Trials / Completed
CompletedNCT04270682
Study to Evaluate Patients With Cerebrotendinous Xanthomatosis (RESTORE)
A Phase 3 Study to Evaluate the Effects of Chenodeoxycholic Acid in Adult and Pediatric Patients With Cerebrotendinous Xanthomatosis
- Status
- Completed
- Phase
- Phase 3
- Study type
- Interventional
- Enrollment
- 19 (actual)
- Sponsor
- Mirum Pharmaceuticals, Inc. · Industry
- Sex
- All
- Age
- 1 Month
- Healthy volunteers
- Not accepted
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)
Conditions
Interventions
| Type | Name | Description |
|---|---|---|
| DRUG | Blinded CDCA 250 mg TID | Adult cohort patients will receive blinded 250 mg CDCA TID or placebo during the double-blind periods based upon their treatment assignment. |
| DRUG | Placebo | Adult cohort patients will receive blinded 250 mg CDCA TID or placebo during the double-blind periods based upon their treatment assignment. |
| DRUG | Open-Label CDCA 250 mg TID | Adult cohort patients will receive open-label 250 mg CDCA TID during the run-in and washout periods of the study or as rescue medication during the double-blind periods, if needed, based on clinical symptoms. |
| DRUG | Rescue Medication CDCA 250 mg TID | CDCA 250 mg TID will be provided as rescue medication during the double-blind periods, if needed, based on laboratory results. |
| DRUG | CDCA Weight-Based Dose TID | Patients in the pediatric cohort will complete a weight-based dose titration to a tolerated dose and will maintain that tolerated dose for the remainder of the study. Pediatric cohort dosing of CDCA will not exceed an equivalent dose of 750 mg/day. |
Timeline
- Start date
- 2020-01-31
- Primary completion
- 2023-07-13
- Completion
- 2023-10-04
- First posted
- 2020-02-17
- Last updated
- 2024-10-28
- Results posted
- 2024-10-28
Locations
12 sites across 2 countries: United States, Brazil
Regulatory
- FDA-regulated drug study
Source: ClinicalTrials.gov record NCT04270682. Inclusion in this directory is not an endorsement.