Status
Study
Lead sponsor
Phase
Enrol
US states
Start
By invitation
QED Therapeutics, a BridgeBio company
Phase 2
135
California, Colorado, District of Columbia, Maryland, Missouri, Ohio, Tennessee, Wisconsin
2026-04-23
Recruiting
BioMarin Pharmaceutical
Phase 2
60
Arizona, California, District of Columbia, Illinois, Maryland, Minnesota, Missouri, Texas
2025-07-30
By invitation
BioMarin Pharmaceutical
Phase 3
140
Arizona, California, Delaware, District of Columbia, Illinois, Maryland, Minnesota, Missouri
2025-06-20
By invitation
QED Therapeutics, a BridgeBio company
Phase 2/3
24
California, Colorado, District of Columbia, Maryland, Missouri, Ohio, Tennessee, Wisconsin
2025-04-22
Active, closed
BioMarin Pharmaceutical
Phase 3
80
Delaware, District of Columbia, Illinois, Wisconsin
2024-06-17
Recruiting
QED Therapeutics, a BridgeBio company
Observational
150
California, Colorado, District of Columbia, Maryland, Missouri, Ohio, Tennessee, Wisconsin
2024-06-05
Recruiting
BioMarin Pharmaceutical
Observational
400
Arizona, California, Delaware, District of Columbia, Illinois, Maryland, Massachusetts, Minnesota
2023-11-27