Medical Information Only. Always consult your healthcare provider before enrolling in any clinical trial.
NCT04270682 · ClinicalTrials.gov registry record · Phase 3
Study to Evaluate Patients With Cerebrotendinous Xanthomatosis (RESTORE)
A Phase 3 study, sponsored by Mirum Pharmaceuticals.
- Completed
- Registry status
- Phase 3
- Development phase
- 19
- Enrollment target
NCT04270682 is a Phase 3 study that has completed, run by Mirum Pharmaceuticals. The registered enrollment target is 19 participants.
The verdict
NCT04270682, a Phase 3 study, has completed, sponsored by Mirum Pharmaceuticals.
- COMPLETED
- Registry status
- Phase 3
- Development phase
- 19 participants
- Enrollment target
Study 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)
Interventions
- DRUG Placebo
- DRUG Blinded CDCA 250 mg TID
- DRUG Open-Label CDCA 250 mg TID
- DRUG Rescue Medication CDCA 250 mg TID
- DRUG CDCA Weight-Based Dose TID
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 19 participants |
| Start Date | 2020-01-31 |
| Est. Completion | 2023-10-04 |
| Phase | Phase 3 |
Interested in This Trial?
Full Details on ClinicalTrials.gov ↗What the Registry Record Tells You About NCT04270682
The ClinicalTrials.gov registry entry for NCT04270682 describes a study currently listed as completed, categorized as Phase 3. The registered enrollment target is 19 participants, a figure that helps gauge the scale of data the investigators plan to collect. The listed sponsor is Mirum Pharmaceuticals, which has 28 total studies on file at ClinicalTrials.gov.
The record links to 0 conditions, and to 5 interventions - of which Placebo is the first listed.
NCT04270682 reports 0 study locations.
Frequently Asked Questions
What is clinical trial NCT04270682 about?
NCT04270682 is a clinical study titled "Study to Evaluate Patients With Cerebrotendinous Xanthomatosis (RESTORE)". 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)
What is the current status of trial NCT04270682?
This trial is currently completed. It is a Phase 3 study. The enrollment target is 19 participants. The study started on 2020-01-31. Estimated completion is 2023-10-04.
What interventions are being tested in trial NCT04270682?
The interventions under investigation include: Placebo (DRUG), Blinded CDCA 250 mg TID (DRUG), Open-Label CDCA 250 mg TID (DRUG), Rescue Medication CDCA 250 mg TID (DRUG), CDCA Weight-Based Dose TID (DRUG).
Who is sponsoring clinical trial NCT04270682?
This trial is sponsored by Mirum Pharmaceuticals, which has 28 total clinical trials registered on ClinicalTrials.gov.
Learn More About Clinical Trials
Explore more on PlainTrial
Read our methodology - how this data is sourced, computed, and verified.
Related
Every figure on PlainTrial is rendered directly from the ClinicalTrials.gov registry, no number is typed in by an editor. This page mirrors this trial's own ClinicalTrials.gov registry record, live from the dataset. See our editorial standards & corrections policy, the methodology behind these numbers, or report a data error.