Status
Study
Lead sponsor
Phase
Enrol
US states
Start
Recruiting
Satellos Bioscience, Inc.
Phase 2
51
California, Colorado, Illinois, Massachusetts, Missouri, Ohio, Washington
2025-12-08
Active, closed
Insmed Gene Therapy LLC
Phase 1
12
Arkansas, California, Georgia, New York, Ohio, Tennessee, Virginia
2025-07-22
Active, closed
Hoffmann-La Roche
Phase 2
30
Arkansas, California, Georgia, Michigan, Texas, Virginia
2025-04-04
Recruiting
Catalyst Pharmaceuticals, Inc.
Observational
250
Arizona, Arkansas, California, District of Columbia, Florida, Illinois, Indiana, Kansas
2024-09-25
Active, closed
NS Pharma, Inc.
Phase 1/2
20
California, Colorado, Illinois, Kansas, Pennsylvania
2024-09-18
By invitation
REGENXBIO Inc.
Observational
66
Arkansas, California, Illinois, Texas, Virginia
2024-05-08
Recruiting
Solid Biosciences Inc.
Phase 1/2
60
Arkansas, California, Georgia, Illinois, Missouri, Ohio, Oregon, Pennsylvania
2024-05-06
Active, closed
Edgewise Therapeutics, Inc.
Phase 2
43
California, Florida, Massachusetts, Missouri, North Carolina, Ohio
2024-03-22
By invitation
Sarepta Therapeutics, Inc.
Observational
500
Arkansas, California, Colorado, Connecticut, District of Columbia, Florida, Illinois, Indiana
2024-02-07
Active, closed
Avidity Biosciences, Inc.
Phase 2
39
Arkansas, California, Georgia, Massachusetts, Minnesota, North Carolina, Ohio, Texas
2024-01-22
By invitation
Sarepta Therapeutics, Inc.
Phase 3
400
Arkansas, California, Colorado, Florida, Illinois, Iowa, Maryland, Massachusetts
2023-09-27
Active, closed
Sarepta Therapeutics, Inc.
Phase 3
148
Arkansas, California, Florida, Illinois, Maryland, Massachusetts, Missouri, New York
2023-05-31
Active, closed
REGENXBIO Inc.
Phase 2/3
65
Arkansas, California, Colorado, Florida, Georgia, Illinois, Iowa, Kansas
2023-01-04
Active, closed
Edgewise Therapeutics, Inc.
Phase 2
76
Arkansas, California, Colorado, Florida, Georgia, Iowa, Kansas, Maryland
2022-10-24
Active, closed
NS Pharma, Inc.
Phase 4
9
California, Illinois, North Carolina, Virginia
2021-06-10
Recruiting
Sarepta Therapeutics, Inc.
Phase 1
83
Arkansas, California, Florida, Illinois, Missouri, North Carolina, Ohio, Texas
2020-11-23
Active, closed
Pfizer
Phase 3
114
Arkansas, California, Florida, Iowa, Kansas, North Carolina, Pennsylvania, Utah
2020-11-05
Active, closed
Capricor Inc.
Phase 2
13
California, Colorado, Missouri, Ohio, Wisconsin
2020-08-05
By invitation
Sarepta Therapeutics, Inc.
Observational
300
Arkansas, California, District of Columbia, Florida, Iowa, Maryland, Missouri, New York
2019-01-07
Active, closed
Solid Biosciences Inc.
Phase 1/2
12
California, Florida
2017-12-06
Recruiting
Italfarmaco
Phase 2/3
206
California, Connecticut, Florida, Georgia, Iowa, Missouri, Oregon, Pennsylvania
2017-10-24