ACTIVE_NOT_RECRUITING

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

Conditions

Amyotrophic Lateral Sclerosis Gene Therapy AAV (adeno-associated virus)ALS SOD1

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Study Overview

This clinical trial focuses on testing the efficacy of different digital interventions to promote re-engagement in cancer-related long-term follow-up care for adolescent and young adult (AYA) survivors of childhood cancer.

Description

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.

Official Title

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).

Quick Facts

Study Start:2024-08-01

Study Completion:2031-06-30

Study Type:Not specified

Phase:Not Applicable

Enrollment:Not specified

Status:ACTIVE_NOT_RECRUITING

Study ID

NCT06100276

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Ages Eligible for Study:18 Years

Sexes Eligible for Study:ALL

Accepts Healthy Volunteers:No

Standard Ages:ADULT, OLDER_ADULT

Inclusion Criteria	Exclusion 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.	* 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.

Inclusion Criteria

Exclusion 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.

* 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.

Contacts and Locations

Principal Investigator

Executive Director, Clinical Development

STUDY_DIRECTOR

UniQure Biopharma B.V.

Study Locations (Sites)

Barrow Neurological Institute

Phoenix, Arizona, 85013

United States

University of California Irvine

Irvine, California, 92697

United States

California Pacific Medical Center

San Francisco, California, 94109

United States

Mayo Clinic Florida

Jacksonville, Florida, 32224

United States

Winship Cancer Institute of Emory University

Atlanta, Georgia, 30322

United States

Northwestern University Feinberg School of Medicine

Chicago, Illinois, 60611

United States

University of Kansas Medical Center

Fairway, Kansas, 66205

United States

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

Boston, Massachusetts, 02114

United States

Mayo Clinic Rochester

Rochester, Minnesota, 55905

United States

Columbia University Irving Medical Center

New York, New York, 10032

United States

University of Pennsylvania School of Medicine

Philadelphia, Pennsylvania, 19104

United States

Collaborators and Investigators

Sponsor: UniQure Biopharma B.V.

Executive Director, Clinical Development, STUDY_DIRECTOR, UniQure Biopharma B.V.

Study Record Dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Registration Dates

Study Start Date2024-08-01

Study Completion Date2031-06-30

Study Record Updates

Study Start Date2024-08-01

Study Completion Date2031-06-30

Terms related to this study

Keywords Provided by Researchers

Gene Therapy
AAV (adeno-associated virus)
ALS
SOD1

Additional Relevant MeSH Terms

Amyotrophic Lateral Sclerosis