Constipation Chronic IdiopathicPhase 2/3NCT06413368
Maralixibat in Patients With Cystic Fibrosis and Constipation
A Phase 2/3 study testing Maralixibat 9.5 MG/ML [Livmarli] in people with Constipation Chronic Idiopathic and Cystic Fibrosis, run by Children's Hospital Los Angeles at 1 site in United States.
Trial summary
Chronic constipation is common in children with cystic fibrosis (CF), likely due to impaired chloride channel function that reduces intestinal secretions. Standard osmotic laxatives often provide inadequate relief in this population. Read more about this study
- Who is it for?
- Ages 1 year to 18 years
- Conditions the study is for
- Constipation Chronic Idiopathic, Cystic Fibrosis
- What is being tested?
- Maralixibat 9.5 MG/ML [Livmarli]
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 20 participants
- Where
- 1 siteUnited States
- Study dates
- 9 Apr 2025to 30 Jun 2027
- Phase
- Phase 2/3
- Are there results?
- Not yet: the study is under way
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 1 year to 18 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Ages 1 to 18 years.
- Proven diagnosis of Cystic Fibrosis confirmed by genetic testing or sweat chloride testing.
- Proven diagnosis of chronic constipation, defined as a Bristol Stool Scale (BSS) score <3 while on a stable conventional constipation therapy regimen.
- Stable conventional constipation medication regimen (no medication changes or dose adjustments) for at least 4 weeks prior to enrollment. Conventional therapy may include stool softeners, stimulant laxatives, or dietary interventions.
Who cannot join
- Uncontrolled fat-soluble vitamin deficiency (Vitamin A, D, E, or K).
- Changes to conventional constipation medication regimen within 4 weeks prior to initiation of Maralixibat.
- Adequately treated chronic constipation, defined as a Bristol Stool Scale (BSS) score >3 on the current regimen.
- Known allergy or sensitivity to Maralixibat or any study-related ingredients.
- Inability or unwillingness of the participant or legal guardian/representative to provide written informed consent.
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
United States1 site, 1 recruiting
- Recruiting
Children's Hospital Los Angeles
Los Angeles, California, 90027
Jaya Punati, MD, jpunati@chla.usc.edu
Jaya Punati, MD
About this study
Official title: Maralixibat in Patients With Cystic Fibrosis and Constipation, A Within-Subjects Pilot Study
Chronic constipation is common in children with cystic fibrosis (CF), likely due to impaired chloride channel function that reduces intestinal secretions. Standard osmotic laxatives often provide inadequate relief in this population.
Maralixibat is an ileal bile acid transporter inhibitor (IBATi) that increases the amount of bile acids reaching the colon. Bile acids can enhance intestinal secretion, reduce transit time, and soften stool. This study will evaluate whether Maralixibat improves stool consistency in children with CF who experience constipation.
Read more
We will enroll 20 children with CF and constipation, defined as a Bristol Stool Scale score <4 for at least one week while on a stable laxative regimen. Each participant will receive Maralixibat for two weeks in addition to their usual laxatives. Families will record stool consistency and ease of defecation before and during treatment.
The primary objective is to determine whether Maralixibat improves stool consistency to a Bristol Stool Scale score >4. The secondary objective is to assess changes in ease of defecation using standardized questionnaires.
Read the detailed description
Constipation is a frequent gastrointestinal complication in children with cystic fibrosis (CF). Impaired CFTR-mediated chloride and water secretion leads to dehydrated intestinal contents, slowed transit, and difficulty with stool passage. Despite routine use of osmotic laxatives, many children with CF continue to experience hard stools, abdominal discomfort, and incomplete evacuation, highlighting the need for alternative therapeutic approaches.
This study will evaluate the effect of Maralixibat on stool consistency and ease of defecation in children with CF who meet criteria for constipation while on a stable laxative regimen. The study uses a within-subjects design in which each participant serves as their own control. After a baseline observation period, participants will receive Maralixibat for two weeks in addition to their existing constipation management. Families will record stool characteristics and defecation symptoms using standardized tools provided by the study team.
Changes in stool consistency and ease of defecation will be assessed by comparing pre-treatment and treatment-period data. The study is designed to generate preliminary evidence regarding the potential utility of IBAT inhibition as an adjunctive therapy for constipation in pediatric CF patients and to inform the feasibility and design of future controlled trials.
- Conditions
- Constipation Chronic Idiopathic, Cystic Fibrosis
- Being tested
- Maralixibat 9.5 MG/ML [Livmarli]
- Keywords
- Maralixibiat, Cystic Fibrosis, Chronic Constipation, Bristol Stool
- Medical subject headings
- Cystic Fibrosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Maralixibat 9.5 MG/ML [Livmarli]Drug
Within Study subjects receiving 2 weeks of treatment with Maralixibat 9.5 MG/ML [Livmarli] and compare to baseline treatment.
| Study group | Role | What they receive |
|---|---|---|
| treatment arm | Other | within - subjects study Receives: Drug: Maralixibat 9.5 MG/ML [Livmarli] |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change in Stool Consistency Measured by the Bristol Stool Scale
Constipation is defined as a Bristol Stool Scale (BSS) score of 1-3. The primary endpoint is the proportion of participants who demonstrate improvement in stool consistency, defined as either an increase of at least 1 point on the BSS from baseline or achieving a post-treatment BSS score greater than 3. The Bristol Stool Scale (BSS) is a clinical tool used to classify stool form into seven categories, ranging from very hard to entirely liquid. It helps quantify stool consistency and is commonly used in constipation and gastrointestinal studies.
Measured: baseline to 4 weeks
Secondary outcomes (1)
Change in subjective scoring in ease of stooling with the addition of Maralixibat to a conventional constipation medication regimen via subjective questionnaire.
Maralixibat inhibits baseline absorption which in turn results in looser stools by osmosis.
We will use a questionnaire to record subjective report of ease of stooling by patients from baseline prior to intervention using a Likert score of 1-5
- - cannot stool
- - Difficulty stooling
- - neither easy nor difficult
- - Easier stooling with medication
- - No issues with stooling
Measured: Baseline - 3 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted14 May 2024
- Study start9 Apr 2025
- Primary completion30 Jun 2027 (estimated)
- Study completion30 Jun 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Constipation Chronic IdiopathicStudies 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 2/3
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 20 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Children's Hospital Los Angeles
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Jaya Punati, Children's Hospital Los Angeles
- Investigators
- Jaya Punati, MD, Principal investigator, Children's Hospital Los Angeles
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06413368
- Sponsor's protocol ID
- CHLA-23-00352
- Status last verified
- Mar 2026
- First submitted
- 9 May 2024
- Last updated
- 21 Apr 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 21 Apr 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Maralixibat in Patients With Cystic Fibrosis and Constipation, A Within-Subjects Pilot Study
- ClinicalTrials.gov ID
- NCT06413368
- Acronym / sponsor ID
- CHLA-23-00352
- Conditions
- Constipation Chronic Idiopathic, Cystic Fibrosis
- Keywords
- Maralixibiat, Cystic Fibrosis, Chronic Constipation, Bristol Stool
- Phase
- Phase 2/3
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 20 participants (estimated)
- Lead sponsor
- Children's Hospital Los AngelesUniversity, hospital or other
- Study start
- 9 Apr 2025 (actual)
- Primary completion
- 30 Jun 2027 (estimated)
- Study completion
- 30 Jun 2027 (estimated)
- First posted
- 14 May 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.
Chronic constipation is common in children with cystic fibrosis (CF), likely due to impaired chloride channel function that reduces intestinal secretions. Standard osmotic laxatives often provide inadequate relief in this population.
Maralixibat is an ileal bile acid transporter inhibitor (IBATi) that increases the amount of bile acids reaching the colon. Bile acids can enhance intestinal secretion, reduce transit time, and soften stool. This study will evaluate whether Maralixibat improves stool consistency in children with CF who experience constipation.
We will enroll 20 children with CF and constipation, defined as a Bristol Stool Scale score <4 for at least one week while on a stable laxative regimen. Each participant will receive Maralixibat for two weeks in addition to their usual laxatives. Families will record stool consistency and ease of defecation before and during treatment.
The primary objective is to determine whether Maralixibat improves stool consistency to a Bristol Stool Scale score >4. The secondary objective is to assess changes in ease of defecation using standardized questionnaires.
Detailed description
Constipation is a frequent gastrointestinal complication in children with cystic fibrosis (CF). Impaired CFTR-mediated chloride and water secretion leads to dehydrated intestinal contents, slowed transit, and difficulty with stool passage. Despite routine use of osmotic laxatives, many children with CF continue to experience hard stools, abdominal discomfort, and incomplete evacuation, highlighting the need for alternative therapeutic approaches.
This study will evaluate the effect of Maralixibat on stool consistency and ease of defecation in children with CF who meet criteria for constipation while on a stable laxative regimen. The study uses a within-subjects design in which each participant serves as their own control. After a baseline observation period, participants will receive Maralixibat for two weeks in addition to their existing constipation management. Families will record stool characteristics and defecation symptoms using standardized tools provided by the study team.
Changes in stool consistency and ease of defecation will be assessed by comparing pre-treatment and treatment-period data. The study is designed to generate preliminary evidence regarding the potential utility of IBAT inhibition as an adjunctive therapy for constipation in pediatric CF patients and to inform the feasibility and design of future controlled trials.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| treatment arm | Other | Drug: Maralixibat 9.5 MG/ML [Livmarli]within - subjects study |
Who may qualify
- Ages
- 1 year to 18 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (4 listed)
- Ages 1 to 18 years.
- Proven diagnosis of Cystic Fibrosis confirmed by genetic testing or sweat chloride testing.
- Proven diagnosis of chronic constipation, defined as a Bristol Stool Scale (BSS) score <3 while on a stable conventional constipation therapy regimen.
- Stable conventional constipation medication regimen (no medication changes or dose adjustments) for at least 4 weeks prior to enrollment. Conventional therapy may include stool softeners, stimulant laxatives, or dietary interventions.
Key exclusion criteria (5 listed)
- Uncontrolled fat-soluble vitamin deficiency (Vitamin A, D, E, or K).
- Changes to conventional constipation medication regimen within 4 weeks prior to initiation of Maralixibat.
- Adequately treated chronic constipation, defined as a Bristol Stool Scale (BSS) score >3 on the current regimen.
- Known allergy or sensitivity to Maralixibat or any study-related ingredients.
- Inability or unwillingness of the participant or legal guardian/representative to provide written informed consent.
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 |
|---|---|---|
| Maralixibat 9.5 MG/ML [Livmarli]Within Study subjects receiving 2 weeks of treatment with Maralixibat 9.5 MG/ML [Livmarli] and compare to baseline treatment. | Drug | treatment arm |
Primary outcome
Change in Stool Consistency Measured by the Bristol Stool Scale
Time frame: baseline to 4 weeks
Constipation is defined as a Bristol Stool Scale (BSS) score of 1-3. The primary endpoint is the proportion of participants who demonstrate improvement in stool consistency, defined as either an increase of at least 1 point on the BSS from baseline or achieving a post-treatment BSS score greater than 3. The Bristol Stool Scale (BSS) is a clinical tool used to classify stool form into seven categories, ranging from very hard to entirely liquid. It helps quantify stool consistency and is commonly used in constipation and gastrointestinal studies.
1 secondary outcome
Change in subjective scoring in ease of stooling with the addition of Maralixibat to a conventional constipation medication regimen via subjective questionnaire.
Time frame: Baseline - 3 weeks
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 21 Apr 2026.
- Individual participant dataSharing planned: No
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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.
Jaya Punati, MD principal investigator
Children's Hospital Los Angeles
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2/3 study testing Maralixibat 9.5 MG/ML [Livmarli] in people with Constipation Chronic Idiopathic and Cystic Fibrosis, run by Children's Hospital Los Angeles at 1 site in United States.
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 4 inclusion and 5 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: looking for participants now, as of the sponsor's last update on 21 Apr 2026. 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 United States. 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 combined phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
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?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Jaya Punati, MD (3233615924, jpunati@chla.usc.edu), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Change in Stool Consistency Measured by the Bristol Stool Scale (time frame: baseline 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 21 Apr 2026.