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Safety, Tolerability, and Exploratory Efficacy Study of Intrathecally Administered Gene Therapy AMT-162 in Adult Participants With SOD1 Amyotrophic Lateral Sclerosis (SOD1-ALS) | Clinical Trials | Clareo Health

ACTIVE_NOT_RECRUITINGPHASE1/PHASE2

Safety, Tolerability, and Exploratory Efficacy Study of Intrathecally Administered Gene Therapy AMT-162 in Adult Participants With SOD1 Amyotrophic Lateral Sclerosis (SOD1-ALS)

A Phase 1/2, Multicenter, Single Ascending Dose Study to Evaluate the Safety, Tolerability, and Exploratory Efficacy of Intrathecally Administered Gene Therapy AMT-162 in Adult Participants With SOD1 Amyotrophic Lateral Sclerosis (SOD1-ALS).

NCT06100276•UniQure Biopharma B.V.

View on ClinicalTrials.gov

Summary

This is the study of AMT-162 in Participants with SOD1-ALS and is designed to evaluate the safety, tolerability, and exploratory efficacy of intrathecally administered gene therapy AMT-162. AMT-162-001 is a Phase 1/2, multi-center, single ascending dose study.

Detailed Description

AMT-162 is an investigational gene therapy that encodes an artificial microribonucleic acid (microRNA or miRNA) targeting the SOD1 gene. This clinical study will test the safety of AMT-162 and explore the hypothesis that it will silence expression of mutant cytosolic SOD1 and thereby ameliorate the course of ALS caused by this mutant gene.

Eligibility

Age

18 - No limit years

Sex

ALL

Healthy Volunteers

Inclusion Criteria

•Confirmed clinical and genetic diagnosis of SOD1-mediated ALS (SOD1-ALS) experiencing signs and/or symptoms of lower motor neuron dysfunction (weakness, atrophy, cramps, fasciculations), with or without upper motor neuron symptoms (weakness, bring reflexes, spasticity).
•ALSFRS-R score ≥ 25 at Screening.
•Slow vital capacity (SVC) ≥50% of predicted normal value.
•Capable of providing informed consent and complying with trial procedures, including: medically able to undergo lumbar puncture and has a responsible caregiver able to attend all clinic visit with the Participant.

Exclusion Criteria

•SOD1 pathogenic or likely pathogenic variants in amino acid regions 43-47.
•Pathogenic repeat expansion in the C9orf72 gene
•Any of the following prior or concomitant treatments:
•* Any prior SOD1 suppression therapy with viral microRNA mediators
•* Prior SOD suppression therapy with antisense oligonucleotide (ASO) mediators such as tofersen (QALSODY™). Exception: Patients who previously received tofersen may be enrolled if the last dose of tofersen was received at least 20 weeks prior to the first Screening assessment and if there were no previous tofersen-related SAEs or ongoing tofersen-related adverse events that would increase the risk of receiving AMT-162, per Investigator judgment.
•* Other ALS medications riluzole (RILUTEK®, TIGLUTIK®), edaravone (RADICAVA®), and sodium phenylbutyrate and taururosdiol combination (RELYVRIO) or bioequivalents are allowed if dose is stable for 30 days prior to immunosuppression.
•* Any prior administration of an AAV gene therapy.
•Participants must be willing to forego new ALS treatments through at least 6 months after infusion of AMT-162. After 6 months, Investigators and participants may decide to add new ALS medications or change existing ALS medications.

Locations (12)

Barrow Neurological Institute

Phoenix, Arizona, United States

University of California Irvine

Irvine, California, United States

California Pacific Medical Center

San Francisco, California, United States

Mayo Clinic Florida

Jacksonville, Florida, United States

Winship Cancer Institute of Emory University

Atlanta, Georgia, United States

Northwestern University Feinberg School of Medicine

Chicago, Illinois, United States

University of Kansas Medical Center

Fairway, Kansas, United States

Massachusetts General Hospital, Sean M. Healey and AMG Center for ALS Research

Boston, Massachusetts, United States

Mayo Clinic Rochester

Rochester, Minnesota, United States

Columbia University Irving Medical Center

New York, New York, United States

Key Dates

Start Date

August 1, 2024

Primary Completion Date

September 30, 2026

Completion Date

June 30, 2031

Last Updated

October 22, 2025

Enrollment

ESTIMATED participants

Conditions

Amyotrophic Lateral Sclerosis

Interventions

AMT-162

DRUG

Pridopidine Phase 3 Study to Evaluate Efficacy and Safety in ALS

NCT07322003

Amyotrophic Lateral Sclerosis

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RECRUITING

Intermuscular Coherence as a Biomarker for ALS

NCT05104710

Amyotrophic Lateral Sclerosis

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RECRUITINGNA

Connect-One: Early Feasibility Study of Connexus® Brain-Computer Interface (BCI)

NCT07357428

Data Source & Attribution

This clinical trial information is sourced from ClinicalTrials.gov, a service of the U.S. National Institutes of Health.

ClinicalTrials.gov last update: October 22, 2025Data synced to Clareo: March 29, 2026

Modifications: This data has been reformatted for display purposes. Eligibility criteria have been parsed into inclusion/exclusion sections. Location data has been geocoded to enable distance-based search. For the authoritative and most current information, please visit ClinicalTrials.gov.

Neither the United States Government nor Clareo Health make any warranties regarding the data. Check ClinicalTrials.gov frequently for updates.

View ClinicalTrials.gov Terms and Conditions

Safety, Tolerability, and Exploratory Efficacy Study of Intrathecally Administered Gene Therapy AMT-162 in Adult Participants With SOD1 Amyotrophic Lateral Sclerosis (SOD1-ALS)

Inclusion Criteria

Exclusion Criteria

Pridopidine Phase 3 Study to Evaluate Efficacy and Safety in ALS

Intermuscular Coherence as a Biomarker for ALS

Connect-One: Early Feasibility Study of Connexus® Brain-Computer Interface (BCI)

UPenn Observational Research Repository on Neurodegenerative Disease

Control Cohort CTRL COH

ASSESS ALL ALS Study