50 and older, any sex, with Geographic Atrophy. 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.
Least Square (LS) Mean Change From Baseline in Square Root GA Lesion Size in the Study Eye at Month 12Primary· Baseline (screening) and Month 12.
The square root GA lesion size (i.e. transformed area of GA) was measured by FAF photographs. Baseline was defined as the last available, non-missing observation prior to first study drug administration.
Group
Value
95% CI
Pegcetacoplan Monthly
0.26
± 0.025
Pegcetacoplan EOM
0.28
± 0.026
Sham Pooled
0.35
± 0.025
Number of Subjects With Treatment Emergent Adverse Events (TEAEs) in the Study Eye, Including by SeverityPrimary· From the time of first study drug administration (Day 1) up to Month 12 (Data cut-off date).
A TEAE was defined as any adverse event (AE) that commenced or worsened on or after time of first study drug administration up to 60 days beyond last dose of study drug. A treatment-related TEAE was defined as a TEAE with a relationship to study drug of possibly related or probably related or not reported. Severity of TEAEs were categorized as mild; moderate; severe; life-threatening or death related to TEAE, according to Common Terminology Criteria for AEs v4.03. A TEAE of special interest (TEAESI) was defined as a TEAE of scientific and medical concern specific to pegcetacoplan, whether seri
TEAEs
Group
Value
95% CI
Pegcetacoplan Monthly
65
Pegcetacoplan EOM
47
Sham Pooled
42
Treatment-related TEAEs
Group
Value
95% CI
Pegcetacoplan Monthly
21
Pegcetacoplan EOM
11
Sham Pooled
0
Serious TEAEs
Group
Value
95% CI
Pegcetacoplan Monthly
4
Pegcetacoplan EOM
2
Sham Pooled
1
TEAEs with mild intensity
Group
Value
95% CI
Pegcetacoplan Monthly
36
Pegcetacoplan EOM
30
Sham Pooled
28
TEAEs with moderate intensity
Group
Value
95% CI
Pegcetacoplan Monthly
23
Pegcetacoplan EOM
14
Sham Pooled
11
TEAEs with severe intensity
Group
Value
95% CI
Pegcetacoplan Monthly
6
Pegcetacoplan EOM
3
Sham Pooled
3
TEAEs with life-threatening intensity
Group
Value
95% CI
Pegcetacoplan Monthly
0
Pegcetacoplan EOM
0
Sham Pooled
0
Death related to TEAEs
Group
Value
95% CI
Pegcetacoplan Monthly
0
Pegcetacoplan EOM
0
Sham Pooled
0
LS Mean Change From Baseline in Untransformed GA Lesion Size in the Study Eye at Month 12Secondary· Baseline (Day 1) and Month 12.
The untransformed area of GA was measured by FAF. Baseline is defined as the last available, non-missing observation prior to first study drug administration.
Group
Value
95% CI
Pegcetacoplan Monthly
1.49
± 0.161
Pegcetacoplan EOM
1.69
± 0.168
Sham Pooled
2.12
± 0.161
LS Mean Change From Baseline in Best-Corrected Visual Acuity (BCVA) Score of the Study Eye at Month 12Secondary· Baseline (Day 1) and Month 12.
The BCVA letter score was determined using Early Treatment Diabetic Retinopathy Study (ETDRS) visual acuity chart assessed at a starting distance of 4 meters. The score ranges from 0 to 100 letters, lower number indicating reduced visual acuity; a positive value of change from baseline indicates visual acuity gain and a negative value indicates visual acuity loss.
Group
Value
95% CI
Pegcetacoplan Monthly
-3.31
± 1.352
Pegcetacoplan EOM
-5.78
± 1.398
Sham Pooled
-4.36
± 1.364
LS Mean Change From Baseline in Low Luminance BCVA (LL-BCVA) Score in the Study Eye at Month 12Secondary· Baseline (Day 1) and Month 12.
The LL-BCVA was measured by placing a 2.0-log-unit neutral density filter over the best correction and having the participant read the normally illuminated ETDRS chart. The score ranges from 0 to 100 letters, lower number indicating worse vision; a positive value of change from baseline indicates visual acuity gain and a negative value indicates visual acuity loss.
Group
Value
95% CI
Pegcetacoplan Monthly
-2.73
± 1.145
Pegcetacoplan EOM
-3.21
± 1.184
Sham Pooled
-0.55
± 1.150
LS Mean Change From Baseline in Low Luminance VA (LL-VA) Deficit Score in the Study Eye at Month 12Secondary· Baseline (Day 1) and Month 12.
The LL-VA deficit score is calculated as BCVA score minus LL-BCVA score. The LL-VA deficit score ranges from 0 to 100 letters, lower number indicating worse deficit.
Group
Value
95% CI
Pegcetacoplan Monthly
-0.76
± 1.382
Pegcetacoplan EOM
-2.40
± 1.424
Sham Pooled
-3.72
± 1.385
LS Mean Change From Baseline in Distance of GA Lesion From the Fovea (Foveal Encroachment) in the Study Eye at Month 12Secondary· Baseline (Day 1) and Month 12.
The foveal encroachment in the study eye was measured by FAF. Baseline is defined as the last available, non-missing observation prior to first study drug administration.
Group
Value
95% CI
Pegcetacoplan Monthly
-0.04
± 0.011
Pegcetacoplan EOM
-0.04
± 0.012
Sham Pooled
-0.06
± 0.011
Number of Subjects With Any Macular Neovascularization (MNV) TEAEs in the Study EyeSecondary· From the time of first study drug administration (Day 1) up to Month 12 (Data cut-off date).
The number of subjects with any MNV TEAEs in the study eye was identified via clinical review of all ocular TEAEs.
Group
Value
95% CI
Pegcetacoplan Monthly
14
Pegcetacoplan EOM
5
Sham Pooled
1
Adverse events — posted to ClinicalTrials.gov
Time frame: TEAE data is reported from the time of first study drug administration (Day 1) up to Month 12 (Data cut-off date)..
Reporting threshold: 5%.
Adverse-event reports describe events observed during the trial — not all are caused by the drug.
The primary objectives of the study are to assess the safety, tolerability and evidence of activity of multiple intravitreal (IVT) injections of pegcetacoplan in subjects with Geographic Atrophy associated with Age-Related Macular Degeneration (AMD).
Publications & conference data
8 peer-reviewed publications reference this trial (live from Europe PMC):
NCT07214298 — Pegcetacoplan in Combination With Modified FOLFIRINOX for the Treatment of Metastatic Pancreatic Ductal Adenocarcinoma
· Phase 1, PHASE2
· 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
Other recruiting trials for Geographic Atrophy
Currently open trials in the same condition.
NCT07215234 — A Safety and Efficacy Study of a One-time Intravitreal Injection of SAR446597 in Participants With Geographic Atrophy Se
· Phase 1, PHASE2
· recruiting
NCT06961370 — A Study to Investigate Safety, Tolerability, Pharmacokinetics (PK), Pharmacodynamics (PD), and Immunogenicity of RO76693
· Phase 1
· recruiting
NCT06970665 — A Study About the Safety of ASP3021 Eye Injections and if They Help People in Japan With Vision Loss From Age-related Ma
· Phase 4
· recruiting
NCT06769048 — A Study to Test Different Doses of BI 1584862 in People With a Distinct Form of Age-related Macular Degeneration (AMD) C
· Phase 2
· active not recruiting
NCT06805474 — A Prospective Observational Study to Assess the Reliability and Validity of the MLSDT
· recruiting
Other Apellis Pharmaceuticals, Inc. trials
Trials by the same sponsor.
NCT07214740 — Study to Evaluate a Pegcetacoplan (Syfovre®) Prefilled Syringe
· Phase 3
· completed
NCT06499571 — Geographic Atrophy Long-Terms Outcomes Study
· completed
NCT05067127 — Phase III Study Assessing the Efficacy and Safety of Pegcetacoplan in Patients With C3 Glomerulopathy or Immune-Complex
· Phase 3
· completed
NCT04770545 — An Extension Study to Evaluate the Long-term Safety and Efficacy of Pegcetacoplan (APL-2) in Subjects With Geographic At
· Phase 3
· completed
NCT04579666 — MERIDIAN: A Study to Evaluate the Efficacy and Safety of Pegcetacoplan in Adults With Amyotrophic Lateral Sclerosis (ALS
· Phase 2
· terminated
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: 6 October 2020
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/NCT02503332.