Skip to main content
OpenTrials
Recruiting

NCT Number: NCT06413368

Maralixibat in Patients With Cystic Fibrosis and Constipation

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.

Recruiting

Interested in participating?

Request Info

Key information

Conditions

Constipation Chronic Idiopathic Congenital, Hereditary, and Neonatal Diseases and Abnormalities Cystic Fibrosis Digestive System Diseases Genetic Diseases, Inborn Infant, Newborn, Diseases Lung Diseases Pancreatic Diseases Respiratory Tract Diseases

Age range

1 year–18 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 2 / Phase 3

Primary location

Children's Hospital Los Angeles

Los Angeles, California, 90027, United States

Location status: Recruiting

Location contact

Jaya Punati, MD

CONTACT

[email protected]

Jaya Punati, MD

PRINCIPAL_INVESTIGATOR

About this study

Constipation is a frequent gastrointestinal complication in children with cystic fibrosis (CF). Impaired CFTR-mediated chloride and water secretion leads to dehydrated intestinal contents, slowed transit, and difficulty with stool passage. Despite routine use of osmotic laxatives, many children with CF continue to experience hard stools, abdominal discomfort, and incomplete evacuation, highlighting the need for alternative therapeutic approaches.

This study will evaluate the effect of Maralixibat on stool consistency and ease of defecation in children with CF who meet criteria for constipation while on a stable laxative regimen. The study uses a within-subjects design in which each participant serves as their own control. After a baseline observation period, participants will receive Maralixibat for two weeks in addition to their existing constipation management. Families will record stool characteristics and defecation symptoms using standardized tools provided by the study team.

Changes in stool consistency and ease of defecation will be assessed by comparing pre-treatment and treatment-period data. The study is designed to generate preliminary evidence regarding the potential utility of IBAT inhibition as an adjunctive therapy for constipation in pediatric CF patients and to inform the feasibility and design of future controlled trials.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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.

Treatment and study plan

Maralixibat 9.5 MG/ML [Livmarli]

Drug

Within Study subjects receiving 2 weeks of treatment with Maralixibat 9.5 MG/ML [Livmarli] and compare to baseline treatment.

Primary outcomes

  1. Change in Stool Consistency Measured by the Bristol Stool Scale

    Time frame: baseline to 4 weeks

    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.

Secondary outcomes

  1. Change in subjective scoring in ease of stooling with the addition of Maralixibat to a conventional constipation medication regimen via subjective questionnaire.

    Time frame: Baseline - 3 weeks

    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

    • - cannot stool
    • - Difficulty stooling
    • - neither easy nor difficult
    • - Easier stooling with medication
    • - No issues with stooling

Study contacts

Contact information is provided by the study sponsor or research team.

Jaya Punati, MD

CONTACT

[email protected]

3233615924

Sponsors and collaborators

Lead sponsor

Children's Hospital Los Angeles

Other

Registry information

Official study title

Maralixibat in Patients With Cystic Fibrosis and Constipation, A Within-Subjects Pilot Study

Important dates

Study start
2025
Primary completion
2027
Study completion
2027
First posted
May 14, 2024
Registry last updated
Apr 21, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

Related clinical trials

Published trials that share one or more normalized conditions with this study.