263. 脳腱黄色腫症
［臨床試験数：5，薬物数：10（DrugBank：2），標的遺伝子数：2，標的パスウェイ数：4］
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 | 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 |