Trial Outcomes & Findings for A Study of Pegcetacoplan for Patients With Transplant-associated Thrombotic Microangiopathy After Hematopoietic Stem Cell Transplantation (NCT NCT05148299)

Last Updated: 2025-11-28

Results Overview

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.

Recruitment status

COMPLETED

Study phase

PHASE2

Target enrollment

12 participants

Primary outcome timeframe

Week 1

Results posted on

2025-11-28

Participant Flow

12 patients were enrolled and treated in study

Participant milestones

Participant milestones
Measure	Pegcetacoplan sterile solution in stoppered glass vial given as infusion, for a total treatment duration of 12 to 16 weeks.
Treatment Period STARTED	12
Treatment Period COMPLETED	9
Treatment Period NOT COMPLETED	3
Follow-up Period STARTED	9
Follow-up Period COMPLETED	6
Follow-up Period NOT COMPLETED	3

Reasons for withdrawal

Reasons for withdrawal
Measure	Pegcetacoplan sterile solution in stoppered glass vial given as infusion, for a total treatment duration of 12 to 16 weeks.
Treatment Period Adverse Event	1
Treatment Period Physician Decision	1
Treatment Period Death	1
Follow-up Period Death	3

Baseline Characteristics

Female patients were analyzed.

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Pegcetacoplan n=12 Participants sterile solution in stoppered glass vial given as infusion, for a total treatment duration of 12 to 16 weeks.
Age, Categorical <=18 years	0 Participants n=12 Participants
Age, Categorical Between 18 and 65 years	10 Participants n=12 Participants
Age, Categorical >=65 years	2 Participants n=12 Participants
Age, Continuous	50.5 Years n=12 Participants
Sex: Female, Male Female	6 Participants n=12 Participants
Sex: Female, Male Male	6 Participants n=12 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	1 Participants n=12 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	11 Participants n=12 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	0 Participants n=12 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=12 Participants
Race (NIH/OMB) Asian	0 Participants n=12 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=12 Participants
Race (NIH/OMB) Black or African American	0 Participants n=12 Participants
Race (NIH/OMB) White	12 Participants n=12 Participants
Race (NIH/OMB) More than one race	0 Participants n=12 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants n=12 Participants
Region of Enrollment Greece	7 participants n=12 Participants
Region of Enrollment United States	3 participants n=12 Participants
Region of Enrollment Spain	2 participants n=12 Participants
Female of childbearing potential Yes	2 Participants n=6 Participants • Female patients were analyzed.
Female of childbearing potential No (surgical sterilization)	0 Participants n=6 Participants • Female patients were analyzed.
Female of childbearing potential No (hysterectomy)	0 Participants n=6 Participants • Female patients were analyzed.
Female of childbearing potential No (post-menopausal more than 1 year)	4 Participants n=6 Participants • Female patients were analyzed.
Male method of contraception Sterilization	0 Participants n=6 Participants • Male patients were analyzed.
Male method of contraception Condom	0 Participants n=6 Participants • Male patients were analyzed.
Male method of contraception Abstinence	6 Participants n=6 Participants • Male patients were analyzed.
Male method of contraception Other	0 Participants n=6 Participants • Male patients were analyzed.
Female method of contraception Male Partner Sterilization	0 Participants n=2 Participants • Female patients of childbearing potential were analyzed.
Female method of contraception Oral contraception	0 Participants n=2 Participants • Female patients of childbearing potential were analyzed.
Female method of contraception Injected or implanted hormonal methods	0 Participants n=2 Participants • Female patients of childbearing potential were analyzed.
Female method of contraception IUD plus barrier method	0 Participants n=2 Participants • Female patients of childbearing potential were analyzed.
Female method of contraception Abstinence	2 Participants n=2 Participants • Female patients of childbearing potential were analyzed.
Female method of contraception Other	0 Participants n=2 Participants • Female patients of childbearing potential were analyzed.
Weight	63.0 kg n=12 Participants
Height	165.75 cm n=12 Participants
Platelet value at diagnosis	21.5 10^9 platelets/L n=12 Participants
Highest platelet value achieved after transplant	117.5 10^9 platelets/L n=12 Participants
Lactate dehydrogenase (LDH) value at diagnosis	740.5 U/L n=12 Participants
Time between HSCT and TA-TMA diagnosis	27.5 Days n=12 Participants
Time between TA-TMA diagnosis and enrollment to the study	6.5 Days n=12 Participants
Presence of schistocytes in the peripheral blood smear Yes	12 Participants n=12 Participants
Presence of schistocytes in the peripheral blood smear No	0 Participants n=12 Participants
Absolute number of schistocytes per high power field (hpf) in the peripheral blood smear	2.4 schistocytes per hpf n=9 Participants • For 3 patients schistocytes were identified but the number per hpf was not available.
Was any biopsy performed to confirm the histologic diagnosis of TA-TMA? Yes	0 Participants n=12 Participants
Was any biopsy performed to confirm the histologic diagnosis of TA-TMA? No	12 Participants n=12 Participants
De novo anemia at diagnosis Yes	5 Participants n=8 Participants • De novo anemia (Yes/No) is available only for patients included using Protocol version 2.0 and could not be derived for Protocol version 1.0.
De novo anemia at diagnosis No	3 Participants n=8 Participants • De novo anemia (Yes/No) is available only for patients included using Protocol version 2.0 and could not be derived for Protocol version 1.0.
The most recent pre-transfusion Hb level at diagnosis	78.0 g/L n=9 Participants • The analysis population consists of the 5 patients with de novo anemia at diagnosis (Protocol version 2.0) and the 4 patients with "Yes" for "Was diagnosis of TA-TMA established as per laboratory markers indicating TMA?" (Protocol version 1.0).
Proteinuria at diagnosis Yes	9 Participants n=12 Participants
Proteinuria at diagnosis No	2 Participants n=12 Participants
Proteinuria at diagnosis Missing	1 Participants n=12 Participants
The most recent random urine protein/creatinine ratio (rUPCR) value at diagnosis	2.0 mg/mg Cr n=7 Participants • One patient had no proteinuria at diagnosis, thus no value collected. One patient had a missing value. Three patients had non available UPCR.
The most recent proteinuria value at diagnosis	0.8475 g/L n=8 Participants • One patient had no proteinuria at diagnosis, thus no value collected. One patient had a missing value. Two patients had UPCR, but no proteinuria recorded.
Elevated plasma concentration of soluble complement 5b-9 (sC5b-9) above upper limit of normal (ULN) Yes	1 Participants n=12 Participants
Elevated plasma concentration of soluble complement 5b-9 (sC5b-9) above upper limit of normal (ULN) No	7 Participants n=12 Participants
Elevated plasma concentration of soluble complement 5b-9 (sC5b-9) above upper limit of normal (ULN) Missing	4 Participants n=12 Participants
Arterial hypertension Yes	6 Participants n=12 Participants
Arterial hypertension No	6 Participants n=12 Participants
The pre-antihypertensive therapy systolic blood pressure	145.0 mmHg n=7 Participants • The analysis population consists of the 6 patients with arterial hypertension (Protocol version 2.0) and the 1 patient that did not have arterial hypertension as derived for Protocol version 1.0 but had sign/symptom of cardiovascular dysfunction.
The pre-antihypertensive therapy diastolic blood pressure	91.0 mmHg n=7 Participants • The analysis population consists of the 6 patients with arterial hypertension (Protocol version 2.0) and the 1 patient that did not have arterial hypertension as derived for Protocol version 1.0 but had sign/symptom of cardiovascular dysfunction.
Is the patient taking any antihypertension medication? Yes	6 Participants n=7 Participants • The analysis population consists of the 6 patients with arterial hypertension (Protocol version 2.0) and the 1 patient that did not have arterial hypertension as derived for Protocol version 1.0 but had sign/symptom of cardiovascular dysfunction.
Is the patient taking any antihypertension medication? No	1 Participants n=7 Participants • The analysis population consists of the 6 patients with arterial hypertension (Protocol version 2.0) and the 1 patient that did not have arterial hypertension as derived for Protocol version 1.0 but had sign/symptom of cardiovascular dysfunction.
Number of ongoing medications excluding diuretics 0 medication	0 Participants n=6 Participants • Patients taking any antihypertension medication were analyzed.
Number of ongoing medications excluding diuretics ≤ 2 medications	4 Participants n=6 Participants • Patients taking any antihypertension medication were analyzed.
Number of ongoing medications excluding diuretics > 2 medications	2 Participants n=6 Participants • Patients taking any antihypertension medication were analyzed.
TA-TMA persisting despite initial management of any triggering condition Yes	12 Participants n=12 Participants
TA-TMA persisting despite initial management of any triggering condition No	0 Participants n=12 Participants
Any sign/symptom of kidney dysfunction Yes	4 Participants n=12 Participants
Any sign/symptom of kidney dysfunction No	8 Participants n=12 Participants
Most recent serum creatinine value	194.4835 µmol/L n=4 Participants • Patients with any sign/symptom of kidney dysfunction were analyzed.
Last serum creatinine value pre-transplant	46.4110 µmol/L n=4 Participants • Patients with any sign/symptom of kidney dysfunction were analyzed.
Patients requiring renal replacement therapy Yes	1 Participants n=4 Participants • Patients with any sign/symptom of kidney dysfunction were analyzed.
Patients requiring renal replacement therapy No	3 Participants n=4 Participants • Patients with any sign/symptom of kidney dysfunction were analyzed.
Any sign/symptom of lungs dysfunction Yes	1 Participants n=12 Participants
Any sign/symptom of lungs dysfunction No	11 Participants n=12 Participants
The lowest oxygen level in the blood	83.0 % n=1 Participants • Patients with any sign/symptom of lungs dysfunction were analyzed.
Patients receiving noninvasive positive pressure ventilation Yes	0 Participants n=1 Participants • Patients with any sign/symptom of lungs dysfunction were analyzed.
Patients receiving noninvasive positive pressure ventilation No	1 Participants n=1 Participants • Patients with any sign/symptom of lungs dysfunction were analyzed.
Patients receiving invasive positive pressure ventilation Yes	1 Participants n=1 Participants • Patients with any sign/symptom of lungs dysfunction were analyzed.
Patients receiving invasive positive pressure ventilation No	0 Participants n=1 Participants • Patients with any sign/symptom of lungs dysfunction were analyzed.
Any sign/symptom of cardiovascular dysfunction Yes	4 Participants n=12 Participants
Any sign/symptom of cardiovascular dysfunction No	8 Participants n=12 Participants
Patients with pulmonary hypertension diagnosis Yes	0 Participants n=4 Participants • Patients with any sign/symptom of cardiovascular dysfunction were analyzed.
Patients with pulmonary hypertension diagnosis No	4 Participants n=4 Participants • Patients with any sign/symptom of cardiovascular dysfunction were analyzed.
Any sign/symptom of serositis Yes	1 Participants n=12 Participants
Any sign/symptom of serositis No	11 Participants n=12 Participants
Patients with pericardial effusion Yes	1 Participants n=1 Participants • Patients with any sign/symptom of serositis were analyzed.
Patients with pericardial effusion No	0 Participants n=1 Participants • Patients with any sign/symptom of serositis were analyzed.
Patients requiring pericardiocentesis Yes	1 Participants n=1 Participants • Patients with any sign/symptom of serositis were analyzed.
Patients requiring pericardiocentesis No	0 Participants n=1 Participants • Patients with any sign/symptom of serositis were analyzed.
Any sign/symptom of central nervous system (CNS) dysfunction Yes	1 Participants n=12 Participants
Any sign/symptom of central nervous system (CNS) dysfunction No	11 Participants n=12 Participants
Patients with diagnosis of posterior reversible encephalopathy syndrome (PRES) Yes	0 Participants n=1 Participants • Patients with any sign/symptom of CNS dysfunction were analyzed.
Patients with diagnosis of posterior reversible encephalopathy syndrome (PRES) No	1 Participants n=1 Participants • Patients with any sign/symptom of CNS dysfunction were analyzed.
Any biopsy-proven GI involvement of TA-TMA Yes	0 Participants n=12 Participants
Any biopsy-proven GI involvement of TA-TMA No	12 Participants n=12 Participants

PRIMARY outcome

Timeframe: 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.

Outcome measures

Outcome measures
Measure	Pegcetacoplan n=12 Participants sterile solution in stoppered glass vial given as infusion, for a total treatment duration of 12 to 16 weeks.
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 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 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 outcome

Timeframe: 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.

Maximum observed serum concentration. Determined using the samples collected post-dose on dosing Days 1, 3 and 5.

Outcome measures

Outcome measures
Measure	Pegcetacoplan n=12 Participants sterile solution in stoppered glass vial given as infusion, for a total treatment duration of 12 to 16 weeks.
Pegcetacoplan PK Parameter Maximal Serum Concentration (Cmax) Day 1	508.1 µg/mL Standard Deviation 134.86
Pegcetacoplan PK Parameter Maximal Serum Concentration (Cmax) Day 3	579.6 µg/mL Standard Deviation 142.11
Pegcetacoplan PK Parameter Maximal Serum Concentration (Cmax) Day 5	705.2 µg/mL Standard Deviation 203.04

PRIMARY outcome

Timeframe: 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.

Time of maximum measured serum concentration. Determined using the samples collected post-dose on dosing Days 1, 3 and 5.

Outcome measures

Outcome measures
Measure	Pegcetacoplan n=12 Participants sterile solution in stoppered glass vial given as infusion, for a total treatment duration of 12 to 16 weeks.
Pegcetacoplan PK Parameter Time to Cmax (Tmax) Day 1	1.0 Hour Interval 0.3 to 48.0
Pegcetacoplan PK Parameter Time to Cmax (Tmax) Day 3	1.02 Hour Interval 0.3 to 8.5
Pegcetacoplan PK Parameter Time to Cmax (Tmax) Day 5	1.05 Hour Interval 0.5 to 24.0

PRIMARY outcome

Timeframe: 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.

Observed serum concentration pre-dose. From Day 8 (Week 1) and onwards, PK samples were taken pre-dose at each visit.

Outcome measures

Outcome measures
Measure	Pegcetacoplan n=12 Participants sterile solution in stoppered glass vial given as infusion, for a total treatment duration of 12 to 16 weeks.
Pegcetacoplan PK Parameter Observed Serum Concentration Pre-dose (Ctrough) Week 1	365.22 µg/mL Interval 308.37 to 432.55
Pegcetacoplan PK Parameter Observed Serum Concentration Pre-dose (Ctrough) Week 2	449.86 µg/mL Interval 344.63 to 587.23
Pegcetacoplan PK Parameter Observed Serum Concentration Pre-dose (Ctrough) Week 3	585.71 µg/mL Interval 456.62 to 751.3
Pegcetacoplan PK Parameter Observed Serum Concentration Pre-dose (Ctrough) Week 4	665.32 µg/mL Interval 501.51 to 882.64
Pegcetacoplan PK Parameter Observed Serum Concentration Pre-dose (Ctrough) Week 6	750.82 µg/mL Interval 602.41 to 935.8
Pegcetacoplan PK Parameter Observed Serum Concentration Pre-dose (Ctrough) Week 8	826.94 µg/mL Interval 655.21 to 1043.69
Pegcetacoplan PK Parameter Observed Serum Concentration Pre-dose (Ctrough) Week 10	761.76 µg/mL Interval 569.21 to 1019.44
Pegcetacoplan PK Parameter Observed Serum Concentration Pre-dose (Ctrough) Week 12	466.29 µg/mL Interval 112.54 to 1931.99
Pegcetacoplan PK Parameter Observed Serum Concentration Pre-dose (Ctrough) Week 14	893.49 µg/mL Interval 769.46 to 1037.5

SECONDARY outcome

Timeframe: 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.

Absolute levels and change from baseline to Week 24 in biomarker of complement activation sC5b-9

Outcome measures

Outcome measures
Measure	Pegcetacoplan n=11 Participants sterile solution in stoppered glass vial given as infusion, for a total treatment duration of 12 to 16 weeks.
Absolute Levels and Change From Baseline in sC5b-9 Baseline Absolute Level	309.2 µg/L Standard Deviation 200.72
Absolute Levels and Change From Baseline in sC5b-9 Week 1 Absolute Level	175.3 µg/L Standard Deviation 81.26
Absolute Levels and Change From Baseline in sC5b-9 Week 1 Change from Baseline	-133.6 µg/L Standard Deviation 176.13
Absolute Levels and Change From Baseline in sC5b-9 Week 2 Absolute Level	152.0 µg/L Standard Deviation 73.95
Absolute Levels and Change From Baseline in sC5b-9 Week 2 Change from Baseline	-163.0 µg/L Standard Deviation 157.36
Absolute Levels and Change From Baseline in sC5b-9 Week 3 Absolute Level	129.8 µg/L Standard Deviation 43.38
Absolute Levels and Change From Baseline in sC5b-9 Week 3 Change from Baseline	-185.2 µg/L Standard Deviation 194.18
Absolute Levels and Change From Baseline in sC5b-9 Week 4 Absolute Level	161.6 µg/L Standard Deviation 78.37
Absolute Levels and Change From Baseline in sC5b-9 Week 4 Change from Baseline	-142.1 µg/L Standard Deviation 160.54
Absolute Levels and Change From Baseline in sC5b-9 Week 6 Absolute Level	111.7 µg/L Standard Deviation 37.98
Absolute Levels and Change From Baseline in sC5b-9 Week 6 Change from Baseline	-204.0 µg/L Standard Deviation 217.36
Absolute Levels and Change From Baseline in sC5b-9 Week 8 Absolute Level	148.0 µg/L Standard Deviation 45.69
Absolute Levels and Change From Baseline in sC5b-9 Week 8 Change from Baseline	-168.3 µg/L Standard Deviation 226.01
Absolute Levels and Change From Baseline in sC5b-9 Week 10 Absolute Level	131.4 µg/L Standard Deviation 70.60
Absolute Levels and Change From Baseline in sC5b-9 Week 10 Change from Baseline	-184.3 µg/L Standard Deviation 179.44
Absolute Levels and Change From Baseline in sC5b-9 Week 12 Absolute Level	141.2 µg/L Standard Deviation 77.97
Absolute Levels and Change From Baseline in sC5b-9 Week 12 Change from Baseline	-214.0 µg/L Standard Deviation 224.06
Absolute Levels and Change From Baseline in sC5b-9 Week 14 Absolute Level	156.8 µg/L Standard Deviation 65.64
Absolute Levels and Change From Baseline in sC5b-9 Week 14 Change from Baseline	-211.8 µg/L Standard Deviation 211.05
Absolute Levels and Change From Baseline in sC5b-9 Week 16 Absolute Level	158.0 µg/L Standard Deviation 54.21
Absolute Levels and Change From Baseline in sC5b-9 Week 16 Change from Baseline	-210.6 µg/L Standard Deviation 240.97
Absolute Levels and Change From Baseline in sC5b-9 Week 20 Absolute Level	190.0 µg/L Standard Deviation 76.28
Absolute Levels and Change From Baseline in sC5b-9 Week 20 Change from Baseline	-171.6 µg/L Standard Deviation 232.00
Absolute Levels and Change From Baseline in sC5b-9 Week 24 Absolute Level	256.0 µg/L Standard Deviation 122.45
Absolute Levels and Change From Baseline in sC5b-9 Week 24 Change from Baseline	-167.8 µg/L Standard Deviation 162.09

SECONDARY outcome

Timeframe: 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.

Absolute levels and change from baseline to Week 24 in biomarker of complement activation C3a

Outcome measures

Outcome measures
Measure	Pegcetacoplan n=11 Participants sterile solution in stoppered glass vial given as infusion, for a total treatment duration of 12 to 16 weeks.
Absolute Levels and Change From Baseline in C3a Baseline Absolute Level	156.9 µg/L Standard Deviation 113.09
Absolute Levels and Change From Baseline in C3a Week 1 Absolute Level	67.6 µg/L Standard Deviation 70.18
Absolute Levels and Change From Baseline in C3a Week 1 Change from Baseline	-93.4 µg/L Standard Deviation 120.73
Absolute Levels and Change From Baseline in C3a Week 2 Absolute Level	126.1 µg/L Standard Deviation 143.32
Absolute Levels and Change From Baseline in C3a Week 2 Change from Baseline	-27.7 µg/L Standard Deviation 201.41
Absolute Levels and Change From Baseline in C3a Week 3 Absolute Level	69.3 µg/L Standard Deviation 75.08
Absolute Levels and Change From Baseline in C3a Week 3 Change from Baseline	-84.4 µg/L Standard Deviation 148.81
Absolute Levels and Change From Baseline in C3a Week 4 Absolute Level	153.6 µg/L Standard Deviation 184.80
Absolute Levels and Change From Baseline in C3a Week 4 Change from Baseline	-11.9 µg/L Standard Deviation 209.85
Absolute Levels and Change From Baseline in C3a Week 6 Absolute Level	54.3 µg/L Standard Deviation 31.97
Absolute Levels and Change From Baseline in C3a Week 6 Change from Baseline	-74.7 µg/L Standard Deviation 61.34
Absolute Levels and Change From Baseline in C3a Week 8 Absolute Level	73.0 µg/L Standard Deviation 51.40
Absolute Levels and Change From Baseline in C3a Week 8 Change from Baseline	-57.5 µg/L Standard Deviation 43.88
Absolute Levels and Change From Baseline in C3a Week 10 Absolute Level	82.7 µg/L Standard Deviation 51.19
Absolute Levels and Change From Baseline in C3a Week 10 Change from Baseline	-46.3 µg/L Standard Deviation 44.74
Absolute Levels and Change From Baseline in C3a Week 12 Absolute Level	218.4 µg/L Standard Deviation 211.57
Absolute Levels and Change From Baseline in C3a Week 12 Change from Baseline	77.8 µg/L Standard Deviation 207.74
Absolute Levels and Change From Baseline in C3a Week 14 Absolute Level	141.0 µg/L Standard Deviation 173.84
Absolute Levels and Change From Baseline in C3a Week 14 Change from Baseline	-3.2 µg/L Standard Deviation 198.30
Absolute Levels and Change From Baseline in C3a Week 16 Absolute Level	150.8 µg/L Standard Deviation 169.23
Absolute Levels and Change From Baseline in C3a Week 16 Change from Baseline	6.6 µg/L Standard Deviation 195.38
Absolute Levels and Change From Baseline in C3a Week 20 Absolute Level	189.8 µg/L Standard Deviation 148.22
Absolute Levels and Change From Baseline in C3a Week 20 Change from Baseline	75.6 µg/L Standard Deviation 157.49
Absolute Levels and Change From Baseline in C3a Week 24 Absolute Level	102.4 µg/L Standard Deviation 75.03
Absolute Levels and Change From Baseline in C3a Week 24 Change from Baseline	-11.5 µg/L Standard Deviation 91.55

SECONDARY outcome

Timeframe: Week 24

Absolute levels and change from baseline to Week 24 in biomarker of complement activation C3

Outcome measures

Outcome measures
Measure	Pegcetacoplan n=11 Participants sterile solution in stoppered glass vial given as infusion, for a total treatment duration of 12 to 16 weeks.
Absolute Levels and Change From Baseline in C3 Baseline Absolute Level	1.095 g/L Standard Deviation 0.2893
Absolute Levels and Change From Baseline in C3 Week 1 Absolute Level	2.500 g/L Standard Deviation 0.6503
Absolute Levels and Change From Baseline in C3 Week 1 Change from Baseline	1.313 g/L Standard Deviation 0.5038
Absolute Levels and Change From Baseline in C3 Week 2 Absolute Level	3.095 g/L Standard Deviation 0.8883
Absolute Levels and Change From Baseline in C3 Week 2 Change from Baseline	1.929 g/L Standard Deviation 0.7034
Absolute Levels and Change From Baseline in C3 Week 3 Absolute Level	2.883 g/L Standard Deviation 0.9188
Absolute Levels and Change From Baseline in C3 Week 3 Change from Baseline	1.824 g/L Standard Deviation 0.7698
Absolute Levels and Change From Baseline in C3 Week 4 Absolute Level	3.716 g/L Standard Deviation 0.9693
Absolute Levels and Change From Baseline in C3 Week 4 Change from Baseline	2.399 g/L Standard Deviation 0.7395
Absolute Levels and Change From Baseline in C3 Week 6 Absolute Level	3.934 g/L Standard Deviation 0.8033
Absolute Levels and Change From Baseline in C3 Week 6 Change from Baseline	2.777 g/L Standard Deviation 0.7718
Absolute Levels and Change From Baseline in C3 Week 8 Absolute Level	4.238 g/L Standard Deviation 0.4511
Absolute Levels and Change From Baseline in C3 Week 8 Change from Baseline	3.040 g/L Standard Deviation 0.3240
Absolute Levels and Change From Baseline in C3 Week 10 Absolute Level	3.920 g/L Standard Deviation 1.0524
Absolute Levels and Change From Baseline in C3 Week 10 Change from Baseline	2.814 g/L Standard Deviation 0.9156
Absolute Levels and Change From Baseline in C3 Week 12 Absolute Level	4.061 g/L Standard Deviation 1.7835
Absolute Levels and Change From Baseline in C3 Week 12 Change from Baseline	2.893 g/L Standard Deviation 1.7103
Absolute Levels and Change From Baseline in C3 Week 14 Absolute Level	4.156 g/L Standard Deviation 1.1392
Absolute Levels and Change From Baseline in C3 Week 14 Change from Baseline	2.957 g/L Standard Deviation 0.9774
Absolute Levels and Change From Baseline in C3 Week 16 Absolute Level	4.320 g/L Standard Deviation 1.2352
Absolute Levels and Change From Baseline in C3 Week 16 Change from Baseline	3.177 g/L Standard Deviation 1.2536
Absolute Levels and Change From Baseline in C3 Week 20 Absolute Level	2.052 g/L Standard Deviation 0.9091
Absolute Levels and Change From Baseline in C3 Week 20 Change from Baseline	0.858 g/L Standard Deviation 0.9097
Absolute Levels and Change From Baseline in C3 Week 24 Absolute Level	1.338 g/L Standard Deviation 0.5070
Absolute Levels and Change From Baseline in C3 Week 24 Change from Baseline	0.047 g/L Standard Deviation 0.3580

SECONDARY outcome

Timeframe: Week 24

Absolute levels and change from baseline to Week 24 in biomarker of complement activation Bb

Outcome measures

Outcome measures
Measure	Pegcetacoplan n=11 Participants sterile solution in stoppered glass vial given as infusion, for a total treatment duration of 12 to 16 weeks.
Absolute Levels and Change From Baseline in Bb Baseline Absolute Level	2.098 mg/L Standard Deviation 1.5087
Absolute Levels and Change From Baseline in Bb Week 1 Absolute Level	0.769 mg/L Standard Deviation 0.4819
Absolute Levels and Change From Baseline in Bb Week 1 Change from Baseline	-1.404 mg/L Standard Deviation 1.2250
Absolute Levels and Change From Baseline in Bb Week 2 Absolute Level	0.726 mg/L Standard Deviation 0.2783
Absolute Levels and Change From Baseline in Bb Week 2 Change from Baseline	-1.354 mg/L Standard Deviation 1.4238
Absolute Levels and Change From Baseline in Bb Week 3 Absolute Level	0.611 mg/L Standard Deviation 0.1335
Absolute Levels and Change From Baseline in Bb Week 3 Change from Baseline	-1.469 mg/L Standard Deviation 1.6091
Absolute Levels and Change From Baseline in Bb Week 4 Absolute Level	0.749 mg/L Standard Deviation 0.1857
Absolute Levels and Change From Baseline in Bb Week 4 Change from Baseline	-1.458 mg/L Standard Deviation 1.6256
Absolute Levels and Change From Baseline in Bb Week 6 Absolute Level	0.617 mg/L Standard Deviation 0.1485
Absolute Levels and Change From Baseline in Bb Week 6 Change from Baseline	-1.720 mg/L Standard Deviation 1.7170
Absolute Levels and Change From Baseline in Bb Week 8 Absolute Level	0.632 mg/L Standard Deviation 0.1308
Absolute Levels and Change From Baseline in Bb Week 8 Change from Baseline	-1.858 mg/L Standard Deviation 1.8627
Absolute Levels and Change From Baseline in Bb Week 10 Absolute Level	0.704 mg/L Standard Deviation 0.2522
Absolute Levels and Change From Baseline in Bb Week 10 Change from Baseline	-1.633 mg/L Standard Deviation 1.6250
Absolute Levels and Change From Baseline in Bb Week 12 Absolute Level	0.746 mg/L Standard Deviation 0.3340
Absolute Levels and Change From Baseline in Bb Week 12 Change from Baseline	-2.034 mg/L Standard Deviation 1.6738
Absolute Levels and Change From Baseline in Bb Week 14 Absolute Level	0.528 mg/L Standard Deviation 0.1555
Absolute Levels and Change From Baseline in Bb Week 14 Change from Baseline	-1.968 mg/L Standard Deviation 1.9232
Absolute Levels and Change From Baseline in Bb Week 16 Absolute Level	0.672 mg/L Standard Deviation 0.3385
Absolute Levels and Change From Baseline in Bb Week 16 Change from Baseline	-1.824 mg/L Standard Deviation 1.9516
Absolute Levels and Change From Baseline in Bb Week 20 Absolute Level	0.600 mg/L Standard Deviation 0.2507
Absolute Levels and Change From Baseline in Bb Week 20 Change from Baseline	-0.966 mg/L Standard Deviation 1.1270
Absolute Levels and Change From Baseline in Bb Week 24 Absolute Level	0.746 mg/L Standard Deviation 0.0631
Absolute Levels and Change From Baseline in Bb Week 24 Change from Baseline	-1.015 mg/L Standard Deviation 1.1970

SECONDARY outcome

Timeframe: Week 24

Absolute levels and change from baseline to Week 24 in biomarker of complement activation C4a

Outcome measures

Outcome measures
Measure	Pegcetacoplan n=11 Participants sterile solution in stoppered glass vial given as infusion, for a total treatment duration of 12 to 16 weeks.
Absolute Levels and Change From Baseline in C4a Baseline Absolute Level	3422.9 ug/L Standard Deviation 2792.23
Absolute Levels and Change From Baseline in C4a Week 1 Absolute Level	1724.6 ug/L Standard Deviation 1219.46
Absolute Levels and Change From Baseline in C4a Week 1 Change from Baseline	-1470.8 ug/L Standard Deviation 3194.16
Absolute Levels and Change From Baseline in C4a Week 2 Absolute Level	4644.1 ug/L Standard Deviation 4426.51
Absolute Levels and Change From Baseline in C4a Week 2 Change from Baseline	1263.3 ug/L Standard Deviation 3370.74
Absolute Levels and Change From Baseline in C4a Week 3 Absolute Level	4118.0 ug/L Standard Deviation 4192.86
Absolute Levels and Change From Baseline in C4a Week 3 Change from Baseline	737.2 ug/L Standard Deviation 3316.77
Absolute Levels and Change From Baseline in C4a Week 4 Absolute Level	4504.8 ug/L Standard Deviation 3690.83
Absolute Levels and Change From Baseline in C4a Week 4 Change from Baseline	811.8 ug/L Standard Deviation 1763.02
Absolute Levels and Change From Baseline in C4a Week 6 Absolute Level	3582.6 ug/L Standard Deviation 3559.59
Absolute Levels and Change From Baseline in C4a Week 6 Change from Baseline	-438.7 ug/L Standard Deviation 5670.06
Absolute Levels and Change From Baseline in C4a Week 8 Absolute Level	2792.5 ug/L Standard Deviation 3573.91
Absolute Levels and Change From Baseline in C4a Week 8 Change from Baseline	-987.2 ug/L Standard Deviation 1295.21
Absolute Levels and Change From Baseline in C4a Week 10 Absolute Level	2234.7 ug/L Standard Deviation 2012.23
Absolute Levels and Change From Baseline in C4a Week 10 Change from Baseline	-1786.6 ug/L Standard Deviation 2007.28
Absolute Levels and Change From Baseline in C4a Week 12 Absolute Level	5171.6 ug/L Standard Deviation 4735.92
Absolute Levels and Change From Baseline in C4a Week 12 Change from Baseline	852.0 ug/L Standard Deviation 4487.81
Absolute Levels and Change From Baseline in C4a Week 14 Absolute Level	2618.2 ug/L Standard Deviation 4142.08
Absolute Levels and Change From Baseline in C4a Week 14 Change from Baseline	-2269.8 ug/L Standard Deviation 4901.62
Absolute Levels and Change From Baseline in C4a Week 16 Absolute Level	1731.8 ug/L Standard Deviation 1285.26
Absolute Levels and Change From Baseline in C4a Week 16 Change from Baseline	-3156.2 ug/L Standard Deviation 3051.14
Absolute Levels and Change From Baseline in C4a Week 20 Absolute Level	4007.8 ug/L Standard Deviation 3648.52
Absolute Levels and Change From Baseline in C4a Week 20 Change from Baseline	-664.6 ug/L Standard Deviation 3821.60
Absolute Levels and Change From Baseline in C4a Week 24 Absolute Level	4329.2 ug/L Standard Deviation 2520.61
Absolute Levels and Change From Baseline in C4a Week 24 Change from Baseline	-403.5 ug/L Standard Deviation 2475.95

SECONDARY outcome

Timeframe: Week 24

Absolute levels and change from baseline to Week 24 in biomarker of complement activation CH50

Outcome measures

Outcome measures
Measure	Pegcetacoplan n=11 Participants sterile solution in stoppered glass vial given as infusion, for a total treatment duration of 12 to 16 weeks.
Absolute Levels and Change From Baseline in Classical Pathway (CH50) Baseline Absolute Level	92.2 U/mL Standard Deviation 17.94
Absolute Levels and Change From Baseline in Classical Pathway (CH50) Week 1 Absolute Level	99.6 U/mL Standard Deviation 29.99
Absolute Levels and Change From Baseline in Classical Pathway (CH50) Week 1 Change from Baseline	7.2 U/mL Standard Deviation 28.48
Absolute Levels and Change From Baseline in Classical Pathway (CH50) Week 2 Absolute Level	116.9 U/mL Standard Deviation 18.98
Absolute Levels and Change From Baseline in Classical Pathway (CH50) Week 2 Change from Baseline	21.9 U/mL Standard Deviation 25.30
Absolute Levels and Change From Baseline in Classical Pathway (CH50) Week 3 Absolute Level	114.9 U/mL Standard Deviation 27.09
Absolute Levels and Change From Baseline in Classical Pathway (CH50) Week 3 Change from Baseline	21.0 U/mL Standard Deviation 35.76
Absolute Levels and Change From Baseline in Classical Pathway (CH50) Week 4 Absolute Level	107.7 U/mL Standard Deviation 21.72
Absolute Levels and Change From Baseline in Classical Pathway (CH50) Week 4 Change from Baseline	12.1 U/mL Standard Deviation 13.64
Absolute Levels and Change From Baseline in Classical Pathway (CH50) Week 6 Absolute Level	114.9 U/mL Standard Deviation 19.36
Absolute Levels and Change From Baseline in Classical Pathway (CH50) Week 6 Change from Baseline	25.1 U/mL Standard Deviation 30.72
Absolute Levels and Change From Baseline in Classical Pathway (CH50) Week 8 Absolute Level	123.4 U/mL Standard Deviation 32.40
Absolute Levels and Change From Baseline in Classical Pathway (CH50) Week 8 Change from Baseline	31.8 U/mL Standard Deviation 35.78
Absolute Levels and Change From Baseline in Classical Pathway (CH50) Week 10 Absolute Level	121.1 U/mL Standard Deviation 34.98
Absolute Levels and Change From Baseline in Classical Pathway (CH50) Week 10 Change from Baseline	25.5 U/mL Standard Deviation 29.59
Absolute Levels and Change From Baseline in Classical Pathway (CH50) Week 12 Absolute Level	103.0 U/mL Standard Deviation 45.15
Absolute Levels and Change From Baseline in Classical Pathway (CH50) Week 12 Change from Baseline	12.7 U/mL Standard Deviation 42.57
Absolute Levels and Change From Baseline in Classical Pathway (CH50) Week 14 Absolute Level	127.4 U/mL Standard Deviation 34.81
Absolute Levels and Change From Baseline in Classical Pathway (CH50) Week 14 Change from Baseline	35.2 U/mL Standard Deviation 44.74
Absolute Levels and Change From Baseline in Classical Pathway (CH50) Week 16 Absolute Level	129.9 U/mL Standard Deviation 30.42
Absolute Levels and Change From Baseline in Classical Pathway (CH50) Week 16 Change from Baseline	32.7 U/mL Standard Deviation 37.69
Absolute Levels and Change From Baseline in Classical Pathway (CH50) Week 20 Absolute Level	119.6 U/mL Standard Deviation 43.93
Absolute Levels and Change From Baseline in Classical Pathway (CH50) Week 20 Change from Baseline	26.3 U/mL Standard Deviation 46.44
Absolute Levels and Change From Baseline in Classical Pathway (CH50) Week 24 Absolute Level	106.8 U/mL Standard Deviation 51.10
Absolute Levels and Change From Baseline in Classical Pathway (CH50) Week 24 Change from Baseline	17.0 U/mL Standard Deviation 73.63

SECONDARY outcome

Timeframe: Week 24

Absolute levels and change from baseline to Week 24 in biomarker of complement activation AH50

Outcome measures

Outcome measures
Measure	Pegcetacoplan n=11 Participants sterile solution in stoppered glass vial given as infusion, for a total treatment duration of 12 to 16 weeks.
Absolute Levels and Change From Baseline in Alternative Pathway (AH50) Baseline Absolute Level	125.8 U/mL Standard Deviation 44.75
Absolute Levels and Change From Baseline in Alternative Pathway (AH50) Week 1 Absolute Level	75.0 U/mL Standard Deviation 57.85
Absolute Levels and Change From Baseline in Alternative Pathway (AH50) Week 1 Change from Baseline	-55.3 U/mL Standard Deviation 49.73
Absolute Levels and Change From Baseline in Alternative Pathway (AH50) Week 2 Absolute Level	65.9 U/mL Standard Deviation 49.10
Absolute Levels and Change From Baseline in Alternative Pathway (AH50) Week 2 Change from Baseline	-47.7 U/mL Standard Deviation 49.15
Absolute Levels and Change From Baseline in Alternative Pathway (AH50) Week 3 Absolute Level	34.7 U/mL Standard Deviation 34.38
Absolute Levels and Change From Baseline in Alternative Pathway (AH50) Week 3 Change from Baseline	-80.7 U/mL Standard Deviation 38.62
Absolute Levels and Change From Baseline in Alternative Pathway (AH50) Week 4 Absolute Level	35.4 U/mL Standard Deviation 38.46
Absolute Levels and Change From Baseline in Alternative Pathway (AH50) Week 4 Change from Baseline	-79.9 U/mL Standard Deviation 36.02
Absolute Levels and Change From Baseline in Alternative Pathway (AH50) Week 6 Absolute Level	24.0 U/mL Standard Deviation 40.27
Absolute Levels and Change From Baseline in Alternative Pathway (AH50) Week 6 Change from Baseline	-85.3 U/mL Standard Deviation 38.05
Absolute Levels and Change From Baseline in Alternative Pathway (AH50) Week 8 Absolute Level	23.6 U/mL Standard Deviation 43.47
Absolute Levels and Change From Baseline in Alternative Pathway (AH50) Week 8 Change from Baseline	-85.8 U/mL Standard Deviation 43.81
Absolute Levels and Change From Baseline in Alternative Pathway (AH50) Week 10 Absolute Level	36.9 U/mL Standard Deviation 54.84
Absolute Levels and Change From Baseline in Alternative Pathway (AH50) Week 10 Change from Baseline	-71.3 U/mL Standard Deviation 55.56
Absolute Levels and Change From Baseline in Alternative Pathway (AH50) Week 12 Absolute Level	60.6 U/mL Standard Deviation 69.54
Absolute Levels and Change From Baseline in Alternative Pathway (AH50) Week 12 Change from Baseline	-49.5 U/mL Standard Deviation 43.34
Absolute Levels and Change From Baseline in Alternative Pathway (AH50) Week 14 Absolute Level	20.0 U/mL Standard Deviation 41.81
Absolute Levels and Change From Baseline in Alternative Pathway (AH50) Week 14 Change from Baseline	-96.5 U/mL Standard Deviation 45.17
Absolute Levels and Change From Baseline in Alternative Pathway (AH50) Week 16 Absolute Level	35.9 U/mL Standard Deviation 51.88
Absolute Levels and Change From Baseline in Alternative Pathway (AH50) Week 16 Change from Baseline	-84.8 U/mL Standard Deviation 37.55
Absolute Levels and Change From Baseline in Alternative Pathway (AH50) Week 20 Absolute Level	118.1 U/mL Standard Deviation 23.74
Absolute Levels and Change From Baseline in Alternative Pathway (AH50) Week 20 Change from Baseline	7.8 U/mL Standard Deviation 55.02
Absolute Levels and Change From Baseline in Alternative Pathway (AH50) Week 24 Absolute Level	129.4 U/mL Standard Deviation 19.03
Absolute Levels and Change From Baseline in Alternative Pathway (AH50) Week 24 Change from Baseline	11.5 U/mL Standard Deviation 63.67

SECONDARY outcome

Timeframe: 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

Outcome measures

Outcome measures
Measure	Pegcetacoplan n=12 Participants sterile solution in stoppered glass vial given as infusion, for a total treatment duration of 12 to 16 weeks.
Number of Participants Reaching Clinical Response at Week 24	0 Participants

SECONDARY outcome

Timeframe: Week 24

A participant will be declared as reaching TMA response upon improvement in laboratory markers of TMA

Outcome measures

Outcome measures
Measure	Pegcetacoplan n=12 Participants sterile solution in stoppered glass vial given as infusion, for a total treatment duration of 12 to 16 weeks.
Number of Participants Reaching TMA Response at Week 24	2 Participants

SECONDARY outcome

Timeframe: Day 100 from diagnosis

Survival

Outcome measures

Outcome measures
Measure	Pegcetacoplan n=12 Participants sterile solution in stoppered glass vial given as infusion, for a total treatment duration of 12 to 16 weeks.
Overall Survival at Day 100	8 Participants

SECONDARY outcome

Timeframe: Week 24 from treatment start

Survival

Outcome measures

Outcome measures
Measure	Pegcetacoplan n=12 Participants sterile solution in stoppered glass vial given as infusion, for a total treatment duration of 12 to 16 weeks.
Overall Survival at Week 24	6 Participants

SECONDARY outcome

Timeframe: 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.

Both clinical response sustained at week 24 and clinical response at any time during the study will be assessed.

Outcome measures

Outcome measures
Measure	Pegcetacoplan n=2 Participants sterile solution in stoppered glass vial given as infusion, for a total treatment duration of 12 to 16 weeks.
Time to Clinical Response	NA Weeks Interval 2.0 to Median is "time to median response" (the time it takes for half of the patients to get response). The median time to clinical response could not be estimated, as there were too few observed responses, and the survival curve never reached a value of 0.5. The confidence interval limits for these values are obtained by seeing when the respective confidence interval limit of the survival function falls below 50%. If it does not do that, then the value is non-estimable.

SECONDARY outcome

Timeframe: 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.

Both TMA response sustained at week 24 and TMA response at any time during the study will be assessed.

Outcome measures

Outcome measures
Measure	Pegcetacoplan n=4 Participants sterile solution in stoppered glass vial given as infusion, for a total treatment duration of 12 to 16 weeks.
Time to TMA Response	24.0 Weeks Interval 6.14 to The confidence interval limits for these values are obtained by seeing when the respective confidence interval limit of the survival function falls below 50%. If it does not do that, then the value is non-estimable.

SECONDARY outcome

Timeframe: 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.

Duration of clinical response sustained at week 24

Outcome measures

Outcome measures
Measure	Pegcetacoplan n=2 Participants sterile solution in stoppered glass vial given as infusion, for a total treatment duration of 12 to 16 weeks.
Duration of Clinical Response	1.6 Weeks Interval 1.3 to 2.0

SECONDARY outcome

Timeframe: 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.

Duration of TMA response sustained at week 24

Outcome measures

Outcome measures
Measure	Pegcetacoplan n=4 Participants sterile solution in stoppered glass vial given as infusion, for a total treatment duration of 12 to 16 weeks.
Duration of TMA Response	4.0 Weeks Interval 2.0 to 6.1

SECONDARY outcome

Timeframe: Week 24

Population: Only 2 patients had available assessment at the visit.

A participant will be declared as relapsing upon appearance of laboratory markers of TMA

Outcome measures

Outcome measures
Measure	Pegcetacoplan n=2 Participants sterile solution in stoppered glass vial given as infusion, for a total treatment duration of 12 to 16 weeks.
TA-TMA Relapse at Week 24	0 Participants

SECONDARY outcome

Timeframe: Week 12

Number of participants reaching clinical response at week 12

Outcome measures

Outcome measures
Measure	Pegcetacoplan n=12 Participants sterile solution in stoppered glass vial given as infusion, for a total treatment duration of 12 to 16 weeks.
Number of Participants Reaching Clinical Response at Week 12	1 Participants

SECONDARY outcome

Timeframe: Week 12

Number of participants reaching TMA response at week 12

Outcome measures

Outcome measures
Measure	Pegcetacoplan n=12 Participants sterile solution in stoppered glass vial given as infusion, for a total treatment duration of 12 to 16 weeks.
Number of Participants Reaching TMA Response at Week 12	2 Participants

OTHER_PRE_SPECIFIED outcome

Timeframe: 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.

Outcome measures

Outcome measures
Measure	Pegcetacoplan n=12 Participants sterile solution in stoppered glass vial given as infusion, for a total treatment duration of 12 to 16 weeks.
Number of Participants With Treatment-emergent Adverse Events	8 Participants

OTHER_PRE_SPECIFIED outcome

Timeframe: 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.

Change from baseline to Week 24 in platelets

Outcome measures

Outcome measures
Measure	Pegcetacoplan n=12 Participants sterile solution in stoppered glass vial given as infusion, for a total treatment duration of 12 to 16 weeks.
Change From Baseline in Platelets Week 1	12.4 10^9 platelets/L Standard Deviation 24.22
Change From Baseline in Platelets Week 2	2.8 10^9 platelets/L Standard Deviation 12.50
Change From Baseline in Platelets Week 3	5.1 10^9 platelets/L Standard Deviation 11.20
Change From Baseline in Platelets Week 4	18.1 10^9 platelets/L Standard Deviation 28.61
Change From Baseline in Platelets Week 6	14.8 10^9 platelets/L Standard Deviation 21.66
Change From Baseline in Platelets Week 8	19.6 10^9 platelets/L Standard Deviation 16.20
Change From Baseline in Platelets Week 10	17.6 10^9 platelets/L Standard Deviation 23.01
Change From Baseline in Platelets Week 12	13.4 10^9 platelets/L Standard Deviation 11.40
Change From Baseline in Platelets Week 14	12.3 10^9 platelets/L Standard Deviation 13.72
Change From Baseline in Platelets Week 16	10.6 10^9 platelets/L Standard Deviation 13.79
Change From Baseline in Platelets Week 20	14.9 10^9 platelets/L Standard Deviation 32.23
Change From Baseline in Platelets Week 24	22.6 10^9 platelets/L Standard Deviation 19.88

OTHER_PRE_SPECIFIED outcome

Timeframe: Week 24

Change from baseline to Week 24 in hemoglobin

Outcome measures

Outcome measures
Measure	Pegcetacoplan n=12 Participants sterile solution in stoppered glass vial given as infusion, for a total treatment duration of 12 to 16 weeks.
Change From Baseline in Hemoglobin Week 1	-3.8 g/L Standard Deviation 14.30
Change From Baseline in Hemoglobin Week 2	8.1 g/L Standard Deviation 12.48
Change From Baseline in Hemoglobin Week 3	3.6 g/L Standard Deviation 14.89
Change From Baseline in Hemoglobin Week 4	8.0 g/L Standard Deviation 21.85
Change From Baseline in Hemoglobin Week 6	2.0 g/L Standard Deviation 13.20
Change From Baseline in Hemoglobin Week 8	9.4 g/L Standard Deviation 15.14
Change From Baseline in Hemoglobin Week 10	13.0 g/L Standard Deviation 15.08
Change From Baseline in Hemoglobin Week 12	10.6 g/L Standard Deviation 16.99
Change From Baseline in Hemoglobin Week 14	5.7 g/L Standard Deviation 19.77
Change From Baseline in Hemoglobin Week 16	7.2 g/L Standard Deviation 17.64
Change From Baseline in Hemoglobin Week 20	13.4 g/L Standard Deviation 11.04
Change From Baseline in Hemoglobin Week 24	14.0 g/L Standard Deviation 11.02

OTHER_PRE_SPECIFIED outcome

Timeframe: Week 24

Change from baseline to Week 24 in LDH

Outcome measures

Outcome measures
Measure	Pegcetacoplan n=12 Participants sterile solution in stoppered glass vial given as infusion, for a total treatment duration of 12 to 16 weeks.
Change From Baseline in Lactate Dehydrogenase (LDH) Week 1	-273.9 U/L Standard Deviation 310.05
Change From Baseline in Lactate Dehydrogenase (LDH) Week 2	-315.9 U/L Standard Deviation 328.13
Change From Baseline in Lactate Dehydrogenase (LDH) Week 3	-348.2 U/L Standard Deviation 346.46
Change From Baseline in Lactate Dehydrogenase (LDH) Week 4	-365.1 U/L Standard Deviation 363.98
Change From Baseline in Lactate Dehydrogenase (LDH) Week 6	-408.0 U/L Standard Deviation 408.28
Change From Baseline in Lactate Dehydrogenase (LDH) Week 8	-406.0 U/L Standard Deviation 427.49
Change From Baseline in Lactate Dehydrogenase (LDH) Week 10	-428.8 U/L Standard Deviation 425.67
Change From Baseline in Lactate Dehydrogenase (LDH) Week 12	-480.9 U/L Standard Deviation 405.14
Change From Baseline in Lactate Dehydrogenase (LDH) Week 14	-336.3 U/L Standard Deviation 406.86
Change From Baseline in Lactate Dehydrogenase (LDH) Week 16	-355.0 U/L Standard Deviation 396.77
Change From Baseline in Lactate Dehydrogenase (LDH) Week 20	-242.1 U/L Standard Deviation 194.73
Change From Baseline in Lactate Dehydrogenase (LDH) Week 24	-198.0 U/L Standard Deviation 235.45

OTHER_PRE_SPECIFIED outcome

Timeframe: Week 24

Change from baseline to Week 24 in haptoglobin

Outcome measures

Outcome measures
Measure	Pegcetacoplan n=12 Participants sterile solution in stoppered glass vial given as infusion, for a total treatment duration of 12 to 16 weeks.
Change From Baseline in Haptoglobin Week 1	0.070 g/L Standard Deviation 0.2280
Change From Baseline in Haptoglobin Week 2	0.244 g/L Standard Deviation 0.4553
Change From Baseline in Haptoglobin Week 3	0.179 g/L Standard Deviation 0.5054
Change From Baseline in Haptoglobin Week 4	0.260 g/L Standard Deviation 0.5674
Change From Baseline in Haptoglobin Week 6	0.590 g/L Standard Deviation 0.6451
Change From Baseline in Haptoglobin Week 8	0.388 g/L Standard Deviation 0.4496
Change From Baseline in Haptoglobin Week 10	0.578 g/L Standard Deviation 0.5832
Change From Baseline in Haptoglobin Week 12	0.625 g/L Standard Deviation 0.6279
Change From Baseline in Haptoglobin Week 14	0.620 g/L Standard Deviation 0.7013
Change From Baseline in Haptoglobin Week 16	0.657 g/L Standard Deviation 0.6629
Change From Baseline in Haptoglobin Week 20	0.686 g/L Standard Deviation 0.7459
Change From Baseline in Haptoglobin Week 24	0.778 g/L Standard Deviation 0.5427

OTHER_PRE_SPECIFIED outcome

Timeframe: Week 24

Change from baseline to Week 24 in indirect bilirubin

Outcome measures

Outcome measures
Measure	Pegcetacoplan n=12 Participants sterile solution in stoppered glass vial given as infusion, for a total treatment duration of 12 to 16 weeks.
Change From Baseline in Indirect Bilirubin Week 1	-7.80 umol/L Standard Deviation 14.213
Change From Baseline in Indirect Bilirubin Week 2	-9.73 umol/L Standard Deviation 12.344
Change From Baseline in Indirect Bilirubin Week 3	-9.71 umol/L Standard Deviation 14.683
Change From Baseline in Indirect Bilirubin Week 4	-9.87 umol/L Standard Deviation 15.239
Change From Baseline in Indirect Bilirubin Week 6	-8.95 umol/L Standard Deviation 18.202
Change From Baseline in Indirect Bilirubin Week 8	-11.98 umol/L Standard Deviation 21.787
Change From Baseline in Indirect Bilirubin Week 10	-10.24 umol/L Standard Deviation 18.390
Change From Baseline in Indirect Bilirubin Week 12	-10.71 umol/L Standard Deviation 23.203
Change From Baseline in Indirect Bilirubin Week 14	-14.03 umol/L Standard Deviation 26.164
Change From Baseline in Indirect Bilirubin Week 16	-13.70 umol/L Standard Deviation 27.536
Change From Baseline in Indirect Bilirubin Week 20	-3.23 umol/L Standard Deviation 9.330
Change From Baseline in Indirect Bilirubin Week 24	-3.65 umol/L Standard Deviation 7.769

OTHER_PRE_SPECIFIED outcome

Timeframe: Week 24

Change from baseline to Week 24 in serum creatinine

Outcome measures

Outcome measures
Measure	Pegcetacoplan n=12 Participants sterile solution in stoppered glass vial given as infusion, for a total treatment duration of 12 to 16 weeks.
Change From Baseline in Serum Creatinine Week 1	12.14 umol/L Standard Deviation 42.106
Change From Baseline in Serum Creatinine Week 2	-3.84 umol/L Standard Deviation 64.131
Change From Baseline in Serum Creatinine Week 3	-14.93 umol/L Standard Deviation 66.231
Change From Baseline in Serum Creatinine Week 4	-28.32 umol/L Standard Deviation 101.178
Change From Baseline in Serum Creatinine Week 6	-8.80 umol/L Standard Deviation 78.912
Change From Baseline in Serum Creatinine Week 8	-6.24 umol/L Standard Deviation 72.867
Change From Baseline in Serum Creatinine Week 10	-7.99 umol/L Standard Deviation 86.096
Change From Baseline in Serum Creatinine Week 12	-22.04 umol/L Standard Deviation 64.786
Change From Baseline in Serum Creatinine Week 14	16.13 umol/L Standard Deviation 43.379
Change From Baseline in Serum Creatinine Week 16	14.13 umol/L Standard Deviation 39.711
Change From Baseline in Serum Creatinine Week 20	17.77 umol/L Standard Deviation 24.444
Change From Baseline in Serum Creatinine Week 24	11.30 umol/L Standard Deviation 17.470

OTHER_PRE_SPECIFIED outcome

Timeframe: Week 24

Change from baseline to Week 24 in urine protein/creatinine ratio

Outcome measures

Outcome measures
Measure	Pegcetacoplan n=12 Participants sterile solution in stoppered glass vial given as infusion, for a total treatment duration of 12 to 16 weeks.
Change From Baseline in Urine Protein/Creatinine Ratio Week 1	-1.0821 mg/mg cr Standard Deviation 1.90348
Change From Baseline in Urine Protein/Creatinine Ratio Week 2	0.2738 mg/mg cr Standard Deviation 3.95639
Change From Baseline in Urine Protein/Creatinine Ratio Week 3	-0.8321 mg/mg cr Standard Deviation 1.74384
Change From Baseline in Urine Protein/Creatinine Ratio Week 4	5.4769 mg/mg cr Standard Deviation 18.92243
Change From Baseline in Urine Protein/Creatinine Ratio Week 6	-0.7370 mg/mg cr Standard Deviation 1.25579
Change From Baseline in Urine Protein/Creatinine Ratio Week 8	-0.5807 mg/mg cr Standard Deviation 1.27999
Change From Baseline in Urine Protein/Creatinine Ratio Week 10	-0.7423 mg/mg cr Standard Deviation 1.32039
Change From Baseline in Urine Protein/Creatinine Ratio Week 12	-0.9154 mg/mg cr Standard Deviation 1.52232
Change From Baseline in Urine Protein/Creatinine Ratio Week 14	-0.9039 mg/mg cr Standard Deviation 1.54815
Change From Baseline in Urine Protein/Creatinine Ratio Week 16	-0.8121 mg/mg cr Standard Deviation 1.78436
Change From Baseline in Urine Protein/Creatinine Ratio Week 20	-0.3050 mg/mg cr Standard Deviation 1.15634
Change From Baseline in Urine Protein/Creatinine Ratio Week 24	-0.7543 mg/mg cr Standard Deviation 0.56060

OTHER_PRE_SPECIFIED outcome

Timeframe: 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.

Outcome measures

Outcome measures
Measure	Pegcetacoplan n=12 Participants sterile solution in stoppered glass vial given as infusion, for a total treatment duration of 12 to 16 weeks.
Change From Baseline in Systolic Blood Pressure Week 1 Pre-dose	-5.0 mmHg Standard Deviation 20.30
Change From Baseline in Systolic Blood Pressure Week 1 Post-dose	-8.7 mmHg Standard Deviation 14.24
Change From Baseline in Systolic Blood Pressure Week 2 Pre-dose	3.9 mmHg Standard Deviation 17.48
Change From Baseline in Systolic Blood Pressure Week 2 Post-dose	-0.4 mmHg Standard Deviation 17.32
Change From Baseline in Systolic Blood Pressure Week 3 Pre-dose	1.3 mmHg Standard Deviation 22.46
Change From Baseline in Systolic Blood Pressure Week 3 Post-dose	-13.2 mmHg Standard Deviation 9.97
Change From Baseline in Systolic Blood Pressure Week 4 Pre-dose	-7.6 mmHg Standard Deviation 15.18
Change From Baseline in Systolic Blood Pressure Week 4 Post-dose	-12.0 mmHg Standard Deviation 14.31
Change From Baseline in Systolic Blood Pressure Week 6 Pre-dose	-10.7 mmHg Standard Deviation 16.71
Change From Baseline in Systolic Blood Pressure Week 6 Post-dose	-24.3 mmHg Standard Deviation 8.66
Change From Baseline in Systolic Blood Pressure Week 8 Pre-dose	-9.1 mmHg Standard Deviation 19.46
Change From Baseline in Systolic Blood Pressure Week 8 Post-dose	-13.2 mmHg Standard Deviation 10.48
Change From Baseline in Systolic Blood Pressure Week 10 Pre-dose	-7.7 mmHg Standard Deviation 16.02
Change From Baseline in Systolic Blood Pressure Week 10 Post-dose	-8.4 mmHg Standard Deviation 17.07
Change From Baseline in Systolic Blood Pressure Week 12 Pre-dose	-10.6 mmHg Standard Deviation 21.47
Change From Baseline in Systolic Blood Pressure Week 12 Post-dose	-15.0 mmHg Standard Deviation 10.15
Change From Baseline in Systolic Blood Pressure Week 14 Pre-dose	-4.0 mmHg Standard Deviation 17.67
Change From Baseline in Systolic Blood Pressure Week 14 Post-dose	-11.3 mmHg Standard Deviation 21.96
Change From Baseline in Systolic Blood Pressure Week 16	-13.3 mmHg Standard Deviation 12.09
Change From Baseline in Systolic Blood Pressure Week 20	-13.7 mmHg Standard Deviation 12.32
Change From Baseline in Systolic Blood Pressure Week 24	-8.3 mmHg Standard Deviation 12.24

OTHER_PRE_SPECIFIED outcome

Timeframe: 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.

Outcome measures

Outcome measures
Measure	Pegcetacoplan n=12 Participants sterile solution in stoppered glass vial given as infusion, for a total treatment duration of 12 to 16 weeks.
Change From Baseline in Diastolic Blood Pressure Week 1 Pre-dose	-4.5 mmHg Standard Deviation 9.21
Change From Baseline in Diastolic Blood Pressure Week 1 Post-dose	-5.1 mmHg Standard Deviation 7.41
Change From Baseline in Diastolic Blood Pressure Week 2 Pre-dose	-2.2 mmHg Standard Deviation 10.28
Change From Baseline in Diastolic Blood Pressure Week 2 Post-dose	-6.0 mmHg Standard Deviation 11.58
Change From Baseline in Diastolic Blood Pressure Week 3 Pre-dose	-2.1 mmHg Standard Deviation 11.50
Change From Baseline in Diastolic Blood Pressure Week 3 Post-dose	-9.2 mmHg Standard Deviation 3.43
Change From Baseline in Diastolic Blood Pressure Week 4 Pre-dose	-7.6 mmHg Standard Deviation 8.88
Change From Baseline in Diastolic Blood Pressure Week 4 Post-dose	-7.3 mmHg Standard Deviation 6.63
Change From Baseline in Diastolic Blood Pressure Week 6 Pre-dose	-7.0 mmHg Standard Deviation 9.42
Change From Baseline in Diastolic Blood Pressure Week 6 Post-dose	-12.8 mmHg Standard Deviation 7.50
Change From Baseline in Diastolic Blood Pressure Week 8 Pre-dose	-7.9 mmHg Standard Deviation 8.98
Change From Baseline in Diastolic Blood Pressure Week 8 Post-dose	-11.3 mmHg Standard Deviation 9.46
Change From Baseline in Diastolic Blood Pressure Week 10 Pre-dose	-7.1 mmHg Standard Deviation 8.88
Change From Baseline in Diastolic Blood Pressure Week 10 Post-dose	-7.2 mmHg Standard Deviation 13.20
Change From Baseline in Diastolic Blood Pressure Week 12 Pre-dose	-8.6 mmHg Standard Deviation 10.86
Change From Baseline in Diastolic Blood Pressure Week 12 Post-dose	-10.8 mmHg Standard Deviation 8.58
Change From Baseline in Diastolic Blood Pressure Week 14 Pre-dose	-5.1 mmHg Standard Deviation 9.04
Change From Baseline in Diastolic Blood Pressure Week 14 Post-dose	-7.7 mmHg Standard Deviation 16.77
Change From Baseline in Diastolic Blood Pressure Week 16	-8.3 mmHg Standard Deviation 8.77
Change From Baseline in Diastolic Blood Pressure Week 20	-10.6 mmHg Standard Deviation 7.28
Change From Baseline in Diastolic Blood Pressure Week 24	-13.2 mmHg Standard Deviation 9.09

OTHER_PRE_SPECIFIED outcome

Timeframe: 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.

Outcome measures

Outcome measures
Measure	Pegcetacoplan n=12 Participants sterile solution in stoppered glass vial given as infusion, for a total treatment duration of 12 to 16 weeks.
Change From Baseline in Respiratory Rate Week 4 Post-dose	1.1 breaths/min Standard Deviation 2.04
Change From Baseline in Respiratory Rate Week 6 Pre-dose	0.4 breaths/min Standard Deviation 1.67
Change From Baseline in Respiratory Rate Week 6 Post-dose	0.5 breaths/min Standard Deviation 1.00
Change From Baseline in Respiratory Rate Week 8 Pre-dose	0.3 breaths/min Standard Deviation 0.87
Change From Baseline in Respiratory Rate Week 8 Post-dose	-0.2 breaths/min Standard Deviation 1.47
Change From Baseline in Respiratory Rate Week 10 Pre-dose	0.0 breaths/min Standard Deviation 1.41
Change From Baseline in Respiratory Rate Week 10 Post-dose	0.0 breaths/min Standard Deviation 1.58
Change From Baseline in Respiratory Rate Week 12 Pre-dose	1.4 breaths/min Standard Deviation 2.62
Change From Baseline in Respiratory Rate Week 12 Post-dose	0.6 breaths/min Standard Deviation 0.55
Change From Baseline in Respiratory Rate Week 14 Pre-dose	0.4 breaths/min Standard Deviation 1.27
Change From Baseline in Respiratory Rate Week 14 Post-dose	0.0 breaths/min Standard Deviation 1.73
Change From Baseline in Respiratory Rate Week 16	0.7 breaths/min Standard Deviation 1.21
Change From Baseline in Respiratory Rate Week 20	0.3 breaths/min Standard Deviation 1.25
Change From Baseline in Respiratory Rate Week 24	0.2 breaths/min Standard Deviation 1.47
Change From Baseline in Respiratory Rate Week 1 Pre-dose	-0.5 breaths/min Standard Deviation 1.69
Change From Baseline in Respiratory Rate Week 1 Post-dose	-0.1 breaths/min Standard Deviation 0.93
Change From Baseline in Respiratory Rate Week 2 Pre-dose	-0.8 breaths/min Standard Deviation 1.69
Change From Baseline in Respiratory Rate Week 2 Post-dose	-0.3 breaths/min Standard Deviation 0.71
Change From Baseline in Respiratory Rate Week 3 Pre-dose	0.0 breaths/min Standard Deviation 1.15
Change From Baseline in Respiratory Rate Week 3 Post-dose	0.5 breaths/min Standard Deviation 0.55
Change From Baseline in Respiratory Rate Week 4 Pre-dose	1.0 breaths/min Standard Deviation 2.45

OTHER_PRE_SPECIFIED outcome

Timeframe: 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.

Outcome measures

Outcome measures
Measure	Pegcetacoplan n=12 Participants sterile solution in stoppered glass vial given as infusion, for a total treatment duration of 12 to 16 weeks.
Change From Baseline in Heart Rate Week 1 Pre-dose	-7.4 bpm Standard Deviation 9.91
Change From Baseline in Heart Rate Week 1 Post-dose	-7.3 bpm Standard Deviation 8.60
Change From Baseline in Heart Rate Week 2 Pre-dose	-4.3 bpm Standard Deviation 9.20
Change From Baseline in Heart Rate Week 2 Post-dose	-3.6 bpm Standard Deviation 11.93
Change From Baseline in Heart Rate Week 3 Pre-dose	-7.8 bpm Standard Deviation 8.73
Change From Baseline in Heart Rate Week 3 Post-dose	-5.5 bpm Standard Deviation 6.06
Change From Baseline in Heart Rate Week 4 Pre-dose	-7.3 bpm Standard Deviation 7.76
Change From Baseline in Heart Rate Week 4 Post-dose	-8.3 bpm Standard Deviation 11.28
Change From Baseline in Heart Rate Week 6 Pre-dose	-6.4 bpm Standard Deviation 3.78
Change From Baseline in Heart Rate Week 6 Post-dose	-8.5 bpm Standard Deviation 4.51
Change From Baseline in Heart Rate Week 8 Pre-dose	-9.2 bpm Standard Deviation 6.44
Change From Baseline in Heart Rate Week 8 Post-dose	-9.3 bpm Standard Deviation 2.25
Change From Baseline in Heart Rate Week 10 Pre-dose	-6.3 bpm Standard Deviation 6.22
Change From Baseline in Heart Rate Week 10 Post-dose	-5.2 bpm Standard Deviation 9.96
Change From Baseline in Heart Rate Week 12 Pre-dose	-5.8 bpm Standard Deviation 8.00
Change From Baseline in Heart Rate Week 12 Post-dose	-11.2 bpm Standard Deviation 2.68
Change From Baseline in Heart Rate Week 14 Pre-dose	-5.1 bpm Standard Deviation 12.33
Change From Baseline in Heart Rate Week 14 Post-dose	1.7 bpm Standard Deviation 7.51
Change From Baseline in Heart Rate Week 16	-12.3 bpm Standard Deviation 10.03
Change From Baseline in Heart Rate Week 20	-8.6 bpm Standard Deviation 9.57
Change From Baseline in Heart Rate Week 24	-6.3 bpm Standard Deviation 9.97

OTHER_PRE_SPECIFIED outcome

Timeframe: 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.

Outcome measures

Outcome measures
Measure	Pegcetacoplan n=12 Participants sterile solution in stoppered glass vial given as infusion, for a total treatment duration of 12 to 16 weeks.
Change From Baseline in Temperature Week 1 Pre-dose	0.10 Celsius Standard Deviation 0.671
Change From Baseline in Temperature Week 1 Post-dose	-0.19 Celsius Standard Deviation 0.298
Change From Baseline in Temperature Week 2 Pre-dose	-0.08 Celsius Standard Deviation 0.311
Change From Baseline in Temperature Week 2 Post-dose	-0.13 Celsius Standard Deviation 0.224
Change From Baseline in Temperature Week 3 Pre-dose	-0.07 Celsius Standard Deviation 0.157
Change From Baseline in Temperature Week 3 Post-dose	-0.05 Celsius Standard Deviation 0.197
Change From Baseline in Temperature Week 4 Pre-dose	-0.03 Celsius Standard Deviation 0.173
Change From Baseline in Temperature Week 4 Post-dose	0.02 Celsius Standard Deviation 0.075
Change From Baseline in Temperature Week 6 Pre-dose	-0.10 Celsius Standard Deviation 0.235
Change From Baseline in Temperature Week 6 Post-dose	0.03 Celsius Standard Deviation 0.150
Change From Baseline in Temperature Week 8 Pre-dose	-0.09 Celsius Standard Deviation 0.322
Change From Baseline in Temperature Week 8 Post-dose	-0.04 Celsius Standard Deviation 0.207
Change From Baseline in Temperature Week 10 Pre-dose	0.14 Celsius Standard Deviation 0.485
Change From Baseline in Temperature Week 10 Post-dose	0.02 Celsius Standard Deviation 0.277
Change From Baseline in Temperature Week 12 Pre-dose	-0.05 Celsius Standard Deviation 0.273
Change From Baseline in Temperature Week 12 Post-dose	0.06 Celsius Standard Deviation 0.152
Change From Baseline in Temperature Week 14 Pre-dose	-0.13 Celsius Standard Deviation 0.256
Change From Baseline in Temperature Week 14 Post-dose	-0.07 Celsius Standard Deviation 0.379
Change From Baseline in Temperature Week 16	-0.14 Celsius Standard Deviation 0.299
Change From Baseline in Temperature Week 20	-0.31 Celsius Standard Deviation 0.430
Change From Baseline in Temperature Week 24	-0.30 Celsius Standard Deviation 0.522

OTHER_PRE_SPECIFIED outcome

Timeframe: Week 24

Population: The analysis population is based on the number of patients with assessment at the visit.

Occurrence of clinically significant abnormal electrocardiogram findings.

Outcome measures

Outcome measures
Measure	Pegcetacoplan n=12 Participants sterile solution in stoppered glass vial given as infusion, for a total treatment duration of 12 to 16 weeks.
Number of Participants With Clinically Significant Changes in Abnormal Electrocardiogram Findings Baseline · Normal	3 Participants
Number of Participants With Clinically Significant Changes in Abnormal Electrocardiogram Findings Baseline · Abnormal, not clinically significant	9 Participants
Number of Participants With Clinically Significant Changes in Abnormal Electrocardiogram Findings Baseline · Abnormal, clinically significant	0 Participants
Number of Participants With Clinically Significant Changes in Abnormal Electrocardiogram Findings Week 1 · Normal	3 Participants
Number of Participants With Clinically Significant Changes in Abnormal Electrocardiogram Findings Week 1 · Abnormal, not clinically significant	8 Participants
Number of Participants With Clinically Significant Changes in Abnormal Electrocardiogram Findings Week 1 · Abnormal, clinically significant	0 Participants
Number of Participants With Clinically Significant Changes in Abnormal Electrocardiogram Findings Week 2 · Normal	4 Participants
Number of Participants With Clinically Significant Changes in Abnormal Electrocardiogram Findings Week 2 · Abnormal, not clinically significant	6 Participants
Number of Participants With Clinically Significant Changes in Abnormal Electrocardiogram Findings Week 2 · Abnormal, clinically significant	0 Participants
Number of Participants With Clinically Significant Changes in Abnormal Electrocardiogram Findings Week 3 · Normal	3 Participants
Number of Participants With Clinically Significant Changes in Abnormal Electrocardiogram Findings Week 3 · Abnormal, not clinically significant	6 Participants
Number of Participants With Clinically Significant Changes in Abnormal Electrocardiogram Findings Week 3 · Abnormal, clinically significant	0 Participants
Number of Participants With Clinically Significant Changes in Abnormal Electrocardiogram Findings Week 4 · Normal	3 Participants
Number of Participants With Clinically Significant Changes in Abnormal Electrocardiogram Findings Week 4 · Abnormal, not clinically significant	7 Participants
Number of Participants With Clinically Significant Changes in Abnormal Electrocardiogram Findings Week 4 · Abnormal, clinically significant	0 Participants
Number of Participants With Clinically Significant Changes in Abnormal Electrocardiogram Findings Week 6 · Normal	4 Participants
Number of Participants With Clinically Significant Changes in Abnormal Electrocardiogram Findings Week 6 · Abnormal, not clinically significant	5 Participants
Number of Participants With Clinically Significant Changes in Abnormal Electrocardiogram Findings Week 6 · Abnormal, clinically significant	0 Participants
Number of Participants With Clinically Significant Changes in Abnormal Electrocardiogram Findings Week 8 · Normal	4 Participants
Number of Participants With Clinically Significant Changes in Abnormal Electrocardiogram Findings Week 8 · Abnormal, not clinically significant	5 Participants
Number of Participants With Clinically Significant Changes in Abnormal Electrocardiogram Findings Week 8 · Abnormal, clinically significant	0 Participants
Number of Participants With Clinically Significant Changes in Abnormal Electrocardiogram Findings Week 10 · Normal	3 Participants
Number of Participants With Clinically Significant Changes in Abnormal Electrocardiogram Findings Week 10 · Abnormal, not clinically significant	6 Participants
Number of Participants With Clinically Significant Changes in Abnormal Electrocardiogram Findings Week 10 · Abnormal, clinically significant	0 Participants
Number of Participants With Clinically Significant Changes in Abnormal Electrocardiogram Findings Week 12 · Normal	3 Participants
Number of Participants With Clinically Significant Changes in Abnormal Electrocardiogram Findings Week 12 · Abnormal, not clinically significant	5 Participants
Number of Participants With Clinically Significant Changes in Abnormal Electrocardiogram Findings Week 12 · Abnormal, clinically significant	0 Participants
Number of Participants With Clinically Significant Changes in Abnormal Electrocardiogram Findings Week 14 · Normal	3 Participants
Number of Participants With Clinically Significant Changes in Abnormal Electrocardiogram Findings Week 14 · Abnormal, not clinically significant	4 Participants
Number of Participants With Clinically Significant Changes in Abnormal Electrocardiogram Findings Week 14 · Abnormal, clinically significant	0 Participants
Number of Participants With Clinically Significant Changes in Abnormal Electrocardiogram Findings Week 16 · Normal	2 Participants
Number of Participants With Clinically Significant Changes in Abnormal Electrocardiogram Findings Week 16 · Abnormal, not clinically significant	4 Participants
Number of Participants With Clinically Significant Changes in Abnormal Electrocardiogram Findings Week 16 · Abnormal, clinically significant	0 Participants
Number of Participants With Clinically Significant Changes in Abnormal Electrocardiogram Findings Week 20 · Normal	2 Participants
Number of Participants With Clinically Significant Changes in Abnormal Electrocardiogram Findings Week 20 · Abnormal, not clinically significant	4 Participants
Number of Participants With Clinically Significant Changes in Abnormal Electrocardiogram Findings Week 20 · Abnormal, clinically significant	0 Participants
Number of Participants With Clinically Significant Changes in Abnormal Electrocardiogram Findings Week 24 · Normal	1 Participants
Number of Participants With Clinically Significant Changes in Abnormal Electrocardiogram Findings Week 24 · Abnormal, not clinically significant	4 Participants
Number of Participants With Clinically Significant Changes in Abnormal Electrocardiogram Findings Week 24 · Abnormal, clinically significant	0 Participants

OTHER_PRE_SPECIFIED outcome

Timeframe: from treatment start to the end of the study, up to 6 months.

Presence of antibodies to pegcetacoplan throughout treatment and follow-up periods.

Outcome measures

Outcome measures
Measure	Pegcetacoplan n=12 Participants sterile solution in stoppered glass vial given as infusion, for a total treatment duration of 12 to 16 weeks.
Number of Participants With Antibodies to Pegcetacoplan Throughout Treatment and Follow-up Periods	0 Participants

OTHER_PRE_SPECIFIED outcome

Timeframe: 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.

Presence of antibodies to PEG throughout treatment and follow-up periods. Baseline represents the situation at treatment start.

Outcome measures

Outcome measures
Measure	Pegcetacoplan n=12 Participants sterile solution in stoppered glass vial given as infusion, for a total treatment duration of 12 to 16 weeks.
Number of Participants With Antibodies to Polyethylene Glycol (PEG) Throughout Treatment and Follow-up Periods Baseline · Positive	10 Participants
Number of Participants With Antibodies to Polyethylene Glycol (PEG) Throughout Treatment and Follow-up Periods Baseline · Negative	1 Participants
Number of Participants With Antibodies to Polyethylene Glycol (PEG) Throughout Treatment and Follow-up Periods Week 1 · Positive	1 Participants
Number of Participants With Antibodies to Polyethylene Glycol (PEG) Throughout Treatment and Follow-up Periods Week 1 · Negative	0 Participants
Number of Participants With Antibodies to Polyethylene Glycol (PEG) Throughout Treatment and Follow-up Periods Week 2 · Positive	6 Participants
Number of Participants With Antibodies to Polyethylene Glycol (PEG) Throughout Treatment and Follow-up Periods Week 2 · Negative	1 Participants
Number of Participants With Antibodies to Polyethylene Glycol (PEG) Throughout Treatment and Follow-up Periods Week 4 · Positive	4 Participants
Number of Participants With Antibodies to Polyethylene Glycol (PEG) Throughout Treatment and Follow-up Periods Week 4 · Negative	2 Participants
Number of Participants With Antibodies to Polyethylene Glycol (PEG) Throughout Treatment and Follow-up Periods Week 12 · Positive	3 Participants
Number of Participants With Antibodies to Polyethylene Glycol (PEG) Throughout Treatment and Follow-up Periods Week 12 · Negative	2 Participants
Number of Participants With Antibodies to Polyethylene Glycol (PEG) Throughout Treatment and Follow-up Periods Week 16 · Positive	1 Participants
Number of Participants With Antibodies to Polyethylene Glycol (PEG) Throughout Treatment and Follow-up Periods Week 16 · Negative	2 Participants
Number of Participants With Antibodies to Polyethylene Glycol (PEG) Throughout Treatment and Follow-up Periods Week 20 · Positive	2 Participants
Number of Participants With Antibodies to Polyethylene Glycol (PEG) Throughout Treatment and Follow-up Periods Week 20 · Negative	2 Participants
Number of Participants With Antibodies to Polyethylene Glycol (PEG) Throughout Treatment and Follow-up Periods Week 24 · Positive	1 Participants
Number of Participants With Antibodies to Polyethylene Glycol (PEG) Throughout Treatment and Follow-up Periods Week 24 · Negative	1 Participants

OTHER_PRE_SPECIFIED outcome

Timeframe: 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.

Outcome measures

Outcome measures
Measure	Pegcetacoplan n=12 Participants sterile solution in stoppered glass vial given as infusion, for a total treatment duration of 12 to 16 weeks.
Number of Participants With Treatment-emergent Anti-PEG Antibodies Throughout Treatment and Follow-up Periods	2 Participants

Adverse Events

Pegcetacoplan

Serious events: 8 serious events

Other events: 5 other events

Deaths: 6 deaths

Serious adverse events

Serious adverse events
Measure	Pegcetacoplan n=12 participants at risk sterile solution in stoppered glass vial given as infusion, for a total treatment duration of 12 to 16 weeks.
Respiratory, thoracic and mediastinal disorders Respiratory failure	16.7% 2/12 • Number of events 2 • From the first dose of IMP and up to 8 weeks after the date of administration of the last dose of IMP, up to 24 weeks
Respiratory, thoracic and mediastinal disorders Acute respiratory failure	8.3% 1/12 • Number of events 1 • From the first dose of IMP and up to 8 weeks after the date of administration of the last dose of IMP, up to 24 weeks
Respiratory, thoracic and mediastinal disorders Pleural effusion	8.3% 1/12 • Number of events 1 • From the first dose of IMP and up to 8 weeks after the date of administration of the last dose of IMP, up to 24 weeks
Respiratory, thoracic and mediastinal disorders Pulmonary embolism	8.3% 1/12 • Number of events 1 • From the first dose of IMP and up to 8 weeks after the date of administration of the last dose of IMP, up to 24 weeks
Nervous system disorders Generalised tonic-clonic seizure	8.3% 1/12 • Number of events 1 • From the first dose of IMP and up to 8 weeks after the date of administration of the last dose of IMP, up to 24 weeks
Nervous system disorders Haemorrhagic stroke	8.3% 1/12 • Number of events 1 • From the first dose of IMP and up to 8 weeks after the date of administration of the last dose of IMP, up to 24 weeks
Nervous system disorders Neuropathy peripheral	8.3% 1/12 • Number of events 1 • From the first dose of IMP and up to 8 weeks after the date of administration of the last dose of IMP, up to 24 weeks
Cardiac disorders Cardiac failure	8.3% 1/12 • Number of events 1 • From the first dose of IMP and up to 8 weeks after the date of administration of the last dose of IMP, up to 24 weeks
Cardiac disorders Cardiac failure congestive	8.3% 1/12 • Number of events 1 • From the first dose of IMP and up to 8 weeks after the date of administration of the last dose of IMP, up to 24 weeks
Infections and infestations Bronchopulmonary aspergillosis	8.3% 1/12 • Number of events 1 • From the first dose of IMP and up to 8 weeks after the date of administration of the last dose of IMP, up to 24 weeks
Infections and infestations Epstein-Barr virus infection reactivation	8.3% 1/12 • Number of events 1 • From the first dose of IMP and up to 8 weeks after the date of administration of the last dose of IMP, up to 24 weeks
Gastrointestinal disorders Ascites	8.3% 1/12 • Number of events 1 • From the first dose of IMP and up to 8 weeks after the date of administration of the last dose of IMP, up to 24 weeks
Immune system disorders Graft versus host disease	8.3% 1/12 • Number of events 1 • From the first dose of IMP and up to 8 weeks after the date of administration of the last dose of IMP, up to 24 weeks
Renal and urinary disorders Acute kidney injury	8.3% 1/12 • Number of events 1 • From the first dose of IMP and up to 8 weeks after the date of administration of the last dose of IMP, up to 24 weeks
Vascular disorders Veno-occlusive disease	8.3% 1/12 • Number of events 1 • From the first dose of IMP and up to 8 weeks after the date of administration of the last dose of IMP, up to 24 weeks

Other adverse events

Other adverse events
Measure	Pegcetacoplan n=12 participants at risk sterile solution in stoppered glass vial given as infusion, for a total treatment duration of 12 to 16 weeks.
Blood and lymphatic system disorders Thrombocytopenia	8.3% 1/12 • Number of events 1 • From the first dose of IMP and up to 8 weeks after the date of administration of the last dose of IMP, up to 24 weeks
Injury, poisoning and procedural complications Foot fracture	8.3% 1/12 • Number of events 1 • From the first dose of IMP and up to 8 weeks after the date of administration of the last dose of IMP, up to 24 weeks
Metabolism and nutrition disorders Metabolic acidosis	8.3% 1/12 • Number of events 1 • From the first dose of IMP and up to 8 weeks after the date of administration of the last dose of IMP, up to 24 weeks
Psychiatric disorders Confusional state	8.3% 1/12 • Number of events 1 • From the first dose of IMP and up to 8 weeks after the date of administration of the last dose of IMP, up to 24 weeks
Psychiatric disorders Delirium	8.3% 1/12 • Number of events 1 • From the first dose of IMP and up to 8 weeks after the date of administration of the last dose of IMP, up to 24 weeks
Psychiatric disorders Insomnia	8.3% 1/12 • Number of events 1 • From the first dose of IMP and up to 8 weeks after the date of administration of the last dose of IMP, up to 24 weeks
Cardiac disorders Atrial fibrillation	8.3% 1/12 • Number of events 1 • From the first dose of IMP and up to 8 weeks after the date of administration of the last dose of IMP, up to 24 weeks
Renal and urinary disorders Acute kidney injury	8.3% 1/12 • Number of events 1 • From the first dose of IMP and up to 8 weeks after the date of administration of the last dose of IMP, up to 24 weeks
Renal and urinary disorders Chromaturia	8.3% 1/12 • Number of events 1 • From the first dose of IMP and up to 8 weeks after the date of administration of the last dose of IMP, up to 24 weeks
Immune system disorders Graft versus host disease	8.3% 1/12 • Number of events 1 • From the first dose of IMP and up to 8 weeks after the date of administration of the last dose of IMP, up to 24 weeks
Musculoskeletal and connective tissue disorders Muscular weakness	8.3% 1/12 • Number of events 1 • From the first dose of IMP and up to 8 weeks after the date of administration of the last dose of IMP, up to 24 weeks
Musculoskeletal and connective tissue disorders Pain in extremity	8.3% 1/12 • Number of events 1 • From the first dose of IMP and up to 8 weeks after the date of administration of the last dose of IMP, up to 24 weeks
Blood and lymphatic system disorders Neutropenia	8.3% 1/12 • Number of events 1 • From the first dose of IMP and up to 8 weeks after the date of administration of the last dose of IMP, up to 24 weeks
Gastrointestinal disorders Diarrhea	25.0% 3/12 • Number of events 4 • From the first dose of IMP and up to 8 weeks after the date of administration of the last dose of IMP, up to 24 weeks
Gastrointestinal disorders Vomiting	16.7% 2/12 • Number of events 3 • From the first dose of IMP and up to 8 weeks after the date of administration of the last dose of IMP, up to 24 weeks
Gastrointestinal disorders Abdominal pain	8.3% 1/12 • Number of events 1 • From the first dose of IMP and up to 8 weeks after the date of administration of the last dose of IMP, up to 24 weeks
Gastrointestinal disorders Hemoperitoneum	8.3% 1/12 • Number of events 1 • From the first dose of IMP and up to 8 weeks after the date of administration of the last dose of IMP, up to 24 weeks
Gastrointestinal disorders Nausea	8.3% 1/12 • Number of events 1 • From the first dose of IMP and up to 8 weeks after the date of administration of the last dose of IMP, up to 24 weeks
Gastrointestinal disorders Oral pain	8.3% 1/12 • Number of events 1 • From the first dose of IMP and up to 8 weeks after the date of administration of the last dose of IMP, up to 24 weeks
Respiratory, thoracic and mediastinal disorders Pleural effusion	8.3% 1/12 • Number of events 1 • From the first dose of IMP and up to 8 weeks after the date of administration of the last dose of IMP, up to 24 weeks
Respiratory, thoracic and mediastinal disorders Bronchospasm	8.3% 1/12 • Number of events 1 • From the first dose of IMP and up to 8 weeks after the date of administration of the last dose of IMP, up to 24 weeks
Respiratory, thoracic and mediastinal disorders Hemoptysis	8.3% 1/12 • Number of events 1 • From the first dose of IMP and up to 8 weeks after the date of administration of the last dose of IMP, up to 24 weeks
Respiratory, thoracic and mediastinal disorders Oropharyngeal pain	8.3% 1/12 • Number of events 1 • From the first dose of IMP and up to 8 weeks after the date of administration of the last dose of IMP, up to 24 weeks
Respiratory, thoracic and mediastinal disorders Pulmonary hemorrhage	8.3% 1/12 • Number of events 1 • From the first dose of IMP and up to 8 weeks after the date of administration of the last dose of IMP, up to 24 weeks
General disorders Pyrexia	16.7% 2/12 • Number of events 2 • From the first dose of IMP and up to 8 weeks after the date of administration of the last dose of IMP, up to 24 weeks
General disorders Generalized oedema	8.3% 1/12 • Number of events 1 • From the first dose of IMP and up to 8 weeks after the date of administration of the last dose of IMP, up to 24 weeks
General disorders Pain	8.3% 1/12 • Number of events 1 • From the first dose of IMP and up to 8 weeks after the date of administration of the last dose of IMP, up to 24 weeks
Infections and infestations Escherichia bacteremia	8.3% 1/12 • Number of events 3 • From the first dose of IMP and up to 8 weeks after the date of administration of the last dose of IMP, up to 24 weeks
Infections and infestations COVID-19	8.3% 1/12 • Number of events 1 • From the first dose of IMP and up to 8 weeks after the date of administration of the last dose of IMP, up to 24 weeks
Infections and infestations Cytomegalovirus infection reactivation	8.3% 1/12 • Number of events 1 • From the first dose of IMP and up to 8 weeks after the date of administration of the last dose of IMP, up to 24 weeks
Infections and infestations Enterococcal bacteremia	8.3% 1/12 • Number of events 1 • From the first dose of IMP and up to 8 weeks after the date of administration of the last dose of IMP, up to 24 weeks
Infections and infestations Oral herpes	8.3% 1/12 • Number of events 1 • From the first dose of IMP and up to 8 weeks after the date of administration of the last dose of IMP, up to 24 weeks
Skin and subcutaneous tissue disorders Decubitus ulcer	16.7% 2/12 • Number of events 2 • From the first dose of IMP and up to 8 weeks after the date of administration of the last dose of IMP, up to 24 weeks
Skin and subcutaneous tissue disorders Herpes simplex	8.3% 1/12 • Number of events 2 • From the first dose of IMP and up to 8 weeks after the date of administration of the last dose of IMP, up to 24 weeks
Psychiatric disorders Agitation	8.3% 1/12 • Number of events 1 • From the first dose of IMP and up to 8 weeks after the date of administration of the last dose of IMP, up to 24 weeks
Psychiatric disorders Anxiety	8.3% 1/12 • Number of events 1 • From the first dose of IMP and up to 8 weeks after the date of administration of the last dose of IMP, up to 24 weeks

Additional Information

Medical Director

Swedish Orphan Biovitrum AB

Phone: +4686972000

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place