Outcome
| Type |
Measure |
Description |
Time frame |
Safety issue |
| Primary |
Least Squares (LS) Mean Change From Baseline to Week 16 in Hemoglobin (Hb) Level During the RCP |
Baseline was the average of measurements recorded before taking the first dose of pegcetacoplan, which included local and central laboratory values during the screening period. Analysis excluded data before the RCP and was censored for transfusions. |
Baseline and Week 16 |
|
| Secondary |
Percentage of Subjects Who Did Not Require a Transfusion (Transfusion Avoidance) During the RCP |
Subjects who experienced more than 1 transfusion during the RCP are only counted once. Subjects who did not have a transfusion but withdrew before Week 16 were considered as having a transfusion in the analysis of transfusion avoidance. |
Day 1 to Week 16 |
|
| Secondary |
LS Mean Change From Baseline to Week 16 in Absolute Reticulocyte Count (ARC) During the RCP |
Baseline was the average of available measurements recorded from central laboratory before taking the first dose of pegcetacoplan. Analysis excluded data before the RCP and was censored for transfusions. |
Baseline and Week 16 |
|
| Secondary |
LS Mean Change From Baseline to Week 16 in Lactate Dehydrogenase (LDH) Level During the RCP |
Baseline was the average of available measurements recorded from central laboratory before taking the first dose of pegcetacoplan. Analysis excluded data before the RCP and was censored for transfusions. |
Baseline and Week 16 |
|
| Secondary |
LS Mean Change From Baseline to Week 16 in Functional Assessment of Chronic Illness Therapy (FACIT) - Fatigue Scale Score During the RCP |
The FACIT-fatigue scale version 4 is a 13-item Likert scaled instrument where the subject was presented with 13 statements and asked to indicate their response as it applied to the past 7 days. The 5 possible responses were 'Not at all' (0), 'A little bit (1), 'Somewhat' (2), 'Quite a bit' (3) and 'Very much' (4). With 13 statements the total score had a range of 0 to 52. A higher score corresponds to a higher quality of life (QoL). Baseline was the last available, nonmissing observation before taking the first dose of pegcetacoplan. Data collected after transfusion is excluded from analysis. |
Baseline and Week 16 |
|
| Secondary |
Percentage of Subjects Who Achieved a Hb Response in the Absence of Transfusions at Week 16 |
Hb response was defined as an increase of at least 1 g/dL in Hb from Baseline at Week 16. Baseline was the average of measurements recorded before taking the first dose of pegcetacoplan, which included local and central laboratory values during the screening period. Analysis excluded data before the RCP and was censored for transfusions. |
Baseline and Week 16 |
|
| Secondary |
Percentage of Subjects Who Achieved Reticulocyte Normalization in the Absence of Transfusions at Week 16 |
Reticulocyte normalization was defined as the ARC being below the upper limit of the gender-specific normal range at Week 16, censored for transfusions. Subjects who received a transfusion between Day 1 and Week 16 or withdrew without providing efficacy data at Week 16 were classified as nonresponders. |
Week 16 |
|
| Secondary |
Percentage of Subjects Who Achieved Hb Normalization in the Absence of Transfusions at Week 16 |
Hb normalization was defined as the Hb level being above the lower limit of the normal range at Week 16, censored for transfusions. Subjects who received a transfusion between Day 1 and Week 16 or withdrew without providing efficacy data at Week 16 are classified as nonnormalization. |
Week 16 |
|
| Secondary |
LS Mean Change From Baseline to Week 16 in Indirect Bilirubin Level During the RCP |
Baseline was the average of available measurements recorded from central laboratory before taking the first dose of pegcetacoplan. Analysis excluded data before the RCP and was censored for transfusions. |
Baseline and Week 16 |
|
| Secondary |
LS Mean Change From Baseline to Week 16 in Haptoglobin Level During the RCP |
Baseline was the average of available measurements recorded from central laboratory before taking the first dose of pegcetacoplan. Analysis excluded data before the RCP and was censored for transfusions. |
Baseline and Week 16 |
|
| Secondary |
LS Mean Change From Baseline to Week 16 in Linear Analog Scale Assessment (LASA) Scores During the RCP |
The LASA consists of 3 items, where the respondents were asked to rate their perceived level of functioning. Specific domains included activity level, ability to carry out daily activities, and an item for overall QoL. Their level of functioning was reported on a 0 to 100 scale with 0 indicates "As low as could be" and 100 indicates "As high as could be". The combined score ranged from 0 to 300, with higher scores corresponding to a higher QoL. |
Baseline and Week 16 |
|
| Secondary |
LS Mean Change From Baseline to Week 16 in European Organisation for Research and Treatment of Cancer (EORTC) Quality of Life Questionnaire - Core 30 Scale (QLQ-C30) Scores During the RCP |
The EORTC QLQ-C30 questionnaire (version 3.0) consists of 30 questions comprised of both multi-item scales and single-item measures to assess overall QoL in subjects. Questions are designated by functional scales, symptom scales, and global subject QoL/overall perceived health status. For the first 28 questions the 4 possible responses are "Not at all' (1), 'A little' (2), 'Quite a bit' (3) and 'Very much' (4). For the remaining 2 questions the response is requested on a 7-point scale from 1 ('Very poor') to 7 ('Excellent'). The raw scale scores were linear transformed, producing scale scores that ranged from 0% to 100%. A high scale score represents a higher response level. Hence for the functional scales and the global health status a higher score indicates a better QoL, whilst for the symptom scale scores this is implied by a lower score. |
Baseline and Week 16 |
|
| Secondary |
Total Number of PRBC Units Transfused During the RCP |
Subjects who withdrew during the RCP before Week 16 will have their number of units of PRBC estimated from the duration they were in the study. |
Day 1 to Week 16 |
|
| Secondary |
Mean Change From Baseline to Week 48 in Hb Level During the Treatment Period |
Baseline was the average of measurements recorded before taking the first dose of pegcetacoplan, which included local and central laboratory values during the screening period. Analysis excluded data before the RCP and was censored for transfusions. |
Baseline and Week 48 |
|
| Secondary |
Mean Change From Week 17 to Week 48 in Hb Level During the Open-label Period |
Baseline was the average of measurements recorded before taking the first dose of pegcetacoplan, which included local and central laboratory values during the screening period. Analysis excluded data before the RCP and was censored for transfusions. |
Week 17 and Week 48 |
|
| Secondary |
Mean Change From Baseline to Week 48 in ARC During the Treatment Period |
Baseline was the average of available measurements recorded from central laboratory before taking the first dose of pegcetacoplan. Analysis excluded data before the RCP and was censored for transfusions. |
Baseline and Week 48 |
|
| Secondary |
Mean Change From Week 17 to Week 48 in ARC During the Open-label Period |
Baseline was the average of available measurements recorded from central laboratory before taking the first dose of pegcetacoplan. Analysis excluded data before the RCP and was censored for transfusions. |
Week 17 and Week 48 |
|
| Secondary |
Mean Change From Baseline to Week 48 in LDH Level During the Treatment Period |
Baseline was the average of available measurements recorded from central laboratory before taking the first dose of pegcetacoplan. Analysis excluded data before the RCP and was censored for transfusions. |
Baseline and Week 48 |
|
| Secondary |
Mean Change From Week 17 to Week 48 in LDH Level During the Open-label Period |
Baseline was the average of available measurements recorded from central laboratory before taking the first dose of pegcetacoplan. Analysis excluded data before the RCP and was censored for transfusions. |
Week 17 and Week 48 |
|
| Secondary |
Mean Change From Baseline to Week 48 in FACIT-Fatigue Scale Score During the Treatment Period |
The FACIT-fatigue scale version 4 is a 13-item Likert scaled instrument where the subject was presented with 13 statements and asked to indicate their response as it applied to the past 7 days. The 5 possible responses were 'Not at all' (0), 'A little bit (1), 'Somewhat' (2), 'Quite a bit' (3) and 'Very much' (4). With 13 statements the total score had a range of 0 to 52. A higher score corresponds to a higher QoL. Baseline was the last available, nonmissing observation before taking the first dose of pegcetacoplan. Data collected after transfusion is excluded from analysis. |
Baseline and Week 48 |
|
| Secondary |
Mean Change From Week 17 to Week 48 in FACIT-Fatigue Scale Score During the Open-label Period |
The FACIT-fatigue scale is a 13 item Likert scaled instrument where the subject was presented with 13 statements and asked to indicate their response as it applied to the past 7 days. The 5 possible responses were 'Not at all' (0), 'A little bit (1), 'Somewhat' (2), 'Quite a bit' (3) and 'Very much' (4). With 13 statements the total score had a range of 0 to 52. Higher score corresponds to a higher QoL. |
Week 17 and Week 48 |
|
| Secondary |
Mean Change From Baseline to Week 48 in LASA Scores During the Treatment Period |
The LASA consists of 3 items, where the respondents were asked to rate their perceived level of functioning. Specific domains included activity level, ability to carry out daily activities, and an item for overall QoL. Their level of functioning was reported on a 0 to 100 scale with 0 indicates "As low as could be" and 100 indicates "As high as could be". The combined score ranged from 0 to 300, with higher scores corresponding to a higher QoL. |
Baseline and Week 48 |
|
| Secondary |
Mean Change From Week 17 to Week 48 in LASA Scores During the Open-label Period |
The FACIT-fatigue scale is a 13 item Likert scaled instrument where the subject was presented with 13 statements and asked to indicate their response as it applied to the past 7 days. The 5 possible responses were 'Not at all' (0), 'A little bit (1), 'Somewhat' (2), 'Quite a bit' (3) and 'Very much' (4). With 13 statements the total score had a range of 0 to 52. Higher score corresponds to a higher QoL. |
Week 17 and Week 48 |
|
| Secondary |
Mean Change From Baseline to Week 48 in QLQ-C30 Scores During the Treatment Period |
The EORTC QLQ-C30 questionnaire (version 3.0) consists of 30 questions comprised of both multi-item scales and single-item measures to assess overall QoL in subjects. Questions are designated by functional scales, symptom scales, and global subject QoL/overall perceived health status. For the first 28 questions the 4 possible responses are 'Not at all' (1), 'A little' (2), 'Quite a bit' (3) and 'Very much' (4). For the remaining 2 questions the response is requested on a 7-point scale from 1 ('Very poor') to 7 ('Excellent'). The raw scale scores were linear transformed, producing scale scores that ranged from 0% to 100%. A high scale score represents a higher response level. Hence for the functional scales and the global health status a higher score indicates a better QoL, whilst for the symptom scale scores this is implied by a lower score. |
Baseline and Week 48 |
|
| Secondary |
Mean Change From Week 17 to Week 48 in QLQ-C30 Scores During the Open-label Period |
The EORTC QLQ-C30 questionnaire (version 3.0) consists of 30 questions comprised of both multi-item scales and single-item measures to assess overall QoL in subjects. Questions are designated by functional scales, symptom scales, and global subject QoL/overall perceived health status. For the first 28 questions the 4 possible responses are 'Not at all' (1), 'A little' (2), 'Quite a bit' (3) and 'Very much' (4). For the remaining 2 questions the response is requested on a 7-point scale from 1 ('Very poor') to 7 ('Excellent'). The raw scale scores were linear transformed, producing scale scores that ranged from 0% to 100%. A high scale score represents a higher response level. Hence for the functional scales and the global health status a higher score indicates a better QoL, whilst for the symptom scale scores this is implied by a lower score. |
Week 17 and Week 48 |
|
| Secondary |
Total Number of PRBC Units Transfused During the Open-Label Period |
Number of units of PRBC transfused to subjects in the open-label period are reported. |
Week 17 to Week 48 |
|
