Safety and Efficacy of NMD670 in Adult Patients with Type 1 and Type 2 Charcot-Marie-Tooth Disease
- Conditions
- Charcot-Marie-Tooth Disease Type 1 and Type 2MedDRA version: 20.0Level: LLTClassification code: 10008414Term: Charcot-Marie-Tooth disease Class: 10010331Therapeutic area: Diseases [C] - Nervous System Diseases [C10]
- Registration Number
- CTIS2023-507892-23-00
- Lead Sponsor
- MD Pharma A/S
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- ot Recruiting
- Sex
- All
- Target Recruitment
- 80
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., The following physical capabilities must be met without assistive devices such as AFOs (orthopaedic inserts are allowed) at screening (both must apply): a. 6MWT total distance within 1 standard deviation of the mean adjusted by age and sex from the provided CMT norm. b. Bilateral ankle dorsiflexion strength of 4 (inclusive) or less on the manual muscle testing scale., Participants already receiving physical or occupational therapy or following a prescribed training regimen for more than 30 days prior to screening should continue their current treatment regimen throughout the study. Participants are not allowed to start physical or occupational therapy or a prescribed training regimen within 30 days of screening or during the study., Participants taking medication for muscle cramps (e.g., magnesium) and/or pain (e.g., nonsteroidal anti-inflammatory drug [NSAID], neuropathic pain medication, or opiates) should be on a stable dose for at least 30 days prior to screening and should maintain a stable dose for the duration of the study., Body mass index between 18 and 35 kg/m2 inclusive at screening and a minimum weight of 40 kg., Female participants who are women of childbearing potential (WOCBP) and male participants with partners who are WOCBP must agree to use a highly effective contraception method during the study. Contraceptive use by men and women must be consistent with local regulations regarding the methods of contraception for those participating in clinical studies (refer to Section 10.4, Appendix 4 for additional information)., Participant is capable of giving signed informed consent.
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 breast cancer, lymphoma, leukaemia, or any malignancy within the past 5 years. An exception of this 5-year requirement is basal cell or squamous epithelial carcinomas of the skin that have been resected with no evidence of metastatic disease., Participants with an ongoing significant psychiatric disorder (e.g., unstable and newly diagnosed major depressive disorder or unstable and newly diagnosed anxiety disorders)., Participants with alcohol abuse as judged by the Investigator or with positive drug screen results (cocaine, heroin, opiates, or marijuana) at screening. a. If positive for marijuana, criteria for drug abuse (as determined by the Investigator) is also needed to meet this exclusion criterion. b. If positive for opiates, the exclusion criterion is not met if opiates have been medically prescribed for the treatment of pain in CMT., Participants with a positive HIV antibody test result., Participants with positive serology test results for hepatitis B (unless due to vaccination, resolved natural infection, or passive immunisation, as confirmed with the Medical Monitor)., Participants positive for hepatitis C antibody., Participants with a clinically significant history of allergic conditions (including drug allergies and anaphylactic reactions) or hypersensitivity to any component of the IMP., Participant with myotonic disorders or those on drugs that induce or mask myotonia., Participants unable to undergo the ophthalmologic evaluation at screening., Participants who require prohibited medication within 30 days of screening (or 5 half-lives of the medication, whichever is shorter) and/or are likely to require them during the study; prohibited medications include drugs showing relevant effects on NMJ transmission and drugs where the effect might be affected by potential metabolic drug-drug interactions with NMD670. Other current and recent (within 1 month prior to screening) treatments will be allowed if judged by the Investigator to be of no relevance for the study., Use of daily assistive devices such as AFOs (orthopaedic inserts are allowed)., 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., Severe deformity or ankle contracture that in the opinion of the Investigator would limit passive range of motion to interfere with performance of the tests., Ankle surgery or other limb-surgery procedures related to CMT within 9 months of screening., Moderate-to-severe neuropathic or inflammatory/musculoskeletal pain that in the opinion of the Investigator would interfere with performance of the tests., Participants with a clinical diagnosis of gout or with serum uric acid greater than the upper limit of normal (ULN) at screening., Study participants with a centrally read 12-lead ECG with findings considered to be clinically significant upon medical review. The clinical significance of the findings needs to be assessed by the Investigator to determine eligibility. Participants with any of the following a
Study & Design
- Study Type
- Interventional clinical trial of medicinal product
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method
- Secondary Outcome Measures
Name Time Method