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Clinical Trials in the USA / NCT06100276
Active, not recruiting Phase 1/2

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

NCT06100276 · tracked via the Priya Life Science USA tracker
Sponsor
UniQure Biopharma B.V.
Phase
Phase 1/2
Started
2024-08-01
Last updated
2025-10-22

Condition(s) studied

Amyotrophic Lateral Sclerosis

Investigational drug(s) / intervention(s)

AMT-162

AMT-162: AMT-162, the investigational product (IP), is a nonreplicating, rep/cap-deleted, self-complementary Recombinant adeno-associated virus (rAAV) vector based on adeno-associated virus (AAV) serotype rh10 and contains complementary deoxyribonucleic acid (cDNA) encoding an artificial miRNA targeting the SOD1 gene.

Study 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.

Eligibility

Sex
ALL
Min age
18 Years
Max age
Healthy volunteers
No
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.

Primary outcome measure(s)

Trial sites (12)

FacilityCityRegionStatus
Barrow Neurological Institute Phoenix Arizona
University of California Irvine Irvine California
California Pacific Medical Center San Francisco California
Mayo Clinic Florida Jacksonville Florida
Winship Cancer Institute of Emory University Atlanta Georgia
Northwestern University Feinberg School of Medicine Chicago Illinois
University of Kansas Medical Center Fairway Kansas
Massachusetts General Hospital, Sean M. Healey and AMG Center for ALS Research Boston Massachusetts
Mayo Clinic Rochester Rochester Minnesota
Columbia University Irving Medical Center New York New York
University of Pennsylvania School of Medicine Philadelphia Pennsylvania
Norrlands Universitetssjukhus Umeå Vasterbottens Ian

More UniQure Biopharma B.V. trials in the USA

Other trials for the same condition

Official registry record

This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.

View NCT06100276 on ClinicalTrials.gov ↗ ← All trials in the USA