NCT06100276View on ClinicalTrials.gov

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

PHASE1/PHASE2Active, not recruitingINTERVENTIONAL
Enrollment

20

Participants

Timeline

Start Date

August 1, 2024

Primary Completion Date

September 30, 2026

Study Completion Date

June 30, 2031

Conditions
Amyotrophic Lateral Sclerosis
Interventions
DRUG

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.

Trial Locations (12)

10032

Columbia University Irving Medical Center, New York

19104

University of Pennsylvania School of Medicine, Philadelphia

30322

Winship Cancer Institute of Emory University, Atlanta

32224

Mayo Clinic Florida, Jacksonville

55905

Mayo Clinic Rochester, Rochester

60611

Northwestern University Feinberg School of Medicine, Chicago

66205

University of Kansas Medical Center, Fairway

85013

Barrow Neurological Institute, Phoenix

92697

University of California Irvine, Irvine

94109

California Pacific Medical Center, San Francisco

02114

Massachusetts General Hospital, Sean M. Healey and AMG Center for ALS Research, Boston

Unknown

Norrlands Universitetssjukhus, Umeå

All Listed Sponsors
lead

UniQure Biopharma B.V.

INDUSTRY

NCT06100276 - Safety, Tolerability, and Exploratory Efficacy Study of Intrathecally Administered Gene Therapy AMT-162 in Adult Participants With SOD1 Amyotrophic Lateral Sclerosis (SOD1-ALS) | Biotech Hunter | Biotech Hunter