Transplant-Associated Thrombotic Microangiopathy
Conditions
Keywords
TA-TMA
Brief summary
The purpose of the study was to assess pharmacokinetics (PK), pharmacodynamics (PD), efficacy, and safety of pegcetacoplan in patients with TA-TMA after HSCT.
Detailed description
This was a pilot study, and the sample size was based on practical rather than statistical aspects. A total of 12 patients were to be included and treated in the study. With 12 patients included, it was estimated that 9 patients would complete at least 4 weeks of treatment, which is deemed sufficient to characterize the PK of pegcetacoplan in patients with TA-TMA to an appropriate precision. In addition, 12 patients would provide a 72 % probability to observe a response rate of at least 8 responders of the 12 patients recruited (assuming the true response rate is 70 %).
Interventions
DRUGPegcetacoplan
20-cc glass vials-1080 mg
Sponsors
Apellis Pharmaceuticals, Inc.
Swedish Orphan Biovitrum
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Intervention model description
open label, single arm, multicenter
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
Inclusion criteria
1. Male and female patients aged ≥ 18 years at the time of informed consent form (ICF) signature. 2. Received allogeneic HSCT. 3. Diagnosis of TA-TMA established, as per laboratory markers indicating TMA. 4. Have a diagnosis of TA-TMA that persists despite initial management of any triggering condition. 5. Have random urine protein/creatinine ratio (rUPCR) ≥ 1 mg/mg. 6. Women of childbearing potential, defined as any women who have experienced menarche and who are NOT permanently sterile or postmenopausal, must have a negative serum pregnancy test at screening and agree to use protocol-defined methods of contraception for the duration of the study and 8 weeks after their last investigational medicinal product (IMP) dose. Note: Postmenopausal is defined as having had 12 consecutive months with no menses without an alternative medical cause. 7. Men must agree to the following for the duration of the study and 8 weeks after their last dose of IMP: 1. Avoid fathering a child. 2. Use protocol-defined methods of contraception. 3. Refrain from donating sperm. 8. Patient and/or legally authorized representative must be capable of giving signed informed consent, which includes compliance with the requirements and restrictions listed in the ICF.
Exclusion criteria
1. Positive direct Coombs test. 2. Known familial or acquired ADAMTS13 deficiency. 3. Known Shiga toxin-related hemolytic uremic syndrome. 4. Known bone marrow or graft failure. 5. Diagnosis of disseminated intravascular coagulation. 6. Diagnosis of veno-occlusive disease (VOD). 7. Active GI bleeding (hematemesis or hematochezia) at baseline. 8. Body weight \< 30 kg and \> 100 kg. 9. Uncontrolled systemic bacterial or fungal infection, presence or suspicion of sepsis. 10. Previously or currently treated with a complement inhibitor (approved or investigational). 11. Pregnancy or breastfeeding. 12. Positive human immunodeficiency virus antibody at screening or documented in pre-HSCT medical record. 13. Hepatitis C virus detectable by polymerase chain reaction at screening or documented in pre-HSCT medical record. 14. Chronic inactive hepatitis B virus with viral loads \> 1000 IU/mL (\> 5000 copies/mL) at screening or documented in pre-HSCT medical record. Eligible patients who are chronic active carriers (≤ 1000 IU/mL) must receive prophylactic antiviral treatment (e.g., entecavir, tenofovir, lamivudine) according to local country guidelines. 15. Known or suspected hereditary fructose intolerance. 16. Hypersensitivity to pegcetacoplan or any of its excipients. 17. Inability to cooperate with study procedures or any condition that, in the opinion of the investigator, could increase the patient's risk by participating in the study or confound the outcome of the study.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Pegcetacoplan Pharmacokinetic (PK) Parameter Area Under the Curve Limited to the End of Dosing Interval (AUC0-tau) | Week 1 | Area under the concentration-time curve limited to the end of the dosing interval. The samples included in the calculation of AUC0-tau were collected at the following times: on dosing Days 1, 3 and 5, PK samples were taken up to 30 minutes pre-dose and at 15 minutes (± 5 min), 30 minutes (± 5 min), 1 hour (± 10 min), 4 hours (± 10 min), 8 hours (± 30 min), and 24 hours (± 30 min) post-dose as well as on Day 8 pre-dose. |
| Pegcetacoplan PK Parameter Observed Serum Concentration Pre-dose (Ctrough) | Week 1 up to Week 14 | Observed serum concentration pre-dose. From Day 8 (Week 1) and onwards, PK samples were taken pre-dose at each visit. |
| Pegcetacoplan PK Parameter Time to Cmax (Tmax) | Week 1 | Time of maximum measured serum concentration. Determined using the samples collected post-dose on dosing Days 1, 3 and 5. |
| Pegcetacoplan PK Parameter Maximal Serum Concentration (Cmax) | Week 1 | Maximum observed serum concentration. Determined using the samples collected post-dose on dosing Days 1, 3 and 5. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Absolute Levels and Change From Baseline in C3 | Week 24 | Absolute levels and change from baseline to Week 24 in biomarker of complement activation C3 |
| Absolute Levels and Change From Baseline in sC5b-9 | Week 24 | Absolute levels and change from baseline to Week 24 in biomarker of complement activation sC5b-9 |
| Absolute Levels and Change From Baseline in C3a | Week 24 | Absolute levels and change from baseline to Week 24 in biomarker of complement activation C3a |
| Absolute Levels and Change From Baseline in Bb | Week 24 | Absolute levels and change from baseline to Week 24 in biomarker of complement activation Bb |
| Absolute Levels and Change From Baseline in C4a | Week 24 | Absolute levels and change from baseline to Week 24 in biomarker of complement activation C4a |
| Absolute Levels and Change From Baseline in Classical Pathway (CH50) | Week 24 | Absolute levels and change from baseline to Week 24 in biomarker of complement activation CH50 |
| Absolute Levels and Change From Baseline in Alternative Pathway (AH50) | Week 24 | Absolute levels and change from baseline to Week 24 in biomarker of complement activation AH50 |
| Number of Participants Reaching Clinical Response at Week 24 | Week 24 | A participant will be declared as reaching a clinical response upon improvement in laboratory markers of TMA and resolution of TMA clinical symptoms |
| Number of Participants Reaching TMA Response at Week 24 | Week 24 | A participant will be declared as reaching TMA response upon improvement in laboratory markers of TMA |
| Overall Survival at Day 100 | Day 100 from diagnosis | Survival |
| Overall Survival at Week 24 | Week 24 from treatment start | Survival |
| Time to Clinical Response | From treatment start to first documentation of attainment of a clinical response, up to 24 weeks | Both clinical response sustained at week 24 and clinical response at any time during the study will be assessed. |
| Time to TMA Response | From treatment start to first documentation of attainment of a TMA response, up to 24 weeks | Both TMA response sustained at week 24 and TMA response at any time during the study will be assessed. |
| Duration of Clinical Response | From the first observed clinical response until the response criteria is no longer fulfilled or until end of study, up to 24 weeks | Duration of clinical response sustained at week 24 |
| Duration of TMA Response | From the first observed TMA response until the response criteria is no longer fulfilled or until end of study, up to 24 weeks | Duration of TMA response sustained at week 24 |
| TA-TMA Relapse at Week 24 | Week 24 | A participant will be declared as relapsing upon appearance of laboratory markers of TMA |
| Number of Participants Reaching Clinical Response at Week 12 | Week 12 | Number of participants reaching clinical response at week 12 |
| Number of Participants Reaching TMA Response at Week 12 | Week 12 | Number of participants reaching TMA response at week 12 |
Other
| Measure | Time frame | Description |
|---|---|---|
| Change From Baseline in Indirect Bilirubin | Week 24 | Change from baseline to Week 24 in indirect bilirubin |
| Change From Baseline in Serum Creatinine | Week 24 | Change from baseline to Week 24 in serum creatinine |
| Change From Baseline in Urine Protein/Creatinine Ratio | Week 24 | Change from baseline to Week 24 in urine protein/creatinine ratio |
| Change From Baseline in Systolic Blood Pressure | Week 24 | Change from baseline to Week 24 in systolic blood pressure. Post-dose is 30 minutes after stop of infusion. The timepoints from Week 16 onward have a single value as no infusions were administered at these study visits. |
| Change From Baseline in Diastolic Blood Pressure | Week 24 | Change from baseline to Week 24 in diastolic blood pressure. Post-dose is 30 minutes after stop of infusion. The timepoints from Week 16 onward have a single value as no infusions were administered at these study visits. |
| Change From Baseline in Respiratory Rate | Week 24 | Change from baseline to Week 24 in respiratory rate. Post-dose is 30 minutes after stop of infusion. The timepoints from Week 16 onward have a single value as no infusions were administered at these study visits. |
| Change From Baseline in Heart Rate | Week 24 | Change from baseline to Week 24 in heart rate. Post-dose is 30 minutes after stop of infusion. The timepoints from Week 16 onward have a single value as no infusions were administered at these study visits. |
| Change From Baseline in Temperature | Week 24 | Change from baseline to Week 24 in temperature. Post-dose is 30 minutes after stop of infusion. The timepoints from Week 16 onward have a single value as no infusions were administered at these study visits. |
| Number of Participants With Clinically Significant Changes in Abnormal Electrocardiogram Findings | Week 24 | Occurrence of clinically significant abnormal electrocardiogram findings. |
| Number of Participants With Antibodies to Pegcetacoplan Throughout Treatment and Follow-up Periods | from treatment start to the end of the study, up to 6 months. | Presence of antibodies to pegcetacoplan throughout treatment and follow-up periods. |
| Number of Participants With Antibodies to Polyethylene Glycol (PEG) Throughout Treatment and Follow-up Periods | from treatment start to the end of the study, up to 6 months. | Presence of antibodies to PEG throughout treatment and follow-up periods. Baseline represents the situation at treatment start. |
| Number of Participants With Treatment-emergent Anti-PEG Antibodies Throughout Treatment and Follow-up Periods | from treatment start to the end of the study, up to 6 months. | Presence of treatment-emergent anti-PEG antibodies throughout treatment and follow-up periods. Conservatively, the anti-PEG antibodies detected in a patient with a missing baseline sample have been considered as treatment-emergent. |
| Change From Baseline in Platelets | Week 24 | Change from baseline to Week 24 in platelets |
| Change From Baseline in Haptoglobin | Week 24 | Change from baseline to Week 24 in haptoglobin |
| Number of Participants With Treatment-emergent Adverse Events | From treatment start to end of study, up to 6 months (8 weeks since last dose of IMP) | Occurrence and severity of treatment-emergent adverse events. |
| Change From Baseline in Lactate Dehydrogenase (LDH) | Week 24 | Change from baseline to Week 24 in LDH |
| Change From Baseline in Hemoglobin | Week 24 | Change from baseline to Week 24 in hemoglobin |
Countries
France, Greece, Italy, Spain, United States
Participant flow
Pre-assignment details
12 patients were enrolled and treated in study
Participants by arm
| Arm | Count |
|---|---|
| Pegcetacoplan sterile solution in stoppered glass vial given as infusion, for a total treatment duration of 12 to 16 weeks. | 12 |
| Total | 12 |
Withdrawals & dropouts
| Period | Reason | FG000 |
|---|---|---|
| Follow-up Period | Death | 3 |
| Treatment Period | Adverse Event | 1 |
| Treatment Period | Death | 1 |
| Treatment Period | Physician Decision | 1 |
Baseline characteristics
| Characteristic | Pegcetacoplan |
|---|---|
| Absolute number of schistocytes per high power field (hpf) in the peripheral blood smear | 2.4 schistocytes per hpf |
| Age, Categorical <=18 years | 0 Participants |
| Age, Categorical >=65 years | 2 Participants |
| Age, Categorical Between 18 and 65 years | 10 Participants |
| Age, Continuous | 50.5 Years |
| Any biopsy-proven GI involvement of TA-TMA No | 12 Participants |
| Any biopsy-proven GI involvement of TA-TMA Yes | 0 Participants |
| Any sign/symptom of cardiovascular dysfunction No | 8 Participants |
| Any sign/symptom of cardiovascular dysfunction Yes | 4 Participants |
| Any sign/symptom of central nervous system (CNS) dysfunction No | 11 Participants |
| Any sign/symptom of central nervous system (CNS) dysfunction Yes | 1 Participants |
| Any sign/symptom of kidney dysfunction No | 8 Participants |
| Any sign/symptom of kidney dysfunction Yes | 4 Participants |
| Any sign/symptom of lungs dysfunction No | 11 Participants |
| Any sign/symptom of lungs dysfunction Yes | 1 Participants |
| Any sign/symptom of serositis No | 11 Participants |
| Any sign/symptom of serositis Yes | 1 Participants |
| Arterial hypertension No | 6 Participants |
| Arterial hypertension Yes | 6 Participants |
| De novo anemia at diagnosis No | 3 Participants |
| De novo anemia at diagnosis Yes | 5 Participants |
| Elevated plasma concentration of soluble complement 5b-9 (sC5b-9) above upper limit of normal (ULN) Missing | 4 Participants |
| Elevated plasma concentration of soluble complement 5b-9 (sC5b-9) above upper limit of normal (ULN) No | 7 Participants |
| Elevated plasma concentration of soluble complement 5b-9 (sC5b-9) above upper limit of normal (ULN) Yes | 1 Participants |
| Ethnicity (NIH/OMB) Hispanic or Latino | 1 Participants |
| Ethnicity (NIH/OMB) Not Hispanic or Latino | 11 Participants |
| Ethnicity (NIH/OMB) Unknown or Not Reported | 0 Participants |
| Female method of contraception Abstinence | 2 Participants |
| Female method of contraception Injected or implanted hormonal methods | 0 Participants |
| Female method of contraception IUD plus barrier method | 0 Participants |
| Female method of contraception Male Partner Sterilization | 0 Participants |
| Female method of contraception Oral contraception | 0 Participants |
| Female method of contraception Other | 0 Participants |
| Female of childbearing potential No (hysterectomy) | 0 Participants |
| Female of childbearing potential No (post-menopausal more than 1 year) | 4 Participants |
| Female of childbearing potential No (surgical sterilization) | 0 Participants |
| Female of childbearing potential Yes | 2 Participants |
| Height | 165.75 cm |
| Highest platelet value achieved after transplant | 117.5 10^9 platelets/L |
| Is the patient taking any antihypertension medication? No | 1 Participants |
| Is the patient taking any antihypertension medication? Yes | 6 Participants |
| Lactate dehydrogenase (LDH) value at diagnosis | 740.5 U/L |
| Last serum creatinine value pre-transplant | 46.4110 µmol/L |
| Male method of contraception Abstinence | 6 Participants |
| Male method of contraception Condom | 0 Participants |
| Male method of contraception Other | 0 Participants |
| Male method of contraception Sterilization | 0 Participants |
| Most recent serum creatinine value | 194.4835 µmol/L |
| Number of ongoing medications excluding diuretics 0 medication | 0 Participants |
| Number of ongoing medications excluding diuretics ≤ 2 medications | 4 Participants |
| Number of ongoing medications excluding diuretics > 2 medications | 2 Participants |
| Patients receiving invasive positive pressure ventilation No | 0 Participants |
| Patients receiving invasive positive pressure ventilation Yes | 1 Participants |
| Patients receiving noninvasive positive pressure ventilation No | 1 Participants |
| Patients receiving noninvasive positive pressure ventilation Yes | 0 Participants |
| Patients requiring pericardiocentesis No | 0 Participants |
| Patients requiring pericardiocentesis Yes | 1 Participants |
| Patients requiring renal replacement therapy No | 3 Participants |
| Patients requiring renal replacement therapy Yes | 1 Participants |
| Patients with diagnosis of posterior reversible encephalopathy syndrome (PRES) No | 1 Participants |
| Patients with diagnosis of posterior reversible encephalopathy syndrome (PRES) Yes | 0 Participants |
| Patients with pericardial effusion No | 0 Participants |
| Patients with pericardial effusion Yes | 1 Participants |
| Patients with pulmonary hypertension diagnosis No | 4 Participants |
| Patients with pulmonary hypertension diagnosis Yes | 0 Participants |
| Platelet value at diagnosis | 21.5 10^9 platelets/L |
| Presence of schistocytes in the peripheral blood smear No | 0 Participants |
| Presence of schistocytes in the peripheral blood smear Yes | 12 Participants |
| Proteinuria at diagnosis Missing | 1 Participants |
| Proteinuria at diagnosis No | 2 Participants |
| Proteinuria at diagnosis Yes | 9 Participants |
| Race (NIH/OMB) American Indian or Alaska Native | 0 Participants |
| Race (NIH/OMB) Asian | 0 Participants |
| Race (NIH/OMB) Black or African American | 0 Participants |
| Race (NIH/OMB) More than one race | 0 Participants |
| Race (NIH/OMB) Native Hawaiian or Other Pacific Islander | 0 Participants |
| Race (NIH/OMB) Unknown or Not Reported | 0 Participants |
| Race (NIH/OMB) White | 12 Participants |
| Region of Enrollment Greece | 7 participants |
| Region of Enrollment Spain | 2 participants |
| Region of Enrollment United States | 3 participants |
| Sex: Female, Male Female | 6 Participants |
| Sex: Female, Male Male | 6 Participants |
| TA-TMA persisting despite initial management of any triggering condition No | 0 Participants |
| TA-TMA persisting despite initial management of any triggering condition Yes | 12 Participants |
| The lowest oxygen level in the blood | 83.0 % |
| The most recent pre-transfusion Hb level at diagnosis | 78.0 g/L |
| The most recent proteinuria value at diagnosis | 0.8475 g/L |
| The most recent random urine protein/creatinine ratio (rUPCR) value at diagnosis | 2.0 mg/mg Cr |
| The pre-antihypertensive therapy diastolic blood pressure | 91.0 mmHg |
| The pre-antihypertensive therapy systolic blood pressure | 145.0 mmHg |
| Time between HSCT and TA-TMA diagnosis | 27.5 Days |
| Time between TA-TMA diagnosis and enrollment to the study | 6.5 Days |
| Was any biopsy performed to confirm the histologic diagnosis of TA-TMA? No | 12 Participants |
| Was any biopsy performed to confirm the histologic diagnosis of TA-TMA? Yes | 0 Participants |
| Weight | 63.0 kg |
Adverse events
| Event type | EG000 affected / at risk |
|---|---|
| deaths Total, all-cause mortality | 6 / 12 |
| other Total, other adverse events | 5 / 12 |
| serious Total, serious adverse events | 8 / 12 |
Outcome results
Primary
Pegcetacoplan Pharmacokinetic (PK) Parameter Area Under the Curve Limited to the End of Dosing Interval (AUC0-tau)
Area under the concentration-time curve limited to the end of the dosing interval. The samples included in the calculation of AUC0-tau were collected at the following times: on dosing Days 1, 3 and 5, PK samples were taken up to 30 minutes pre-dose and at 15 minutes (± 5 min), 30 minutes (± 5 min), 1 hour (± 10 min), 4 hours (± 10 min), 8 hours (± 30 min), and 24 hours (± 30 min) post-dose as well as on Day 8 pre-dose.
Time frame: Week 1
Population: On Day 1 and 5 the AUC0-tau parameters did not meet lambda-z acceptance criteria for 2 patients, so they were not included. On Day 8 the AUC0-tau parameter did not meet lambda-z acceptance criteria for 1 patient, so they were not included.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Pegcetacoplan | Pegcetacoplan Pharmacokinetic (PK) Parameter Area Under the Curve Limited to the End of Dosing Interval (AUC0-tau) | Day 1-Day 3 | 14934.65 µg*h/mL | Standard Deviation 4448.067 |
| Pegcetacoplan | Pegcetacoplan Pharmacokinetic (PK) Parameter Area Under the Curve Limited to the End of Dosing Interval (AUC0-tau) | Day 3-Day 5 | 20089.79 µg*h/mL | Standard Deviation 5162.653 |
| Pegcetacoplan | Pegcetacoplan Pharmacokinetic (PK) Parameter Area Under the Curve Limited to the End of Dosing Interval (AUC0-tau) | Day 5-Day 8 | 36208.37 µg*h/mL | Standard Deviation 10685.626 |
Primary
Pegcetacoplan PK Parameter Maximal Serum Concentration (Cmax)
Maximum observed serum concentration. Determined using the samples collected post-dose on dosing Days 1, 3 and 5.
Time frame: Week 1
Population: On Day 1, Cmax could not be estimated for 2 patients due to a) no sample provided; and b) Cmax considered unreliable due to pegcetacoplan positive pre-dose sample. On Day 3 and Day 5, Cmax could not be estimated for 1 patient due to a limited number of samples provided.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Pegcetacoplan | Pegcetacoplan PK Parameter Maximal Serum Concentration (Cmax) | Day 1 | 508.1 µg/mL | Standard Deviation 134.86 |
| Pegcetacoplan | Pegcetacoplan PK Parameter Maximal Serum Concentration (Cmax) | Day 3 | 579.6 µg/mL | Standard Deviation 142.11 |
| Pegcetacoplan | Pegcetacoplan PK Parameter Maximal Serum Concentration (Cmax) | Day 5 | 705.2 µg/mL | Standard Deviation 203.04 |
Primary
Pegcetacoplan PK Parameter Observed Serum Concentration Pre-dose (Ctrough)
Observed serum concentration pre-dose. From Day 8 (Week 1) and onwards, PK samples were taken pre-dose at each visit.
Time frame: Week 1 up to Week 14
Population: The number of patients in the analysis population for Ctrough decreased over the course of the study, from 11 at Week 1 to 7 at Week 14, reflecting the limited number of samples available due to study discontinuation of patients.
| Arm | Measure | Group | Value (GEOMETRIC_MEAN) |
|---|---|---|---|
| Pegcetacoplan | Pegcetacoplan PK Parameter Observed Serum Concentration Pre-dose (Ctrough) | Week 1 | 365.22 µg/mL |
| Pegcetacoplan | Pegcetacoplan PK Parameter Observed Serum Concentration Pre-dose (Ctrough) | Week 2 | 449.86 µg/mL |
| Pegcetacoplan | Pegcetacoplan PK Parameter Observed Serum Concentration Pre-dose (Ctrough) | Week 3 | 585.71 µg/mL |
| Pegcetacoplan | Pegcetacoplan PK Parameter Observed Serum Concentration Pre-dose (Ctrough) | Week 4 | 665.32 µg/mL |
| Pegcetacoplan | Pegcetacoplan PK Parameter Observed Serum Concentration Pre-dose (Ctrough) | Week 6 | 750.82 µg/mL |
| Pegcetacoplan | Pegcetacoplan PK Parameter Observed Serum Concentration Pre-dose (Ctrough) | Week 8 | 826.94 µg/mL |
| Pegcetacoplan | Pegcetacoplan PK Parameter Observed Serum Concentration Pre-dose (Ctrough) | Week 10 | 761.76 µg/mL |
| Pegcetacoplan | Pegcetacoplan PK Parameter Observed Serum Concentration Pre-dose (Ctrough) | Week 12 | 466.29 µg/mL |
| Pegcetacoplan | Pegcetacoplan PK Parameter Observed Serum Concentration Pre-dose (Ctrough) | Week 14 | 893.49 µg/mL |
Primary
Pegcetacoplan PK Parameter Time to Cmax (Tmax)
Time of maximum measured serum concentration. Determined using the samples collected post-dose on dosing Days 1, 3 and 5.
Time frame: Week 1
Population: On Day 1, Tmax could not be estimated for 2 patients due to a) no sample provided; and b) Tmax considered unreliable due to pegcetacoplan positive pre-dose sample. On Day 3 and Day 5, Tmax could not be estimated for one patient due to a limited number of samples provided.
| Arm | Measure | Group | Value (MEDIAN) |
|---|---|---|---|
| Pegcetacoplan | Pegcetacoplan PK Parameter Time to Cmax (Tmax) | Day 1 | 1.0 Hour |
| Pegcetacoplan | Pegcetacoplan PK Parameter Time to Cmax (Tmax) | Day 3 | 1.02 Hour |
| Pegcetacoplan | Pegcetacoplan PK Parameter Time to Cmax (Tmax) | Day 5 | 1.05 Hour |
Secondary
Absolute Levels and Change From Baseline in Alternative Pathway (AH50)
Absolute levels and change from baseline to Week 24 in biomarker of complement activation AH50
Time frame: Week 24
Population: The PD set includes all patients in the ITT set who receive IMP and have at least 1 evaluable post-dose PD measurement. The number of patients in the analysis population decreased over the course of the study reflecting the limited number of samples available due to study discontinuation of patients. Patients with an available measure both at the analyzed time point and at baseline are included. In some cases, there were also missing samples.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Pegcetacoplan | Absolute Levels and Change From Baseline in Alternative Pathway (AH50) | Baseline Absolute Level | 125.8 U/mL | Standard Deviation 44.75 |
| Pegcetacoplan | Absolute Levels and Change From Baseline in Alternative Pathway (AH50) | Week 1 Absolute Level | 75.0 U/mL | Standard Deviation 57.85 |
| Pegcetacoplan | Absolute Levels and Change From Baseline in Alternative Pathway (AH50) | Week 1 Change from Baseline | -55.3 U/mL | Standard Deviation 49.73 |
| Pegcetacoplan | Absolute Levels and Change From Baseline in Alternative Pathway (AH50) | Week 2 Absolute Level | 65.9 U/mL | Standard Deviation 49.1 |
| Pegcetacoplan | Absolute Levels and Change From Baseline in Alternative Pathway (AH50) | Week 2 Change from Baseline | -47.7 U/mL | Standard Deviation 49.15 |
| Pegcetacoplan | Absolute Levels and Change From Baseline in Alternative Pathway (AH50) | Week 3 Absolute Level | 34.7 U/mL | Standard Deviation 34.38 |
| Pegcetacoplan | Absolute Levels and Change From Baseline in Alternative Pathway (AH50) | Week 3 Change from Baseline | -80.7 U/mL | Standard Deviation 38.62 |
| Pegcetacoplan | Absolute Levels and Change From Baseline in Alternative Pathway (AH50) | Week 4 Absolute Level | 35.4 U/mL | Standard Deviation 38.46 |
| Pegcetacoplan | Absolute Levels and Change From Baseline in Alternative Pathway (AH50) | Week 4 Change from Baseline | -79.9 U/mL | Standard Deviation 36.02 |
| Pegcetacoplan | Absolute Levels and Change From Baseline in Alternative Pathway (AH50) | Week 6 Absolute Level | 24.0 U/mL | Standard Deviation 40.27 |
| Pegcetacoplan | Absolute Levels and Change From Baseline in Alternative Pathway (AH50) | Week 6 Change from Baseline | -85.3 U/mL | Standard Deviation 38.05 |
| Pegcetacoplan | Absolute Levels and Change From Baseline in Alternative Pathway (AH50) | Week 8 Absolute Level | 23.6 U/mL | Standard Deviation 43.47 |
| Pegcetacoplan | Absolute Levels and Change From Baseline in Alternative Pathway (AH50) | Week 8 Change from Baseline | -85.8 U/mL | Standard Deviation 43.81 |
| Pegcetacoplan | Absolute Levels and Change From Baseline in Alternative Pathway (AH50) | Week 10 Absolute Level | 36.9 U/mL | Standard Deviation 54.84 |
| Pegcetacoplan | Absolute Levels and Change From Baseline in Alternative Pathway (AH50) | Week 10 Change from Baseline | -71.3 U/mL | Standard Deviation 55.56 |
| Pegcetacoplan | Absolute Levels and Change From Baseline in Alternative Pathway (AH50) | Week 12 Absolute Level | 60.6 U/mL | Standard Deviation 69.54 |
| Pegcetacoplan | Absolute Levels and Change From Baseline in Alternative Pathway (AH50) | Week 12 Change from Baseline | -49.5 U/mL | Standard Deviation 43.34 |
| Pegcetacoplan | Absolute Levels and Change From Baseline in Alternative Pathway (AH50) | Week 14 Absolute Level | 20.0 U/mL | Standard Deviation 41.81 |
| Pegcetacoplan | Absolute Levels and Change From Baseline in Alternative Pathway (AH50) | Week 14 Change from Baseline | -96.5 U/mL | Standard Deviation 45.17 |
| Pegcetacoplan | Absolute Levels and Change From Baseline in Alternative Pathway (AH50) | Week 16 Absolute Level | 35.9 U/mL | Standard Deviation 51.88 |
| Pegcetacoplan | Absolute Levels and Change From Baseline in Alternative Pathway (AH50) | Week 16 Change from Baseline | -84.8 U/mL | Standard Deviation 37.55 |
| Pegcetacoplan | Absolute Levels and Change From Baseline in Alternative Pathway (AH50) | Week 20 Absolute Level | 118.1 U/mL | Standard Deviation 23.74 |
| Pegcetacoplan | Absolute Levels and Change From Baseline in Alternative Pathway (AH50) | Week 20 Change from Baseline | 7.8 U/mL | Standard Deviation 55.02 |
| Pegcetacoplan | Absolute Levels and Change From Baseline in Alternative Pathway (AH50) | Week 24 Absolute Level | 129.4 U/mL | Standard Deviation 19.03 |
| Pegcetacoplan | Absolute Levels and Change From Baseline in Alternative Pathway (AH50) | Week 24 Change from Baseline | 11.5 U/mL | Standard Deviation 63.67 |
Secondary
Absolute Levels and Change From Baseline in Bb
Absolute levels and change from baseline to Week 24 in biomarker of complement activation Bb
Time frame: Week 24
Population: The PD set includes all patients in the ITT set who receive IMP and have at least 1 evaluable post-dose PD measurement. The number of patients in the analysis population decreased over the course of the study reflecting the limited number of samples available due to study discontinuation of patients. Patients with an available measure both at the analyzed time point and at baseline are included. In some cases, there were also missing samples.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Pegcetacoplan | Absolute Levels and Change From Baseline in Bb | Baseline Absolute Level | 2.098 mg/L | Standard Deviation 1.5087 |
| Pegcetacoplan | Absolute Levels and Change From Baseline in Bb | Week 1 Absolute Level | 0.769 mg/L | Standard Deviation 0.4819 |
| Pegcetacoplan | Absolute Levels and Change From Baseline in Bb | Week 1 Change from Baseline | -1.404 mg/L | Standard Deviation 1.225 |
| Pegcetacoplan | Absolute Levels and Change From Baseline in Bb | Week 2 Absolute Level | 0.726 mg/L | Standard Deviation 0.2783 |
| Pegcetacoplan | Absolute Levels and Change From Baseline in Bb | Week 2 Change from Baseline | -1.354 mg/L | Standard Deviation 1.4238 |
| Pegcetacoplan | Absolute Levels and Change From Baseline in Bb | Week 3 Absolute Level | 0.611 mg/L | Standard Deviation 0.1335 |
| Pegcetacoplan | Absolute Levels and Change From Baseline in Bb | Week 3 Change from Baseline | -1.469 mg/L | Standard Deviation 1.6091 |
| Pegcetacoplan | Absolute Levels and Change From Baseline in Bb | Week 4 Absolute Level | 0.749 mg/L | Standard Deviation 0.1857 |
| Pegcetacoplan | Absolute Levels and Change From Baseline in Bb | Week 4 Change from Baseline | -1.458 mg/L | Standard Deviation 1.6256 |
| Pegcetacoplan | Absolute Levels and Change From Baseline in Bb | Week 6 Absolute Level | 0.617 mg/L | Standard Deviation 0.1485 |
| Pegcetacoplan | Absolute Levels and Change From Baseline in Bb | Week 6 Change from Baseline | -1.720 mg/L | Standard Deviation 1.717 |
| Pegcetacoplan | Absolute Levels and Change From Baseline in Bb | Week 8 Absolute Level | 0.632 mg/L | Standard Deviation 0.1308 |
| Pegcetacoplan | Absolute Levels and Change From Baseline in Bb | Week 8 Change from Baseline | -1.858 mg/L | Standard Deviation 1.8627 |
| Pegcetacoplan | Absolute Levels and Change From Baseline in Bb | Week 10 Absolute Level | 0.704 mg/L | Standard Deviation 0.2522 |
| Pegcetacoplan | Absolute Levels and Change From Baseline in Bb | Week 10 Change from Baseline | -1.633 mg/L | Standard Deviation 1.625 |
| Pegcetacoplan | Absolute Levels and Change From Baseline in Bb | Week 12 Absolute Level | 0.746 mg/L | Standard Deviation 0.334 |
| Pegcetacoplan | Absolute Levels and Change From Baseline in Bb | Week 12 Change from Baseline | -2.034 mg/L | Standard Deviation 1.6738 |
| Pegcetacoplan | Absolute Levels and Change From Baseline in Bb | Week 14 Absolute Level | 0.528 mg/L | Standard Deviation 0.1555 |
| Pegcetacoplan | Absolute Levels and Change From Baseline in Bb | Week 14 Change from Baseline | -1.968 mg/L | Standard Deviation 1.9232 |
| Pegcetacoplan | Absolute Levels and Change From Baseline in Bb | Week 16 Absolute Level | 0.672 mg/L | Standard Deviation 0.3385 |
| Pegcetacoplan | Absolute Levels and Change From Baseline in Bb | Week 16 Change from Baseline | -1.824 mg/L | Standard Deviation 1.9516 |
| Pegcetacoplan | Absolute Levels and Change From Baseline in Bb | Week 20 Absolute Level | 0.600 mg/L | Standard Deviation 0.2507 |
| Pegcetacoplan | Absolute Levels and Change From Baseline in Bb | Week 20 Change from Baseline | -0.966 mg/L | Standard Deviation 1.127 |
| Pegcetacoplan | Absolute Levels and Change From Baseline in Bb | Week 24 Absolute Level | 0.746 mg/L | Standard Deviation 0.0631 |
| Pegcetacoplan | Absolute Levels and Change From Baseline in Bb | Week 24 Change from Baseline | -1.015 mg/L | Standard Deviation 1.197 |
Secondary
Absolute Levels and Change From Baseline in C3
Absolute levels and change from baseline to Week 24 in biomarker of complement activation C3
Time frame: Week 24
Population: The PD set includes all patients in the ITT set who receive IMP and have at least 1 evaluable post-dose PD measurement. The number of patients in the analysis population decreased over the course of the study reflecting the limited number of samples available due to study discontinuation of patients. Patients with an available measure both at the analyzed time point and at baseline are included. In some cases, there were also missing samples.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Pegcetacoplan | Absolute Levels and Change From Baseline in C3 | Baseline Absolute Level | 1.095 g/L | Standard Deviation 0.2893 |
| Pegcetacoplan | Absolute Levels and Change From Baseline in C3 | Week 1 Absolute Level | 2.500 g/L | Standard Deviation 0.6503 |
| Pegcetacoplan | Absolute Levels and Change From Baseline in C3 | Week 1 Change from Baseline | 1.313 g/L | Standard Deviation 0.5038 |
| Pegcetacoplan | Absolute Levels and Change From Baseline in C3 | Week 2 Absolute Level | 3.095 g/L | Standard Deviation 0.8883 |
| Pegcetacoplan | Absolute Levels and Change From Baseline in C3 | Week 2 Change from Baseline | 1.929 g/L | Standard Deviation 0.7034 |
| Pegcetacoplan | Absolute Levels and Change From Baseline in C3 | Week 3 Absolute Level | 2.883 g/L | Standard Deviation 0.9188 |
| Pegcetacoplan | Absolute Levels and Change From Baseline in C3 | Week 3 Change from Baseline | 1.824 g/L | Standard Deviation 0.7698 |
| Pegcetacoplan | Absolute Levels and Change From Baseline in C3 | Week 4 Absolute Level | 3.716 g/L | Standard Deviation 0.9693 |
| Pegcetacoplan | Absolute Levels and Change From Baseline in C3 | Week 4 Change from Baseline | 2.399 g/L | Standard Deviation 0.7395 |
| Pegcetacoplan | Absolute Levels and Change From Baseline in C3 | Week 6 Absolute Level | 3.934 g/L | Standard Deviation 0.8033 |
| Pegcetacoplan | Absolute Levels and Change From Baseline in C3 | Week 6 Change from Baseline | 2.777 g/L | Standard Deviation 0.7718 |
| Pegcetacoplan | Absolute Levels and Change From Baseline in C3 | Week 8 Absolute Level | 4.238 g/L | Standard Deviation 0.4511 |
| Pegcetacoplan | Absolute Levels and Change From Baseline in C3 | Week 8 Change from Baseline | 3.040 g/L | Standard Deviation 0.324 |
| Pegcetacoplan | Absolute Levels and Change From Baseline in C3 | Week 10 Absolute Level | 3.920 g/L | Standard Deviation 1.0524 |
| Pegcetacoplan | Absolute Levels and Change From Baseline in C3 | Week 10 Change from Baseline | 2.814 g/L | Standard Deviation 0.9156 |
| Pegcetacoplan | Absolute Levels and Change From Baseline in C3 | Week 12 Absolute Level | 4.061 g/L | Standard Deviation 1.7835 |
| Pegcetacoplan | Absolute Levels and Change From Baseline in C3 | Week 12 Change from Baseline | 2.893 g/L | Standard Deviation 1.7103 |
| Pegcetacoplan | Absolute Levels and Change From Baseline in C3 | Week 14 Absolute Level | 4.156 g/L | Standard Deviation 1.1392 |
| Pegcetacoplan | Absolute Levels and Change From Baseline in C3 | Week 14 Change from Baseline | 2.957 g/L | Standard Deviation 0.9774 |
| Pegcetacoplan | Absolute Levels and Change From Baseline in C3 | Week 16 Absolute Level | 4.320 g/L | Standard Deviation 1.2352 |
| Pegcetacoplan | Absolute Levels and Change From Baseline in C3 | Week 16 Change from Baseline | 3.177 g/L | Standard Deviation 1.2536 |
| Pegcetacoplan | Absolute Levels and Change From Baseline in C3 | Week 20 Absolute Level | 2.052 g/L | Standard Deviation 0.9091 |
| Pegcetacoplan | Absolute Levels and Change From Baseline in C3 | Week 20 Change from Baseline | 0.858 g/L | Standard Deviation 0.9097 |
| Pegcetacoplan | Absolute Levels and Change From Baseline in C3 | Week 24 Absolute Level | 1.338 g/L | Standard Deviation 0.507 |
| Pegcetacoplan | Absolute Levels and Change From Baseline in C3 | Week 24 Change from Baseline | 0.047 g/L | Standard Deviation 0.358 |
Secondary
Absolute Levels and Change From Baseline in C3a
Absolute levels and change from baseline to Week 24 in biomarker of complement activation C3a
Time frame: Week 24
Population: The PD set includes all patients in the ITT set who receive IMP and have at least 1 evaluable post-dose PD measurement. The number of patients in the analysis population decreased over the course of the study reflecting the limited number of samples available due to study discontinuation of patients. Patients with an available measure both at the analyzed time point and at baseline are included. In some cases, there were also missing samples.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Pegcetacoplan | Absolute Levels and Change From Baseline in C3a | Baseline Absolute Level | 156.9 µg/L | Standard Deviation 113.09 |
| Pegcetacoplan | Absolute Levels and Change From Baseline in C3a | Week 1 Absolute Level | 67.6 µg/L | Standard Deviation 70.18 |
| Pegcetacoplan | Absolute Levels and Change From Baseline in C3a | Week 1 Change from Baseline | -93.4 µg/L | Standard Deviation 120.73 |
| Pegcetacoplan | Absolute Levels and Change From Baseline in C3a | Week 2 Absolute Level | 126.1 µg/L | Standard Deviation 143.32 |
| Pegcetacoplan | Absolute Levels and Change From Baseline in C3a | Week 2 Change from Baseline | -27.7 µg/L | Standard Deviation 201.41 |
| Pegcetacoplan | Absolute Levels and Change From Baseline in C3a | Week 3 Absolute Level | 69.3 µg/L | Standard Deviation 75.08 |
| Pegcetacoplan | Absolute Levels and Change From Baseline in C3a | Week 3 Change from Baseline | -84.4 µg/L | Standard Deviation 148.81 |
| Pegcetacoplan | Absolute Levels and Change From Baseline in C3a | Week 4 Absolute Level | 153.6 µg/L | Standard Deviation 184.8 |
| Pegcetacoplan | Absolute Levels and Change From Baseline in C3a | Week 4 Change from Baseline | -11.9 µg/L | Standard Deviation 209.85 |
| Pegcetacoplan | Absolute Levels and Change From Baseline in C3a | Week 6 Absolute Level | 54.3 µg/L | Standard Deviation 31.97 |
| Pegcetacoplan | Absolute Levels and Change From Baseline in C3a | Week 6 Change from Baseline | -74.7 µg/L | Standard Deviation 61.34 |
| Pegcetacoplan | Absolute Levels and Change From Baseline in C3a | Week 8 Absolute Level | 73.0 µg/L | Standard Deviation 51.4 |
| Pegcetacoplan | Absolute Levels and Change From Baseline in C3a | Week 8 Change from Baseline | -57.5 µg/L | Standard Deviation 43.88 |
| Pegcetacoplan | Absolute Levels and Change From Baseline in C3a | Week 10 Absolute Level | 82.7 µg/L | Standard Deviation 51.19 |
| Pegcetacoplan | Absolute Levels and Change From Baseline in C3a | Week 10 Change from Baseline | -46.3 µg/L | Standard Deviation 44.74 |
| Pegcetacoplan | Absolute Levels and Change From Baseline in C3a | Week 12 Absolute Level | 218.4 µg/L | Standard Deviation 211.57 |
| Pegcetacoplan | Absolute Levels and Change From Baseline in C3a | Week 12 Change from Baseline | 77.8 µg/L | Standard Deviation 207.74 |
| Pegcetacoplan | Absolute Levels and Change From Baseline in C3a | Week 14 Absolute Level | 141.0 µg/L | Standard Deviation 173.84 |
| Pegcetacoplan | Absolute Levels and Change From Baseline in C3a | Week 14 Change from Baseline | -3.2 µg/L | Standard Deviation 198.3 |
| Pegcetacoplan | Absolute Levels and Change From Baseline in C3a | Week 16 Absolute Level | 150.8 µg/L | Standard Deviation 169.23 |
| Pegcetacoplan | Absolute Levels and Change From Baseline in C3a | Week 16 Change from Baseline | 6.6 µg/L | Standard Deviation 195.38 |
| Pegcetacoplan | Absolute Levels and Change From Baseline in C3a | Week 20 Absolute Level | 189.8 µg/L | Standard Deviation 148.22 |
| Pegcetacoplan | Absolute Levels and Change From Baseline in C3a | Week 20 Change from Baseline | 75.6 µg/L | Standard Deviation 157.49 |
| Pegcetacoplan | Absolute Levels and Change From Baseline in C3a | Week 24 Absolute Level | 102.4 µg/L | Standard Deviation 75.03 |
| Pegcetacoplan | Absolute Levels and Change From Baseline in C3a | Week 24 Change from Baseline | -11.5 µg/L | Standard Deviation 91.55 |
Secondary
Absolute Levels and Change From Baseline in C4a
Absolute levels and change from baseline to Week 24 in biomarker of complement activation C4a
Time frame: Week 24
Population: The PD set includes all patients in the ITT set who receive IMP and have at least 1 evaluable post-dose PD measurement. The number of patients in the analysis population decreased over the course of the study reflecting the limited number of samples available due to study discontinuation of patients. Patients with an available measure both at the analyzed time point and at baseline are included. In some cases, there were also missing samples.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Pegcetacoplan | Absolute Levels and Change From Baseline in C4a | Baseline Absolute Level | 3422.9 ug/L | Standard Deviation 2792.23 |
| Pegcetacoplan | Absolute Levels and Change From Baseline in C4a | Week 1 Absolute Level | 1724.6 ug/L | Standard Deviation 1219.46 |
| Pegcetacoplan | Absolute Levels and Change From Baseline in C4a | Week 1 Change from Baseline | -1470.8 ug/L | Standard Deviation 3194.16 |
| Pegcetacoplan | Absolute Levels and Change From Baseline in C4a | Week 2 Absolute Level | 4644.1 ug/L | Standard Deviation 4426.51 |
| Pegcetacoplan | Absolute Levels and Change From Baseline in C4a | Week 2 Change from Baseline | 1263.3 ug/L | Standard Deviation 3370.74 |
| Pegcetacoplan | Absolute Levels and Change From Baseline in C4a | Week 3 Absolute Level | 4118.0 ug/L | Standard Deviation 4192.86 |
| Pegcetacoplan | Absolute Levels and Change From Baseline in C4a | Week 3 Change from Baseline | 737.2 ug/L | Standard Deviation 3316.77 |
| Pegcetacoplan | Absolute Levels and Change From Baseline in C4a | Week 4 Absolute Level | 4504.8 ug/L | Standard Deviation 3690.83 |
| Pegcetacoplan | Absolute Levels and Change From Baseline in C4a | Week 4 Change from Baseline | 811.8 ug/L | Standard Deviation 1763.02 |
| Pegcetacoplan | Absolute Levels and Change From Baseline in C4a | Week 6 Absolute Level | 3582.6 ug/L | Standard Deviation 3559.59 |
| Pegcetacoplan | Absolute Levels and Change From Baseline in C4a | Week 6 Change from Baseline | -438.7 ug/L | Standard Deviation 5670.06 |
| Pegcetacoplan | Absolute Levels and Change From Baseline in C4a | Week 8 Absolute Level | 2792.5 ug/L | Standard Deviation 3573.91 |
| Pegcetacoplan | Absolute Levels and Change From Baseline in C4a | Week 8 Change from Baseline | -987.2 ug/L | Standard Deviation 1295.21 |
| Pegcetacoplan | Absolute Levels and Change From Baseline in C4a | Week 10 Absolute Level | 2234.7 ug/L | Standard Deviation 2012.23 |
| Pegcetacoplan | Absolute Levels and Change From Baseline in C4a | Week 10 Change from Baseline | -1786.6 ug/L | Standard Deviation 2007.28 |
| Pegcetacoplan | Absolute Levels and Change From Baseline in C4a | Week 12 Absolute Level | 5171.6 ug/L | Standard Deviation 4735.92 |
| Pegcetacoplan | Absolute Levels and Change From Baseline in C4a | Week 12 Change from Baseline | 852.0 ug/L | Standard Deviation 4487.81 |
| Pegcetacoplan | Absolute Levels and Change From Baseline in C4a | Week 14 Absolute Level | 2618.2 ug/L | Standard Deviation 4142.08 |
| Pegcetacoplan | Absolute Levels and Change From Baseline in C4a | Week 14 Change from Baseline | -2269.8 ug/L | Standard Deviation 4901.62 |
| Pegcetacoplan | Absolute Levels and Change From Baseline in C4a | Week 16 Absolute Level | 1731.8 ug/L | Standard Deviation 1285.26 |
| Pegcetacoplan | Absolute Levels and Change From Baseline in C4a | Week 16 Change from Baseline | -3156.2 ug/L | Standard Deviation 3051.14 |
| Pegcetacoplan | Absolute Levels and Change From Baseline in C4a | Week 20 Absolute Level | 4007.8 ug/L | Standard Deviation 3648.52 |
| Pegcetacoplan | Absolute Levels and Change From Baseline in C4a | Week 20 Change from Baseline | -664.6 ug/L | Standard Deviation 3821.6 |
| Pegcetacoplan | Absolute Levels and Change From Baseline in C4a | Week 24 Absolute Level | 4329.2 ug/L | Standard Deviation 2520.61 |
| Pegcetacoplan | Absolute Levels and Change From Baseline in C4a | Week 24 Change from Baseline | -403.5 ug/L | Standard Deviation 2475.95 |
Secondary
Absolute Levels and Change From Baseline in Classical Pathway (CH50)
Absolute levels and change from baseline to Week 24 in biomarker of complement activation CH50
Time frame: Week 24
Population: The PD set includes all patients in the ITT set who receive IMP and have at least 1 evaluable post-dose PD measurement. The number of patients in the analysis population decreased over the course of the study reflecting the limited number of samples available due to study discontinuation of patients. Patients with an available measure both at the analyzed time point and at baseline are included. In some cases, there were also missing samples.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Pegcetacoplan | Absolute Levels and Change From Baseline in Classical Pathway (CH50) | Baseline Absolute Level | 92.2 U/mL | Standard Deviation 17.94 |
| Pegcetacoplan | Absolute Levels and Change From Baseline in Classical Pathway (CH50) | Week 1 Absolute Level | 99.6 U/mL | Standard Deviation 29.99 |
| Pegcetacoplan | Absolute Levels and Change From Baseline in Classical Pathway (CH50) | Week 1 Change from Baseline | 7.2 U/mL | Standard Deviation 28.48 |
| Pegcetacoplan | Absolute Levels and Change From Baseline in Classical Pathway (CH50) | Week 2 Absolute Level | 116.9 U/mL | Standard Deviation 18.98 |
| Pegcetacoplan | Absolute Levels and Change From Baseline in Classical Pathway (CH50) | Week 2 Change from Baseline | 21.9 U/mL | Standard Deviation 25.3 |
| Pegcetacoplan | Absolute Levels and Change From Baseline in Classical Pathway (CH50) | Week 3 Absolute Level | 114.9 U/mL | Standard Deviation 27.09 |
| Pegcetacoplan | Absolute Levels and Change From Baseline in Classical Pathway (CH50) | Week 3 Change from Baseline | 21.0 U/mL | Standard Deviation 35.76 |
| Pegcetacoplan | Absolute Levels and Change From Baseline in Classical Pathway (CH50) | Week 4 Absolute Level | 107.7 U/mL | Standard Deviation 21.72 |
| Pegcetacoplan | Absolute Levels and Change From Baseline in Classical Pathway (CH50) | Week 4 Change from Baseline | 12.1 U/mL | Standard Deviation 13.64 |
| Pegcetacoplan | Absolute Levels and Change From Baseline in Classical Pathway (CH50) | Week 6 Absolute Level | 114.9 U/mL | Standard Deviation 19.36 |
| Pegcetacoplan | Absolute Levels and Change From Baseline in Classical Pathway (CH50) | Week 6 Change from Baseline | 25.1 U/mL | Standard Deviation 30.72 |
| Pegcetacoplan | Absolute Levels and Change From Baseline in Classical Pathway (CH50) | Week 8 Absolute Level | 123.4 U/mL | Standard Deviation 32.4 |
| Pegcetacoplan | Absolute Levels and Change From Baseline in Classical Pathway (CH50) | Week 8 Change from Baseline | 31.8 U/mL | Standard Deviation 35.78 |
| Pegcetacoplan | Absolute Levels and Change From Baseline in Classical Pathway (CH50) | Week 10 Absolute Level | 121.1 U/mL | Standard Deviation 34.98 |
| Pegcetacoplan | Absolute Levels and Change From Baseline in Classical Pathway (CH50) | Week 10 Change from Baseline | 25.5 U/mL | Standard Deviation 29.59 |
| Pegcetacoplan | Absolute Levels and Change From Baseline in Classical Pathway (CH50) | Week 12 Absolute Level | 103.0 U/mL | Standard Deviation 45.15 |
| Pegcetacoplan | Absolute Levels and Change From Baseline in Classical Pathway (CH50) | Week 12 Change from Baseline | 12.7 U/mL | Standard Deviation 42.57 |
| Pegcetacoplan | Absolute Levels and Change From Baseline in Classical Pathway (CH50) | Week 14 Absolute Level | 127.4 U/mL | Standard Deviation 34.81 |
| Pegcetacoplan | Absolute Levels and Change From Baseline in Classical Pathway (CH50) | Week 14 Change from Baseline | 35.2 U/mL | Standard Deviation 44.74 |
| Pegcetacoplan | Absolute Levels and Change From Baseline in Classical Pathway (CH50) | Week 16 Absolute Level | 129.9 U/mL | Standard Deviation 30.42 |
| Pegcetacoplan | Absolute Levels and Change From Baseline in Classical Pathway (CH50) | Week 16 Change from Baseline | 32.7 U/mL | Standard Deviation 37.69 |
| Pegcetacoplan | Absolute Levels and Change From Baseline in Classical Pathway (CH50) | Week 20 Absolute Level | 119.6 U/mL | Standard Deviation 43.93 |
| Pegcetacoplan | Absolute Levels and Change From Baseline in Classical Pathway (CH50) | Week 20 Change from Baseline | 26.3 U/mL | Standard Deviation 46.44 |
| Pegcetacoplan | Absolute Levels and Change From Baseline in Classical Pathway (CH50) | Week 24 Absolute Level | 106.8 U/mL | Standard Deviation 51.1 |
| Pegcetacoplan | Absolute Levels and Change From Baseline in Classical Pathway (CH50) | Week 24 Change from Baseline | 17.0 U/mL | Standard Deviation 73.63 |
Secondary
Absolute Levels and Change From Baseline in sC5b-9
Absolute levels and change from baseline to Week 24 in biomarker of complement activation sC5b-9
Time frame: Week 24
Population: The pharmacodynamic (PD) set includes patients in the intent-to-treat (ITT) set who receive investigational medicinal product (IMP) and have at least 1 evaluable post-dose PD measurement. The number of patients in the analysis population decreased over the course of the study reflecting the limited number of samples available due to study discontinuation of patients. Patients with an available measure both at the analyzed time point and at baseline are included. There were also missing samples.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Pegcetacoplan | Absolute Levels and Change From Baseline in sC5b-9 | Week 8 Change from Baseline | -168.3 µg/L | Standard Deviation 226.01 |
| Pegcetacoplan | Absolute Levels and Change From Baseline in sC5b-9 | Baseline Absolute Level | 309.2 µg/L | Standard Deviation 200.72 |
| Pegcetacoplan | Absolute Levels and Change From Baseline in sC5b-9 | Week 1 Absolute Level | 175.3 µg/L | Standard Deviation 81.26 |
| Pegcetacoplan | Absolute Levels and Change From Baseline in sC5b-9 | Week 1 Change from Baseline | -133.6 µg/L | Standard Deviation 176.13 |
| Pegcetacoplan | Absolute Levels and Change From Baseline in sC5b-9 | Week 2 Absolute Level | 152.0 µg/L | Standard Deviation 73.95 |
| Pegcetacoplan | Absolute Levels and Change From Baseline in sC5b-9 | Week 2 Change from Baseline | -163.0 µg/L | Standard Deviation 157.36 |
| Pegcetacoplan | Absolute Levels and Change From Baseline in sC5b-9 | Week 3 Absolute Level | 129.8 µg/L | Standard Deviation 43.38 |
| Pegcetacoplan | Absolute Levels and Change From Baseline in sC5b-9 | Week 10 Absolute Level | 131.4 µg/L | Standard Deviation 70.6 |
| Pegcetacoplan | Absolute Levels and Change From Baseline in sC5b-9 | Week 3 Change from Baseline | -185.2 µg/L | Standard Deviation 194.18 |
| Pegcetacoplan | Absolute Levels and Change From Baseline in sC5b-9 | Week 4 Absolute Level | 161.6 µg/L | Standard Deviation 78.37 |
| Pegcetacoplan | Absolute Levels and Change From Baseline in sC5b-9 | Week 4 Change from Baseline | -142.1 µg/L | Standard Deviation 160.54 |
| Pegcetacoplan | Absolute Levels and Change From Baseline in sC5b-9 | Week 6 Absolute Level | 111.7 µg/L | Standard Deviation 37.98 |
| Pegcetacoplan | Absolute Levels and Change From Baseline in sC5b-9 | Week 6 Change from Baseline | -204.0 µg/L | Standard Deviation 217.36 |
| Pegcetacoplan | Absolute Levels and Change From Baseline in sC5b-9 | Week 8 Absolute Level | 148.0 µg/L | Standard Deviation 45.69 |
| Pegcetacoplan | Absolute Levels and Change From Baseline in sC5b-9 | Week 10 Change from Baseline | -184.3 µg/L | Standard Deviation 179.44 |
| Pegcetacoplan | Absolute Levels and Change From Baseline in sC5b-9 | Week 12 Absolute Level | 141.2 µg/L | Standard Deviation 77.97 |
| Pegcetacoplan | Absolute Levels and Change From Baseline in sC5b-9 | Week 12 Change from Baseline | -214.0 µg/L | Standard Deviation 224.06 |
| Pegcetacoplan | Absolute Levels and Change From Baseline in sC5b-9 | Week 14 Absolute Level | 156.8 µg/L | Standard Deviation 65.64 |
| Pegcetacoplan | Absolute Levels and Change From Baseline in sC5b-9 | Week 14 Change from Baseline | -211.8 µg/L | Standard Deviation 211.05 |
| Pegcetacoplan | Absolute Levels and Change From Baseline in sC5b-9 | Week 16 Absolute Level | 158.0 µg/L | Standard Deviation 54.21 |
| Pegcetacoplan | Absolute Levels and Change From Baseline in sC5b-9 | Week 16 Change from Baseline | -210.6 µg/L | Standard Deviation 240.97 |
| Pegcetacoplan | Absolute Levels and Change From Baseline in sC5b-9 | Week 20 Absolute Level | 190.0 µg/L | Standard Deviation 76.28 |
| Pegcetacoplan | Absolute Levels and Change From Baseline in sC5b-9 | Week 20 Change from Baseline | -171.6 µg/L | Standard Deviation 232 |
| Pegcetacoplan | Absolute Levels and Change From Baseline in sC5b-9 | Week 24 Absolute Level | 256.0 µg/L | Standard Deviation 122.45 |
| Pegcetacoplan | Absolute Levels and Change From Baseline in sC5b-9 | Week 24 Change from Baseline | -167.8 µg/L | Standard Deviation 162.09 |
Secondary
Duration of Clinical Response
Duration of clinical response sustained at week 24
Time frame: From the first observed clinical response until the response criteria is no longer fulfilled or until end of study, up to 24 weeks
Population: Analysis population based on patients who achieved clinical response at any point during the study.
| Arm | Measure | Value (MEDIAN) |
|---|---|---|
| Pegcetacoplan | Duration of Clinical Response | 1.6 Weeks |
Secondary
Duration of TMA Response
Duration of TMA response sustained at week 24
Time frame: From the first observed TMA response until the response criteria is no longer fulfilled or until end of study, up to 24 weeks
Population: Analysis population based on patients who achieved TMA response at any point during the study.
| Arm | Measure | Value (MEDIAN) |
|---|---|---|
| Pegcetacoplan | Duration of TMA Response | 4.0 Weeks |
Secondary
Number of Participants Reaching Clinical Response at Week 12
Number of participants reaching clinical response at week 12
Time frame: Week 12
| Arm | Measure | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|
| Pegcetacoplan | Number of Participants Reaching Clinical Response at Week 12 | 1 Participants |
Secondary
Number of Participants Reaching Clinical Response at Week 24
A participant will be declared as reaching a clinical response upon improvement in laboratory markers of TMA and resolution of TMA clinical symptoms
Time frame: Week 24
| Arm | Measure | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|
| Pegcetacoplan | Number of Participants Reaching Clinical Response at Week 24 | 0 Participants |
Secondary
Number of Participants Reaching TMA Response at Week 12
Number of participants reaching TMA response at week 12
Time frame: Week 12
| Arm | Measure | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|
| Pegcetacoplan | Number of Participants Reaching TMA Response at Week 12 | 2 Participants |
Secondary
Number of Participants Reaching TMA Response at Week 24
A participant will be declared as reaching TMA response upon improvement in laboratory markers of TMA
Time frame: Week 24
| Arm | Measure | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|
| Pegcetacoplan | Number of Participants Reaching TMA Response at Week 24 | 2 Participants |
Secondary
Overall Survival at Day 100
Survival
Time frame: Day 100 from diagnosis
| Arm | Measure | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|
| Pegcetacoplan | Overall Survival at Day 100 | 8 Participants |
Secondary
Overall Survival at Week 24
Survival
Time frame: Week 24 from treatment start
| Arm | Measure | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|
| Pegcetacoplan | Overall Survival at Week 24 | 6 Participants |
Secondary
TA-TMA Relapse at Week 24
A participant will be declared as relapsing upon appearance of laboratory markers of TMA
Time frame: Week 24
Population: Only 2 patients had available assessment at the visit.
| Arm | Measure | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|
| Pegcetacoplan | TA-TMA Relapse at Week 24 | 0 Participants |
Secondary
Time to Clinical Response
Both clinical response sustained at week 24 and clinical response at any time during the study will be assessed.
Time frame: From treatment start to first documentation of attainment of a clinical response, up to 24 weeks
Population: Analysis population based on patients who achieved clinical response at any point during the study.
| Arm | Measure | Value (MEDIAN) |
|---|---|---|
| Pegcetacoplan | Time to Clinical Response | NA Weeks |
Secondary
Time to TMA Response
Both TMA response sustained at week 24 and TMA response at any time during the study will be assessed.
Time frame: From treatment start to first documentation of attainment of a TMA response, up to 24 weeks
Population: Analysis population based on patients who achieved TMA response at any point during the study.
| Arm | Measure | Value (MEDIAN) |
|---|---|---|
| Pegcetacoplan | Time to TMA Response | 24.0 Weeks |
Other Pre-specified
Change From Baseline in Diastolic Blood Pressure
Change from baseline to Week 24 in diastolic blood pressure. Post-dose is 30 minutes after stop of infusion. The timepoints from Week 16 onward have a single value as no infusions were administered at these study visits.
Time frame: Week 24
Population: The number of patients in the analysis population reflects the limited number of samples available due to study discontinuation of patients. Patients with an available measure are included.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Pegcetacoplan | Change From Baseline in Diastolic Blood Pressure | Week 1 Pre-dose | -4.5 mmHg | Standard Deviation 9.21 |
| Pegcetacoplan | Change From Baseline in Diastolic Blood Pressure | Week 1 Post-dose | -5.1 mmHg | Standard Deviation 7.41 |
| Pegcetacoplan | Change From Baseline in Diastolic Blood Pressure | Week 2 Pre-dose | -2.2 mmHg | Standard Deviation 10.28 |
| Pegcetacoplan | Change From Baseline in Diastolic Blood Pressure | Week 2 Post-dose | -6.0 mmHg | Standard Deviation 11.58 |
| Pegcetacoplan | Change From Baseline in Diastolic Blood Pressure | Week 3 Pre-dose | -2.1 mmHg | Standard Deviation 11.5 |
| Pegcetacoplan | Change From Baseline in Diastolic Blood Pressure | Week 3 Post-dose | -9.2 mmHg | Standard Deviation 3.43 |
| Pegcetacoplan | Change From Baseline in Diastolic Blood Pressure | Week 4 Pre-dose | -7.6 mmHg | Standard Deviation 8.88 |
| Pegcetacoplan | Change From Baseline in Diastolic Blood Pressure | Week 4 Post-dose | -7.3 mmHg | Standard Deviation 6.63 |
| Pegcetacoplan | Change From Baseline in Diastolic Blood Pressure | Week 6 Pre-dose | -7.0 mmHg | Standard Deviation 9.42 |
| Pegcetacoplan | Change From Baseline in Diastolic Blood Pressure | Week 6 Post-dose | -12.8 mmHg | Standard Deviation 7.5 |
| Pegcetacoplan | Change From Baseline in Diastolic Blood Pressure | Week 8 Pre-dose | -7.9 mmHg | Standard Deviation 8.98 |
| Pegcetacoplan | Change From Baseline in Diastolic Blood Pressure | Week 8 Post-dose | -11.3 mmHg | Standard Deviation 9.46 |
| Pegcetacoplan | Change From Baseline in Diastolic Blood Pressure | Week 10 Pre-dose | -7.1 mmHg | Standard Deviation 8.88 |
| Pegcetacoplan | Change From Baseline in Diastolic Blood Pressure | Week 10 Post-dose | -7.2 mmHg | Standard Deviation 13.2 |
| Pegcetacoplan | Change From Baseline in Diastolic Blood Pressure | Week 12 Pre-dose | -8.6 mmHg | Standard Deviation 10.86 |
| Pegcetacoplan | Change From Baseline in Diastolic Blood Pressure | Week 12 Post-dose | -10.8 mmHg | Standard Deviation 8.58 |
| Pegcetacoplan | Change From Baseline in Diastolic Blood Pressure | Week 14 Pre-dose | -5.1 mmHg | Standard Deviation 9.04 |
| Pegcetacoplan | Change From Baseline in Diastolic Blood Pressure | Week 14 Post-dose | -7.7 mmHg | Standard Deviation 16.77 |
| Pegcetacoplan | Change From Baseline in Diastolic Blood Pressure | Week 16 | -8.3 mmHg | Standard Deviation 8.77 |
| Pegcetacoplan | Change From Baseline in Diastolic Blood Pressure | Week 20 | -10.6 mmHg | Standard Deviation 7.28 |
| Pegcetacoplan | Change From Baseline in Diastolic Blood Pressure | Week 24 | -13.2 mmHg | Standard Deviation 9.09 |
Other Pre-specified
Change From Baseline in Haptoglobin
Change from baseline to Week 24 in haptoglobin
Time frame: Week 24
Population: The number of patients in the analysis population reflects the limited number of samples available due to study discontinuation of patients. Patients with an available measure are included.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Pegcetacoplan | Change From Baseline in Haptoglobin | Week 1 | 0.070 g/L | Standard Deviation 0.228 |
| Pegcetacoplan | Change From Baseline in Haptoglobin | Week 2 | 0.244 g/L | Standard Deviation 0.4553 |
| Pegcetacoplan | Change From Baseline in Haptoglobin | Week 3 | 0.179 g/L | Standard Deviation 0.5054 |
| Pegcetacoplan | Change From Baseline in Haptoglobin | Week 4 | 0.260 g/L | Standard Deviation 0.5674 |
| Pegcetacoplan | Change From Baseline in Haptoglobin | Week 6 | 0.590 g/L | Standard Deviation 0.6451 |
| Pegcetacoplan | Change From Baseline in Haptoglobin | Week 8 | 0.388 g/L | Standard Deviation 0.4496 |
| Pegcetacoplan | Change From Baseline in Haptoglobin | Week 10 | 0.578 g/L | Standard Deviation 0.5832 |
| Pegcetacoplan | Change From Baseline in Haptoglobin | Week 12 | 0.625 g/L | Standard Deviation 0.6279 |
| Pegcetacoplan | Change From Baseline in Haptoglobin | Week 14 | 0.620 g/L | Standard Deviation 0.7013 |
| Pegcetacoplan | Change From Baseline in Haptoglobin | Week 16 | 0.657 g/L | Standard Deviation 0.6629 |
| Pegcetacoplan | Change From Baseline in Haptoglobin | Week 20 | 0.686 g/L | Standard Deviation 0.7459 |
| Pegcetacoplan | Change From Baseline in Haptoglobin | Week 24 | 0.778 g/L | Standard Deviation 0.5427 |
Other Pre-specified
Change From Baseline in Heart Rate
Change from baseline to Week 24 in heart rate. Post-dose is 30 minutes after stop of infusion. The timepoints from Week 16 onward have a single value as no infusions were administered at these study visits.
Time frame: Week 24
Population: The number of patients in the analysis population reflects the limited number of samples available due to study discontinuation of patients. Patients with an available measure are included.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Pegcetacoplan | Change From Baseline in Heart Rate | Week 1 Pre-dose | -7.4 bpm | Standard Deviation 9.91 |
| Pegcetacoplan | Change From Baseline in Heart Rate | Week 1 Post-dose | -7.3 bpm | Standard Deviation 8.6 |
| Pegcetacoplan | Change From Baseline in Heart Rate | Week 2 Pre-dose | -4.3 bpm | Standard Deviation 9.2 |
| Pegcetacoplan | Change From Baseline in Heart Rate | Week 2 Post-dose | -3.6 bpm | Standard Deviation 11.93 |
| Pegcetacoplan | Change From Baseline in Heart Rate | Week 3 Pre-dose | -7.8 bpm | Standard Deviation 8.73 |
| Pegcetacoplan | Change From Baseline in Heart Rate | Week 3 Post-dose | -5.5 bpm | Standard Deviation 6.06 |
| Pegcetacoplan | Change From Baseline in Heart Rate | Week 4 Pre-dose | -7.3 bpm | Standard Deviation 7.76 |
| Pegcetacoplan | Change From Baseline in Heart Rate | Week 4 Post-dose | -8.3 bpm | Standard Deviation 11.28 |
| Pegcetacoplan | Change From Baseline in Heart Rate | Week 6 Pre-dose | -6.4 bpm | Standard Deviation 3.78 |
| Pegcetacoplan | Change From Baseline in Heart Rate | Week 6 Post-dose | -8.5 bpm | Standard Deviation 4.51 |
| Pegcetacoplan | Change From Baseline in Heart Rate | Week 8 Pre-dose | -9.2 bpm | Standard Deviation 6.44 |
| Pegcetacoplan | Change From Baseline in Heart Rate | Week 8 Post-dose | -9.3 bpm | Standard Deviation 2.25 |
| Pegcetacoplan | Change From Baseline in Heart Rate | Week 10 Pre-dose | -6.3 bpm | Standard Deviation 6.22 |
| Pegcetacoplan | Change From Baseline in Heart Rate | Week 10 Post-dose | -5.2 bpm | Standard Deviation 9.96 |
| Pegcetacoplan | Change From Baseline in Heart Rate | Week 12 Pre-dose | -5.8 bpm | Standard Deviation 8 |
| Pegcetacoplan | Change From Baseline in Heart Rate | Week 12 Post-dose | -11.2 bpm | Standard Deviation 2.68 |
| Pegcetacoplan | Change From Baseline in Heart Rate | Week 14 Pre-dose | -5.1 bpm | Standard Deviation 12.33 |
| Pegcetacoplan | Change From Baseline in Heart Rate | Week 14 Post-dose | 1.7 bpm | Standard Deviation 7.51 |
| Pegcetacoplan | Change From Baseline in Heart Rate | Week 16 | -12.3 bpm | Standard Deviation 10.03 |
| Pegcetacoplan | Change From Baseline in Heart Rate | Week 20 | -8.6 bpm | Standard Deviation 9.57 |
| Pegcetacoplan | Change From Baseline in Heart Rate | Week 24 | -6.3 bpm | Standard Deviation 9.97 |
Other Pre-specified
Change From Baseline in Hemoglobin
Change from baseline to Week 24 in hemoglobin
Time frame: Week 24
Population: The number of patients in the analysis population reflects the limited number of samples available due to study discontinuation of patients. Patients with an available measure are included.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Pegcetacoplan | Change From Baseline in Hemoglobin | Week 1 | -3.8 g/L | Standard Deviation 14.3 |
| Pegcetacoplan | Change From Baseline in Hemoglobin | Week 2 | 8.1 g/L | Standard Deviation 12.48 |
| Pegcetacoplan | Change From Baseline in Hemoglobin | Week 3 | 3.6 g/L | Standard Deviation 14.89 |
| Pegcetacoplan | Change From Baseline in Hemoglobin | Week 4 | 8.0 g/L | Standard Deviation 21.85 |
| Pegcetacoplan | Change From Baseline in Hemoglobin | Week 6 | 2.0 g/L | Standard Deviation 13.2 |
| Pegcetacoplan | Change From Baseline in Hemoglobin | Week 8 | 9.4 g/L | Standard Deviation 15.14 |
| Pegcetacoplan | Change From Baseline in Hemoglobin | Week 10 | 13.0 g/L | Standard Deviation 15.08 |
| Pegcetacoplan | Change From Baseline in Hemoglobin | Week 12 | 10.6 g/L | Standard Deviation 16.99 |
| Pegcetacoplan | Change From Baseline in Hemoglobin | Week 14 | 5.7 g/L | Standard Deviation 19.77 |
| Pegcetacoplan | Change From Baseline in Hemoglobin | Week 16 | 7.2 g/L | Standard Deviation 17.64 |
| Pegcetacoplan | Change From Baseline in Hemoglobin | Week 20 | 13.4 g/L | Standard Deviation 11.04 |
| Pegcetacoplan | Change From Baseline in Hemoglobin | Week 24 | 14.0 g/L | Standard Deviation 11.02 |
Other Pre-specified
Change From Baseline in Indirect Bilirubin
Change from baseline to Week 24 in indirect bilirubin
Time frame: Week 24
Population: The number of patients in the analysis population reflects the limited number of samples available due to study discontinuation of patients. Patients with an available measure are included.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Pegcetacoplan | Change From Baseline in Indirect Bilirubin | Week 1 | -7.80 umol/L | Standard Deviation 14.213 |
| Pegcetacoplan | Change From Baseline in Indirect Bilirubin | Week 2 | -9.73 umol/L | Standard Deviation 12.344 |
| Pegcetacoplan | Change From Baseline in Indirect Bilirubin | Week 3 | -9.71 umol/L | Standard Deviation 14.683 |
| Pegcetacoplan | Change From Baseline in Indirect Bilirubin | Week 4 | -9.87 umol/L | Standard Deviation 15.239 |
| Pegcetacoplan | Change From Baseline in Indirect Bilirubin | Week 6 | -8.95 umol/L | Standard Deviation 18.202 |
| Pegcetacoplan | Change From Baseline in Indirect Bilirubin | Week 8 | -11.98 umol/L | Standard Deviation 21.787 |
| Pegcetacoplan | Change From Baseline in Indirect Bilirubin | Week 10 | -10.24 umol/L | Standard Deviation 18.39 |
| Pegcetacoplan | Change From Baseline in Indirect Bilirubin | Week 12 | -10.71 umol/L | Standard Deviation 23.203 |
| Pegcetacoplan | Change From Baseline in Indirect Bilirubin | Week 14 | -14.03 umol/L | Standard Deviation 26.164 |
| Pegcetacoplan | Change From Baseline in Indirect Bilirubin | Week 16 | -13.70 umol/L | Standard Deviation 27.536 |
| Pegcetacoplan | Change From Baseline in Indirect Bilirubin | Week 20 | -3.23 umol/L | Standard Deviation 9.33 |
| Pegcetacoplan | Change From Baseline in Indirect Bilirubin | Week 24 | -3.65 umol/L | Standard Deviation 7.769 |
Other Pre-specified
Change From Baseline in Lactate Dehydrogenase (LDH)
Change from baseline to Week 24 in LDH
Time frame: Week 24
Population: The number of patients in the analysis population reflects the limited number of samples available due to study discontinuation of patients. Patients with an available measure are included.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Pegcetacoplan | Change From Baseline in Lactate Dehydrogenase (LDH) | Week 1 | -273.9 U/L | Standard Deviation 310.05 |
| Pegcetacoplan | Change From Baseline in Lactate Dehydrogenase (LDH) | Week 2 | -315.9 U/L | Standard Deviation 328.13 |
| Pegcetacoplan | Change From Baseline in Lactate Dehydrogenase (LDH) | Week 3 | -348.2 U/L | Standard Deviation 346.46 |
| Pegcetacoplan | Change From Baseline in Lactate Dehydrogenase (LDH) | Week 4 | -365.1 U/L | Standard Deviation 363.98 |
| Pegcetacoplan | Change From Baseline in Lactate Dehydrogenase (LDH) | Week 6 | -408.0 U/L | Standard Deviation 408.28 |
| Pegcetacoplan | Change From Baseline in Lactate Dehydrogenase (LDH) | Week 8 | -406.0 U/L | Standard Deviation 427.49 |
| Pegcetacoplan | Change From Baseline in Lactate Dehydrogenase (LDH) | Week 10 | -428.8 U/L | Standard Deviation 425.67 |
| Pegcetacoplan | Change From Baseline in Lactate Dehydrogenase (LDH) | Week 12 | -480.9 U/L | Standard Deviation 405.14 |
| Pegcetacoplan | Change From Baseline in Lactate Dehydrogenase (LDH) | Week 14 | -336.3 U/L | Standard Deviation 406.86 |
| Pegcetacoplan | Change From Baseline in Lactate Dehydrogenase (LDH) | Week 16 | -355.0 U/L | Standard Deviation 396.77 |
| Pegcetacoplan | Change From Baseline in Lactate Dehydrogenase (LDH) | Week 20 | -242.1 U/L | Standard Deviation 194.73 |
| Pegcetacoplan | Change From Baseline in Lactate Dehydrogenase (LDH) | Week 24 | -198.0 U/L | Standard Deviation 235.45 |
Other Pre-specified
Change From Baseline in Platelets
Change from baseline to Week 24 in platelets
Time frame: Week 24
Population: The number of patients in the analysis population reflects the limited number of samples available due to study discontinuation of patients. Patients with an available measure are included.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Pegcetacoplan | Change From Baseline in Platelets | Week 1 | 12.4 10^9 platelets/L | Standard Deviation 24.22 |
| Pegcetacoplan | Change From Baseline in Platelets | Week 2 | 2.8 10^9 platelets/L | Standard Deviation 12.5 |
| Pegcetacoplan | Change From Baseline in Platelets | Week 3 | 5.1 10^9 platelets/L | Standard Deviation 11.2 |
| Pegcetacoplan | Change From Baseline in Platelets | Week 4 | 18.1 10^9 platelets/L | Standard Deviation 28.61 |
| Pegcetacoplan | Change From Baseline in Platelets | Week 6 | 14.8 10^9 platelets/L | Standard Deviation 21.66 |
| Pegcetacoplan | Change From Baseline in Platelets | Week 8 | 19.6 10^9 platelets/L | Standard Deviation 16.2 |
| Pegcetacoplan | Change From Baseline in Platelets | Week 10 | 17.6 10^9 platelets/L | Standard Deviation 23.01 |
| Pegcetacoplan | Change From Baseline in Platelets | Week 12 | 13.4 10^9 platelets/L | Standard Deviation 11.4 |
| Pegcetacoplan | Change From Baseline in Platelets | Week 14 | 12.3 10^9 platelets/L | Standard Deviation 13.72 |
| Pegcetacoplan | Change From Baseline in Platelets | Week 16 | 10.6 10^9 platelets/L | Standard Deviation 13.79 |
| Pegcetacoplan | Change From Baseline in Platelets | Week 20 | 14.9 10^9 platelets/L | Standard Deviation 32.23 |
| Pegcetacoplan | Change From Baseline in Platelets | Week 24 | 22.6 10^9 platelets/L | Standard Deviation 19.88 |
Other Pre-specified
Change From Baseline in Respiratory Rate
Change from baseline to Week 24 in respiratory rate. Post-dose is 30 minutes after stop of infusion. The timepoints from Week 16 onward have a single value as no infusions were administered at these study visits.
Time frame: Week 24
Population: The number of patients in the analysis population reflects the limited number of samples available due to study discontinuation of patients. Patients with an available measure are included.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Pegcetacoplan | Change From Baseline in Respiratory Rate | Week 10 Pre-dose | 0.0 breaths/min | Standard Deviation 1.41 |
| Pegcetacoplan | Change From Baseline in Respiratory Rate | Week 1 Pre-dose | -0.5 breaths/min | Standard Deviation 1.69 |
| Pegcetacoplan | Change From Baseline in Respiratory Rate | Week 1 Post-dose | -0.1 breaths/min | Standard Deviation 0.93 |
| Pegcetacoplan | Change From Baseline in Respiratory Rate | Week 2 Pre-dose | -0.8 breaths/min | Standard Deviation 1.69 |
| Pegcetacoplan | Change From Baseline in Respiratory Rate | Week 2 Post-dose | -0.3 breaths/min | Standard Deviation 0.71 |
| Pegcetacoplan | Change From Baseline in Respiratory Rate | Week 3 Pre-dose | 0.0 breaths/min | Standard Deviation 1.15 |
| Pegcetacoplan | Change From Baseline in Respiratory Rate | Week 3 Post-dose | 0.5 breaths/min | Standard Deviation 0.55 |
| Pegcetacoplan | Change From Baseline in Respiratory Rate | Week 4 Pre-dose | 1.0 breaths/min | Standard Deviation 2.45 |
| Pegcetacoplan | Change From Baseline in Respiratory Rate | Week 4 Post-dose | 1.1 breaths/min | Standard Deviation 2.04 |
| Pegcetacoplan | Change From Baseline in Respiratory Rate | Week 6 Pre-dose | 0.4 breaths/min | Standard Deviation 1.67 |
| Pegcetacoplan | Change From Baseline in Respiratory Rate | Week 6 Post-dose | 0.5 breaths/min | Standard Deviation 1 |
| Pegcetacoplan | Change From Baseline in Respiratory Rate | Week 8 Pre-dose | 0.3 breaths/min | Standard Deviation 0.87 |
| Pegcetacoplan | Change From Baseline in Respiratory Rate | Week 8 Post-dose | -0.2 breaths/min | Standard Deviation 1.47 |
| Pegcetacoplan | Change From Baseline in Respiratory Rate | Week 10 Post-dose | 0.0 breaths/min | Standard Deviation 1.58 |
| Pegcetacoplan | Change From Baseline in Respiratory Rate | Week 12 Pre-dose | 1.4 breaths/min | Standard Deviation 2.62 |
| Pegcetacoplan | Change From Baseline in Respiratory Rate | Week 12 Post-dose | 0.6 breaths/min | Standard Deviation 0.55 |
| Pegcetacoplan | Change From Baseline in Respiratory Rate | Week 14 Pre-dose | 0.4 breaths/min | Standard Deviation 1.27 |
| Pegcetacoplan | Change From Baseline in Respiratory Rate | Week 14 Post-dose | 0.0 breaths/min | Standard Deviation 1.73 |
| Pegcetacoplan | Change From Baseline in Respiratory Rate | Week 16 | 0.7 breaths/min | Standard Deviation 1.21 |
| Pegcetacoplan | Change From Baseline in Respiratory Rate | Week 20 | 0.3 breaths/min | Standard Deviation 1.25 |
| Pegcetacoplan | Change From Baseline in Respiratory Rate | Week 24 | 0.2 breaths/min | Standard Deviation 1.47 |
Other Pre-specified
Change From Baseline in Serum Creatinine
Change from baseline to Week 24 in serum creatinine
Time frame: Week 24
Population: The number of patients in the analysis population reflects the limited number of samples available due to study discontinuation of patients. Patients with an available measure are included.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Pegcetacoplan | Change From Baseline in Serum Creatinine | Week 1 | 12.14 umol/L | Standard Deviation 42.106 |
| Pegcetacoplan | Change From Baseline in Serum Creatinine | Week 2 | -3.84 umol/L | Standard Deviation 64.131 |
| Pegcetacoplan | Change From Baseline in Serum Creatinine | Week 3 | -14.93 umol/L | Standard Deviation 66.231 |
| Pegcetacoplan | Change From Baseline in Serum Creatinine | Week 4 | -28.32 umol/L | Standard Deviation 101.178 |
| Pegcetacoplan | Change From Baseline in Serum Creatinine | Week 6 | -8.80 umol/L | Standard Deviation 78.912 |
| Pegcetacoplan | Change From Baseline in Serum Creatinine | Week 8 | -6.24 umol/L | Standard Deviation 72.867 |
| Pegcetacoplan | Change From Baseline in Serum Creatinine | Week 10 | -7.99 umol/L | Standard Deviation 86.096 |
| Pegcetacoplan | Change From Baseline in Serum Creatinine | Week 12 | -22.04 umol/L | Standard Deviation 64.786 |
| Pegcetacoplan | Change From Baseline in Serum Creatinine | Week 14 | 16.13 umol/L | Standard Deviation 43.379 |
| Pegcetacoplan | Change From Baseline in Serum Creatinine | Week 16 | 14.13 umol/L | Standard Deviation 39.711 |
| Pegcetacoplan | Change From Baseline in Serum Creatinine | Week 20 | 17.77 umol/L | Standard Deviation 24.444 |
| Pegcetacoplan | Change From Baseline in Serum Creatinine | Week 24 | 11.30 umol/L | Standard Deviation 17.47 |
Other Pre-specified
Change From Baseline in Systolic Blood Pressure
Change from baseline to Week 24 in systolic blood pressure. Post-dose is 30 minutes after stop of infusion. The timepoints from Week 16 onward have a single value as no infusions were administered at these study visits.
Time frame: Week 24
Population: The number of patients in the analysis population reflects the limited number of samples available due to study discontinuation of patients. Patients with an available measure are included.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Pegcetacoplan | Change From Baseline in Systolic Blood Pressure | Week 1 Pre-dose | -5.0 mmHg | Standard Deviation 20.3 |
| Pegcetacoplan | Change From Baseline in Systolic Blood Pressure | Week 1 Post-dose | -8.7 mmHg | Standard Deviation 14.24 |
| Pegcetacoplan | Change From Baseline in Systolic Blood Pressure | Week 2 Pre-dose | 3.9 mmHg | Standard Deviation 17.48 |
| Pegcetacoplan | Change From Baseline in Systolic Blood Pressure | Week 2 Post-dose | -0.4 mmHg | Standard Deviation 17.32 |
| Pegcetacoplan | Change From Baseline in Systolic Blood Pressure | Week 3 Pre-dose | 1.3 mmHg | Standard Deviation 22.46 |
| Pegcetacoplan | Change From Baseline in Systolic Blood Pressure | Week 3 Post-dose | -13.2 mmHg | Standard Deviation 9.97 |
| Pegcetacoplan | Change From Baseline in Systolic Blood Pressure | Week 4 Pre-dose | -7.6 mmHg | Standard Deviation 15.18 |
| Pegcetacoplan | Change From Baseline in Systolic Blood Pressure | Week 4 Post-dose | -12.0 mmHg | Standard Deviation 14.31 |
| Pegcetacoplan | Change From Baseline in Systolic Blood Pressure | Week 6 Pre-dose | -10.7 mmHg | Standard Deviation 16.71 |
| Pegcetacoplan | Change From Baseline in Systolic Blood Pressure | Week 6 Post-dose | -24.3 mmHg | Standard Deviation 8.66 |
| Pegcetacoplan | Change From Baseline in Systolic Blood Pressure | Week 8 Pre-dose | -9.1 mmHg | Standard Deviation 19.46 |
| Pegcetacoplan | Change From Baseline in Systolic Blood Pressure | Week 8 Post-dose | -13.2 mmHg | Standard Deviation 10.48 |
| Pegcetacoplan | Change From Baseline in Systolic Blood Pressure | Week 10 Pre-dose | -7.7 mmHg | Standard Deviation 16.02 |
| Pegcetacoplan | Change From Baseline in Systolic Blood Pressure | Week 10 Post-dose | -8.4 mmHg | Standard Deviation 17.07 |
| Pegcetacoplan | Change From Baseline in Systolic Blood Pressure | Week 12 Pre-dose | -10.6 mmHg | Standard Deviation 21.47 |
| Pegcetacoplan | Change From Baseline in Systolic Blood Pressure | Week 12 Post-dose | -15.0 mmHg | Standard Deviation 10.15 |
| Pegcetacoplan | Change From Baseline in Systolic Blood Pressure | Week 14 Pre-dose | -4.0 mmHg | Standard Deviation 17.67 |
| Pegcetacoplan | Change From Baseline in Systolic Blood Pressure | Week 14 Post-dose | -11.3 mmHg | Standard Deviation 21.96 |
| Pegcetacoplan | Change From Baseline in Systolic Blood Pressure | Week 16 | -13.3 mmHg | Standard Deviation 12.09 |
| Pegcetacoplan | Change From Baseline in Systolic Blood Pressure | Week 20 | -13.7 mmHg | Standard Deviation 12.32 |
| Pegcetacoplan | Change From Baseline in Systolic Blood Pressure | Week 24 | -8.3 mmHg | Standard Deviation 12.24 |
Other Pre-specified
Change From Baseline in Temperature
Change from baseline to Week 24 in temperature. Post-dose is 30 minutes after stop of infusion. The timepoints from Week 16 onward have a single value as no infusions were administered at these study visits.
Time frame: Week 24
Population: The number of patients in the analysis population reflects the limited number of samples available due to study discontinuation of patients. Patients with an available measure are included.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Pegcetacoplan | Change From Baseline in Temperature | Week 8 Pre-dose | -0.09 Celsius | Standard Deviation 0.322 |
| Pegcetacoplan | Change From Baseline in Temperature | Week 1 Pre-dose | 0.10 Celsius | Standard Deviation 0.671 |
| Pegcetacoplan | Change From Baseline in Temperature | Week 1 Post-dose | -0.19 Celsius | Standard Deviation 0.298 |
| Pegcetacoplan | Change From Baseline in Temperature | Week 2 Pre-dose | -0.08 Celsius | Standard Deviation 0.311 |
| Pegcetacoplan | Change From Baseline in Temperature | Week 2 Post-dose | -0.13 Celsius | Standard Deviation 0.224 |
| Pegcetacoplan | Change From Baseline in Temperature | Week 3 Pre-dose | -0.07 Celsius | Standard Deviation 0.157 |
| Pegcetacoplan | Change From Baseline in Temperature | Week 3 Post-dose | -0.05 Celsius | Standard Deviation 0.197 |
| Pegcetacoplan | Change From Baseline in Temperature | Week 4 Pre-dose | -0.03 Celsius | Standard Deviation 0.173 |
| Pegcetacoplan | Change From Baseline in Temperature | Week 4 Post-dose | 0.02 Celsius | Standard Deviation 0.075 |
| Pegcetacoplan | Change From Baseline in Temperature | Week 6 Pre-dose | -0.10 Celsius | Standard Deviation 0.235 |
| Pegcetacoplan | Change From Baseline in Temperature | Week 6 Post-dose | 0.03 Celsius | Standard Deviation 0.15 |
| Pegcetacoplan | Change From Baseline in Temperature | Week 8 Post-dose | -0.04 Celsius | Standard Deviation 0.207 |
| Pegcetacoplan | Change From Baseline in Temperature | Week 10 Pre-dose | 0.14 Celsius | Standard Deviation 0.485 |
| Pegcetacoplan | Change From Baseline in Temperature | Week 10 Post-dose | 0.02 Celsius | Standard Deviation 0.277 |
| Pegcetacoplan | Change From Baseline in Temperature | Week 12 Pre-dose | -0.05 Celsius | Standard Deviation 0.273 |
| Pegcetacoplan | Change From Baseline in Temperature | Week 12 Post-dose | 0.06 Celsius | Standard Deviation 0.152 |
| Pegcetacoplan | Change From Baseline in Temperature | Week 14 Pre-dose | -0.13 Celsius | Standard Deviation 0.256 |
| Pegcetacoplan | Change From Baseline in Temperature | Week 14 Post-dose | -0.07 Celsius | Standard Deviation 0.379 |
| Pegcetacoplan | Change From Baseline in Temperature | Week 16 | -0.14 Celsius | Standard Deviation 0.299 |
| Pegcetacoplan | Change From Baseline in Temperature | Week 20 | -0.31 Celsius | Standard Deviation 0.43 |
| Pegcetacoplan | Change From Baseline in Temperature | Week 24 | -0.30 Celsius | Standard Deviation 0.522 |
Other Pre-specified
Change From Baseline in Urine Protein/Creatinine Ratio
Change from baseline to Week 24 in urine protein/creatinine ratio
Time frame: Week 24
Population: The number of patients in the analysis population reflects the limited number of samples available due to study discontinuation of patients. Patients with an available measure are included.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Pegcetacoplan | Change From Baseline in Urine Protein/Creatinine Ratio | Week 6 | -0.7370 mg/mg cr | Standard Deviation 1.25579 |
| Pegcetacoplan | Change From Baseline in Urine Protein/Creatinine Ratio | Week 1 | -1.0821 mg/mg cr | Standard Deviation 1.90348 |
| Pegcetacoplan | Change From Baseline in Urine Protein/Creatinine Ratio | Week 2 | 0.2738 mg/mg cr | Standard Deviation 3.95639 |
| Pegcetacoplan | Change From Baseline in Urine Protein/Creatinine Ratio | Week 3 | -0.8321 mg/mg cr | Standard Deviation 1.74384 |
| Pegcetacoplan | Change From Baseline in Urine Protein/Creatinine Ratio | Week 4 | 5.4769 mg/mg cr | Standard Deviation 18.92243 |
| Pegcetacoplan | Change From Baseline in Urine Protein/Creatinine Ratio | Week 8 | -0.5807 mg/mg cr | Standard Deviation 1.27999 |
| Pegcetacoplan | Change From Baseline in Urine Protein/Creatinine Ratio | Week 10 | -0.7423 mg/mg cr | Standard Deviation 1.32039 |
| Pegcetacoplan | Change From Baseline in Urine Protein/Creatinine Ratio | Week 12 | -0.9154 mg/mg cr | Standard Deviation 1.52232 |
| Pegcetacoplan | Change From Baseline in Urine Protein/Creatinine Ratio | Week 14 | -0.9039 mg/mg cr | Standard Deviation 1.54815 |
| Pegcetacoplan | Change From Baseline in Urine Protein/Creatinine Ratio | Week 16 | -0.8121 mg/mg cr | Standard Deviation 1.78436 |
| Pegcetacoplan | Change From Baseline in Urine Protein/Creatinine Ratio | Week 20 | -0.3050 mg/mg cr | Standard Deviation 1.15634 |
| Pegcetacoplan | Change From Baseline in Urine Protein/Creatinine Ratio | Week 24 | -0.7543 mg/mg cr | Standard Deviation 0.5606 |
Other Pre-specified
Number of Participants With Antibodies to Pegcetacoplan Throughout Treatment and Follow-up Periods
Presence of antibodies to pegcetacoplan throughout treatment and follow-up periods.
Time frame: from treatment start to the end of the study, up to 6 months.
| Arm | Measure | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|
| Pegcetacoplan | Number of Participants With Antibodies to Pegcetacoplan Throughout Treatment and Follow-up Periods | 0 Participants |
Other Pre-specified
Number of Participants With Antibodies to Polyethylene Glycol (PEG) Throughout Treatment and Follow-up Periods
Presence of antibodies to PEG throughout treatment and follow-up periods. Baseline represents the situation at treatment start.
Time frame: from treatment start to the end of the study, up to 6 months.
Population: The number of patients in the analysis population decreased over the course of the study, reflecting the limited number of samples available due to study discontinuation of patients. In some cases, there were also missing samples.
| Arm | Measure | Group | Category | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|---|---|
| Pegcetacoplan | Number of Participants With Antibodies to Polyethylene Glycol (PEG) Throughout Treatment and Follow-up Periods | Week 2 | Positive | 6 Participants |
| Pegcetacoplan | Number of Participants With Antibodies to Polyethylene Glycol (PEG) Throughout Treatment and Follow-up Periods | Week 2 | Negative | 1 Participants |
| Pegcetacoplan | Number of Participants With Antibodies to Polyethylene Glycol (PEG) Throughout Treatment and Follow-up Periods | Baseline | Positive | 10 Participants |
| Pegcetacoplan | Number of Participants With Antibodies to Polyethylene Glycol (PEG) Throughout Treatment and Follow-up Periods | Baseline | Negative | 1 Participants |
| Pegcetacoplan | Number of Participants With Antibodies to Polyethylene Glycol (PEG) Throughout Treatment and Follow-up Periods | Week 1 | Positive | 1 Participants |
| Pegcetacoplan | Number of Participants With Antibodies to Polyethylene Glycol (PEG) Throughout Treatment and Follow-up Periods | Week 1 | Negative | 0 Participants |
| Pegcetacoplan | Number of Participants With Antibodies to Polyethylene Glycol (PEG) Throughout Treatment and Follow-up Periods | Week 4 | Positive | 4 Participants |
| Pegcetacoplan | Number of Participants With Antibodies to Polyethylene Glycol (PEG) Throughout Treatment and Follow-up Periods | Week 4 | Negative | 2 Participants |
| Pegcetacoplan | Number of Participants With Antibodies to Polyethylene Glycol (PEG) Throughout Treatment and Follow-up Periods | Week 12 | Positive | 3 Participants |
| Pegcetacoplan | Number of Participants With Antibodies to Polyethylene Glycol (PEG) Throughout Treatment and Follow-up Periods | Week 12 | Negative | 2 Participants |
| Pegcetacoplan | Number of Participants With Antibodies to Polyethylene Glycol (PEG) Throughout Treatment and Follow-up Periods | Week 16 | Positive | 1 Participants |
| Pegcetacoplan | Number of Participants With Antibodies to Polyethylene Glycol (PEG) Throughout Treatment and Follow-up Periods | Week 16 | Negative | 2 Participants |
| Pegcetacoplan | Number of Participants With Antibodies to Polyethylene Glycol (PEG) Throughout Treatment and Follow-up Periods | Week 20 | Positive | 2 Participants |
| Pegcetacoplan | Number of Participants With Antibodies to Polyethylene Glycol (PEG) Throughout Treatment and Follow-up Periods | Week 20 | Negative | 2 Participants |
| Pegcetacoplan | Number of Participants With Antibodies to Polyethylene Glycol (PEG) Throughout Treatment and Follow-up Periods | Week 24 | Positive | 1 Participants |
| Pegcetacoplan | Number of Participants With Antibodies to Polyethylene Glycol (PEG) Throughout Treatment and Follow-up Periods | Week 24 | Negative | 1 Participants |
Other Pre-specified
Number of Participants With Clinically Significant Changes in Abnormal Electrocardiogram Findings
Occurrence of clinically significant abnormal electrocardiogram findings.
Time frame: Week 24
Population: The analysis population is based on the number of patients with assessment at the visit.
| Arm | Measure | Group | Category | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|---|---|
| Pegcetacoplan | Number of Participants With Clinically Significant Changes in Abnormal Electrocardiogram Findings | Week 10 | Normal | 3 Participants |
| Pegcetacoplan | Number of Participants With Clinically Significant Changes in Abnormal Electrocardiogram Findings | Baseline | Normal | 3 Participants |
| Pegcetacoplan | Number of Participants With Clinically Significant Changes in Abnormal Electrocardiogram Findings | Baseline | Abnormal, not clinically significant | 9 Participants |
| Pegcetacoplan | Number of Participants With Clinically Significant Changes in Abnormal Electrocardiogram Findings | Baseline | Abnormal, clinically significant | 0 Participants |
| Pegcetacoplan | Number of Participants With Clinically Significant Changes in Abnormal Electrocardiogram Findings | Week 1 | Normal | 3 Participants |
| Pegcetacoplan | Number of Participants With Clinically Significant Changes in Abnormal Electrocardiogram Findings | Week 1 | Abnormal, not clinically significant | 8 Participants |
| Pegcetacoplan | Number of Participants With Clinically Significant Changes in Abnormal Electrocardiogram Findings | Week 1 | Abnormal, clinically significant | 0 Participants |
| Pegcetacoplan | Number of Participants With Clinically Significant Changes in Abnormal Electrocardiogram Findings | Week 2 | Normal | 4 Participants |
| Pegcetacoplan | Number of Participants With Clinically Significant Changes in Abnormal Electrocardiogram Findings | Week 2 | Abnormal, not clinically significant | 6 Participants |
| Pegcetacoplan | Number of Participants With Clinically Significant Changes in Abnormal Electrocardiogram Findings | Week 2 | Abnormal, clinically significant | 0 Participants |
| Pegcetacoplan | Number of Participants With Clinically Significant Changes in Abnormal Electrocardiogram Findings | Week 3 | Normal | 3 Participants |
| Pegcetacoplan | Number of Participants With Clinically Significant Changes in Abnormal Electrocardiogram Findings | Week 3 | Abnormal, not clinically significant | 6 Participants |
| Pegcetacoplan | Number of Participants With Clinically Significant Changes in Abnormal Electrocardiogram Findings | Week 3 | Abnormal, clinically significant | 0 Participants |
| Pegcetacoplan | Number of Participants With Clinically Significant Changes in Abnormal Electrocardiogram Findings | Week 4 | Normal | 3 Participants |
| Pegcetacoplan | Number of Participants With Clinically Significant Changes in Abnormal Electrocardiogram Findings | Week 4 | Abnormal, not clinically significant | 7 Participants |
| Pegcetacoplan | Number of Participants With Clinically Significant Changes in Abnormal Electrocardiogram Findings | Week 4 | Abnormal, clinically significant | 0 Participants |
| Pegcetacoplan | Number of Participants With Clinically Significant Changes in Abnormal Electrocardiogram Findings | Week 6 | Normal | 4 Participants |
| Pegcetacoplan | Number of Participants With Clinically Significant Changes in Abnormal Electrocardiogram Findings | Week 6 | Abnormal, not clinically significant | 5 Participants |
| Pegcetacoplan | Number of Participants With Clinically Significant Changes in Abnormal Electrocardiogram Findings | Week 6 | Abnormal, clinically significant | 0 Participants |
| Pegcetacoplan | Number of Participants With Clinically Significant Changes in Abnormal Electrocardiogram Findings | Week 8 | Normal | 4 Participants |
| Pegcetacoplan | Number of Participants With Clinically Significant Changes in Abnormal Electrocardiogram Findings | Week 8 | Abnormal, not clinically significant | 5 Participants |
| Pegcetacoplan | Number of Participants With Clinically Significant Changes in Abnormal Electrocardiogram Findings | Week 8 | Abnormal, clinically significant | 0 Participants |
| Pegcetacoplan | Number of Participants With Clinically Significant Changes in Abnormal Electrocardiogram Findings | Week 10 | Abnormal, not clinically significant | 6 Participants |
| Pegcetacoplan | Number of Participants With Clinically Significant Changes in Abnormal Electrocardiogram Findings | Week 10 | Abnormal, clinically significant | 0 Participants |
| Pegcetacoplan | Number of Participants With Clinically Significant Changes in Abnormal Electrocardiogram Findings | Week 12 | Normal | 3 Participants |
| Pegcetacoplan | Number of Participants With Clinically Significant Changes in Abnormal Electrocardiogram Findings | Week 12 | Abnormal, not clinically significant | 5 Participants |
| Pegcetacoplan | Number of Participants With Clinically Significant Changes in Abnormal Electrocardiogram Findings | Week 12 | Abnormal, clinically significant | 0 Participants |
| Pegcetacoplan | Number of Participants With Clinically Significant Changes in Abnormal Electrocardiogram Findings | Week 14 | Normal | 3 Participants |
| Pegcetacoplan | Number of Participants With Clinically Significant Changes in Abnormal Electrocardiogram Findings | Week 14 | Abnormal, not clinically significant | 4 Participants |
| Pegcetacoplan | Number of Participants With Clinically Significant Changes in Abnormal Electrocardiogram Findings | Week 14 | Abnormal, clinically significant | 0 Participants |
| Pegcetacoplan | Number of Participants With Clinically Significant Changes in Abnormal Electrocardiogram Findings | Week 16 | Normal | 2 Participants |
| Pegcetacoplan | Number of Participants With Clinically Significant Changes in Abnormal Electrocardiogram Findings | Week 16 | Abnormal, not clinically significant | 4 Participants |
| Pegcetacoplan | Number of Participants With Clinically Significant Changes in Abnormal Electrocardiogram Findings | Week 16 | Abnormal, clinically significant | 0 Participants |
| Pegcetacoplan | Number of Participants With Clinically Significant Changes in Abnormal Electrocardiogram Findings | Week 20 | Normal | 2 Participants |
| Pegcetacoplan | Number of Participants With Clinically Significant Changes in Abnormal Electrocardiogram Findings | Week 20 | Abnormal, not clinically significant | 4 Participants |
| Pegcetacoplan | Number of Participants With Clinically Significant Changes in Abnormal Electrocardiogram Findings | Week 20 | Abnormal, clinically significant | 0 Participants |
| Pegcetacoplan | Number of Participants With Clinically Significant Changes in Abnormal Electrocardiogram Findings | Week 24 | Normal | 1 Participants |
| Pegcetacoplan | Number of Participants With Clinically Significant Changes in Abnormal Electrocardiogram Findings | Week 24 | Abnormal, not clinically significant | 4 Participants |
| Pegcetacoplan | Number of Participants With Clinically Significant Changes in Abnormal Electrocardiogram Findings | Week 24 | Abnormal, clinically significant | 0 Participants |
Other Pre-specified
Number of Participants With Treatment-emergent Adverse Events
Occurrence and severity of treatment-emergent adverse events.
Time frame: From treatment start to end of study, up to 6 months (8 weeks since last dose of IMP)
| Arm | Measure | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|
| Pegcetacoplan | Number of Participants With Treatment-emergent Adverse Events | 8 Participants |
Other Pre-specified
Number of Participants With Treatment-emergent Anti-PEG Antibodies Throughout Treatment and Follow-up Periods
Presence of treatment-emergent anti-PEG antibodies throughout treatment and follow-up periods. Conservatively, the anti-PEG antibodies detected in a patient with a missing baseline sample have been considered as treatment-emergent.
Time frame: from treatment start to the end of the study, up to 6 months.
| Arm | Measure | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|
| Pegcetacoplan | Number of Participants With Treatment-emergent Anti-PEG Antibodies Throughout Treatment and Follow-up Periods | 2 Participants |