A Study of Chenodeoxycholic Acid (CDCA) in Newly Diagnosed Participants With Cerebrotendinous Xanthomatosis (CTX)
Phase 3
Withdrawn
- Conditions
- Cerebrotendinous Xanthomatoses
- Interventions
- Drug: Chenodeoxycholic acid Placebo
- Registration Number
- NCT06260748
- Lead Sponsor
- Leadiant Biosciences, Inc.
- Brief Summary
This study is designed to demonstrate the beneficial effect of CDCA in the treatment of CTX-associated diarrhea in approximately 10 participants aged 2-75 years old with newly diagnosed CTX or suspected CTX who have never received treatment with CDCA.
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- WITHDRAWN
- Sex
- All
- Target Recruitment
- 10
Inclusion Criteria
- Signed informed consent form (or assent form as appliable)
- Aged from 2 to 75 years old
- Has a new or suspected diagnosis of CTX as defined by an elevated plasma cholestanol concentration (>10 mg/L/>25.7 μmol/L) in conjunction with a clinical presentation consistent with the diseased as assessed by the investigator
- Has never received treatment with CDCA
- Has never received treatment with other bile acid products
Exclusion Criteria
- Any medical condition that, in the opinion of the investigator, precludes the participant's participation in the study
- Presence of known hepatocyte dysfunction or bile ductal abnormalities such as intrahepatic cholestasis, primary biliary cirrhosis or sclerosing cholangitis
- Inability to adhere to treatment and visit schedule
- Female participants who are pregnant
- Female participants who are breast feeding
- Female participants who are using estrogen-containing compounds and cannot/will not discontinue them for the duration of the study
- Female participants of childbearing potential who are not using locally approved birth control method(s) or double barrier contraception (ie, condom and diaphragm, condom or diaphragm and spermicidal gel or foam)
- Taking any of the following medications: bile acid products; inhibitors of bile acid transporters; bile acid binding resins; aluminum-based antacids; coumarin and its derivatives; cholestyramine; ciclosporin; sirolimus; or phenobarbital
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- PARALLEL
- Arm && Interventions
Group Intervention Description Group 1 Diarrhea-Evaluable Chenodeoxycholic acid Placebo Diarrhea-evaluable participants aged 12-75 years old who did not use continence aids that precluded assessment or evaluation of stool frequency and consistency during the screening/baseline period; randomized 1:1 to receive either placebo or CDCA. Group 1 Diarrhea-Evaluable Chenodeoxycholic acid Diarrhea-evaluable participants aged 12-75 years old who did not use continence aids that precluded assessment or evaluation of stool frequency and consistency during the screening/baseline period; randomized 1:1 to receive either placebo or CDCA. Group 2b Pediatrics Chenodeoxycholic acid Participants aged 2 to less than 12 years old with or without clinically burdensome diarrhea. Group 2a Non-Diarrhea Evaluable Chenodeoxycholic acid Non-diarrhea-evaluable participants aged 12-75 years old who do not have stable, clinically burdensome diarrhea or their diarrhea cannot be fully characterized.
- Primary Outcome Measures
Name Time Method Change from baseline at Week 4 in average number of stools with a BSS score of 6 or 7 per day Up to 4 weeks
- Secondary Outcome Measures
Name Time Method Change in urine and/or plasma bile alcohol levels from baseline to Week 12 Up to 12 weeks All groups combined
Incidence of, severity/intensity of, and relationship to study drug of SAEs Up to 20 weeks Incidence of, severity/intensity of, and changes in physical examination Up to 20 weeks Incidence of, severity/intensity of, and changes in laboratory values Up to 20 weeks Incidence of, severity/intensity of, and relationship to study drug of AEs Up to 20 weeks Incidence of, severity/intensity of, and changes in vital signs Up to 20 weeks Incidence of, severity/intensity of AESIs Up to 20 weeks Diarrhea and hepatic dysfunction
Number of participants with discontinuations due to AEs Up to 20 weeks Change in plasma cholestanol levels from baseline to Week 12 Up to 12 weeks All groups combined
Trial Locations
- Locations (1)
Sheba Medical Center
🇮🇱Tel HaShomer, Israel
Sheba Medical Center🇮🇱Tel HaShomer, Israel