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Maralixibat in Patients With Cystic Fibrosis and Constipation
Maralixibat in Patients With Cystic Fibrosis and Constipation, A Within-Subjects Pilot Study
Who can join
Ages 1 to 18 · All sexes
Full eligibility 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.
About the 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.
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.
What is being tested
- Maralixibat 9.5 MG/ML [Livmarli] (drug)
Sponsor: Children's Hospital Los Angeles · Participants: 20 · Started: Apr 9, 2025
Contact the study team
- Jaya Punati, MD · Phone: 3233615924
Official record on ClinicalTrials.gov — NCT06413368
Locations in the U.S.
| California | Children's Hospital Los Angeles, Los Angeles |
Conditions
From ClinicalTrials.gov, data retrieved Sep 30, 2026. Each study sets its own eligibility; the study team decides who can join.