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

StatusActive, Not Recruiting
PhasePhase 1/2
Started2024-08-01
View on ClinicalTrials.gov ↗
Record status
This record was last updated October 22, 2025 (before its estimated September 30, 2026 completion). Its status may not reflect the trial's current state.

Amendment history

2025-10-20
Recruiting→Active, Not Recruiting Study Status, Contacts/Locations v12
Trial statusRecruiting→Active, Not Recruiting
Completion date2031-03-30→2031-06-30
Study sites10→12
2025-09-01
Study Identification, Study Status, Contacts/Locations v11
Study sitesdetails revised at 10 of 10 sites
Study titlerevised
[...] lly Administered Gene Therapy AMT-162 in Adult Participants withWith SOD1 Amyotrophic Lateral Sclerosis (SOD1-ALS)
2024-10-08
Study Status, Contacts/Locations v10
Study sitesdetails revised at 3 of 10 sites
2024-09-18
Study Status, Eligibility, Contacts/Locations v9
Study sites6→10
Eligibility criteria+232 characters
[...] uppression. 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.
2024-08-01
Not Yet Recruiting→Recruiting Study Identification, Study Status, Oversight, Contacts/Locations, IPDSharing v8
Trial statusNot Yet Recruiting→Recruiting
Start dateestimated 2024-07-30→confirmed 2024-08-01
Study sitesdetails revised at 6 of 6 sites
Study titlerevised
[...] lly Administered Gene Therapy AMT-162 in Adult Participants Withwith SOD1 Amyotrophic Lateral Sclerosis (SOD1-ALS)
Show 7 earlier versions
2024-07-19
Study Status, Study Design, Arms and Interventions, Contacts/Locations v7
Study arms1→2
2024-07-05
Study Status, Study Design, Arms and Interventions v6
Study arms2→1
2024-07-03
Study Status, Study Design, Arms and Interventions, Eligibility, Contacts/Locations v5
Start date2024-05-30→2024-07-30
Enrollment target12→20
Study arms1→2
Eligibility criteria+61 characters
[...] LSFRS-R score ≥ 25 at Screening. Slow vital capacity (SVC) ≥6550% of predicted normal value. Capable of providing informed c [...] ide (ASO) mediators such as tofersen (QALSODY™). Exception: PatientPatients who previously received tofersen may be enrolled if the las [...] g assessment and if there were no previous tofersen-related serious adverse eventsSAEs or ongoing tofersen-related adverse events that would increase the risk of receiving AMT-162, per Investigator judgment. Other ALS medications riluzole (RILUTEK®, TIGLUTIK®), edara [...]
Study sites6 entries, revised
University of PensylvaniaPennsylvania School of Medicine
2024-05-22
Study Status, Contacts/Locations v4
Study sites4→6
2024-05-13
Study Identification, Study Status, Study Description, Conditions, Study Design, Arms and Interventions, Outcome Measures, Eligibility, Contacts/Locations v3
Primary completion date2026-06-30→2026-09-30
Completion date2032-02-27→2031-03-30
Start date2024-02-01→2024-05-30
Enrollment target42→12
Study sites0→4
Study arms3→1
Eligibility criteria-3,464 characters
Inclusion Criteria: SubjectsConfirmed diagnosedclinical withand mutantgenetic 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). Subjects with rapidly progressing disease ("fast" progressors), defined as average ALS Functional Rating Scale - Revised decline ≥1.0 per month calculated from score at onset of symptoms compared to score at Screening ALSFRS-R. ALSFRS-R score ≥ 25 at Screening. Slow vital capacity [...] [...]
Secondary endpoints2 entries, revised
EfficacyCharacterization of AMT-162 EfficacyImmune ofResponse to AMT-162 comparedand toShedding placeboof intrathecally administered AMT-162. Characterization of the Effect of intrathecally administered AMT-162
Primary endpoints3 to 1 entries
CharacterizationTo evaluate the safety and tolerability of Kinetics,ascending Immune Response and Sheddingdoses of AMT-162. Efficacyintrathecally ofadministered AMT-162 comparedin toParticipants placebo Incidencewith of Treatment Emergent Adverse Events (TEAEs).SOD1-ALS
Study descriptionrevised
AMT-162 is an investigational gene therapy that encodes an artificial micro-ribonucleicmicroribonucleic acid (microRNA or miRNA) targeting the SOD1 gene. This clin [...] ant cytosolic SOD1 and thereby ameliorate the course of ALS causecaused by this mutant gene.
Study title+17 characters
Safety, Tolerability, and Exploratory Efficacy Study of Intrathecally Administered Gene Therapy AMT-162 in Adult Participants With SOD1 Amyotrophic Lateral Sclerosis (SOD1-ALS) Patients With SOD1 Mutations
2023-11-13
Study Status v2
Start date2023-11-01→2024-02-01
2023-11-07
Study Identification, Study Status, Arms and Interventions, Outcome Measures, Eligibility v1
Eligibility criteria+43 characters
[...] h control for the entire study period History of structural Central Nervous System (CNS) disease, may impact intrathecal infusion or cause difficult [...] tigational study medication within 90 days prior to signing IC.FInformed Consent (ICF) Planned treatment with live vaccines during Lead-in period [...]
Secondary endpointsdetails revised at 2 of 2 entries
Study title+32 characters
Safety, Tolerability, and Efficacy Study of Intrathecally Administered Gene Therapy AMT-162 in Amyotrophic Lateral Sclerosis (ALS) Patients With SOD1 Mutations
2023-10-20
Original filing
UniQure Biopharma B.V.
5 trials monitored
See 1 trial at risk
ALS
384 trials monitored
See 17 trials at risk
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.
Trial Details
NCT Number NCT06100276
Lead Sponsor UniQure Biopharma B.V.
Conditions Amyotrophic Lateral Sclerosis
Enrollment 20 participants
Start Date 2024-08-01
Primary Completion 2026-09-30 (estimated)
Study Completion 2031-06-30 (estimated)
Updated on ClinicalTrials.gov 2025-10-22