NCT04270682Phase 3Completed

Study to Evaluate Patients With Cerebrotendinous Xanthomatosis (RESTORE)

Study sponsor: Mirum Pharmaceuticals, Inc. · MIRM

Compare the available registry edits below. Primary completion is the date data collection ends for the main study measures; it does not tell you when results will be published.

Open ClinicalTrials.gov record CSV export · Pro

Primary completion2023-07-13Actual

Enrollment · participants19Actual

Source updated2024-10-28Last update listed in the registry record

Version history

Changes to this trial

Last 12 months

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No recorded changes are available for this view and time period. Our history may be incomplete; this does not establish that the trial stayed unchanged.

Study details

Conditions studied

CTX

Treatments or interventions studied

Blinded CDCA 250 mg TIDDrug
PlaceboDrug
Open-Label CDCA 250 mg TIDDrug
Rescue Medication CDCA 250 mg TIDDrug
CDCA Weight-Based Dose TIDDrug

Primary outcomes · main study measures

measure: Change From Baseline in Urine 23S-Pentol During the Two Double-Blind Periods; description: Primary Analysis of Change from Baseline in Urine 23S-Pentol (Natural Log-Transformed) during the Two Double-blind Periods- Paired T-test; timeFrame: Two double-blind periods: Week 0 to Week 4, Week 12 to Week 16

History coverage

Research sample: selected versions from May 2023 onward plus a preceding baseline where available. Only selected fields were retained; endpoint measures were truncated to 300 characters. This is not complete trial or sponsor history. Daily snapshots begin when collection starts.

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