Study to Evaluate Patients With Cerebrotendinous Xanthomatosis

  • STATUS
    Recruiting
  • participants needed
    12
  • sponsor
    Retrophin, Inc.
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Updated on 19 February 2024
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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)

Details
Condition Cyclophosphamide, CEREBROTENDINOUS XANTHOMATOSIS
Age 1years - 100years
Treatment Placebo, Blinded CDCA 250 mg TID, Open-Label CDCA 250 mg TID, Rescue Medication CDCA 250 mg TID, CDCA Weight-Based Dose TID
Clinical Study IdentifierNCT04270682
SponsorRetrophin, Inc.
Last Modified on19 February 2024

Eligibility

 Yes No Not Sure

Inclusion Criteria

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 Criteria

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
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