Safety and Efficacy of NMD670 in Adult Patients With Type 1 and Type 2 Charcot-Marie-Tooth Disease
Purpose
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.
Condition
- Charcot-Marie-Tooth Disease
Eligibility
- Eligible Ages
- Between 18 Years and 70 Years
- Eligible Genders
- All
- Accepts Healthy Volunteers
- No
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.
Study Design
- Phase
- Phase 2
- Study Type
- Interventional
- Allocation
- Randomized
- Intervention Model
- Parallel Assignment
- Primary Purpose
- Treatment
- Masking
- Triple (Participant, Investigator, Outcomes Assessor)
Arm Groups
| Arm | Description | Assigned Intervention |
|---|---|---|
|
Experimental NMD670 |
|
|
|
Placebo Comparator Placebo |
|
Recruiting Locations
Mass General Neurology
Boston, Massachusetts 02114
Boston, Massachusetts 02114
More Details
- Status
- Recruiting
- Sponsor
- NMD Pharma A/S