263. Cerebrotendinous xanthomatosis
5 clinical trials, 10 drugs (DrugBank: 2 drugs), 2 drug target genes, 4 drug target pathways
Searched query = "Cerebrotendinous xanthomatosis", "27-hydroxylase deficiency", "CYP27 deficiency"
The queries were searched in Public_title, Scientific_title, and Condition. Export date: 03/15/2021. Trials are sorted by Date_enrollment from most recent to oldest in the table.
| No. | TrialID | Date_ enrollment | Date_ registration | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| 1 | JPRN-JapicCTI-205157 | 03/8/2020 | 13/02/2020 | An open-label study of FPF1011 in patients with cerebrotendinous xanthomatosis (phase 3) | An open-label study of FPF1011 in patients with cerebrotendinous xanthomatosis (phase 3) | Cerebrotendinous xanthomatosis | Intervention name : FPF1011 INN of the intervention : Chenodeoxycholic acid Dosage And administration of the intervention : Dose of chenodeoxycholic acid is 750 mg/day for patients aged 20 or older, and 15 mg/kg/day under ages 20, administered orally in three divided doses a day. Control intervention name : - INN of the control intervention : - Dosage And administration of the control intervention : - | Fujimoto Pharmaceutical Corporation | NULL | recruiting | 0 | BOTH | 5 | Phase 3 | Japan | |
| 2 | NCT04270682 (ClinicalTrials.gov) | January 31, 2020 | 12/2/2020 | Study to Evaluate Patients With Cerebrotendinous Xanthomatosis | A Phase 3 Study to Evaluate the Effects of Chenodeoxycholic Acid in Adult and Pediatric Patients With Cerebrotendinous Xanthomatosis | CTX | Drug: Blinded CDCA 250 mg TID;Drug: Placebo;Drug: Open-Label CDCA 250 mg TID;Drug: Rescue Medication CDCA 250 mg TID;Drug: CDCA Weight-Based Dose TID | Travere Therapeutics, Inc. | NULL | Recruiting | 1 Month | N/A | All | 12 | Phase 3 | United States |
| 3 | NCT00018694 (ClinicalTrials.gov) | October 1999 | 3/7/2001 | Cholestanol in Humans | Biologic Significance of Cholestanol in Man | Cerebrotendinous Xanthomatosis | Drug: Chenodeoxycholic Acid | Department of Veterans Affairs | NULL | Withdrawn | 5 Years | 80 Years | Both | 0 | N/A | United States |
| 4 | NCT00004346 (ClinicalTrials.gov) | January 1996 | 18/10/1999 | Phase II Study of Cholesterol- and Cholestanol-Free Diet, Lovastatin, and Chenodeoxycholic Acid for Cerebrotendinous Xanthomatosis | Effects of Diet and Medication in Patients With Cerebrotendinous Xanthomatosis (CTX) | Cerebrotendinous Xanthomatosis | Drug: chenodeoxycholic acid;Drug: lovastatin | National Center for Research Resources (NCRR) | Oregon Health and Science University | Recruiting | N/A | N/A | Both | 5 | Phase 2 | United States |