ALS Phase II Study of NX210c

Amyotrophic Lateral Sclerosis Clinical Trial

— SEALS  

Official title:

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

Clinical Trial Details — Status: Not yet recruiting

Administrative data

• NCT number: NCT06365216
• Other study ID #: AXO-CLI-210c-03
• Secondary ID: 2023-508895-13-0
• Status: Not yet recruiting
• Phase: Phase 2
• Start date: September 1, 2024
• Est. completion date: February 1, 2026

Study information

• Verified date: June 2024
• Source: Axoltis Pharma
• Contact: Sébastien Marie
• Phone: 0033 (0)4 72 62 17 36
• Email: contact[@]axoltis.com
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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).

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.

Recruitment information / eligibility

• Status: Not yet recruiting
• Enrollment: 80
• Est. completion date: February 1, 2026
• Est. primary completion date: February 1, 2026
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years and older

Eligibility

Inclusion Criteria:

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

• Aged = 18 years, inclusive at screening.

• King's Clinical Staging Stage =3, disease duration of = 36 calendar months and estimated life expectancy > 6 months at baseline.

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.

Related Conditions & MeSH terms

• Amyotrophic Lateral Sclerosis
• Motor Neuron Disease
• Sclerosis

Intervention

Drug:
• NX210c
3 times a week, for 4 weeks
• Placebo
3 times a week, for 4 weeks

Locations

Country	Name	City	State
France	CHU de Lyon HCL	Bron

Sponsors (2)

Lead Sponsor	Collaborator
Axoltis Pharma	ACT4ALS network

Country where clinical trial is conducted

France, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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 2-month follow-up