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
84
All subjects will receive Maralixibat oral solution (up to 600 microgram per kilogram \[mcg/kg\]) twice daily
Mean Change From Baseline Over Time in the Average Morning ItchRO(Obs) Severity Score
The Score on the ItchRo scale is a score on a 5-point scale from 0 (no itch) to 4 (very severe itch)
Time frame: From Baseline to Weeks 75-78
Maintenance of ItchRO(Obs) Response (Weeks 15 - 26)
Maintenance of treatment effect is defined as the proportion of participants in the MRX-MRX treatment group (MRX-502 and MRX-503 data for subjects on MRX in the MRX-502 study) who obtain an ItchRO (Obs) response from Week 15 to Week 26 in Study MRX-503 in the primary cohort and PFIC cohort.
Time frame: Week 15 - 26
Proportion of ItchRO(Obs) Responders Over Time
Proportion of ItchRO responders over time at each study visit using the 4-week study period prior to the visit as per ItchRO(Obs) responder definition.
Time frame: Baseline to EOT
Mean Change From Baseline Over Time in the Average Morning ItchRO(Obs) Frequency Score
The Score on the ItchRo scale is a score on a 5-point scale from 0 (no itch) to 4 (very severe itch)
Time frame: From Baseline to Weeks 75-78
Mean Change From Baseline Over Time in Total Serum Bile Acid (sBA) Levels
Mean change from baseline over time in total serum bile acid (sBA) levels
Time frame: Baseline to week 70
Proportion of Subjects Who Experience an sBA Control Over Time From Week 18 to Week 26
Proportion of subjects who experience an sBA control over time from Week 18 to Week 26
Time frame: From Week 18 to Week 26
This platform is for informational purposes only and does not constitute medical advice. Always consult a qualified healthcare professional.
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
Change From Baseline in Height Z-score
Height-for-age Z-score measures a participant's height relative to a reference population of children of the same age and sex. Z-scores were derived using World Health Organization growth charts for participants less than 24 months of age and Centers for Disease Control and Prevention growth charts for participants 24 months of age or older. A Z-score of 0 represents the population mean for age and sex. Positive Z-scores indicate height above the reference population mean, and negative Z-scores indicate height below the reference population mean. Higher Z-scores generally indicate greater linear growth relative to the reference population. Change from baseline was calculated as post-baseline height-for-age Z-score minus baseline height-for-age Z-score.
Time frame: From baseline to Week 70
Change From Baseline in Weight Z-score
Weight-for-age Z-score measures a participant's weight relative to a reference population of children of the same age and sex. Z-scores were derived using World Health Organization growth charts for participants less than 24 months of age and Centers for Disease Control and Prevention growth charts for participants 24 months of age or older. A Z-score of 0 represents the population mean for age and sex. Positive Z-scores indicate weight above the reference population mean, and negative Z-scores indicate weight below the reference population mean. Higher Z-scores generally indicate greater body weight relative to the reference population. Change from baseline was calculated as post-baseline weight-for-age Z-score minus baseline weight-for-age Z-score.
Time frame: From Baseline to Week 70