Charcot-Marie-Tooth Disease
Conditions
Brief 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.
Interventions
DRUGNMD670
Tablets taken twice daily for 21 days
DRUGPlacebo
Tablets taken twice daily for 21 days
Sponsors
NMD Pharma A/S
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
TRIPLE (Subject, Investigator, Outcomes Assessor)
Eligibility
Sex/Gender
ALL
Age
18 Years to 70 Years
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.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Change from baseline to day 21 in 6-minute walk test total distance for NMD670 vs placebo | Baseline to day 21 |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Change from baseline to day 21 in the time to complete the 10MW/RT for NMD670 vs placebo | Baseline to day 21 | — |
| Change from baseline to day 21 in 6-minute walk test fatigue index for NMD670 vs placebo | Baseline to day 21 | — |
| Change from baseline to day 21 in Overall Neuropathy Limitation Scale total score and individual items for NMD670 vs placebo | Baseline to day 21 | The Overall Neuropathy Limitation Scale consists of an arm and a leg scale. Scale goes from 0-12 and a higher score indicates worse symptomatology |
| Change from baseline to day 21 in CMT Health Index total score and individual domains for NMD670 vs placebo | Baseline to day 21 | The CMT Health Index has 18 domains. Scale goes from 0-100 and a higher score indicates worse symptomatology |
| Change from baseline to day 21 in SF-36 total score and individual domains for NMD670 vs placebo | Baseline to day 21 | The SF-36 has 8 domains. Scale goes from 0-100 and a lower score indicates worse symptomatology |
| Change from baseline to day 21 in jitter and blocking for NMD670 vs placebo | Baseline to day 21 | — |
| Incidence of treatment emergent adverse events | Over 21 days of dosing | Summarised per treatment |
| Incidence of serious treatment emergent adverse events | Over 21 days of dosing | Summarised per treatment |
| Incidence of clinically significant abnormalities on physical examinations | Over 21 days of dosing | Summarised per treatment |
| Change from baseline to day 21 in CMT Functional Outcome Measure Total Score and Individual Items for NMD670 vs placebo | Baseline to day 21 | CMT Functional Outcome Meausre is a 12-item scale. Scale goes from 0-100 and a higher score indicates worse symptomatology |
| Incidence of clinically significant vital signs abnormalities | Over 21 days of dosing | Summarised per treatment |
| Incidence of clinically significant ECG abnormalities | Over 21 days of dosing | Summarised per treatment |
| Incidence of Suicidal Ideation or Suicidal Behavior | Over 21 days of dosing | Summarised per treatment |
| Incidence of clinically significant abnormalities on opthalmological examinations | From screening (day -28 to day -1) until follow up (day 28)] | Summarised per treatment |
| Proportion of participants with clinically meaningful change from baseline in CMT-FOM total score and individual items for NMD670 vs placebo | Baseline to day 21 | — |
| Proportion of participants with clinically meaningful change from baseline in 10MW/RT for NMD670 vs placebo | Baseline to day 21 | — |
| Proportion of participants with clinically meaningful change from baseline in ONLS total score for NMD670 vs placebo | Baseline to day 21 | — |
| Proportion of participants with clinically meaningful change from baseline in CMT-HI total score and individual domains for NMD670 vs placebo | Baseline to day 21 | — |
| Proportion of participants with clinically meaningful change from baseline in SF-36 total score and individual domains for NMD670 vs placebo | Baseline to day 21 | — |
| Incidence of clinically significant abnormalities on safety laboratory parameters | Over 21 days of dosing | Summarised per treatment |
Countries
Belgium, Denmark, France, Spain, United States
Outcome results
None listed