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A Study to Evaluate the Safety and Tolerability of Maralixibat in Infant Participants With Cholestatic Liver Diseases Including Progressive Familial Intrahepatic Cholestasis (PFIC) and Alagille Syndrome (ALGS). (RISE)

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details. Identifier: NCT04729751
Recruitment Status : Recruiting
First Posted : January 28, 2021
Last Update Posted : July 19, 2022
Information provided by (Responsible Party):
Mirum Pharmaceuticals, Inc.

Brief Summary:
This study is designed to assess whether the investigational drug maralixibat, is safe and well tolerated in children <12 months of age with Alagille Syndrome [ALGS] or Progressive Familial Intrahepatic Cholestasis [PFIC].

Condition or disease Intervention/treatment Phase
Progressive Familial Intrahepatic Cholestasis Alagille Syndrome Cholestatic Liver Disease Drug: Maralixibat Phase 2

Detailed Description:
This is an open label study where all participants will receive maralixibat treatment.

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 12 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Intervention Model Description: Single group with 2 cohorts - ≥ 6 participants each from ALGS and PFIC.
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Open-Label, Phase 2 Study to Evaluate the Safety and Tolerability of Maralixibat in the Treatment of Infants With Cholestatic Liver Diseases Including Progressive Familial Intrahepatic Cholestasis and Alagille Syndrome
Actual Study Start Date : September 9, 2021
Estimated Primary Completion Date : March 2023
Estimated Study Completion Date : August 2023

Arm Intervention/treatment
Experimental: Maralixibat
Participants will receive up to 600 μg/kg twice daily (PFIC) or up to 400 μg/kg once daily (ALGS) over 13 weeks in the core study and for the duration of the Long Term Extension (LTE) where applicable.
Drug: Maralixibat

Maralixibat chloride provided in the form of an oral solution (i.e., 5, 10, 15, and 20 mg/mL)

  • 400 μg/kg maralixibat chloride is equivalent to 380 µg/kg maralixibat free base
  • 600 μg/kg maralixibat chloride is equivalent to 570 µg/kg maralixibat free base
Other Name: Formerly LUM001 and SHP625

Primary Outcome Measures :
  1. Frequency of treatment-emergent adverse events [TEAEs] [ Time Frame: From Baseline through to Week 13 ]

Secondary Outcome Measures :
  1. Change in fasting serum bile acid (sBA) levels [ Time Frame: From Baseline through to Week 13 ]
  2. To evaluate the effect on liver enzymes (ALT, AST) and bilirubin [ Time Frame: From Baseline through to Week 13 ]
  3. To evaluate the effect on LSVs [ Time Frame: From Baseline through to Week 13 ]
  4. To assess the plasma level of maralixibat in infant participants [ Time Frame: At Baseline, Week 6, Week 10, Week 13 or Early Termination Visit ]

Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.

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Ages Eligible for Study:   0 Days to 364 Days   (Child)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

Inclusion Criteria:

  1. Body weight of ≥2.5 kg
  2. <12 months of age at the baseline visit (ROW). >31 days and <12 months of age at the baseline visit (US).
  3. Gestational age ≥36 weeks at birth. For children born with gestational age between 32 and 36 weeks, a postmenstrual age of ≥36 weeks is required.
  4. Diagnosis of PFIC or ALGS

Exclusion criteria:

  1. Predicted complete absence of bile salt excretion pump (BSEP) function
  2. History of surgical disruption of the enterohepatic circulation
  3. History of liver transplant or imminent need for liver transplant
  4. Decompensated cirrhosis
  5. Presence of any other disease or condition known to interfere with the absorption, distribution, metabolism, or excretion of drugs, including bile salt metabolism in the intestine (e.g., inflammatory bowel disease), per investigator discretion
  6. Presence of other significant liver disease or any other conditions or abnormalities which, in the opinion of the investigator or medical monitor, may compromise the safety of the participant or interfere with the participant's participation in or completion of the study

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT04729751

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Contact: Clinical Trials Mirum +16506674085
Contact: Mirum Medical Information

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United States, California
Children Hospital LA Recruiting
Los Angeles, California, United States, 90027
Contact: Jillene Wong    323-361-3403   
Principal Investigator: Chuan-Hao Lin, MD         
University of California - San Francisco Recruiting
San Francisco, California, United States, 94158
Contact: Camille Langlois    415-476-1756   
Principal Investigator: Philip Rosenthal, MD         
United States, District of Columbia
Medstar Georgetown University Hospital Recruiting
Washington, District of Columbia, United States, 20007
Contact: Carissa Vinovskis    202-444-3133   
Principal Investigator: Udeme Ekong, MD         
United States, Louisiana
Ochsner Hospital for Children Recruiting
New Orleans, Louisiana, United States, 70121
Contact: Shawan Stewart    504-894-2873   
Principal Investigator: Ryan Himes, MD         
United States, Pennsylvania
Children's Hospital of Pittsburgh Recruiting
Pittsburgh, Pennsylvania, United States, 15224
Contact: Susan Richey    412-692-6337   
Principal Investigator: Simon Horslen, MD         
United States, Texas
Texas Children's Hospital Recruiting
Houston, Texas, United States, 77030
Contact: Murali Gali    832-822-3565   
Principal Investigator: Anna Banc-Husu, MD         
United States, Washington
Seattle Children's Hospital Recruiting
Seattle, Washington, United States, 98105
Contact: Melissa Young    206-987-1037   
Principal Investigator: Evelyn Hsu, MD         
Cliniques Universitaires Saint-Luc Recruiting
Brussels, Belgium
Contact: Julia Versavau    +32 2764 1933   
Principal Investigator: Etienne Sokal, MD         
Sociedade Beneficente de Senhoras - Hospital Sírio-Libanês Not yet recruiting
São Paulo, Brazil, 01308-000
Principal Investigator: Gilda Porta, MD         
Hôpital Kremlin Bicêtre Recruiting
Le Kremlin-Bicêtre, France
Contact: Hubert Lesur    +33 1 45 21 31 67   
Principal Investigator: Emmanuel Gonzales, MD         
Hopital Necker Recruiting
Paris, France
Contact: Mme Tauliaut    +33 1 44 49 44 12   
Principal Investigator: Florence Lacaille, MD         
Consultorio de Joshue David Covarrubias Esquer Not yet recruiting
Zapopan, Mexico, 45050
Principal Investigator: Joshue Covarrubias Esquer, MD         
Instytut Pomnik-Centrum Zdrowia Dziecka Recruiting
Warsaw, Poland
Principal Investigator: Irena Jankowska, MD         
United Kingdom
King's College Hospital Recruiting
London, United Kingdom
Principal Investigator: Richard Thompson, MD         
Sponsors and Collaborators
Mirum Pharmaceuticals, Inc.
Additional Information:
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Responsible Party: Mirum Pharmaceuticals, Inc. Identifier: NCT04729751    
Other Study ID Numbers: MRX-801
2020-004628-40 ( EudraCT Number )
First Posted: January 28, 2021    Key Record Dates
Last Update Posted: July 19, 2022
Last Verified: July 2022

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Mirum Pharmaceuticals, Inc.:
Bile Duct Diseases
Liver Diseases
Biliary Tract Diseases
Additional relevant MeSH terms:
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Liver Diseases
Cholestasis, Intrahepatic
Alagille Syndrome
Pathologic Processes
Digestive System Diseases
Bile Duct Diseases
Biliary Tract Diseases
Heart Defects, Congenital
Cardiovascular Abnormalities
Cardiovascular Diseases
Abnormalities, Multiple
Congenital Abnormalities
Genetic Diseases, Inborn