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

NCT06260748 | Withdrawn Phase 3 DMC FDA Drug

• 2 other identifiers: LBI-CDCA-0012023-505759-29-00
• Study Type: interventional
• Target: N/A
• Locations: 1 country
• Sites: 1

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.

Conditions

Cerebrotendinous Xanthomatoses

Outcome Measures

Primary Outcomes (1)

• 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 Outcomes (9)

• Change in plasma cholestanol levels from baseline to Week 12

  Up to 12 weeks

• Change in urine and/or plasma bile alcohol levels from baseline to Week 12

  Up to 12 weeks

• Incidence of, severity/intensity of, and relationship to study drug of AEs

  Up to 20 weeks

• Incidence of, severity/intensity of, and relationship to study drug of SAEs

  Up to 20 weeks

• Incidence of, severity/intensity of AESIs

  Up to 20 weeks

Study Arms (3)

Group 1 Diarrhea-Evaluable

EXPERIMENTAL

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.

Drug: Chenodeoxycholic acid; Drug: Chenodeoxycholic acid Placebo

Group 2a Non-Diarrhea Evaluable

ACTIVE COMPARATOR

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

Drug: Chenodeoxycholic acid

Group 2b Pediatrics

ACTIVE COMPARATOR

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

Drug: Chenodeoxycholic acid

Interventions

Chenodeoxycholic acid DRUG

250mg capsules

Also known as: CDCA

Group 1 Diarrhea-Evaluable; Group 2a Non-Diarrhea Evaluable; Group 2b Pediatrics

Chenodeoxycholic acid Placebo DRUG

Placebo to match

Group 1 Diarrhea-Evaluable

Eligibility Criteria

• Age: 2 Years - 75 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• Leadiant Biosciences, Inc.: lead

Study Sites (1)

Sheba Medical Center

Tel Litwinsky, Israel

Location

MeSH Terms

Conditions

Xanthomatosis, Cerebrotendinous

Interventions

Chenodeoxycholic Acid

Condition Hierarchy (Ancestors)

Lipid Metabolism, Inborn Errors; Metabolism, Inborn Errors; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Lipid Metabolism Disorders; Metabolic Diseases; Nutritional and Metabolic Diseases; Xanthomatosis

Intervention Hierarchy (Ancestors)

Deoxycholic Acid; Cholic Acids; Bile Acids and Salts; Steroids; Fused-Ring Compounds; Polycyclic Compounds; Cholanes

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: February 1, 2024
• First Posted: February 15, 2024
• Study Start: May 27, 2024
• Primary Completion: November 30, 2025
• Study Completion: December 31, 2025
• Last Updated: November 18, 2024

Record last verified: 2024-11

Locations

IL (1)