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NCT06100276Not yet recruiting

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

A Phase 1/2, Multicenter, Three-part Study to Evaluate the Safety, Tolerability, and Efficacy of Intrathecally Administered Gene Therapy AMT-162 in Patients With SOD1 Amyotrophic Lateral Sclerosis (SOD1-ALS).
UniQure Biopharma B.V.Phase 1/2ClinicalTrials.govdrug
Verified
Study focus
Disease/Condition
Amyotrophic Lateral Sclerosis
Drug/Device/Intervention
AMT-162
Study type
Interventional
Intervention type
drug
Sponsor & location
Primary sponsor
UniQure Biopharma B.V.
Timeline & enrollment
Phase
Phase 1/2
Start date
Feb 01, 2024
End date
Feb 27, 2032
Enrollment
42 participants
Primary outcome

Incidence of Treatment Emergent Adverse Events (TEAEs).,Characterization of Kinetics, Immune Response and Shedding of AMT-162.,Efficacy of AMT-162 compared to placebo

Summary

This is the study of AMT-162 in rapidly progressive ALS Patients with SOD1 Mutations and is
 designed to evaluate the Safety, Tolerability, and Efficacy of Intrathecally Administered
 Gene Therapy AMT-162. AMT-162-001 is a Phase 1/2, Multi-center, Three-part Study : Part I
 Single Ascending Dose, Part II Randomized, Double-blind, Placebo-controlled, and Part III
 Extended Follow-up.

ICD-10 classifications
Neuralgic amyotrophyMotor neuron diseaseOther degenerative diseases of the nervous systemOther degenerative disorders of nervous system in diseases classified elsewhereMultiple sclerosis
Data source
Registry
ClinicalTrials.gov
Trial ID
NCT06100276
Type
Non-Device Trial
About this record

Access comprehensive clinical trial information for NCT06100276 through Pure Global AI's free database. This Phase 1/2 trial is sponsored by UniQure Biopharma B.V. and is currently Not yet recruiting. The study focuses on Amyotrophic Lateral Sclerosis. Target enrollment is 42 participants.

This page provides complete trial specifications, intervention details, outcomes, and location information. Pure Global AI offers free access to ClinicalTrials.gov data, helping medical device and pharmaceutical companies navigate clinical research efficiently.