CI

At a glance

ClinicalIndex Comparison Record
Phase 3Completed· 19 enrolled
Drug / intervention
Blinded CDCA 250 mg TID +4 moredrug
Likely dose
Blinded CDCA 250 mg TIDfrom record
Structured eligibility isn't available for this trial yet — see the full criteria in the Eligibility tab below.

Standardized by ClinicalIndex from the ClinicalTrials.gov record · verify against the source.

Search/NCT04270682
NCT04270682Phase 3Completed
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A Phase 3 Study to Evaluate the Effects of Chenodeoxycholic Acid in Adult and Pediatric Patients With Cerebrotendinous Xanthomatosis

Mirum Pharmaceuticals, Inc.·interventional·Posted Feb 17, 2020·Updated Oct 28, 2024

In Brief

A Phase 3 clinical trial evaluating Blinded CDCA 250 mg TID, Placebo, and 3 other interventions for CTX. Completed, enrolled 19 participants across 12 sites in 2 countries.

Detailed 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)

Study Details

Study Typeinterventional
Allocation--
Masking--
Primary Purpose--
ConditionsCTX
CountriesBrazil, United States
Collaborators--

Timeline

Phase 3CompletedFinished
2020202120222023202420252026
First PostedFeb 17, 2020
Enrollment StartJan 31, 2020
Primary CompletionJul 13, 2023
Study CompletionOct 4, 2023
TodayJul 2, 2026
Enrollment to primary: 3.4 yearsPosted 6.4 years ago

Interventions

Blinded CDCA 250 mg TIDdrug

Adult cohort patients will receive blinded 250 mg CDCA TID or placebo during the double-blind periods based upon their treatment assignment.

Placebodrug

Adult cohort patients will receive blinded 250 mg CDCA TID or placebo during the double-blind periods based upon their treatment assignment.

Open-Label CDCA 250 mg TIDdrug

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.

Rescue Medication CDCA 250 mg TIDdrug

CDCA 250 mg TID will be provided as rescue medication during the double-blind periods, if needed, based on laboratory results.

CDCA Weight-Based Dose TIDdrug

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.