Study to Evaluate Patients With Cerebrotendinous Xanthomatosis (RESTORE)
Public ClinicalTrials.gov record NCT04270682. Field values are reproduced from the official study page; the official ClinicalTrials.gov record remains the source of truth for eligibility, enrollment, and contact information.
Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.
Official title
A Phase 3 Study to Evaluate the Effects of Chenodeoxycholic Acid in Adult and Pediatric Patients With Cerebrotendinous Xanthomatosis
Brief summary
Reproduced verbatim from the official ClinicalTrials.gov record. Not medical advice.
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)
Study identification
- NCT ID
- NCT04270682
- Recruitment status
- Completed
- Study type
- Interventional
- Phase
- Phase 3
- Enrollment
- 19 participants
Conditions and interventions
Conditions
Interventions
- 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 Drug
Drug
Eligibility (public fields only)
- Age range
- 1 Month and older
- Sex
- All
- Healthy volunteers
- Healthy volunteers not accepted
This page does not interpret eligibility. Detailed inclusion and exclusion criteria are on the official ClinicalTrials.gov record.
Study timeline
- Start date
- Jan 31, 2020 (Actual)
- Primary completion
- Jul 13, 2023 (Actual)
- Completion
- Oct 4, 2023 (Actual)
- Last update posted
- Oct 28, 2024
2020 – 2023
United States locations
- U.S. sites
- 9
- U.S. states
- 8
- U.S. cities
- 8
| Facility | City | State | ZIP | Site status |
|---|---|---|---|---|
| Travere Investigational Site | Aurora | Colorado | 80045 | — |
| Travere Investigational Site | Orlando | Florida | 32806 | — |
| Travere Investigational Site | Iowa City | Iowa | 52242 | — |
| Travere Investigational Site | New Orleans | Louisiana | 70112 | — |
| Travere Investigational Site | New Orleans | Louisiana | 70121 | — |
| Travere Investigational Site | Great Neck | New York | 11021 | — |
| Travere Investigational Site | Columbus | Ohio | 43221 | — |
| Travere Investigational Site | Austin | Texas | 78723 | — |
| Travere Investigational Site | Seattle | Washington | 98195 | — |
Site contact phone numbers, emails, and investigator names are intentionally not displayed here. Open the official ClinicalTrials.gov record for site contact information.
Non-U.S. locations
This page focuses on the U.S. directory. The official record also lists 3 non-U.S. sites.
About this trial record page
- What this page shows
- Public field values for ClinicalTrials.gov record NCT04270682, including study identification, conditions, interventions, eligibility (age, sex, healthy volunteer), timeline, and U.S. site list.
- What this page does not do
- No medical advice, eligibility judgments, treatment recommendations, study quality scoring, or AI-generated medical summaries. No site contact phone numbers, emails, or investigator names.
- Where the data comes from
- Sourced from the official ClinicalTrials.gov public API. The official record is the source of truth.
- Last refresh
- Last update posted Oct 28, 2024 · Synced Oct 22, 2026
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Open the official record
The complete public registry record, listed eligibility criteria, and available contact information for NCT04270682 live on ClinicalTrials.gov.