Maralixibat for Cystic Fibrosis and Constipation
What You Need to Know Before You Apply
What is the purpose of this trial?
Chronic constipation is a feature of children with cystic fibrosis (CF). This is postulated to be a result of inhibition of secretory activity of the gastrointestinal luminal cells due to ineffective chloride channel function. Typical laxatives that work as osmotic agents fail to produce adequate relief in this population.Maralixibat is a non-systemic bile acid transport inhibitor (IBATi) that acts by interrupting bile acid reabsorption in the ileum thus interrupting the normal enterohepatic circulation. This interruption results in a larger volume of bile acids reaching the colon and being excreted in stool. Bile acids are known to decrease bowel transit time, increase mucosal permeability and secretions, as well as alter gut microbiota resulting in diarrhea.The overarching hypothesis of the study is that Maralixibat will improve stool consistency in children (Age \<18 years) with cystic fibrosis and constipation (Bristol Stool Scale \<4). Specifically, we aim to test the hypothesis that IBATi improves the consistency of stool to Bristol scale \>4 in children with CF and constipation.We will recruit a total of 20 patients with CF and constipation (defined as Bristol Stool Scale \<4 for 1 week prior to enrollment while on a stable laxative regimen for at least 4 weeks.) Design is a 'Within-Subjects' study by which each enrolled patient will take Maralixibat for 2 weeks total in addition to their stable laxative regimen during the study. Stool consistency \& ease of defecation will be recorded before and during the study period by families of enrolled patients via materials provided by the investigators. Stool consistency and ease of defecation will be compared before and after initiation of Maralixibat.The primary endpoint: Improvement in stool consistency to Bristol scale \>4 in children with CF and constipation. The secondary endpoint: Improvement in ease of defecation in children with CF and constipation. This will be measured via survey using a standardized scale (Bristol Stool Scale) and questionnaires developed by the research team. Analysis will involve comparison of pre-intervention to post-intervention stool consistency \& survey
Will I have to stop taking my current medications?
No, you will not have to stop taking your current medications. The trial requires that you stay on your stable laxative regimen while taking Maralixibat.
Who Is on the Research Team?
Jaya Punati, MD
Principal Investigator
Children's Hospital Los Angeles
Are You a Good Fit for This Trial?
This trial is for children under 18 with cystic fibrosis who suffer from chronic constipation, despite being on a stable laxative regimen for at least four weeks. They must have a Bristol Stool Scale score of less than 4 for one week prior to enrollment.Inclusion Criteria
Exclusion Criteria
Timeline for a Trial Participant
Screening
Participants are screened for eligibility to participate in the trial
Baseline
Participants maintain a stable laxative regimen for at least 4 weeks prior to starting Maralixibat
Treatment
Participants receive Maralixibat in addition to their stable laxative regimen
Follow-up
Participants are monitored for changes in stool consistency and ease of defecation after treatment
What Are the Treatments Tested in This Trial?
Interventions
- Maralixibat
Maralixibat is already approved in United States, European Union, Canada for the following indications:
Find a Clinic Near You
Who Is Running the Clinical Trial?
Children's Hospital Los Angeles
Lead Sponsor