Maralixibat for constipation in children with cystic fibrosis
Maralixibat in Patients With Cystic Fibrosis and Constipation, A Within-Subjects Pilot Study
We will try maralixibat for two weeks to see if it softens stools and makes bowel movements easier in children with cystic fibrosis who still have constipation despite their usual laxatives.
Quick facts
| Phase | Phase2; Phase3 |
|---|---|
| Study type | Interventional |
| Enrollment | 20 (estimated) |
| Ages | 1 Year to 18 Years |
| Sex | All |
| Sponsor | Children's Hospital Los Angeles Academic / other |
| Locations | 1 site (Los Angeles, California) |
| Trial ID | NCT06413368 on ClinicalTrials.gov |
What this trial studies
This open-label, within-subject pilot enrolls 20 children with cystic fibrosis and chronic constipation and adds maralixibat to each participant's stable laxative regimen for two weeks. Maralixibat is an ileal bile acid transporter inhibitor that increases bile acids reaching the colon, which may increase intestinal secretion, speed transit, and soften stool. Families record stool consistency (Bristol Stool Scale) and ease of defecation before and during treatment to compare baseline and on-treatment outcomes. The trial is designed to generate preliminary evidence on symptom change and to inform feasibility and design of larger controlled studies.
Who should consider this trial
Good fit: Children aged 1 to 18 years with genetically or sweat-test confirmed cystic fibrosis and chronic constipation (Bristol Stool Scale score less than 3) who have been on a stable conventional constipation regimen for at least four weeks are ideal candidates.
Not a fit: Patients who already have adequate constipation control on current therapy, who have uncontrolled fat-soluble vitamin deficiencies, or who cannot take maralixibat due to allergy are unlikely to benefit from joining this pilot.
Why it matters
Potential benefit: If successful, maralixibat could provide a new adjunctive option to soften stool and make bowel movements easier for children with CF who do not get enough relief from standard laxatives.
How similar studies have performed: IBAT inhibitors have shown stool-softening effects in other conditions, but using maralixibat specifically for CF-related constipation is largely untested and remains preliminary.
Eligibility criteria
Show full inclusion / 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.
Where this trial is running
Los Angeles, California
- Children's Hospital Los Angeles — Los Angeles, California, United States (Recruiting)
Study contacts
- Principal investigator: Jaya Punati, MD — Children's Hospital Los Angeles
- Study coordinator: Jaya Punati, MD
- Email: jpunati@chla.usc.edu
- Phone: 3233615924
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.