Long-Term SafEty and Clinical Outcomes of LivmArli in Patients in the United States (LEAP-US)

Part of paid clinical trials in Los Angeles, California.

Sponsor
Mirum Pharmaceuticals, Inc.
Study ID
NCT06193928
Status
Recruiting
Apply to this trial

Conditions

  • Alagille Syndrome
  • Progressive Familial Intrahepatic Cholestasis

Eligibility Criteria

Sex
ALL
Age
N/A - N/A
Healthy Volunteers
Not accepted

Interventions

  • Livmarli — DRUG
    The recommended dosage is 380 mcg/kg once daily.
  • Livmarli — DRUG
    The recommended dosage us 570 mcg/kg twice daily.

Study Details

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.

Key Dates

Start date
Sep 21, 2023
Status verified
May 2025
Primary completion
Sep 20, 2028
Completion
Sep 20, 2030

Study Design

Enrollment
70 participants (estimated)

Arms

  • Arm: Alagille syndrome (ALGS)
    * A clinically and/or genetically confirmed ALGS diagnosis * Participant prescribed Livmarli
  • Arm: Progressive familial intrahepatic cholestasis (PFIC)
    * A clinically and/or genetically confirmed PFIC diagnosis * Prescribed Livmarli

Primary Outcome Measure

Incidence of Long-Term Clinical Outcomes [ Time Frame: Long-term clinical outcomes (SBD, LT, portal hypertension, all-cause mortality) up to 180 days after discontinuation of Livmarli will be recorded. ]

Central Contacts

Locations (8)

FacilityCityStateZIPSite coordinators
Children's Hospital Los Angeles CHLALos AngelesCalifornia90027
Chuan-Hao Lin, MD
[email protected]
George Yanni
Chuan-Hao Lin (PRINCIPAL_INVESTIGATOR)
Section of Gastroenterology, Hepatology and Nutrition, Department of Pediatrics and the Digestive Health Institute, Children's Hospital of Colorado and University of ColoradoAuroraColorado80045
Shikha S. Sundaram
[email protected]
Shikha S. Sundaram (PRINCIPAL_INVESTIGATOR)
Children's Healthcare of Atlanta - Emory University School of MedicineAtlantaGeorgia30322
Angela Stallworth
[email protected]
404-785-3279
Chrissy Lopez (PRINCIPAL_INVESTIGATOR)
Children's Mercy Kansas City, Department of Gastroenterology, Section of HepatologyKansas CityMissouri64108
Ryan T. Fischer
[email protected]
Ryan T. Fischer (PRINCIPAL_INVESTIGATOR)
Oregon Health and Science University, Division of Pediatric Gastroenterology, Department of PediatricsPortlandOregon97239
Henry C. Lin
[email protected]
Henry C. Lin (PRINCIPAL_INVESTIGATOR)
Children's Hospital of PhiladelphiaPhiladelphiaPennsylvania19104
Kathleen Loomes
[email protected]
267-426-7223
Kathleen Loomes, MD (PRINCIPAL_INVESTIGATOR)
Children Hospital of PittsburghPittsburghPennsylvania15260
Simon Horslen, MD
[email protected]
James E. Squires
Simon Horslen (PRINCIPAL_INVESTIGATOR)
University of Utah, Division of Pediatric Gastroenterology, Hepatology and NutritionSalt Lake CityUtah84112
M. Kyle Jensen
[email protected]
M. Kyle Jensen (PRINCIPAL_INVESTIGATOR)

Find similar trials in Los Angeles, CA

By research site
Children's Hospital Los Angeles CHLA· Los Angeles, CASection of Gastroenterology, Hepatology and Nutrition, Department of Pediatrics and the Digestive Health Institute, Children's Hospital of Colorado and University of Colorado· Aurora, COChildren's Healthcare of Atlanta - Emory University School of Medicine· Atlanta, GAChildren's Mercy Kansas City, Department of Gastroenterology, Section of Hepatology· Kansas City, MOOregon Health and Science University, Division of Pediatric Gastroenterology, Department of Pediatrics· Portland, ORChildren's Hospital of Philadelphia· Philadelphia, PA

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