Status:
COMPLETED
Study to Evaluate Patients With Cerebrotendinous Xanthomatosis (RESTORE)
Lead Sponsor:
Mirum Pharmaceuticals, Inc.
Conditions:
CTX
Eligibility:
All Genders
1+ years
Phase:
PHASE3
Brief 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 a...
Eligibility Criteria
Inclusion
- Male or female at least 1 month or older at screening.
- Clinical diagnosis of CTX with biochemical confirmation.
- Women of childbearing potential must agree to the use of one highly reliable method of contraception during the study, plus one additional barrier method during sexual activity.
- Males must be surgically sterile, or males and their sexual partners must together agree to use medically accepted methods of contraception that are considered highly reliable during the course of the study.
Exclusion
- Genetic testing does not confirm CTX.
- Malabsorption disorder or confounding inflammatory gastrointestinal condition (for example, irritable bowel syndrome).
- Documented history of heart failure.
- Treated with medications which impact bile acid absorption such as bile acid sequestering agents (eg, cholestyramine, colestipol, aluminum-based antacids.
- Treated with cholic acid medication.
- Female patient who is pregnant, plans to become pregnant during the course of the study, or is breastfeeding.
- Positive at screening for the human immunodeficiency virus (HIV) or markers indicating acute or chronic hepatitis B infection or hepatitis C infection.
Key Trial Info
Start Date :
January 31 2020
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
October 4 2023
Estimated Enrollment :
19 Patients enrolled
Trial Details
Trial ID
NCT04270682
Start Date
January 31 2020
End Date
October 4 2023
Last Update
October 28 2024
Active Locations (12)
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1
Travere Investigational Site
Aurora, Colorado, United States, 80045
2
Travere Investigational Site
Orlando, Florida, United States, 32806
3
Travere Investigational Site
Iowa City, Iowa, United States, 52242
4
Travere Investigational Site
New Orleans, Louisiana, United States, 70112