Safety, Tolerability, and Efficacy Study of Intrathecally Administered Gene Therapy AMT-162 in Amyotrophic Lateral Sclerosis (ALS) Patients With SOD1 Mutations - Trial NCT06100276
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.
Study Focus
Sponsor & Location
UniQure Biopharma B.V.
Timeline & Enrollment
Phase 1/2
Feb 01, 2024
Feb 27, 2032
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
Data Source
ClinicalTrials.gov
NCT06100276
Non-Device Trial