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

NMD670-02-0003

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.

2024-09-25

2026-03-16

2026-03-16

Not yet recruiting

Early phase I

80

Inclusion Criteria: * 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 Exclusion Criteria: * 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 1. * 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.

{'studyType': 'INTERVENTIONAL', 'phases': ['PHASE2'], 'designInfo': {'allocation': 'RANDOMIZED', 'interventionModel': 'PARALLEL', 'primaryPurpose': 'TREATMENT', 'maskingInfo': {'masking': 'TRIPLE', 'whoMasked': ['PARTICIPANT', 'INVESTIGATOR', 'OUTCOMES_ASSESSOR']}}, 'enrollmentInfo': {'count': 80, 'type': 'ESTIMATED'}}

2024-07-01