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)

Status

Recruiting

Phases

Phase 1Phase 2

Study type

Interventional

Source

EU CTIS

Registry ID

CTIS2024-517244-57-00

Acronym

AMT-162-001

Enrollment

Registered

2025-01-28

Start date

2025-03-03

Completion date

Unknown

Last updated

2025-10-23

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Amyotrophic Lateral Sclerosis (ALS)

Brief summary

Incidence of treatment-emergent adverse events (TEAEs), Mean change from baseline (BL) in clinical safety laboratory tests, Mean change from BL in vital sign parameters, Mean change from BL in electrocardiogram (ECG) parameters

Detailed description

AMT-162 vector shedding, B-cell and T-cell immunological response (neutralizing antibodies to AAVrh10 viral capsid, anti-AAV total IgG/IgM antibodies, and T cell response), Change from BL in slow vital capacity (SVC) percent of predicted value, Hand-held dynamometry (HHD) scores, Neurofilament light chain (NfL) protein levels in serum up to 5 years post-treatment

Interventions

None listed

Eligibility

Sex/Gender

All

Age

18 Years to No maximum

Design outcomes

Primary

Measure	Time frame
Incidence of treatment-emergent adverse events (TEAEs), Mean change from baseline (BL) in clinical safety laboratory tests, Mean change from BL in vital sign parameters, Mean change from BL in electrocardiogram (ECG) parameters	—

Secondary

Measure	Time frame
AMT-162 vector shedding, B-cell and T-cell immunological response (neutralizing antibodies to AAVrh10 viral capsid, anti-AAV total IgG/IgM antibodies, and T cell response), Change from BL in slow vital capacity (SVC) percent of predicted value, Hand-held dynamometry (HHD) scores, Neurofilament light chain (NfL) protein levels in serum up to 5 years post-treatment	—

Countries

Sweden

Outcome results