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WithdrawnNCT06260748Updated Nov 18, 2024

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

A Phase 3 interventional study of Chenodeoxycholic acid and Chenodeoxycholic acid Placebo in Cerebrotendinous Xanthomatoses, sponsored by Leadiant Biosciences, Inc.. Withdrawn at 1 site in Israel. Open to participants aged 2 Years to 75 Years. Per ClinicalTrials.gov, last updated 2024-11-18.

Sponsored by Leadiant Biosciences, Inc. · Phase 3, Interventional, and Treatment

Why this study was withdrawn
Lack of feasibility
Phase
Phase 3
Study type
Interventional
Enrollment
0
Allocation
Randomized
Ages
2 Years to 75 Years
Sex
All
01

Study 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.

02

Conditions studied

  • Cerebrotendinous Xanthomatoses
03

In context

Lead sponsor

Leadiant Biosciences, Inc. is the lead sponsor of 5 studies on the registry; none are open to participants now.

Counted across the registry records on this site, refreshed daily.

04

Who can participate

Ages eligible
2 Years to 75 Years
Sexes eligible
All
Accepts healthy volunteers
No

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

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
05

Study design

Phase
Phase 3
Primary purpose
Treatment
Allocation
Randomized
Intervention model
Parallel assignment
Masking
Quadruple (Participant, Care provider, Investigator, Outcomes assessor)
Enrollment
0 participants (actual)

Study arms

  • Experimental
    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.

    Drug: Chenodeoxycholic acid · Drug: Chenodeoxycholic acid Placebo

  • Active comparator
    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.

    Drug: Chenodeoxycholic acid

  • Active comparator
    Group 2b Pediatrics

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

    Drug: Chenodeoxycholic acid

Interventions

  • DrugChenodeoxycholic acid

    250mg capsules

    Also known as: CDCA

  • DrugChenodeoxycholic acid Placebo

    Placebo to match

06

What researchers measure

Primary outcomes

  1. Change from baseline at Week 4 in average number of stools with a BSS score of 6 or 7 per day

    Time frame: Up to 4 weeks

Secondary outcomes

  1. Change in plasma cholestanol levels from baseline to Week 12

    All groups combined

    Time frame: Up to 12 weeks

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

    All groups combined

    Time frame: Up to 12 weeks

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

    Time frame: Up to 20 weeks

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

    Time frame: Up to 20 weeks

  5. Incidence of, severity/intensity of AESIs

    Diarrhea and hepatic dysfunction

    Time frame: Up to 20 weeks

  6. Incidence of, severity/intensity of, and changes in laboratory values

    Time frame: Up to 20 weeks

  7. Incidence of, severity/intensity of, and changes in physical examination

    Time frame: Up to 20 weeks

  8. Incidence of, severity/intensity of, and changes in vital signs

    Time frame: Up to 20 weeks

  9. Number of participants with discontinuations due to AEs

    Time frame: Up to 20 weeks

07

Study locations

1 site
  • Sheba Medical Center
    Tel HaShomer, Israel
08

Updates

Tracking since Sep 25, 2026
No changes since tracking began. The registry record was last updated on Nov 18, 2024, before this site started recording changes on Sep 25, 2026. Its history is on ClinicalTrials.gov ↗
09

Registry details

Key details

Study ID
NCT06260748
Lead sponsor
Leadiant Biosciences, Inc.
Responsible party
Sponsor
First posted
Feb 15, 2024
Start date
May 27, 2024
Primary completion
Nov 30, 2025 (estimated)
Completion
Dec 31, 2025 (estimated)
Last update
Nov 18, 2024

Oversight

Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No
View the source record on ClinicalTrials.gov ↗

Not currently enrolling

This study is withdrawn, as verified in Nov 2024. You cannot join it, but the record below documents what was studied.

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Discussion

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Other studies in this condition

Xanthomatosis, Cerebrotendinous

Other studies from this sponsor

Leadiant Biosciences, Inc.

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