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CompletedNCT05067127VALIANTUpdated Jan 29, 2026Results posted

Phase III Study Assessing the Efficacy and Safety of Pegcetacoplan in Patients With C3 Glomerulopathy or Immune-Complex Membranoproliferative Glomerulonephritis

A Phase 3 interventional study of Pegcetacoplan and Placebo in C3G, IC-MPGN and C3 Glomerulopathy, sponsored by Apellis Pharmaceuticals, Inc.. Completed at 125 sites in 19 countries. Open to participants aged 12 Years and older. Per ClinicalTrials.gov, last updated 2026-01-29.

Sponsored by Apellis Pharmaceuticals, Inc. · Phase 3, Interventional, and Treatment

Phase
Phase 3
Study type
Interventional
Enrollment
124
Allocation
Randomized
Ages
12 Years and older
Sex
All
01

Study summary

This is a Phase 3 study to assess the efficacy and safety of twice-weekly subcutaneous (SC) doses of pegcetacoplan compared to placebo in patients with C3 glomerulopathy (C3G) or immune-complex membranoproliferative glomerulonephritis (IC-MPGN) on the basis of a reduction in proteinuria.

02

Conditions studied

  • C3G
  • IC-MPGN
  • C3 Glomerulopathy
  • C3 Glomerulonephritis
  • Complement 3 Glomerulopathy
  • Complement 3 Glomerulopathy (C3G)
  • Complement 3 Glomerulonephritis
  • Dense Deposit Disease
  • DDD
  • Membranoproliferative Glomerulonephritis
  • Membranoproliferative Glomerulonephritis (MPGN)
  • Immune Complex Membranoproliferative Glomerulonephritis (IC-MPGN)
03

In context

Glomerulonephritis, Membranoproliferative

28 studies on the registry are indexed under Glomerulonephritis, Membranoproliferative; 8 are open to participants now.

This study's enrollment of 124 is above the median of 22 across 22 interventional studies indexed under Glomerulonephritis, Membranoproliferative.

Browse Glomerulonephritis, Membranoproliferative studies →

Lead sponsor

Apellis Pharmaceuticals, Inc. is the lead sponsor of 28 studies on the registry; 3 are open to participants now.

Of its 18 completed or terminated interventional studies of FDA-regulated products, 14 (78%) have results posted.

Counted across the registry records on this site, refreshed daily.

04

Who can participate

Ages eligible
12 Years and older
Sexes eligible
All
Accepts healthy volunteers
No

Inclusion criteria

  1. Aged at least 18 years; where approved, adolescents (aged 12-17 years) weighing at least 30 kg may also be enrolled.
  2. A diagnosis of primary C3G or IC-MPGN (with or without previous renal transplant).

  3. 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.
  4. No more than 50% global glomerulosclerosis or interstitial fibrosis on the baseline biopsy for adult participants or adolescent participants providing a baseline biopsy.
  5. 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.
  6. 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.

  7. 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.
  8. 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

Exclusion Criteria:

  1. Previous exposure to pegcetacoplan.
  2. 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.
  3. 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.
  4. Body weight greater than 100 kg at screening.
  5. Hypersensitivity to pegcetacoplan or to any of the excipients.
  6. History of meningococcal disease.

  7. 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
  8. Severe infection (eg, requiring IV antibiotic therapy) within 14 days prior to the first dose of pegcetacoplan.
  9. An absolute neutrophil count \<1000 cells/mm3 at screening.
  10. 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.
  11. 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.
  12. 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.
  13. Known or suspected hereditary fructose intolerance.
05

Study design

Phase
Phase 3
Primary purpose
Treatment
Allocation
Randomized
Intervention model
Parallel assignment
Masking
Triple (Participant, Care provider, Investigator)
Enrollment
124 participants (actual)

Study arms

  • Experimental
    Group 1: Pegcetacoplan administration

    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

  • Placebo comparator
    Group 2: Placebo administration

    Subcutaneous infusion of either 10mL, 12mL, 15mL, or 20mL, twice weekly

    Other: Placebo

Interventions

  • DrugPegcetacoplan

    Complement (C3) Inhibitor

  • OtherPlacebo

    Sterile solution of equal volume to active arm

06

What researchers measure

Primary outcomes

  1. 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.

    Time frame: Baseline (Day -70 to Day 1) to Week 26

Secondary outcomes

  1. 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.

    Time frame: Week 26

  2. 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.

    Time frame: Baseline (Day -70 to Day 1) and Week 26

  3. 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.

    Time frame: Baseline (Day 1) and Week 26

  4. 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.

    Time frame: Baseline (Day 1) and Week 26

  5. 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.

    Time frame: Baseline (Day 1) and Week 26

  6. 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.

    Time frame: Week 24

  7. 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.

    Time frame: Week 26

  8. 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.

    Time frame: Week 26

  9. 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.

    Time frame: Baseline (Day 1) and Week 26

  10. 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.

    Time frame: Baseline (Day 1) and Week 26

07

Results

Posted Aug 6, 2025

Participant flow

This Phase 3 randomized, placebo-controlled, double-blinded study was conducted in subjects with complement 3 glomerulopathy (C3G) or primary immune-complex membranoproliferative glomerulonephritis (IC-MPGN) at 122 sites.

Randomized Controlled Period (26 weeks)
Participant flow — Randomized Controlled Period (26 weeks)
MilestoneRandomized Controlled Period: PegcetacoplanRandomized Controlled Period: PlaceboOpen-Label Period: Pegcetacoplan to PegcetacoplanOpen-Label Period: Placebo to Pegcetacoplan
Started636100
Completed615700
Not completed2400
Withdrew: Lost to follow-up0100
Withdrew: Withdrawal by subject0200
Withdrew: Investigator or medical monitor decision1000
Withdrew: Pregnancy0100
Withdrew: Death1000
Open-Label Period (26 weeks)
Participant flow — Open-Label Period (26 weeks)
MilestoneRandomized Controlled Period: PegcetacoplanRandomized Controlled Period: PlaceboOpen-Label Period: Pegcetacoplan to PegcetacoplanOpen-Label Period: Placebo to Pegcetacoplan
Started006157
Completed005955
Not completed0022
Withdrew: Withdrawal by subject0001
Withdrew: Investigator or medical monitor decision0021

Outcome measures

PrimaryRandomized 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.

Time frame:
Baseline (Day -70 to Day 1) to Week 26
Reported as:
Least squares mean · log (uPCR)
Randomized Controlled Period: Change From Baseline in Log-Transformed Urine Protein-to-Creatinine Ratio (uPCR) at Week 26
log (uPCR)Randomized Controlled Period: PegcetacoplanRandomized Controlled Period: Placebo
Randomized Controlled Period: Change From Baseline in Log-Transformed Urine Protein-to-Creatinine Ratio (uPCR) at Week 26-1.114 (-1.380 to -0.848)0.029 (-0.090 to 0.148)
Statistical analysis
  • Randomized Controlled Period: Pegcetacoplan vs Randomized Controlled Period: Placebo · MMRM · p = <0.0001 · Difference in least squares (ls) mean: -1.143 · 95% CI -1.436 to -0.85
SecondaryRandomized 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.

Time frame:
Week 26
Reported as:
Number · percentage of subjects
Randomized Controlled Period: Percentage of Subjects Who Achieved the Composite Renal Endpoint at Week 26
percentage of subjectsRandomized Controlled Period: PegcetacoplanRandomized Controlled Period: Placebo
Randomized Controlled Period: Percentage of Subjects Who Achieved the Composite Renal Endpoint at Week 2649.213.28
Statistical analysis
  • Randomized Controlled Period: Pegcetacoplan vs Randomized Controlled Period: Placebo · Logistic · p = <0.0001 · Odds ratio (or): 27.516 · 95% CI 6.105 to 124.026
SecondaryRandomized 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.

Time frame:
Baseline (Day -70 to Day 1) and Week 26
Reported as:
Number · percentage of subjects
Randomized Controlled Period: Percentage of Subjects With a Reduction of At Least 50% From Baseline in Urine Protein-to-Creatinine Ratio at Week 26
percentage of subjectsRandomized Controlled Period: PegcetacoplanRandomized Controlled Period: Placebo
Randomized Controlled Period: Percentage of Subjects With a Reduction of At Least 50% From Baseline in Urine Protein-to-Creatinine Ratio at Week 2660.324.92
Statistical analysis
  • Randomized Controlled Period: Pegcetacoplan vs Randomized Controlled Period: Placebo · Logistic · p = <0.0001 · Odds ratio (or): 30.932 · 95% CI 8.401 to 113.897
SecondaryRandomized 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.

Time frame:
Baseline (Day 1) and Week 26
Reported as:
Least squares mean · units on a scale
Randomized Controlled Period: Change From Baseline in the C3 Glomerulopathy (C3G) Histologic Index Activity Score at Week 26
units on a scaleRandomized Controlled Period: PegcetacoplanRandomized Controlled Period: Placebo
Randomized Controlled Period: Change From Baseline in the C3 Glomerulopathy (C3G) Histologic Index Activity Score at Week 26-3.482 (-4.721 to -2.244)-2.480 (-3.775 to -1.186)
Statistical analysis
  • Randomized Controlled Period: Pegcetacoplan vs Randomized Controlled Period: Placebo · ANCOVA · p = 0.2753 · Difference in ls mean: -1.002 · 95% CI -2.803 to 0.798
SecondaryRandomized 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.

Time frame:
Baseline (Day 1) and Week 26
Reported as:
Number · percentage of subjects
Randomized Controlled Period: Percentage of Subjects Who Showed Decrease in C3c Staining on Renal Biopsy From Baseline at Week 26
percentage of subjectsRandomized Controlled Period: PegcetacoplanRandomized Controlled Period: Placebo
Randomized Controlled Period: Percentage of Subjects Who Showed Decrease in C3c Staining on Renal Biopsy From Baseline at Week 2674.2911.76
Statistical analysis
  • Randomized Controlled Period: Pegcetacoplan vs Randomized Controlled Period: Placebo · Logistic · p = <0.0001 · Odds ratio (or): 27.392 · 95% CI 6.477 to 115.852
SecondaryRandomized 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.

Time frame:
Baseline (Day 1) and Week 26
Reported as:
Least squares mean · milliliter (mL)/minute/1.73 m^2
Randomized Controlled Period: Change From Baseline in Estimated Glomerular Filtration Rate at Week 26
milliliter (mL)/minute/1.73 m^2Randomized Controlled Period: PegcetacoplanRandomized Controlled Period: Placebo
Randomized Controlled Period: Change From Baseline in Estimated Glomerular Filtration Rate at Week 26-1.497 (-5.892 to 2.899)-7.808 (-11.570 to -4.047)
Statistical analysis
  • Randomized Controlled Period: Pegcetacoplan vs Randomized Controlled Period: Placebo · MMRM · p = 0.0333 · Difference in ls mean: 6.312 · 95% CI 0.501 to 12.122
SecondaryRandomized 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.

Time frame:
Week 24
Reported as:
Number · percentage of subjects
Randomized Controlled Period: Percentage of Subjects Who Achieved Proteinuria <1 Gram (g)/Day at Week 24
percentage of subjectsRandomized Controlled Period: PegcetacoplanRandomized Controlled Period: Placebo
Randomized Controlled Period: Percentage of Subjects Who Achieved Proteinuria <1 Gram (g)/Day at Week 2436.5111.48
Statistical analysis
  • Randomized Controlled Period: Pegcetacoplan vs Randomized Controlled Period: Placebo · Logistic · p = 0.0006 · Odds ratio (or): 5.753 · 95% CI 2.106 to 15.716
SecondaryRandomized 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.

Time frame:
Week 26
Reported as:
Number · percentage of subjects
Randomized Controlled Period: Percentage of Subjects With Normalization of Serum Albumin Levels at Week 26
percentage of subjectsRandomized Controlled Period: PegcetacoplanRandomized Controlled Period: Placebo
Randomized Controlled Period: Percentage of Subjects With Normalization of Serum Albumin Levels at Week 2677.784.35
Statistical analysis
  • Randomized Controlled Period: Pegcetacoplan vs Randomized Controlled Period: Placebo · Logistic · p = 0.0001 · Odds ratio (or): 88.341 · 95% CI 8.863 to 880.544
SecondaryRandomized 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.

Time frame:
Week 26
Reported as:
Number · percentage of subjects
Randomized Controlled Period: Percentage of Subjects With Serum C3 Levels Above the Lower Limit of Normal at Week 26
percentage of subjectsRandomized Controlled Period: PegcetacoplanRandomized Controlled Period: Placebo
Randomized Controlled Period: Percentage of Subjects With Serum C3 Levels Above the Lower Limit of Normal at Week 2690.246.12
Statistical analysis
  • Randomized Controlled Period: Pegcetacoplan vs Randomized Controlled Period: Placebo · Logistic · p = 0.0094 · Odds ratio (or): 999.999 · 95% CI 12.175 to 9999.999
SecondaryRandomized 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.

Time frame:
Baseline (Day 1) and Week 26
Reported as:
Least squares mean · units on a scale
Randomized Controlled Period: Change From Baseline in the Functional Assessment of Chronic Illness Therapy-Fatigue (FACIT-Fatigue) Score at Week 26
units on a scaleRandomized Controlled Period: PegcetacoplanRandomized Controlled Period: Placebo
Randomized Controlled Period: Change From Baseline in the Functional Assessment of Chronic Illness Therapy-Fatigue (FACIT-Fatigue) Score at Week 260.929 (-1.549 to 3.407)0.367 (-1.949 to 2.683)
Statistical analysis
  • Randomized Controlled Period: Pegcetacoplan vs Randomized Controlled Period: Placebo · ANCOVA · p = 0.7384 · Difference in ls mean: 0.562 · 95% CI -2.739 to 3.863
SecondaryRandomized 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.

Time frame:
Baseline (Day 1) and Week 26
Reported as:
Least squares mean · units on a scale
Randomized Controlled Period: Change From Baseline in the Kidney Disease Quality of Life (KDQOL) Score at Week 26
units on a scaleRandomized Controlled Period: PegcetacoplanRandomized Controlled Period: Placebo
Randomized Controlled Period: Change From Baseline in the Kidney Disease Quality of Life (KDQOL) Score at Week 260.757 (-2.385 to 3.900)-0.587 (-3.847 to 2.672)
Statistical analysis
  • Randomized Controlled Period: Pegcetacoplan vs Randomized Controlled Period: Placebo · ANCOVA · p = 0.5648 · Difference in ls mean: 1.345 · 95% CI -3.237 to 5.927

Adverse events

Collected over TEAE data is reported from first dose of study drug (Day 1) up to 56 days after the last dose of study drug (Week 52), up to 60 weeks. All-cause mortality: From first dose of study drug (Day 1) up to end of the study, approximately 137 weeks.. Non-serious events are listed at a 5% frequency threshold.

Adverse event summary by group
GroupDeathsSeriousOther
Randomized Controlled Period: Pegcetacoplan1/63 (1.6%)6/63 (9.5%)53/63 (84.1%)
Randomized Controlled Period: Placebo0/61 (0%)6/61 (9.8%)56/61 (91.8%)
Open-Label Period: Pegcetacoplan to Pegcetacoplan0/61 (0%)6/61 (9.8%)47/61 (77%)
Open-Label Period: Placebo to Pegcetacoplan0/57 (0%)4/57 (7%)42/57 (73.7%)
Most frequent serious events
Showing 10 of 29
Most frequent serious events
EventRandomized Controlled Period: PegcetacoplanRandomized Controlled Period: PlaceboOpen-Label Period: Pegcetacoplan to PegcetacoplanOpen-Label Period: Placebo to Pegcetacoplan
Acute kidney injuryRenal and urinary disorders1/632/612/611/57
VomitingGastrointestinal disorders0/631/610/611/57
PyrexiaGeneral disorders1/630/610/611/57
GastroenteritisInfections and infestations0/630/610/611/57
Herpes zoster meningoencephalitisInfections and infestations0/630/610/611/57
Shunt infectionInfections and infestations0/630/610/611/57
Shunt malfunctionInjury, poisoning and procedural complications0/630/610/611/57
Shunt thrombosisInjury, poisoning and procedural complications0/630/610/611/57
DehydrationMetabolism and nutrition disorders0/630/611/611/57
Abdominal painGastrointestinal disorders0/630/611/610/57
Most frequent other events
Showing 10 of 27
Most frequent other events
EventRandomized Controlled Period: PegcetacoplanRandomized Controlled Period: PlaceboOpen-Label Period: Pegcetacoplan to PegcetacoplanOpen-Label Period: Placebo to Pegcetacoplan
PyrexiaGeneral disorders12/637/615/614/57
HeadacheNervous system disorders8/6311/614/617/57
NasopharyngitisInfections and infestations11/637/617/613/57
VomitingGastrointestinal disorders5/639/617/612/57
DiarrhoeaGastrointestinal disorders2/637/614/618/57
Injection site swellingGeneral disorders2/637/611/614/57
NauseaGastrointestinal disorders6/634/616/612/57
DizzinessNervous system disorders0/636/613/610/57
HypertensionVascular disorders3/635/616/612/57
InfluenzaInfections and infestations6/633/614/611/57

Baseline characteristics

The intent-to-treat (ITT) analysis set included all randomized subjects.

Age, Continuous
Age, Continuous(years)Randomized Controlled Period: PegcetacoplanRandomized Controlled Period: PlaceboTotal
Mean28.2 ± 17.0823.6 ± 14.2626.0 ± 15.86
Sex: Female, Male
Sex: Female, Male(Participants)Randomized Controlled Period: PegcetacoplanRandomized Controlled Period: PlaceboTotal
Female373370
Male262854
Race/Ethnicity, Customized
Race/Ethnicity, Customized(Participants)Randomized Controlled Period: PegcetacoplanRandomized Controlled Period: PlaceboTotal
American Indian or Alaskan Native101
Asian9918
Black or African American101
Native Hawaiian or Other Pacific Islander000
White454691
Other7613
Race/Ethnicity, Customized
Race/Ethnicity, Customized(Participants)Randomized Controlled Period: PegcetacoplanRandomized Controlled Period: PlaceboTotal
Hispanic151025
Not Hispanic or Latino414788
Not Reported628
Unknown123
08

Study locations

125 sites
  • University of Alabama at Birmingham
    Birmingham, Alabama 35294, United States
  • Academic Medical Research Institute
    Los Angeles, California 90022, United States
  • Keck School of Medicine, University of Southern California
    Los Angeles, California 90033, United States
  • Ronald Reagan UCLA Medical Center (01035)
    Los Angeles, California 90095, United States
  • UCI Center for Clinical Research
    Orange, California 92868, United States
  • UC Davis Medical Center (Transplant Research) (01016)
    Sacramento, California 95817, United States
  • Children's Hospital Colorado
    Aurora, Colorado 80045, United States
  • University of Florida
    Gainesville, Florida 32610, United States
  • Emory University School of Medicine
    Atlanta, Georgia 30322, United States
  • Fides Clinical Research, LLC (01042)
    Atlanta, Georgia 30342, United States
  • Institute for Public Health and Medicine Northwestern University Northwestern University (01041)
    Chicago, Illinois 60611, United States
  • NANIU Research Chicago (01040)
    Oak Brook, Illinois 60523, United States
  • Nephrology Associates of Northern IL and Inn (01043)
    Fort Wayne, Indiana 46804, United States
  • The University of Iowa
    Iowa City, Iowa 52242, United States
  • Boston Children's Hospital (01013)
    Boston, Massachusetts 02115, United States
  • Renal and Transplant Associates of New England, PC
    Springfield, Massachusetts 01107, United States
  • University of Michigan Medical Center
    Ann Arbor, Michigan 48109, United States
  • Children's Mercy Hospital
    Kansas City, Missouri 64102, United States
  • Hackensack Meridian Health
    Hackensack, New Jersey 07601, United States
  • Cohen Children Hospital
    New Hyde Park, New York 11040, United States
  • CUIMC - Columbia Nephrology
    New York, New York 10032, United States
  • The Ohio State University Medical Center
    Columbus, Ohio 43210, United States
  • Oregon Health & Science University (01038)
    Portland, Oregon 97239, United States
  • Northeast Clinical Research Center, LLC
    Bethlehem, Pennsylvania 18017, United States
  • MedResearch Inc
    El Paso, Texas 79902, United States
  • Texas Children's Hospital
    Houston, Texas 77030, United States
  • Hospital Universitario Austral
    Buenos Aires, 1629, Argentina
  • Hospital Privado-Universitario de Cordoba
    Córdoba, CPA X5016KEH, Argentina
  • Clinica Privada Velez Sarsfield
    Córdoba, X5000, Argentina
  • Canberra Hospital - Renal Clinical Trials & Research Unit
    Garran, Australian Capital Territory 2605, Australia
  • Princess Alexandra Hospital
    Woolloongabba, Queensland QLD 4102, Australia
  • Monash University
    Box Hill, VIC 3128, Australia
  • St. Vincents Melbourne
    Fitzroy, VIC 3065, Australia
  • Princess Alexandra Hospital
    Woolloongabba, QLD 4102, Australia
  • Medical University Hospital Innsbruck (43004)
    Innsbruck, 6020, Austria
  • Medizinische Universität Wien
    Vienna, A-1090, Austria
  • Hopital Erasme HUB Service Pharmacie
    Brussels, 1070, Belgium
  • University Hospital Antwerp (32004)
    Edegem, 2650, Belgium
  • Catholic University of Leuven
    Leuven, B-3000, Belgium
  • CHU Sart-Tilman
    Liège, B-4000, Belgium
  • Clinical Trials CHU de Liège
    Liège, B-4000, Belgium
  • Santa Casa de Misericordia de Belo Horizonte
    Belo Horizonte, Minas Gerais 30150-221, Brazil
  • Centro de Tratamento de Doencas Renais
    Juiz de Fora, Minas Gerais 36025-340, Brazil
  • Irmandade da Santa Casa de Misericordia de Porto Alegre
    Porto Alegre, Rio Grande do Sul 90020-090, Brazil
  • HC UNESP Botucatu
    Botucatu, 18618-687, Brazil
  • Hospital Universitario Walter Cantidio
    Fortaleza, 60430-372, Brazil
  • Irmandade da Santa Casa de Misericordia de Porto Alegre
    Porto Alegre, 90020-090, Brazil
  • Hospital de Clinicas de Porto Alegre
    Porto Alegre, 90035-903, Brazil
  • Real Hospital Portuguas de Beneficancia em Pernambuco
    Recife, 52010-095, Brazil
  • Hospital das Clinicas de Ribeirao Preto, Division of Nephrology
    Ribeirão Preto, 14110-000, Brazil
  • Nefrologia I-Dor
    Rio de Janeiro, 22211-225, Brazil
  • Ruschel Medicina E Pesquisa Clinica
    Rio de Janeiro, 22270-060, Brazil
  • Hospital de Base
    São José do Rio Preto, 150900-000, Brazil
  • UNIFESP - Hospital Sao Paulo
    São Paulo, 04038-002, Brazil
  • Instituto da Crianca-Hospital das Clinicas University of Sao Paulo
    São Paulo, 05403-000, Brazil
  • HCFMUSP-Hospital Clinicas da Faculdade Medicina da Universidade de São Paulo
    São Paulo, 05403-900, Brazil
  • Hospital for Sick Children (11003)
    Toronto, Ontario M5G 1X8, Canada
  • Hopital Maisonneuve-Rosemont
    Montreal, Quebec QC H1T2M4, Canada
  • Institute for Clinical and Experimental Medicine
    Prague, 140 21, Czechia
  • Faculty Hospital Kralovske Vinohrady (42002)
    Prague, Czechia
  • CHU de Bordeaux - Hopital Pellegrin
    Bordeaux, 33076, France
  • Hopital Henri-Mondor
    Créteil, 94010, France
  • Hospital Edouard Herriot, Hospices Civils de Lyon
    Lyon, 69437, France
  • CHU Montpellier, Hopital Lapeyronie
    Montpellier, 34295, France
  • Nantes University Hospital
    Nantes, 44093, France
  • Lille Regional University Hospital Center, Claude Huriez Hospital, Department of Nephrology
    Paris, 59037, France
  • Hopital Necker (33014)
    Paris, 75015, France
  • Hôpital Européen Georges-Pompidou
    Paris, 75015, France
  • CHU de Saint Etienne, Hospital Nord
    Saint-Priest-en-Jarez, 42055, France
  • University Hospital Strasbourg
    Strasbourg, 67091, France
  • Rangueil Hospital-University Hospital Center (CHU) of Toulouse
    Toulouse, 31059, France
  • Charite Universitatsmedizin (49007)
    Berlin, 10117, Germany
  • Universitatsklinikum Essen (AoR), Zentrum fur Kinder (49005)
    Essen, D-45147, Germany
  • Medizinische Hochschule Hannover, Studienzentrum fur Nieren und Hochdruckerkrankungen
    Hanover, 30625, Germany
  • Universitatsmedizin Mainz
    Mainz, 55131, Germany
  • Universitatsklinikum Munster
    Münster, 48149, Germany
  • University Hospital Regensburg (49004)
    Regensburg, 93053, Germany
  • Rambam Health Care Campus
    Haifa, 3109601, Israel
  • Institute of Pediatric Nephrology
    Petah Tikva, 4920235, Israel
  • Policlinico di Bari
    Bari, 70123, Italy
  • Policlinico Sant Orsola-Malpighi
    Bologna, 40138, Italy
  • IRCCS Istituto Giannina Gaslini (39012)
    Genova, 16147, Italy
  • Universita degli Studi di Messina
    Messina, 98125, Italy
  • Istituto di Ricerche Farmacologiche Mario Negri IRCCS
    Milan, 20156, Italy
  • ASST Grande Ospedale Metropolitano Niguarda
    Milan, 20162, Italy
  • Azienda Ospedaliera Universitaria di Padova (39011)
    Padova, 35128, Italy
  • Instituti Clinici Scientifici Maugeri SPA-IRCCS
    Pavia, 27100, Italy
  • Ospedale Pediatrico Bambino Gesu
    Rome, 00165, Italy
  • Nagoya University Hospital (81003)
    Nagoya, Aichi-ken 466-8560, Japan
  • Aichi Children's Health and Medical Center
    Ōbu, Aichi-ken 474-8710, Japan
  • Gunma University Hospital (81006)
    Maebashi, Gunma 371-8511, Japan
  • NHO Kanazawa Medical Center
    Kanazawa, Ishikawa-ken 9208650, Japan
  • Nagasaki University Hospital (81005)
    Nagasaki, Nagasaki 852-8501, Japan
  • Seirei Hamamatsu General Hospital (81004)
    Hamamatsu, Shizuoka 430-8558, Japan
  • Kitano Hospital
    Osaka, 530-8480, Japan
  • Kyorin University Hospital (81009)
    Tokyo, 181-8611, Japan
  • Emma Kinderziekenhuis, Amsterdam UMC
    Amsterdam, 1105 AZ, Netherlands
  • University Medical Center Groningen
    Groningen, 9713 GZ, Netherlands
  • Radboud University Medical Center
    Nijmegen, 6500 HB, Netherlands
  • Samodzielny Publiczny Zaklad Opieki Zdrowotnej Uniwersytecki Szpital Kliniczny Nr 1 im. Norberta Barlickiego Uniwersytetu Medycznego w Lodzi
    Lodz, 90-153, Poland

Showing the first 100 of 125 sites across 19 countries.

09

References and documents

Publications

  • 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. PubMed 42133432 ↗
  • Fakhouri F, Bomback AS, Ariceta G, Delmas Y, Dixon BP, Gale DP, Greenbaum LA, Han SH, Isbel N, Le Quintrec M, Licht C, Mastrangelo A, Mizuno M, Neves de Holanda MI, Pickering MC, Remuzzi G, Van De Kar N, Vivarelli M, Walker PD, Wallace D, Zecher D, Francois C, Deschatelets P, Li L, Wang Z, Abad-Franch L, Kinnman N, Lopez-Lazaro L, Szamosi J, Nester CM; VALIANT Trial Investigators Group. Trial of Pegcetacoplan in C3 Glomerulopathy and Immune-Complex MPGN. N Engl J Med. 2025 Dec 4;393(22):2210-2220. doi: 10.1056/NEJMoa2501510. PubMed 41337715 ↗

Study documents

  • Study protocol · Apr 25, 2024
  • Statistical analysis plan · Feb 10, 2025

Documents are hosted by the registry — open the source record to download them.

10

Updates

Tracking since Sep 25, 2026
No changes since tracking began. The registry record was last updated on Jan 29, 2026, before this site started recording changes on Sep 25, 2026. Its history is on ClinicalTrials.gov ↗
11

Registry details

Key details

Study ID
NCT05067127
Lead sponsor
Apellis Pharmaceuticals, Inc.
Responsible party
Sponsor
First posted
Oct 5, 2021
Start date
Nov 12, 2021
Primary completion
Jun 26, 2024
Completion
Jan 14, 2025
Results posted
Aug 6, 2025
Last update
Jan 29, 2026

Oversight

Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No
View the source record on ClinicalTrials.gov ↗

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This study is completed, as verified in Aug 2025. You cannot join it, but the record below documents what was studied.

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Apellis Pharmaceuticals, Inc.

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