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ALS Phase II Study of NX210c

NCT ID: NCT06365216

Last Updated: 2025-12-24

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

ACTIVE_NOT_RECRUITING

Clinical Phase

PHASE2

Total Enrollment

80 participants

Study Classification

INTERVENTIONAL

Study Start Date

2024-10-25

Study Completion Date

2026-09-30

Brief Summary

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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).

Detailed Description

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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.

Conditions

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Amyotrophic Lateral Sclerosis

Keywords

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ALS

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Double-blind, randomized, placebo-controlled, multicentric, phase II study in adult patients with ALS to assess efficacy, safety, tolerability and pharmacokinetics of multiple intravenous infusions of NX210c.

Two doses of NX210c (5 mg/kg and 10 mg/Kg) will be investigated, along with a placebo group which will serve as a reference. Patients will be randomized to one of 3 arms in a 3:3:2 allocation ratio.

Patients will participate up to approximately 44 weeks: up to 30 days for screening, 26 days of treatment and 9 months of follow-up (3-month initial follow-up and 6-month extension). Lumbar punctures will assess CSF NfL and other key CSF biomarkers related to ALS and drug mechanism of action. Blood sampling will be drawn for safety, PK and biomarkers testing. Urine will be collected for biomarkers. A comprehensive assessment of the disease, e.g., the ALSFRS-R will be performed. Additional follow-up extension will be offered to all patients that have/are participating to the study.
Primary Study Purpose

TREATMENT

Blinding Strategy

DOUBLE

Participants Investigators

Study Groups

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NX210c 5 mg/kg

Dose: 5 mg/kg

Group Type EXPERIMENTAL

NX210c

Intervention Type DRUG

3 times a week, for 4 weeks

NX210c 10 mg/kg

Dose: 10 mg/kg

Group Type EXPERIMENTAL

NX210c

Intervention Type DRUG

3 times a week, for 4 weeks

Placebo

Group Type PLACEBO_COMPARATOR

Placebo

Intervention Type DRUG

3 times a week, for 4 weeks

Interventions

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NX210c

3 times a week, for 4 weeks

Intervention Type DRUG

Placebo

3 times a week, for 4 weeks

Intervention Type DRUG

Eligibility Criteria

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Inclusion Criteria

* Aged ≥ 18 years, inclusive at screening.
* Patients diagnosed as having possible, probable, probable laboratory-supported, or definite ALS according to El Escorial Revised criteria.
* King's Clinical Staging Stage ≤3, disease duration of ≤ 36 calendar months

Exclusion Criteria

* Patients with any cognitive or psychological disorder, intellectual disability or other significant impairment that would result in an inability to understand and sign the informed consent.
* History of any clinically significant or unstable medical, neurological, psychiatric condition, disorder or disease (other than ALS) or social circumstances that, based on the investigator's judgment, would interfere with the conduct of the study or pose a risk to the patient if they were to participate.
Minimum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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ACT4ALS network

UNKNOWN

Sponsor Role collaborator

Axoltis Pharma

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Locations

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Centre Hospitalier Regional Et Universitaire De Brest

Brest, France, France

Site Status

Centre Hospitalier Universitaire De Caen Normandie

Caen, France, France

Site Status

Hôpital La Timone - APHM

Marseille, France, France

Site Status

CHRU De Nancy

Nancy, France, France

Site Status

Centre Hospitalier Universitaire d'Angers

Angers, , France

Site Status

Centre Hospitalier Universitaire De Bordeaux

Bordeaux, , France

Site Status

CHU de Lyon HCL

Bron, , France

Site Status

Centre Hospitalier Universitaire de Clermont-Ferrand - Hôpital Gabriel Montpied

Clermont-Ferrand, , France

Site Status

Centre Hospitalier Universitaire de Lille

Lille, , France

Site Status

Centre Hospitalier Et Universitaire de Limoges

Limoges, , France

Site Status

Centre Hospitalier Universitaire de Montpellier

Montpellier, , France

Site Status

Centre Hospitalier Universitaire de Nantes - Hôpital Nord Laennec

Nantes, , France

Site Status

Centre Hospitalier Universitaire de Nice

Nice, , France

Site Status

Hôpital de la Pitié Salpêtrière

Paris, , France

Site Status

Centre Hospitalier Universitaire de Rennes

Rennes, , France

Site Status

Centre Hospitalier Regional Universitaire de Tours

Tours, , France

Site Status

Countries

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France

Other Identifiers

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2023-508895-13-00

Identifier Type: CTIS

Identifier Source: secondary_id

AXO-CLI-210c-03

Identifier Type: -

Identifier Source: org_study_id