Education and Training
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).
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].
Stanford is currently accepting patients for this trial.
Stanford Investigator(s):
Intervention(s):
- drug: Maralixibat
Eligibility
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
Ages Eligible for Study
0 Days - 364 Days
Genders Eligible for Study
All
Now accepting new patients
Contact Information
Stanford University
School of Medicine
300 Pasteur Drive
Stanford,
CA
94305
Recruiting