The RESTORE study is a Phase 3 clinical trial looking at an investigational medication called chenodeoxycholic acid (also called Chenodal™, or CDCA). We are inviting people with Cerebrotendinous Xanthomatosis (CTX) who may be interested in participating in a research study.
The RESTORE study is enrolling pediatric (1 month to 16 years old) and adult (16 years and older) participants throughout the United States. The study will last approximately 6-7 months and involve about 18 visits to a study center. If the study is right for you and you choose to participate, all study-related care and study medication will be provided at no cost during your participation.
CTX is a rare, progressive, and underdiagnosed bile acid synthesis disorder affecting many parts of the body. Patients with CTX have a genetic mutation that prevents their body from making CDCA. As a result, the body is unable to break down cholesterol properly causing toxins (e.g., cholestanol and bile alcohols) to build up throughout the body over time. In CTX, cholestanol builds up in the eyes, tendons (i.e., tissues that connect muscle to the bone), brain, and other tissues and can cause a number of different problems throughout a patient’s life.
The investigational medicine in the RESTORE study, CDCA, is a bile acid replacement therapy that is recognized as a potential treatment for CTX1,2; however, it has not been approved by the U.S. Food and Drug Administration (FDA) for use in CTX in the United States. CDCA is approved in the United States as Chenodal™ for the treatment of a certain type of gallstones.3
The goal of the RESTORE study is to better understand how the body responds, as measured in blood and urine, when people with CTX use CDCA. It will also help the sponsor (Travere Therapeutics, Inc.) learn more about the safety of CDCA, or what side effects, if any, appear with CDCA use. For the purposes of the RESTORE study, CDCA is considered to be an investigational study drug.
Eligible participants (pediatric and adult) will need to visit a study center about 18 times. There will also be times when participants, or their caregivers, will need to speak by telephone to the study team at a study center. The study will last approximately 25 to 28 weeks (6-7 months).
In the Adult Group (16 years and older), participants will receive, at different times, CDCA or placebo (a substance that looks like CDCA but does not contain any active CDCA) to compare how the body responds. Both of these study medications will be given as tablets that need to be taken 3 times daily by mouth.
In the Pediatric Group (1 month to 16 years old), all participants will receive CDCA (there will be no placebo). The study medication will be given in liquid form that will need to be taken 3 times daily by mouth.
In both the Adult and Pediatric Groups, several tests and assessments will be performed to monitor the participant’s health. These will include:
The RESTORE Study consists of 6 study periods for adults and 4 study periods for pediatrics. See below for more details.
You* may be eligible to participate in the RESTORE study if you meet the following:
You must meet all other study criteria to take part in the RESTORE study. The full list of eligibility criteria is available at clinicaltrials.gov.Email firstname.lastname@example.org for more information!
*If you are reviewing these details on behalf of a child under the age of 16 years, the word "you" refers to the child
** Any personal data you provide Travere Therapeutics will be processed according to our Privacy Policies.
Clinical trials are divided into different phases. Each phase is designed to collect specific information about the study drug or regimen.
Phase 4 trials are conducted after the Regulatory Authorities have approved a drug and after the drug is on the market. Phase 4 trials typically involve a large number of participants. They may evaluate new uses of existing therapies or be used to detect side effects that did not appear during Phase 3 or earlier clinical trials and do not involve a placebo group.
For more information on clinical trials, please visit travere.com.
REFERENCES: 1. Mignarri A, Gallus GN, Dotti MT, Federico A. A suspicion index for early diagnosis and treatment of cerebrotendinous xanthomatosis. J Inherit Metab Dis. 2014;37(3):421-429. 2. National Organization for Rare Disorders. Rare Disease Database: Cerebrotendinous xanthomatosis. https://rarediseases.org/rare-diseases/cerebrotendinous-xanthomatosis/ Accessed April 26, 2020. 3. CHENODAL [package insert]. Fort Collins, CO: Manchester Pharmaceuticals, Inc; 2009.