Alagille SyndromeLEAPNCT06193928
Long-Term SafEty and Clinical Outcomes of LivmArli in Patients in the United States (LEAP-US)
An observational study of Alagille Syndrome and Progressive Familial Intrahepatic Cholestasis, run by Mirum Pharmaceuticals, Inc. at 8 sites in United States.
Trial 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. Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- Alagille Syndrome, Progressive Familial Intrahepatic Cholestasis
- What is being tested?
- Livmarli
- How many people
- 70 participants
- Where
- 8 sitesUnited States
- Study dates
- 21 Sep 2023to 20 Sep 2030
- How long will I take part?
- 5 Years
- Phase
- Observational
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Any age
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Study population: All patients with ALGS and PFIC prescribed Livmarli at a participating study center and who consent/assent to participation (if required by the local IRB/IEC) will be included in the study at the discretion of the center investigator.
Who may be able to join
- A clinically and/or genetically confirmed ALGS diagnosis or PFIC diagnosis
- Participant prescribed Livmarli
Who cannot join
- 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
Trial locations and contacts
8 sites in 1 country. Contact the study team or a site near you to ask about taking part.
8 sites in 1 country
United States8 sites, 8 recruiting
- Recruiting
Children's Healthcare of Atlanta - Emory University School of Medicine
Atlanta, Georgia, 30322
Angela Stallworth, 404-785-3279, Angela.Stallworth@choa.org
Chrissy Lopez
- 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, 80045
Shikha S. Sundaram, Shikha.Sundaram@childrenscolorado.org
Shikha S. Sundaram
- Recruiting
Children's Mercy Kansas City, Department of Gastroenterology, Section of Hepatology
Kansas City, Missouri, 64108
Ryan T. Fischer, rtfischer@cmh.edu
Ryan T. Fischer
- Recruiting
Children's Hospital Los Angeles CHLA
Los Angeles, California, 90027
Rohit Kohli, MD, rokohli@chla.usc.edu
George Yanni
- Recruiting
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104
Kathleen Loomes, 267-426-7223, LOOMES@email.chop.edu
Kathleen Loomes, MD
- Recruiting
Children Hospital of Pittsburgh
Pittsburgh, Pennsylvania, 15260
Simon Horslen, MD, horslensp@upmc.edu
James E. Squires
- Recruiting
Oregon Health and Science University, Division of Pediatric Gastroenterology, Department of Pediatrics
Portland, Oregon, 97239
Henry C. Lin, linhe@ohsu.edu
Henry C. Lin
- Recruiting
University of Utah, Division of Pediatric Gastroenterology, Hepatology and Nutrition
Salt Lake City, Utah, 84112
M. Kyle Jensen, kyle.jensen@hsc.utah.edu
M. Kyle Jensen
About this study
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.
Read the detailed description
Livmarli® is a novel, minimally absorbed, pharmacological product that inhibits the ileal bile acid transporter (IBAT) in the terminal ileum, leading to reduced levels of bile acids. Livmarli (maralixibat) has been developed by Mirum Pharmaceuticals and was the first treatment approved by the US Food and Drug Administration (FDA) for the treatment of cholestatic pruritus in patients 3 months of age and older with Alagille syndrome (ALGS). Subsequently, Livmarli was approved by the FDA for the treatment of cholestatic pruritus in patients 12 months of age and older with Progressive familial intrahepatic cholestasis (PFIC). To be eligible for the study, participants must meet the following criteria:
- A clinically and/or genetically confirmed ALGS diagnosis or PFIC diagnosis
- Prescribed Livmarli
- Being tested
- Livmarli
- Keywords
- ALGS, Liver Diseases, PFIC, Livmarli, Alagille Syndrome, Progressive Familial Intrahepatic Cholestasis
- Medical subject headings
- Alagille Syndrome, Cholestasis, progressive familial intrahepatic 1, Liver Diseases
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
LivmarliDrug
Also called Maralixibat
The recommended dosage is 380 mcg/kg once daily.
LivmarliDrug
Also called Maralixibat
The recommended dosage us 570 mcg/kg twice daily.
| Study group | Role | What they receive |
|---|---|---|
| Alagille syndrome (ALGS) | * A clinically and/or genetically confirmed ALGS diagnosis
* Participant prescribed Livmarli Receives: Drug: Livmarli | |
| Progressive familial intrahepatic cholestasis (PFIC) | * A clinically and/or genetically confirmed PFIC diagnosis
* Prescribed Livmarli Receives: Drug: Livmarli |
What is being measured
The primary outcome is the main question the study is designed to answer.
Incidence of Long-Term Clinical Outcomes
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.
Measured: Long-term clinical outcomes (SBD, LT, portal hypertension, all-cause mortality) up to 180 days after discontinuation of Livmarli will be recorded.
Liver Transplant Indication and Waitlist Status
LT waitlist status will be collected, including when placed on or removed from LT waitlist.
Measured: LT waitlist status will be collected at enrollment and every 6 months for 5 years.
Assessment of Growth and Development
Height and weight will be collected both at the time the participant started Livmarli and at the time of enrollment in the study. Subsequent weight will be collected for up to 5 years.
Weight z-score (kilograms) and height z-score (centimeters) will be assessed and reported every year for 5 years.
Measured: Weight (kilograms) and height (centimeters) z-scores will be collected every year for 5 years.
Incidence of Clinical Events Potentially Related to Fat-Soluble Vitamin Deficiencies and Their Long-Term Sequelae
Bleeding events (including all gastrointestinal [GI] or non-GI bleeding requiring hospitalization, emergency department care, or transfusion) and fracture events will be reported.
Measured: The incidence of events will be assessed and reported every year for 5 years.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted5 Jan 2024
- Study start21 Sep 2023
- Primary completion20 Sep 2028 (estimated)
- Study completion20 Sep 2030 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Alagille SyndromeStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 70 participants (estimated)
- Target duration
- 5 Years
Sponsor and investigators
- Lead sponsor
- Mirum Pharmaceuticals, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06193928
- Sponsor's protocol ID
- MRX-310
- Acronym
- LEAP
- Status last verified
- Oct 2026
- First submitted
- 11 Dec 2023
- Last updated
- 8 Oct 2026
- Results posted
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No