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

Leadiant Biosciences

Status and phase: Withdrawn; Phase 3

Conditions: Cerebrotendinous Xanthomatoses

Treatments: Drug: Chenodeoxycholic acid; Drug: Chenodeoxycholic acid Placebo

Study type: Interventional

Funder types: Industry

Identifiers

NCT06260748

LBI-CDCA-001

2023-505759-29-00 (Other Identifier)

Details and patient eligibility

About

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.

Sex: All

Ages: 2 to 75 years old

Volunteers: No Healthy Volunteers

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

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Quadruple Blind

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0 participants in 3 patient groups

Group 1 Diarrhea-Evaluable

Experimental group

Description:

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.

Treatment:

Drug: Chenodeoxycholic acid Placebo

Drug: Chenodeoxycholic acid

Group 2a Non-Diarrhea Evaluable

Active Comparator group

Description:

Non-diarrhea-evaluable participants aged 12-75 years old who do not have stable, clinically burdensome diarrhea or their diarrhea cannot be fully characterized.

Treatment:

Drug: Chenodeoxycholic acid

Group 2b Pediatrics

Active Comparator group

Description:

Participants aged 2 to less than 12 years old with or without clinically burdensome diarrhea.

Treatment:

Drug: Chenodeoxycholic acid