Maralixibat in Patients With Cystic Fibrosis and Constipation (NCT06413368) | Clinical Trial Compass
RecruitingPhase 2/3
Maralixibat in Patients With Cystic Fibrosis and Constipation
United States20 participantsStarted 2025-04-09
Plain-language 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.
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.
Who can participate
Age range
1 Year – 18 Years
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
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.
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Maralixibat was originally developed for liver conditions in children — given that this trial is now testing it for constipation in people with cystic fibrosis specifically, what do we know so far about how safe it is for CF patients?
2Since this is a Phase 2/3 trial, what does that mean for how much we already know about whether this drug actually works for CF-related constipation compared to the treatments I might already be using?
3The trial measures success using the Bristol Stool Scale to track changes in stool consistency — is that level of improvement meaningful enough in your view to be worth participating in a study, versus just adjusting my current CF bowel management plan?
4Constipation in cystic fibrosis can sometimes be related to DIOS or other serious bowel complications — would joining this trial still allow my care team to treat those issues the usual way if they came up while I'm enrolled?
5Before considering this trial, are there standard treatments or adjustments to my current CF regimen — like enzyme dosing or hydration — that we haven't fully tried yet that might help my constipation without the added demands of being in a study?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
What they're measuring
1
Change in Stool Consistency Measured by the Bristol Stool Scale