This is a multicentre, extension study of LUM001 in children diagnosed with Alagille Syndrome who have completed participation in a core LUM001 treatment protocol. The primary objective is to evaluate long-term safety and tolerability of LUM001. Efficacy will be assessed by evaluating the effect of LUM001 on the biochemical markers and pruritus associated with Alagille Syndrome.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
DOUBLE
Enrollment
34
Dosing of LUM001 also known as Maralixibat (MRX) with the objective of achieving optimal control of pruritus at a dose level that is tolerated by the participant and up to a maximum daily dose of 280 micrograms per kilogram (mcg/kg).
Children's Hospital Los Angeles
Los Angeles, California, United States
University of California at San Francisco Children's Hospital
San Francisco, California, United States
Children's Hospital Colorado
Aurora, Colorado, United States
Change From MRX Baseline to Week 48 in Fasting Serum Bile Acid (sBA)
This primacy efficacy endpoint is the mean change from MRX baseline to week 48 in fasting sBA levels.
Time frame: Baseline to Week 48
Change From MRX Baseline to Week 216 in Fasting Serum Bile Acid (sBA)
The secondary endpoint of this study was the mean change from MRX baseline to week 216 fasting in sBA levels.
Time frame: Baseline to week 216
Change From Baseline to Week 218 in Pruritus
This secondary efficacy endpoint is the mean change from MRX baseline over time to week 218 in pruritus as measured by ItchRO(Obs) weekly average morning severity score. ItchRO scores range from 0 to 4; the higher score indicates increasing itch severity (0 = none; 4 = very severe). Results reported here are the long-term results.
Time frame: Baseline to Week 218
Change From Baseline to Week 216 in Alanine Aminotransferase
This secondary efficacy endpoint is the mean change from MRX baseline to week 216 in ALT levels.
Time frame: Baseline to week 216
Change From Baseline to End of Treatment in Alkaline Phosphatase
This secondary efficacy endpoint is the mean change from MRX baseline to week 216 in ALP levels.
Time frame: Baseline to Week 216
Change From MRX Baseline to Week 216 in Aspartate Aminotransferase
This secondary efficacy endpoint is the mean change from MRX baseline to week 216 in AST levels.
Time frame: Baseline to week 216
Change From MRX Baseline to Week 216 in Clinician Xanthoma Severity Score
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Riley Hospital for Children
Indianapolis, Indiana, United States
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, United States
The Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States
Children's Hospital of Pittsburgh of UPMC
Pittsburgh, Pennsylvania, United States
Baylor College of Medicine/Texas Children's Hospital
Houston, Texas, United States
University of Utah
Salt Lake City, Utah, United States
Seattle Children's Hospital
Seattle, Washington, United States
...and 1 more locations
This secondary efficacy endpoint is the mean change from MRX baseline to week 216 in clinician xanthoma severity scores. It is based on a 0-4 scale to rate the number of lesions present and the degree to which the lesions interfere or limit activities. Clinician xanthoma severity scores range from 0 to 4, with a score of zero representing no evidence of xanthomatosis and a score of 4 representing xanthoma so severe that it is disabling.
Time frame: Baseline to week 216
Change From Baseline to Week 216/LOFC Clinician Scratch Scale (CSS) Score
This secondary efficacy endpoint is the mean change from MRX baseline over time to week 216/LOCF in pruritus as measured by the Clinician Scratch Scale (CSS). The Clinician Scratch Scale uses a 5-point scale, where 0 = none; 1 = rubbing or mild scratching when undistracted; 2 = active scratching without evident skin abrasions; 3 = abrasion evident; 4 = cutaneous mutilation, haemorrhage and scarring evident.
Time frame: Baseline to Week 216
Change From MRX Baseline to Week 216 in Gamma Glutamyltransferase
This secondary efficacy endpoint is the mean change from MRX baseline to week 216 in GGT.
Time frame: Baseline to Week 216
Mean Change From MRX Baseline to Week 216 in Total Bilirubin
This secondary efficacy endpoint is the mean change from MRX baseline to week 216 in total bilirubin.
Time frame: Baseline to week 216
Mean Change From MRX Baseline to Week 216 in Direct Bilirubin
This secondary efficacy endpoint is the mean change from MRX baseline to week 216 in direct bilirubin.
Time frame: Baseline to week 216