263. Cerebrotendinous xanthomatosis Disease details / Clinical trials / Drug dev / DR info
Clinical trials : 8 / Drugs : 9 - (DrugBank : 2) / Drug target genes : 2 - Drug target pathways : 3
Drugs, Primary sponsors and Trial info (Enrollment year, Phase, Trial ID, Countries)
Blinded cdca 250 MG TID
Mirum Pharmaceuticals, Inc.
2020 Phase 3 NCT04270682 Brazil;United States
Blood tests
Sheba Medical Center
2010 - NCT01613898 Israel
Cdca weight-based dose TID
Mirum Pharmaceuticals, Inc.
2020 Phase 3 NCT04270682 Brazil;United States
Chenodeoxycholic acid
Department of Veterans Affairs
1999 - NCT00018694 United States
Fujimoto Pharmaceutical Corporation
2020 Phase 3 JPRN-jRCT2080225062 Japan
Leadiant Biosciences, Inc.
2024 Phase 3 NCT06260748 Israel
National Center for Research Resources (NCRR)
1996 Phase 2 NCT00004346 United States
Chenodeoxycholic acid leadiant
Humanis Saglik Anonim Sirketi
2022 Phase 1 NCT06180057 Jordan
Lovastatin
National Center for Research Resources (NCRR)
1996 Phase 2 NCT00004346 United States
Mutation analysis
TRPHARM
2019 - NCT04218006 Turkey
OPEN-label cdca 250 MG TID
Mirum Pharmaceuticals, Inc.
2020 Phase 3 NCT04270682 Brazil;United States
Rescue medication cdca 250 MG TID
Mirum Pharmaceuticals, Inc.
2020 Phase 3 NCT04270682 Brazil;United States
Mirum Pharmaceuticals, Inc.
2020 Phase 3 NCT04270682 Brazil;United States
Blood tests
Sheba Medical Center
2010 - NCT01613898 Israel
Cdca weight-based dose TID
Mirum Pharmaceuticals, Inc.
2020 Phase 3 NCT04270682 Brazil;United States
Chenodeoxycholic acid
Department of Veterans Affairs
1999 - NCT00018694 United States
Fujimoto Pharmaceutical Corporation
2020 Phase 3 JPRN-jRCT2080225062 Japan
Leadiant Biosciences, Inc.
2024 Phase 3 NCT06260748 Israel
National Center for Research Resources (NCRR)
1996 Phase 2 NCT00004346 United States
Chenodeoxycholic acid leadiant
Humanis Saglik Anonim Sirketi
2022 Phase 1 NCT06180057 Jordan
Lovastatin
National Center for Research Resources (NCRR)
1996 Phase 2 NCT00004346 United States
Mutation analysis
TRPHARM
2019 - NCT04218006 Turkey
OPEN-label cdca 250 MG TID
Mirum Pharmaceuticals, Inc.
2020 Phase 3 NCT04270682 Brazil;United States
Rescue medication cdca 250 MG TID
Mirum Pharmaceuticals, Inc.
2020 Phase 3 NCT04270682 Brazil;United States