Paroxysmal Nocturnal Hemoglobinuria
Conditions
Brief summary
Evaluation of the Efficacy and Safety of Pegcetacoplan in Patients with Paroxysmal Nocturnal Hemoglobinuria .
Interventions
DRUGAPL-2
Complement (C3) Inhibitor
Sponsors
Apellis Pharmaceuticals, Inc.
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
Inclusion criteria
* Be at least 18 years old (inclusive). * Have LDH ≥1.5 x ULN at the screening visit. * Have PNH diagnosis, confirmed by high sensitivity flow cytometry (granulocyte or monocyte clone \>10%). * Have Hb less than the lower limit of normal (LLN) at the screening visit. * Have ferritin greater than/equal to the LLN, or total iron binding capacity (TIBC) less than/equal to ULN at the screening visit, based on central laboratory reference ranges. If a subject is receiving iron supplements at screening, the Investigator must ensure that the subject's dose has been stable for 4 weeks prior to screening, and it must be maintained throughout the study. Subjects not receiving iron at screening must not start iron supplementation during the course of the study. * Body mass index (BMI) ≤ 35 kg/m2 at the screening visit. * Have a platelet count of \>50,000/mm3 at the screening visit. * Have an absolute neutrophil count \>500/mm3 at the screening visit.
Exclusion criteria
* Treatment with any complement inhibitor (eg, eculizumab) within 3 months prior to screening. * Hereditary complement deficiency. * History of bone marrow transplantation. * Concomitant use of any of the following medications is prohibited if not on a stable regimen for the time period indicated below prior to screening: * Erythropoietin or immunosuppressants for at least 8 weeks * Systemic corticosteroids for at least 4 weeks * Vitamin K antagonists (eg, warfarin) with a stable international normalized ratio (INR) for at least 4 weeks * Iron supplements, vitamin B12, or folic acid for at least 4 weeks * Low-molecular-weight heparin for at least 4 weeks
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Number of Subjects Who Achieved Hemoglobin (Hb) Stabilization | From Baseline (Day 1) up to Week 26 | The Hb stabilization was defined as avoidance of a \>1 gram per deciliter (g/dL) decrease in Hb concentration from Baseline in the absence of transfusion through Week 26. |
| Change From Baseline in Lactate Dehydrogenase (LDH) Concentration At Week 26 | Baseline (Day 1) and Week 26 | The LDH concentration was analyzed using an analysis of covariance (ANCOVA) model with a last observation carried forward (LOCF) and a baseline observation carried forward (BOCF) approach for handling missing data. Baseline was defined as average of measurements prior to first dose of pegcetacoplan or on or prior to randomization of SoC. Post baseline missing values are imputed using multiple imputation method with Markov Chain Mont Carlo method. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Number of Subjects With an Hb Response in the Absence of Transfusions | Baseline and Week 26 | An Hb response was defined as a =\>1 g/dL increase in Hb from baseline at Week 26. |
| Change From Baseline in Absolute Reticulocyte Count (ARC) at Week 26 | Baseline and Week 26 | Blood samples were collected via direct venipuncture at the specific time points to determine ARC. Baseline was defined as average of measurements prior to first dose of pegcetacoplan or on or prior to randomization of SoC. Post baseline missing values are imputed using multiple imputation method with Markov Chain Mont Carlo method. |
| Change From Baseline in Hb Concentration at Week 26 | Baseline and Week 26 | Baseline was defined as average of measurements prior to first dose of pegcetacoplan or on or prior to randomization of SoC. Post baseline missing values are imputed using multiple imputation method with Markov Chain Mont Carlo method. |
| Percentage of Subjects Who Received Transfusion or Decrease of Hb >2 g/dL From Baseline | At Week 26 | Transfusion refers to any transfusion of PRBC, leukocyte-depleted red blood cells (LDPRC), leukocyte poor packed red blood cell (LPRC), leukocyte poor blood (LPB) or whole blood. |
| Percentage of Subjects With Transfusion Avoidance | At Week 26 | Transfusion avoidance was defined as the percentage of subjects who did not require a transfusion during the RCP. Transfusion refers to any transfusion of PRBC, LDPRC, LPRC, LPB or whole blood. |
| Number of PRBC Units Transfused From Baseline Through Week 26 | Up to Week 26 | The number of units of PRBC transfusions was estimated. In one transfusion subjects received one or more units. |
| Change From Baseline in Functional Assessment of Chronic Illness Therapy- (FACIT-Fatigue) Scale Score at Week 26 | Baseline and Week 26 | The FACIT-Fatigue Scale is a 13-item Likert scaled instrument that is self-administered by the subjects during clinic visits. Subjects were presented with 13 statements and asked to indicate their responses as it applied to the past 7 days. The 5 possible responses are Not at all (0), A little bit (1), Somewhat (2), Quite a bit (3), and Very much (4). With 13 statements, the total score has a range of 0 to 52. The higher score corresponded to a higher quality of life. Baseline is defined as average of measurements prior to first dose of pegcetacoplan or on or prior to randomization of SoC. Post baseline missing values are imputed using multiple imputation method with Markov Chain Mont Carlo method. |
| Percentage of Subjects With Hb Normalization Levels at Week 26 | Baseline and Week 26 | Normalization of Hb levels defined as \>= 1x LLN at Week 26 in the absence of transfusion. Transfusion refers to any transfusion of PRBC, LDPRC, LPRC, LPB or whole blood. |
| Percentage of Subjects With LDH Normalization at Week 26 | At Week 26 | The LDH normalization was defined as LDH \<= 1xupper limit of normal (ULN) of normal range at week 26 in the absence of transfusion. Transfusion refers to any transfusion of PRBC, LDPRC, LPPRC, LPRC, LPB or whole blood. |
| Change From Baseline in European Organization for Research and Treatment of Cancer (EORTC) 30-item Core Quality of Life Questionnaire (QLQ-C30) Scores at Week 26 | Baseline and Week 26 | The EORTC QLQ-C30 questionnaire (version 3.0) consisted of 30 questions comprised of both multi-item scales and single-item measures to assess overall quality of life in subjects. Questions were 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'). Each scale has a range of 0% - 100%. A high scale score represents a higher response level. Baseline is defined as average of measurements prior to first dose of pegcetacoplan or on or prior to randomization of SoC. Post baseline missing values are imputed using multiple imputation method with Markov Chain Mont Carlo method. |
| Change From Baseline in Linear Analog Assessment (LASA) Scales Score at Week 26 | Baseline and Week 26 | The LASA consisted of 3 items asking respondents to rate their perceived level of functioning. Specific domains include activity level, ability to carry out daily activities, and an item for overall QOL. Their level of functioning was reported on a 0-100 scale with 0 representing As low as could be and 100 representing As high as could be. |
| Percentage of Subjects With ARC Normalization | At Week 26 | Absolute reticulocyte count normalization is defined as ARC \< 1x ULN of the gender-specific normal range at week 26 in the absence of transfusion. Subjects who received a transfusion or withdraw from study or escaped from SoC to pegcetacoplan treatment group or lost to follow up without providing efficacy data at Week 26 were classified as non-responders. Transfusion refers to any transfusion of PRBC, LDPRC, LPRC, LPB or whole blood. |
| Number of Subjects With Failure of Hb Stabilization | Up to Week 26 | Hb stabilization is defined as avoidance of a \>1 g/dL decrease in Hb levels from baseline through Week 26 in the absence of transfusion. Transfusion refers to any transfusion of PRBC, LDPRC, LPRC, LPB or whole blood. |
| Time to First PRBC Transfusion | Up to Week 26 | Time to first-on-study PRBC transfusions during RCP were reported. Here NA indicates not estimable. |
Countries
Colombia, Hong Kong, Malaysia, Mexico, Peru, Philippines, Poland, Serbia, Singapore, Thailand
Participant flow
Recruitment details
This study was conducted in subjects with paroxysmal nocturnal hemoglobinuria (PNH) at 22 investigational sites. The study consisted of a screening period (up to 4 weeks), followed by a randomized controlled period (RCP) (26 weeks). All subjects who completed RCP rolled over into a separate open-label, long-term extension study (APL2-307) or completed the safety follow-up (34 weeks).
Pre-assignment details
A total of 68 subjects were screened. Of which, 53 subjects with PNH who met all of the inclusion criteria and none of the exclusion criteria were randomized in a 2:1 ratio either to receive pegcetacoplan or to remain on their current standard of care (SoC) in RCP.
Participants by arm
| Arm | Count |
|---|---|
| Pegcetacoplan Subjects were received SC infusion of pegcetacoplan 1080 milligram (mg) twice weekly or every 3 days up to end of the RCP (Week 26). Subjects were not allowed to receive treatment with other complement inhibitors. | 35 |
| Standard of Care Subjects continued to receive SoC treatment but were not allowed to receive treatment with a complement inhibitor unless they qualified for pegcetacoplan escape therapy. | 18 |
| Total | 53 |
Withdrawals & dropouts
| Period | Reason | FG000 | FG001 |
|---|---|---|---|
| Overall Study | Death | 1 | 1 |
| Overall Study | Lost to Follow-up | 1 | 0 |
Baseline characteristics
| Characteristic | Pegcetacoplan | Standard of Care | Total |
|---|---|---|---|
| Age, Continuous | 42.2 years STANDARD_DEVIATION 12.7 | 49.1 years STANDARD_DEVIATION 15.64 | 44.5 years STANDARD_DEVIATION 14 |
| Age, Customized >=65 and <75 years | 2 Participants | 4 Participants | 6 Participants |
| Age, Customized <65 years | 33 Participants | 14 Participants | 47 Participants |
| Ethnicity (NIH/OMB) Hispanic or Latino | 12 Participants | 2 Participants | 14 Participants |
| Ethnicity (NIH/OMB) Not Hispanic or Latino | 23 Participants | 16 Participants | 39 Participants |
| Ethnicity (NIH/OMB) Unknown or Not Reported | 0 Participants | 0 Participants | 0 Participants |
| Race/Ethnicity, Customized American Indian or Alaska Native | 9 Participants | 2 Participants | 11 Participants |
| Race/Ethnicity, Customized Asian | 23 Participants | 16 Participants | 39 Participants |
| Race/Ethnicity, Customized Black or African American | 2 Participants | 0 Participants | 2 Participants |
| Race/Ethnicity, Customized Other | 1 Participants | 0 Participants | 1 Participants |
| Sex: Female, Male Female | 16 Participants | 8 Participants | 24 Participants |
| Sex: Female, Male Male | 19 Participants | 10 Participants | 29 Participants |
Adverse events
| Event type | EG000 affected / at risk | EG001 affected / at risk |
|---|---|---|
| deaths Total, all-cause mortality | 1 / 46 | 1 / 18 |
| other Total, other adverse events | 35 / 46 | 12 / 18 |
| serious Total, serious adverse events | 6 / 46 | 3 / 18 |
Outcome results
Primary
Change From Baseline in Lactate Dehydrogenase (LDH) Concentration At Week 26
The LDH concentration was analyzed using an analysis of covariance (ANCOVA) model with a last observation carried forward (LOCF) and a baseline observation carried forward (BOCF) approach for handling missing data. Baseline was defined as average of measurements prior to first dose of pegcetacoplan or on or prior to randomization of SoC. Post baseline missing values are imputed using multiple imputation method with Markov Chain Mont Carlo method.
Time frame: Baseline (Day 1) and Week 26
Population: The ITT set included all subjects assigned to treatment.
| Arm | Measure | Value (LEAST_SQUARES_MEAN) | Dispersion |
|---|---|---|---|
| Pegcetacoplan | Change From Baseline in Lactate Dehydrogenase (LDH) Concentration At Week 26 | -1870.47 Units/Liter (U/L) | Standard Error 100.971 |
| Standard of Care | Change From Baseline in Lactate Dehydrogenase (LDH) Concentration At Week 26 | -400.09 Units/Liter (U/L) | Standard Error 312.988 |
p-value: <0.000195% CI: [-2113.44, -827.32]ANCOVA
Primary
Number of Subjects Who Achieved Hemoglobin (Hb) Stabilization
The Hb stabilization was defined as avoidance of a \>1 gram per deciliter (g/dL) decrease in Hb concentration from Baseline in the absence of transfusion through Week 26.
Time frame: From Baseline (Day 1) up to Week 26
Population: The ITT set included all subjects assigned to treatment.
| Arm | Measure | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|
| Pegcetacoplan | Number of Subjects Who Achieved Hemoglobin (Hb) Stabilization | 30 Participants |
| Standard of Care | Number of Subjects Who Achieved Hemoglobin (Hb) Stabilization | 0 Participants |
p-value: <0.000195% CI: [0.572, 0.8902]Cochran-Mantel-Haenszel
Secondary
Change From Baseline in Absolute Reticulocyte Count (ARC) at Week 26
Blood samples were collected via direct venipuncture at the specific time points to determine ARC. Baseline was defined as average of measurements prior to first dose of pegcetacoplan or on or prior to randomization of SoC. Post baseline missing values are imputed using multiple imputation method with Markov Chain Mont Carlo method.
Time frame: Baseline and Week 26
Population: The ITT set included all subjects assigned to treatment.
| Arm | Measure | Value (LEAST_SQUARES_MEAN) | Dispersion |
|---|---|---|---|
| Pegcetacoplan | Change From Baseline in Absolute Reticulocyte Count (ARC) at Week 26 | -123.26 10^9 cells/L | Standard Error 9.164 |
| Standard of Care | Change From Baseline in Absolute Reticulocyte Count (ARC) at Week 26 | -19.44 10^9 cells/L | Standard Error 25.209 |
p-value: 0.000295% CI: [-158.9, -48.74]ANCOVA
Secondary
Change From Baseline in European Organization for Research and Treatment of Cancer (EORTC) 30-item Core Quality of Life Questionnaire (QLQ-C30) Scores at Week 26
The EORTC QLQ-C30 questionnaire (version 3.0) consisted of 30 questions comprised of both multi-item scales and single-item measures to assess overall quality of life in subjects. Questions were 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'). Each scale has a range of 0% - 100%. A high scale score represents a higher response level. Baseline is defined as average of measurements prior to first dose of pegcetacoplan or on or prior to randomization of SoC. Post baseline missing values are imputed using multiple imputation method with Markov Chain Mont Carlo method.
Time frame: Baseline and Week 26
Population: The ITT set included all subjects assigned to treatment.
| Arm | Measure | Value (LEAST_SQUARES_MEAN) | Dispersion |
|---|---|---|---|
| Pegcetacoplan | Change From Baseline in European Organization for Research and Treatment of Cancer (EORTC) 30-item Core Quality of Life Questionnaire (QLQ-C30) Scores at Week 26 | 18.90 scores on a scale | Standard Error 2.909 |
| Standard of Care | Change From Baseline in European Organization for Research and Treatment of Cancer (EORTC) 30-item Core Quality of Life Questionnaire (QLQ-C30) Scores at Week 26 | -2.85 scores on a scale | Standard Error 5.703 |
p-value: 0.000695% CI: [9.35, 34.16]ANCOVA
Secondary
Change From Baseline in Functional Assessment of Chronic Illness Therapy- (FACIT-Fatigue) Scale Score at Week 26
The FACIT-Fatigue Scale is a 13-item Likert scaled instrument that is self-administered by the subjects during clinic visits. Subjects were presented with 13 statements and asked to indicate their responses as it applied to the past 7 days. The 5 possible responses are Not at all (0), A little bit (1), Somewhat (2), Quite a bit (3), and Very much (4). With 13 statements, the total score has a range of 0 to 52. The higher score corresponded to a higher quality of life. Baseline is defined as average of measurements prior to first dose of pegcetacoplan or on or prior to randomization of SoC. Post baseline missing values are imputed using multiple imputation method with Markov Chain Mont Carlo method.
Time frame: Baseline and Week 26
Population: The ITT set included all subjects assigned to treatment.
| Arm | Measure | Value (LEAST_SQUARES_MEAN) | Dispersion |
|---|---|---|---|
| Pegcetacoplan | Change From Baseline in Functional Assessment of Chronic Illness Therapy- (FACIT-Fatigue) Scale Score at Week 26 | 7.78 scores on a scale | Standard Error 1.21 |
| Standard of Care | Change From Baseline in Functional Assessment of Chronic Illness Therapy- (FACIT-Fatigue) Scale Score at Week 26 | 3.26 scores on a scale | Standard Error 2.113 |
p-value: 0.06195% CI: [-0.21, 9.24]ANCOVA
Secondary
Change From Baseline in Hb Concentration at Week 26
Baseline was defined as average of measurements prior to first dose of pegcetacoplan or on or prior to randomization of SoC. Post baseline missing values are imputed using multiple imputation method with Markov Chain Mont Carlo method.
Time frame: Baseline and Week 26
Population: The ITT set included all subjects assigned to treatment.
| Arm | Measure | Value (LEAST_SQUARES_MEAN) | Dispersion |
|---|---|---|---|
| Pegcetacoplan | Change From Baseline in Hb Concentration at Week 26 | 2.94 g/dL | Standard Error 0.383 |
| Standard of Care | Change From Baseline in Hb Concentration at Week 26 | 0.27 g/dL | Standard Error 0.759 |
p-value: 0.001995% CI: [0.99, 4.35]ANCOVA
Secondary
Change From Baseline in Linear Analog Assessment (LASA) Scales Score at Week 26
The LASA consisted of 3 items asking respondents to rate their perceived level of functioning. Specific domains include activity level, ability to carry out daily activities, and an item for overall QOL. Their level of functioning was reported on a 0-100 scale with 0 representing As low as could be and 100 representing As high as could be.
Time frame: Baseline and Week 26
Population: The ITT set included all subjects assigned to treatment.
| Arm | Measure | Value (LEAST_SQUARES_MEAN) | Dispersion |
|---|---|---|---|
| Pegcetacoplan | Change From Baseline in Linear Analog Assessment (LASA) Scales Score at Week 26 | 50.39 scores on a scale | Standard Error 9.062 |
| Standard of Care | Change From Baseline in Linear Analog Assessment (LASA) Scales Score at Week 26 | -5.39 scores on a scale | Standard Error 17.689 |
p-value: 0.00595% CI: [16.83, 94.74]ANCOVA
Secondary
Number of PRBC Units Transfused From Baseline Through Week 26
The number of units of PRBC transfusions was estimated. In one transfusion subjects received one or more units.
Time frame: Up to Week 26
Population: The ITT set included all subjects assigned to treatment.
| Arm | Measure | Value (MEDIAN) |
|---|---|---|
| Pegcetacoplan | Number of PRBC Units Transfused From Baseline Through Week 26 | 0.0 PRBC transfusions |
| Standard of Care | Number of PRBC Units Transfused From Baseline Through Week 26 | 3.0 PRBC transfusions |
p-value: <0.000195% CI: [2, 4]Wilcoxon Rank-Sum Test
Secondary
Number of Subjects With an Hb Response in the Absence of Transfusions
An Hb response was defined as a =\>1 g/dL increase in Hb from baseline at Week 26.
Time frame: Baseline and Week 26
Population: The ITT set included all subjects assigned to treatment.
| Arm | Measure | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|
| Pegcetacoplan | Number of Subjects With an Hb Response in the Absence of Transfusions | 25 Participants |
| Standard of Care | Number of Subjects With an Hb Response in the Absence of Transfusions | 1 Participants |
p-value: <0.000195% CI: [0.339, 0.7431]Cochran-Mantel-Haenszel
Secondary
Number of Subjects With Failure of Hb Stabilization
Hb stabilization is defined as avoidance of a \>1 g/dL decrease in Hb levels from baseline through Week 26 in the absence of transfusion. Transfusion refers to any transfusion of PRBC, LDPRC, LPRC, LPB or whole blood.
Time frame: Up to Week 26
Population: The ITT set included all subjects assigned to treatment.
| Arm | Measure | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|
| Pegcetacoplan | Number of Subjects With Failure of Hb Stabilization | 4 Participants |
| Standard of Care | Number of Subjects With Failure of Hb Stabilization | 18 Participants |
p-value: <0.000195% CI: [0.004, 0.091]Stratified Wilcoxon
Secondary
Percentage of Subjects Who Received Transfusion or Decrease of Hb >2 g/dL From Baseline
Transfusion refers to any transfusion of PRBC, leukocyte-depleted red blood cells (LDPRC), leukocyte poor packed red blood cell (LPRC), leukocyte poor blood (LPB) or whole blood.
Time frame: At Week 26
Population: The ITT set included all subjects assigned to treatment.
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| Pegcetacoplan | Percentage of Subjects Who Received Transfusion or Decrease of Hb >2 g/dL From Baseline | 11.4 percentage of subjects |
| Standard of Care | Percentage of Subjects Who Received Transfusion or Decrease of Hb >2 g/dL From Baseline | 100 percentage of subjects |
p-value: <0.000195% CI: [-0.9041, -0.5969]Cochran-Mantel-Haenszel
Secondary
Percentage of Subjects With ARC Normalization
Absolute reticulocyte count normalization is defined as ARC \< 1x ULN of the gender-specific normal range at week 26 in the absence of transfusion. Subjects who received a transfusion or withdraw from study or escaped from SoC to pegcetacoplan treatment group or lost to follow up without providing efficacy data at Week 26 were classified as non-responders. Transfusion refers to any transfusion of PRBC, LDPRC, LPRC, LPB or whole blood.
Time frame: At Week 26
Population: The ITT set included all subjects assigned to treatment.
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| Pegcetacoplan | Percentage of Subjects With ARC Normalization | 60.0 percentage of subjects |
| Standard of Care | Percentage of Subjects With ARC Normalization | 5.6 percentage of subjects |
p-value: 0.000295% CI: [0.2529, 0.675]Cochran-Mantel-Haenszel
Secondary
Percentage of Subjects With Hb Normalization Levels at Week 26
Normalization of Hb levels defined as \>= 1x LLN at Week 26 in the absence of transfusion. Transfusion refers to any transfusion of PRBC, LDPRC, LPRC, LPB or whole blood.
Time frame: Baseline and Week 26
Population: The ITT set included all subjects assigned to treatment.
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| Pegcetacoplan | Percentage of Subjects With Hb Normalization Levels at Week 26 | 45.7 percentage of subjects |
| Standard of Care | Percentage of Subjects With Hb Normalization Levels at Week 26 | 0 percentage of subjects |
p-value: 0.00195% CI: [0.1648, 0.5642]Cochran-Mantel-Haenszel
Secondary
Percentage of Subjects With LDH Normalization at Week 26
The LDH normalization was defined as LDH \<= 1xupper limit of normal (ULN) of normal range at week 26 in the absence of transfusion. Transfusion refers to any transfusion of PRBC, LDPRC, LPPRC, LPRC, LPB or whole blood.
Time frame: At Week 26
Population: The ITT set included all subjects assigned to treatment.
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| Pegcetacoplan | Percentage of Subjects With LDH Normalization at Week 26 | 65.7 percentage of subjects |
| Standard of Care | Percentage of Subjects With LDH Normalization at Week 26 | 0 percentage of subjects |
p-value: <0.000195% CI: [0.3682, 0.7502]Cochran-Mantel-Haenszel
Secondary
Percentage of Subjects With Transfusion Avoidance
Transfusion avoidance was defined as the percentage of subjects who did not require a transfusion during the RCP. Transfusion refers to any transfusion of PRBC, LDPRC, LPRC, LPB or whole blood.
Time frame: At Week 26
Population: The ITT set included all subjects assigned to treatment.
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| Pegcetacoplan | Percentage of Subjects With Transfusion Avoidance | 91.4 percentage of subjects |
| Standard of Care | Percentage of Subjects With Transfusion Avoidance | 5.6 percentage of subjects |
p-value: <0.000195% CI: [0.5583, 0.8899]Cochran-Mantel-Haenszel
Secondary
Time to First PRBC Transfusion
Time to first-on-study PRBC transfusions during RCP were reported. Here NA indicates not estimable.
Time frame: Up to Week 26
Population: The ITT set included all subjects assigned to treatment.
| Arm | Measure | Value (MEDIAN) |
|---|---|---|
| Pegcetacoplan | Time to First PRBC Transfusion | NA weeks |
| Standard of Care | Time to First PRBC Transfusion | 7.000 weeks |
p-value: <0.000195% CI: [0.005, 0.121]Stratified Wilcoxon