Maralixibat in Patients With Cystic Fibrosis and Constipation

NCT06413368 | Recruiting Phase 2 FDA Drug

• 1 other identifier: CHLA-23-00352
• Study Type: interventional
• Target: 20
• Locations: 1 country
• Sites: 1

Brief 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.

Conditions

Constipation Chronic Idiopathic; Cystic Fibrosis

Keywords

Maralixibiat; Cystic Fibrosis; Chronic Constipation; Bristol Stool

Outcome Measures

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.

  baseline to 4 weeks

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.

  Baseline - 3 weeks

Study Arms (1)

treatment arm

OTHER

within - subjects study

Drug: Maralixibat 9.5 MG/ML [Livmarli]

Interventions

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.

treatment arm

Eligibility Criteria

• Age: 1 Year - 18 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Children's Hospital Los Angeles: lead

Study Sites (1)

Children's Hospital Los Angeles

Los Angeles, California, 90027, United States

RECRUITING

MeSH Terms

Conditions

Cystic Fibrosis

Interventions

maralixibat

Condition Hierarchy (Ancestors)

Pancreatic Diseases; Digestive System Diseases; Lung Diseases; Respiratory Tract Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Infant, Newborn, Diseases

Study Officials

• Jaya Punati, MD

  Children's Hospital Los Angeles

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Jaya Punati, MD

CONTACT

3233615924; jpunati@chla.usc.edu

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Section Chief, General Gastroenterology; Clinical Operations Director

Study Record Dates

• First Submitted: May 9, 2024
• First Posted: May 14, 2024
• Study Start: April 9, 2025
• Primary Completion (Estimated): June 30, 2027
• Study Completion (Estimated): June 30, 2027
• Last Updated: April 21, 2026

Record last verified: 2026-03

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)