Status:
COMPLETED
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).
Lead Sponsor:
Mirum Pharmaceuticals, Inc.
Conditions:
Progressive Familial Intrahepatic Cholestasis
Alagille Syndrome
Eligibility:
All Genders
Up to 364 years
Phase:
PHASE2
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 Intrahe...
Detailed Description
This is an open label study where all participants will receive maralixibat treatment.
Eligibility Criteria
Inclusion
- Inclusion Criteria:
- Body weight of ≥2.5 kg
- \<12 months of age at the baseline visit (ROW). \>31 days and \<12 months of age at the baseline visit (US).
- 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.
- Diagnosis of PFIC or ALGS
- Exclusion criteria:
- Predicted complete absence of bile salt excretion pump (BSEP) function
- History of surgical disruption of the enterohepatic circulation
- History of liver transplant or imminent need for liver transplant
- Decompensated cirrhosis
- 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
- 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
Exclusion
Key Trial Info
Start Date :
September 9 2021
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
December 17 2024
Estimated Enrollment :
27 Patients enrolled
Trial Details
Trial ID
NCT04729751
Start Date
September 9 2021
End Date
December 17 2024
Last Update
February 6 2025
Active Locations (14)
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1
Children Hospital LA
Los Angeles, California, United States, 90027
2
University of California - San Francisco
San Francisco, California, United States, 94158
3
Medstar Georgetown University Hospital
Washington D.C., District of Columbia, United States, 20007
4
Ochsner Hospital for Children
New Orleans, Louisiana, United States, 70121