Maralixibat in Patients With Cystic Fibrosis and Constipation

Recruiting Phase 2 Phase 3 Interventional Study
Constipation Chronic Idiopathic Cystic Fibrosis
No Placebo Group Every participant receives an active treatment — no one gets a placebo. Pivotal Trial This treatment is in the last trial phase before FDA approval. Prior Safety Data This treatment has already been tested in at least one earlier human trial.
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At a Glance
Age
1 – 18
Sex
Any
Trial phase
Phase 2/3
Study type
Interventional
Purpose
Treatment
Participants needed
20 (estimated)
Sponsor
Children's Hospital Los Angeles · Other
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About This Trial
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 secreti…
Trial Locations
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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 c…
Contacts

Jaya Punati, MD

3233615924

jpunati@chla.usc.edu

CONTACT