Clinical trial NCT06482437: Safety and Efficacy of NMD670 in Adult Patients with Type 1 and Type 2 Charcot-Marie-Tooth Disease
Official title: A Phase 2a, Randomised, Double-Blind, Placebo-Controlled Study to Evaluate the Efficacy, Safety, and Tolerability of NMD670 Over 21 Days in Ambulatory Adult Patients with Type 1 and Type 2 Charcot-Marie-Tooth Disease
Study type: INTERVENTIONAL
Overall status: RECRUITING
Phase: PHASE2
Conditions: Charcot-Marie-Tooth Disease
Lead sponsor: NMD Pharma A/S
Sponsor class: INDUSTRY
First posted: 2024-07-01
Enrollment: 80
Sex: ALL
Minimum age (years): 18
Maximum age (years): 70
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

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.


Eligibility criteria: 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.

Primary outcomes:
measure: Change from baseline to day 21 in 6-minute walk test total distance for NMD670 vs placebo
timeFrame: Baseline to day 21
measure: Change from baseline to day 21 in the time to complete 10 meter walk/run test for NMD670 vs placebo
timeFrame: Baseline to day 21
measure: Change from baseline to day 21 in the time to complete timed-up-and-go for NMD670 vs placebo
timeFrame: Baseline to day 21
Secondary outcomes:
measure: Change from baseline to day 21 in CMT Functional Outcome Measure Total Score and Individual Items for NMD670 vs placebo
timeFrame: Baseline to day 21
measure: Change from baseline to day 21 in 6-minute walk test fatigue index for NMD670 vs placebo
timeFrame: Baseline to day 21
measure: Change from baseline to day 21 in Overall Neuropathy Limitation Scale total score and individual items for NMD670 vs placebo
timeFrame: Baseline to day 21
measure: Change from baseline to day 21 in CMT Health Index total score and individual domains for NMD670 vs placebo
timeFrame: Baseline to day 21
measure: Change from baseline to day 21 in SF-36 total score and individual domains for NMD670 vs placebo
timeFrame: Baseline to day 21
measure: Incidence of treatment emergent adverse events
timeFrame: Over 21 days of dosing
measure: Incidence of serious treatment emergent adverse events
timeFrame: Over 21 days of dosing
measure: Incidence of clinically significant abnormalities on physical examinations
timeFrame: Over 21 days of dosing
measure: Incidence of clinically significant abnormalities on safety laboratory parameters
timeFrame: Over 21 days of dosing
measure: Incidence of clinically significant vital signs abnormalities
timeFrame: Over 21 days of dosing
measure: Incidence of clinically significant ECG abnormalities
timeFrame: Over 21 days of dosing
measure: Incidence of Suicidal Ideation or Suicidal Behavior
timeFrame: Over 21 days of dosing
measure: Incidence of clinically significant abnormalities on opthalmological examinations
timeFrame: From screening (day -28 to day -1) until follow up (day 28)]
Other outcomes:
Locations:
city: Kansas City
country: United States
facility: University of Kansas Medical Center, Department of Neurology
state: Kansas
city: Boston
country: United States
facility: Mass General Neurology
state: Massachusetts
city: Columbia
country: United States
facility: NextGen Precision Health
state: Missouri
city: New York
country: United States
facility: Columbia University Medical Center
state: New York
city: Rochester
country: United States
facility: University of Rochester Neuromuscular Disease Center
state: New York
city: Columbus
country: United States
facility: OSU Department of Neurology Division of Neuromuscular Diseases
state: Ohio
city: Austin
country: United States
facility: National Neuromuscular research Institute, PLLC
state: Texas
city: Spokane
country: United States
facility: Providence Medical Research Center
state: Washington
city: Leuven
country: Belgium
facility: University Hospitals Leuven, Department of Neurology
city: Liege
country: Belgium
facility: CHR de la Citadelle- Site Citadelle Neurolgie Boulevard du 12eme de Ligne 1
city: Aarhus
country: Denmark
facility: Aarhus University Hospital
city: Copenhagen
country: Denmark
facility: Rigshospitalet, Department of Neurology
city: Marseille
country: France
facility: CHU Marseille, Reference centre for neuromuscular diseases and ALS Department of Neuromuscular Diseases
city: Nantes
country: France
facility: Laboratoire d'Explorations Fonctionnelles, CHU Nantes
city: Nice
country: France
facility: Centre Hospitalier Universitaire De Nice
city: Paris
country: France
facility: Bicêtre University Hospital
city: Barcelona
country: Spain
facility: Hospital Universitari Vall d'Hebron
city: Valencia
country: Spain
facility: Hospital Universitari i Politecnic La Fe de Valencia
Study officials:
References: