Recruiting

Livmarli

Sponsor:

Mirum Pharmaceuticals, Inc.

Code:

NCT06193928

Conditions

Alagille Syndrome

Progressive Familial Intrahepatic Cholestasis

Eligibility Criteria

Sex: All

Age: 0+

Healthy Volunteers: Not accepted

Interventions

Livmarli

Livmarli

Study Details

Brief 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.

Conditions

Alagille Syndrome

Progressive Familial Intrahepatic Cholestasis

Study ID

NCT06193928

Start date

Sep 21, 2023

Status verified date

May, 2025

Completion date

Sep 20, 2030

Anticipated

Primary completion date

Sep 20, 2028

Anticipated

Eligibility Criteria

Eligibility Criteria

Sex: All

Age: 0+

Healthy Volunteers: Not accepted

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

Study Design

Enrollment

70 participants

Anticipated

Interventions and Outcome Measures

Arms

Alagille syndrome (ALGS)

  • A clinically and/or genetically confirmed ALGS diagnosis
  • Participant prescribed Livmarli

Progressive familial intrahepatic cholestasis (PFIC)

  • A clinically and/or genetically confirmed PFIC diagnosis
  • Prescribed Livmarli

Interventions

Livmarli

The recommended dosage is 380 mcg/kg once daily.

Livmarli

The recommended dosage us 570 mcg/kg twice daily.

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. ]
  • Liver Transplant Indication and Waitlist Status [ Time Frame: LT waitlist status will be collected at enrollment and every 6 months for 5 years. ]
  • Assessment of Growth and Development [ Time Frame: 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 [ Time Frame: The incidence of events will be assessed and reported every year for 5 years. ]

Central Contacts and Locations

Central contacts

Clinical Trials Mirum

+16506674085Clinical Trials <clinicaltrials@mirumpharma.com>;

Locations

Children's Hospital Los Angeles CHLA

Recruiting

Los Angeles, California, United States, 90027

Contacts

Chuan-Hao Lin, MD

ChLin@chla.usc.edu

Principal Investigator:

Chuan-Hao Lin

Section of Gastroenterology, Hepatology and Nutrition, Department of Pediatrics and the Digestive Health Institute, Children's Hospital of Colorado and University of Colorado

Recruiting

Aurora, Colorado, United States, 80045

Contacts

Shikha S. Sundaram

Shikha.Sundaram@childrenscolorado.org

Principal Investigator:

Shikha S. Sundaram

Children's Healthcare of Atlanta - Emory University School of Medicine

Recruiting

Atlanta, Georgia, United States, 30322

Contacts

Angela Stallworth

404-785-3279Angela.Stallworth@choa.org

Principal Investigator:

Chrissy Lopez

Children's Mercy Kansas City, Department of Gastroenterology, Section of Hepatology

Recruiting

Kansas City, Missouri, United States, 64108

Contacts

Ryan T. Fischer

rtfischer@cmh.edu

Principal Investigator:

Ryan T. Fischer

Oregon Health and Science University, Division of Pediatric Gastroenterology, Department of Pediatrics

Recruiting

Portland, Oregon, United States, 97239

Contacts

Henry C. Lin

linhe@ohsu.edu

Principal Investigator:

Henry C. Lin

Children's Hospital of Philadelphia

Recruiting

Philadelphia, Pennsylvania, United States, 19104

Contacts

Kathleen Loomes

267-426-7223LOOMES@email.chop.edu

Principal Investigator:

Kathleen Loomes, MD

Children Hospital of Pittsburgh

Recruiting

Pittsburgh, Pennsylvania, United States, 15260

Contacts

Simon Horslen, MD

horslensp@upmc.edu

Principal Investigator:

Simon Horslen

University of Utah, Division of Pediatric Gastroenterology, Hepatology and Nutrition

Recruiting

Salt Lake City, Utah, United States, 84112

Contacts

M. Kyle Jensen

kyle.jensen@hsc.utah.edu

Principal Investigator:

M. Kyle Jensen

More Information

Sponsor

Mirum Pharmaceuticals, Inc.

Last update posted

May 25, 2025

Last verified

May, 2025

Keywords

  • ALGS
  • Liver Diseases
  • PFIC
  • Livmarli
  • Alagille Syndrome
  • Progressive Familial Intrahepatic Cholestasis

Trial information was received from ClinicalTrials.gov and was last updated on 2026-09-08. This information was provided to ClinicalTrials.gov by Mirum Pharmaceuticals, Inc. on 2025-05-25.