Outcome
Type |
Measure |
Description |
Time frame |
Safety issue |
Primary |
Change From MRX Baseline to Week 48 in Fasting sBA Levels |
The primary endpoint of this study was the mean change from MRX baseline to Week 48 in fasting sBA level. |
MRX baseline to Week 48 |
|
Secondary |
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. |
MRX baseline to End of Treatment (maximum exposure was 336 weeks) |
|
Secondary |
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). |
MRX baseline to Week 48 |
|
Secondary |
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. |
MRX baseline to End of Treatment (maximum exposure was 336 weeks) |
|
Secondary |
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. |
MRX baseline to Week 48 |
|
Secondary |
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. |
MRX baseline to End of Treatment (maximum exposure was 336 weeks) |
|
Secondary |
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. |
MRX baseline to Week 48 |
|
Secondary |
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. |
MRX baseline to end of treatment (maximum exposure was 336 weeks) |
|
Secondary |
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. |
MRX baseline to Week 48 |
|
Secondary |
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. |
MRX baseline to End of Treatment (maximum exposure was 336 weeks) |
|
Secondary |
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. |
MRX baseline to Week 48 |
|
Secondary |
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. |
MRX baseline to End of treatment (maximum exposure was 336 weeks) |
|
Secondary |
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. |
MRX baseline to Week 48 |
|
Secondary |
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. |
MRX baseline to End of Treatment (maximum exposure was 336 weeks) |
|
Secondary |
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. |
MRX baseline to Week 48 |
|
Secondary |
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. |
MRX baseline to End of Treatment (maximum exposure was 336 weeks) |
|