Charcot-Marie-Tooth DiseasePhase 2SYNAPSE-CMTNCT06482437
Safety and Efficacy of NMD670 in Adult Patients With Type 1 and Type 2 Charcot-Marie-Tooth Disease
A Phase 2 study testing NMD670 in people with Charcot-Marie-Tooth Disease, run by NMD Pharma A/S at 19 sites in 5 countries.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 to 70 years
- Conditions the study is for
- Charcot-Marie-Tooth Disease
- What is being tested?
- NMD670
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 81 participants
- Where
- 19 sitesin 5 countries
- Study dates
- 30 Sep 2024to 4 Nov 2025
- Phase
- Phase 2
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 70 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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
Who cannot join
- 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.
Trial locations and contacts
19 sites in 5 countries.
19 sites in 5 countries
United States8 sites
National Neuromuscular research Institute, PLLC
Austin, Texas, 78759
Mass General Neurology
Boston, Massachusetts, 02114
NextGen Precision Health
Columbia, Missouri, 65211
OSU Department of Neurology Division of Neuromuscular Diseases
Columbus, Ohio, 43221
University of Kansas Medical Center, Department of Neurology
Kansas City, Kansas, 66160
Columbia University Medical Center
New York, New York, 10032
University of Rochester Neuromuscular Disease Center
Rochester, New York, 14642
Providence Medical Research Center
Spokane, Washington, 99204
France5 sites
CHU Marseille, Reference centre for neuromuscular diseases and ALS Department of Neuromuscular Diseases
Marseille, 13005
Laboratoire d'Explorations Fonctionnelles, CHU Nantes
Nantes
Centre Hospitalier Universitaire De Nice
Nice, 06001
Institut de Myologie Groupe Hospitalier Pitié-Salpêtrière
Paris, 75651
Bicêtre University Hospital
Paris, 94275
Belgium2 sites
University Hospitals Leuven, Department of Neurology
Leuven, 3000
CHR de la Citadelle- Site Citadelle Neurolgie Boulevard du 12eme de Ligne 1
Liège, 4000
Denmark2 sites
Aarhus University Hospital
Aarhus
Rigshospitalet, Department of Neurology
Copenhagen
Spain2 sites
Hospital Universitari Vall d'Hebron
Barcelona, 8035
Hospital Universitari i Politecnic La Fe de Valencia
Valencia, 46026
About this study
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
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.
- Conditions
- Charcot-Marie-Tooth Disease
- Being tested
- NMD670, Placebo
- Medical subject headings
- Charcot-Marie-Tooth Disease
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
NMD670Drug
Tablets taken twice daily for 21 days
PlaceboDrug
Tablets taken twice daily for 21 days
| Study group | Role | What they receive |
|---|---|---|
| NMD670 | Experimental | Not described Receives: Drug: NMD670 |
| Placebo | Placebo comparator | Not described Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change from baseline to day 21 in 6-minute walk test total distance for NMD670 vs placebo
Measured: Baseline to day 21
Secondary outcomes (20)
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
Measured: Baseline to day 21
Change from baseline to day 21 in the time to complete the 10MW/RT for NMD670 vs placebo
Measured: Baseline to day 21
Change from baseline to day 21 in 6-minute walk test fatigue index for NMD670 vs placebo
Measured: 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
Measured: 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
Measured: 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
Measured: Baseline to day 21
Change from baseline to day 21 in jitter and blocking for NMD670 vs placebo
Measured: Baseline to day 21
Incidence of treatment emergent adverse events
Summarised per treatment
Measured: Over 21 days of dosing
Incidence of serious treatment emergent adverse events
Summarised per treatment
Measured: Over 21 days of dosing
Incidence of clinically significant abnormalities on physical examinations
Summarised per treatment
Measured: Over 21 days of dosing
Incidence of clinically significant abnormalities on safety laboratory parameters
Summarised per treatment
Measured: Over 21 days of dosing
Incidence of clinically significant vital signs abnormalities
Summarised per treatment
Measured: Over 21 days of dosing
Incidence of clinically significant ECG abnormalities
Summarised per treatment
Measured: Over 21 days of dosing
Incidence of Suicidal Ideation or Suicidal Behavior
Summarised per treatment
Measured: Over 21 days of dosing
Incidence of clinically significant abnormalities on opthalmological examinations
Summarised per treatment
Measured: 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
Measured: Baseline to day 21
Proportion of participants with clinically meaningful change from baseline in 10MW/RT for NMD670 vs placebo
Measured: Baseline to day 21
Proportion of participants with clinically meaningful change from baseline in ONLS total score for NMD670 vs placebo
Measured: 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
Measured: 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
Measured: Baseline to day 21
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted1 Jul 2024
- Study start30 Sep 2024
- Primary completion28 Oct 2025
- Study completion4 Nov 2025
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Charcot-Marie-Tooth DiseaseStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Triple blind
- Who is masked
- Participant, Investigator, Outcomes assessor
- Enrolment
- 81 participants (actual)
Sponsor and investigators
- Lead sponsor
- NMD Pharma A/S
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06482437
- Sponsor's protocol ID
- NMD670-02-0003
- Acronym
- SYNAPSE-CMT
- Status last verified
- Nov 2025
- First submitted
- 25 Jun 2024
- Last updated
- 12 Nov 2025
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
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.
PubMed 41224659
Study at a glance
From the registry record, last updated by the sponsor on 12 Nov 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT06482437
- Acronym / sponsor ID
- SYNAPSE-CMT / NMD670-02-0003
- Conditions
- Charcot-Marie-Tooth Disease
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 81 participants (actual)
- Lead sponsor
- NMD Pharma A/SIndustry (pharma, biotech, devices)
- Study start
- 30 Sep 2024 (actual)
- Primary completion
- 28 Oct 2025 (actual)
- Study completion
- 4 Nov 2025 (actual)
- First posted
- 1 Jul 2024 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Triple blind (participant, investigator, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| NMD670 | Experimental | Drug: NMD670 |
| Placebo | Placebo comparator | Drug: Placebo |
Who may qualify
- Ages
- 18 to 70 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (5 listed)
- 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
Key exclusion criteria (5 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| NMD670Tablets taken twice daily for 21 days | Drug | NMD670 |
| PlaceboTablets taken twice daily for 21 days | Drug | Placebo |
Primary outcome
Change from baseline to day 21 in 6-minute walk test total distance for NMD670 vs placebo
Time frame: Baseline to day 21
20 secondary outcomes
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
Change from baseline to day 21 in the time to complete the 10MW/RT for NMD670 vs placebo
Time frame: Baseline to day 21
Change from baseline to day 21 in 6-minute walk test fatigue index for NMD670 vs placebo
Time frame: Baseline to day 21
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
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
Change from baseline to day 21 in SF-36 total score and individual domains for NMD670 vs placebo
Time frame: Baseline to day 21
Change from baseline to day 21 in jitter and blocking for NMD670 vs placebo
Time frame: Baseline to day 21
Incidence of treatment emergent adverse events
Time frame: Over 21 days of dosing
Incidence of serious treatment emergent adverse events
Time frame: Over 21 days of dosing
Incidence of clinically significant abnormalities on physical examinations
Time frame: Over 21 days of dosing
Incidence of clinically significant abnormalities on safety laboratory parameters
Time frame: Over 21 days of dosing
Incidence of clinically significant vital signs abnormalities
Time frame: Over 21 days of dosing
Incidence of clinically significant ECG abnormalities
Time frame: Over 21 days of dosing
Incidence of Suicidal Ideation or Suicidal Behavior
Time frame: Over 21 days of dosing
Incidence of clinically significant abnormalities on opthalmological examinations
Time frame: 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
Time frame: Baseline to day 21
Proportion of participants with clinically meaningful change from baseline in 10MW/RT for NMD670 vs placebo
Time frame: Baseline to day 21
Proportion of participants with clinically meaningful change from baseline in ONLS total score for NMD670 vs placebo
Time frame: 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
Time frame: 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
Time frame: Baseline to day 21
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 12 Nov 2025.
- Derived from this study: PubMed 41224659Moss 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.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
19 sites in 5 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing NMD670 in people with Charcot-Marie-Tooth Disease, run by NMD Pharma A/S at 19 sites in 5 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 5 inclusion and 5 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 12 Nov 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 19 sites in United States, France, Belgium, Denmark, Spain. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Change from baseline to day 21 in 6-minute walk test total distance for NMD670 vs placebo (time frame: Baseline to day 21). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT06482437, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 12 Nov 2025.