Safety and Efficacy of NMD670 in Adult Patients With Type 1 and Type 2 Charcot-Marie-Tooth Disease (SYNAPSE-CMT)

NMD Pharma

Status and phase

Enrolling

Phase 2

Conditions

Charcot-Marie-Tooth Disease

Treatments

Drug: NMD670

Drug: Placebo

Study type

Interventional

Funder types

Industry

Identifiers

NCT06482437

NMD670-02-0003

Details and patient eligibility

About

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.

Enrollment

80 estimated patients

Sex

All

Ages

18 to 70 years old

Volunteers

No Healthy Volunteers

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.