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New drug tested for rare nerve disease that weakens muscles

NCT ID NCT06482437

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

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.

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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

Conditions

Explore the condition pages connected to this study.

CHARCOT MARIE TOOTH DISEASE CMT