Maralixibat in Patients With Cystic Fibrosis and Constipation
Trial statusRecruiting
Trial phasePhase 2, Phase 3
Trial typeInterventional
Biological sexAll
Age1-18
SponsorChildren's Hospital Los Angeles
About this trial
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.
Eligibility criteria
Qualifiers
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.
Disqualifiers
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.
Trial design
Treatments tested in this trial
- Maralixibat 9.5 MG/ML [Livmarli]
Treatment groups
20 Participants
are divided into 1 treatment groupSponsors and collaborators
Source ClinicalTrials.gov