Overview

Study to Evaluate Patients With Cerebrotendinous Xanthomatosis

Status:
Recruiting
Trial end date:
2023-03-01
Target enrollment:
Participant gender:
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)
Phase:
Phase 3
Details
Lead Sponsor:
Retrophin, Inc.
Travere Therapeutics, Inc.
Treatments:
Chenodeoxycholic Acid