An Extension Study to Evaluate the Long-Term Safety and Durability of Effect of LUM001 in the Treatment of Cholestatic Liver Disease in Subjects With Alagille Syndrome (ALGS)

Change From MRX Baseline Over Time in Fasting sBA Levels

This secondary efficacy endpoint is the mean change from MRX baseline over time in fasting sBA levels. Results reported here are the long-term results.

Time frame: MRX baseline to End of Treatment (maximum exposure was 336 weeks)

Change From MRX Baseline to Week 48 in Pruritus

This secondary efficacy endpoint is the change from MRX baseline to Week 48 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).

Time frame: MRX baseline to Week 48

Change From MRX Baseline Over Time in Pruritus

This secondary efficacy endpoint is the change from MRX baseline over time 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: MRX baseline to End of Treatment (maximum exposure was 336 weeks)

Change From MRX Baseline to Week 48 in Clinician Xanthoma Severity Score

This secondary efficacy endpoint is the mean change from MRX baseline to Week 48 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 participant's lesions interfere or limit his or her activities. Clinician xanthoma severity scores range from 0 to 4, with a xanthoma score of zero representing no evidence of xanthomatosis and a score of 4 representing xanthoma so severe that it is disabling. Clinician xanthoma severity scores were not assessed in Study LUM001-302 so mean clinician xanthoma severity score at MRX baseline was calculated from the 5 participants who were assigned to placebo in Study LUM001-302, and analysis of change from MRX baseline is not presented.

Time frame: MRX baseline to Week 48

Change From MRX Baseline Over Time in Clinician Xanthoma Severity Score

This secondary efficacy endpoint is the mean change from MRX baseline over time (with Week 252 chosen as the end point, as the last analysis visit with at least 6 participants) 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. Clinician xanthoma severity scores were not assessed in Study LUM001-302 so mean clinician xanthoma severity score at MRX baseline was calculated from the 5 participants assigned to placebo in Study LUM001-302, and analysis of change from MRX baseline is not presented. Results reported here are the long-term results.

Time frame: MRX baseline to End of Treatment (maximum exposure was 336 weeks)

Secondary: Change From MRX Baseline to Week 48 in Alkaline Phosphatase

This secondary efficacy endpoint is the mean change from MRX baseline to Week 48 in ALP.

Time frame: MRX baseline to Week 48

Change From MRX Baseline Over Time in Alkaline Phosphatase

This secondary efficacy endpoint is the mean change from MRX baseline over time in ALP. Results reported here are the long-term results.

Time frame: MRX baseline to end of treatment (maximum exposure was 336 weeks)

Change From MRX Baseline to Week 48 in Alanine Aminotransferase

This secondary efficacy endpoint is the mean change from MRX baseline to Week 48 in ALT.

Time frame: MRX baseline to Week 48

Change From MRX Baseline Over Time in Alanine Aminotransferase

This secondary efficacy endpoint is the mean change from MRX baseline over time in ALT levels. Results reported here are the long-term results.

Time frame: MRX baseline to End of Treatment (maximum exposure was 336 weeks)

Change From MRX Baseline to Week 48 in Aspartate Aminotransferase

This secondary efficacy endpoint is the mean change from MRX baseline to Week 48 in AST levels.

Time frame: MRX baseline to Week 48

Change From MRX Baseline Over Time in Aspartate Aminotransferase

This secondary efficacy endpoint is the mean change from MRX baseline over time in AST levels. Results reported here are the long-term results.

Time frame: MRX baseline to End of treatment (maximum exposure was 336 weeks)

Change From MRX Baseline to Week 48 in Gamma Glutamyltransferase

This secondary efficacy endpoint is the mean change from MRX baseline to Week 48 in GGT.

Time frame: MRX baseline to Week 48

Change From MRX Baseline Over Time in Gamma Glutamyltransferase

This secondary efficacy endpoint is the mean change from MRX baseline over time in GGT. Results reported here are the long-term results.

Time frame: MRX baseline to End of Treatment (maximum exposure was 336 weeks)

Change From MRX Baseline to Week 48 in Total and Direct Bilirubin

This secondary efficacy endpoint is the mean change from MRX baseline to Week 48 in total bilirubin and direct bilirubin.

Time frame: MRX baseline to Week 48

Change From MRX Baseline Over Time in Total and Direct Bilirubin

This secondary efficacy endpoint is the mean change from MRX baseline over time in total bilirubin and direct bilirubin. Results reported here are the long-term results.

Time frame: MRX baseline to End of Treatment (maximum exposure was 336 weeks)