ALS Phase II Study of NX210c

Phase 2

Recruiting

• Conditions: Amyotrophic Lateral Sclerosis
• Interventions: Drug: NX210c; Drug: Placebo

• Registration Number: NCT06365216
• Lead Sponsor: Axoltis Pharma

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).

Detailed 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.

Study Timeline

• Study First Submitted: 2024-04-09
• Study First Submitted QC: 2024-04-12
• Study First Posted: 2024-04-15
• Study Start: 2024-10-25
• Status Verified: 2025-02
• Last Update Submitted: 2025-02-04
• Last Update Posted: 2025-02-05
• Primary Completion: 2026-02-01
• Study Completion: 2026-02-01

Recruitment & Eligibility

• Status: RECRUITING
• Sex: All
• Target Recruitment: 80

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.

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
NX210c 5 mg/kg	NX210c	Dose: 5 mg/kg
NX210c 10 mg/kg	NX210c	Dose: 10 mg/kg
Placebo	Placebo	-

Primary Outcome Measures

Name	Time	Method
The effect of NX210c on blood neurofilament light chain (NfL) or on a blood and cerebrospinal fluid (CSF) biomarker of BBB integrity.	From predose to 6-week follow-up	Changes in serum NfL or changes in Albumin CSF/serum quotient (Qalb)

Trial Locations

Locations (12)

Centre Hospitalier Universitaire d'Angers; 🇫🇷 Angers, France

Centre Hospitalier Universitaire De Bordeaux; 🇫🇷 Bordeaux, France

CHU de Lyon HCL; 🇫🇷 Bron, France

Centre Hospitalier Universitaire de Clermont-Ferrand - Hôpital Gabriel Montpied; 🇫🇷 Clermont-Ferrand Cedex 1, France

Centre Hospitalier Universitaire de Lille; 🇫🇷 Lille, France

Centre Hospitalier Et Universitaire de Limoges; 🇫🇷 Limoges Cedex 1, France

Centre Hospitalier Universitaire de Montpellier; 🇫🇷 Montpellier Cedex 5, France

Centre Hospitalier Universitaire de Nantes - Hôpital Nord Laennec; 🇫🇷 Nantes Cedex 01, France

Centre Hospitalier Universitaire de Nice; 🇫🇷 Nice, France

Hôpital de la Pitié Salpêtrière; 🇫🇷 Paris, France

Centre Hospitalier Universitaire de Rennes; 🇫🇷 Rennes, France

Centre Hospitalier Regional Universitaire de Tours; 🇫🇷 Tours Cedex 9, France