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.
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.
Study Type
INTERVENTIONAL
Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Enrollment
20
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.
Barrow Neurological Institute
Phoenix, Arizona, United States
University of California Irvine
Irvine, California, United States
California Pacific Medical Center
San Francisco, California, United States
To evaluate the safety and tolerability of ascending doses of intrathecally administered AMT-162 in Participants with SOD1-ALS
Occurrence of TEAEs upon administration of ascending doses of AMT-162
Time frame: up to 5 years
Characterization of Immune Response to AMT-162 and Shedding of intrathecally administered AMT-162.
Any positive results in shedding samples will be summarized at each timepoint. Immunogenicity parameters will be summarized for each visit.
Time frame: up to 5 years
Characterization of the Effect of intrathecally administered AMT-162
Change from Baseline in Slow Vital Capacity (SVC) percent of predict value, Hand-held dynamometry (HHD) scores, and Neurofilament light chain (NfL) protein levels in serum. Immunogenicity parameters will be summarized for each visit.
Time frame: up to 5 years
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Mayo Clinic Florida
Jacksonville, Florida, United States
Winship Cancer Institute of Emory University
Atlanta, Georgia, United States
Northwestern University Feinberg School of Medicine
Chicago, Illinois, United States
University of Kansas Medical Center
Fairway, Kansas, United States
Massachusetts General Hospital, Sean M. Healey and AMG Center for ALS Research
Boston, Massachusetts, United States
Mayo Clinic Rochester
Rochester, Minnesota, United States
Columbia University Irving Medical Center
New York, New York, United States
...and 2 more locations