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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. Read our disclaimer for details.
ClinicalTrials.gov Identifier: NCT04729751
Recruitment Status : Active, not recruiting
First Posted : January 28, 2021
Last Update Posted : May 19, 2023
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 : July 2023
Estimated Study Completion Date : May 2024

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

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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 ClinicalTrials.gov identifier (NCT number): NCT04729751

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United States, California
Children Hospital LA
Los Angeles, California, United States, 90027
University of California - San Francisco
San Francisco, California, United States, 94158
United States, District of Columbia
Medstar Georgetown University Hospital
Washington, District of Columbia, United States, 20007
United States, Louisiana
Ochsner Hospital for Children
New Orleans, Louisiana, United States, 70121
United States, Pennsylvania
Children's Hospital of Pittsburgh
Pittsburgh, Pennsylvania, United States, 15224
United States, Texas
Texas Children's Hospital
Houston, Texas, United States, 77030
United States, Washington
Seattle Children's Hospital
Seattle, Washington, United States, 98105
Cliniques Universitaires Saint-Luc
Brussels, Belgium
Sociedade Beneficente de Senhoras - Hospital Sírio-Libanês
São Paulo, Brazil, 01308-000
Hôpital Kremlin Bicêtre
Le Kremlin-Bicêtre, France
Hopital Necker
Paris, France
Consultorio de Joshue David Covarrubias Esquer
Zapopan, Mexico, 45050
Instytut Pomnik-Centrum Zdrowia Dziecka
Warsaw, Poland
United Kingdom
King's College Hospital
London, United Kingdom
Sponsors and Collaborators
Mirum Pharmaceuticals, Inc.
Additional Information:
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Responsible Party: Mirum Pharmaceuticals, Inc.
ClinicalTrials.gov Identifier: NCT04729751    
Other Study ID Numbers: MRX-801
2020-004628-40 ( EudraCT Number )
First Posted: January 28, 2021    Key Record Dates
Last Update Posted: May 19, 2023
Last Verified: May 2023

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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