Status:
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)
Lead Sponsor:
UniQure Biopharma B.V.
Conditions:
Amyotrophic Lateral Sclerosis
Eligibility:
All Genders
18+ years
Phase:
PHASE1
PHASE2
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-162-00...
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...
Eligibility Criteria
Inclusion
- 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
- 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.
Key Trial Info
Start Date :
August 1 2024
Trial Type :
INTERVENTIONAL
Allocation :
ESTIMATED
End Date :
June 30 2031
Estimated Enrollment :
20 Patients enrolled
Trial Details
Trial ID
NCT06100276
Start Date
August 1 2024
End Date
June 30 2031
Last Update
October 22 2025
Active Locations (12)
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1
Barrow Neurological Institute
Phoenix, Arizona, United States, 85013
2
University of California Irvine
Irvine, California, United States, 92697
3
California Pacific Medical Center
San Francisco, California, United States, 94109
4
Mayo Clinic Florida
Jacksonville, Florida, United States, 32224