| NCT ID | Title | Phase | Status | Enrollment | Velocity | Design | Start | Completion | Last Updated | Sites | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|
| NCT03500549 | Study to Evaluate the Efficacy and Safety of APL-2 in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) | PHASE3 | COMPLETED | 80 | — | — | Jun 14, 2018 | Aug 13, 2020 | Mar 25, 2022 | 53 | United States, Australia +9 |
| NCT02588833 | Pilot Study to Assess Safety, Preliminary Efficacy and Pharmacokinetics of S.C. Pegcetacoplan (APL-2) in PNH Subjects. | PHASE1 | COMPLETED | 23 | — | — | Dec 1, 2015 | Aug 26, 2019 | Jan 11, 2021 | 7 | Hong Kong, Malaysia +2 |
Baseline was the average of measurements recorded before taking the first dose of pegcetacoplan, which included local and central laboratory values during the screening period. Analysis excluded data before the RCP and was censored for transfusions.
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 graded according to Common Terminology Criteria for Adverse Events v4.03 based on: Grade 1: Mild; Grade 2: Moderate; Grade 3: Severe; Grade 4: Life-threatening; Grade 5: Death related to AE.
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.
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.
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.
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.
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.
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.
| Arm | Type | Description |
|---|---|---|
| Pegcetacoplan | EXPERIMENTAL | 1080 mg pegcetacoplan administered subcutaneously twice-weekly or every three days. |
| Eculizumab | ACTIVE_COMPARATOR | Complement (C5) Inhibitor. |
| Cohort 1 | EXPERIMENTAL | 180 mg pegcetacoplan/day |
| Cohort 2 | EXPERIMENTAL | 270 mg pegcetacoplan/day |
| Name | Type | Description |
|---|---|---|
| Pegcetacoplan | DRUG | Complement (C3) Inhibitor |
| Soliris | DRUG | Complement (C5) Inhibitor |
Inclusion Criteria: * At least 18 years of age * Primary diagnosis of PNH confirmed by high-sensitivity flow cytometry * On treatment with eculizumab. Dose of eculizumab must have been stable for at least 3 months prior to the Screening Visit * Hb \<10.5 g/dL at the Screening Visit * Absolute retic...