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New drug shows promise for nerve disease that affects walking

NCT ID NCT06482437

Symptom relief Sponsor: NMD Pharma A/S Source: ClinicalTrials.gov ↗

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Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing
Running, but no longer taking on new participants.
Completed This study
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Jun 27, 2026 · Last updated Jun 27, 2026

Trial info Results

Early CMT pill trial shows mixed but encouraging signs

This study tested ignaseclant, an experimental pill for adults with Charcot-Marie-Tooth disease (CMT). The trial did not improve the main walking test, but it did show encouraging signs in other areas like hand strength, fine motor function, and patient-reported symptoms. In simple terms: the medicine did not open the front door, but it may have found a side door worth exploring. The drug was also reported to be safe and well tolerated over the short study period, with no serious side effects reported in the treatment group.

Based on sponsor-reported topline results

This was a Phase 2a, randomized, double-blind, placebo-controlled study of ignaseclant in adults with genetically confirmed CMT type 1 or type 2. Patients took the study drug or placebo twice a day for 21 days, with follow-up at day 28. NMD Pharma’s press release says the study enrolled 81 adults, while the conference poster reports 80 people were randomized, so “about 80 adults” is the most careful way to describe it publicly.

The main goal was to see whether people could do better on the 6-minute walk test after treatment. That main goal was not met. But the story did not end there. The late-breaking conference poster reported improvement trends in several other measures, including the CMT Functional Outcome Measure, handgrip strength, fine hand function, and patient-reported health impact. For example, the poster says more patients on ignaseclant were responders on the CMT-Health Index at day 21 than patients on placebo, and handgrip strength reached statistical significance versus placebo at day 21 and day 28. It is a bit like a runner who does not win the race, but still shows stronger legs, steadier hands, and better balance along the way.

Safety looked encouraging in this short study. According to the poster, treatment-emergent side effects were more common with ignaseclant than placebo, but they were mild to moderate, did not lead to treatment discontinuation, and no serious adverse events were seen in the ignaseclant group. The sponsor’s press release also described the drug as safe and well tolerated.

So what do these results mean for people with CMT? The clearest answer is: mixed, but promising enough to keep going. The study missed its main walking endpoint, so this was not a clean win. But the secondary and exploratory findings suggest the drug may still be helping in ways that matter to daily life, especially in hand strength and function. That makes this result feel less like a dead end and more like a trail marker pointing researchers toward a longer and better-designed next study.

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 2

Tests whether the treatment actually works, and watches for side effects, in a larger group.

Participants

81 people

The number who actually took part.

Started

Sep 2024

Finished

Nov 2025

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

18 to 70 years

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

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.

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Conditions

The condition(s) this trial relates to.

Charcot-Marie-Tooth disease Charcot-Marie-Tooth disease type 1 Charcot-Marie-Tooth disease type 2

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Charcot-Marie-Tooth disease CMT

Contacts and locations

Locations

  • Aarhus University Hospital

    Aarhus, Denmark

  • Bicêtre University Hospital

    Paris, 94275, France

  • CHR de la Citadelle- Site Citadelle Neurolgie Boulevard du 12eme de Ligne 1

    Liège, 4000, Belgium

  • CHU Marseille, Reference centre for neuromuscular diseases and ALS Department of Neuromuscular Diseases

    Marseille, 13005, France

  • Centre Hospitalier Universitaire De Nice

    Nice, 06001, France

  • Columbia University Medical Center

    New York, New York, 10032, United States

  • Hospital Universitari Vall d'Hebron

    Barcelona, 8035, Spain

  • Hospital Universitari i Politecnic La Fe de Valencia

    Valencia, 46026, Spain

  • Institut de Myologie Groupe Hospitalier Pitié-Salpêtrière

    Paris, 75651, France

  • Laboratoire d'Explorations Fonctionnelles, CHU Nantes

    Nantes, France

  • Mass General Neurology

    Boston, Massachusetts, 02114, United States

  • National Neuromuscular research Institute, PLLC

    Austin, Texas, 78759, United States

  • NextGen Precision Health

    Columbia, Missouri, 65211, United States

  • OSU Department of Neurology Division of Neuromuscular Diseases

    Columbus, Ohio, 43221, United States

  • Providence Medical Research Center

    Spokane, Washington, 99204, United States

  • Rigshospitalet, Department of Neurology

    Copenhagen, Denmark

  • University Hospitals Leuven, Department of Neurology

    Leuven, 3000, Belgium

  • University of Kansas Medical Center, Department of Neurology

    Kansas City, Kansas, 66160, United States

  • University of Rochester Neuromuscular Disease Center

    Rochester, New York, 14642, United States

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