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
This trial does not accept healthy volunteersQualifiers
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
Design model
Single group
Treatments tested in this trial
Maralixibat 9.5 MG/ML [Livmarli]
DrugWithin Study subjects receiving 2 weeks of treatment with Maralixibat 9.5 MG/ML \[Livmarli\] and compare to baseline treatment.
Treatment groups
20 Participants
are divided into 1 treatment groupGroup A: treatment armOther 1 intervention
Trial outcomes
Primary outcomes
1
Change in Stool Consistency Measured by the Bristol Stool Scale
Constipation is defined as a Bristol Stool Scale (BSS) score of 1-3. The primary endpoint is the proportion of participants who demonstrate improvement in stool consistency, defined as either an increase of at least 1 point on the BSS from baseline or achieving a post-treatment BSS score greater than 3. The Bristol Stool Scale (BSS) is a clinical tool used to classify stool form into seven categories, ranging from very hard to entirely liquid. It helps quantify stool consistency and is commonly used in constipation and gastrointestinal studies.
Time frame
baseline to 4 weeksSecondary outcomes
1
Change in subjective scoring in ease of stooling with the addition of Maralixibat to a conventional constipation medication regimen via subjective questionnaire.
Maralixibat inhibits baseline absorption which in turn results in looser stools by osmosis. We will use a questionnaire to record subjective report of ease of stooling by patients from baseline prior to intervention using a Likert score of 1-5 1. \- cannot stool 2. \- Difficulty stooling 3. \- neither easy nor difficult 4. \- Easier stooling with medication 5. \- No issues with stooling
Time frame
Baseline - 3 weeksSponsors and contacts
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Source ClinicalTrials.gov