Evaluate the long-term safety of maralixibat (MRX) in subjects with cholestatic liver disease including, but not limited to, Alagille Syndrome (ALGS), Progressive Familial Intrahepatic Cholestasis (PFIC) and Biliary Atresia.
This is a multicenter, open-label study of maralixibat in subjects diagnosed with cholestatic liver disease (including, but not limited to ALGS, PFIC or Biliary Atresia) who have previously participated in a maralixibat clinical study. All subjects received maralixibat in the previous studies and will continues to receive maralixibat in this study.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
52
Maralixibat chloride oral solution orally twice daily (up to 1200\* mcg/kg/day), and according to indication. \*equivalent to 1140 mcg/kg/day maralixibat
Children'S Hospital Los Angeles
Los Angeles, California, United States
Riley Hospital For Children
Indianapolis, Indiana, United States
Incidence of Treatment-Emergent Adverse Events
TEAE = Treatment-emergent Adverse Event; AESI = Adverse Event of Special Interest..
Time frame: From informed consent through approximately 4.5 years, including 30 days after last dose.
Long-Term Effect on Pruritus
Change from Baseline in Pruritus Severity assessed using the Clinician Scratch Score (CSS), a 5-point scale where 0 indicates no evidence of scratching and 4 indicates cutaneous mutilation with bleeding, hemorrhage, and scarring.
Time frame: From Baseline through Week 160, including Change from Baseline values.
Long-Term Effect on Serum Bile Acid Levels
Time frame: From Baseline through Week 160, including Change from Baseline values.
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Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States
Children Hospital of Pittsburgh
Pittsburgh, Pennsylvania, United States
Baylor College of Medicine/Texas Children's Hospital
Houston, Texas, United States
Seattle Children's Hospital
Seattle, Washington, United States
Children's Hospital Westmead
Westmead, New South Wales, Australia
The Royal Children'S Hospital Melbourne
Parkville, Victoria, Australia
Cliniques Universitaires Saint-Luc
Brussels, Belgium
Hospital for Sick Children
Toronto, Canada
...and 7 more locations