18 and older, any sex, with Paroxysmal Nocturnal Hemoglobinuria. Patients with the condition only — healthy volunteers not accepted.
Results — posted to ClinicalTrials.gov
Per-arm endpoint measurements with 95% confidence intervals where reported. Source: trial results section.
Number of Subjects With Treatment Emergent Adverse Events (TEAEs) Including by SeverityPrimary· From first dose of study drug (Day 1) up to 30 days after the last dose of study drug, up to approximately 563 days.
TEAEs were defined as adverse events (AEs) that occurred after dosing on Day 1 and up to 30 days after the last dose of study drug. A treatment-related TEAE is defined as a TEAE with a relationship to study drug of probably, possibly, unlikely, or unrelated. A serious adverse event (SAE) is defined as any AE that resulted in death; was life-threatening; required hospitalization or prolongation of existing hospitalization; resulted in persistent or significant incapacity or substantial disruption of ability to conduct normal life functions; was a congenital anomaly or birth defect. TEAEs were g
All TEAEs
Group
Value
95% CI
Cohort 1
2
Cohort 2
18
Treatment related TEAEs
Group
Value
95% CI
Cohort 1
2
Cohort 2
9
Serious AEs
Group
Value
95% CI
Cohort 1
1
Cohort 2
6
TEAEs leading to study drug discontinuation
Group
Value
95% CI
Cohort 1
1
Cohort 2
2
TEAEs leading to death
Group
Value
95% CI
Cohort 1
0
Cohort 2
1
TEAEs with mild intensity
Group
Value
95% CI
Cohort 1
0
Cohort 2
3
TEAEs with moderate intensity
Group
Value
95% CI
Cohort 1
2
Cohort 2
8
TEAEs with severe intensity
Group
Value
95% CI
Cohort 1
0
Cohort 2
5
Mean Change From Baseline in Lactate Dehydrogenase (LDH) at Day 365Primary· Baseline (Day 1) and Day 365.
Serum chemistry assessments of LDH were made at the last measurement prior to the first dose of pegcetacoplan (baseline) and periodically throughout the different parts of the treatment period. LDH results were summarized for Cohort 2 only.
Group
Value
95% CI
Cohort 2
-2105.2
± 1078.79
Mean Percentage Change From Baseline in LDH at Day 365Primary· Baseline (Day 1) and Day 365.
Serum chemistry assessments of LDH were made at the last measurement prior to the first dose of pegcetacoplan (baseline) and periodically throughout the different parts of the treatment period. LDH results were summarized for Cohort 2 only.
Group
Value
95% CI
Cohort 2
-84.8
± 14.04
Mean Change From Baseline in Haptoglobin at Day 365Primary· Baseline (Day 1) and Day 365.
Serum chemistry assessments of haptoglobin were made at the last measurement prior to the first dose of pegcetacoplan (baseline) and periodically throughout the different parts of the treatment period. Haptoglobin results were summarized for Cohort 2 only.
Group
Value
95% CI
Cohort 2
0.066
± 0.1245
Mean Percentage Change From Baseline in Haptoglobin at Day 365Primary· Baseline (Day 1) and Day 365.
Serum chemistry assessments of haptoglobin were made at the last measurement prior to the first dose of pegcetacoplan (baseline) and periodically throughout the different parts of the treatment period. Haptoglobin results were summarized for Cohort 2 only.
Group
Value
95% CI
Cohort 2
166.176
± 311.3656
Mean Change From Baseline in Hemoglobin at Day 365Primary· Baseline (Day 1) and Day 365.
Hematology assessments of hemoglobin were made at the last measurement prior to the first dose of pegcetacoplan (baseline) and periodically throughout the different parts of the treatment period. Hemoglobin results were summarized for Cohort 2 only.
Group
Value
95% CI
Cohort 2
3.68
± 2.690
Mean Percentage Change From Baseline in Hemoglobin at Day 365Primary· Baseline (Day 1) and Day 365.
Hematology assessments of hemoglobin were made at the last measurement prior to the first dose of pegcetacoplan (baseline) and periodically throughout the different parts of the treatment period. Hemoglobin results were summarized for Cohort 2 only.
Group
Value
95% CI
Cohort 2
49.86
± 43.254
Mean Change From Baseline in Functional Assessment of Chronic Illness Therapy (FACIT)-Fatigue Score at Day 365Secondary· Baseline (Day 1) and Day 365.
The FACIT-Fatigue Scale is a 13 item Likert scaled instrument where the subject was presented with 13 statements and asked to indicate their response 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). There are 13 statements with the total score ranging from 0 to 52 and higher scores indicating better quality of life. The FACIT-Fatigue Scale results were summarized for Cohort 2 only.
Group
Value
95% CI
Cohort 2
7.1
± 11.09
Mean Change From Baseline in Absolute Reticulocyte Count (ARC) at Day 365Secondary· Baseline (Day 1) and Day 365.
Hematology assessments of ARC were made at the last measurement prior to the first dose of pegcetacoplan (baseline) and periodically throughout the different parts of the treatment period. ARC results were summarized for Cohort 2 only.
Group
Value
95% CI
Cohort 2
-105.9
± 70.28
Mean Percentage Change From Baseline in ARC at Day 365Secondary· Baseline (Day 1) and Day 365.
Hematology assessments of ARC were made at the last measurement prior to the first dose of pegcetacoplan (baseline) and periodically throughout the different parts of the treatment period. ARC results were summarized for Cohort 2 only.
Group
Value
95% CI
Cohort 2
-47.5
± 26.86
Mean Change From Baseline in Total Bilirubin at Day 365Secondary· Baseline (Day 1) and Day 365.
Serum chemistry assessments of total bilirubin were made at the last measurement prior to the first dose of pegcetacoplan (baseline) and periodically throughout the 3 parts of the treatment period. Total bilirubin results were summarized for Cohort 2 only.
Group
Value
95% CI
Cohort 2
-29.9
± 24.34
Mean Percentage Change From Baseline in Total Bilirubin at Day 365Secondary· Baseline (Day 1) and Day 365.
Serum chemistry assessments of total bilirubin were made at the last measurement prior to the first dose of pegcetacoplan (baseline) and periodically throughout the 3 parts of the treatment period. Total bilirubin results were summarized for Cohort 2 only.
Group
Value
95% CI
Cohort 2
-60.9
± 19.00
Adverse events — posted to ClinicalTrials.gov
Time frame: TEAE data is reported from first dose of study drug (Day 1) up to 30 days after the last dose of study drug, up to approximately 563 days..
Reporting threshold: 0%.
Adverse-event reports describe events observed during the trial — not all are caused by the drug.
Cohort 1
Serious: 1/3 (33%)
Deaths: 0/3
Cohort 2
Serious: 6/20 (30%)
Deaths: 1/20
Serious adverse events (11 terms)
Reaction
System
Cohort 1
Cohort 2
Haemolysis
Blood and lymphatic system disorders
—
—
Hypersensitivity
Immune system disorders
—
—
Abdominal neoplasm
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
The objectives of the study are to assess the safety, tolerability, preliminary efficacy and PK of multiple subcutaneous (SC) doses of pegcetacoplan in subjects with paroxysmal nocturnal hemoglobinuria (PNH) who have not received treatment with eculizumab in the past.
An exploratory objective of the study is to assess the pharmacodynamics (PD) of multiple SC doses of pegcetacoplan when administered to PNH patients.
Publications & conference data
8 peer-reviewed publications reference this trial (live from Europe PMC):
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· recruiting
NCT06722157 — A Study to Test Whether BI 771716 Helps People With an Advanced Form of Age-related Macular Degeneration (AMD) Called Ge
· Phase 2
· active not recruiting
NCT05776472 — A Real World Effectiveness Study of Pegcetacoplan in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH)
· recruiting
NCT04919629 — APL-2 and Pembrolizumab Versus APL-2, Pembrolizumab and Bevacizumab Versus Bevacizumab Alone for the Treatment of Recurr
· Phase 2
· recruiting
NCT05096403 — A Study to Evaluate the Efficacy and Safety of Pegcetacoplan in Patients With Cold Agglutinin Disease (CAD)
· Phase 3
· completed
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Trials by the same sponsor.
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Publications: Europe PMC API search by NCT ID, retrieved 10 June 2026
Drug + disease cross-links: matched in real time against Drug Landscape's normalised drug + company + condition tables
Sponsor: as reported to ClinicalTrials.gov by Apellis Pharmaceuticals, Inc.
Last refreshed: 11 January 2021
Drug Landscape aggregates and links these public records for informational use only. Always verify against the primary source before clinical or regulatory decisions. Canonical URL: https://druglandscape.com/trial/NCT02588833.