NCT06413368 · Children's Hospital Los Angeles
Maralixibat in Patients With Cystic Fibrosis and Constipation
What this study is about
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.
View original scientific description
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.
Interventions
DRUG
Maralixibat 9.5 MG/ML [Livmarli]
Within Study subjects receiving 2 weeks of treatment with Maralixibat 9.5 MG/ML \[Livmarli\] and compare to baseline treatment.
Primary outcome measures
Change in Stool Consistency Measured by the Bristol Stool Scale
Time frame: baseline to 4 weeks
Constipation is defined as a Bristol Stool Scale (BSS) score of 1-3. The primary endpoint is the proportion of participants who demonstrate improvement in stool consistency, defined as either an increase of at least 1 point on the BSS from baseline or achieving a post-treatment BSS score greater than 3. The Bristol Stool Scale (BSS) is a clinical tool used to classify stool form into seven categories, ranging from very hard to entirely liquid. It helps quantify stool consistency and is commonly used in constipation and gastrointestinal studies.
Who can participate
This study lists these criteria on ClinicalTrials.gov. A study coordinator reviews eligibility during screening — this page does not determine whether you qualify.
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.
Where
- Los Angeles, California
Related conditions & keywords
Frequently asked questions
What is a clinical trial?
A clinical trial is a research study that tests new medical treatments, drugs, devices, or procedures to determine their safety and effectiveness. Trials are carefully designed and monitored to protect participants while advancing medical knowledge.
Is it safe to participate?
Clinical trials follow strict safety guidelines and ethical standards. Trials must be reviewed and approved, and participants are closely monitored by medical professionals throughout the study. You can withdraw at any time if you choose.
Will I be compensated?
Many clinical trials offer compensation for your time, travel expenses, and inconvenience. The specific compensation varies by study and will be discussed during the screening process. All study-related medical care is typically provided at no cost to participants.
Will I receive a placebo instead of treatment?
When effective treatment exists, participants typically receive either the standard treatment plus the study intervention, or the standard treatment plus placebo. You would not be denied effective care. Placebos are primarily used when no proven treatment is available, or in addition to standard care. Your trial consent form will clearly explain what treatments you may receive.
Can I leave a trial if I change my mind?
Absolutely. Participation in clinical trials is completely voluntary. You have the right to withdraw from the study at any time, for any reason, without penalty or loss of benefits to which you are otherwise entitled.
How long does a clinical trial last?
Trial duration varies widely depending on the study design and purpose. Some trials last just a few weeks, while others may continue for months or years. The study coordinator will provide specific timeline information during your screening call.
Data: ClinicalTrials.gov · synced Apr 21, 2026 · Source of record for eligibility and locations