 days of dosing|Incidence of clinically significant abnormalities on safety laboratory parameters, Summarised per treatment, Over 21 days of dosing|Incidence of clinically significant vital signs abnormalities, Summarised per treatment, Over 21 days of dosing|Incidence of clinically significant ECG abnormalities, Summarised per treatment, Over 21 days of dosing|Incidence of Suicidal Ideation or Suicidal Behavior, Summarised per treatment, Over 21 days of dosing|Incidence of clinically significant abnormalities on opthalmological examinations, Summarised per treatment, From screening (day -28 to day -1) until follow up (day 28)]