Maralixibat in Patients With Cystic Fibrosis and Constipation

Recruitment Status
RECRUITING
(See Contacts and Locations)Verified February 2026 by Children's Hospital Los Angeles
Sponsor
Children's Hospital Los Angeles
Information Provided by (Responsible Party)
Jaya Punati
Clinicaltrials.gov Identifier
NCT06413368
Other Study ID Numbers:
CHLA-23-00352
First Submitted
May 8, 2024
First Posted
May 13, 2024
Last Update Posted
April 20, 2026
Last Verified
February 2026

ClinicalTrials.gov processed this data on April 2026. Link to the current ClinicalTrials.gov record .

History of Changes

Study Details

Study 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.

Condition or DiseaseIntervention/Treatment
Constipation Chronic IdiopathicCystic Fibrosis
Drug: Maralixibat 9.5 MG/ML [Livmarli]

Study Design

Study TypeInterventional
Actual Enrollment20 participants
Design AllocationN/A
Interventional ModelSingle Group Assignment
MaskingNone (Open Label)
Primary PurposeTreatment
Official TitleMaralixibat in Patients With Cystic Fibrosis and Constipation, A Within-Subjects Pilot Study
Study Start DateApril 8, 2025
Actual Primary Completion Date1yr 1mo from now
Actual Study Completion Date1yr 1mo from now

Groups and Cohorts

Group/CohortIntervention/Treatment
treatment arm
within - subjects study
Drug: 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.

Outcome Measures

Primary Outcome Measures
  1. 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.
Secondary Outcome Measures
  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 1. \- cannot stool 2. \- Difficulty stooling 3. \- neither easy nor difficult 4. \- Easier stooling with medication 5. \- No issues with stooling

Eligibility Criteria

Ages Eligible for Study(Child, Adult)
Sexes Eligible for StudyAll
Accepts Healthy VolunteersNo
Inclusion Criteria
Inclusion Criteria
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.
Exclusion Criteria
Inclusion Criteria
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. Exclusion Criteria
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.

Contacts and Locations

Sponsors and CollaboratorsChildren's Hospital Los Angeles
Locations
Children's Hospital Los Angeles | Los Angeles California, United States, 90027
Investigators
Principal Investigator: Jaya Punati, MD, Children's Hospital Los Angeles