Status
Study
Lead sponsor
Phase
Enrol
US states
Start
Recruiting
Amicus Therapeutics
Observational
450
Alabama, Arkansas, Georgia, Missouri, New York, Pennsylvania, Texas, Virginia
2026-02-13
Recruiting
UniQure Biopharma B.V.
Phase 1/2
12
Alabama, Georgia, Illinois, Minnesota, New York, Pennsylvania, Utah, Virginia
2024-06-05
Active, closed
Sanofi
Phase 3
104
Alabama, California, Georgia, Illinois, New York, Texas, Utah, Virginia
2022-05-03
Active, closed
Sanofi
Phase 3
122
Alabama, California, Florida, Georgia, New York, Ohio, Pennsylvania, Texas
2022-03-11
Active, closed
Sangamo Therapeutics
Observational
32
California, Florida, Georgia, Iowa, Minnesota, New York, Ohio, Virginia
2021-08-16