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NCT06193928 · ClinicalTrials.gov registry record
Long-Term SafEty and Clinical Outcomes of LivmArli in Patients in the United States (LEAP-US)
A clinical trial of Alagille Syndrome and Progressive Familial Intrahepatic Cholestasis, sponsored by Mirum Pharmaceuticals.
- Recruiting
- Registry status
- 70
- Enrollment target
- 8
- Study locations
NCT06193928: Recruiting study of Alagille Syndrome and Progressive Familial Intrahepatic Cholestasis, sponsored by Mirum Pharmaceuticals.
NCT06193928 is a study of Alagille Syndrome and Progressive Familial Intrahepatic Cholestasis that is actively recruiting participants, run by Mirum Pharmaceuticals. The registered enrollment target is 70 participants, above the 45-participant average among 3 other Alagille Syndrome trials with a reported enrollment target (56% higher). The trial reports 8 study locations across 7 states. According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT06193928, a study of Alagille Syndrome and Progressive Familial Intrahepatic Cholestasis, is actively recruiting participants, sponsored by Mirum Pharmaceuticals.
- RECRUITING
- Registry status
- 70 participants
- Enrollment target
- 8
- Study locations
Study Summary
The objective of this 5-year, prospective, observational cohort study is to evaluate the long-term safety and clinical outcomes of patients with Alagille syndrome (ALGS) or Progressive familial intrahepatic cholestasis (PFIC) treated with Livmarli.
Primary Outcome
The dates, and reasons for the following first events (of this first endpoint) will be collected: Surgical Biliary Diversion (SBD), Liver Transplant (LT), and all-cause mortality. In addition, manifestations of clinically evident portal hypertension (CEPH) will be captured during each interval event assessments.
Conditions Studied
Interventions
- DRUG Livmarli
Study Locations (8)
Pennsylvania
- Children's Hospital of Philadelphia - Philadelphia
- Children Hospital of Pittsburgh - Pittsburgh
California
- Children's Hospital Los Angeles CHLA - Los Angeles
Colorado
- Section of Gastroenterology, Hepatology and Nutrition, Department of Pediatrics and the Digestive Health Institute, Children's Hospital of Colorado and University of Colorado - Aurora
Georgia
- Children's Healthcare of Atlanta - Emory University School of Medicine - Atlanta
Missouri
- Children's Mercy Kansas City, Department of Gastroenterology, Section of Hepatology - Kansas City
Oregon
- Oregon Health and Science University, Division of Pediatric Gastroenterology, Department of Pediatrics - Portland
Utah
- University of Utah, Division of Pediatric Gastroenterology, Hepatology and Nutrition - Salt Lake City
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 70 participants |
| Start Date | 2023-09-21 |
| Est. Completion | 2030-09-20 |
What NCT06193928 shows while recruiting
NCT06193928 is an observational study that tracks outcomes without assigning an intervention. The registry caps enrollment at 70 participants, a relatively small participant target, above the 45-participant average among 3 other Alagille Syndrome trials with a reported enrollment target (56% higher).
The record links to 2 conditions, with Alagille Syndrome appearing as the primary indexed condition, and to 1 intervention - of which Livmarli is the first listed.
NCT06193928 lists 8 locations in 7 states (Pennsylvania, California, Colorado).
Frequently Asked Questions
What is clinical trial NCT06193928 about?
NCT06193928 is a clinical study titled "Long-Term SafEty and Clinical Outcomes of LivmArli in Patients in the United States (LEAP-US)". The objective of this 5-year, prospective, observational cohort study is to evaluate the long-term safety and clinical outcomes of patients with Alagille syndrome (ALGS) or Progressive familial intrahepatic cholestasis (PFIC) treated with Livmarli.
What is the current status of trial NCT06193928?
This trial is currently recruiting. The enrollment target is 70 participants. The study started on 2023-09-21. Estimated completion is 2030-09-20.
What conditions does trial NCT06193928 study?
This clinical trial studies the following conditions: Alagille Syndrome, Progressive Familial Intrahepatic Cholestasis.
What interventions are being tested in trial NCT06193928?
The interventions under investigation include: Livmarli (DRUG).
Who is sponsoring clinical trial NCT06193928?
This trial is sponsored by Mirum Pharmaceuticals, which has 28 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT06193928 being conducted?
This trial has 8 study locations across California, Colorado, Georgia, Missouri, Oregon. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
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