NCT06413368 Maralixibat in Patients With Cystic Fibrosis and Constipation
| NCT ID | NCT06413368 |
| Status | Recruiting |
| Phase | Phase 2, Phase 3 |
| Sponsor | Children's Hospital Los Angeles |
| Condition | Constipation Chronic Idiopathic |
| Study Type | INTERVENTIONAL |
| Enrollment | 20 participants |
| Start Date | 2025-04-09 |
| Primary Completion | 2027-06-30 |
Eligibility & Interventions
Eligibility Fast-Check
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What to Expect as a Participant
You will actively receive the study intervention — which may be a drug, biologic, device, or procedure.
In Phase 2, researchers evaluate early signs of effectiveness. You may be randomized to receive the active treatment or a comparator. Monitoring continues closely.
This trial targets 20 participants in total. It began in 2025-04-09 with a primary completion date of 2027-06-30.
⚠ This information is for research awareness only. Always consult your physician before joining any clinical trial. Participation is voluntary and you may withdraw at any time.
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.
Eligibility Criteria
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.
Contact & Investigator
Jaya Punati, MD
PRINCIPAL INVESTIGATOR
Children's Hospital Los Angeles
Frequently Asked Questions
Who can join the NCT06413368 clinical trial?
This trial is open to participants of all sexes, aged 1 Year or older, up to 18 Years, studying Constipation Chronic Idiopathic. Full inclusion and exclusion criteria are listed in the Eligibility Criteria section. Always confirm your eligibility with the research team before applying.
What phase is the NCT06413368 trial and what does that mean for participants?
Phase 2 trials evaluate whether the treatment shows signs of effectiveness while continuing to monitor safety. More participants are enrolled than in Phase 1 to help refine the treatment protocol.
Is NCT06413368 currently recruiting?
Yes, NCT06413368 is actively recruiting participants. Contact the research team at jpunati@chla.usc.edu for enrollment information.
Where is the NCT06413368 trial being conducted?
This trial is being conducted at Los Angeles, United States.
Who is sponsoring the NCT06413368 clinical trial?
NCT06413368 is sponsored by Children's Hospital Los Angeles. The principal investigator is Jaya Punati, MD at Children's Hospital Los Angeles. The trial plans to enroll 20 participants.