NCT06365216

ALS Phase II Study of NX210c

A Double-blind, Randomized, Placebo-controlled, Multicentric, Phase II Study in Adult Patients With Amyotrophic Lateral Sclerosis (ALS) to Assess Efficacy, Safety, Tolerability and Pharmacokinetics of Multiple Intravenous Infusions of NX210c

• Status: Active Not Recruiting
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 80 (estimated)
• Sponsor: Axoltis Pharma · Industry
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

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.

Conditions

• Amyotrophic Lateral Sclerosis

Interventions

Type	Name	Description
DRUG	NX210c	3 times a week, for 4 weeks
DRUG	Placebo	3 times a week, for 4 weeks

Timeline

• Start date: 2024-10-25
• Primary completion: 2026-02-01
• Completion: 2026-09-30
• First posted: 2024-04-15
• Last updated: 2025-12-24

Locations

16 sites across 1 country: France