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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.
This is a Phase Ib, open-label, multiple ascending dose, pilot study in patients with PNH who have not received eculizumab (Soliris)® in the past. Two cohorts of 3 subjects are planned for evaluation.
Safety will be assessed throughout the study; serial blood and urine samples will be collected for these assessments. Blood samples will be collected for the assessment of pegcetacoplan PK. Additional samples for assessment of PD will also be collected.
The study will consist of four parts;
Subjects will be entered into Part 1 of the study on Day 1 at a time designated by the PI. During Part 1, the first 3 daily SC doses of pegcetacoplan (Day 1 to 3) as well as doses on Day 8, 15 and 22 will be administered at the clinical site. From Day 4 to Day 28 daily doses of pegcetacoplan will be administered off-site by a trained study nurse at the subject's home, workplace, or other location convenient to the subject with the exception of those days where dosing is at the clinical site. Following ongoing review of available safety, PK and PD data by the investigator and sponsor, subjects showing evidence of perceived clinical benefit may progress to Part 2A and then to Part 2B of the study and continue to receive daily doses of pegcetacoplan until Day 84 and then until Day 364.
Cohort 2 will not be initiated until all subjects in Cohort 1 have reached the Day 29 visit and the SMC has reviewed emerging safety and efficacy data and determined that, at the initial dose, pegcetacoplan has an acceptable safety and tolerability profile.
The planned length of participation in the study for each subject in Cohort 2 is approximately 444 days (14.5 months) from Day 30 through completion of the Day 414 Exit visit procedures).
The study is planned to take place over approximately 24 months (from screening of Cohort 1 through completion of Cohort 2).
This time period may change in the event that the study is terminated early, additional cohorts are enrolled, additional time is required to review safety between cohorts, or extended safety and PK sampling is added for a cohort.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Cohort 1 | Experimental | 180 mg pegcetacoplan/day |
|
| Cohort 2 | Experimental | 270 mg pegcetacoplan/day |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Pegcetacoplan | Drug | Complement (C3) Inhibitor |
|
| Measure | Description | Time Frame |
|---|---|---|
| Number of Subjects With Treatment Emergent Adverse Events (TEAEs) Including by Severity | 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. | From first dose of study drug (Day 1) up to 30 days after the last dose of study drug, up to approximately 563 days. |
| Mean Change From Baseline in Lactate Dehydrogenase (LDH) at 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. | Baseline (Day 1) and Day 365. |
| Mean Percentage Change From Baseline in LDH at 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. | Baseline (Day 1) and Day 365. |
| Mean Change From Baseline in Haptoglobin at 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. |
| Measure | Description | Time Frame |
|---|---|---|
| Mean Change From Baseline in Functional Assessment of Chronic Illness Therapy (FACIT)-Fatigue Score at 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. |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Federico Grossi, MD, PhD | Apellis Pharmaceuticals, Inc. | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Prince of Wales Hospital | Hong Kong | Hong Kong | ||||
| Queen Mary Hospital |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 39079163 | Derived | Panse J, Daguindau N, Okuyama S, Peffault de Latour R, Schafhausen P, Straetmans N, Al-Adhami M, Persson E, Wong RSM. Improvements in hematologic markers and decreases in fatigue with pegcetacoplan for patients with paroxysmal nocturnal hemoglobinuria and mild or moderate anemia (hemoglobin >/=10 g/dL) who had received eculizumab or were naive to complement inhibitors. PLoS One. 2024 Jul 29;19(7):e0306407. doi: 10.1371/journal.pone.0306407. eCollection 2024. | |
| 35869170 |
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Subjects could participate in more than 1 cohort. The study consisted of 3 parts: Part 1 for Cohorts 1 and 2, Part 2 (Part 2A, Part 2B and Part 2C) for Cohort 2 only and Part 3 (safety follow-up). Cohort 2 dosing was not initiated until all subjects in Cohort 1 had completed Part 1 and review of emerging safety and efficacy data. Overall, 2 cohorts of 22 unique subjects were evaluated.
This Phase 1b, pilot study was conducted at 9 sites in 5 countries (Hong Kong, Malaysia, New Zealand, Thailand and United States) in subjects with paroxysmal nocturnal hemoglobinuria (PNH) who had not received treatment with eculizumab (Soliris®) in the past, between 01 December 2015 and 26 August 2019. A total of 22 subjects were enrolled.
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| ID | Title | Description |
|---|---|---|
| FG000 | Cohort 1 | Subjects received subcutaneous (SC) injections of pegcetacoplan 180 milligrams (mg)/day for up to 28 days. |
| FG001 | Cohort 2 | Subjects received SC injections or infusions of pegcetacoplan 270 mg/day for up to 364 days if entering the open-label extension study or, if entering Part 2C, until enrollment in the open-label extension study became available. If clinically indicated on the basis of response, the pegcetacoplan dosage could be increased up to 360 mg/day. |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| Part 1 |
|
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| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| Prot | Yes | No | No | Study Protocol | Oct 25, 2018 | Jul 22, 2020 |
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| Baseline (Day 1) and Day 365. |
| Mean Percentage Change From Baseline in Haptoglobin at 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. | Baseline (Day 1) and Day 365. |
| Mean Change From Baseline in Hemoglobin at 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. | Baseline (Day 1) and Day 365. |
| Mean Percentage Change From Baseline in Hemoglobin at 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. | Baseline (Day 1) and Day 365. |
| Baseline (Day 1) and Day 365. |
| Mean Change From Baseline in Absolute Reticulocyte Count (ARC) at 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. | Baseline (Day 1) and Day 365. |
| Mean Percentage Change From Baseline in ARC at 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. | Baseline (Day 1) and Day 365. |
| Mean Change From Baseline in Total Bilirubin at 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. | Baseline (Day 1) and Day 365. |
| Mean Percentage Change From Baseline in Total Bilirubin at 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. | Baseline (Day 1) and Day 365. |
| Number of Subjects Receiving Red Blood Cell (RBC) Transfusions | The number of on-study RBC transfusions were monitored throughout the treatment period. | From Day 1 up to Day 533. |
| Hong Kong |
| Hong Kong |
| Hospital Ampang | Ampang | Selangor | 68000 | Malaysia |
| Waikato Hospital | Hamilton | Waikato Region | 3240 | New Zealand |
| Ramathibodi Hospital | Bangkok | 10400 | Thailand |
| Siriraj hospital | Bangkok | 10700 | Thailand |
| Phramongkutklao Hospital | Bangkok | Thailand |
| Derived |
| Wong RSM, Pullon HWH, Amine I, Bogdanovic A, Deschatelets P, Francois CG, Ignatova K, Issaragrisil S, Niparuck P, Numbenjapon T, Roman E, Sathar J, Xu R, Al-Adhami M, Tan L, Tse E, Grossi FV. Inhibition of C3 with pegcetacoplan results in normalization of hemolysis markers in paroxysmal nocturnal hemoglobinuria. Ann Hematol. 2022 Sep;101(9):1971-1986. doi: 10.1007/s00277-022-04903-x. Epub 2022 Jul 22. |
| COMPLETED |
|
| NOT COMPLETED |
|
|
| Part 2A |
|
|
| Part 2B |
|
|
| Part 2C |
|
|
| Part 3 (Safety Follow-up) |
|
|
The Intent-To-Treat (ITT) population set included all subjects eligible to receive study drug and who received ≥1 dose of study drug. In this study, subjects could participate in more than 1 cohort. Overall, 2 cohorts of 22 unique subjects were evaluated as one subject participated in cohorts 1 and 2.
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| ID | Title | Description |
|---|---|---|
| BG000 | Cohort 1 | Subjects received SC injections of pegcetacoplan 180 mg/day for up to 28 days. |
| BG001 | Cohort 2 | Subjects received SC injections or infusions of pegcetacoplan 270 mg/day for up to 364 days if entering the open-label extension study or, if entering Part 2C, until enrollment in the open-label extension study became available. If clinically indicated on the basis of response, the pegcetacoplan dosage could be increased up to 360 mg/day. |
| BG002 | Total | Total of all reporting groups |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes | ||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Age, Categorical | Count of Participants | Participants | No |
| |||||||||||||||||
| Sex: Female, Male | Count of Participants | Participants | No |
| |||||||||||||||||
| Race/Ethnicity, Customized | Count of Participants | Participants | No |
| |||||||||||||||||
| Race/Ethnicity, Customized | Count of Participants | Participants | No |
|
| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | ||||||||||||||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | Number of Subjects With Treatment Emergent Adverse Events (TEAEs) Including by Severity | 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. | The safety population set included all subjects eligible to receive study drug and who received ≥1 dose of study drug. | Posted | Count of Participants | Participants | No | From first dose of study drug (Day 1) up to 30 days after the last dose of study drug, up to approximately 563 days. |
|
|
| |||||||||||||||||||||||||||||||||||
| Primary | Mean Change From Baseline in Lactate Dehydrogenase (LDH) at 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. | The ITT population set included all subjects eligible to receive study drug and who received ≥1 dose of study drug. Only subjects analyzed at Day 365 are reported. | Posted | Mean | Standard Deviation | units per liter (U/L) | Baseline (Day 1) and Day 365. |
|
| ||||||||||||||||||||||||||||||||||||
| Primary | Mean Percentage Change From Baseline in LDH at 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. | The ITT population set included all subjects eligible to receive study drug and who received ≥1 dose of study drug. Only subjects analyzed at Day 365 are reported. | Posted | Mean | Standard Deviation | percent change | Baseline (Day 1) and Day 365. |
|
| ||||||||||||||||||||||||||||||||||||
| Primary | Mean Change From Baseline in Haptoglobin at 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. | The ITT population set included all subjects eligible to receive study drug and who received ≥1 dose of study drug. Only subjects analyzed at Day 365 are reported. | Posted | Mean | Standard Deviation | gram per liter (g/L) | Baseline (Day 1) and Day 365. |
|
| ||||||||||||||||||||||||||||||||||||
| Primary | Mean Percentage Change From Baseline in Haptoglobin at 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. | The ITT population set included all subjects eligible to receive study drug and who received ≥1 dose of study drug. Only subjects analyzed at Day 365 are reported. | Posted | Mean | Standard Deviation | percent change | Baseline (Day 1) and Day 365. |
|
| ||||||||||||||||||||||||||||||||||||
| Primary | Mean Change From Baseline in Hemoglobin at 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. | The ITT population set included all subjects eligible to receive study drug and who received ≥1 dose of study drug. Only subjects analyzed at Day 365 are reported. | Posted | Mean | Standard Deviation | g/L | Baseline (Day 1) and Day 365. |
|
| ||||||||||||||||||||||||||||||||||||
| Primary | Mean Percentage Change From Baseline in Hemoglobin at 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. | The ITT population set included all subjects eligible to receive study drug and who received ≥1 dose of study drug. Only subjects analyzed at Day 365 are reported. | Posted | Mean | Standard Deviation | percent change | Baseline (Day 1) and Day 365. |
|
| ||||||||||||||||||||||||||||||||||||
| Secondary | Mean Change From Baseline in Functional Assessment of Chronic Illness Therapy (FACIT)-Fatigue Score at 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. | The ITT population set included all subjects eligible to receive study drug and who received ≥1 dose of study drug. Only subjects analyzed at Day 365 are reported. | Posted | Mean | Standard Deviation | score on a scale | Baseline (Day 1) and Day 365. |
|
| ||||||||||||||||||||||||||||||||||||
| Secondary | Mean Change From Baseline in Absolute Reticulocyte Count (ARC) at 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. | The ITT population set included all subjects eligible to receive study drug and who received ≥1 dose of study drug. Only subjects analyzed at Day 365 are reported. | Posted | Mean | Standard Deviation | 10^9 cells/L | Baseline (Day 1) and Day 365. |
|
| ||||||||||||||||||||||||||||||||||||
| Secondary | Mean Percentage Change From Baseline in ARC at 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. | The ITT population set included all subjects eligible to receive study drug and who received ≥1 dose of study drug. Only subjects analyzed at Day 365 are reported. | Posted | Mean | Standard Deviation | percent change | Baseline (Day 1) and Day 365. |
|
| ||||||||||||||||||||||||||||||||||||
| Secondary | Mean Change From Baseline in Total Bilirubin at 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. | The ITT population set included all subjects eligible to receive study drug and who received ≥1 dose of study drug. Only subjects analyzed at Day 365 are reported. | Posted | Mean | Standard Deviation | micromole per liter (umol/L) | Baseline (Day 1) and Day 365. |
|
| ||||||||||||||||||||||||||||||||||||
| Secondary | Mean Percentage Change From Baseline in Total Bilirubin at 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. | The ITT population set included all subjects eligible to receive study drug and who received ≥1 dose of study drug. Only subjects analyzed at Day 365 are reported. | Posted | Mean | Standard Deviation | percent change | Baseline (Day 1) and Day 365. |
|
| ||||||||||||||||||||||||||||||||||||
| Secondary | Number of Subjects Receiving Red Blood Cell (RBC) Transfusions | The number of on-study RBC transfusions were monitored throughout the treatment period. | The ITT population set included all subjects eligible to receive study drug and who received ≥1 dose of study drug. | Posted | Count of Participants | Participants | No | From Day 1 up to Day 533. |
|
|
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.
The safety population set included all subjects eligible to receive study drug and who received ≥1 dose of study drug. One subject was enrolled in both cohorts.
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| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | Cohort 1 | Subjects received SC injections of pegcetacoplan 180 mg/day for up to 28 days. | 0 | 3 | 1 | 3 | 2 | 3 |
| EG001 | Cohort 2 | Subjects received SC injections or infusions of pegcetacoplan 270 mg/day for up to 364 days if entering the open-label extension study or, if entering Part 2C, until enrollment in the open-label extension study became available. If clinically indicated on the basis of response, the pegcetacoplan dosage could be increased up to 360 mg/day. | 1 | 20 | 6 | 20 | 17 | 20 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Hypersensitivity | Immune system disorders | MedDRA 21.0 | Systematic Assessment |
| |
| Abdominal neoplasm | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA 21.0 | Systematic Assessment |
| |
| Aplastic anaemia | Blood and lymphatic system disorders | MedDRA 21.0 | Systematic Assessment |
| |
| Intravascular haemolysis | Blood and lymphatic system disorders | MedDRA 21.0 | Systematic Assessment |
| |
| Acute kidney injury | Renal and urinary disorders | MedDRA 21.0 | Systematic Assessment |
| |
| Joint dislocation | Injury, poisoning and procedural complications | MedDRA 21.0 | Systematic Assessment |
| |
| Pneumonitis | Respiratory, thoracic and mediastinal disorders | MedDRA 21.0 | Systematic Assessment |
| |
| Haemolysis | Blood and lymphatic system disorders | MedDRA 21.0 | Systematic Assessment |
| |
| Paroxysmal nocturnal haemoglobinuria | Blood and lymphatic system disorders | MedDRA 21.0 | Systematic Assessment |
| |
| Cholecystitis acute | Hepatobiliary disorders | MedDRA 21.0 | Systematic Assessment |
| |
| Abdominal pain | Gastrointestinal disorders | MedDRA 21.0 | Systematic Assessment |
|
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Upper respiratory tract infection | Infections and infestations | MedDRA 21.0 | Systematic Assessment |
| |
| Pharyngitis | Infections and infestations | MedDRA 21.0 | Systematic Assessment |
| |
| Hordeolum | Infections and infestations | MedDRA 21.0 | Systematic Assessment |
| |
| Nasal abscess | Infections and infestations | MedDRA 21.0 | Systematic Assessment |
| |
| Nasopharyngitis | Infections and infestations | MedDRA 21.0 | Systematic Assessment |
| |
| Ophthalmic herpes zoster | Infections and infestations | MedDRA 21.0 | Systematic Assessment |
| |
| Paronychia | Infections and infestations | MedDRA 21.0 | Systematic Assessment |
| |
| Urinary tract infection | Infections and infestations | MedDRA 21.0 | Systematic Assessment |
| |
| Epistaxis | Respiratory, thoracic and mediastinal disorders | MedDRA 21.0 | Systematic Assessment |
| |
| Cough | Respiratory, thoracic and mediastinal disorders | MedDRA 21.0 | Systematic Assessment |
| |
| Nasal congestion | Respiratory, thoracic and mediastinal disorders | MedDRA 21.0 | Systematic Assessment |
| |
| Oropharyngeal pain | Respiratory, thoracic and mediastinal disorders | MedDRA 21.0 | Systematic Assessment |
| |
| Pleural effusion | Respiratory, thoracic and mediastinal disorders | MedDRA 21.0 | Systematic Assessment |
| |
| Productive cough | Respiratory, thoracic and mediastinal disorders | MedDRA 21.0 | Systematic Assessment |
| |
| Rhinorrhoea | Respiratory, thoracic and mediastinal disorders | MedDRA 21.0 | Systematic Assessment |
| |
| Injection site erythema | General disorders | MedDRA 21.0 | Systematic Assessment |
| |
| Pyrexia | General disorders | MedDRA 21.0 | Systematic Assessment |
| |
| Injection site induration | General disorders | MedDRA 21.0 | Systematic Assessment |
| |
| Injection site pain | General disorders | MedDRA 21.0 | Systematic Assessment |
| |
| Injection site swelling | General disorders | MedDRA 21.0 | Systematic Assessment |
| |
| Oedema peripheral | General disorders | MedDRA 21.0 | Systematic Assessment |
| |
| Abdominal pain upper | Gastrointestinal disorders | MedDRA 21.0 | Systematic Assessment |
| |
| Constipation | Gastrointestinal disorders | MedDRA 21.0 | Systematic Assessment |
| |
| Diarrhoea | Gastrointestinal disorders | MedDRA 21.0 | Systematic Assessment |
| |
| Gingival bleeding | Gastrointestinal disorders | MedDRA 21.0 | Systematic Assessment |
| |
| Mouth ulceration | Gastrointestinal disorders | MedDRA 21.0 | Systematic Assessment |
| |
| Vomiting | Gastrointestinal disorders | MedDRA 21.0 | Systematic Assessment |
| |
| Electrocardiogram QT prolonged | Investigations | MedDRA 21.0 | Systematic Assessment |
| |
| Alanine aminotransferase increased | Investigations | MedDRA 21.0 | Systematic Assessment |
| |
| Blood alkaline phosphatase increased | Investigations | MedDRA 21.0 | Systematic Assessment |
| |
| Blood lactate dehydrogenase increased | Investigations | MedDRA 21.0 | Systematic Assessment |
| |
| Gamma-glutamyltransferase increased | Investigations | MedDRA 21.0 | Systematic Assessment |
| |
| Liver function test abnormal | Investigations | MedDRA 21.0 | Systematic Assessment |
| |
| Serum ferritin decreased | Investigations | MedDRA 21.0 | Systematic Assessment |
| |
| Rash maculo-papular | Skin and subcutaneous tissue disorders | MedDRA 21.0 | Systematic Assessment |
| |
| Dry skin | Skin and subcutaneous tissue disorders | MedDRA 21.0 | Systematic Assessment |
| |
| Erythema | Skin and subcutaneous tissue disorders | MedDRA 21.0 | Systematic Assessment |
| |
| Petechiae | Skin and subcutaneous tissue disorders | MedDRA 21.0 | Systematic Assessment |
| |
| Purpura | Skin and subcutaneous tissue disorders | MedDRA 21.0 | Systematic Assessment |
| |
| Rash | Skin and subcutaneous tissue disorders | MedDRA 21.0 | Systematic Assessment |
| |
| Skin hypopigmentation | Skin and subcutaneous tissue disorders | MedDRA 21.0 | Systematic Assessment |
| |
| Neutropenia | Blood and lymphatic system disorders | MedDRA 21.0 | Systematic Assessment |
| |
| Haemolysis | Blood and lymphatic system disorders | MedDRA 21.0 | Systematic Assessment |
| |
| Thrombocytopenia | Blood and lymphatic system disorders | MedDRA 21.0 | Systematic Assessment |
| |
| Anaemia | Blood and lymphatic system disorders | MedDRA 21.0 | Systematic Assessment |
| |
| Febrile neutropenia | Blood and lymphatic system disorders | MedDRA 21.0 | Systematic Assessment |
| |
| Haemolytic anaemia | Blood and lymphatic system disorders | MedDRA 21.0 | Systematic Assessment |
| |
| Acute kidney injury | Renal and urinary disorders | MedDRA 21.0 | Systematic Assessment |
| |
| Chromaturia | Renal and urinary disorders | MedDRA 21.0 | Systematic Assessment |
| |
| Haematuria | Renal and urinary disorders | MedDRA 21.0 | Systematic Assessment |
| |
| Chronic kidney disease | Renal and urinary disorders | MedDRA 21.0 | Systematic Assessment |
| |
| Haemoglobinuria | Renal and urinary disorders | MedDRA 21.0 | Systematic Assessment |
| |
| Contusion | Injury, poisoning and procedural complications | MedDRA 21.0 | Systematic Assessment |
| |
| Ligament sprain | Injury, poisoning and procedural complications | MedDRA 21.0 | Systematic Assessment |
| |
| Post procedural haemorrhage | Injury, poisoning and procedural complications | MedDRA 21.0 | Systematic Assessment |
| |
| Road traffic accident | Injury, poisoning and procedural complications | MedDRA 21.0 | Systematic Assessment |
| |
| Hypokalaemia | Metabolism and nutrition disorders | MedDRA 21.0 | Systematic Assessment |
| |
| Hypoalbuminaemia | Metabolism and nutrition disorders | MedDRA 21.0 | Systematic Assessment |
| |
| Hypocalcaemia | Metabolism and nutrition disorders | MedDRA 21.0 | Systematic Assessment |
| |
| Dizziness | Nervous system disorders | MedDRA 21.0 | Systematic Assessment |
| |
| Hypoaesthesia | Nervous system disorders | MedDRA 21.0 | Systematic Assessment |
| |
| Lethargy | Nervous system disorders | MedDRA 21.0 | Systematic Assessment |
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| Cataract | Eye disorders | MedDRA 21.0 | Systematic Assessment |
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| Conjunctival haemorrhage | Eye disorders | MedDRA 21.0 | Systematic Assessment |
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| Dry eye | Eye disorders | MedDRA 21.0 | Systematic Assessment |
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| Arthralgia | Musculoskeletal and connective tissue disorders | MedDRA 21.0 | Systematic Assessment |
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| Musculoskeletal pain | Musculoskeletal and connective tissue disorders | MedDRA 21.0 | Systematic Assessment |
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| Pain in extremity | Musculoskeletal and connective tissue disorders | MedDRA 21.0 | Systematic Assessment |
| |
| Cholangitis | Hepatobiliary disorders | MedDRA 21.0 | Systematic Assessment |
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| Hepatic steatosis | Hepatobiliary disorders | MedDRA 21.0 | Systematic Assessment |
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| Haematoma | Vascular disorders | MedDRA 21.0 | Systematic Assessment |
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| Hypotension | Vascular disorders | MedDRA 21.0 | Systematic Assessment |
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| Bradycardia | Cardiac disorders | MedDRA 21.0 | Systematic Assessment |
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| Uterine haemorrhage | Reproductive system and breast disorders | MedDRA 21.0 | Systematic Assessment |
|
Not provided
Not provided
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Apellis Clinical Trial Information Line | Apellis Pharmaceuticals, Inc | 1-833-284-6361 | clinicaltrials@apellis.com |
| Prot_000.pdf |
| SAP | No | Yes | No | Statistical Analysis Plan | Dec 19, 2019 | Jul 22, 2020 | SAP_001.pdf |
| ID | Term |
|---|---|
| D006457 | Hemoglobinuria, Paroxysmal |
| D000740 | Anemia |
| D006456 | Hemoglobinuria |
| D006461 | Hemolysis |
| D006402 | Hematologic Diseases |
| ID | Term |
|---|---|
| D000743 | Anemia, Hemolytic |
| D006425 | Hemic and Lymphatic Diseases |
| D009190 | Myelodysplastic Syndromes |
| D001855 | Bone Marrow Diseases |
| D011507 | Proteinuria |
| D014555 | Urination Disorders |
| D014570 | Urologic Diseases |
| D052776 | Female Urogenital Diseases |
| D005261 | Female Urogenital Diseases and Pregnancy Complications |
| D000091642 | Urogenital Diseases |
| D052801 | Male Urogenital Diseases |
| D020924 | Urological Manifestations |
| D012816 | Signs and Symptoms |
| D013568 | Pathological Conditions, Signs and Symptoms |
| D010335 | Pathologic Processes |
Not provided
Not provided
| ID | Term |
|---|---|
| C000716074 | pegcetacoplan |
Not provided
Not provided
Not provided
| >=65 years |
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| Male |
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| Asian |
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| Native Hawaiian or Other Pacific Islander |
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| Maori |
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| Other |
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| Unknown |
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| Serious AEs |
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| TEAEs leading to study drug discontinuation |
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| TEAEs leading to death |
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| TEAEs with mild intensity |
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| TEAEs with moderate intensity |
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| TEAEs with severe intensity |
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| TEAEs with life threatening intensity |
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