Study to Evaluate Patients With Cerebrotendinous Xanthomatosis
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- STATUS
- Recruiting
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- participants needed
- 12
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- sponsor
- Retrophin, Inc.
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 | 1-100 years |
| 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 Identifier | NCT04270682 |
| Sponsor | Retrophin, Inc. |
| Last Modified on | 19 February 2024 |
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Additional screening procedures may be conducted by the study team before you can be confirmed eligible to participate.
Learn moreIf you are confirmed eligible after full screening, you will be required to understand and sign the informed consent if you decide to enroll in the study. Once enrolled you may be asked to make scheduled visits over a period of time.
Learn moreComplete your scheduled study participation activities and then you are done. You may receive summary of study results if provided by the sponsor.
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