ALS Phase II Study of NX210c

NCT06365216 | Active Not Recruiting Phase 2

• 2 other identifiers: AXO-CLI-210c-032023-508895-13-00
• Study Type: interventional
• Target: 80
• Locations: 1 country
• Sites: 16

Brief 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).

Conditions

Amyotrophic Lateral Sclerosis

Keywords

ALS

Outcome Measures

Primary Outcomes (1)

• The effect of NX210c on blood neurofilament light chain (NfL) or on a blood and cerebrospinal fluid (CSF) biomarker of BBB integrity.

  Changes in serum NfL or changes in Albumin CSF/serum quotient (Qalb)

  From predose to 6-week follow-up

Study Arms (3)

NX210c 5 mg/kg

EXPERIMENTAL

Dose: 5 mg/kg

Drug: NX210c

NX210c 10 mg/kg

EXPERIMENTAL

Dose: 10 mg/kg

Drug: NX210c

Placebo

PLACEBO COMPARATOR

Drug: Placebo

Interventions

NX210c DRUG

3 times a week, for 4 weeks

NX210c 10 mg/kg; NX210c 5 mg/kg

Placebo DRUG

3 times a week, for 4 weeks

Placebo

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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.

Sponsors & Collaborators

• Axoltis Pharma: lead
• ACT4ALS network: collaborator

Study Sites (16)

Centre Hospitalier Regional Et Universitaire De Brest

Brest, France, 29200, France

Location

Centre Hospitalier Universitaire De Caen Normandie

Caen, France, 14033, France

Location

Hôpital La Timone - APHM

Marseille, France, 13005, France

Location

CHRU De Nancy

Nancy, France, 54000, France

Location

Centre Hospitalier Universitaire d'Angers

Angers, 49100, France

Location

Centre Hospitalier Universitaire De Bordeaux

Bordeaux, 33000, France

Location

CHU de Lyon HCL

Bron, 69500, France

Location

Centre Hospitalier Universitaire de Clermont-Ferrand - Hôpital Gabriel Montpied

Clermont-Ferrand, 63003, France

Location

Centre Hospitalier Universitaire de Lille

Lille, 59037, France

Location

Centre Hospitalier Et Universitaire de Limoges

Limoges, 87042, France

Location

Centre Hospitalier Universitaire de Montpellier

Montpellier, 34295, France

Location

Centre Hospitalier Universitaire de Nantes - Hôpital Nord Laennec

Nantes, 44093, France

Location

Centre Hospitalier Universitaire de Nice

Nice, 06000, France

Location

Hôpital de la Pitié Salpêtrière

Paris, 75013, France

Location

Centre Hospitalier Universitaire de Rennes

Rennes, 35000, France

Location

Centre Hospitalier Regional Universitaire de Tours

Tours, 37044, France

Location

MeSH Terms

Conditions

Amyotrophic Lateral Sclerosis

Condition Hierarchy (Ancestors)

Spinal Cord Diseases; Central Nervous System Diseases; Nervous System Diseases; Motor Neuron Disease; Neurodegenerative Diseases; TDP-43 Proteinopathies; Neuromuscular Diseases; Proteostasis Deficiencies; Metabolic Diseases; Nutritional and Metabolic Diseases

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: DOUBLE
• Who Masked: PARTICIPANT, INVESTIGATOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Model Details: 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.

Study Record Dates

• First Submitted: April 9, 2024
• First Posted: April 15, 2024
• Study Start: October 25, 2024
• Primary Completion: February 1, 2026
• Study Completion (Estimated): September 30, 2026
• Last Updated: December 24, 2025

Record last verified: 2025-11

Data Sharing

• IPD Sharing: Will not share

Locations

FR (16)