Maralixibat for constipation in children with cystic fibrosis

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

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.

Why it matters

Who can join: the full rules

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 it's running

• 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

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How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.