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Gene Therapy Trial Report

Summary

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


NCTID NCT06100276 (View at clinicaltrials.gov)
Description
Development Status Active
Indication Amyotrophic Lateral Sclerosis (ALS)
Disease Ontology Term DOID:0060193
Compound Name AMT-162
Sponsor UniQure Biopharma B.V.
Funder Type Industry
Recruitment Status
Active not recruiting
Enrollment Count 20 (ESTIMATED)
Results Posted Not Available

Therapy Information


Target Gene/Variant MiSOD1
Therapy Type Gene transfer
Therapy Route In-vivo
Mechanism of Action MiRNA knockdown of mutant/aberrant gene
Route of Administration Intrathecal
Drug Product Type Viral vector
Delivery System Viral transduction
Vector Type AAVrh10
Editor Type none
Dose 1 Undisclosed low dose
Dose 2 Undisclosed intermediate dose
Dose 3 Undisclosed high dose

Study Record Dates


Current Stage Phase1, Phase2
Submit Date 2023-10-20
Completion Date 2031-06-30
Last Update 2025-10-22

Participation Criteria


Eligible Age >=18 Years
Standard Ages Adult, Older adult
Sexes Eligible for Study ALL
Eligibility Criteria
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.
View Inclusion and Exclusion Criteria at ClinicalTrials.gov

Locations


No.of Trial Sites 12
Locations Sweden,United States

Regulatory Information


Has US IND True
FDA Designations Fast Track, Orphan Drug Designation
Recent Updates Dose-limiting toxicity resulted in an SAE during 3Q 2025

Resources/Links