ALS Phase II Study of NX210c | NCT06365216

Studies
ALS Phase II Study of NX210c

NCT06365216

Sponsor

Axoltis Pharma

Status

Active, not recruiting

Last Update Posted

Dec 24, 2025Actual

Enrollment

80Estimated

Phase

Phase 2

Conditions

Amyotrophic Lateral Sclerosis

Interventions

NX210c
Placebo

Countries

France

Protocol Section

Identification Module Status Module Sponsor/Collaborators Module Oversight Module Description Module Conditions Module Design Module Arms/Interventions Module Outcomes Module Eligibility Module Contacts/Locations Module References Module IPD Sharing Statement Module

Results Section

Participant Flow Module Baseline Characteristics Module Outcome Measures Module Adverse Events Module More Info Module

Annotation Section

Annotation Module

Document Section

Large Document Module

Derived Section

Miscellaneous Info Module Condition Browse Module Intervention Browse Module

Protocol Section

Identification Module

NCT ID

Results Section

No data available

No data is available for this block.

Annotation Section

No data available

No data is available for this block.

Document Section

No data available

No data is available for this block.

Derived Section

Miscellaneous Info Module

Version Holder

ID	Type	Description	Link
2023-508895-13-00	EU Trial (CTIS) Number

Status Module

Record Verification Date

Nov 2025

Overall Recruitment Status or Expanded Access Status

Active, not recruiting

Last Known Status

Not provided

Delayed Posting

Not provided

Why Stopped

Not provided

Expanded Access Info

Start Date

Oct 25, 2024Actual

Primary Completion Date

Feb 1, 2026Estimated

Completion Date

Sep 30, 2026Estimated

First Submitted Date

Apr 9, 2024

First Submission Date that Met QC Criteria

Apr 12, 2024

First Posted Date

Apr 15, 2024Actual

Results Waived

Not provided

Results First Submitted Date

Not provided

Results First Submitted that Met QC Criteria

Not provided

Results First Posted Date

Not provided

Certification/Extension (aka Delayed Results) First Submitted Date

Not provided

Certification/Extension First Submitted that Passed QC Review

Not provided

Certification/Extension First Posted Date

Not provided

Last Update Submitted Date

Dec 18, 2025

Last Update Posted Date

Dec 24, 2025Actual

Name	Class
ACT4ALS network	UNKNOWN

Oversight Module

Has Data Monitoring Committee (DMC)

Is FDA Regulated Drug

Is FDA Regulated Device

Is Unapproved Device

Not provided

Pediatric Postmarket Surveillance of a Device Product

Not provided

Product Exported from US

Not provided

FDAAA801 Violation

Not provided

Description Module

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.

Design Module

Study Type

Interventional

Number of References to an Expanded Access Study

Not provided

Expanded Access Types

Not provided

Patient Registry

Not provided

Target Follow-Up Duration

Not provided

Phases

Phase 2

Interventional Study Design

Allocation

Biospecimen

No data available

No data is available for this block.

Enrollment

80Estimated

Arms/Interventions Module

Arm Groups

Label	Type	Description	Intervention Names
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

Name	Type	Description	Arm Group Labels	Other Names
NX210c	Drug	3 times a week, for 4 weeks	NX210c 10 mg/kg NX210c 5 mg/kg

Outcomes Module

Primary Outcomes

Measure	Description	Time Frame
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

Secondary Outcomes

Not provided

Other Outcomes

Not provided

Eligibility Module

Eligibility Criteria

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.

Accepts Healthy Volunteers

Sex

All

Sex/Gender Based

Not provided

Sex/Gender Description

Not provided

Minimum Age

18 Years

Maximum Age

Not provided

Standard Ages

AdultOlder Adult

Study Population

Not provided

Sampling Method

Not provided

Contacts/Locations Module

Central Contacts

Not provided

Overall Officials

Not provided

Locations

Facility	Status	City	State	ZIP	Country	Contacts
Centre Hospitalier Regional Et Universitaire De Brest		Brest	France	29200	France
Centre Hospitalier Universitaire De Caen Normandie

IPD Sharing Statement Module

Plan to Share IPD

Description

Anonymized subject data to be shared based on reasonable request

Types

Not provided

Time Frame

Not provided

Access Criteria

Not provided

URL

Not provided

Condition Browse Module

MeSH Terms

ID	Term
D000690	Amyotrophic Lateral Sclerosis

Ancestor Terms

ID	Term
D013118	Spinal Cord Diseases
D002493	Central Nervous System Diseases
D009422	Nervous System Diseases
D016472	Motor Neuron Disease

Browse Leaves

Not provided

Browse Branches

Not provided

Intervention Model Description

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.