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 \[PFIC\].
This is an open label study where all participants will receive maralixibat treatment.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
27
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
Children Hospital LA
Los Angeles, California, United States
University of California - San Francisco
San Francisco, California, United States
Medstar Georgetown University Hospital
Washington D.C., District of Columbia, United States
Ochsner Hospital for Children
New Orleans, Louisiana, United States
Children's Hospital of Pittsburgh
Pittsburgh, Pennsylvania, United States
Texas Children's Hospital
Houston, Texas, United States
Seattle Children's Hospital
Seattle, Washington, United States
Cliniques Universitaires Saint-Luc
Brussels, Belgium
Sociedade Beneficente de Senhoras - Hospital Sírio-Libanês
São Paulo, Brazil
Hôpital Kremlin Bicêtre
Le Kremlin-Bicêtre, France
...and 4 more locations
Frequency of treatment-emergent adverse events [TEAEs]
Time frame: From Baseline through to Week 13
Change in fasting serum bile acid (sBA) levels
Time frame: From Baseline through to Week 13
To evaluate the effect on liver enzymes (ALT, AST) and bilirubin
Time frame: From Baseline through to Week 13
To evaluate the effect on LSVs
Time frame: From Baseline through to Week 13
To assess the plasma level of maralixibat in infant participants
Time frame: At Baseline, Week 6, Week 10, Week 13 or Early Termination Visit
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