Maralixibat in Patients With Cystic Fibrosis and Constipation

Part of paid clinical trials in Los Angeles, California.

Sponsor
Children's Hospital Los Angeles
Study ID
NCT06413368
Phase
PHASE2/PHASE3
Status
Recruiting
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Conditions

  • Constipation Chronic Idiopathic
  • Cystic Fibrosis

Eligibility Criteria

Sex
ALL
Age
1 Year - 18 Years
Healthy Volunteers
Not accepted

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.

Study Details

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.

Key Dates

Start date
Apr 9, 2025
Status verified
Mar 2026
Primary completion
Jun 30, 2027
Completion
Jun 30, 2027

Study Design

Enrollment
20 participants (estimated)
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT

Arms

  • Other: treatment arm
    within - subjects study

Primary Outcome Measure

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

Central Contacts

Locations (1)

FacilityCityStateZIPSite coordinators
Children's Hospital Los AngelesLos AngelesCalifornia90027
Jaya Punati, MD
[email protected]
Jaya Punati, MD (PRINCIPAL_INVESTIGATOR)

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