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| ID | Type | Description | Link |
|---|---|---|---|
| Parsaclisib | Other Identifier | Incyte Corporation | |
| 2017-003652-22 | EudraCT Number |
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The purpose of this study is to evaluate the safety and efficacy of parsaclisib administered orally to participants with autoimmune hemolytic anemia (AIHA) who have decreased hemoglobin and evidence of ongoing hemolysis that requires treatment intervention.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Parsaclisib 1 mg QD | Experimental | Parsaclisib at 1 milligram (mg) once daily (QD) for 12 weeks followed by extension period, with a dose-increase option (to 2.5 mg QD) at Week 6 for participants who fulfill dose increase criteria. |
|
| Parsaclisib 2.5 mg QD | Experimental | Parsaclisib at 2.5 mg QD for 12 weeks followed by extension period. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Parsaclisib | Drug | Parsaclisib administered orally. |
|
| Measure | Description | Time Frame |
|---|---|---|
| Percentage of Participants Attaining a Complete Response at Any Visit From Week 6 to Week 12 | A complete response was defined as hemoglobin >12 grams per deciliter (g/dL) not attributed to a transfusion effect and the normalization of hemolytic markers. No transfusion effect was defined as > 1 week since the last transfusion. | Week 6 to Week 12 |
| Percentage of Participants Attaining a Partial Response at Any Visit From Week 6 to Week 12 | A partial response was defined as hemoglobin 10-12 g/dL or at least a 2 g/dL increase from Baseline not attributed to a transfusion effect and the normalization of hemolytic markers. No transfusion effect was defined as > 1 week since the last transfusion. | Week 6 to Week 12 |
| Number of Participants With Any Treatment-emergent Adverse Event (TEAE) | An adverse event (AE) was defined as any untoward medical occurrence associated with the use of a drug in humans, whether or not considered drug related, that occurred after a participant provided informed consent. Abnormal laboratory values or test results occurring after informed consent constituted AEs only if they induced clinical signs or symptoms, were considered clinically meaningful, required therapy, or required changes in the study drug. Anemia and transfusions should not have been reported as AEs unless they represented a clinically meaningful decrease from Baseline in hemoglobin. A TEAE was defined as any AE either reported for the first time or the worsening of a pre-existing event after the first dose of study drug. | up to 1638 days |
| Measure | Description | Time Frame |
|---|---|---|
| Percentage of Participants Attaining a Complete Response During Post-Baseline Visits | A complete response was defined as hemoglobin >12 g/dL) not attributed to a transfusion effect and the normalization of hemolytic markers. No transfusion effect was defined as > 1 week since the last transfusion. | up to 1638 days |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Kathleen Butler, MD | Incyte Corporation | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Georgetown University Hospital | Washington D.C. | District of Columbia | 20007 | United States | ||
| University of Minnesota |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 39435908 | Derived | Barcellini W, Pane F, Patriarca A, Murakhovskaya I, Terriou L, DeSancho MT, Hanna WT, Leopold L, Rappold E, Szeto K, Wei S, Jager U. Parsaclisib for the treatment of primary autoimmune hemolytic anemia: Results from a phase 2, open-label study. Am J Hematol. 2024 Dec;99(12):2313-2320. doi: 10.1002/ajh.27493. Epub 2024 Oct 22. |
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The study was conducted in 25 participants enrolled in 8 sites in Austria, France, Italy, and the United States.
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| ID | Title | Description |
|---|---|---|
| FG000 | Parsaclisib 1 mg QD | Participants received oral parsaclisib once daily (QD) for 12 weeks. Participants initially received parsaclisib 1 milligram (mg) QD. At Week 6, participants who continued to require transfusions or did not attain a meaningful clinical response (at least a stabilization of a ≥ 2 grams per deciliter [g/dL] increase in hemoglobin from Baseline to Week 6) may have had their dose increased, with sponsor preapproval, to parsaclisib 2.5 mg QD until Week 12. If there were any tolerability issues in individual participants who received 2.5 mg, the dose could have been decreased to 1 mg. Participants who continued to receive clinical benefit after the 12-week Treatment Period could have entered into an Extension Period, during which they, with sponsor approval, continued to receive parsaclisib 1 mg QD or 2.5 mg QD until the drug was available through another clinical study, or was commercially available, or the sponsor stopped the study or the study drug development. Following the last dose of parsaclisib, in either the Treatment Period (for participants not entering the Extension Period) or the Extension Period, participants were eligible for a 12-week (3-month) follow-up period. |
| FG001 | Parsaclisib 2.5 mg QD | Participants received oral parsaclisib 2.5 mg QD for 12 weeks. If there were any tolerability issues in individual participants, the dose could have been decreased to 1 mg. Participants who continued to receive clinical benefit after the 12-week Treatment Period could have entered into an Extension Period, during which they, with sponsor approval, continued to receive parsaclisib 2.5 mg QD until the drug was available through another clinical study, or was commercially available, or the sponsor stopped the study or the study drug development. Following the last dose of parsaclisib, in either the Treatment Period (for participants not entering the Extension Period) or the Extension Period, participants were eligible for a 12-week (3-month) follow-up period. |
| Title | Milestones | Reasons Not Completed | |||||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| 12-Week Treatment Period |
|
| |||||||||||||||||||||
| Treatment Extension Period |
|
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| ID | Title | Description |
|---|---|---|
| BG000 | Parsaclisib 1 mg QD | Participants received oral parsaclisib once daily (QD) for 12 weeks. Participants initially received parsaclisib 1 milligram (mg) QD. At Week 6, participants who continued to require transfusions or did not attain a meaningful clinical response (at least a stabilization of a ≥ 2 grams per deciliter [g/dL] increase in hemoglobin from Baseline to Week 6) may have had their dose increased, with sponsor preapproval, to parsaclisib 2.5 mg QD until Week 12. If there were any tolerability issues in individual participants who received 2.5 mg, the dose could have been decreased to 1 mg. Participants who continued to receive clinical benefit after the 12-week Treatment Period could have entered into an Extension Period, during which they, with sponsor approval, continued to receive parsaclisib 1 mg QD or 2.5 mg QD until the drug was available through another clinical study, or was commercially available, or the sponsor stopped the study or the study drug development. Following the last dose of parsaclisib, in either the Treatment Period (for participants not entering the Extension Period) or the Extension Period, participants were eligible for a 12-week (3-month) follow-up period. |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes |
|---|---|---|---|---|---|---|---|---|---|
| Age, Continuous | Mean |
| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | |||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | Percentage of Participants Attaining a Complete Response at Any Visit From Week 6 to Week 12 | A complete response was defined as hemoglobin >12 grams per deciliter (g/dL) not attributed to a transfusion effect and the normalization of hemolytic markers. No transfusion effect was defined as > 1 week since the last transfusion. | Full Analysis Set: all participants who enrolled in the study who received at least 1 dose of study drug | Posted | Number | percentage of participants | Week 6 to Week 12 |
|
up to a maximum of 1638 days
TEAEs (adverse events either reported for the first time or the worsening of pre-existing events after the first dose of study drug) have been reported. As dose increases and decreases, based on clinical benefit and tolerability issues, respectively, were allowed during the course of the study for participants randomized to receive both 1 mg and 2.5 mg, AEs are reported per the treatment participants were randomized to rather than the dose level that was administered at the time the AE occurred.
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| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | Parsaclisib 1 mg QD | Participants received oral parsaclisib once daily (QD) for 12 weeks. Participants initially received parsaclisib 1 milligram (mg) QD. At Week 6, participants who continued to require transfusions or did not attain a meaningful clinical response (at least a stabilization of a ≥ 2 grams per deciliter [g/dL] increase in hemoglobin from Baseline to Week 6) may have had their dose increased, with sponsor preapproval, to parsaclisib 2.5 mg QD until Week 12. If there were any tolerability issues in individual participants who received 2.5 mg, the dose could have been decreased to 1 mg. Participants who continued to receive clinical benefit after the 12-week Treatment Period could have entered into an Extension Period, during which they, with sponsor approval, continued to receive parsaclisib 1 mg QD or 2.5 mg QD until the drug was available through another clinical study, or was commercially available, or the sponsor stopped the study or the study drug development. Following the last dose of parsaclisib, in either the Treatment Period (for participants not entering the Extension Period) or the Extension Period, participants were eligible for a 12-week (3-month) follow-up period. |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Acute respiratory failure | Respiratory, thoracic and mediastinal disorders | MedDRA 23 | Systematic Assessment |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Abdominal distension | Gastrointestinal disorders | MedDRA 23 | Systematic Assessment |
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| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Study Director | Incyte Corporation | 1-855-463-3463 | medinfo@incyte.com |
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| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| Prot | Yes | No | No | Study Protocol | Nov 2, 2020 | Aug 1, 2022 | Prot_000.pdf |
| SAP | No | Yes | No | Statistical Analysis Plan | Mar 3, 2022 | Aug 1, 2022 | SAP_001.pdf |
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| ID | Term |
|---|---|
| D000744 | Anemia, Hemolytic, Autoimmune |
| D009477 | Hereditary Sensory and Autonomic Neuropathies |
| ID | Term |
|---|---|
| D000743 | Anemia, Hemolytic |
| D000740 | Anemia |
| D006402 | Hematologic Diseases |
| D006425 | Hemic and Lymphatic Diseases |
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| ID | Term |
|---|---|
| C000656179 | parsaclisib |
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| Percentage of Participants Attaining a Partial Response During Post-Baseline Visits |
A partial response was defined as hemoglobin 10-12 g/dL or at least a 2 g/dL increase from Baseline not attributed to a transfusion effect and the normalization of hemolytic markers. No transfusion effect was defined as > 1 week since the last transfusion. |
| up to 1638 days |
| Percentage of Participants Attaining a ≥ 2 g/dL Increase in Hemoglobin From Baseline | Hemoglobin levels were assessed throughout the study. | up to 1638 days |
| Change From Baseline in Hemoglobin | Change from Baseline was calculated as the post-Baseline value minus the Baseline value. | Baseline; up to 1638 days |
| Percentage Change From Baseline in Hemoglobin | Percentage change from Baseline was calculated as: ([post-Baseline value minus the Baseline value] / Baseline value) x 100. | Baseline; up to 1638 days |
| Percentage of Participants Requiring Transfusions | A participant was defined to have required a transfusion if his or her last transfusion was within 7 days of the visit date. | Baseline; up to 1638 days |
| Percentage of Participants Who Achieved Normalization of Hemoglobin, Haptoglobin, Lactate Dehydrogenase (LDH), Reticulocyte Count, Total Bilirubin, Direct Bilirubin, and Indirect Bilirubin | Normalization was determined by the Investigator based on normal ranges for the clinical reference laboratory. | up to 1638 days |
| Percentage of Participants Requiring a Prednisone Dose Change (Increase or Decrease) | Prednisone use was monitored throughout the study. | up to 1638 days |
| Change From Baseline in the Functional Assessment of Chronic Illness Therapy - Fatigue (FACIT-F) Subscale Scores | The FACIT-F subscale is a 13-item instrument designed to assess fatigue/tiredness and its impact on daily activities and functioning in a number of chronic diseases. Participants were asked to respond to 13 statements that people with the illness have said are important on the following scale: 0, not at all; 1, a little bit; 2, somewhat; 3, quite a bit; 4, very much. Participants were asked to indicate the response as it applied to the last 7 days. The total fatigue subscale score ranges from 0 to 52; a higher score indicates more severe impact on daily activities and functioning. | Baseline; up to 1638 days |
| Mean Cmax of Parsaclisib | Cmax was defined as the maximum observed concentration. Participants in Cohort 1 were not eligible to receive parsaclisib 2.5 mg at Week 2. | predose at Weeks 1 and 12; predose and 1, 2, and 4 hours postdose at Weeks 2 and 8 |
| Mean Tmax of Parsaclisib | tmax was defined as the time to the maximum concentration. Participants in Cohort 1 were not eligible to receive parsaclisib 2.5 mg at Week 2. | predose at Weeks 1 and 12; predose and 1, 2, and 4 hours postdose at Weeks 2 and 8 |
| Mean Cmin of Parsaclisib | Cmin was defined as the minimum observed concentration over the dose interval. Participants in Cohort 1 were not eligible to receive parsaclisib 2.5 mg at Week 2. | predose at Weeks 1 and 12; predose and 1, 2, and 4 hours postdose at Weeks 2 and 8 |
| Mean AUC0-4 of Parsaclisib | AUC0-4 was defined as the area under the concentration-time curve from time = 0 to 4 hours postdose. Participants in Cohort 1 were not eligible to receive parsaclisib 2.5 mg at Week 2. | predose at Weeks 1 and 12; predose and 1, 2, and 4 hours postdose at Weeks 2 and 8 |
| Mean AUC0-t of Parsaclisib | AUC0-t was defined as the area under the concentration-time curve from time = 0 to the last measureable concentration at time = t. Participants in Cohort 1 were not eligible to receive parsaclisib 2.5 mg at Week 2. | predose at Weeks 1 and 12; predose and 1, 2, and 4 hours postdose at Weeks 2 and 8 |
| Mean Clast of Parsaclisib | Clast was defined as the last measurable concentration (above the quantification limit). Participants in Cohort 1 were not eligible to receive parsaclisib 2.5 mg at Week 2. | predose at Weeks 1 and 12; predose and 1, 2, and 4 hours postdose at Weeks 2 and 8 |
| Mean Tlast of Parsaclisib | Tlast was defined as the time of the last measurable concentration (above the quantification limit). Participants in Cohort 1 were not eligible to receive parsaclisib 2.5 mg at Week 2. | predose at Weeks 1 and 12; predose and 1, 2, and 4 hours postdose at Weeks 2 and 8 |
| Change From Baseline in Reticulocyte Count | Change from Baseline was calculated as the post-Baseline value minus the Baseline value | Baseline; up to 1638 days |
| Change From Baseline in Cardiolipin Immunoglobulin G (IgG) Antibody and Cardiolipin Immunoglobulin M (IgM) Antibody | Change from Baseline was calculated as the post-Baseline value minus the Baseline value. | Baseline; Week 12 |
| Change From Baseline in Cold Hemagglutinin Levels | Change from Baseline was calculated as the post-Baseline value minus the Baseline value. | Baseline; Week 12 |
| Change From Baseline in Haptoglobin, Total Bilirubin, Direct Bilirubin, and Indirect Bilirubin | Change from Baseline was calculated as the post-Baseline value minus the Baseline value. | Baseline; up to 1638 days |
| Change From Baseline in Lactate Dehydrogenase (LDH) | Change from Baseline was calculated as the post-Baseline value minus the Baseline value. | Baseline; up to 1638 days |
| Change From Baseline in CH50 | Change from Baseline was calculated as the post-Baseline value minus the Baseline value. | Baseline; up to 1638 days |
| Change From Baseline in Complement C3 and Complement C4 | Change from Baseline was calculated as the post-Baseline value minus the Baseline value. | Baseline; up to 1638 days |
| Minneapolis |
| Minnesota |
| 55455 |
| United States |
| Washington University School of Medicine | St Louis | Missouri | 63110 | United States |
| Weill Medical College of Cornell University | New York | New York | 10021 | United States |
| Montefiore Medical Center | The Bronx | New York | 10467 | United States |
| University Health System Inc., Dba the University of Tn Medical Center | Knoxville | Tennessee | 37920 | United States |
| Allgemeines Krankenhaus Der Stadt Wien | Vienna | 01090 | Austria |
| Centre Hospitalier Universitaire Henri Mondor | Créteil | 94010 | France |
| Centre Hospitalier Regional Universitaire (Chru) de Lille | Lille | 59037 | France |
| Fondazione Irccs Ca Granda Ospedale Maggiore | Milan | 20122 | Italy |
| UNIVERSIT� DI NAPOLI FEDERICO II | Naples | 80131 | Italy |
| AZIENDA OSPEDALIERO UNIVERSITARIA MAGGIORE DELLA CARIT� DI NOVARA | Novara | 28100 | Italy |
| NOT COMPLETED |
|
|
| BG001 | Parsaclisib 2.5 mg QD | Participants received oral parsaclisib 2.5 mg QD for 12 weeks. If there were any tolerability issues in individual participants, the dose could have been decreased to 1 mg. Participants who continued to receive clinical benefit after the 12-week Treatment Period could have entered into an Extension Period, during which they, with sponsor approval, continued to receive parsaclisib 2.5 mg QD until the drug was available through another clinical study, or was commercially available, or the sponsor stopped the study or the study drug development. Following the last dose of parsaclisib, in either the Treatment Period (for participants not entering the Extension Period) or the Extension Period, participants were eligible for a 12-week (3-month) follow-up period. |
| BG002 | Total | Total of all reporting groups |
| years |
|
| Sex: Female, Male | Count of Participants | Participants |
|
| Ethnicity (NIH/OMB) | Count of Participants | Participants |
|
| Race/Ethnicity, Customized | Count of Participants | Participants |
|
| Hemoglobin | Mean | Standard Deviation | grams per deciliter (g/dL) |
|
| OG001 | Parsaclisib 2.5 mg QD | Participants received oral parsaclisib 2.5 mg QD for 12 weeks. If there were any tolerability issues in individual participants, the dose could have been decreased to 1 mg. Participants who continued to receive clinical benefit after the 12-week Treatment Period could have entered into an Extension Period, during which they, with sponsor approval, continued to receive parsaclisib 2.5 mg QD until the drug was available through another clinical study, or was commercially available, or the sponsor stopped the study or the study drug development. Following the last dose of parsaclisib, in either the Treatment Period (for participants not entering the Extension Period) or the Extension Period, participants were eligible for a 12-week (3-month) follow-up period. |
|
|
| Primary | Percentage of Participants Attaining a Partial Response at Any Visit From Week 6 to Week 12 | A partial response was defined as hemoglobin 10-12 g/dL or at least a 2 g/dL increase from Baseline not attributed to a transfusion effect and the normalization of hemolytic markers. No transfusion effect was defined as > 1 week since the last transfusion. | Full Analysis Set | Posted | Number | percentage of participants | Week 6 to Week 12 |
|
|
|
| Primary | Number of Participants With Any Treatment-emergent Adverse Event (TEAE) | An adverse event (AE) was defined as any untoward medical occurrence associated with the use of a drug in humans, whether or not considered drug related, that occurred after a participant provided informed consent. Abnormal laboratory values or test results occurring after informed consent constituted AEs only if they induced clinical signs or symptoms, were considered clinically meaningful, required therapy, or required changes in the study drug. Anemia and transfusions should not have been reported as AEs unless they represented a clinically meaningful decrease from Baseline in hemoglobin. A TEAE was defined as any AE either reported for the first time or the worsening of a pre-existing event after the first dose of study drug. | Full Analysis Set | Posted | Count of Participants | Participants | up to 1638 days |
|
|
|
| Secondary | Percentage of Participants Attaining a Complete Response During Post-Baseline Visits | A complete response was defined as hemoglobin >12 g/dL) not attributed to a transfusion effect and the normalization of hemolytic markers. No transfusion effect was defined as > 1 week since the last transfusion. | Full Analysis Set. Only participants with available data were analyzed. | Posted | Number | percentage of participants | up to 1638 days |
|
|
|
| Secondary | Percentage of Participants Attaining a Partial Response During Post-Baseline Visits | A partial response was defined as hemoglobin 10-12 g/dL or at least a 2 g/dL increase from Baseline not attributed to a transfusion effect and the normalization of hemolytic markers. No transfusion effect was defined as > 1 week since the last transfusion. | Full Analysis Set. Only participants with available data were analyzed. | Posted | Number | percentage of participants | up to 1638 days |
|
|
|
| Secondary | Percentage of Participants Attaining a ≥ 2 g/dL Increase in Hemoglobin From Baseline | Hemoglobin levels were assessed throughout the study. | Full Analysis Set. Only participants with available data were analyzed. | Posted | Number | percentage of participants | up to 1638 days |
|
|
|
| Secondary | Change From Baseline in Hemoglobin | Change from Baseline was calculated as the post-Baseline value minus the Baseline value. | Full Analysis Set. Only participants with available data were analyzed. | Posted | Mean | Standard Deviation | grams per deciliter (g/dL) | Baseline; up to 1638 days |
|
|
|
| Secondary | Percentage Change From Baseline in Hemoglobin | Percentage change from Baseline was calculated as: ([post-Baseline value minus the Baseline value] / Baseline value) x 100. | Full Analysis Set. Only participants with available data were analyzed. | Posted | Mean | Standard Deviation | percent change | Baseline; up to 1638 days |
|
|
|
| Secondary | Percentage of Participants Requiring Transfusions | A participant was defined to have required a transfusion if his or her last transfusion was within 7 days of the visit date. | Full Analysis Set. Only participants with available data were analyzed. | Posted | Number | percentage of participants | Baseline; up to 1638 days |
|
|
|
| Secondary | Percentage of Participants Who Achieved Normalization of Hemoglobin, Haptoglobin, Lactate Dehydrogenase (LDH), Reticulocyte Count, Total Bilirubin, Direct Bilirubin, and Indirect Bilirubin | Normalization was determined by the Investigator based on normal ranges for the clinical reference laboratory. | Full Analysis Set. Only participants with available data were analyzed. | Posted | Number | percentage of participants | up to 1638 days |
|
|
|
| Secondary | Percentage of Participants Requiring a Prednisone Dose Change (Increase or Decrease) | Prednisone use was monitored throughout the study. | Full Analysis Set | Posted | Number | percentage of participants | up to 1638 days |
|
|
|
| Secondary | Change From Baseline in the Functional Assessment of Chronic Illness Therapy - Fatigue (FACIT-F) Subscale Scores | The FACIT-F subscale is a 13-item instrument designed to assess fatigue/tiredness and its impact on daily activities and functioning in a number of chronic diseases. Participants were asked to respond to 13 statements that people with the illness have said are important on the following scale: 0, not at all; 1, a little bit; 2, somewhat; 3, quite a bit; 4, very much. Participants were asked to indicate the response as it applied to the last 7 days. The total fatigue subscale score ranges from 0 to 52; a higher score indicates more severe impact on daily activities and functioning. | Full Analysis Set. Only participants with available data were analyzed. | Posted | Mean | Standard Deviation | scores on a scale | Baseline; up to 1638 days |
|
|
|
| Secondary | Mean Cmax of Parsaclisib | Cmax was defined as the maximum observed concentration. Participants in Cohort 1 were not eligible to receive parsaclisib 2.5 mg at Week 2. | Pharmacokinetic (PK)/Pharmacodynamic (PD) Evaluable Population: all participants who received at least 1 dose of study drug and provided at least 1 postdose blood sample for PK or biomarker assessment. Only participants with available data were analyzed. | Posted | Geometric Mean | Geometric Coefficient of Variation | nanomoles per Liter (nmol/L) | predose at Weeks 1 and 12; predose and 1, 2, and 4 hours postdose at Weeks 2 and 8 |
|
|
|
|
| Secondary | Mean Tmax of Parsaclisib | tmax was defined as the time to the maximum concentration. Participants in Cohort 1 were not eligible to receive parsaclisib 2.5 mg at Week 2. | PK/PD Evaluable Population. Only participants with available data were analyzed. | Posted | Median | Full Range | hours | predose at Weeks 1 and 12; predose and 1, 2, and 4 hours postdose at Weeks 2 and 8 |
|
|
|
|
| Secondary | Mean Cmin of Parsaclisib | Cmin was defined as the minimum observed concentration over the dose interval. Participants in Cohort 1 were not eligible to receive parsaclisib 2.5 mg at Week 2. | PK/PD Evaluable Population. Only participants with available data were analyzed. | Posted | Geometric Mean | Geometric Coefficient of Variation | nmol/L | predose at Weeks 1 and 12; predose and 1, 2, and 4 hours postdose at Weeks 2 and 8 |
|
|
|
|
| Secondary | Mean AUC0-4 of Parsaclisib | AUC0-4 was defined as the area under the concentration-time curve from time = 0 to 4 hours postdose. Participants in Cohort 1 were not eligible to receive parsaclisib 2.5 mg at Week 2. | PK/PD Evaluable Population. Only participants with available data were analyzed. | Posted | Geometric Mean | Geometric Coefficient of Variation | hours*nmol/L | predose at Weeks 1 and 12; predose and 1, 2, and 4 hours postdose at Weeks 2 and 8 |
|
|
|
|
| Secondary | Mean AUC0-t of Parsaclisib | AUC0-t was defined as the area under the concentration-time curve from time = 0 to the last measureable concentration at time = t. Participants in Cohort 1 were not eligible to receive parsaclisib 2.5 mg at Week 2. | PK/PD Evaluable Population. Only participants with available data were analyzed. | Posted | Geometric Mean | Geometric Coefficient of Variation | hours*nmol/L | predose at Weeks 1 and 12; predose and 1, 2, and 4 hours postdose at Weeks 2 and 8 |
|
|
|
| Secondary | Mean Clast of Parsaclisib | Clast was defined as the last measurable concentration (above the quantification limit). Participants in Cohort 1 were not eligible to receive parsaclisib 2.5 mg at Week 2. | PK/PD Evaluable Population. Only participants with available data were analyzed. | Posted | Geometric Mean | Geometric Coefficient of Variation | nmol/L | predose at Weeks 1 and 12; predose and 1, 2, and 4 hours postdose at Weeks 2 and 8 |
|
|
|
| Secondary | Mean Tlast of Parsaclisib | Tlast was defined as the time of the last measurable concentration (above the quantification limit). Participants in Cohort 1 were not eligible to receive parsaclisib 2.5 mg at Week 2. | PK/PD Evaluable Population. Only participants with available data were analyzed. | Posted | Geometric Mean | Geometric Coefficient of Variation | hours | predose at Weeks 1 and 12; predose and 1, 2, and 4 hours postdose at Weeks 2 and 8 |
|
|
|
| Secondary | Change From Baseline in Reticulocyte Count | Change from Baseline was calculated as the post-Baseline value minus the Baseline value | Full Analysis Set. Only participants with available data were analyzed. | Posted | Mean | Standard Deviation | 10^9 cells per Liter (10^9 cells/L) | Baseline; up to 1638 days |
|
|
|
| Secondary | Change From Baseline in Cardiolipin Immunoglobulin G (IgG) Antibody and Cardiolipin Immunoglobulin M (IgM) Antibody | Change from Baseline was calculated as the post-Baseline value minus the Baseline value. | Full Analysis Set. Only participants with available data were analyzed. | Posted | Mean | Standard Deviation | micrograms per milliliter (µg/mL) | Baseline; Week 12 |
|
|
|
| Secondary | Change From Baseline in Cold Hemagglutinin Levels | Change from Baseline was calculated as the post-Baseline value minus the Baseline value. | Full Analysis Set. Only participants with available data were analyzed. | Posted | Mean | Standard Deviation | titer | Baseline; Week 12 |
|
|
|
| Secondary | Change From Baseline in Haptoglobin, Total Bilirubin, Direct Bilirubin, and Indirect Bilirubin | Change from Baseline was calculated as the post-Baseline value minus the Baseline value. | Full Analysis Set. Only participants with available data were analyzed. | Posted | Mean | Standard Deviation | micromoles (µmol)/L | Baseline; up to 1638 days |
|
|
|
| Secondary | Change From Baseline in Lactate Dehydrogenase (LDH) | Change from Baseline was calculated as the post-Baseline value minus the Baseline value. | Full Analysis Set. Only participants with available data were analyzed. | Posted | Mean | Standard Deviation | Units (U)/L | Baseline; up to 1638 days |
|
|
|
| Secondary | Change From Baseline in CH50 | Change from Baseline was calculated as the post-Baseline value minus the Baseline value. | Full Analysis Set. Only participants with available data were analyzed. | Posted | Mean | Standard Deviation | microgram equivalents per Liter (µgEq/L) | Baseline; up to 1638 days |
|
|
|
| Secondary | Change From Baseline in Complement C3 and Complement C4 | Change from Baseline was calculated as the post-Baseline value minus the Baseline value. | Full Analysis Set. Only participants with available data were analyzed. | Posted | Mean | Standard Deviation | grams per Liter (g/L) | Baseline; up to 1638 days |
|
|
|
| 0 |
| 10 |
| 6 |
| 10 |
| 10 |
| 10 |
| EG001 | Parsaclisib 2.5 mg QD | Participants received oral parsaclisib 2.5 mg QD for 12 weeks. If there were any tolerability issues in individual participants, the dose could have been decreased to 1 mg. Participants who continued to receive clinical benefit after the 12-week Treatment Period could have entered into an Extension Period, during which they, with sponsor approval, continued to receive parsaclisib 2.5 mg QD until the drug was available through another clinical study, or was commercially available, or the sponsor stopped the study or the study drug development. Following the last dose of parsaclisib, in either the Treatment Period (for participants not entering the Extension Period) or the Extension Period, participants were eligible for a 12-week (3-month) follow-up period. | 1 | 15 | 5 | 15 | 15 | 15 |
| Amoebic dysentery | Infections and infestations | MedDRA 23 | Systematic Assessment |
|
| Aortic thrombosis | Vascular disorders | MedDRA 23 | Systematic Assessment |
|
| Atypical mycobacterial infection | Infections and infestations | MedDRA 23 | Systematic Assessment |
|
| Autoimmune haemolytic anaemia | Blood and lymphatic system disorders | MedDRA 23 | Systematic Assessment |
|
| Bacterial sepsis | Infections and infestations | MedDRA 23 | Systematic Assessment |
|
| COVID-19 | Infections and infestations | MedDRA 23 | Systematic Assessment |
|
| COVID-19 pneumonia | Infections and infestations | MedDRA 23 | Systematic Assessment |
|
| Campylobacter colitis | Infections and infestations | MedDRA 23 | Systematic Assessment |
|
| Cytomegalovirus infection reactivation | Infections and infestations | MedDRA 23 | Systematic Assessment |
|
| Diabetes insipidus | Endocrine disorders | MedDRA 23 | Systematic Assessment |
|
| Diarrhoea | Gastrointestinal disorders | MedDRA 23 | Systematic Assessment |
|
| Enterococcal infection | Infections and infestations | MedDRA 23 | Systematic Assessment |
|
| Fall | Injury, poisoning and procedural complications | MedDRA 23 | Systematic Assessment |
|
| Femur fracture | Injury, poisoning and procedural complications | MedDRA 23 | Systematic Assessment |
|
| Haemolytic anaemia | Blood and lymphatic system disorders | MedDRA 23 | Systematic Assessment |
|
| Hypernatraemia | Metabolism and nutrition disorders | MedDRA 23 | Systematic Assessment |
|
| Interstitial lung disease | Respiratory, thoracic and mediastinal disorders | MedDRA 23 | Systematic Assessment |
|
| Invasive ductal breast carcinoma | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA 23 | Systematic Assessment |
|
| Pneumonia | Infections and infestations | MedDRA 23 | Systematic Assessment |
|
| Pseudomonas infection | Infections and infestations | MedDRA 23 | Systematic Assessment |
|
| Psoriasis | Skin and subcutaneous tissue disorders | MedDRA 23 | Systematic Assessment |
|
| Pulmonary fibrosis | Respiratory, thoracic and mediastinal disorders | MedDRA 23 | Systematic Assessment |
|
| Pulmonary haemorrhage | Respiratory, thoracic and mediastinal disorders | MedDRA 23 | Systematic Assessment |
|
| Respiratory failure | Respiratory, thoracic and mediastinal disorders | MedDRA 23 | Systematic Assessment |
|
| Sepsis | Infections and infestations | MedDRA 23 | Systematic Assessment |
|
| Staphylococcal sepsis | Infections and infestations | MedDRA 23 | Systematic Assessment |
|
| Tibia fracture | Injury, poisoning and procedural complications | MedDRA 23 | Systematic Assessment |
|
| Abdominal pain | Gastrointestinal disorders | MedDRA 23 | Systematic Assessment |
|
| Agitation | Psychiatric disorders | MedDRA 23 | Systematic Assessment |
|
| Anaemia | Blood and lymphatic system disorders | MedDRA 23 | Systematic Assessment |
|
| Angina pectoris | Cardiac disorders | MedDRA 23 | Systematic Assessment |
|
| Arrhythmia | Cardiac disorders | MedDRA 23 | Systematic Assessment |
|
| Arthralgia | Musculoskeletal and connective tissue disorders | MedDRA 23 | Systematic Assessment |
|
| Asthenia | General disorders | MedDRA 23 | Systematic Assessment |
|
| Autoimmune haemolytic anaemia | Blood and lymphatic system disorders | MedDRA 23 | Systematic Assessment |
|
| Bacteraemia | Infections and infestations | MedDRA 23 | Systematic Assessment |
|
| Blister | Skin and subcutaneous tissue disorders | MedDRA 23 | Systematic Assessment |
|
| Blood bilirubin increased | Investigations | MedDRA 23 | Systematic Assessment |
|
| Blood creatinine increased | Investigations | MedDRA 23 | Systematic Assessment |
|
| Blood potassium decreased | Investigations | MedDRA 23 | Systematic Assessment |
|
| Bone pain | Musculoskeletal and connective tissue disorders | MedDRA 23 | Systematic Assessment |
|
| Breath sounds abnormal | Investigations | MedDRA 23 | Systematic Assessment |
|
| COVID-19 | Infections and infestations | MedDRA 23 | Systematic Assessment |
|
| Cardiac discomfort | Cardiac disorders | MedDRA 23 | Systematic Assessment |
|
| Chest discomfort | General disorders | MedDRA 23 | Systematic Assessment |
|
| Chromaturia | Renal and urinary disorders | MedDRA 23 | Systematic Assessment |
|
| Chronic obstructive pulmonary disease | Respiratory, thoracic and mediastinal disorders | MedDRA 23 | Systematic Assessment |
|
| Complement factor C4 decreased | Investigations | MedDRA 23 | Systematic Assessment |
|
| Conjunctivitis | Infections and infestations | MedDRA 23 | Systematic Assessment |
|
| Constipation | Gastrointestinal disorders | MedDRA 23 | Systematic Assessment |
|
| Cough | Respiratory, thoracic and mediastinal disorders | MedDRA 23 | Systematic Assessment |
|
| Crohn's disease | Gastrointestinal disorders | MedDRA 23 | Systematic Assessment |
|
| Cystitis | Infections and infestations | MedDRA 23 | Systematic Assessment |
|
| Cytomegalovirus infection reactivation | Infections and infestations | MedDRA 23 | Systematic Assessment |
|
| Decreased appetite | Metabolism and nutrition disorders | MedDRA 23 | Systematic Assessment |
|
| Delirium | Psychiatric disorders | MedDRA 23 | Systematic Assessment |
|
| Dermatitis contact | Skin and subcutaneous tissue disorders | MedDRA 23 | Systematic Assessment |
|
| Diarrhoea | Gastrointestinal disorders | MedDRA 23 | Systematic Assessment |
|
| Disorientation | Psychiatric disorders | MedDRA 23 | Systematic Assessment |
|
| Disturbance in attention | Nervous system disorders | MedDRA 23 | Systematic Assessment |
|
| Dizziness | Nervous system disorders | MedDRA 23 | Systematic Assessment |
|
| Drug eruption | Skin and subcutaneous tissue disorders | MedDRA 23 | Systematic Assessment |
|
| Dry mouth | Gastrointestinal disorders | MedDRA 23 | Systematic Assessment |
|
| Dry skin | Skin and subcutaneous tissue disorders | MedDRA 23 | Systematic Assessment |
|
| Dysphagia | Gastrointestinal disorders | MedDRA 23 | Systematic Assessment |
|
| Dyspnoea | Respiratory, thoracic and mediastinal disorders | MedDRA 23 | Systematic Assessment |
|
| Dysuria | Renal and urinary disorders | MedDRA 23 | Systematic Assessment |
|
| Eosinophilia | Blood and lymphatic system disorders | MedDRA 23 | Systematic Assessment |
|
| Eosinophilic colitis | Gastrointestinal disorders | MedDRA 23 | Systematic Assessment |
|
| Fluid overload | Metabolism and nutrition disorders | MedDRA 23 | Systematic Assessment |
|
| Fluid retention | Metabolism and nutrition disorders | MedDRA 23 | Systematic Assessment |
|
| Flushing | Vascular disorders | MedDRA 23 | Systematic Assessment |
|
| Fungal infection | Infections and infestations | MedDRA 23 | Systematic Assessment |
|
| Gastroenteritis | Infections and infestations | MedDRA 23 | Systematic Assessment |
|
| Gastroenteritis viral | Infections and infestations | MedDRA 23 | Systematic Assessment |
|
| Gastrooesophageal reflux disease | Gastrointestinal disorders | MedDRA 23 | Systematic Assessment |
|
| Haemarthrosis | Musculoskeletal and connective tissue disorders | MedDRA 23 | Systematic Assessment |
|
| Haemolysis | Blood and lymphatic system disorders | MedDRA 23 | Systematic Assessment |
|
| Headache | Nervous system disorders | MedDRA 23 | Systematic Assessment |
|
| Hepatotoxicity | Hepatobiliary disorders | MedDRA 23 | Systematic Assessment |
|
| Humerus fracture | Injury, poisoning and procedural complications | MedDRA 23 | Systematic Assessment |
|
| Hypertension | Vascular disorders | MedDRA 23 | Systematic Assessment |
|
| Hyperthyroidism | Endocrine disorders | MedDRA 23 | Systematic Assessment |
|
| Hyperuricaemia | Metabolism and nutrition disorders | MedDRA 23 | Systematic Assessment |
|
| Hypokalaemia | Metabolism and nutrition disorders | MedDRA 23 | Systematic Assessment |
|
| Hypomagnesaemia | Metabolism and nutrition disorders | MedDRA 23 | Systematic Assessment |
|
| Hyponatraemia | Metabolism and nutrition disorders | MedDRA 23 | Systematic Assessment |
|
| Hypotension | Vascular disorders | MedDRA 23 | Systematic Assessment |
|
| Hypoxia | Respiratory, thoracic and mediastinal disorders | MedDRA 23 | Systematic Assessment |
|
| Ileus | Gastrointestinal disorders | MedDRA 23 | Systematic Assessment |
|
| Influenza | Infections and infestations | MedDRA 23 | Systematic Assessment |
|
| Influenza like illness | General disorders | MedDRA 23 | Systematic Assessment |
|
| Injection site pain | General disorders | MedDRA 23 | Systematic Assessment |
|
| Insomnia | Psychiatric disorders | MedDRA 23 | Systematic Assessment |
|
| Irritable bowel syndrome | Gastrointestinal disorders | MedDRA 23 | Systematic Assessment |
|
| Jaundice | Hepatobiliary disorders | MedDRA 23 | Systematic Assessment |
|
| Leukopenia | Blood and lymphatic system disorders | MedDRA 23 | Systematic Assessment |
|
| Lichen planus | Skin and subcutaneous tissue disorders | MedDRA 23 | Systematic Assessment |
|
| Lipids increased | Investigations | MedDRA 23 | Systematic Assessment |
|
| Lipoma | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA 23 | Systematic Assessment |
|
| Lymphocyte count decreased | Investigations | MedDRA 23 | Systematic Assessment |
|
| Macular oedema | Eye disorders | MedDRA 23 | Systematic Assessment |
|
| Mitral valve incompetence | Cardiac disorders | MedDRA 23 | Systematic Assessment |
|
| Muscle spasms | Musculoskeletal and connective tissue disorders | MedDRA 23 | Systematic Assessment |
|
| Muscular weakness | Musculoskeletal and connective tissue disorders | MedDRA 23 | Systematic Assessment |
|
| Musculoskeletal pain | Musculoskeletal and connective tissue disorders | MedDRA 23 | Systematic Assessment |
|
| Myalgia | Musculoskeletal and connective tissue disorders | MedDRA 23 | Systematic Assessment |
|
| Nausea | Gastrointestinal disorders | MedDRA 23 | Systematic Assessment |
|
| Neutropenia | Blood and lymphatic system disorders | MedDRA 23 | Systematic Assessment |
|
| Neutrophil count decreased | Investigations | MedDRA 23 | Systematic Assessment |
|
| Non-Hodgkin's lymphoma | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA 23 | Systematic Assessment |
|
| Oedema genital | Reproductive system and breast disorders | MedDRA 23 | Systematic Assessment |
|
| Oedema peripheral | General disorders | MedDRA 23 | Systematic Assessment |
|
| Oral candidiasis | Infections and infestations | MedDRA 23 | Systematic Assessment |
|
| Oropharyngeal candidiasis | Infections and infestations | MedDRA 23 | Systematic Assessment |
|
| Oropharyngeal pain | Respiratory, thoracic and mediastinal disorders | MedDRA 23 | Systematic Assessment |
|
| Osteoarthritis | Musculoskeletal and connective tissue disorders | MedDRA 23 | Systematic Assessment |
|
| Osteoporosis | Musculoskeletal and connective tissue disorders | MedDRA 23 | Systematic Assessment |
|
| Pain in extremity | Musculoskeletal and connective tissue disorders | MedDRA 23 | Systematic Assessment |
|
| Palmoplantar keratoderma | Skin and subcutaneous tissue disorders | MedDRA 23 | Systematic Assessment |
|
| Palpitations | Cardiac disorders | MedDRA 23 | Systematic Assessment |
|
| Plantar fasciitis | Musculoskeletal and connective tissue disorders | MedDRA 23 | Systematic Assessment |
|
| Pleural effusion | Respiratory, thoracic and mediastinal disorders | MedDRA 23 | Systematic Assessment |
|
| Pneumonia | Infections and infestations | MedDRA 23 | Systematic Assessment |
|
| Pneumonitis | Respiratory, thoracic and mediastinal disorders | MedDRA 23 | Systematic Assessment |
|
| Pneumothorax | Respiratory, thoracic and mediastinal disorders | MedDRA 23 | Systematic Assessment |
|
| Productive cough | Respiratory, thoracic and mediastinal disorders | MedDRA 23 | Systematic Assessment |
|
| Pruritus | Skin and subcutaneous tissue disorders | MedDRA 23 | Systematic Assessment |
|
| Psoriasis | Skin and subcutaneous tissue disorders | MedDRA 23 | Systematic Assessment |
|
| Purpura | Skin and subcutaneous tissue disorders | MedDRA 23 | Systematic Assessment |
|
| Pyrexia | General disorders | MedDRA 23 | Systematic Assessment |
|
| Rash | Skin and subcutaneous tissue disorders | MedDRA 23 | Systematic Assessment |
|
| Rash erythematous | Skin and subcutaneous tissue disorders | MedDRA 23 | Systematic Assessment |
|
| Rash macular | Skin and subcutaneous tissue disorders | MedDRA 23 | Systematic Assessment |
|
| Rash pruritic | Skin and subcutaneous tissue disorders | MedDRA 23 | Systematic Assessment |
|
| SARS-CoV-2 test positive | Investigations | MedDRA 23 | Systematic Assessment |
|
| Sciatica | Nervous system disorders | MedDRA 23 | Systematic Assessment |
|
| Skin ulcer | Skin and subcutaneous tissue disorders | MedDRA 23 | Systematic Assessment |
|
| Supraventricular extrasystoles | Cardiac disorders | MedDRA 23 | Systematic Assessment |
|
| Tachypnoea | Respiratory, thoracic and mediastinal disorders | MedDRA 23 | Systematic Assessment |
|
| Thrombocytopenia | Blood and lymphatic system disorders | MedDRA 23 | Systematic Assessment |
|
| Total complement activity decreased | Investigations | MedDRA 23 | Systematic Assessment |
|
| Tremor | Nervous system disorders | MedDRA 23 | Systematic Assessment |
|
| Upper respiratory tract infection | Infections and infestations | MedDRA 23 | Systematic Assessment |
|
| Urinary tract obstruction | Renal and urinary disorders | MedDRA 23 | Systematic Assessment |
|
| Urine protein/creatinine ratio increased | Investigations | MedDRA 23 | Systematic Assessment |
|
| Vertigo | Ear and labyrinth disorders | MedDRA 23 | Systematic Assessment |
|
| Vitreous haemorrhage | Eye disorders | MedDRA 23 | Systematic Assessment |
|
| Vomiting | Gastrointestinal disorders | MedDRA 23 | Systematic Assessment |
|
Following the first publication, the Institution and/or Principal Investigator may publish data or results from the Study, provided, however, that the Institution and/or Principal Investigator submits the proposed publication to the Sponsor for review at least sixty (60) days prior to the date of the proposed publication. Sponsor may remove from the proposed publication any information that is considered confidential and/or proprietary other than Study data and results.
| D001327 |
| Autoimmune Diseases |
| D007154 | Immune System Diseases |
| D009421 | Nervous System Malformations |
| D009422 | Nervous System Diseases |
| D020271 | Heredodegenerative Disorders, Nervous System |
| D019636 | Neurodegenerative Diseases |
| D011115 | Polyneuropathies |
| D010523 | Peripheral Nervous System Diseases |
| D009468 | Neuromuscular Diseases |
| D000013 | Congenital Abnormalities |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D030342 | Genetic Diseases, Inborn |
| Week 2 |
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| Week 4 |
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| Week 6 |
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| Week 8 |
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| Week 10 |
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| Week 12 |
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| Follow-up Month 1 |
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| Follow-up Month 2 |
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| Follow-up Month 3 |
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| Extension End of Treatment |
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| Week 2 |
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| Week 4 |
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| Week 6 |
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| Week 8 |
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| Week 10 |
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| Week 12 |
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| Follow-up Month 1 |
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| Follow-up Month 2 |
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| Follow-up Month 3 |
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| Extension End of Treatment |
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| Week 2 |
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| Week 4 |
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| Week 6 |
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| Week 8 |
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| Week 10 |
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| Week 12 |
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| Follow-up Month 1 |
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| Follow-up Month 2 |
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| Follow-up Month 3 |
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| Extension End of Treatment |
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| Change from Baseline at Week 1 |
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| Change from Baseline at Week 2 |
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| Change from Baseline at Week 4 |
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| Change from Baseline at Week 6 |
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| Change from Baseline at Week 8 |
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| Change from Baseline at Week 10 |
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| Change from Baseline at Week 12 |
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| Change from Baseline at Follow-up Month 1 |
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| Change from Baseline at Follow-up Month 2 |
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| Change from Baseline at Follow-up Month 3 |
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| Change from Baseline at Extension End of Treatment |
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| Week 2 |
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| Week 4 |
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| Week 6 |
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| Week 8 |
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| Week 10 |
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| Week 12 |
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| Follow-up Month 1 |
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| Follow-up Month 2 |
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| Follow-up Month 3 |
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| Extension End of Treatment |
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| Week 2 |
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| Week 4 |
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| Week 6 |
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| Week 8 |
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| Week 10 |
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| Week 12 |
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| Follow-up Month 1 |
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| Follow-up Month 2 |
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| Follow-up Month 3 |
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| Extension End of Treatment |
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| Hemoglobin, Week 2 |
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| Hemoglobin, Week 4 |
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| Hemoglobin, Week 6 |
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| Hemoglobin, Week 8 |
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| Hemoglobin, Week 10 |
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| Hemoglobin, Week 12 |
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| Hemoglobin, Follow-up Month 1 |
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| Hemoglobin, Follow-up Month 2 |
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| Hemoglobin, Follow-up Month 3 |
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| Haptoglobin, Week 1 |
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| Haptoglobin, Week 2 |
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| Haptoglobin, Week 4 |
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| Haptoglobin, Week 6 |
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| Haptoglobin, Week 8 |
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| Haptoglobin, Week 10 |
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| Haptoglobin, Week 12 |
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| Haptoglobin, Follow-up Month 2 |
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| LDH, Week 1 |
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| LDH, Week 2 |
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| LDH, Week 4 |
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| LDH, Week 6 |
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| LDH, Week 8 |
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| LDH, Week 10 |
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| LDH, Week 12 |
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| LDH, Follow-up Month 2 |
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| Reticulocytes, Week 1 |
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| Reticulocytes, Week 2 |
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| Reticulocytes, Week 4 |
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| Reticulocytes, Week 6 |
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| Reticulocytes, Week 8 |
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| Reticulocytes, Week 10 |
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| Reticulocytes, Week 12 |
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| Reticulocytes, Follow-up Month 1 |
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| Reticulocytes, Follow-up Month 2 |
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| Reticulocytes, Follow-up Month 3 |
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| Total bilirubin, Week 1 |
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| Total bilirubin, Week 2 |
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| Total bilirubin, Week 4 |
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| Total bilirubin, Week 6 |
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| Total bilirubin, Week 8 |
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| Total bilirubin, Week 10 |
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| Total bilirubin, Week 12 |
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| Total bilirubin, Follow-up Month 2 |
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| Direct bilirubin, Week 1 |
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| Direct bilirubin, Week 2 |
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| Direct bilirubin, Week 4 |
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| Direct bilirubin, Week 6 |
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| Direct bilirubin, Week 8 |
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| Direct bilirubin, Week 10 |
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| Direct bilirubin, Week 12 |
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| Indirect bilirubin, Week 1 |
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| Indirect bilirubin, Week 2 |
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| Indirect bilirubin, Week 4 |
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| Indirect bilirubin, Week 6 |
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| Indirect bilirubin, Week 8 |
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| Indirect bilirubin, Week 10 |
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| Indirect bilirubin, Week 12 |
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| Week 2, increased |
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| Week 2, decreased |
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| Week 4, increased |
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| Week 4, decreased |
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| Week 6, increased |
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| Week 6, decreased |
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| Week 8, increased |
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| Week 8, decreased |
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| Week 10, increased |
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| Week 10, decreased |
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| Week 12, increased |
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| Week 12, decreased |
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| Extension End of Treatment, increased |
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| Extension End of Treatment, decreased |
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| Week 6 |
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| Week 12 |
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| Follow-up Month 1 |
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| Follow-up Month 2 |
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| Follow-up Month 3 |
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| Week 8 |
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| Week 8 |
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| Week 8 |
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| Week 8 |
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| Week 8 |
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| Week 8 |
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| Week 8 |
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| Change from Baseline at Week 1 of the TP |
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| Change from Baseline at Week 2 of the TP |
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| Change from Baseline at Week 4 of the TP |
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| Change from Baseline at Week 6 of the TP |
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| Change from Baseline at Week 8 of the TP |
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| Change from Baseline at Week 10 of the TP |
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| Change from Baseline at Week 12 of the TP |
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| Change from Baseline at Follow-up Month 1 of the TP |
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| Change from Baseline at Follow-up Month 2 of the TP |
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| Change from Baseline at Follow-up Month 3 of the TP |
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| Baseline of the Extension Period (EP) |
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| Change from Baseline at End of Treatment during the EP |
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| Change from Baseline in IgG at Week 12 |
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| IgM at Baseline |
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| Change from Baseline in IgM at Week 12 |
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| Change from Baseline at Week 12 |
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| Haptoglobin, Change from Baseline at Week 1 of the TP |
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| Haptoglobin, Change from Baseline at Week 2 of the TP |
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| Haptoglobin, Change from Baseline at Week 4 of the TP |
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| Haptoglobin, Change from Baseline at Week 6 of the TP |
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| Haptoglobin, Change from Baseline at Week 8 of the TP |
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| Haptoglobin, Change from Baseline at Week 10 of the TP |
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| Haptoglobin, Change from Baseline at Week 12 of the TP |
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| Haptoglobin, Change from Baseline at Follow-up Month 2 of the TP |
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| Haptoglobin, Baseline of the EP |
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| Haptoglobin, Change from Baseline at Extension End of Treatment |
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| Indirect bilirubin, Baseline of the TP |
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| Indirect bilirubin, Change from Baseline at Week 1 of the TP |
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| Indirect bilirubin, Change from Baseline at Week 2 of the TP |
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| Indirect bilirubin, Change from Baseline at Week 4 of the TP |
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| Indirect bilirubin, Change from Baseline at Week 6 of the TP |
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| Indirect bilirubin, Change from Baseline at Week 8 of the TP |
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| Indirect bilirubin, Change from Baseline at Week 10 of the TP |
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| Indirect bilirubin, Change from Baseline at Week 12 of the TP |
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| Indirect bilirubin, Baseline of the EP |
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| Indirect bilirubin, Change from Baseline at Extension End of Treatment |
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| Total bilirubin, Baseline of the TP |
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| Total bilirubin, Change from Baseline at Week 1 of the TP |
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| Total bilirubin, Change from Baseline at Week 2 of the TP |
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| Total bilirubin, Change from Baseline at Week 4 of the TP |
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| Total bilirubin, Change from Baseline at Week 6 of the TP |
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| Total bilirubin, Change from Baseline at Week 8 of the TP |
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| Total bilirubin, Change from Baseline at Week 10 of the TP |
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| Total bilirubin, Change from Baseline at Week 12 of the TP |
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| Total bilirubin, Change from Baseline at Follow-up Month 2 of the TP |
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| Total bilirubin, Baseline of the EP |
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| Total bilirubin, Change from Baseline at Extension End of Treatment |
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| Direct bilirubin, Baseline of the TP |
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| Direct bilirubin, Change from Baseline at Week 1 of the TP |
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| Direct bilirubin, Change from Baseline at Week 2 of the TP |
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| Direct bilirubin, Change from Baseline at Week 4 of the TP |
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| Direct bilirubin, Change from Baseline at Week 6 of the TP |
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| Direct bilirubin, Change from Baseline at Week 8 of the TP |
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| Direct bilirubin, Change from Baseline at Week 10 of the TP |
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| Direct bilirubin, Change from Baseline at Week 12 of the TP |
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| Direct bilirubin, Baseline of the EP |
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| Direct bilirubin, Change from Baseline at Extension End of Treatment |
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| Change from Baseline at Week 1 of the TP |
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| Change from Baseline at Week 2 of the TP |
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| Change from Baseline at Week 4 of the TP |
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| Change from Baseline at Week 6 of the TP |
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| Change from Baseline at Week 8 of the TP |
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| Change from Baseline at Week 10 of the TP |
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| Change from Baseline at Week 12 of the TP |
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| Change from Baseline at Follow-up Month 2 of the TP |
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| Baseline of the EP |
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| Change from Baseline at Extension End of Treatment |
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| Change from Baseline at Week 12 in the TP |
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| Baseline in the EP |
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| Change from Baseline at Extension End of Treatment |
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| Complement C3, Change from Baseline at Week 2 of the TP |
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| Complement C3, Change from Baseline at Week 6 of the TP |
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| Complement C3, Change from Baseline at Week 12 of the TP |
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| Complement C3, Baseline of the EP |
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| Complement C3, Change from Baseline at Extension End of Treatment |
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| Complement C4, Baseline of the TP |
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| Complement C4, Change from Baseline at Week 2 of the TP |
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| Complement C4, Change from Baseline at Week 6 of the TP |
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| Complement C4, Change from Baseline at Week 12 of the TP |
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| Complement C4, Baseline of the EP |
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| Complement C4, Change from Baseline at Extension End of Treatment |
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|