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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

No Placebo-Only Group

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.

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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:

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I am not allergic to the study drugs or their ingredients.

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I do not have an uncontrolled deficiency in vitamins A, E, D, or K.

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My chronic constipation is managed and I have normal stool consistency.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ baseline - 3 weeks

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

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

Change in stool consistency by 1 point in Bristol scale or transition to Bristol scale > 3 after study drug

Secondary study objectives

Change in subjective scoring in ease of stooling with the addition of Maralixibat to a conventional constipation medication regimen via subjective questionnaire.