Safety and Efficacy of NMD670 in Adult Patients With Type 1 and Type 2 Charcot-Marie-Tooth Disease (NCT06482437) | Clinical Trial Compass
CompletedPhase 2
Safety and Efficacy of NMD670 in Adult Patients With Type 1 and Type 2 Charcot-Marie-Tooth Disease
United States81 participantsStarted 2024-09-30
Plain-language 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 2.
Who can participate
Age range18 Years – 70 Years
SexALL
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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.
What they're measuring
1
Change from baseline to day 21 in 6-minute walk test total distance for NMD670 vs placebo