ALS Phase II Study of NX210c (SEALS)

Axoltis Pharma

Status and phase: Enrolling; Phase 2

Conditions: Amyotrophic Lateral Sclerosis

Treatments: Drug: NX210c; Drug: Placebo

Study type: Interventional

Funder types: Industry; Other

Identifiers

NCT06365216

2023-508895-13-00

AXO-CLI-210c-03

Details and patient eligibility

About

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).

Full 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.

Enrollment: 80 estimated patients

Sex: All

Ages: 18+ years old

Volunteers: No Healthy Volunteers

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.

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Double Blind

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80 participants in 3 patient groups, including a placebo group

NX210c 5 mg/kg

Experimental group

Description:

Dose: 5 mg/kg

Treatment:

Drug: NX210c

NX210c 10 mg/kg

Experimental group

Description:

Dose: 10 mg/kg

Treatment:

Drug: NX210c

Placebo

Placebo Comparator group

Treatment:

Drug: Placebo