Safety, Tolerability, and Exploratory Efficacy Study of Intrathecally Administered Gene Therapy AMT-162 in Adult Participants With SOD1 Amyotrophic Lateral Sclerosis (SOD1-ALS)
Condition(s) studied
Investigational drug(s) / intervention(s)
AMT-162: AMT-162, the investigational product (IP), is a nonreplicating, rep/cap-deleted, self-complementary Recombinant adeno-associated virus (rAAV) vector based on adeno-associated virus (AAV) serotype rh10 and contains complementary deoxyribonucleic acid (cDNA) encoding an artificial miRNA targeting the SOD1 gene.
Study 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.
Eligibility
Primary outcome measure(s)
- To evaluate the safety and tolerability of ascending doses of intrathecally administered AMT-162 in Participants with SOD1-ALS — up to 5 years
Occurrence of TEAEs upon administration of ascending doses of AMT-162
Trial sites (12)
| Facility | City | Region | Status |
|---|---|---|---|
| Barrow Neurological Institute | Phoenix | Arizona | |
| University of California Irvine | Irvine | California | |
| California Pacific Medical Center | San Francisco | California | |
| Mayo Clinic Florida | Jacksonville | Florida | |
| Winship Cancer Institute of Emory University | Atlanta | Georgia | |
| Northwestern University Feinberg School of Medicine | Chicago | Illinois | |
| University of Kansas Medical Center | Fairway | Kansas | |
| Massachusetts General Hospital, Sean M. Healey and AMG Center for ALS Research | Boston | Massachusetts | |
| Mayo Clinic Rochester | Rochester | Minnesota | |
| Columbia University Irving Medical Center | New York | New York | |
| University of Pennsylvania School of Medicine | Philadelphia | Pennsylvania | |
| Norrlands Universitetssjukhus | Umeå | Vasterbottens Ian |
More UniQure Biopharma B.V. trials in the USA
Other trials for the same condition
This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.
View NCT06100276 on ClinicalTrials.gov ↗ ← All trials in the USA