drugset / Trial / NCT04270682
Study to Evaluate Patients With Cerebrotendinous Xanthomatosis (RESTORE)
RandomizedCrossoverQuadruple-blindTreatment
Summary
The study is made up of two cohorts: a randomized double-blind crossover (placebo withdrawal with rescue) study among patients ≥ 16 years of age (adult cohort) and an open-label dose titration study among pediatric patients ≥1 month and \<16 years of age (pediatric cohort)
Timeline
- Start
- 2020-01-31
- Primary completion
- 2023-07-13
- Completion
- 2023-10-04
Drugs
| Evaluation | Drug | Modality | Dose | Route |
|---|---|---|---|---|
| Subject | Chenodeoxycholic Acid | Small molecule | 250 mg | — |
Indications
No indication recorded.