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Completed

NCT Number: NCT06482437

Safety and Efficacy of NMD670 in Adult Patients With Type 1 and Type 2 Charcot-Marie-Tooth Disease

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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Key information

Conditions

Charcot-Marie-Tooth Disease Congenital Abnormalities Congenital, Hereditary, and Neonatal Diseases and Abnormalities Genetic Diseases, Inborn Hereditary Sensory and Motor Neuropathy Heredodegenerative Disorders, Nervous System Nervous System Diseases Nervous System Malformations Neurodegenerative Diseases Neuromuscular Diseases Peripheral Nervous System Diseases Polyneuropathies

Age range

18 year–70 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 2

Primary location

University Hospitals Leuven, Department of Neurology, Leuven, Belgium

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Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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.

Treatment and study plan

NMD670

Drug

Tablets taken twice daily for 21 days

Placebo

Drug

Tablets taken twice daily for 21 days

Primary outcomes

  1. Change from baseline to day 21 in 6-minute walk test total distance for NMD670 vs placebo

    Time frame: Baseline to day 21

Secondary outcomes

  1. Change from baseline to day 21 in CMT Functional Outcome Measure Total Score and Individual Items for NMD670 vs placebo

    Time frame: 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

  2. Change from baseline to day 21 in the time to complete the 10MW/RT for NMD670 vs placebo

    Time frame: Baseline to day 21

  3. Change from baseline to day 21 in 6-minute walk test fatigue index for NMD670 vs placebo

    Time frame: Baseline to day 21

  4. Change from baseline to day 21 in Overall Neuropathy Limitation Scale total score and individual items for NMD670 vs placebo

    Time frame: 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

  5. Change from baseline to day 21 in CMT Health Index total score and individual domains for NMD670 vs placebo

    Time frame: Baseline to day 21

    The CMT Health Index has 18 domains. Scale goes from 0-100 and a higher score indicates worse symptomatology

  6. Change from baseline to day 21 in SF-36 total score and individual domains for NMD670 vs placebo

    Time frame: Baseline to day 21

    The SF-36 has 8 domains. Scale goes from 0-100 and a lower score indicates worse symptomatology

  7. Change from baseline to day 21 in jitter and blocking for NMD670 vs placebo

    Time frame: Baseline to day 21

  8. Incidence of treatment emergent adverse events

    Time frame: Over 21 days of dosing

    Summarised per treatment

  9. Incidence of serious treatment emergent adverse events

    Time frame: Over 21 days of dosing

    Summarised per treatment

  10. Incidence of clinically significant abnormalities on physical examinations

    Time frame: Over 21 days of dosing

    Summarised per treatment

  11. Incidence of clinically significant abnormalities on safety laboratory parameters

    Time frame: Over 21 days of dosing

    Summarised per treatment

  12. Incidence of clinically significant vital signs abnormalities

    Time frame: Over 21 days of dosing

    Summarised per treatment

  13. Incidence of clinically significant ECG abnormalities

    Time frame: Over 21 days of dosing

    Summarised per treatment

  14. Incidence of Suicidal Ideation or Suicidal Behavior

    Time frame: Over 21 days of dosing

    Summarised per treatment

  15. Incidence of clinically significant abnormalities on opthalmological examinations

    Time frame: From screening (day -28 to day -1) until follow up (day 28)]

    Summarised per treatment

  16. Proportion of participants with clinically meaningful change from baseline in CMT-FOM total score and individual items for NMD670 vs placebo

    Time frame: Baseline to day 21

  17. Proportion of participants with clinically meaningful change from baseline in 10MW/RT for NMD670 vs placebo

    Time frame: Baseline to day 21

  18. Proportion of participants with clinically meaningful change from baseline in ONLS total score for NMD670 vs placebo

    Time frame: Baseline to day 21

  19. Proportion of participants with clinically meaningful change from baseline in CMT-HI total score and individual domains for NMD670 vs placebo

    Time frame: Baseline to day 21

  20. Proportion of participants with clinically meaningful change from baseline in SF-36 total score and individual domains for NMD670 vs placebo

    Time frame: Baseline to day 21

Sponsors and collaborators

Lead sponsor

NMD Pharma A/S

Industry

Registry information

Official study 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

Acronym: SYNAPSE-CMT

Important dates

Study start
2024
Primary completion
2025
Study completion
2025
First posted
Jul 1, 2024
Registry last updated
Nov 12, 2025

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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