Study to Evaluate Patients With Cerebrotendinous Xanthomatosis (RESTORE)

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)

Timeline

Start

2020-01-31

Primary completion

2023-07-13

Completion

2023-10-04

Outcome

Met primary endpoint

registry analysis (equivalence test); CDCA TID vs Placebo TID; p <0.0001; Mean Difference (Final Values) -3.06 (95% CI -3.794 to -2.331); paired t-test NCT04270682 ↗

Drugs

Indications

No indication recorded.