Evaluating the long-term safety and outcomes of Livmarli in patients with liver diseases
Long-Term SafEty and Clinical Outcomes of LivmArli in Patients in the United States (LEAP-US)
This study is testing how safe and effective the medication Livmarli is for people with Alagille syndrome or Progressive familial intrahepatic cholestasis over a five-year period.
Quick facts
| Study type | Observational |
|---|---|
| Enrollment | 70 (estimated) |
| Sex | All |
| Sponsor | Mirum Pharmaceuticals, Inc. Industry-sponsored |
| Locations | 8 sites (Los Angeles, California and 7 other locations) |
| Trial ID | NCT06193928 on ClinicalTrials.gov |
What this trial studies
This is a 5-year, prospective, observational cohort study aimed at assessing the long-term safety and clinical outcomes of patients diagnosed with Alagille syndrome (ALGS) or Progressive familial intrahepatic cholestasis (PFIC) who are treated with Livmarli. Livmarli is a novel medication that works by inhibiting the ileal bile acid transporter, thereby reducing bile acid levels in the body. The study will include participants who have a confirmed diagnosis of ALGS or PFIC and are prescribed Livmarli, allowing researchers to gather data on its effectiveness and safety over time.
Who should consider this trial
Good fit: Ideal candidates for this study are patients with a clinically and/or genetically confirmed diagnosis of Alagille syndrome or Progressive familial intrahepatic cholestasis who have been prescribed Livmarli.
Not a fit: Patients who have previously participated in a clinical study involving Livmarli or have received an investigational drug within 30 days prior to the study may not benefit from this study.
Why it matters
Potential benefit: If successful, this study could provide valuable insights into the long-term safety and efficacy of Livmarli, potentially improving treatment options for patients with ALGS and PFIC.
How similar studies have performed: Other studies have shown promising results with similar approaches in treating cholestatic liver diseases, but this specific observational study is novel in its long-term focus on Livmarli.
Eligibility criteria
Show full inclusion / exclusion criteria
Inclusion Criteria: * A clinically and/or genetically confirmed ALGS diagnosis or PFIC diagnosis * Participant prescribed Livmarli Exclusion Criteria: * Refusal to provide informed consent/assent (if required by the local IRB) * Previously or currently on Livmarli through participation in a clinical study or expanded access program * Participants who have previously received an SBD or LT * Any condition or abnormalities that, in the opinion of the investigator, may interfere with the participant participating in or completing the study * Participants who have received an investigational drug within 30 days of the first dose of Livmarli
Where this trial is running
Los Angeles, California and 7 other locations
- Children's Hospital Los Angeles CHLA — Los Angeles, California, United States (Recruiting)
- Section of Gastroenterology, Hepatology and Nutrition, Department of Pediatrics and the Digestive Health Institute, Children's Hospital of Colorado and University of Colorado — Aurora, Colorado, United States (Recruiting)
- Children's Healthcare of Atlanta - Emory University School of Medicine — Atlanta, Georgia, United States (Recruiting)
- Children's Mercy Kansas City, Department of Gastroenterology, Section of Hepatology — Kansas City, Missouri, United States (Recruiting)
- Oregon Health and Science University, Division of Pediatric Gastroenterology, Department of Pediatrics — Portland, Oregon, United States (Recruiting)
- Children's Hospital of Philadelphia — Philadelphia, Pennsylvania, United States (Recruiting)
- Children Hospital of Pittsburgh — Pittsburgh, Pennsylvania, United States (Recruiting)
- University of Utah, Division of Pediatric Gastroenterology, Hepatology and Nutrition — Salt Lake City, Utah, United States (Recruiting)
Study contacts
- Study coordinator: Clinical Trials Mirum
- Email: Clinical Trials <clinicaltrials@mirumpharma.com>;
- Phone: +16506674085
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.