Phase III Study Assessing the Efficacy and Safety of Pegc... | NCT05067127

NCT05067127

Status Module

Label	Type	Description	Intervention Names
Group 1: Pegcetacoplan administration	Experimental	Subcutaneous infusion of 20mL (1080 mg), twice weekly (for adults or adolescents >50kg), and the three other weight-based doses either of 10mL (540mg), 12mL (648mg), or 15mL (810mg)	Drug: Pegcetacoplan
Group 2: Placebo administration	Placebo Comparator	Subcutaneous infusion of either 10mL, 12mL, 15mL, or 20mL, twice weekly	Other: Placebo

Name	Type	Description	Arm Group Labels	Other Names
Pegcetacoplan	Drug	Complement (C3) Inhibitor	Group 1: Pegcetacoplan administration

Outcomes Module

Primary Outcomes

Measure	Description	Time Frame
Randomized Controlled Period: Change From Baseline in Log-Transformed Urine Protein-to-Creatinine Ratio (uPCR) at Week 26	Baseline uPCR value was calculated as the average of the uPCR measurements from at least 6 of the 9 first-morning spot urine (FMU) samples collected between the start of screening and Day 1, inclusive. The uPCR values used to calculate baseline included those from the samples collected on Day -2, Day -1, and before dosing on Day 1. In situations where less than 6 samples or more than 9 samples were collected, the average of all collected samples was used for baseline derivation. The difference between treatment groups using a composite contrast of equal-weighted average over Weeks 24, 25, and 26 was estimated.	Baseline (Day -70 to Day 1) to Week 26

Secondary Outcomes

Measure	Description	Time Frame
Randomized Controlled Period: Percentage of Subjects Who Achieved the Composite Renal Endpoint at Week 26	Subject who achieved a composite renal endpoint was defined as: (1) a stable or improved estimated glomerular filtration rate (eGFR) compared to baseline (<=15% reduction in eGFR), and (2) a >=50% reduction in uPCR compared to baseline. Percentages are rounded off to the hundredth decimal place.

Eligibility Module

Eligibility Criteria

Inclusion Criteria:

Aged at least 18 years; where approved, adolescents (aged 12-17 years) weighing at least 30 kg may also be enrolled.
A diagnosis of primary C3G or IC-MPGN (with or without previous renal transplant).
Evidence of active renal disease, based on one or more of the following:
1. In adults or adolescents with a baseline renal biopsy (either one collected during screening or a historic biopsy collected within 28 weeks prior to randomization), at least 2+ C3c staining on the baseline renal biopsy.
2. In adolescents not providing a baseline renal biopsy, at least one of the following:
  - Plasma sC5b-9 level above the upper limit of normal during screening
  - Serum C3 below the LLN during screening
  - Presence of an active urine sediment during screening, as evidenced by hematuria with at least 5 red blood cells (RBCs) per high-power field (HPF) and/or red blood cell casts on local or central microscopic analysis of urine.
  - Presence of C3 nephritic factor within 6 months of screening, based on central laboratory results or medical history.
No more than 50% global glomerulosclerosis or interstitial fibrosis on the baseline biopsy for adult participants or adolescent participants providing a baseline biopsy.
At least 1 g/day of proteinuria on a screening 24-hour urine collection and a uPCR of at least 1000 mg/g in at least 2 first-morning spot urine samples collected during screening.
eGFR ≥30 mL/min/1.73 m2 calculated by the Chronic Kidney Disease-Epidemiology Collaboration creatinine equation for adults or the Bedside Schwartz equation for adolescents.
Stable regimen for C3G/IC-MPGN treatment, as described below:
1. Angiotensin-converting enzyme inhibitor/, angiotensin receptor blocker, and/or sodium-glucose cotransporter-2 inhibitor therapy that is stable and optimized, in the opinion of the investigator, for at least 12 weeks prior to randomization
2. Stable doses of other medications that can affect proteinuria (eg, steroids, mycophenolate mofetil, and/or other allowed immunosuppressants that the participant is receiving for treatment of C3G or IC-MPGN) for at least 12 weeks prior to the baseline renal biopsy and randomization.
3. If a participant is on prednisone (or other systemic corticosteroid) for C3G or IC-MPGN treatment, the dosage is stable and no higher than 20 mg/day (or equivalent dosage of a corticosteroid other than prednisone) for at least 12 weeks prior to randomization.
Have received vaccinations against S pneumoniae, N meningitidis (types A, C, W, Y, and B), and H influenzae (type B) within 5 years prior to randomization or agree to receive vaccinations during screening.

Exclusion Criteria:

Previous exposure to pegcetacoplan.
C3G/IC-MPGN secondary to another condition (eg, infection, malignancy, monoclonal gammopathy, a systemic autoimmune disease such as systemic lupus erythematosus, chronic antibody-mediated rejection, or a medication), in the opinion of the investigator.
Current or prior diagnosis of human immunodeficiency virus (HIV), hepatitis B (HBV), or hepatitis C (HCV) infection or positive serology during screening that is indicative of infection with any of these viruses.
Body weight greater than 100 kg at screening.
Hypersensitivity to pegcetacoplan or to any of the excipients.
History of meningococcal disease.
Malignancy, except for the following:
1. Cured basal or squamous cell skin cancer
2. Curatively treated in situ disease
3. Malignancy-free and off treatment for ≥5 years
Severe infection (eg, requiring IV antibiotic therapy) within 14 days prior to the first dose of pegcetacoplan.
An absolute neutrophil count <1000 cells/mm3 at screening.
Use of rituximab, belimumab, or any approved or investigational anticomplement therapy other than pegcetacoplan within 5 half-lives of that product prior to the screening period.
Female participants who are pregnant or who are currently breastfeeding and are unwilling to discontinue for the duration of the study and for at least 90 days after the final dose of study drug.
Presence or suspicion of severe infection during the screening period (including but not limited to recurrent or chronic infections) that, in the opinion of the investigator, may place the participant at unacceptable risk by study participation.
Known or suspected hereditary fructose intolerance.

Facility	Status	City	State	ZIP	Country	Contacts
University of Alabama at Birmingham		Birmingham	Alabama	35294	United States
Academic Medical Research Institute

PubMed Identifier	Type	Citation	Retractions
42133432	Derived	Vivarelli M, Ariceta G, Borovitz Y, Dixon BP, Greenbaum LA, Licht C, Mastrangelo A, Melhem N, Fujita N, van de Kar NCAJ, Wallace D, Khankin E, Wang Z, Lopez-Lazaro L, Szamosi J, Nester CM. Pegcetacoplan for Adolescents with C3 Glomerulopathy or Primary Immune Complex Membranoproliferative GN: Phase 3 VALIANT Subgroup Analysis. Clin J Am Soc Nephrol. 2026 May 14. doi: 10.2215/CJN.0000001077. Online ahead of print.
41337715

Results Section

Participant Flow Module

Arm/Group Information

ID	Title	Description
FG000	Randomized Controlled Period: Pegcetacoplan	All adult subjects (regardless of weight) and adolescent subjects with body weight >=50 kilogram (kg) received pegcetacoplan 1080 milligram (mg) subcutaneous (SC) infusion twice weekly for 26 weeks in RCP. Adolescent subjects with body weight 35 to <50 kg received pegcetacoplan 648 mg for the first SC infusion and 810 mg SC infusion thereafter twice weekly for 26 weeks in RCP. Adolescent subjects with body weight 30 to <35 kg received pegcetacoplan 540 mg for the first 2 SC infusions and 648 mg SC infusion thereafter twice weekly for 26 weeks in RCP.

Baseline Characteristics Module

Outcome Measures Module

Adverse Events Module

More Info Module

Type	Includes Protocol	Includes SAP	Includes ICF	Document Label	Document Date	Document Uploaded Date	Document File Name
Prot	Yes	No	No	Study Protocol	Apr 25, 2024	Jun 26, 2025

Type

Title

Description

Population Description

Reporting Status

Anticipated Posting Date

Parameter Type

Dispersion Type

Unit of Measure

Calculate Percentage

Time Frame

Units Analyzed

Denominator Units Selected

Arm/Group Information

Denominators

Classes

Analyses

Primary

Randomized Controlled Period: Change From Baseline in Log-Transformed Urine Protein-to-Creatinine Ratio (uPCR) at Week 26

Baseline uPCR value was calculated as the average of the uPCR measurements from at least 6 of the 9 first-morning spot urine (FMU) samples collected between the start of screening and Day 1, inclusive. The uPCR values used to calculate baseline included those from the samples collected on Day -2, Day -1, and before dosing on Day 1. In situations where less than 6 samples or more than 9 samples were collected, the average of all collected samples was used for baseline derivation. The difference between treatment groups using a composite contrast of equal-weighted average over Weeks 24, 25, and 26 was estimated.

The ITT analysis set included all randomized subjects.

Posted

Least Squares Mean

95% Confidence Interval

log (uPCR)

Baseline (Day -70 to Day 1) to Week 26

ID	Title	Description
OG000	Randomized Controlled Period: Pegcetacoplan	All adult subjects (regardless of weight) and adolescent subjects with body weight >=50 kg received pegcetacoplan 1080 mg SC infusion twice weekly for 26 weeks in RCP. Adolescent subjects with body weight 35 to <50 kg received pegcetacoplan 648 mg for the first SC infusion and 810 mg SC infusion thereafter twice weekly for 26 weeks in RCP. Adolescent subjects with body weight 30 to <35 kg received pegcetacoplan 540 mg for the first 2 SC infusions and 648 mg SC infusion thereafter twice weekly for 26 weeks in RCP.
OG001	Randomized Controlled Period: Placebo	All adult subjects (regardless of weight) and adolescent subjects (with body weight: 30 to <35 kg, 35 to <50 kg, and >=50 kg) received placebo matching with pegcetacoplan SC infusion twice weekly for 26 weeks in RCP.

Units	Counts
Participants	OG00063 OG00161

Title

Denominators

Categories

Title	Measurements
	OG000-1.114(-1.380 to -0.848) OG0010.029(-0.090 to 0.148)

Group IDs	Group Description	Statistical Method	Statistical Comment	P-Value	P-Value Comment	Parameter Type	Parameter Value	Dispersion Type	Dispersion Value	Confidence Interval Sides	Confidence Interval %	CI Lower Limit	CI Upper Limit	CI Lower Limit Comment	CI Upper Limit Comment	Estimate Comment	Tested Non-Inferiority	Non-Inferiority Type	Non-Inferiority Comment	Other Analysis Description
OG000 OG001	An mixed-effect model for repeated measures (MMRM) including fixed categorical effect for treatment group, visit, disease type, baseline immunosuppressants use, stratification factors, and the visit-by-treatment group interactions as well as the continuous, fixed covariate of baseline log-transformed uPCR, was utilized to analyze the log-transformed ratio of uPCR at Week 26 compared to baseline.	MMRM		<0.0001		Difference in least squares (LS) mean	-1.143			2-Sided	95	-1.436	-0.85					Superiority

Secondary

Randomized Controlled Period: Percentage of Subjects Who Achieved the Composite Renal Endpoint at Week 26

Subject who achieved a composite renal endpoint was defined as: (1) a stable or improved estimated glomerular filtration rate (eGFR) compared to baseline (<=15% reduction in eGFR), and (2) a >=50% reduction in uPCR compared to baseline. Percentages are rounded off to the hundredth decimal place.

The ITT analysis set included all randomized subjects.

Posted

Number

percentage of subjects

Week 26

ID	Title	Description
OG000	Randomized Controlled Period: Pegcetacoplan	All adult subjects (regardless of weight) and adolescent subjects with body weight >=50 kg received pegcetacoplan 1080 mg SC infusion twice weekly for 26 weeks in RCP. Adolescent subjects with body weight 35 to <50 kg received pegcetacoplan 648 mg for the first SC infusion and 810 mg SC infusion thereafter twice weekly for 26 weeks in RCP. Adolescent subjects with body weight 30 to <35 kg received pegcetacoplan 540 mg for the first 2 SC infusions and 648 mg SC infusion thereafter twice weekly for 26 weeks in RCP.
OG001	Randomized Controlled Period: Placebo	All adult subjects (regardless of weight) and adolescent subjects (with body weight: 30 to <35 kg, 35 to <50 kg, and >=50 kg) received placebo matching with pegcetacoplan SC infusion twice weekly for 26 weeks in RCP.

Secondary

Randomized Controlled Period: Percentage of Subjects With a Reduction of At Least 50% From Baseline in Urine Protein-to-Creatinine Ratio at Week 26

Baseline uPCR value was calculated as the average of the uPCR measurements from at least 6 of the 9 FMU samples collected between the start of screening and Day 1, inclusive. The uPCR values used to calculate baseline included those from the samples collected on Day -2, Day -1, and before dosing on Day 1. In situations where less than 6 samples or more than 9 samples were collected, the average of all collected samples was used for baseline derivation. Percentages are rounded off to the hundredth decimal place.

The ITT analysis set included all randomized subjects.

Posted

Number

percentage of subjects

Baseline (Day -70 to Day 1) and Week 26

ID	Title	Description
OG000	Randomized Controlled Period: Pegcetacoplan	All adult subjects (regardless of weight) and adolescent subjects with body weight >=50 kg received pegcetacoplan 1080 mg SC infusion twice weekly for 26 weeks in RCP. Adolescent subjects with body weight 35 to <50 kg received pegcetacoplan 648 mg for the first SC infusion and 810 mg SC infusion thereafter twice weekly for 26 weeks in RCP. Adolescent subjects with body weight 30 to <35 kg received pegcetacoplan 540 mg for the first 2 SC infusions and 648 mg SC infusion thereafter twice weekly for 26 weeks in RCP.
OG001	Randomized Controlled Period: Placebo	All adult subjects (regardless of weight) and adolescent subjects (with body weight: 30 to <35 kg, 35 to <50 kg, and >=50 kg) received placebo matching with pegcetacoplan SC infusion twice weekly for 26 weeks in RCP.

Secondary

Randomized Controlled Period: Change From Baseline in the C3 Glomerulopathy (C3G) Histologic Index Activity Score at Week 26

The C3G histologic index used to assess disease activity and chronicity in C3G. The C3G total activity score ranges from 0 (worse) to 21 (best). Higher scores indicate better outcome. Baseline was defined as the most recent non-missing measurement prior to taking the first dose of study drug.

The ITT analysis set included all randomized subjects. Since adolescents subjects were not required to provide post-screening biopsies, this endpoint was analyzed based on adult subjects only.

Posted

Least Squares Mean

95% Confidence Interval

units on a scale

Baseline (Day 1) and Week 26

ID	Title	Description
OG000	Randomized Controlled Period: Pegcetacoplan	All adult subjects (regardless of weight) and adolescent subjects with body weight >=50 kg received pegcetacoplan 1080 mg SC infusion twice weekly for 26 weeks in RCP. Adolescent subjects with body weight 35 to <50 kg received pegcetacoplan 648 mg for the first SC infusion and 810 mg SC infusion thereafter twice weekly for 26 weeks in RCP. Adolescent subjects with body weight 30 to <35 kg received pegcetacoplan 540 mg for the first 2 SC infusions and 648 mg SC infusion thereafter twice weekly for 26 weeks in RCP.
OG001	Randomized Controlled Period: Placebo	All adult subjects (regardless of weight) and adolescent subjects (with body weight: 30 to <35 kg, 35 to <50 kg, and >=50 kg) received placebo matching with pegcetacoplan SC infusion twice weekly for 26 weeks in RCP.

Secondary

Randomized Controlled Period: Percentage of Subjects Who Showed Decrease in C3c Staining on Renal Biopsy From Baseline at Week 26

Subject who showed decrease in C3c staining was defined as decrease of at least 2 orders of magnitude of intensity from baseline. Percentages are rounded off to the hundredth decimal place.

The ITT analysis set included all randomized subjects. Since adolescents subjects were not required to provide post-screening biopsies, this endpoint was analyzed based on adult subjects only.

Posted

Number

percentage of subjects

Baseline (Day 1) and Week 26

ID	Title	Description
OG000	Randomized Controlled Period: Pegcetacoplan	All adult subjects (regardless of weight) and adolescent subjects with body weight >=50 kg received pegcetacoplan 1080 mg SC infusion twice weekly for 26 weeks in RCP. Adolescent subjects with body weight 35 to <50 kg received pegcetacoplan 648 mg for the first SC infusion and 810 mg SC infusion thereafter twice weekly for 26 weeks in RCP. Adolescent subjects with body weight 30 to <35 kg received pegcetacoplan 540 mg for the first 2 SC infusions and 648 mg SC infusion thereafter twice weekly for 26 weeks in RCP.
OG001	Randomized Controlled Period: Placebo	All adult subjects (regardless of weight) and adolescent subjects (with body weight: 30 to <35 kg, 35 to <50 kg, and >=50 kg) received placebo matching with pegcetacoplan SC infusion twice weekly for 26 weeks in RCP.

Secondary

Randomized Controlled Period: Change From Baseline in Estimated Glomerular Filtration Rate at Week 26

Serum samples were collected to determine the eGFR, calculated by using chronic kidney disease-epidemiology collaboration (CKD-EPI) creatinine equation for adults and the Bedside Schwartz equation for adolescents. Baseline eGFR value was calculated using the last non-missing assessment prior to first dose of study drug.

The ITT analysis set included all randomized subjects.

Posted

Least Squares Mean

95% Confidence Interval

milliliter (mL)/minute/1.73 m^2

Baseline (Day 1) and Week 26

ID	Title	Description
OG000	Randomized Controlled Period: Pegcetacoplan	All adult subjects (regardless of weight) and adolescent subjects with body weight >=50 kg received pegcetacoplan 1080 mg SC infusion twice weekly for 26 weeks in RCP. Adolescent subjects with body weight 35 to <50 kg received pegcetacoplan 648 mg for the first SC infusion and 810 mg SC infusion thereafter twice weekly for 26 weeks in RCP. Adolescent subjects with body weight 30 to <35 kg received pegcetacoplan 540 mg for the first 2 SC infusions and 648 mg SC infusion thereafter twice weekly for 26 weeks in RCP.
OG001	Randomized Controlled Period: Placebo	All adult subjects (regardless of weight) and adolescent subjects (with body weight: 30 to <35 kg, 35 to <50 kg, and >=50 kg) received placebo matching with pegcetacoplan SC infusion twice weekly for 26 weeks in RCP.

Secondary

Randomized Controlled Period: Percentage of Subjects Who Achieved Proteinuria <1 Gram (g)/Day at Week 24

Urine samples were collected to determine the proteinuria. Percentage of subjects who achieved proteinuria <1 g/day was assessed by 24-hour urine protein. Percentages are rounded off to the hundredth decimal place.

The ITT analysis set included all randomized subjects.

Posted

Number

percentage of subjects

Week 24

ID	Title	Description
OG000	Randomized Controlled Period: Pegcetacoplan	All adult subjects (regardless of weight) and adolescent subjects with body weight >=50 kg received pegcetacoplan 1080 mg SC infusion twice weekly for 26 weeks in RCP. Adolescent subjects with body weight 35 to <50 kg received pegcetacoplan 648 mg for the first SC infusion and 810 mg SC infusion thereafter twice weekly for 26 weeks in RCP. Adolescent subjects with body weight 30 to <35 kg received pegcetacoplan 540 mg for the first 2 SC infusions and 648 mg SC infusion thereafter twice weekly for 26 weeks in RCP.
OG001	Randomized Controlled Period: Placebo	All adult subjects (regardless of weight) and adolescent subjects (with body weight: 30 to <35 kg, 35 to <50 kg, and >=50 kg) received placebo matching with pegcetacoplan SC infusion twice weekly for 26 weeks in RCP.

Secondary

Randomized Controlled Period: Percentage of Subjects With Normalization of Serum Albumin Levels at Week 26

Baseline serum albumin value was calculated as the average of up to 2 serum albumin measurements preceding and including Day 1. Week 26 serum albumin values was calculated as the average of up to 2 serum albumin measurements preceding and including Week 26, no earlier than Week 20 measurement. Percentages are rounded off to the hundredth decimal place.

The ITT analysis set included all randomized subjects. Only subjects with serum albumin levels below lower limit of normal (LLN) at baseline are analyzed.

Posted

Number

percentage of subjects

Week 26

ID	Title	Description
OG000	Randomized Controlled Period: Pegcetacoplan	All adult subjects (regardless of weight) and adolescent subjects with body weight >=50 kg received pegcetacoplan 1080 mg SC infusion twice weekly for 26 weeks in RCP. Adolescent subjects with body weight 35 to <50 kg received pegcetacoplan 648 mg for the first SC infusion and 810 mg SC infusion thereafter twice weekly for 26 weeks in RCP. Adolescent subjects with body weight 30 to <35 kg received pegcetacoplan 540 mg for the first 2 SC infusions and 648 mg SC infusion thereafter twice weekly for 26 weeks in RCP.
OG001	Randomized Controlled Period: Placebo	All adult subjects (regardless of weight) and adolescent subjects (with body weight: 30 to <35 kg, 35 to <50 kg, and >=50 kg) received placebo matching with pegcetacoplan SC infusion twice weekly for 26 weeks in RCP.

Secondary

Randomized Controlled Period: Percentage of Subjects With Serum C3 Levels Above the Lower Limit of Normal at Week 26

Baseline serum C3 value was calculated as the average of up to 2 serum C3 measurements preceding and including Day 1. Week 26 serum C3 value was calculated as the average of up to 2 serum C3 measurements preceding and including Week 26, no earlier than Week 20 measurement. Percentages are rounded off to the hundredth decimal place.

The ITT analysis set included all randomized subjects. Only subjects with serum C3 levels below LLN at baseline are analyzed.

Posted

Number

percentage of subjects

Week 26

ID	Title	Description
OG000	Randomized Controlled Period: Pegcetacoplan	All adult subjects (regardless of weight) and adolescent subjects with body weight >=50 kg received pegcetacoplan 1080 mg SC infusion twice weekly for 26 weeks in RCP. Adolescent subjects with body weight 35 to <50 kg received pegcetacoplan 648 mg for the first SC infusion and 810 mg SC infusion thereafter twice weekly for 26 weeks in RCP. Adolescent subjects with body weight 30 to <35 kg received pegcetacoplan 540 mg for the first 2 SC infusions and 648 mg SC infusion thereafter twice weekly for 26 weeks in RCP.
OG001	Randomized Controlled Period: Placebo	All adult subjects (regardless of weight) and adolescent subjects (with body weight: 30 to <35 kg, 35 to <50 kg, and >=50 kg) received placebo matching with pegcetacoplan SC infusion twice weekly for 26 weeks in RCP.

Secondary

Randomized Controlled Period: Change From Baseline in the Functional Assessment of Chronic Illness Therapy-Fatigue (FACIT-Fatigue) Score at Week 26

The FACIT-Fatigue scale was a 13-item Likert scaled instrument that was self-administered by subjects. Subjects were presented with 13 statements and asked to indicate their responses as it applied to the past 7 days. The 5 possible responses were "not at all" (0), "a little bit" (1), "somewhat" (2), "quite a bit" (3) and "very much" (4). With 13 statements the total score has a range of 0 (worse health-related quality of life) to 52 (best health-related quality of life). Higher scores indicate better quality of life. Baseline was defined as the most recent non-missing measurement prior to taking the first dose of study drug.

The ITT analysis set included all randomized subjects.

Posted

Least Squares Mean

95% Confidence Interval

units on a scale

Baseline (Day 1) and Week 26

ID	Title	Description
OG000	Randomized Controlled Period: Pegcetacoplan	All adult subjects (regardless of weight) and adolescent subjects with body weight >=50 kg received pegcetacoplan 1080 mg SC infusion twice weekly for 26 weeks in RCP. Adolescent subjects with body weight 35 to <50 kg received pegcetacoplan 648 mg for the first SC infusion and 810 mg SC infusion thereafter twice weekly for 26 weeks in RCP. Adolescent subjects with body weight 30 to <35 kg received pegcetacoplan 540 mg for the first 2 SC infusions and 648 mg SC infusion thereafter twice weekly for 26 weeks in RCP.
OG001	Randomized Controlled Period: Placebo

Secondary

Randomized Controlled Period: Change From Baseline in the Kidney Disease Quality of Life (KDQOL) Score at Week 26

The KDQOL score was constructed as the KDQOL-36 Summary Score (KSS) by averaging the 24 items from Burden of Kidney Disease, Symptoms and Problems of Kidney Disease, and Effects of Kidney Disease on scale ranging from 0 (worse health-related quality of life) to 100 (best health-related quality of life). Higher scores indicate better quality of life. Baseline was defined as the most recent non-missing measurement prior to taking the first dose of study drug.

The ITT analysis set included all randomized subjects.

Posted

Least Squares Mean

95% Confidence Interval

units on a scale

Baseline (Day 1) and Week 26

ID	Title	Description
OG000	Randomized Controlled Period: Pegcetacoplan	All adult subjects (regardless of weight) and adolescent subjects with body weight >=50 kg received pegcetacoplan 1080 mg SC infusion twice weekly for 26 weeks in RCP. Adolescent subjects with body weight 35 to <50 kg received pegcetacoplan 648 mg for the first SC infusion and 810 mg SC infusion thereafter twice weekly for 26 weeks in RCP. Adolescent subjects with body weight 30 to <35 kg received pegcetacoplan 540 mg for the first 2 SC infusions and 648 mg SC infusion thereafter twice weekly for 26 weeks in RCP.
OG001	Randomized Controlled Period: Placebo	All adult subjects (regardless of weight) and adolescent subjects (with body weight: 30 to <35 kg, 35 to <50 kg, and >=50 kg) received placebo matching with pegcetacoplan SC infusion twice weekly for 26 weeks in RCP.

Arm/Groups

ID	Title	Description	Deaths (Affected)	Deaths (At Risk)	Serious Events (Affected)	Serious Events (At Risk)	Other Events (Affected)	Other Events (At Risk)
EG000	Randomized Controlled Period: Pegcetacoplan	All adult subjects (regardless of weight) and adolescent subjects with body weight >=50 kg received pegcetacoplan 1080 mg SC infusion twice weekly for 26 weeks in RCP. Adolescent subjects with body weight 35 to <50 kg received pegcetacoplan 648 mg for the first SC infusion and 810 mg SC infusion thereafter twice weekly for 26 weeks in RCP. Adolescent subjects with body weight 30 to <35 kg received pegcetacoplan 540 mg for the first 2 SC infusions and 648 mg SC infusion thereafter twice weekly for 26 weeks in RCP.	1	63	6	63	53	63
EG001	Randomized Controlled Period: Placebo	All adult subjects (regardless of weight) and adolescent subjects (with body weight: 30 to <35 kg, 35 to <50 kg, and >=50 kg) received placebo matching with pegcetacoplan SC infusion twice weekly for 26 weeks in RCP.	0	61	6	61	56	61
EG002	Open-Label Period: Pegcetacoplan to Pegcetacoplan	All eligible adult subjects (regardless of weight) and adolescent subjects with body weight >=50 kg who received pegcetacoplan in RCP continued to receive pegcetacoplan 1080 mg SC infusion twice weekly for 26 weeks in OLP. Eligible adolescent subjects with body weight 35 to <50 kg who received pegcetacoplan in RCP continued to receive pegcetacoplan 810 mg SC infusion twice weekly for 26 weeks in OLP. Eligible adolescent subjects with body weight 30 to <35 kg who received pegcetacoplan in RCP continued to receive pegcetacoplan 648 mg SC infusion twice weekly for 26 weeks in OLP.	0	61	6	61	47	61
EG003	Open-Label Period: Placebo to Pegcetacoplan	All eligible adult subjects (regardless of weight) and adolescent subjects with body weight >=50 kg who received placebo in RCP entered OLP to receive pegcetacoplan 1080 mg SC infusion twice weekly for 26 weeks in OLP. Eligible adolescent subjects with body weight 35 to <50 kg who received placebo in RCP entered OLP to receive pegcetacoplan 810 mg SC infusion twice weekly for 26 weeks in OLP. Eligible adolescent subjects with body weight 30 to <35 kg who received placebo in RCP entered OLP to receive pegcetacoplan 648 mg SC infusion twice weekly for 26 weeks in OLP.	0	57	4	57	42	57

Serious Adverse Events

Term	Organ System	Source Vocabulary	Assessment Type	Statistical Information
Abdominal pain	Gastrointestinal disorders	MedDRA 26.0	Systematic Assessment	EG0000 events0 affected63 at risk EG0010 events0 affected61 at risk EG0021 events1 affected61 at risk EG0030 events0 affected57 at risk
Diarrhoea	Gastrointestinal disorders	MedDRA 26.0	Systematic Assessment	EG0000 events0 affected63 at risk EG0010 events0 affected61 at risk EG0021 events1 affected61 at risk EG003
Vomiting	Gastrointestinal disorders	MedDRA 26.0	Systematic Assessment	EG0000 events0 affected63 at risk EG0012 events1 affected61 at risk EG0020 events0 affected61 at risk EG003
Pyrexia	General disorders	MedDRA 26.0	Systematic Assessment	EG0001 events1 affected63 at risk EG0010 events0 affected61 at risk EG0020 events0 affected61 at risk EG003
COVID-19 pneumonia	Infections and infestations	MedDRA 26.0	Systematic Assessment	EG0001 events1 affected63 at risk EG0010 events0 affected61 at risk EG0020 events0 affected61 at risk EG003
Gastroenteritis	Infections and infestations	MedDRA 26.0	Systematic Assessment	EG0000 events0 affected63 at risk EG0010 events0 affected61 at risk EG0020 events0 affected61 at risk EG003
Herpes zoster meningoencephalitis	Infections and infestations	MedDRA 26.0	Systematic Assessment	EG0000 events0 affected63 at risk EG0010 events0 affected61 at risk EG0020 events0 affected61 at risk EG003
Influenza	Infections and infestations	MedDRA 26.0	Systematic Assessment	EG0001 events1 affected63 at risk EG0010 events0 affected61 at risk EG0020 events0 affected61 at risk EG003
Pneumonia	Infections and infestations	MedDRA 26.0	Systematic Assessment	EG0001 events1 affected63 at risk EG0010 events0 affected61 at risk EG0020 events0 affected61 at risk EG003
Pneumonia pneumococcal	Infections and infestations	MedDRA 26.0	Systematic Assessment	EG0000 events0 affected63 at risk EG0010 events0 affected61 at risk EG0021 events1 affected61 at risk EG003
Shunt infection	Infections and infestations	MedDRA 26.0	Systematic Assessment	EG0000 events0 affected63 at risk EG0010 events0 affected61 at risk EG0020 events0 affected61 at risk EG003
Viral infection	Infections and infestations	MedDRA 26.0	Systematic Assessment	EG0000 events0 affected63 at risk EG0011 events1 affected61 at risk EG0021 events1 affected61 at risk EG003
Maternal exposure during pregnancy	Injury, poisoning and procedural complications	MedDRA 26.0	Systematic Assessment	EG0000 events0 affected63 at risk EG0011 events1 affected61 at risk EG0020 events0 affected61 at risk EG003
Post procedural haematoma	Injury, poisoning and procedural complications	MedDRA 26.0	Systematic Assessment	EG0000 events0 affected63 at risk EG0010 events0 affected61 at risk EG0021 events1 affected61 at risk EG003
Shunt malfunction	Injury, poisoning and procedural complications	MedDRA 26.0	Systematic Assessment	EG0000 events0 affected63 at risk EG0010 events0 affected61 at risk EG0020 events0 affected61 at risk EG003
Shunt thrombosis	Injury, poisoning and procedural complications	MedDRA 26.0	Systematic Assessment	EG0000 events0 affected63 at risk EG0010 events0 affected61 at risk EG0020 events0 affected61 at risk EG003
Blood creatinine increased	Investigations	MedDRA 26.0	Systematic Assessment	EG0000 events0 affected63 at risk EG0011 events1 affected61 at risk EG0020 events0 affected61 at risk EG003
Dehydration	Metabolism and nutrition disorders	MedDRA 26.0	Systematic Assessment	EG0000 events0 affected63 at risk EG0010 events0 affected61 at risk EG0021 events1 affected61 at risk EG003
Abortion spontaneous	Pregnancy, puerperium and perinatal conditions	MedDRA 26.0	Systematic Assessment	EG0000 events0 affected63 at risk EG0011 events1 affected61 at risk EG0020 events0 affected61 at risk EG003
Acute kidney injury	Renal and urinary disorders	MedDRA 26.0	Systematic Assessment	EG0001 events1 affected63 at risk EG0012 events2 affected61 at risk EG0022 events2 affected61 at risk EG003
End stage renal disease	Renal and urinary disorders	MedDRA 26.0	Systematic Assessment	EG0000 events0 affected63 at risk EG0010 events0 affected61 at risk EG0021 events1 affected61 at risk EG003
Nephrotic syndrome	Renal and urinary disorders	MedDRA 26.0	Systematic Assessment	EG0002 events1 affected63 at risk EG0010 events0 affected61 at risk EG0021 events1 affected61 at risk EG003
Proteinuria	Renal and urinary disorders	MedDRA 26.0	Systematic Assessment	EG0000 events0 affected63 at risk EG0011 events1 affected61 at risk EG0020 events0 affected61 at risk EG003
Renal failure	Renal and urinary disorders	MedDRA 26.0	Systematic Assessment	EG0000 events0 affected63 at risk EG0010 events0 affected61 at risk EG0021 events1 affected61 at risk EG003
Renal impairment	Renal and urinary disorders	MedDRA 26.0	Systematic Assessment	EG0000 events0 affected63 at risk EG0010 events0 affected61 at risk EG0021 events1 affected61 at risk EG003
Tubulointerstitial nephritis	Renal and urinary disorders	MedDRA 26.0	Systematic Assessment	EG0000 events0 affected63 at risk EG0011 events1 affected61 at risk EG0020 events0 affected61 at risk EG003
Respiratory failure	Respiratory, thoracic and mediastinal disorders	MedDRA 26.0	Systematic Assessment	EG0001 events1 affected63 at risk EG0010 events0 affected61 at risk EG0020 events0 affected61 at risk EG003
Hypertension	Vascular disorders	MedDRA 26.0	Systematic Assessment	EG0001 events1 affected63 at risk EG0010 events0 affected61 at risk EG0020 events0 affected61 at risk EG003
Hypertensive urgency	Vascular disorders	MedDRA 26.0	Systematic Assessment	EG0000 events0 affected63 at risk EG0010 events0 affected61 at risk EG0021 events1 affected61 at risk EG003

Other Adverse Events

Term	Organ System	Source Vocabulary	Assessment Type	Statistical Information
Anaemia	Blood and lymphatic system disorders	MedDRA 26.0	Systematic Assessment	EG0000 events0 affected63 at risk EG0014 events4 affected61 at risk EG0025 events5 affected61 at risk EG0034 events4 affected57 at risk
Abdominal pain	Gastrointestinal disorders	MedDRA 26.0	Systematic Assessment	EG0006 events4 affected63 at risk EG0015 events4 affected61 at risk EG0022 events2 affected61 at risk EG003
Diarrhoea	Gastrointestinal disorders	MedDRA 26.0	Systematic Assessment	EG0003 events2 affected63 at risk EG0018 events7 affected61 at risk EG0025 events4 affected61 at risk EG003
Nausea	Gastrointestinal disorders	MedDRA 26.0	Systematic Assessment	EG0008 events6 affected63 at risk EG0014 events4 affected61 at risk EG00210 events6 affected61 at risk EG003
Vomiting	Gastrointestinal disorders	MedDRA 26.0	Systematic Assessment	EG0007 events5 affected63 at risk EG00113 events9 affected61 at risk EG00210 events7 affected61 at risk EG003
Asthenia	General disorders	MedDRA 26.0	Systematic Assessment	EG0000 events0 affected63 at risk EG0014 events4 affected61 at risk EG0020 events0 affected61 at risk EG003
Fatigue	General disorders	MedDRA 26.0	Systematic Assessment	EG0004 events4 affected63 at risk EG0011 events1 affected61 at risk EG0022 events2 affected61 at risk EG003
Infusion site erythema	General disorders	MedDRA 26.0	Systematic Assessment	EG0004 events4 affected63 at risk EG0011 events1 affected61 at risk EG0020 events0 affected61 at risk EG003
Infusion site pain	General disorders	MedDRA 26.0	Systematic Assessment	EG0001 events1 affected63 at risk EG0015 events4 affected61 at risk EG0020 events0 affected61 at risk EG003
Infusion site swelling	General disorders	MedDRA 26.0	Systematic Assessment	EG0000 events0 affected63 at risk EG0012 events1 affected61 at risk EG0021 events1 affected61 at risk EG003
Injection site pain	General disorders	MedDRA 26.0	Systematic Assessment	EG0002 events2 affected63 at risk EG00111 events5 affected61 at risk EG0020 events0 affected61 at risk EG003
Injection site swelling	General disorders	MedDRA 26.0	Systematic Assessment	EG00033 events2 affected63 at risk EG00148 events7 affected61 at risk EG00244 events1 affected61 at risk EG003
Oedema	General disorders	MedDRA 26.0	Systematic Assessment	EG0004 events3 affected63 at risk EG0016 events5 affected61 at risk EG0021 events1 affected61 at risk EG003
Pyrexia	General disorders	MedDRA 26.0	Systematic Assessment	EG00014 events12 affected63 at risk EG0017 events7 affected61 at risk EG0027 events5 affected61 at risk EG003
COVID-19	Infections and infestations	MedDRA 26.0	Systematic Assessment	EG0002 events2 affected63 at risk EG0014 events4 affected61 at risk EG0023 events3 affected61 at risk EG003
Influenza	Infections and infestations	MedDRA 26.0	Systematic Assessment	EG0006 events6 affected63 at risk EG0014 events3 affected61 at risk EG0024 events4 affected61 at risk EG003
Nasopharyngitis	Infections and infestations	MedDRA 26.0	Systematic Assessment	EG00015 events11 affected63 at risk EG0018 events7 affected61 at risk EG0029 events7 affected61 at risk EG003
Upper respiratory tract infection	Infections and infestations	MedDRA 26.0	Systematic Assessment	EG0004 events4 affected63 at risk EG0015 events5 affected61 at risk EG0025 events4 affected61 at risk EG003
Contusion	Injury, poisoning and procedural complications	MedDRA 26.0	Systematic Assessment	EG0004 events4 affected63 at risk EG0012 events1 affected61 at risk EG0020 events0 affected61 at risk EG003
Dizziness	Nervous system disorders	MedDRA 26.0	Systematic Assessment	EG0000 events0 affected63 at risk EG0016 events6 affected61 at risk EG0023 events3 affected61 at risk EG003
Headache	Nervous system disorders	MedDRA 26.0	Systematic Assessment	EG00020 events8 affected63 at risk EG00114 events11 affected61 at risk EG0027 events4 affected61 at risk EG003
Proteinuria	Renal and urinary disorders	MedDRA 26.0	Systematic Assessment	EG0000 events0 affected63 at risk EG0014 events4 affected61 at risk EG0021 events1 affected61 at risk EG003
Cough	Respiratory, thoracic and mediastinal disorders	MedDRA 26.0	Systematic Assessment	EG0007 events6 affected63 at risk EG0011 events1 affected61 at risk EG0025 events4 affected61 at risk EG003
Acne	Skin and subcutaneous tissue disorders	MedDRA 26.0	Systematic Assessment	EG0000 events0 affected63 at risk EG0014 events4 affected61 at risk EG0020 events0 affected61 at risk EG003
Pruritus	Skin and subcutaneous tissue disorders	MedDRA 26.0	Systematic Assessment	EG0002 events2 affected63 at risk EG0010 events0 affected61 at risk EG0024 events4 affected61 at risk EG003
Hypertension	Vascular disorders	MedDRA 26.0	Systematic Assessment	EG0005 events3 affected63 at risk EG0015 events5 affected61 at risk EG0026 events6 affected61 at risk EG003
Hypotension	Vascular disorders	MedDRA 26.0	Systematic Assessment	EG0002 events2 affected63 at risk EG0010 events0 affected61 at risk EG0020 events0 affected61 at risk EG003

ID	Term
D005921	Glomerulonephritis
D009393	Nephritis
D007674	Kidney Diseases
D014570	Urologic Diseases
D052776	Female Urogenital Diseases
D005261	Female Urogenital Diseases and Pregnancy Complications
D000091642	Urogenital Diseases
D052801	Male Urogenital Diseases
D007154	Immune System Diseases

ID	Title	Description
BG000	Randomized Controlled Period: Pegcetacoplan	All adult subjects (regardless of weight) and adolescent subjects with body weight >=50 kg received pegcetacoplan 1080 mg SC infusion twice weekly for 26 weeks in RCP. Adolescent subjects with body weight 35 to <50 kg received pegcetacoplan 648 mg for the first SC infusion and 810 mg SC infusion thereafter twice weekly for 26 weeks in RCP. Adolescent subjects with body weight 30 to <35 kg received pegcetacoplan 540 mg for the first 2 SC infusions and 648 mg SC infusion thereafter twice weekly for 26 weeks in RCP.
BG001	Randomized Controlled Period: Placebo	All adult subjects (regardless of weight) and adolescent subjects (with body weight: 30 to <35 kg, 35 to <50 kg, and >=50 kg) received placebo matching with pegcetacoplan SC infusion twice weekly for 26 weeks in RCP.
BG002	Total	Total of all reporting groups