A Study of Chenodeoxycholic Acid (CDCA) in Newly Diagnosed Participants With Cerebrotendinous Xanthomatosis (CTX)

Launched by LEADIANT BIOSCIENCES, INC. · Feb 12, 2024

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Eligibility criteria

• 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