Summary

Eligibility
for people ages 18 years and up (full criteria)
Location
at Irvine, California and other locations
Dates
study started
completion around

Description

Summary

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.

Official Title

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).

Details

AMT-162 is an investigational gene therapy that encodes an artificial microribonucleic acid (microRNA or miRNA) targeting the SOD1 gene. This clinical study will test the safety of AMT-162 and explore the hypothesis that it will silence expression of mutant cytosolic SOD1 and thereby ameliorate the course of ALS caused by this mutant gene.

Keywords

Amyotrophic Lateral Sclerosis, Gene Therapy, AAV (adeno-associated virus), ALS, SOD1, Motor Neuron Disease, Sclerosis, AMT-162

Eligibility

Locations

  • University of California Irvine accepting new patients
    Irvine California 92697 United States
  • California Pacific Medical Center accepting new patients
    San Francisco California 94109 United States

Details

Status
accepting new patients
Start Date
Completion Date
(estimated)
Sponsor
UniQure Biopharma B.V.
ID
NCT06100276
Phase
Phase 1/2 Amyotropic Lateral Sclerosis (ALS) Research Study
Study Type
Interventional
Participants
Expecting 20 study participants
Last Updated