Trial Outcomes & Findings for A Study to Evaluate the Efficacy and Safety of Pegcetacoplan in Patients With PNH (NCT NCT04085601)

NCT ID: NCT04085601

Last Updated: 2022-10-21

Results Overview

The Hb stabilization was defined as avoidance of a \>1 gram per deciliter (g/dL) decrease in Hb concentration from Baseline in the absence of transfusion through Week 26.

• Recruitment status: COMPLETED
• Study phase: PHASE3
• Target enrollment: 53 participants
• Primary outcome timeframe: From Baseline (Day 1) up to Week 26
• Results posted on: 2022-10-21

Participant Flow

This study was conducted in subjects with paroxysmal nocturnal hemoglobinuria (PNH) at 22 investigational sites. The study consisted of a screening period (up to 4 weeks), followed by a randomized controlled period (RCP) (26 weeks). All subjects who completed RCP rolled over into a separate open-label, long-term extension study (APL2-307) or completed the safety follow-up (34 weeks).

A total of 68 subjects were screened. Of which, 53 subjects with PNH who met all of the inclusion criteria and none of the exclusion criteria were randomized in a 2:1 ratio either to receive pegcetacoplan or to remain on their current standard of care (SoC) in RCP.

Participant milestones

Measure	Pegcetacoplan Subjects were received subcutaneous (SC) infusion of pegcetacoplan 1080 milligram (mg) twice weekly or every 3 days up to end of the RCP (Week 26). Subjects were not allowed to receive treatment with other complement inhibitors.	Standard of Care Subjects continued to receive SoC treatment but were not allowed to receive treatment with a complement inhibitor unless they qualified for pegcetacoplan escape therapy.
Overall Study STARTED	35	18
Overall Study SoC to Pegcetacoplan (Escape Therapy)	0	11
Overall Study COMPLETED	33	17
Overall Study NOT COMPLETED	2	1

Reasons for withdrawal

Measure	Pegcetacoplan Subjects were received subcutaneous (SC) infusion of pegcetacoplan 1080 milligram (mg) twice weekly or every 3 days up to end of the RCP (Week 26). Subjects were not allowed to receive treatment with other complement inhibitors.	Standard of Care Subjects continued to receive SoC treatment but were not allowed to receive treatment with a complement inhibitor unless they qualified for pegcetacoplan escape therapy.
Overall Study Lost to Follow-up	1	0
Overall Study Death	1	1

Baseline Characteristics

A Study to Evaluate the Efficacy and Safety of Pegcetacoplan in Patients With PNH

Baseline characteristics by cohort

Measure	Pegcetacoplan n=35 Participants Subjects were received SC infusion of pegcetacoplan 1080 milligram (mg) twice weekly or every 3 days up to end of the RCP (Week 26). Subjects were not allowed to receive treatment with other complement inhibitors.	Standard of Care n=18 Participants Subjects continued to receive SoC treatment but were not allowed to receive treatment with a complement inhibitor unless they qualified for pegcetacoplan escape therapy.	Total n=53 Participants Total of all reporting groups
Age, Continuous	42.2 years STANDARD_DEVIATION 12.70 • n=5 Participants	49.1 years STANDARD_DEVIATION 15.64 • n=7 Participants	44.5 years STANDARD_DEVIATION 14.00 • n=5 Participants
Age, Customized <65 years	33 Participants n=5 Participants	14 Participants n=7 Participants	47 Participants n=5 Participants
Age, Customized >=65 and <75 years	2 Participants n=5 Participants	4 Participants n=7 Participants	6 Participants n=5 Participants
Sex: Female, Male Female	16 Participants n=5 Participants	8 Participants n=7 Participants	24 Participants n=5 Participants
Sex: Female, Male Male	19 Participants n=5 Participants	10 Participants n=7 Participants	29 Participants n=5 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	12 Participants n=5 Participants	2 Participants n=7 Participants	14 Participants n=5 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	23 Participants n=5 Participants	16 Participants n=7 Participants	39 Participants n=5 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race/Ethnicity, Customized Black or African American	2 Participants n=5 Participants	0 Participants n=7 Participants	2 Participants n=5 Participants
Race/Ethnicity, Customized American Indian or Alaska Native	9 Participants n=5 Participants	2 Participants n=7 Participants	11 Participants n=5 Participants
Race/Ethnicity, Customized Asian	23 Participants n=5 Participants	16 Participants n=7 Participants	39 Participants n=5 Participants
Race/Ethnicity, Customized Other	1 Participants n=5 Participants	0 Participants n=7 Participants	1 Participants n=5 Participants

PRIMARY outcome

Timeframe: From Baseline (Day 1) up to Week 26

Population: The ITT set included all subjects assigned to treatment.

The Hb stabilization was defined as avoidance of a >1 gram per deciliter (g/dL) decrease in Hb concentration from Baseline in the absence of transfusion through Week 26.

Outcome measures

Measure	Pegcetacoplan n=35 Participants Subjects were received SC infusion of pegcetacoplan 1080 mg twice weekly or every 3 days up to end of the RCP (Week 26). Subjects were not allowed to receive treatment with other complement inhibitors.	Standard of Care n=18 Participants Subjects continued to receive SoC treatment but were not allowed to receive treatment with a complement inhibitor unless they qualified for pegcetacoplan escape therapy.
Number of Subjects Who Achieved Hemoglobin (Hb) Stabilization	30 Participants	0 Participants

PRIMARY outcome

Timeframe: Baseline (Day 1) and Week 26

Population: The ITT set included all subjects assigned to treatment.

The LDH concentration was analyzed using an analysis of covariance (ANCOVA) model with a last observation carried forward (LOCF) and a baseline observation carried forward (BOCF) approach for handling missing data. Baseline was defined as average of measurements prior to first dose of pegcetacoplan or on or prior to randomization of SoC. Post baseline missing values are imputed using multiple imputation method with Markov Chain Mont Carlo method.

Outcome measures

Measure	Pegcetacoplan n=35 Participants Subjects were received SC infusion of pegcetacoplan 1080 mg twice weekly or every 3 days up to end of the RCP (Week 26). Subjects were not allowed to receive treatment with other complement inhibitors.	Standard of Care n=18 Participants Subjects continued to receive SoC treatment but were not allowed to receive treatment with a complement inhibitor unless they qualified for pegcetacoplan escape therapy.
Change From Baseline in Lactate Dehydrogenase (LDH) Concentration At Week 26	-1870.47 Units/Liter (U/L) Standard Error 100.971	-400.09 Units/Liter (U/L) Standard Error 312.988

SECONDARY outcome

Timeframe: Baseline and Week 26

Population: The ITT set included all subjects assigned to treatment.

An Hb response was defined as a =>1 g/dL increase in Hb from baseline at Week 26.

Outcome measures

Measure	Pegcetacoplan n=35 Participants Subjects were received SC infusion of pegcetacoplan 1080 mg twice weekly or every 3 days up to end of the RCP (Week 26). Subjects were not allowed to receive treatment with other complement inhibitors.	Standard of Care n=18 Participants Subjects continued to receive SoC treatment but were not allowed to receive treatment with a complement inhibitor unless they qualified for pegcetacoplan escape therapy.
Number of Subjects With an Hb Response in the Absence of Transfusions	25 Participants	1 Participants

SECONDARY outcome

Timeframe: Baseline and Week 26

Population: The ITT set included all subjects assigned to treatment.

Blood samples were collected via direct venipuncture at the specific time points to determine ARC. Baseline was defined as average of measurements prior to first dose of pegcetacoplan or on or prior to randomization of SoC. Post baseline missing values are imputed using multiple imputation method with Markov Chain Mont Carlo method.

Outcome measures

Measure	Pegcetacoplan n=35 Participants Subjects were received SC infusion of pegcetacoplan 1080 mg twice weekly or every 3 days up to end of the RCP (Week 26). Subjects were not allowed to receive treatment with other complement inhibitors.	Standard of Care n=18 Participants Subjects continued to receive SoC treatment but were not allowed to receive treatment with a complement inhibitor unless they qualified for pegcetacoplan escape therapy.
Change From Baseline in Absolute Reticulocyte Count (ARC) at Week 26	-123.26 10^9 cells/L Standard Error 9.164	-19.44 10^9 cells/L Standard Error 25.209

SECONDARY outcome

Timeframe: Baseline and Week 26

Population: The ITT set included all subjects assigned to treatment.

Baseline was defined as average of measurements prior to first dose of pegcetacoplan or on or prior to randomization of SoC. Post baseline missing values are imputed using multiple imputation method with Markov Chain Mont Carlo method.

Outcome measures

Measure	Pegcetacoplan n=35 Participants Subjects were received SC infusion of pegcetacoplan 1080 mg twice weekly or every 3 days up to end of the RCP (Week 26). Subjects were not allowed to receive treatment with other complement inhibitors.	Standard of Care n=18 Participants Subjects continued to receive SoC treatment but were not allowed to receive treatment with a complement inhibitor unless they qualified for pegcetacoplan escape therapy.
Change From Baseline in Hb Concentration at Week 26	2.94 g/dL Standard Error 0.383	0.27 g/dL Standard Error 0.759

SECONDARY outcome

Timeframe: At Week 26

Population: The ITT set included all subjects assigned to treatment.

Transfusion refers to any transfusion of PRBC, leukocyte-depleted red blood cells (LDPRC), leukocyte poor packed red blood cell (LPRC), leukocyte poor blood (LPB) or whole blood.

Outcome measures

Measure	Pegcetacoplan n=35 Participants Subjects were received SC infusion of pegcetacoplan 1080 mg twice weekly or every 3 days up to end of the RCP (Week 26). Subjects were not allowed to receive treatment with other complement inhibitors.	Standard of Care n=18 Participants Subjects continued to receive SoC treatment but were not allowed to receive treatment with a complement inhibitor unless they qualified for pegcetacoplan escape therapy.
Percentage of Subjects Who Received Transfusion or Decrease of Hb >2 g/dL From Baseline	11.4 percentage of subjects	100 percentage of subjects

SECONDARY outcome

Timeframe: At Week 26

Population: The ITT set included all subjects assigned to treatment.

Transfusion avoidance was defined as the percentage of subjects who did not require a transfusion during the RCP. Transfusion refers to any transfusion of PRBC, LDPRC, LPRC, LPB or whole blood.

Outcome measures

Measure	Pegcetacoplan n=35 Participants Subjects were received SC infusion of pegcetacoplan 1080 mg twice weekly or every 3 days up to end of the RCP (Week 26). Subjects were not allowed to receive treatment with other complement inhibitors.	Standard of Care n=18 Participants Subjects continued to receive SoC treatment but were not allowed to receive treatment with a complement inhibitor unless they qualified for pegcetacoplan escape therapy.
Percentage of Subjects With Transfusion Avoidance	91.4 percentage of subjects	5.6 percentage of subjects

SECONDARY outcome

Timeframe: Up to Week 26

Population: The ITT set included all subjects assigned to treatment.

The number of units of PRBC transfusions was estimated. In one transfusion subjects received one or more units.

Outcome measures

Measure	Pegcetacoplan n=35 Participants Subjects were received SC infusion of pegcetacoplan 1080 mg twice weekly or every 3 days up to end of the RCP (Week 26). Subjects were not allowed to receive treatment with other complement inhibitors.	Standard of Care n=18 Participants Subjects continued to receive SoC treatment but were not allowed to receive treatment with a complement inhibitor unless they qualified for pegcetacoplan escape therapy.
Number of PRBC Units Transfused From Baseline Through Week 26	0.0 PRBC transfusions Interval 0.0 to 19.0	3.0 PRBC transfusions Interval 0.0 to 13.0

SECONDARY outcome

Timeframe: Baseline and Week 26

Population: The ITT set included all subjects assigned to treatment.

The FACIT-Fatigue Scale is a 13-item Likert scaled instrument that is self-administered by the subjects during clinic visits. Subjects were presented with 13 statements and asked to indicate their responses as it applied to the past 7 days. The 5 possible responses are "Not at all" (0), "A little bit" (1), "Somewhat" (2), "Quite a bit" (3), and "Very much" (4). With 13 statements, the total score has a range of 0 to 52. The higher score corresponded to a higher quality of life. Baseline is defined as average of measurements prior to first dose of pegcetacoplan or on or prior to randomization of SoC. Post baseline missing values are imputed using multiple imputation method with Markov Chain Mont Carlo method.

Outcome measures

Measure	Pegcetacoplan n=35 Participants Subjects were received SC infusion of pegcetacoplan 1080 mg twice weekly or every 3 days up to end of the RCP (Week 26). Subjects were not allowed to receive treatment with other complement inhibitors.	Standard of Care n=18 Participants Subjects continued to receive SoC treatment but were not allowed to receive treatment with a complement inhibitor unless they qualified for pegcetacoplan escape therapy.
Change From Baseline in Functional Assessment of Chronic Illness Therapy- (FACIT-Fatigue) Scale Score at Week 26	7.78 scores on a scale Standard Error 1.210	3.26 scores on a scale Standard Error 2.113

SECONDARY outcome

Timeframe: Baseline and Week 26

Population: The ITT set included all subjects assigned to treatment.

Normalization of Hb levels defined as >= 1x LLN at Week 26 in the absence of transfusion. Transfusion refers to any transfusion of PRBC, LDPRC, LPRC, LPB or whole blood.

Outcome measures

Measure	Pegcetacoplan n=35 Participants Subjects were received SC infusion of pegcetacoplan 1080 mg twice weekly or every 3 days up to end of the RCP (Week 26). Subjects were not allowed to receive treatment with other complement inhibitors.	Standard of Care n=18 Participants Subjects continued to receive SoC treatment but were not allowed to receive treatment with a complement inhibitor unless they qualified for pegcetacoplan escape therapy.
Percentage of Subjects With Hb Normalization Levels at Week 26	45.7 percentage of subjects	0 percentage of subjects

SECONDARY outcome

Timeframe: At Week 26

Population: The ITT set included all subjects assigned to treatment.

The LDH normalization was defined as LDH <= 1xupper limit of normal (ULN) of normal range at week 26 in the absence of transfusion. Transfusion refers to any transfusion of PRBC, LDPRC, LPPRC, LPRC, LPB or whole blood.

Outcome measures

Measure	Pegcetacoplan n=35 Participants Subjects were received SC infusion of pegcetacoplan 1080 mg twice weekly or every 3 days up to end of the RCP (Week 26). Subjects were not allowed to receive treatment with other complement inhibitors.	Standard of Care n=18 Participants Subjects continued to receive SoC treatment but were not allowed to receive treatment with a complement inhibitor unless they qualified for pegcetacoplan escape therapy.
Percentage of Subjects With LDH Normalization at Week 26	65.7 percentage of subjects	0 percentage of subjects

SECONDARY outcome

Timeframe: Baseline and Week 26

Population: The ITT set included all subjects assigned to treatment.

The EORTC QLQ-C30 questionnaire (version 3.0) consisted of 30 questions comprised of both multi-item scales and single-item measures to assess overall quality of life in subjects. Questions were designated by functional scales, symptom scales, and global subject QOL/overall perceived health status. For the first 28 questions the 4 possible responses are "Not at all' (1), 'A little' (2), 'Quite a bit' (3) and 'Very much' (4). For the remaining 2 questions the response is requested on a 7-point scale from 1 ('Very poor') to 7 ('Excellent'). Each scale has a range of 0% - 100%. A high scale score represents a higher response level. Baseline is defined as average of measurements prior to first dose of pegcetacoplan or on or prior to randomization of SoC. Post baseline missing values are imputed using multiple imputation method with Markov Chain Mont Carlo method.

Outcome measures

Measure	Pegcetacoplan n=35 Participants Subjects were received SC infusion of pegcetacoplan 1080 mg twice weekly or every 3 days up to end of the RCP (Week 26). Subjects were not allowed to receive treatment with other complement inhibitors.	Standard of Care n=18 Participants Subjects continued to receive SoC treatment but were not allowed to receive treatment with a complement inhibitor unless they qualified for pegcetacoplan escape therapy.
Change From Baseline in European Organization for Research and Treatment of Cancer (EORTC) 30-item Core Quality of Life Questionnaire (QLQ-C30) Scores at Week 26	18.90 scores on a scale Standard Error 2.909	-2.85 scores on a scale Standard Error 5.703

SECONDARY outcome

Timeframe: Baseline and Week 26

Population: The ITT set included all subjects assigned to treatment.

The LASA consisted of 3 items asking respondents to rate their perceived level of functioning. Specific domains include activity level, ability to carry out daily activities, and an item for overall QOL. Their level of functioning was reported on a 0-100 scale with 0 representing "As low as could be" and 100 representing "As high as could be".

Outcome measures

Measure	Pegcetacoplan n=35 Participants Subjects were received SC infusion of pegcetacoplan 1080 mg twice weekly or every 3 days up to end of the RCP (Week 26). Subjects were not allowed to receive treatment with other complement inhibitors.	Standard of Care n=18 Participants Subjects continued to receive SoC treatment but were not allowed to receive treatment with a complement inhibitor unless they qualified for pegcetacoplan escape therapy.
Change From Baseline in Linear Analog Assessment (LASA) Scales Score at Week 26	50.39 scores on a scale Standard Error 9.062	-5.39 scores on a scale Standard Error 17.689

SECONDARY outcome

Timeframe: At Week 26

Population: The ITT set included all subjects assigned to treatment.

Absolute reticulocyte count normalization is defined as ARC < 1x ULN of the gender-specific normal range at week 26 in the absence of transfusion. Subjects who received a transfusion or withdraw from study or escaped from SoC to pegcetacoplan treatment group or lost to follow up without providing efficacy data at Week 26 were classified as non-responders. Transfusion refers to any transfusion of PRBC, LDPRC, LPRC, LPB or whole blood.

Outcome measures

Measure	Pegcetacoplan n=35 Participants Subjects were received SC infusion of pegcetacoplan 1080 mg twice weekly or every 3 days up to end of the RCP (Week 26). Subjects were not allowed to receive treatment with other complement inhibitors.	Standard of Care n=18 Participants Subjects continued to receive SoC treatment but were not allowed to receive treatment with a complement inhibitor unless they qualified for pegcetacoplan escape therapy.
Percentage of Subjects With ARC Normalization	60.0 percentage of subjects	5.6 percentage of subjects

SECONDARY outcome

Timeframe: Up to Week 26

Population: The ITT set included all subjects assigned to treatment.

Hb stabilization is defined as avoidance of a >1 g/dL decrease in Hb levels from baseline through Week 26 in the absence of transfusion. Transfusion refers to any transfusion of PRBC, LDPRC, LPRC, LPB or whole blood.

Outcome measures

Measure	Pegcetacoplan n=35 Participants Subjects were received SC infusion of pegcetacoplan 1080 mg twice weekly or every 3 days up to end of the RCP (Week 26). Subjects were not allowed to receive treatment with other complement inhibitors.	Standard of Care n=18 Participants Subjects continued to receive SoC treatment but were not allowed to receive treatment with a complement inhibitor unless they qualified for pegcetacoplan escape therapy.
Number of Subjects With Failure of Hb Stabilization	4 Participants	18 Participants

SECONDARY outcome

Timeframe: Up to Week 26

Population: The ITT set included all subjects assigned to treatment.

Time to first-on-study PRBC transfusions during RCP were reported. Here NA indicates not estimable.

Outcome measures

Measure	Pegcetacoplan n=35 Participants Subjects were received SC infusion of pegcetacoplan 1080 mg twice weekly or every 3 days up to end of the RCP (Week 26). Subjects were not allowed to receive treatment with other complement inhibitors.	Standard of Care n=18 Participants Subjects continued to receive SoC treatment but were not allowed to receive treatment with a complement inhibitor unless they qualified for pegcetacoplan escape therapy.
Time to First PRBC Transfusion	NA weeks NA indicates that it was not possible to calculate a median and 95% confidence interval for time to first PRBC transfusion as fewer than 10% of subjects in the pegcetacoplan arm were transfused.	7.000 weeks Interval 4.143 to 10.286

Adverse Events

Overall Pegcetacoplan

Serious events: 6 serious events

Other events: 35 other events

Deaths: 1 deaths

Standard of Care

Serious events: 3 serious events

Other events: 12 other events

Deaths: 1 deaths

Serious adverse events

Measure	Overall Pegcetacoplan n=46 participants at risk Subjects were received SC infusion of pegcetacoplan 1080 mg twice weekly or every 3 days up to end of the RCP (Week 26). Subjects were not allowed to receive treatment with other complement inhibitors. During the study, any subject assigned to the SoC reporting group (excluding complement inhibitors) who had an Hb concentration \>=2 g/dL below baseline or who presented with a qualifying thromboembolic event secondary to PNH was offered early escape therapy with pegcetacoplan.	Standard of Care n=18 participants at risk Subjects continued to receive SoC treatment but were not allowed to receive treatment with a complement inhibitor unless they qualified for pegcetacoplan escape therapy.
Blood and lymphatic system disorders Febrile neutropenia	2.2% 1/46 • Number of events 1 • Treatment-emergent adverse events were reported from the first dose of study medication and for up to 8 weeks after the last dose of study medication, approximately 34 weeks. The safety set included all subjects who received at least 1 dose of pegcetacoplan and all subjects who were randomized to SoC. Overall Pegcetacoplan included 11 subjects who escaped from the SoC group to pegcetacoplan group.	5.6% 1/18 • Number of events 1 • Treatment-emergent adverse events were reported from the first dose of study medication and for up to 8 weeks after the last dose of study medication, approximately 34 weeks. The safety set included all subjects who received at least 1 dose of pegcetacoplan and all subjects who were randomized to SoC. Overall Pegcetacoplan included 11 subjects who escaped from the SoC group to pegcetacoplan group.
Blood and lymphatic system disorders Neutropenia	2.2% 1/46 • Number of events 1 • Treatment-emergent adverse events were reported from the first dose of study medication and for up to 8 weeks after the last dose of study medication, approximately 34 weeks. The safety set included all subjects who received at least 1 dose of pegcetacoplan and all subjects who were randomized to SoC. Overall Pegcetacoplan included 11 subjects who escaped from the SoC group to pegcetacoplan group.	0.00% 0/18 • Treatment-emergent adverse events were reported from the first dose of study medication and for up to 8 weeks after the last dose of study medication, approximately 34 weeks. The safety set included all subjects who received at least 1 dose of pegcetacoplan and all subjects who were randomized to SoC. Overall Pegcetacoplan included 11 subjects who escaped from the SoC group to pegcetacoplan group.
Blood and lymphatic system disorders Pancytopenia	2.2% 1/46 • Number of events 1 • Treatment-emergent adverse events were reported from the first dose of study medication and for up to 8 weeks after the last dose of study medication, approximately 34 weeks. The safety set included all subjects who received at least 1 dose of pegcetacoplan and all subjects who were randomized to SoC. Overall Pegcetacoplan included 11 subjects who escaped from the SoC group to pegcetacoplan group.	0.00% 0/18 • Treatment-emergent adverse events were reported from the first dose of study medication and for up to 8 weeks after the last dose of study medication, approximately 34 weeks. The safety set included all subjects who received at least 1 dose of pegcetacoplan and all subjects who were randomized to SoC. Overall Pegcetacoplan included 11 subjects who escaped from the SoC group to pegcetacoplan group.
Blood and lymphatic system disorders Bone marrow failure	0.00% 0/46 • Treatment-emergent adverse events were reported from the first dose of study medication and for up to 8 weeks after the last dose of study medication, approximately 34 weeks. The safety set included all subjects who received at least 1 dose of pegcetacoplan and all subjects who were randomized to SoC. Overall Pegcetacoplan included 11 subjects who escaped from the SoC group to pegcetacoplan group.	5.6% 1/18 • Number of events 1 • Treatment-emergent adverse events were reported from the first dose of study medication and for up to 8 weeks after the last dose of study medication, approximately 34 weeks. The safety set included all subjects who received at least 1 dose of pegcetacoplan and all subjects who were randomized to SoC. Overall Pegcetacoplan included 11 subjects who escaped from the SoC group to pegcetacoplan group.
Blood and lymphatic system disorders Thrombocytopenia	0.00% 0/46 • Treatment-emergent adverse events were reported from the first dose of study medication and for up to 8 weeks after the last dose of study medication, approximately 34 weeks. The safety set included all subjects who received at least 1 dose of pegcetacoplan and all subjects who were randomized to SoC. Overall Pegcetacoplan included 11 subjects who escaped from the SoC group to pegcetacoplan group.	5.6% 1/18 • Number of events 1 • Treatment-emergent adverse events were reported from the first dose of study medication and for up to 8 weeks after the last dose of study medication, approximately 34 weeks. The safety set included all subjects who received at least 1 dose of pegcetacoplan and all subjects who were randomized to SoC. Overall Pegcetacoplan included 11 subjects who escaped from the SoC group to pegcetacoplan group.
Congenital, familial and genetic disorders Dermoid cyst	2.2% 1/46 • Number of events 1 • Treatment-emergent adverse events were reported from the first dose of study medication and for up to 8 weeks after the last dose of study medication, approximately 34 weeks. The safety set included all subjects who received at least 1 dose of pegcetacoplan and all subjects who were randomized to SoC. Overall Pegcetacoplan included 11 subjects who escaped from the SoC group to pegcetacoplan group.	0.00% 0/18 • Treatment-emergent adverse events were reported from the first dose of study medication and for up to 8 weeks after the last dose of study medication, approximately 34 weeks. The safety set included all subjects who received at least 1 dose of pegcetacoplan and all subjects who were randomized to SoC. Overall Pegcetacoplan included 11 subjects who escaped from the SoC group to pegcetacoplan group.
Infections and infestations Pneumocystis jirovecii pneumonia	0.00% 0/46 • Treatment-emergent adverse events were reported from the first dose of study medication and for up to 8 weeks after the last dose of study medication, approximately 34 weeks. The safety set included all subjects who received at least 1 dose of pegcetacoplan and all subjects who were randomized to SoC. Overall Pegcetacoplan included 11 subjects who escaped from the SoC group to pegcetacoplan group.	5.6% 1/18 • Number of events 1 • Treatment-emergent adverse events were reported from the first dose of study medication and for up to 8 weeks after the last dose of study medication, approximately 34 weeks. The safety set included all subjects who received at least 1 dose of pegcetacoplan and all subjects who were randomized to SoC. Overall Pegcetacoplan included 11 subjects who escaped from the SoC group to pegcetacoplan group.
Metabolism and nutrition disorders Metabolic acidosis	0.00% 0/46 • Treatment-emergent adverse events were reported from the first dose of study medication and for up to 8 weeks after the last dose of study medication, approximately 34 weeks. The safety set included all subjects who received at least 1 dose of pegcetacoplan and all subjects who were randomized to SoC. Overall Pegcetacoplan included 11 subjects who escaped from the SoC group to pegcetacoplan group.	5.6% 1/18 • Number of events 1 • Treatment-emergent adverse events were reported from the first dose of study medication and for up to 8 weeks after the last dose of study medication, approximately 34 weeks. The safety set included all subjects who received at least 1 dose of pegcetacoplan and all subjects who were randomized to SoC. Overall Pegcetacoplan included 11 subjects who escaped from the SoC group to pegcetacoplan group.
Blood and lymphatic system disorders Haemolysis	2.2% 1/46 • Number of events 1 • Treatment-emergent adverse events were reported from the first dose of study medication and for up to 8 weeks after the last dose of study medication, approximately 34 weeks. The safety set included all subjects who received at least 1 dose of pegcetacoplan and all subjects who were randomized to SoC. Overall Pegcetacoplan included 11 subjects who escaped from the SoC group to pegcetacoplan group.	0.00% 0/18 • Treatment-emergent adverse events were reported from the first dose of study medication and for up to 8 weeks after the last dose of study medication, approximately 34 weeks. The safety set included all subjects who received at least 1 dose of pegcetacoplan and all subjects who were randomized to SoC. Overall Pegcetacoplan included 11 subjects who escaped from the SoC group to pegcetacoplan group.
Hepatobiliary disorders Bile duct stone	2.2% 1/46 • Number of events 1 • Treatment-emergent adverse events were reported from the first dose of study medication and for up to 8 weeks after the last dose of study medication, approximately 34 weeks. The safety set included all subjects who received at least 1 dose of pegcetacoplan and all subjects who were randomized to SoC. Overall Pegcetacoplan included 11 subjects who escaped from the SoC group to pegcetacoplan group.	0.00% 0/18 • Treatment-emergent adverse events were reported from the first dose of study medication and for up to 8 weeks after the last dose of study medication, approximately 34 weeks. The safety set included all subjects who received at least 1 dose of pegcetacoplan and all subjects who were randomized to SoC. Overall Pegcetacoplan included 11 subjects who escaped from the SoC group to pegcetacoplan group.
Infections and infestations Septic shock	2.2% 1/46 • Number of events 1 • Treatment-emergent adverse events were reported from the first dose of study medication and for up to 8 weeks after the last dose of study medication, approximately 34 weeks. The safety set included all subjects who received at least 1 dose of pegcetacoplan and all subjects who were randomized to SoC. Overall Pegcetacoplan included 11 subjects who escaped from the SoC group to pegcetacoplan group.	5.6% 1/18 • Number of events 1 • Treatment-emergent adverse events were reported from the first dose of study medication and for up to 8 weeks after the last dose of study medication, approximately 34 weeks. The safety set included all subjects who received at least 1 dose of pegcetacoplan and all subjects who were randomized to SoC. Overall Pegcetacoplan included 11 subjects who escaped from the SoC group to pegcetacoplan group.
Infections and infestations Herpes virus infection	0.00% 0/46 • Treatment-emergent adverse events were reported from the first dose of study medication and for up to 8 weeks after the last dose of study medication, approximately 34 weeks. The safety set included all subjects who received at least 1 dose of pegcetacoplan and all subjects who were randomized to SoC. Overall Pegcetacoplan included 11 subjects who escaped from the SoC group to pegcetacoplan group.	5.6% 1/18 • Number of events 1 • Treatment-emergent adverse events were reported from the first dose of study medication and for up to 8 weeks after the last dose of study medication, approximately 34 weeks. The safety set included all subjects who received at least 1 dose of pegcetacoplan and all subjects who were randomized to SoC. Overall Pegcetacoplan included 11 subjects who escaped from the SoC group to pegcetacoplan group.
Infections and infestations Pulmonary tuberculosis	0.00% 0/46 • Treatment-emergent adverse events were reported from the first dose of study medication and for up to 8 weeks after the last dose of study medication, approximately 34 weeks. The safety set included all subjects who received at least 1 dose of pegcetacoplan and all subjects who were randomized to SoC. Overall Pegcetacoplan included 11 subjects who escaped from the SoC group to pegcetacoplan group.	5.6% 1/18 • Number of events 1 • Treatment-emergent adverse events were reported from the first dose of study medication and for up to 8 weeks after the last dose of study medication, approximately 34 weeks. The safety set included all subjects who received at least 1 dose of pegcetacoplan and all subjects who were randomized to SoC. Overall Pegcetacoplan included 11 subjects who escaped from the SoC group to pegcetacoplan group.
Infections and infestations Urinary tract infection	0.00% 0/46 • Treatment-emergent adverse events were reported from the first dose of study medication and for up to 8 weeks after the last dose of study medication, approximately 34 weeks. The safety set included all subjects who received at least 1 dose of pegcetacoplan and all subjects who were randomized to SoC. Overall Pegcetacoplan included 11 subjects who escaped from the SoC group to pegcetacoplan group.	5.6% 1/18 • Number of events 1 • Treatment-emergent adverse events were reported from the first dose of study medication and for up to 8 weeks after the last dose of study medication, approximately 34 weeks. The safety set included all subjects who received at least 1 dose of pegcetacoplan and all subjects who were randomized to SoC. Overall Pegcetacoplan included 11 subjects who escaped from the SoC group to pegcetacoplan group.
Respiratory, thoracic and mediastinal disorders Respiratory failure	0.00% 0/46 • Treatment-emergent adverse events were reported from the first dose of study medication and for up to 8 weeks after the last dose of study medication, approximately 34 weeks. The safety set included all subjects who received at least 1 dose of pegcetacoplan and all subjects who were randomized to SoC. Overall Pegcetacoplan included 11 subjects who escaped from the SoC group to pegcetacoplan group.	5.6% 1/18 • Number of events 1 • Treatment-emergent adverse events were reported from the first dose of study medication and for up to 8 weeks after the last dose of study medication, approximately 34 weeks. The safety set included all subjects who received at least 1 dose of pegcetacoplan and all subjects who were randomized to SoC. Overall Pegcetacoplan included 11 subjects who escaped from the SoC group to pegcetacoplan group.
Blood and lymphatic system disorders Anaemia	2.2% 1/46 • Number of events 1 • Treatment-emergent adverse events were reported from the first dose of study medication and for up to 8 weeks after the last dose of study medication, approximately 34 weeks. The safety set included all subjects who received at least 1 dose of pegcetacoplan and all subjects who were randomized to SoC. Overall Pegcetacoplan included 11 subjects who escaped from the SoC group to pegcetacoplan group.	0.00% 0/18 • Treatment-emergent adverse events were reported from the first dose of study medication and for up to 8 weeks after the last dose of study medication, approximately 34 weeks. The safety set included all subjects who received at least 1 dose of pegcetacoplan and all subjects who were randomized to SoC. Overall Pegcetacoplan included 11 subjects who escaped from the SoC group to pegcetacoplan group.

Other adverse events

Measure	Overall Pegcetacoplan n=46 participants at risk Subjects were received SC infusion of pegcetacoplan 1080 mg twice weekly or every 3 days up to end of the RCP (Week 26). Subjects were not allowed to receive treatment with other complement inhibitors. During the study, any subject assigned to the SoC reporting group (excluding complement inhibitors) who had an Hb concentration \>=2 g/dL below baseline or who presented with a qualifying thromboembolic event secondary to PNH was offered early escape therapy with pegcetacoplan.	Standard of Care n=18 participants at risk Subjects continued to receive SoC treatment but were not allowed to receive treatment with a complement inhibitor unless they qualified for pegcetacoplan escape therapy.
General disorders Pyrexia	8.7% 4/46 • Number of events 4 • Treatment-emergent adverse events were reported from the first dose of study medication and for up to 8 weeks after the last dose of study medication, approximately 34 weeks. The safety set included all subjects who received at least 1 dose of pegcetacoplan and all subjects who were randomized to SoC. Overall Pegcetacoplan included 11 subjects who escaped from the SoC group to pegcetacoplan group.	0.00% 0/18 • Treatment-emergent adverse events were reported from the first dose of study medication and for up to 8 weeks after the last dose of study medication, approximately 34 weeks. The safety set included all subjects who received at least 1 dose of pegcetacoplan and all subjects who were randomized to SoC. Overall Pegcetacoplan included 11 subjects who escaped from the SoC group to pegcetacoplan group.
General disorders Fatigue	2.2% 1/46 • Number of events 1 • Treatment-emergent adverse events were reported from the first dose of study medication and for up to 8 weeks after the last dose of study medication, approximately 34 weeks. The safety set included all subjects who received at least 1 dose of pegcetacoplan and all subjects who were randomized to SoC. Overall Pegcetacoplan included 11 subjects who escaped from the SoC group to pegcetacoplan group.	5.6% 1/18 • Number of events 2 • Treatment-emergent adverse events were reported from the first dose of study medication and for up to 8 weeks after the last dose of study medication, approximately 34 weeks. The safety set included all subjects who received at least 1 dose of pegcetacoplan and all subjects who were randomized to SoC. Overall Pegcetacoplan included 11 subjects who escaped from the SoC group to pegcetacoplan group.
General disorders Malaise	0.00% 0/46 • Treatment-emergent adverse events were reported from the first dose of study medication and for up to 8 weeks after the last dose of study medication, approximately 34 weeks. The safety set included all subjects who received at least 1 dose of pegcetacoplan and all subjects who were randomized to SoC. Overall Pegcetacoplan included 11 subjects who escaped from the SoC group to pegcetacoplan group.	5.6% 1/18 • Number of events 1 • Treatment-emergent adverse events were reported from the first dose of study medication and for up to 8 weeks after the last dose of study medication, approximately 34 weeks. The safety set included all subjects who received at least 1 dose of pegcetacoplan and all subjects who were randomized to SoC. Overall Pegcetacoplan included 11 subjects who escaped from the SoC group to pegcetacoplan group.
Skin and subcutaneous tissue disorders Ecchymosis	6.5% 3/46 • Number of events 4 • Treatment-emergent adverse events were reported from the first dose of study medication and for up to 8 weeks after the last dose of study medication, approximately 34 weeks. The safety set included all subjects who received at least 1 dose of pegcetacoplan and all subjects who were randomized to SoC. Overall Pegcetacoplan included 11 subjects who escaped from the SoC group to pegcetacoplan group.	0.00% 0/18 • Treatment-emergent adverse events were reported from the first dose of study medication and for up to 8 weeks after the last dose of study medication, approximately 34 weeks. The safety set included all subjects who received at least 1 dose of pegcetacoplan and all subjects who were randomized to SoC. Overall Pegcetacoplan included 11 subjects who escaped from the SoC group to pegcetacoplan group.
Skin and subcutaneous tissue disorders Erythema	6.5% 3/46 • Number of events 4 • Treatment-emergent adverse events were reported from the first dose of study medication and for up to 8 weeks after the last dose of study medication, approximately 34 weeks. The safety set included all subjects who received at least 1 dose of pegcetacoplan and all subjects who were randomized to SoC. Overall Pegcetacoplan included 11 subjects who escaped from the SoC group to pegcetacoplan group.	0.00% 0/18 • Treatment-emergent adverse events were reported from the first dose of study medication and for up to 8 weeks after the last dose of study medication, approximately 34 weeks. The safety set included all subjects who received at least 1 dose of pegcetacoplan and all subjects who were randomized to SoC. Overall Pegcetacoplan included 11 subjects who escaped from the SoC group to pegcetacoplan group.
Infections and infestations Viral infection	6.5% 3/46 • Number of events 3 • Treatment-emergent adverse events were reported from the first dose of study medication and for up to 8 weeks after the last dose of study medication, approximately 34 weeks. The safety set included all subjects who received at least 1 dose of pegcetacoplan and all subjects who were randomized to SoC. Overall Pegcetacoplan included 11 subjects who escaped from the SoC group to pegcetacoplan group.	0.00% 0/18 • Treatment-emergent adverse events were reported from the first dose of study medication and for up to 8 weeks after the last dose of study medication, approximately 34 weeks. The safety set included all subjects who received at least 1 dose of pegcetacoplan and all subjects who were randomized to SoC. Overall Pegcetacoplan included 11 subjects who escaped from the SoC group to pegcetacoplan group.
Infections and infestations Upper respiratory tract infection	2.2% 1/46 • Number of events 1 • Treatment-emergent adverse events were reported from the first dose of study medication and for up to 8 weeks after the last dose of study medication, approximately 34 weeks. The safety set included all subjects who received at least 1 dose of pegcetacoplan and all subjects who were randomized to SoC. Overall Pegcetacoplan included 11 subjects who escaped from the SoC group to pegcetacoplan group.	11.1% 2/18 • Number of events 2 • Treatment-emergent adverse events were reported from the first dose of study medication and for up to 8 weeks after the last dose of study medication, approximately 34 weeks. The safety set included all subjects who received at least 1 dose of pegcetacoplan and all subjects who were randomized to SoC. Overall Pegcetacoplan included 11 subjects who escaped from the SoC group to pegcetacoplan group.
Infections and infestations Influenza	0.00% 0/46 • Treatment-emergent adverse events were reported from the first dose of study medication and for up to 8 weeks after the last dose of study medication, approximately 34 weeks. The safety set included all subjects who received at least 1 dose of pegcetacoplan and all subjects who were randomized to SoC. Overall Pegcetacoplan included 11 subjects who escaped from the SoC group to pegcetacoplan group.	5.6% 1/18 • Number of events 1 • Treatment-emergent adverse events were reported from the first dose of study medication and for up to 8 weeks after the last dose of study medication, approximately 34 weeks. The safety set included all subjects who received at least 1 dose of pegcetacoplan and all subjects who were randomized to SoC. Overall Pegcetacoplan included 11 subjects who escaped from the SoC group to pegcetacoplan group.
Metabolism and nutrition disorders Hypokalaemia	13.0% 6/46 • Number of events 7 • Treatment-emergent adverse events were reported from the first dose of study medication and for up to 8 weeks after the last dose of study medication, approximately 34 weeks. The safety set included all subjects who received at least 1 dose of pegcetacoplan and all subjects who were randomized to SoC. Overall Pegcetacoplan included 11 subjects who escaped from the SoC group to pegcetacoplan group.	11.1% 2/18 • Number of events 2 • Treatment-emergent adverse events were reported from the first dose of study medication and for up to 8 weeks after the last dose of study medication, approximately 34 weeks. The safety set included all subjects who received at least 1 dose of pegcetacoplan and all subjects who were randomized to SoC. Overall Pegcetacoplan included 11 subjects who escaped from the SoC group to pegcetacoplan group.
Metabolism and nutrition disorders Hyperuricaemia	2.2% 1/46 • Number of events 1 • Treatment-emergent adverse events were reported from the first dose of study medication and for up to 8 weeks after the last dose of study medication, approximately 34 weeks. The safety set included all subjects who received at least 1 dose of pegcetacoplan and all subjects who were randomized to SoC. Overall Pegcetacoplan included 11 subjects who escaped from the SoC group to pegcetacoplan group.	5.6% 1/18 • Number of events 1 • Treatment-emergent adverse events were reported from the first dose of study medication and for up to 8 weeks after the last dose of study medication, approximately 34 weeks. The safety set included all subjects who received at least 1 dose of pegcetacoplan and all subjects who were randomized to SoC. Overall Pegcetacoplan included 11 subjects who escaped from the SoC group to pegcetacoplan group.
Metabolism and nutrition disorders Dehydration	0.00% 0/46 • Treatment-emergent adverse events were reported from the first dose of study medication and for up to 8 weeks after the last dose of study medication, approximately 34 weeks. The safety set included all subjects who received at least 1 dose of pegcetacoplan and all subjects who were randomized to SoC. Overall Pegcetacoplan included 11 subjects who escaped from the SoC group to pegcetacoplan group.	5.6% 1/18 • Number of events 1 • Treatment-emergent adverse events were reported from the first dose of study medication and for up to 8 weeks after the last dose of study medication, approximately 34 weeks. The safety set included all subjects who received at least 1 dose of pegcetacoplan and all subjects who were randomized to SoC. Overall Pegcetacoplan included 11 subjects who escaped from the SoC group to pegcetacoplan group.
Blood and lymphatic system disorders Anaemia	6.5% 3/46 • Number of events 3 • Treatment-emergent adverse events were reported from the first dose of study medication and for up to 8 weeks after the last dose of study medication, approximately 34 weeks. The safety set included all subjects who received at least 1 dose of pegcetacoplan and all subjects who were randomized to SoC. Overall Pegcetacoplan included 11 subjects who escaped from the SoC group to pegcetacoplan group.	5.6% 1/18 • Number of events 1 • Treatment-emergent adverse events were reported from the first dose of study medication and for up to 8 weeks after the last dose of study medication, approximately 34 weeks. The safety set included all subjects who received at least 1 dose of pegcetacoplan and all subjects who were randomized to SoC. Overall Pegcetacoplan included 11 subjects who escaped from the SoC group to pegcetacoplan group.
Blood and lymphatic system disorders Haemolysis	6.5% 3/46 • Number of events 3 • Treatment-emergent adverse events were reported from the first dose of study medication and for up to 8 weeks after the last dose of study medication, approximately 34 weeks. The safety set included all subjects who received at least 1 dose of pegcetacoplan and all subjects who were randomized to SoC. Overall Pegcetacoplan included 11 subjects who escaped from the SoC group to pegcetacoplan group.	0.00% 0/18 • Treatment-emergent adverse events were reported from the first dose of study medication and for up to 8 weeks after the last dose of study medication, approximately 34 weeks. The safety set included all subjects who received at least 1 dose of pegcetacoplan and all subjects who were randomized to SoC. Overall Pegcetacoplan included 11 subjects who escaped from the SoC group to pegcetacoplan group.
Blood and lymphatic system disorders Thrombocytopenia	6.5% 3/46 • Number of events 3 • Treatment-emergent adverse events were reported from the first dose of study medication and for up to 8 weeks after the last dose of study medication, approximately 34 weeks. The safety set included all subjects who received at least 1 dose of pegcetacoplan and all subjects who were randomized to SoC. Overall Pegcetacoplan included 11 subjects who escaped from the SoC group to pegcetacoplan group.	0.00% 0/18 • Treatment-emergent adverse events were reported from the first dose of study medication and for up to 8 weeks after the last dose of study medication, approximately 34 weeks. The safety set included all subjects who received at least 1 dose of pegcetacoplan and all subjects who were randomized to SoC. Overall Pegcetacoplan included 11 subjects who escaped from the SoC group to pegcetacoplan group.
Musculoskeletal and connective tissue disorders Pain in extremity	13.0% 6/46 • Number of events 8 • Treatment-emergent adverse events were reported from the first dose of study medication and for up to 8 weeks after the last dose of study medication, approximately 34 weeks. The safety set included all subjects who received at least 1 dose of pegcetacoplan and all subjects who were randomized to SoC. Overall Pegcetacoplan included 11 subjects who escaped from the SoC group to pegcetacoplan group.	0.00% 0/18 • Treatment-emergent adverse events were reported from the first dose of study medication and for up to 8 weeks after the last dose of study medication, approximately 34 weeks. The safety set included all subjects who received at least 1 dose of pegcetacoplan and all subjects who were randomized to SoC. Overall Pegcetacoplan included 11 subjects who escaped from the SoC group to pegcetacoplan group.
Musculoskeletal and connective tissue disorders Arthralgia	10.9% 5/46 • Number of events 9 • Treatment-emergent adverse events were reported from the first dose of study medication and for up to 8 weeks after the last dose of study medication, approximately 34 weeks. The safety set included all subjects who received at least 1 dose of pegcetacoplan and all subjects who were randomized to SoC. Overall Pegcetacoplan included 11 subjects who escaped from the SoC group to pegcetacoplan group.	0.00% 0/18 • Treatment-emergent adverse events were reported from the first dose of study medication and for up to 8 weeks after the last dose of study medication, approximately 34 weeks. The safety set included all subjects who received at least 1 dose of pegcetacoplan and all subjects who were randomized to SoC. Overall Pegcetacoplan included 11 subjects who escaped from the SoC group to pegcetacoplan group.
Musculoskeletal and connective tissue disorders Musculoskeletal pain	6.5% 3/46 • Number of events 5 • Treatment-emergent adverse events were reported from the first dose of study medication and for up to 8 weeks after the last dose of study medication, approximately 34 weeks. The safety set included all subjects who received at least 1 dose of pegcetacoplan and all subjects who were randomized to SoC. Overall Pegcetacoplan included 11 subjects who escaped from the SoC group to pegcetacoplan group.	0.00% 0/18 • Treatment-emergent adverse events were reported from the first dose of study medication and for up to 8 weeks after the last dose of study medication, approximately 34 weeks. The safety set included all subjects who received at least 1 dose of pegcetacoplan and all subjects who were randomized to SoC. Overall Pegcetacoplan included 11 subjects who escaped from the SoC group to pegcetacoplan group.
Musculoskeletal and connective tissue disorders Plantar fasciitis	0.00% 0/46 • Treatment-emergent adverse events were reported from the first dose of study medication and for up to 8 weeks after the last dose of study medication, approximately 34 weeks. The safety set included all subjects who received at least 1 dose of pegcetacoplan and all subjects who were randomized to SoC. Overall Pegcetacoplan included 11 subjects who escaped from the SoC group to pegcetacoplan group.	5.6% 1/18 • Number of events 1 • Treatment-emergent adverse events were reported from the first dose of study medication and for up to 8 weeks after the last dose of study medication, approximately 34 weeks. The safety set included all subjects who received at least 1 dose of pegcetacoplan and all subjects who were randomized to SoC. Overall Pegcetacoplan included 11 subjects who escaped from the SoC group to pegcetacoplan group.
Gastrointestinal disorders Abdominal pain	6.5% 3/46 • Number of events 3 • Treatment-emergent adverse events were reported from the first dose of study medication and for up to 8 weeks after the last dose of study medication, approximately 34 weeks. The safety set included all subjects who received at least 1 dose of pegcetacoplan and all subjects who were randomized to SoC. Overall Pegcetacoplan included 11 subjects who escaped from the SoC group to pegcetacoplan group.	0.00% 0/18 • Treatment-emergent adverse events were reported from the first dose of study medication and for up to 8 weeks after the last dose of study medication, approximately 34 weeks. The safety set included all subjects who received at least 1 dose of pegcetacoplan and all subjects who were randomized to SoC. Overall Pegcetacoplan included 11 subjects who escaped from the SoC group to pegcetacoplan group.
Gastrointestinal disorders Abdominal pain upper	6.5% 3/46 • Number of events 4 • Treatment-emergent adverse events were reported from the first dose of study medication and for up to 8 weeks after the last dose of study medication, approximately 34 weeks. The safety set included all subjects who received at least 1 dose of pegcetacoplan and all subjects who were randomized to SoC. Overall Pegcetacoplan included 11 subjects who escaped from the SoC group to pegcetacoplan group.	5.6% 1/18 • Number of events 1 • Treatment-emergent adverse events were reported from the first dose of study medication and for up to 8 weeks after the last dose of study medication, approximately 34 weeks. The safety set included all subjects who received at least 1 dose of pegcetacoplan and all subjects who were randomized to SoC. Overall Pegcetacoplan included 11 subjects who escaped from the SoC group to pegcetacoplan group.
Gastrointestinal disorders Dyspepsia	2.2% 1/46 • Number of events 1 • Treatment-emergent adverse events were reported from the first dose of study medication and for up to 8 weeks after the last dose of study medication, approximately 34 weeks. The safety set included all subjects who received at least 1 dose of pegcetacoplan and all subjects who were randomized to SoC. Overall Pegcetacoplan included 11 subjects who escaped from the SoC group to pegcetacoplan group.	5.6% 1/18 • Number of events 1 • Treatment-emergent adverse events were reported from the first dose of study medication and for up to 8 weeks after the last dose of study medication, approximately 34 weeks. The safety set included all subjects who received at least 1 dose of pegcetacoplan and all subjects who were randomized to SoC. Overall Pegcetacoplan included 11 subjects who escaped from the SoC group to pegcetacoplan group.
Respiratory, thoracic and mediastinal disorders Cough	6.5% 3/46 • Number of events 3 • Treatment-emergent adverse events were reported from the first dose of study medication and for up to 8 weeks after the last dose of study medication, approximately 34 weeks. The safety set included all subjects who received at least 1 dose of pegcetacoplan and all subjects who were randomized to SoC. Overall Pegcetacoplan included 11 subjects who escaped from the SoC group to pegcetacoplan group.	0.00% 0/18 • Treatment-emergent adverse events were reported from the first dose of study medication and for up to 8 weeks after the last dose of study medication, approximately 34 weeks. The safety set included all subjects who received at least 1 dose of pegcetacoplan and all subjects who were randomized to SoC. Overall Pegcetacoplan included 11 subjects who escaped from the SoC group to pegcetacoplan group.
Respiratory, thoracic and mediastinal disorders Epistaxis	6.5% 3/46 • Number of events 3 • Treatment-emergent adverse events were reported from the first dose of study medication and for up to 8 weeks after the last dose of study medication, approximately 34 weeks. The safety set included all subjects who received at least 1 dose of pegcetacoplan and all subjects who were randomized to SoC. Overall Pegcetacoplan included 11 subjects who escaped from the SoC group to pegcetacoplan group.	0.00% 0/18 • Treatment-emergent adverse events were reported from the first dose of study medication and for up to 8 weeks after the last dose of study medication, approximately 34 weeks. The safety set included all subjects who received at least 1 dose of pegcetacoplan and all subjects who were randomized to SoC. Overall Pegcetacoplan included 11 subjects who escaped from the SoC group to pegcetacoplan group.
Respiratory, thoracic and mediastinal disorders Dyspnoea	2.2% 1/46 • Number of events 1 • Treatment-emergent adverse events were reported from the first dose of study medication and for up to 8 weeks after the last dose of study medication, approximately 34 weeks. The safety set included all subjects who received at least 1 dose of pegcetacoplan and all subjects who were randomized to SoC. Overall Pegcetacoplan included 11 subjects who escaped from the SoC group to pegcetacoplan group.	5.6% 1/18 • Number of events 1 • Treatment-emergent adverse events were reported from the first dose of study medication and for up to 8 weeks after the last dose of study medication, approximately 34 weeks. The safety set included all subjects who received at least 1 dose of pegcetacoplan and all subjects who were randomized to SoC. Overall Pegcetacoplan included 11 subjects who escaped from the SoC group to pegcetacoplan group.
Respiratory, thoracic and mediastinal disorders Oropharyngeal discomfort	2.2% 1/46 • Number of events 1 • Treatment-emergent adverse events were reported from the first dose of study medication and for up to 8 weeks after the last dose of study medication, approximately 34 weeks. The safety set included all subjects who received at least 1 dose of pegcetacoplan and all subjects who were randomized to SoC. Overall Pegcetacoplan included 11 subjects who escaped from the SoC group to pegcetacoplan group.	5.6% 1/18 • Number of events 1 • Treatment-emergent adverse events were reported from the first dose of study medication and for up to 8 weeks after the last dose of study medication, approximately 34 weeks. The safety set included all subjects who received at least 1 dose of pegcetacoplan and all subjects who were randomized to SoC. Overall Pegcetacoplan included 11 subjects who escaped from the SoC group to pegcetacoplan group.
Respiratory, thoracic and mediastinal disorders Rhinitis allergic	2.2% 1/46 • Number of events 1 • Treatment-emergent adverse events were reported from the first dose of study medication and for up to 8 weeks after the last dose of study medication, approximately 34 weeks. The safety set included all subjects who received at least 1 dose of pegcetacoplan and all subjects who were randomized to SoC. Overall Pegcetacoplan included 11 subjects who escaped from the SoC group to pegcetacoplan group.	5.6% 1/18 • Number of events 1 • Treatment-emergent adverse events were reported from the first dose of study medication and for up to 8 weeks after the last dose of study medication, approximately 34 weeks. The safety set included all subjects who received at least 1 dose of pegcetacoplan and all subjects who were randomized to SoC. Overall Pegcetacoplan included 11 subjects who escaped from the SoC group to pegcetacoplan group.
Nervous system disorders Dizziness	10.9% 5/46 • Number of events 10 • Treatment-emergent adverse events were reported from the first dose of study medication and for up to 8 weeks after the last dose of study medication, approximately 34 weeks. The safety set included all subjects who received at least 1 dose of pegcetacoplan and all subjects who were randomized to SoC. Overall Pegcetacoplan included 11 subjects who escaped from the SoC group to pegcetacoplan group.	0.00% 0/18 • Treatment-emergent adverse events were reported from the first dose of study medication and for up to 8 weeks after the last dose of study medication, approximately 34 weeks. The safety set included all subjects who received at least 1 dose of pegcetacoplan and all subjects who were randomized to SoC. Overall Pegcetacoplan included 11 subjects who escaped from the SoC group to pegcetacoplan group.
Nervous system disorders Headache	8.7% 4/46 • Number of events 9 • Treatment-emergent adverse events were reported from the first dose of study medication and for up to 8 weeks after the last dose of study medication, approximately 34 weeks. The safety set included all subjects who received at least 1 dose of pegcetacoplan and all subjects who were randomized to SoC. Overall Pegcetacoplan included 11 subjects who escaped from the SoC group to pegcetacoplan group.	0.00% 0/18 • Treatment-emergent adverse events were reported from the first dose of study medication and for up to 8 weeks after the last dose of study medication, approximately 34 weeks. The safety set included all subjects who received at least 1 dose of pegcetacoplan and all subjects who were randomized to SoC. Overall Pegcetacoplan included 11 subjects who escaped from the SoC group to pegcetacoplan group.
Nervous system disorders Somnolence	6.5% 3/46 • Number of events 3 • Treatment-emergent adverse events were reported from the first dose of study medication and for up to 8 weeks after the last dose of study medication, approximately 34 weeks. The safety set included all subjects who received at least 1 dose of pegcetacoplan and all subjects who were randomized to SoC. Overall Pegcetacoplan included 11 subjects who escaped from the SoC group to pegcetacoplan group.	0.00% 0/18 • Treatment-emergent adverse events were reported from the first dose of study medication and for up to 8 weeks after the last dose of study medication, approximately 34 weeks. The safety set included all subjects who received at least 1 dose of pegcetacoplan and all subjects who were randomized to SoC. Overall Pegcetacoplan included 11 subjects who escaped from the SoC group to pegcetacoplan group.
Investigations Blood creatinine increased	6.5% 3/46 • Number of events 3 • Treatment-emergent adverse events were reported from the first dose of study medication and for up to 8 weeks after the last dose of study medication, approximately 34 weeks. The safety set included all subjects who received at least 1 dose of pegcetacoplan and all subjects who were randomized to SoC. Overall Pegcetacoplan included 11 subjects who escaped from the SoC group to pegcetacoplan group.	0.00% 0/18 • Treatment-emergent adverse events were reported from the first dose of study medication and for up to 8 weeks after the last dose of study medication, approximately 34 weeks. The safety set included all subjects who received at least 1 dose of pegcetacoplan and all subjects who were randomized to SoC. Overall Pegcetacoplan included 11 subjects who escaped from the SoC group to pegcetacoplan group.
Renal and urinary disorders Acute kidney injury	0.00% 0/46 • Treatment-emergent adverse events were reported from the first dose of study medication and for up to 8 weeks after the last dose of study medication, approximately 34 weeks. The safety set included all subjects who received at least 1 dose of pegcetacoplan and all subjects who were randomized to SoC. Overall Pegcetacoplan included 11 subjects who escaped from the SoC group to pegcetacoplan group.	5.6% 1/18 • Number of events 1 • Treatment-emergent adverse events were reported from the first dose of study medication and for up to 8 weeks after the last dose of study medication, approximately 34 weeks. The safety set included all subjects who received at least 1 dose of pegcetacoplan and all subjects who were randomized to SoC. Overall Pegcetacoplan included 11 subjects who escaped from the SoC group to pegcetacoplan group.
Injury, poisoning and procedural complications Skin abrasion	0.00% 0/46 • Treatment-emergent adverse events were reported from the first dose of study medication and for up to 8 weeks after the last dose of study medication, approximately 34 weeks. The safety set included all subjects who received at least 1 dose of pegcetacoplan and all subjects who were randomized to SoC. Overall Pegcetacoplan included 11 subjects who escaped from the SoC group to pegcetacoplan group.	5.6% 1/18 • Number of events 1 • Treatment-emergent adverse events were reported from the first dose of study medication and for up to 8 weeks after the last dose of study medication, approximately 34 weeks. The safety set included all subjects who received at least 1 dose of pegcetacoplan and all subjects who were randomized to SoC. Overall Pegcetacoplan included 11 subjects who escaped from the SoC group to pegcetacoplan group.
Psychiatric disorders Anxiety	0.00% 0/46 • Treatment-emergent adverse events were reported from the first dose of study medication and for up to 8 weeks after the last dose of study medication, approximately 34 weeks. The safety set included all subjects who received at least 1 dose of pegcetacoplan and all subjects who were randomized to SoC. Overall Pegcetacoplan included 11 subjects who escaped from the SoC group to pegcetacoplan group.	5.6% 1/18 • Number of events 1 • Treatment-emergent adverse events were reported from the first dose of study medication and for up to 8 weeks after the last dose of study medication, approximately 34 weeks. The safety set included all subjects who received at least 1 dose of pegcetacoplan and all subjects who were randomized to SoC. Overall Pegcetacoplan included 11 subjects who escaped from the SoC group to pegcetacoplan group.
Infections and infestations Urinary tract infection	0.00% 0/46 • Treatment-emergent adverse events were reported from the first dose of study medication and for up to 8 weeks after the last dose of study medication, approximately 34 weeks. The safety set included all subjects who received at least 1 dose of pegcetacoplan and all subjects who were randomized to SoC. Overall Pegcetacoplan included 11 subjects who escaped from the SoC group to pegcetacoplan group.	5.6% 1/18 • Number of events 1 • Treatment-emergent adverse events were reported from the first dose of study medication and for up to 8 weeks after the last dose of study medication, approximately 34 weeks. The safety set included all subjects who received at least 1 dose of pegcetacoplan and all subjects who were randomized to SoC. Overall Pegcetacoplan included 11 subjects who escaped from the SoC group to pegcetacoplan group.

Additional Information

Apellis Clinical Trial Information Line

Apellis Pharmaceuticals, Inc

Phone: 1-833-284-6361

Email: clinicaltrials@apellis.com

Results disclosure agreements

• Principal investigator is a sponsor employee
• Publication restrictions are in place

Restriction type: OTHER