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

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

Get a concise snapshot of the trial, including recruitment status, study phase, enrollment targets, and key timeline milestones.

Recruitment Status

WITHDRAWN

Clinical Phase

PHASE3

Study Classification

INTERVENTIONAL

Study Start Date

2024-05-27

Study Completion Date

2025-12-31

Brief Summary

Review the sponsor-provided synopsis that highlights what the study is about and why it is being conducted.

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.

Related Clinical Trials

Explore similar clinical trials based on study characteristics and research focus.

Safety and Efficacy of CDCA in CTX Chenodeoxycholic Acid (CDCA) in Patients Affected by Cerebrotendinous Xanthomatosis (CTX)

NCT05499026

Cerebrotendinous Xanthomatoses

COMPLETED

Study to Evaluate Patients With Cerebrotendinous Xanthomatosis (RESTORE)

NCT04270682

CTX

COMPLETED PHASE3

Phase II Study of Cholesterol- and Cholestanol-Free Diet, Lovastatin, and Chenodeoxycholic Acid for Cerebrotendinous Xanthomatosis

NCT00004346

Cerebrotendinous Xanthomatosis

UNKNOWN PHASE2

Cholestanol in Humans

NCT00018694

Cerebrotendinous Xanthomatosis

WITHDRAWN NA

Obeticholic Acid in Bile Acid Diarrhoea

NCT01585025

Primary Bile Acid Malabsorption Secondary Bile Acid Malabsorption Chronic Diarrhoea

COMPLETED PHASE2

Detailed Description

Dive into the extended narrative that explains the scientific background, objectives, and procedures in greater depth.

Conditions

See the medical conditions and disease areas that this research is targeting or investigating.

Cerebrotendinous Xanthomatoses

Study Design

Understand how the trial is structured, including allocation methods, masking strategies, primary purpose, and other design elements.

Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

QUADRUPLE

Participants Caregivers Investigators Outcome Assessors

Study Groups

Review each arm or cohort in the study, along with the interventions and objectives associated with them.

Group 1 Diarrhea-Evaluable

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 Type EXPERIMENTAL

Chenodeoxycholic acid

Intervention Type DRUG

250mg capsules

Chenodeoxycholic acid Placebo

Intervention Type DRUG

Placebo to match

Group 2a Non-Diarrhea Evaluable

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

Group Type ACTIVE_COMPARATOR

Chenodeoxycholic acid

Intervention Type DRUG

250mg capsules

Group 2b Pediatrics

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

Group Type ACTIVE_COMPARATOR

Chenodeoxycholic acid

Intervention Type DRUG

250mg capsules

Interventions

Learn about the drugs, procedures, or behavioral strategies being tested and how they are applied within this trial.

Chenodeoxycholic acid

250mg capsules

Intervention Type DRUG

Chenodeoxycholic acid Placebo

Placebo to match

Intervention Type DRUG

Other Intervention Names

Discover alternative or legacy names that may be used to describe the listed interventions across different sources.

CDCA

Eligibility Criteria

Check the participation requirements, including inclusion and exclusion rules, age limits, and whether healthy volunteers are accepted.

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

Minimum Eligible Age

2 Years

Maximum Eligible Age

75 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No