Trial · NCT02588833
Pilot Study to Assess Safety, Preliminary Efficacy and Pharmacokinetics of S.C. Pegcetacoplan (APL-2) in PNH Subjects.
NCT02588833, Phase 1, Completed, Apellis Pharmaceuticals, Inc.; results first posted 2021-01-11
3 sourcestrials-2026-11-12-0.5.0
Download JSON
Cite this record
ConductScience Library. Pilot Study to Assess Safety, Preliminary Efficacy and Pharmacokinetics of S.C. Pegcetacoplan (APL-2) in PNH Subjects. Trials, record NCT02588833. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT02588833
On this page
Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Pilot Study to Assess Safety, Preliminary Efficacy and Pharmacokinetics of S.C. Pegcetacoplan (APL-2) in PNH Subjects.
- NCT number
- NCT02588833
- Status
- Completed
- Phases
- Phase 1
- Enrollment
- 23 participants, actual
- Lead sponsor
- Apellis Pharmaceuticals, Inc.
- Conditions
- Paroxysmal Nocturnal Hemoglobinuria
- Interventions
- Pegcetacoplan
- First posted
- 2015-10-28
- Last updated
- 2021-01-11
- Results first posted
- 2021-01-11
Source ClinicalTrials.gov
Registered outcomes13
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Number of Subjects With Treatment Emergent Adverse Events (TEAEs) Including by Severity | 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 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. |
| Primary | Mean Change From Baseline in Lactate Dehydrogenase (LDH) at Day 365 | 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. |
| Primary | Mean Percentage Change From Baseline in LDH at Day 365 | 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. |
| Primary | Mean Change From Baseline in Haptoglobin at Day 365 | 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. |
| Primary | Mean Percentage Change From Baseline in Haptoglobin at Day 365 | 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. |
| Primary | Mean Change From Baseline in Hemoglobin at Day 365 | 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. |
| Primary | Mean Percentage Change From Baseline in Hemoglobin at Day 365 | 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. |
| Secondary | Mean Change From Baseline in Functional Assessment of Chronic Illness Therapy (FACIT)-Fatigue Score at Day 365 | 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. |
| Secondary | Mean Change From Baseline in Absolute Reticulocyte Count (ARC) at Day 365 | 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. |
| Secondary | Mean Percentage Change From Baseline in ARC at Day 365 | 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. |
1 to 10 of 13Source ClinicalTrials.gov
See all 13 registered outcomesOutcomes13
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Number of Subjects With Treatment Emergent Adverse Events (TEAEs) Including by Severity | 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. | Participants | COUNT_OF_PARTICIPANTS |
| PRIMARY | Mean Change From Baseline in Lactate Dehydrogenase (LDH) at Day 365 | Baseline (Day 1) and Day 365. | 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. | units per liter (U/L) | MEAN |
| PRIMARY | Mean Percentage Change From Baseline in LDH at Day 365 | Baseline (Day 1) and Day 365. | 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. | percent change | MEAN |
| PRIMARY | Mean Change From Baseline in Haptoglobin at Day 365 | Baseline (Day 1) and Day 365. | 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. | gram per liter (g/L) | MEAN |
| PRIMARY | Mean Percentage Change From Baseline in Haptoglobin at Day 365 | Baseline (Day 1) and Day 365. | 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. | percent change | MEAN |
| PRIMARY | Mean Change From Baseline in Hemoglobin at Day 365 | Baseline (Day 1) and Day 365. | 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. | g/L | MEAN |
| PRIMARY | Mean Percentage Change From Baseline in Hemoglobin at Day 365 | Baseline (Day 1) and Day 365. | 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. | percent change | MEAN |
| SECONDARY | Mean Change From Baseline in Functional Assessment of Chronic Illness Therapy (FACIT)-Fatigue Score at Day 365 | Baseline (Day 1) and Day 365. | 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. | score on a scale | MEAN |
| SECONDARY | Mean Change From Baseline in Absolute Reticulocyte Count (ARC) at Day 365 | Baseline (Day 1) and Day 365. | 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. | 10^9 cells/L | MEAN |
| SECONDARY | Mean Percentage Change From Baseline in ARC at Day 365 | Baseline (Day 1) and Day 365. | 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. | percent change | MEAN |
1 to 10 of 13Source ClinicalTrials.gov
See all 13 outcomesResults by group31
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Number of Subjects With Treatment Emergent Adverse Events (TEAEs) Including by Severity | Cohort 1 | All TEAEs | 2 | Participants | |
| Number of Subjects With Treatment Emergent Adverse Events (TEAEs) Including by Severity | Cohort 2 | All TEAEs | 18 | Participants | |
| Number of Subjects With Treatment Emergent Adverse Events (TEAEs) Including by Severity | Cohort 1 | Treatment related TEAEs | 2 | Participants | |
| Number of Subjects With Treatment Emergent Adverse Events (TEAEs) Including by Severity | Cohort 2 | Treatment related TEAEs | 9 | Participants | |
| Number of Subjects With Treatment Emergent Adverse Events (TEAEs) Including by Severity | Cohort 1 | Serious AEs | 1 | Participants | |
| Number of Subjects With Treatment Emergent Adverse Events (TEAEs) Including by Severity | Cohort 2 | Serious AEs | 6 | Participants | |
| Number of Subjects With Treatment Emergent Adverse Events (TEAEs) Including by Severity | Cohort 1 | TEAEs leading to study drug discontinuation | 1 | Participants | |
| Number of Subjects With Treatment Emergent Adverse Events (TEAEs) Including by Severity | Cohort 2 | TEAEs leading to study drug discontinuation | 2 | Participants | |
| Number of Subjects With Treatment Emergent Adverse Events (TEAEs) Including by Severity | Cohort 1 | TEAEs leading to death | 0 | Participants | |
| Number of Subjects With Treatment Emergent Adverse Events (TEAEs) Including by Severity | Cohort 2 | TEAEs leading to death | 1 | Participants |
1 to 10 of 31Source ClinicalTrials.gov
See all 31 results by groupParticipant flow30
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Part 1 | STARTED | Cohort 1 | 3 | |
| Part 1 | STARTED | Cohort 2 | 20 | |
| Part 1 | COMPLETED | Cohort 1 | 2 | |
| Part 1 | COMPLETED | Cohort 2 | 20 | |
| Part 1 | NOT COMPLETED | Cohort 1 | 1 | |
| Part 1 | NOT COMPLETED | Cohort 2 | 0 | |
| Part 2A | STARTED | Cohort 1 | 0 | |
| Part 2A | STARTED | Cohort 2 | 20 | |
| Part 2A | COMPLETED | Cohort 1 | 0 | |
| Part 2A | COMPLETED | Cohort 2 | 18 |
1 to 10 of 30Source ClinicalTrials.gov
See all 30 participant flowAdverse events170
Each adverse event the trial posted, group by group: the participants affected out of those at risk.
| Type | Event | Organ system | Group | Affected | At risk | Events |
|---|---|---|---|---|---|---|
| other | Upper respiratory tract infection | Infections and infestations | Cohort 1 | 0 | 3 | 0 |
| other | Upper respiratory tract infection | Infections and infestations | Cohort 2 | 5 | 20 | 6 |
| other | Pharyngitis | Infections and infestations | Cohort 1 | 0 | 3 | 0 |
| other | Pharyngitis | Infections and infestations | Cohort 2 | 2 | 20 | 2 |
| other | Hordeolum | Infections and infestations | Cohort 1 | 0 | 3 | 0 |
| other | Hordeolum | Infections and infestations | Cohort 2 | 1 | 20 | 2 |
| other | Nasal abscess | Infections and infestations | Cohort 1 | 0 | 3 | 0 |
| other | Nasal abscess | Infections and infestations | Cohort 2 | 1 | 20 | 1 |
| other | Nasopharyngitis | Infections and infestations | Cohort 1 | 0 | 3 | 0 |
| other | Nasopharyngitis | Infections and infestations | Cohort 2 | 1 | 20 | 1 |
1 to 10 of 170Source ClinicalTrials.gov
See all 170 adverse eventsAdverse event totals6
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Cohort 1 | 0 | 3 |
| Total, all-cause mortality | Cohort 2 | 1 | 20 |
| Total, other adverse events | Cohort 1 | 2 | 3 |
| Total, other adverse events | Cohort 2 | 17 | 20 |
| Total, serious adverse events | Cohort 1 | 1 | 3 |
| Total, serious adverse events | Cohort 2 | 6 | 20 |
Source ClinicalTrials.gov
Papers2
The papers the trial lists on ClinicalTrials.gov.
Grants0
The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.
None in this release.
Trial comparisons3
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
| Comparison | Source |
|---|---|
| Cohort 1 vs Cohort 2: Number of Subjects Receiving Red Blood Cell (RBC) Transfusions | Trial comparisons |
| Cohort 1 vs Cohort 2: deaths from any cause | Trial comparisons |
| Cohort 1 vs Cohort 2: serious adverse events | Trial comparisons |
Tests0
The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.
None in this release.
Data dates
The day each source was copied for this release.
Sources 3
- ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
- ClinicalTrials.govNo license stated
- NIH RePORTER's clinical studies and project filesPublic domain
Each source's terms
- ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
- ClinicalTrials.govnone stated
- NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)
ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.
For software
Read this trial from software.
The record and each of its lists are JSON, twenty rows a page. No key.
GET https://conductscience.org/api/trials/v1/NCT02588833
GET https://conductscience.org/api/trials/v1/NCT02588833/results?page=2