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

ArmDescriptionAssigned Intervention
Experimental
NMD670
  • Drug: NMD670
    Tablets taken twice daily for 21 days
Placebo Comparator
Placebo
  • Drug: Placebo
    Tablets taken twice daily for 21 days

Recruiting Locations

Mass General Neurology
Boston, Massachusetts 02114
Contact:
Reza Seyedsadjadi
919-280-5582
rseyedsadjadi@mgh.harvard.edu

More Details

Status
Recruiting
Sponsor
NMD Pharma A/S

Study Contact

contact@nmdpharma.com
contact@nmdpharma.com
contact@nmdpharma.com