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Not recruiting
Phase 1
Phase 2

AMT-162

Sponsor:

UniQure Biopharma B.V.

Code:

NCT06100276

Conditions

Amyotrophic Lateral Sclerosis

Eligibility Criteria

Sex: All

Age: 18+

Healthy Volunteers: Not accepted

Interventions

AMT-162

Study Details

Brief 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.

Conditions

Amyotrophic Lateral Sclerosis

Study ID

NCT06100276

Start date

Aug 1, 2024

Status verified date

Sep, 2024

Completion date

Mar 30, 2031

Anticipated

Primary completion date

Sep 30, 2026

Anticipated

Eligibility Criteria

Eligibility Criteria

Sex: All

Age: 18+

Healthy Volunteers: Not accepted

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.

Study Design

Enrollment

20 participants

Anticipated

Allocation

Non randomized

Intervention Model

Sequential

Primary purpose

Treatment

Interventions and Outcome Measures

Arms

experimental: 3 single Ascending Dose Levels

Experimental: 3 single Ascending Dose Levels

The study will be open-label with an initial plan to explore 3 dose levels of AMT-162 in approximately 6 to 12 Participants in total. Each Participant will receive a single dose of AMT-162 delivered via an intrathecal (IT) infusion and will be followed for up to 5 years after AMT-162 administration.

experimental: EXPANSION COHORT

Expansion cohort: To further test selected dose from the SAD part in approximately 6 to 8 participants

The study will be open-label. Each Participant will receive a single dose of AMT-162 delivered via an intrathecal (IT) infusion and will be followed for up to 5 years after AMT-162 administration.

Interventions

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.

Primary outcome measure

  • To evaluate the safety and tolerability of ascending doses of intrathecally administered AMT-162 in Participants with SOD1-ALS [ Time Frame: up to 5 years ]

Central Contacts and Locations

Central contacts

Director Clinical Operations

1-866-520-1257medinfo@uniqure.com

Locations

University of California Irvine

Recruiting

Irvine, California, United States, 92697

Contacts

Genisis Lopez-Martinez

714-456-8437galopezm@hs.uci.edu

Mihaela Nistor

(714) 456-5956mnistor@hs.uci.edu

Principal Investigator:

Namita Goyal, MD

California Pacific Medical Center

Recruiting

San Francisco, California, United States, 94109

Contacts

Daniela Sanchez

415-600-1764daniela.sanchez@sutterhealth.org

Mackenzie Kenneally

415-600-0484mackenzie.kenneally@sutterhealth.org

Principal Investigator:

Jonathan S Katz, MD

Mayo Clinic Florida

Recruiting

Jacksonville, Florida, United States, 32224

Contacts

Jany Dagher

904-956-3730Dagher.Jany@mayo.edu

Principal Investigator:

Björn Oskarsson, MD

Winship Cancer Institute of Emory University

Recruiting

Atlanta, Georgia, United States, 30322

Contacts

Wanda Sanchez

404-727-1273wanda.sanchez@emory.edu

Principal Investigator:

Jonathan Glass, ND

University of Kansas Medical Center

Recruiting

Fairway, Kansas, United States, 66205

Contacts

Katie Lillig

913-945-9932Kjennens2@kumc.edu

Principal Investigator:

Omar Jawdat, MD

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

Recruiting

Boston, Massachusetts, United States, 02114

Contacts

Alison Wheeler

617-643-8449AWHEELER7@mgh.harvard.edu

Principal Investigator:

James Berry, MD

Mayo Clinic Rochester

Recruiting

Rochester, Minnesota, United States, 55905

Contacts

Jeff Laivell

507-538-8095Laivell.Jeffrey@mayo.edu

Principal Investigator:

Nathan Staff, MD, PhD

Columbia University Irving Medical Center

Recruiting

New York, New York, United States, 10032

Contacts

Helen Mejía Santana, MA

212-305-9183hm28@cumc.columbia.edu

Principal Investigator:

Matthew Harms, MD

University of Pennsylvania School of Medicine

Recruiting

Philadelphia, Pennsylvania, United States, 19104

Contacts

Stephanie Hansen, MD

267-438-7518stephanie.hansen@pennmedicine.upenn.edu

Abigail Browngoehl

267-343-6395abigail.browngoehl@pennmedicine.upenn.edu

Principal Investigator:

Colin Quinn, MD

More Information

Sponsor

UniQure Biopharma B.V.

Last update posted

Sep 8, 2025

Last verified

Sep, 2024

Keywords

  • Gene Therapy
  • AAV (adeno-associated virus)
  • ALS
  • SOD1

Trial information was received from ClinicalTrials.gov and was last updated on 2026-09-29. This information was provided to ClinicalTrials.gov by UniQure Biopharma B.V. on 2025-09-08. Recruitment status is synced daily from ClinicalTrials.gov and may not reflect the sponsor's current status. Confirm during your call.