12
Participants
Start Date
June 5, 2024
Primary Completion Date
December 1, 2027
Study Completion Date
April 30, 2031
AMT-191
"A recombinant serotype 5 based adeno-associated viral vector (AMT-191) for one-time intravenous (IV) administration will be investigated in this study. This recombinant AAV5-based vector contains a coding deoxyribonucleic acid (DNA) sequence for human α-galactosidase A.~Delivery of AMT-191 to the systemic circulation is expected to result in a therapeutic effect by promoting the liver expression of the lysosomal enzyme GLA in plasma levels in patients with Fabry disease."
RECRUITING
NYC Health + Hospitals/Metropolitan, New York
RECRUITING
UPMC Children's Hospital of Pittsburgh, Pittsburgh
RECRUITING
Lysosomal & Rare Disorders Research and Treatment Center, Inc, Fairfax
RECRUITING
Emory University School of Medicine, Atlanta
RECRUITING
The Kirklin Clinic Of university of Alabama Birmingham Hospital, Birmingham
RECRUITING
MHealth Fairview University of Minnesota Medical Center East Bank, Minneapolis
RECRUITING
Ann & Robert H. Lurie Children's Hospital of Chicago, Chicago
RECRUITING
University of Utah, Clinical and Translational Sciences Institute, Salt Lake City
UniQure Biopharma B.V.
INDUSTRY