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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: Completed Phase 3 study, sponsored by Mirum Pharmaceuticals.
NCT04270682 is a Phase 3 study that has completed, run by Mirum Pharmaceuticals. The registered enrollment target is 19 participants, below the 772-participant average among 15,282 other Phase 3 trials with a reported enrollment target (98% lower). According to ClinicalTrials.gov, the official US trial registry.
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)
Primary Outcome
Primary Analysis of Change from Baseline in Urine 23S-Pentol (Natural Log-Transformed) during the Two Double-blind Periods- Paired T-test
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 |
What the finished NCT04270682 record still lists
NCT04270682 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 19 participants, a relatively small participant target, below the 772-participant average among 15,282 other Phase 3 trials with a reported enrollment target (98% lower).
The record links to 0 conditions, and to 5 interventions - of which Placebo is the first listed.
NCT04270682 does not publish any study locations in the registry export this page uses.
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
How this trial's enrollment target compares
Where NCT04270682's enrollment target sits among peer trials
19 1969th of 2000 higher than 31 of 2,000 other Phase 3 trials
participants (enrollment target), bucketed by value
Each bar is a band; taller bars hold more other Phase 3 trials. The dashed line + filled bar mark this entry. Hover or tap any bar for its full count and share, and where it sits relative to this entry.
Source ClinicalTrials.gov registry export · 2026-08-08
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