NCT06413368RecruitingPhase 2/3
Maralixibat in Patients With Cystic Fibrosis and Constipation
Maralixibat in Patients With Cystic Fibrosis and Constipation, A Within-Subjects Pilot Study
Source: ClinicalTrials.gov · Last updated 21 Apr 2026
Brief summary (registry, in English)
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.
- Status (registry)
- Recruiting
- Phase (registry)
- Phase 2/3
- Study type
- Interventional
- Enrollment
- 20 (estimated)
- First submitted
- 9 May 2024
- Start date
- 9 Apr 2025
- Primary completion date
- 30 Jun 2027
- Lead sponsor
- Children's Hospital Los Angeles
- Diseases
- Cystic fibrosis
- NCT number
- NCT06413368
What you commit to
- Study type: Interventional · Phase 2/3
- Planned participants: 20
- From 9 Apr 2025 to the primary completion date, 30 Jun 2027
- 1 site in 1 country
- Visits and tests: ask the trial team, the registry does not list them
Built from the registry fields of ClinicalTrials.gov. It describes the study, not your situation.
Who cannot take part
Copied verbatim from ClinicalTrials.gov and grouped by topic. Only the trial team decides who can take part.
Prior treatment
- Changes to conventional constipation medication regimen within 4 weeks prior to initiation of Maralixibat.
Other illness
- Known allergy or sensitivity to Maralixibat or any study-related ingredients.
Other
- Uncontrolled fat-soluble vitamin deficiency (Vitamin A, D, E, or K).
- Adequately treated chronic constipation, defined as a Bristol Stool Scale (BSS) score \>3 on the current regimen.
- Inability or unwillingness of the participant or legal guardian/representative to provide written informed consent.
Who the study is looking for
- 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.
Age in the registry: 1 year to 18 years · Sex: All · Does not accept healthy volunteers
Sites (1)
Sites in registry order. Pick your city on the disease page to sort them by distance.
| Site | City | Country | Site status |
|---|---|---|---|
| Children's Hospital Los Angeles Coordinator of this site? Confirm its status | Los Angeles | United States | Recruiting |
This page is information, not medical advice. Discuss any trial with your doctor.