Study to Evaluate Patients With Cerebrotendinous Xanthomatosis (RESTORE)

NCT04270682 · clinicaltrials.gov ↗

PHASE3

Phase

COMPLETED

Status

19

Enrollment

INDUSTRY

Sponsor class

Conditions

CTX

Interventions

DRUG: Blinded CDCA 250 mg TID
DRUG: Placebo
DRUG: Open-Label CDCA 250 mg TID
DRUG: Rescue Medication CDCA 250 mg TID
DRUG: CDCA Weight-Based Dose TID

Sponsor

Mirum Pharmaceuticals, Inc.