Trial · NCT03500549
Study to Evaluate the Efficacy and Safety of APL-2 in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH)
NCT03500549, Phase 3, Completed, Apellis Pharmaceuticals, Inc.; results first posted 2022-03-25
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. Study to Evaluate the Efficacy and Safety of APL-2 in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH). Trials, record NCT03500549. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT03500549
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Study to Evaluate the Efficacy and Safety of APL-2 in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH)
- NCT number
- NCT03500549
- Status
- Completed
- Phases
- Phase 3
- Enrollment
- 80 participants, actual
- Lead sponsor
- Apellis Pharmaceuticals, Inc.
- Conditions
- Paroxysmal Nocturnal Hemoglobinuria
- Interventions
- Pegcetacoplan, Soliris
- First posted
- 2018-04-18
- Last updated
- 2022-03-25
- Results first posted
- 2022-03-25
Source ClinicalTrials.gov
Registered outcomes26
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Least Squares (LS) Mean Change From Baseline to Week 16 in Hemoglobin (Hb) Level During the RCP | Baseline and Week 16 | 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. |
| Secondary | Percentage of Subjects Who Did Not Require a Transfusion (Transfusion Avoidance) During the RCP | Day 1 to Week 16 | Subjects who experienced more than 1 transfusion during the RCP are only counted once. Subjects who did not have a transfusion but withdrew before Week 16 were considered as having a transfusion in the analysis of transfusion avoidance. |
| Secondary | LS Mean Change From Baseline to Week 16 in Absolute Reticulocyte Count (ARC) During the RCP | Baseline and Week 16 | Baseline was the average of available measurements recorded from central laboratory before taking the first dose of pegcetacoplan. Analysis excluded data before the RCP and was censored for transfusions. |
| Secondary | LS Mean Change From Baseline to Week 16 in Lactate Dehydrogenase (LDH) Level During the RCP | Baseline and Week 16 | Baseline was the average of available measurements recorded from central laboratory before taking the first dose of pegcetacoplan. Analysis excluded data before the RCP and was censored for transfusions. |
| Secondary | LS Mean Change From Baseline to Week 16 in Functional Assessment of Chronic Illness Therapy (FACIT) - Fatigue Scale Score During the RCP | Baseline and Week 16 | The FACIT-fatigue scale version 4 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). With 13 statements the total score had a range of 0 to 52. A higher score corresponds to a higher quality of life (QoL). Baseline was the last available, nonmissing observation before taking the first dose of pegcetacoplan. Data collected after transfusion is excluded from analysis. |
| Secondary | Percentage of Subjects Who Achieved a Hb Response in the Absence of Transfusions at Week 16 | Baseline and Week 16 | Hb response was defined as an increase of at least 1 g/dL in Hb from Baseline at Week 16. 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. |
| Secondary | Percentage of Subjects Who Achieved Reticulocyte Normalization in the Absence of Transfusions at Week 16 | Week 16 | Reticulocyte normalization was defined as the ARC being below the upper limit of the gender-specific normal range at Week 16, censored for transfusions. Subjects who received a transfusion between Day 1 and Week 16 or withdrew without providing efficacy data at Week 16 were classified as nonresponders. |
| Secondary | Percentage of Subjects Who Achieved Hb Normalization in the Absence of Transfusions at Week 16 | Week 16 | Hb normalization was defined as the Hb level being above the lower limit of the normal range at Week 16, censored for transfusions. Subjects who received a transfusion between Day 1 and Week 16 or withdrew without providing efficacy data at Week 16 are classified as nonnormalization. |
| Secondary | LS Mean Change From Baseline to Week 16 in Indirect Bilirubin Level During the RCP | Baseline and Week 16 | Baseline was the average of available measurements recorded from central laboratory before taking the first dose of pegcetacoplan. Analysis excluded data before the RCP and was censored for transfusions. |
| Secondary | LS Mean Change From Baseline to Week 16 in Haptoglobin Level During the RCP | Baseline and Week 16 | Baseline was the average of available measurements recorded from central laboratory before taking the first dose of pegcetacoplan. Analysis excluded data before the RCP and was censored for transfusions. |
1 to 10 of 26Source ClinicalTrials.gov
See all 26 registered outcomesOutcomes26
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Least Squares (LS) Mean Change From Baseline to Week 16 in Hemoglobin (Hb) Level During the RCP | Baseline and Week 16 | The ITT set included all randomized subjects. | Grams per deciliter (g/dL) | LEAST_SQUARES_MEAN |
| SECONDARY | Percentage of Subjects Who Did Not Require a Transfusion (Transfusion Avoidance) During the RCP | Day 1 to Week 16 | The ITT set included all randomized subjects. | Percentage of subjects | NUMBER |
| SECONDARY | LS Mean Change From Baseline to Week 16 in Absolute Reticulocyte Count (ARC) During the RCP | Baseline and Week 16 | The ITT set included all randomized subjects. | 10^9 cells/ liter (L) | LEAST_SQUARES_MEAN |
| SECONDARY | LS Mean Change From Baseline to Week 16 in Lactate Dehydrogenase (LDH) Level During the RCP | Baseline and Week 16 | The ITT set included all randomized subjects. | Units (U)/L | LEAST_SQUARES_MEAN |
| SECONDARY | LS Mean Change From Baseline to Week 16 in Functional Assessment of Chronic Illness Therapy (FACIT) - Fatigue Scale Score During the RCP | Baseline and Week 16 | The ITT set included all randomized subjects. | Score on a scale | LEAST_SQUARES_MEAN |
| SECONDARY | Percentage of Subjects Who Achieved a Hb Response in the Absence of Transfusions at Week 16 | Baseline and Week 16 | The ITT set included all randomized subjects. | Percentage of subjects | NUMBER |
| SECONDARY | Percentage of Subjects Who Achieved Reticulocyte Normalization in the Absence of Transfusions at Week 16 | Week 16 | The ITT set includes all randomized subjects. | Percentage of subjects | NUMBER |
| SECONDARY | Percentage of Subjects Who Achieved Hb Normalization in the Absence of Transfusions at Week 16 | Week 16 | The ITT set included all randomized subjects. | Percentage of subjects | NUMBER |
| SECONDARY | LS Mean Change From Baseline to Week 16 in Indirect Bilirubin Level During the RCP | Baseline and Week 16 | The ITT set included all randomized subjects. | Micromole (μmol)/L | LEAST_SQUARES_MEAN |
| SECONDARY | LS Mean Change From Baseline to Week 16 in Haptoglobin Level During the RCP | Baseline and Week 16 | The ITT set included all randomized subjects. | g/L | LEAST_SQUARES_MEAN |
1 to 10 of 26Source ClinicalTrials.gov
See all 26 outcomesResults by group137
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Least Squares (LS) Mean Change From Baseline to Week 16 in Hemoglobin (Hb) Level During the RCP | Pegcetacoplan to Pegcetacoplan | 2.37 | Standard Error 0.363 | Grams per deciliter (g/dL) | |
| Least Squares (LS) Mean Change From Baseline to Week 16 in Hemoglobin (Hb) Level During the RCP | Eculizumab to Pegcetacoplan | -1.47 | Standard Error 0.666 | Grams per deciliter (g/dL) | |
| Percentage of Subjects Who Did Not Require a Transfusion (Transfusion Avoidance) During the RCP | Pegcetacoplan to Pegcetacoplan | 85.4 | Percentage of subjects | ||
| Percentage of Subjects Who Did Not Require a Transfusion (Transfusion Avoidance) During the RCP | Eculizumab to Pegcetacoplan | 15.4 | Percentage of subjects | ||
| LS Mean Change From Baseline to Week 16 in Absolute Reticulocyte Count (ARC) During the RCP | Pegcetacoplan to Pegcetacoplan | -135.82 | Standard Error 6.543 | 10^9 cells/ liter (L) | |
| LS Mean Change From Baseline to Week 16 in Absolute Reticulocyte Count (ARC) During the RCP | Eculizumab to Pegcetacoplan | 27.79 | Standard Error 11.859 | 10^9 cells/ liter (L) | |
| LS Mean Change From Baseline to Week 16 in Lactate Dehydrogenase (LDH) Level During the RCP | Pegcetacoplan to Pegcetacoplan | -14.76 | Standard Error 42.708 | Units (U)/L | |
| LS Mean Change From Baseline to Week 16 in Lactate Dehydrogenase (LDH) Level During the RCP | Eculizumab to Pegcetacoplan | -10.12 | Standard Error 71.025 | Units (U)/L | |
| LS Mean Change From Baseline to Week 16 in Functional Assessment of Chronic Illness Therapy (FACIT) - Fatigue Scale Score During the RCP | Pegcetacoplan to Pegcetacoplan | 9.22 | Standard Error 1.607 | Score on a scale | |
| LS Mean Change From Baseline to Week 16 in Functional Assessment of Chronic Illness Therapy (FACIT) - Fatigue Scale Score During the RCP | Eculizumab to Pegcetacoplan | -2.65 | Standard Error 2.821 | Score on a scale |
1 to 10 of 137Source ClinicalTrials.gov
See all 137 results by groupParticipant flow18
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Run-in Period (Day -28 to ≤Day 1) | STARTED | Pegcetacoplan to Pegcetacoplan | 41 | |
| Run-in Period (Day -28 to ≤Day 1) | STARTED | Eculizumab to Pegcetacoplan | 39 | |
| Run-in Period (Day -28 to ≤Day 1) | COMPLETED | Pegcetacoplan to Pegcetacoplan | 41 | |
| Run-in Period (Day -28 to ≤Day 1) | COMPLETED | Eculizumab to Pegcetacoplan | 39 | |
| Run-in Period (Day -28 to ≤Day 1) | NOT COMPLETED | Pegcetacoplan to Pegcetacoplan | 0 | |
| Run-in Period (Day -28 to ≤Day 1) | NOT COMPLETED | Eculizumab to Pegcetacoplan | 0 | |
| RCP (Day 1 - Week 16) | STARTED | Pegcetacoplan to Pegcetacoplan | 41 | |
| RCP (Day 1 - Week 16) | STARTED | Eculizumab to Pegcetacoplan | 39 | |
| RCP (Day 1 - Week 16) | COMPLETED | Pegcetacoplan to Pegcetacoplan | 38 | |
| RCP (Day 1 - Week 16) | COMPLETED | Eculizumab to Pegcetacoplan | 39 |
1 to 10 of 18Source ClinicalTrials.gov
See all 18 participant flowAdverse events336
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 | Hypertension | Vascular disorders | Run-in Period: Pegcetacoplan + Eculizumab | 0 | 80 | 0 |
| other | Hypertension | Vascular disorders | Open-label Period: Pegcetacoplan | 3 | 77 | 4 |
| other | Hypertension | Vascular disorders | RCP: Eculizumab | 1 | 39 | 1 |
| other | Hypertension | Vascular disorders | RCP: Pegcetacoplan | 3 | 41 | 3 |
| other | Injection site erythema | General disorders | Run-in Period: Pegcetacoplan + Eculizumab | 33 | 80 | 75 |
| other | Injection site erythema | General disorders | Open-label Period: Pegcetacoplan | 9 | 77 | 127 |
| other | Injection site erythema | General disorders | RCP: Eculizumab | 0 | 39 | 0 |
| other | Injection site erythema | General disorders | RCP: Pegcetacoplan | 7 | 41 | 44 |
| other | Fatigue | General disorders | Run-in Period: Pegcetacoplan + Eculizumab | 5 | 80 | 5 |
| other | Fatigue | General disorders | Open-label Period: Pegcetacoplan | 8 | 77 | 11 |
1 to 10 of 336Source ClinicalTrials.gov
See all 336 adverse eventsAdverse event totals12
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Run-in Period: Pegcetacoplan + Eculizumab | 0 | 80 |
| Total, all-cause mortality | Open-label Period: Pegcetacoplan | 1 | 77 |
| Total, all-cause mortality | RCP: Eculizumab | 0 | 39 |
| Total, all-cause mortality | RCP: Pegcetacoplan | 0 | 41 |
| Total, other adverse events | Run-in Period: Pegcetacoplan + Eculizumab | 71 | 80 |
| Total, other adverse events | Open-label Period: Pegcetacoplan | 71 | 77 |
| Total, other adverse events | RCP: Eculizumab | 36 | 39 |
| Total, other adverse events | RCP: Pegcetacoplan | 36 | 41 |
| Total, serious adverse events | Run-in Period: Pegcetacoplan + Eculizumab | 4 | 80 |
| Total, serious adverse events | Open-label Period: Pegcetacoplan | 18 | 77 |
1 to 10 of 12Source ClinicalTrials.gov
See all 12 adverse event totalsPapers11
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 comparisons0
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
None in this release.
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/NCT03500549
GET https://conductscience.org/api/trials/v1/NCT03500549/registered?page=2