Providing education, support, advocacy, and promoting research
for CTX patients, their families, and medical professionals
who treat and study this rare disease.
Current CTX Clinical Trials
Study to Evaluate Patients with Cerebrotendinous Xanthomatosis sponsored by Travere Therapeutics
In the RESTORE study, doctors will look at markers of CTX in the urine, to see if they are lowered when CDCA is used to treat CTX. Doctors will also look at the safety and potential side effects of CDCA treatment.
The study has 2 groups: Adult (16 years of age and older) and Pediatric (under 16 years of age). In the Adult group, patients will switch from taking CDCA to taking a placebo for a short period. Temporarily taking a placebo will allow the doctors to better understand what effects CDCA has in these patients. In the Pediatric group, no placebo will be given. All patients will receive an appropriate dose of CDCA, based on their weight and how well they tolerate the drug.
In both groups, doctors will look at different markers of the disease, such as laboratory tests, weight, and disease severity.
More About Clinical Trials
The investigational therapies explored in clinical trials are key to improved therapies among the whole CTX community. ClinicalTrials.gov is a database of privately and publicly funded clinical studies conducted around the world. This comprehensive database is managed by the US National Library of Medicine.
To find a study related to CTX, use the search box on clinicaltrials.gov and search on CTX or any other related term (i.e., cerebrotendinous, xanthomatosis, chenodeoxycholic, diarrhea, pediatric cataracts, CDCA). You can view studies that are not yet recruiting, recruiting, enrolling by invitation, active (not recruiting), suspended, terminated, completed, or withdrawn. Many other details are available for each individual trial.
Additional information sources about clinical trials can be found by contacting the Patient Recruitment Office at the National Institutes of Health (NIH) Clinical Center in Bethesda, Maryland at 800.411.1222 or 866.441.1010 (TTY) or by email. You many also access the EU Clinical Trials Register for information on trials conducted in Europe.