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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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| NMD670 | Experimental |
| |
| Placebo | Placebo Comparator |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| NMD670 | Drug | Tablets taken twice daily for 21 days |
| |
| Placebo |
| Measure | Description | Time Frame |
|---|---|---|
| Change from baseline to day 21 in 6-minute walk test total distance for NMD670 vs placebo | Baseline to day 21 |
| Measure | Description | Time Frame |
|---|---|---|
| Change from baseline to day 21 in CMT Functional Outcome Measure Total Score and Individual Items for NMD670 vs placebo | CMT Functional Outcome Meausre is a 12-item scale. Scale goes from 0-100 and a higher score indicates worse symptomatology | Baseline to day 21 |
| Change from baseline to day 21 in the time to complete the 10MW/RT for NMD670 vs placebo |
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Inclusion Criteria:
Exclusion Criteria:
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| University of Kansas Medical Center, Department of Neurology | Kansas City | Kansas | 66160 | United States | ||
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 41224659 | Derived | Moss KR, Darvishi FB, Badawi Y, Fish LA, Funke JR, Pedersen TH, Robitaille R, Arnold WD, Burgess RW, Meriney SD, Nishimune H, Saxena S. The Neuromuscular Junction: A Shared Vulnerability in Aging and Disease. J Neurosci. 2025 Nov 12;45(46):e1353252025. doi: 10.1523/JNEUROSCI.1353-25.2025. |
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| Drug |
Tablets taken twice daily for 21 days |
|
| 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 | 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 | Baseline to day 21 |
| Change from baseline to day 21 in CMT Health Index total score and individual domains for NMD670 vs placebo | The CMT Health Index has 18 domains. Scale goes from 0-100 and a higher score indicates worse symptomatology | Baseline to day 21 |
| Change from baseline to day 21 in SF-36 total score and individual domains for NMD670 vs placebo | The SF-36 has 8 domains. Scale goes from 0-100 and a lower score indicates worse symptomatology | Baseline to day 21 |
| Change from baseline to day 21 in jitter and blocking for NMD670 vs placebo | Baseline to day 21 |
| Incidence of treatment emergent adverse events | Summarised per treatment | Over 21 days of dosing |
| Incidence of serious treatment emergent adverse events | Summarised per treatment | Over 21 days of dosing |
| Incidence of clinically significant abnormalities on physical examinations | Summarised per treatment | Over 21 days of dosing |
| Incidence of clinically significant abnormalities on safety laboratory parameters | Summarised per treatment | Over 21 days of dosing |
| Incidence of clinically significant vital signs abnormalities | Summarised per treatment | Over 21 days of dosing |
| Incidence of clinically significant ECG abnormalities | Summarised per treatment | Over 21 days of dosing |
| Incidence of Suicidal Ideation or Suicidal Behavior | Summarised per treatment | Over 21 days of dosing |
| Incidence of clinically significant abnormalities on opthalmological examinations | Summarised per treatment | From screening (day -28 to day -1) until follow up (day 28)] |
| 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 |
| Mass General Neurology |
| Boston |
| Massachusetts |
| 02114 |
| United States |
| NextGen Precision Health | Columbia | Missouri | 65211 | United States |
| Columbia University Medical Center | New York | New York | 10032 | United States |
| University of Rochester Neuromuscular Disease Center | Rochester | New York | 14642 | United States |
| OSU Department of Neurology Division of Neuromuscular Diseases | Columbus | Ohio | 43221 | United States |
| National Neuromuscular research Institute, PLLC | Austin | Texas | 78759 | United States |
| Providence Medical Research Center | Spokane | Washington | 99204 | United States |
| University Hospitals Leuven, Department of Neurology | Leuven | 3000 | Belgium |
| CHR de la Citadelle- Site Citadelle Neurolgie Boulevard du 12eme de Ligne 1 | Liège | 4000 | Belgium |
| Aarhus University Hospital | Aarhus | Denmark |
| Rigshospitalet, Department of Neurology | Copenhagen | Denmark |
| CHU Marseille, Reference centre for neuromuscular diseases and ALS Department of Neuromuscular Diseases | Marseille | 13005 | France |
| Laboratoire d'Explorations Fonctionnelles, CHU Nantes | Nantes | France |
| Centre Hospitalier Universitaire De Nice | Nice | 06001 | France |
| Institut de Myologie Groupe Hospitalier Pitié-Salpêtrière | Paris | 75651 | France |
| Bicêtre University Hospital | Paris | 94275 | France |
| Hospital Universitari Vall d'Hebron | Barcelona | 8035 | Spain |
| Hospital Universitari i Politecnic La Fe de Valencia | Valencia | 46026 | Spain |
| ID | Term |
|---|---|
| D002607 | Charcot-Marie-Tooth Disease |
| ID | Term |
|---|---|
| D015417 | Hereditary Sensory and Motor Neuropathy |
| D009421 | Nervous System Malformations |
| D009422 | Nervous System Diseases |
| D020271 | Heredodegenerative Disorders, Nervous System |
| D019636 | Neurodegenerative Diseases |
| D011115 | Polyneuropathies |
| D010523 | Peripheral Nervous System Diseases |
| D009468 | Neuromuscular Diseases |
| D000013 | Congenital Abnormalities |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D030342 | Genetic Diseases, Inborn |
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