Cerebrotendinous XanthomatosesPhase 3NCT06260748
A Study of Chenodeoxycholic Acid (CDCA) in Newly Diagnosed Participants With Cerebrotendinous Xanthomatosis (CTX)
A Phase 3 study testing Chenodeoxycholic acid in people with Cerebrotendinous Xanthomatoses, run by Leadiant Biosciences, Inc. at 1 site in Israel.
Trial 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. Read more about this study
- Who is it for?
- Ages 2 to 75 years
- Conditions the study is for
- Cerebrotendinous Xanthomatoses
- What is being tested?
- Chenodeoxycholic acid
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 0 participants
- Where
- 1 siteIsrael
- Study dates
- 27 May 2024to 31 Dec 2025
- Phase
- Phase 3
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 2 to 75 years
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- 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
Who cannot join
- 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 locations and contacts
1 site in 1 country.
About this study
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)
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
- Being tested
- Chenodeoxycholic acid, Chenodeoxycholic acid Placebo
- Medical subject headings
- Xanthomatosis, Cerebrotendinous
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Chenodeoxycholic acidDrug
Also called CDCA
250mg capsules
Chenodeoxycholic acid PlaceboDrug
Placebo to match
| Study group | Role | What they receive |
|---|---|---|
| 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. Receives: 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. Receives: Drug: Chenodeoxycholic acid |
| Group 2b Pediatrics | Active comparator | Participants aged 2 to less than 12 years old with or without clinically burdensome diarrhea. Receives: Drug: Chenodeoxycholic acid |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change from baseline at Week 4 in average number of stools with a BSS score of 6 or 7 per day
Measured: Up to 4 weeks
Secondary outcomes (9)
Change in plasma cholestanol levels from baseline to Week 12
All groups combined
Measured: Up to 12 weeks
Change in urine and/or plasma bile alcohol levels from baseline to Week 12
All groups combined
Measured: Up to 12 weeks
Incidence of, severity/intensity of, and relationship to study drug of AEs
Measured: Up to 20 weeks
Incidence of, severity/intensity of, and relationship to study drug of SAEs
Measured: Up to 20 weeks
Incidence of, severity/intensity of AESIs
Diarrhea and hepatic dysfunction
Measured: Up to 20 weeks
Incidence of, severity/intensity of, and changes in laboratory values
Measured: Up to 20 weeks
Incidence of, severity/intensity of, and changes in physical examination
Measured: Up to 20 weeks
Incidence of, severity/intensity of, and changes in vital signs
Measured: Up to 20 weeks
Number of participants with discontinuations due to AEs
Measured: Up to 20 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted15 Feb 2024
- Study start27 May 2024
- Primary completion30 Nov 2025 (estimated)
- Study completion31 Dec 2025 (estimated)
Why it stopped: Lack of feasibility
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Cerebrotendinous XanthomatosesStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 0 participants (actual)
Sponsor and investigators
- Lead sponsor
- Leadiant Biosciences, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06260748
- Sponsor's protocol ID
- LBI-CDCA-001
- Other IDs
- 2023-505759-29-00, 2023
- Status last verified
- Nov 2024
- First submitted
- 1 Feb 2024
- Last updated
- 18 Nov 2024
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 18 Nov 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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)
- ClinicalTrials.gov ID
- NCT06260748
- Acronym / sponsor ID
- LBI-CDCA-001
- Conditions
- Cerebrotendinous Xanthomatoses
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- WithdrawnReason stopped: Lack of feasibility
- Enrolment
- 0 participants (actual)
- Lead sponsor
- Leadiant Biosciences, Inc.Industry (pharma, biotech, devices)
- Study start
- 27 May 2024 (actual)
- Primary completion
- 30 Nov 2025 (estimated)
- Study completion
- 31 Dec 2025 (estimated)
- First posted
- 15 Feb 2024 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| Group 1 Diarrhea-Evaluable | Experimental | Drug: Chenodeoxycholic acid, Drug: Chenodeoxycholic acid PlaceboDiarrhea-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 2a Non-Diarrhea Evaluable | Active comparator | Drug: Chenodeoxycholic acidNon-diarrhea-evaluable participants aged 12-75 years old who do not have stable, clinically burdensome diarrhea or their diarrhea cannot be fully characterized. |
| Group 2b Pediatrics | Active comparator | Drug: Chenodeoxycholic acidParticipants aged 2 to less than 12 years old with or without clinically burdensome diarrhea. |
Who may qualify
- Ages
- 2 to 75 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (5 listed)
- 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
Key exclusion criteria (8 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Chenodeoxycholic acidAlso: CDCA250mg capsules | Drug | Group 1 Diarrhea-Evaluable, Group 2a Non-Diarrhea Evaluable, Group 2b Pediatrics |
| Chenodeoxycholic acid PlaceboPlacebo to match | Drug | Group 1 Diarrhea-Evaluable |
Primary outcome
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
9 secondary outcomes
Change in plasma cholestanol levels from baseline to Week 12
Time frame: Up to 12 weeks
Change in urine and/or plasma bile alcohol levels from baseline to Week 12
Time frame: Up to 12 weeks
Incidence of, severity/intensity of, and relationship to study drug of AEs
Time frame: Up to 20 weeks
Incidence of, severity/intensity of, and relationship to study drug of SAEs
Time frame: Up to 20 weeks
Incidence of, severity/intensity of AESIs
Time frame: Up to 20 weeks
Incidence of, severity/intensity of, and changes in laboratory values
Time frame: Up to 20 weeks
Incidence of, severity/intensity of, and changes in physical examination
Time frame: Up to 20 weeks
Incidence of, severity/intensity of, and changes in vital signs
Time frame: Up to 20 weeks
Number of participants with discontinuations due to AEs
Time frame: Up to 20 weeks
Results and safety
The study was withdrawn before enrolling participants, so there are no results.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 18 Nov 2024.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing Chenodeoxycholic acid in people with Cerebrotendinous Xanthomatoses, run by Leadiant Biosciences, Inc. at 1 site in Israel.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 5 inclusion and 8 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: withdrawn, as of the sponsor's last update on 18 Nov 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in Israel. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study was withdrawn before enrolling participants, so there are no results.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
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). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 18 Nov 2024.