ALS Phase II Study of NX210c

Last updated: February 4, 2025
Sponsor: Axoltis Pharma
Overall Status: Active - Recruiting

Phase

2

Condition

Amyotrophic Lateral Sclerosis (Als)

Myasthenia Gravis (Chronic Weakness)

Scar Tissue

Treatment

Placebo

NX210c

Clinical Study ID

NCT06365216
AXO-CLI-210c-03
2023-508895-13-00
  • Ages > 18
  • All Genders

Study Summary

This study will investigate the efficacy, safety, tolerability and pharmacokinetics (PK) of multiple intravenous infusions of NX210c, at two dose levels, in patients with Amyotrophic lateral sclerosis (ALS).

Eligibility Criteria

Inclusion

Inclusion Criteria:

  • Aged ≥ 18 years, inclusive at screening.

  • Patients diagnosed as having possible, probable, probable laboratory-supported, ordefinite ALS according to El Escorial Revised criteria.

  • King's Clinical Staging Stage ≤3, disease duration of ≤ 36 calendar months

Exclusion

Exclusion Criteria:

  • Patients with any cognitive or psychological disorder, intellectual disability orother significant impairment that would result in an inability to understand andsign the informed consent.

  • History of any clinically significant or unstable medical, neurological, psychiatriccondition, disorder or disease (other than ALS) or social circumstances that, basedon the investigator's judgment, would interfere with the conduct of the study orpose a risk to the patient if they were to participate.

Study Design

Total Participants: 80
Treatment Group(s): 2
Primary Treatment: Placebo
Phase: 2
Study Start date:
October 25, 2024
Estimated Completion Date:
February 01, 2026

Study Description

Amyotrophic lateral sclerosis (ALS) is a fatal neurodegenerative disease that predominantly affects motor neurons of both the brain and the spinal cord. This leads to muscular atrophy and paralysis, with the majority of patients succumbing to respiratory failure 3-4 years from symptom onset. To date, therapeutic options for ALS are limited and there is no curative treatment. Management of ALS is otherwise supportive and palliative.

There is strong evidence for blood-brain and blood-spinal cord barrier dysfunction in the early stages of ALS. Preclinical in vitro and in vivo data have shown that NX210c exhibits important properties that may be suitable for the treatment of neurological disorders in humans i.e., blood-brain barrier (BBB) integrity restoration, neurotransmission enhancement and neuroprotection.

Based on the safety data collected in a first-in-human single ascending dose study and a multiple ascending dose (MAD) study in healthy elderly subjects, NX210c has been demonstrated to be well tolerated and without safety concerns.

Connect with a study center

  • Centre Hospitalier Universitaire d'Angers

    Angers, 49100
    France

    Active - Recruiting

  • Centre Hospitalier Universitaire De Bordeaux

    Bordeaux, 33000
    France

    Site Not Available

  • CHU de Lyon HCL

    Bron, 69500
    France

    Active - Recruiting

  • Centre Hospitalier Universitaire de Clermont-Ferrand - Hôpital Gabriel Montpied

    Clermont-Ferrand Cedex 1, 63003
    France

    Site Not Available

  • Centre Hospitalier Universitaire de Lille

    Lille, 59037
    France

    Site Not Available

  • Centre Hospitalier Et Universitaire de Limoges

    Limoges Cedex 1, 87042
    France

    Site Not Available

  • Centre Hospitalier Universitaire de Montpellier

    Montpellier Cedex 5, 34295
    France

    Active - Recruiting

  • Centre Hospitalier Universitaire de Nantes - Hôpital Nord Laennec

    Nantes Cedex 01, 44093
    France

    Site Not Available

  • Centre Hospitalier Universitaire de Nice

    Nice, 06000
    France

    Site Not Available

  • Hôpital de la Pitié Salpêtrière

    Paris, 75013
    France

    Active - Recruiting

  • Centre Hospitalier Universitaire de Rennes

    Rennes, 35000
    France

    Site Not Available

  • Centre Hospitalier Regional Universitaire de Tours

    Tours Cedex 9, 37044
    France

    Site Not Available

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