Clinical trial NCT06260748: A Study of Chenodeoxycholic Acid (CDCA) in Newly Diagnosed Participants With Cerebrotendinous Xanthomatosis (CTX)
Official title: A Phase 3, Prospective, Randomized, Double-Blind, Placebo-Controlled, Multicenter Study of Chenodeoxycholic Acid (CDCA), With an Open-Label Cohort, in Newly Diagnosed Participants With Cerebrotendinous Xanthomatosis (CTX)
Study type: INTERVENTIONAL
Overall status: WITHDRAWN
Phase: PHASE3
Conditions: Cerebrotendinous Xanthomatoses
Lead sponsor: Leadiant Biosciences, Inc.
Sponsor class: INDUSTRY
First posted: 2024-02-15
Enrollment: 0
Sex: ALL
Minimum age (years): 2
Maximum age (years): 75
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

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.


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

Primary outcomes:
measure: Change from baseline at Week 4 in average number of stools with a BSS score of 6 or 7 per day
timeFrame: Up to 4 weeks
Secondary outcomes:
measure: Change in plasma cholestanol levels from baseline to Week 12
timeFrame: Up to 12 weeks
measure: Change in urine and/or plasma bile alcohol levels from baseline to Week 12
timeFrame: Up to 12 weeks
measure: Incidence of, severity/intensity of, and relationship to study drug of AEs
timeFrame: Up to 20 weeks
measure: Incidence of, severity/intensity of, and relationship to study drug of SAEs
timeFrame: Up to 20 weeks
measure: Incidence of, severity/intensity of AESIs
timeFrame: Up to 20 weeks
measure: Incidence of, severity/intensity of, and changes in laboratory values
timeFrame: Up to 20 weeks
measure: Incidence of, severity/intensity of, and changes in physical examination
timeFrame: Up to 20 weeks
measure: Incidence of, severity/intensity of, and changes in vital signs
timeFrame: Up to 20 weeks
measure: Number of participants with discontinuations due to AEs
timeFrame: Up to 20 weeks
Other outcomes:
Locations:
city: Tel HaShomer
country: Israel
facility: Sheba Medical Center
Study officials:
References: