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Bile Acid Transport Inhibitor

Maralixibat for Cystic Fibrosis and Constipation

Phase 2 & 3
Waitlist Available
Led By Jaya Punati, MD
Research Sponsored by Children's Hospital Los Angeles
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Proven diagnosis of Cystic Fibrosis (via genetic testing or sweat chloride testing)
Age between 1-18 years
Must not have
Allergy or sensitivity to the study drugs or their ingredients
Uncontrolled fat-soluble vitamin deficiency (Vitamin A, E, D, or K)
Timeline
Screening 3 weeks
Treatment Varies
Follow Up baseline - 3 weeks
Awards & highlights

Summary

"This trial aims to test whether a new medication, Maralixibat, can improve stool consistency in children under 18 years old with cystic fibrosis and constipation. The medication works by

Who is the study for?
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.
What is being tested?
The study tests Maralixibat's ability to improve stool consistency in these children. It's a 'Within-Subjects' pilot study where each child will add Maralixibat to their current laxative treatment for two weeks and track changes in stool consistency and ease of defecation.
What are the potential side effects?
Potential side effects may include diarrhea due to increased bile acids reaching the colon, which can also affect mucosal permeability, secretions, and gut microbiota.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
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I have been diagnosed with Cystic Fibrosis through genetic or sweat tests.
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I am between 1 and 18 years old.
Select...
I have chronic constipation despite treatment, with hard stools.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:
Select...
I am not allergic to the study drugs or their ingredients.
Select...
I do not have an uncontrolled deficiency in vitamins A, E, D, or K.
Select...
My chronic constipation is managed and I have normal stool consistency.

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~baseline - 3 weeks
This trial's timeline: 3 weeks for screening, Varies for treatment, and baseline - 3 weeks for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.
Primary outcome measures
Change in stool consistency by 1 point in Bristol scale or transition to Bristol scale > 3 after study drug
Secondary outcome measures
Change in subjective scoring in ease of stooling with the addition of Maralixibat to a conventional constipation medication regimen via subjective questionnaire.

Trial Design

1Treatment groups
Experimental Treatment
Group I: treatment armExperimental Treatment1 Intervention
within - subjects study

Find a Location

Who is running the clinical trial?

Children's Hospital Los AngelesLead Sponsor
237 Previous Clinical Trials
5,077,130 Total Patients Enrolled
1 Trials studying Cystic Fibrosis
240 Patients Enrolled for Cystic Fibrosis
Jaya Punati, MDPrincipal InvestigatorChildren's Hospital Los Angeles
~13 spots leftby Jun 2026
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