Recruiting
Phase 2
Phase 3

Maralixibat

Sponsor:

Children's Hospital Los Angeles

Code:

NCT06413368

Conditions

Constipation Chronic Idiopathic

Cystic Fibrosis

Eligibility Criteria

Sex: All

Age: 1 - 18

Healthy Volunteers: Not accepted

Interventions

Maralixibat 9.5 MG/ML [Livmarli]

Study Details

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

Study ID

NCT06413368

Start date

Apr 9, 2025

Status verified date

Mar, 2026

Completion date

Jun 30, 2027

Anticipated

Primary completion date

Jun 30, 2027

Anticipated

Eligibility Criteria

Eligibility Criteria

Sex: All

Age: 1 - 18

Healthy Volunteers: Not accepted

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.

Study Design

Enrollment

20 participants

Anticipated

Intervention Model

Single group

Primary purpose

Treatment

Interventions and Outcome Measures

Arms

other: treatment arm

within - subjects study

Interventions

Maralixibat 9.5 MG/ML [Livmarli]

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

Primary outcome measure

  • Change in Stool Consistency Measured by the Bristol Stool Scale [ Time Frame: baseline to 4 weeks ]

Central Contacts and Locations

Central contacts

Jaya Punati, MD

3233615924jpunati@chla.usc.edu

Locations

Children's Hospital Los Angeles

Recruiting

Los Angeles, California, United States, 90027

Contacts

Jaya Punati, MD

jpunati@chla.usc.edu

Principal Investigator:

Jaya Punati, MD

More Information

Sponsor

Children's Hospital Los Angeles

Last update posted

Apr 21, 2026

Last verified

Mar, 2026

Keywords

  • Maralixibiat
  • Cystic Fibrosis
  • Chronic Constipation
  • Bristol Stool

Trial information was received from ClinicalTrials.gov and was last updated on 2026-09-09. This information was provided to ClinicalTrials.gov by Children's Hospital Los Angeles on 2026-04-21.