The primary objective of this open label extension study is to evaluate the long-term safety and tolerability of maralixibat.
The study will be conducted at multiple sites in North America, Europe, Asia, and South America.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
90
All subjects will receive Maralixibat oral solution (up to 600 microgram per kilogram \[mcg/kg\]) twice daily
Incidence of Treatment Emergent Adverse Events (TEAEs) during the study
Time frame: From baseline through time of interim analysis, up to 120 weeks
Change from maralixibat baseline over the course of the study in the weekly average morning ItchRO(Obs)™ (Itch Reported Outcome) severity score
Time frame: From baseline through study completion, up to approximately 4 years
Maintenance of treatment effect based on the average morning ItchRO(Obs)™ severity scores over the time
Time frame: From baseline through study completion, up to approximately 4 years
Mean change from baseline over time in serum bile acid (sBA) levels
Time frame: From baseline through study completion, up to approximately 4 years
Change from maralixibat baseline over the course of the study in the weekly average morning ItchRO(Obs)™ (Itch Reported Outcome) frequency score
Time frame: From baseline through study completion, up to approximately 4 years
Mean change from baseline over time in height and weight z-scores
Time frame: From baseline through study completion, up to approximately 4 years
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Children's Hospital Los Angeles
Los Angeles, California, United States
Medstar Georgetown University Hospital
Washington D.C., District of Columbia, United States
Advent Health
Orlando, Florida, United States
Children's Hospital at Montefiore
The Bronx, New York, United States
Cincinnati Children's Hospital
Cincinnati, Ohio, United States
Children Hospital of Pittsburgh
Pittsburgh, Pennsylvania, United States
Medical University of South Carolina
Charleston, South Carolina, United States
University of Texas Southwestern Medical Center
Dallas, Texas, United States
University of Texas, Health Science Center San Antonio
San Antonio, Texas, United States
Seattle Children's Hospital
Seattle, Washington, United States
...and 18 more locations