18 and older, any sex, with Paroxysmal Nocturnal Hemoglobinuria (PNH). 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 Severity, During Single-dose PhasePrimary· From single dose of study drug (Day 1) up to 30 days
TEAEs were defined as AEs that developed or worsened after first dose of study drug (Day 1), and up to 30 days after last dose of study drug. The Investigator assessed AEs for severity and relatedness to study drug. AEs were graded according to the Common Terminology Criteria for Adverse Events (CTCAE, v4.03) based on: Grade 1: Mild; Grade 2: Moderate; Grade 3: Severe; Grade 4: Life-threatening; Grade 5: Death related to AE.
Any TEAE
Group
Value
95% CI
Cohort 1 Single-dose Phase
1
Cohort 2 Single-dose Phase
2
TEAE at least possibly related to study drug
Group
Value
95% CI
Cohort 1 Single-dose Phase
0
Cohort 2 Single-dose Phase
1
Serious TEAE
Group
Value
95% CI
Cohort 1 Single-dose Phase
0
Cohort 2 Single-dose Phase
0
TEAE leading to study drug discontinuation
Group
Value
95% CI
Cohort 1 Single-dose Phase
0
Cohort 2 Single-dose Phase
0
TEAE leading to death
Group
Value
95% CI
Cohort 1 Single-dose Phase
0
Cohort 2 Single-dose Phase
0
Maximum severity of all TEAEs: mild
Group
Value
95% CI
Cohort 1 Single-dose Phase
0
Cohort 2 Single-dose Phase
1
Maximum severity of all TEAEs: moderate
Group
Value
95% CI
Cohort 1 Single-dose Phase
1
Cohort 2 Single-dose Phase
1
Maximum severity of all TEAEs: severe
Group
Value
95% CI
Cohort 1 Single-dose Phase
0
Cohort 2 Single-dose Phase
0
Number of Subjects With TEAEs, Including by Severity, During Multiple-dose PhasePrimary· From first dose of study drug up to 30 days after last dose of study drug (Cohorts 1-3: up to 58 days; Cohort 4: up to 759 days).
TEAEs were defined as AEs that developed or worsened after first dose of study drug (Day 1), and up to 30 days after last dose of study drug. The Investigator assessed AEs for severity and relatedness to study drug. AEs were graded according to CTCAE, v4.03 based on: Grade 1: Mild; Grade 2: Moderate; Grade 3: Severe; Grade 4: Life-threatening; Grade 5: Death related to AE.
Any TEAE
Group
Value
95% CI
Cohort 1 Multiple-dose Phase
1
Cohort 2 Multiple-dose Phase
2
Cohort 3 Multiple-dose Phase
1
Cohort 4 Multiple-dose Phase
6
TEAE at least possibly related to study drug
Group
Value
95% CI
Cohort 1 Multiple-dose Phase
0
Cohort 2 Multiple-dose Phase
1
Cohort 3 Multiple-dose Phase
1
Cohort 4 Multiple-dose Phase
4
Serious TEAE
Group
Value
95% CI
Cohort 1 Multiple-dose Phase
0
Cohort 2 Multiple-dose Phase
0
Cohort 3 Multiple-dose Phase
0
Cohort 4 Multiple-dose Phase
2
TEAE leading to study drug discontinuation
Group
Value
95% CI
Cohort 1 Multiple-dose Phase
1
Cohort 2 Multiple-dose Phase
0
Cohort 3 Multiple-dose Phase
0
Cohort 4 Multiple-dose Phase
0
TEAE leading to death
Group
Value
95% CI
Cohort 1 Multiple-dose Phase
0
Cohort 2 Multiple-dose Phase
0
Cohort 3 Multiple-dose Phase
0
Cohort 4 Multiple-dose Phase
0
Maximum severity of all TEAEs: mild
Group
Value
95% CI
Cohort 1 Multiple-dose Phase
0
Cohort 2 Multiple-dose Phase
0
Cohort 3 Multiple-dose Phase
1
Cohort 4 Multiple-dose Phase
0
Maximum severity of all TEAEs: moderate
Group
Value
95% CI
Cohort 1 Multiple-dose Phase
0
Cohort 2 Multiple-dose Phase
2
Cohort 3 Multiple-dose Phase
0
Cohort 4 Multiple-dose Phase
2
Maximum severity of all TEAEs: severe
Group
Value
95% CI
Cohort 1 Multiple-dose Phase
1
Cohort 2 Multiple-dose Phase
0
Cohort 3 Multiple-dose Phase
0
Cohort 4 Multiple-dose Phase
4
Area Under the Curve (AUC) From Time 0 to the Last Measurable Concentration (AUC0-t) Over the Multiple Dosing Phase for Cohort 4Primary· Blood samples for PK assessment were collected pre-dose and 4 hours post dose on Day 1 and pre-dose (trough) on Day 2 and up to Day 785.
Assessment of AUC0-t of pegcetacoplan over the multiple dosing phase, estimated using a non-compartmental approach and calculated by the linear-log trapezoidal method. Pegcetacoplan pharmacokinetic (PK) parameters were summarized for Cohort 4 only.
Group
Value
95% CI
Cohort 4 Multiple-dose Phase
6,500,000
± 3,990,000
Maximum Pre-dose Serum Concentration (Ctrough,Max) Over the Multiple Dosing Phase for Cohort 4Primary· Blood samples for PK assessment were collected pre-dose and 4 hours post dose on Day 1 and pre-dose (trough) on Day 2 and up to Day 785.
Assessment of Ctrough,max of pegcetacoplan over the multiple dosing phase, estimated using a non-compartmental approach. Pegcetacoplan PK parameters were summarized for Cohort 4 only. Ctrough,max was calculated for both 270 mg/day and 360 mg/day where subjects received both doses. Note: 1 subject in Cohort 4 who was receiving 360 mg/day was granted Sponsor and institutional review board approval to increase the dose further to the equivalent of 440 mg/day and Ctrough,max is also reported for this dose.
270 mg Dose
Group
Value
95% CI
Cohort 4 Multiple-dose Phase
627
± 207
360 mg Dose
Group
Value
95% CI
Cohort 4 Multiple-dose Phase
543
± 192
440 mg Dose
Group
Value
95% CI
Cohort 4 Multiple-dose Phase
624
± NA
Adverse events — posted to ClinicalTrials.gov
Time frame: TEAE data is reported from first dose of study drug up to 30 days after last dose of study drug (Cohorts 1 and 2, single-dose phase: up to 30 days; Cohorts 1-3, multiple-dose phase: up to 58 days; Cohort 4, multiple-dose phase: up to 759 days)..
Reporting threshold: 0%.
Adverse-event reports describe events observed during the trial — not all are caused by the drug.
Cohort 1 Single-dose Phase
Serious: 0/2 (0%)
Deaths: 1/2
Cohort 2 Single-dose Phase
Serious: 0/2 (0%)
Deaths: 0/2
Cohort 1 Multiple-dose Phase
Serious: 0/1 (0%)
Deaths: 0/1
Cohort 2 Multiple-dose Phase
Serious: 0/2 (0%)
Deaths: 0/2
Cohort 3 Multiple-dose Phase
Serious: 0/2 (0%)
Deaths: 0/2
Cohort 4 Multiple-dose Phase
Serious: 2/6 (33%)
Deaths: 0/6
Serious adverse events (7 terms)
Reaction
System
Cohort 1 Single-dose Phase
Cohort 2 Single-dose Phase
Cohort 1 Multiple-dose Phase
Cohort 2 Multiple-dose Phase
Cohort 3 Multiple-dose Phase
Cohort 4 Multiple-dose Phase
Anaemia
Blood and lymphatic system disorders
—
—
—
—
—
—
Lower gastrointestinal haemorrhage
Gastrointestinal disorders
—
—
—
—
—
—
Pancreatitis
Gastrointestinal disorders
—
—
—
—
—
—
Portal vein thrombosis
Hepatobiliary disorders
—
—
—
—
—
—
Sepsis
Infections and infestations
—
—
—
—
—
—
Alanine aminotransferase increased
Investigations
—
—
—
—
—
—
Aspartate aminotransferase increased
Investigations
—
—
—
—
—
—
Other adverse events (106 terms — click to expand)
This study will be the initial exploration of pegcetacoplan in patients with PNH. The assessments of the safety, tolerability, PK, and PD following administration of single and multiples doses of pegcetacoplan will guide decisions to further develop the drug.
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 Paroxysmal Nocturnal Hemoglobinuria (PNH)
Currently open trials in the same condition.
NCT07187401 — A First-in-Human Safety and Efficacy Study of ALN-CFB, a Small Interfering RNA (siRNA) Targeting Complement Factor B, in
· Phase 1, PHASE2
· recruiting
NCT07229235 — REAL-CARE: Real-world Effectiveness of Iptacopan in Italian Patients With Paroxysmal Nocturnal Hemoglobinuria
· recruiting
NCT06934967 — Study to Assess the Pharmacokinetics, Safety, and Tolerability of Iptacopan in Pediatric PNH Patients
· Phase 3
· recruiting
NCT06312644 — Study of Ultomiris® (Ravulizumab) Safety in Pregnancy
· recruiting
NCT06933914 — Long-term Safety and Tolerability of MY008211A Tablets in Patients With Paroxysmal Nocturnal Hemoglobinuria
· Phase 2, PHASE3
· 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: 8 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/NCT02264639.