Trial · NCT04085601
A Study to Evaluate the Efficacy and Safety of Pegcetacoplan in Patients With PNH
NCT04085601, Phase 3, Completed, Apellis Pharmaceuticals, Inc.; results first posted 2022-10-21
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. A Study to Evaluate the Efficacy and Safety of Pegcetacoplan in Patients With PNH. Trials, record NCT04085601. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT04085601
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- A Study to Evaluate the Efficacy and Safety of Pegcetacoplan in Patients With PNH
- NCT number
- NCT04085601
- Status
- Completed
- Phases
- Phase 3
- Enrollment
- 53 participants, actual
- Lead sponsor
- Apellis Pharmaceuticals, Inc.
- Conditions
- Paroxysmal Nocturnal Hemoglobinuria
- Interventions
- APL-2
- First posted
- 2019-09-11
- Last updated
- 2022-10-21
- Results first posted
- 2022-10-21
Source ClinicalTrials.gov
Registered outcomes16
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Number of Subjects Who Achieved Hemoglobin (Hb) Stabilization | From Baseline (Day 1) up to Week 26 | The Hb stabilization was defined as avoidance of a \>1 gram per deciliter (g/dL) decrease in Hb concentration from Baseline in the absence of transfusion through Week 26. |
| Primary | Change From Baseline in Lactate Dehydrogenase (LDH) Concentration At Week 26 | Baseline (Day 1) and Week 26 | The LDH concentration was analyzed using an analysis of covariance (ANCOVA) model with a last observation carried forward (LOCF) and a baseline observation carried forward (BOCF) approach for handling missing data. Baseline was defined as average of measurements prior to first dose of pegcetacoplan or on or prior to randomization of SoC. Post baseline missing values are imputed using multiple imputation method with Markov Chain Mont Carlo method. |
| Secondary | Number of Subjects With an Hb Response in the Absence of Transfusions | Baseline and Week 26 | An Hb response was defined as a =\>1 g/dL increase in Hb from baseline at Week 26. |
| Secondary | Change From Baseline in Absolute Reticulocyte Count (ARC) at Week 26 | Baseline and Week 26 | Blood samples were collected via direct venipuncture at the specific time points to determine ARC. Baseline was defined as average of measurements prior to first dose of pegcetacoplan or on or prior to randomization of SoC. Post baseline missing values are imputed using multiple imputation method with Markov Chain Mont Carlo method. |
| Secondary | Change From Baseline in Hb Concentration at Week 26 | Baseline and Week 26 | Baseline was defined as average of measurements prior to first dose of pegcetacoplan or on or prior to randomization of SoC. Post baseline missing values are imputed using multiple imputation method with Markov Chain Mont Carlo method. |
| Secondary | Percentage of Subjects Who Received Transfusion or Decrease of Hb >2 g/dL From Baseline | At Week 26 | Transfusion refers to any transfusion of PRBC, leukocyte-depleted red blood cells (LDPRC), leukocyte poor packed red blood cell (LPRC), leukocyte poor blood (LPB) or whole blood. |
| Secondary | Percentage of Subjects With Transfusion Avoidance | At Week 26 | Transfusion avoidance was defined as the percentage of subjects who did not require a transfusion during the RCP. Transfusion refers to any transfusion of PRBC, LDPRC, LPRC, LPB or whole blood. |
| Secondary | Number of PRBC Units Transfused From Baseline Through Week 26 | Up to Week 26 | The number of units of PRBC transfusions was estimated. In one transfusion subjects received one or more units. |
| Secondary | Change From Baseline in Functional Assessment of Chronic Illness Therapy- (FACIT-Fatigue) Scale Score at Week 26 | Baseline and Week 26 | The FACIT-Fatigue Scale is a 13-item Likert scaled instrument that is self-administered by the subjects during clinic visits. Subjects were presented with 13 statements and asked to indicate their responses as it applied to the past 7 days. The 5 possible responses are "Not at all" (0), "A little bit" (1), "Somewhat" (2), "Quite a bit" (3), and "Very much" (4). With 13 statements, the total score has a range of 0 to 52. The higher score corresponded to a higher quality of life. Baseline is defined as average of measurements prior to first dose of pegcetacoplan or on or prior to randomization of SoC. Post baseline missing values are imputed using multiple imputation method with Markov Chain Mont Carlo method. |
| Secondary | Percentage of Subjects With Hb Normalization Levels at Week 26 | Baseline and Week 26 | Normalization of Hb levels defined as \>= 1x LLN at Week 26 in the absence of transfusion. Transfusion refers to any transfusion of PRBC, LDPRC, LPRC, LPB or whole blood. |
1 to 10 of 16Source ClinicalTrials.gov
See all 16 registered outcomesOutcomes16
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Number of Subjects Who Achieved Hemoglobin (Hb) Stabilization | From Baseline (Day 1) up to Week 26 | The ITT set included all subjects assigned to treatment. | Participants | COUNT_OF_PARTICIPANTS |
| PRIMARY | Change From Baseline in Lactate Dehydrogenase (LDH) Concentration At Week 26 | Baseline (Day 1) and Week 26 | The ITT set included all subjects assigned to treatment. | Units/Liter (U/L) | LEAST_SQUARES_MEAN |
| SECONDARY | Number of Subjects With an Hb Response in the Absence of Transfusions | Baseline and Week 26 | The ITT set included all subjects assigned to treatment. | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Change From Baseline in Absolute Reticulocyte Count (ARC) at Week 26 | Baseline and Week 26 | The ITT set included all subjects assigned to treatment. | 10^9 cells/L | LEAST_SQUARES_MEAN |
| SECONDARY | Change From Baseline in Hb Concentration at Week 26 | Baseline and Week 26 | The ITT set included all subjects assigned to treatment. | g/dL | LEAST_SQUARES_MEAN |
| SECONDARY | Percentage of Subjects Who Received Transfusion or Decrease of Hb >2 g/dL From Baseline | At Week 26 | The ITT set included all subjects assigned to treatment. | percentage of subjects | NUMBER |
| SECONDARY | Percentage of Subjects With Transfusion Avoidance | At Week 26 | The ITT set included all subjects assigned to treatment. | percentage of subjects | NUMBER |
| SECONDARY | Number of PRBC Units Transfused From Baseline Through Week 26 | Up to Week 26 | The ITT set included all subjects assigned to treatment. | PRBC transfusions | MEDIAN |
| SECONDARY | Change From Baseline in Functional Assessment of Chronic Illness Therapy- (FACIT-Fatigue) Scale Score at Week 26 | Baseline and Week 26 | The ITT set included all subjects assigned to treatment. | scores on a scale | LEAST_SQUARES_MEAN |
| SECONDARY | Percentage of Subjects With Hb Normalization Levels at Week 26 | Baseline and Week 26 | The ITT set included all subjects assigned to treatment. | percentage of subjects | NUMBER |
1 to 10 of 16Source ClinicalTrials.gov
See all 16 outcomesResults by group32
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Number of Subjects Who Achieved Hemoglobin (Hb) Stabilization | Pegcetacoplan | 30 | Participants | ||
| Number of Subjects Who Achieved Hemoglobin (Hb) Stabilization | Standard of Care | 0 | Participants | ||
| Change From Baseline in Lactate Dehydrogenase (LDH) Concentration At Week 26 | Pegcetacoplan | -1870.47 | Standard Error 100.971 | Units/Liter (U/L) | |
| Change From Baseline in Lactate Dehydrogenase (LDH) Concentration At Week 26 | Standard of Care | -400.09 | Standard Error 312.988 | Units/Liter (U/L) | |
| Number of Subjects With an Hb Response in the Absence of Transfusions | Pegcetacoplan | 25 | Participants | ||
| Number of Subjects With an Hb Response in the Absence of Transfusions | Standard of Care | 1 | Participants | ||
| Change From Baseline in Absolute Reticulocyte Count (ARC) at Week 26 | Pegcetacoplan | -123.26 | Standard Error 9.164 | 10^9 cells/L | |
| Change From Baseline in Absolute Reticulocyte Count (ARC) at Week 26 | Standard of Care | -19.44 | Standard Error 25.209 | 10^9 cells/L | |
| Change From Baseline in Hb Concentration at Week 26 | Pegcetacoplan | 2.94 | Standard Error 0.383 | g/dL | |
| Change From Baseline in Hb Concentration at Week 26 | Standard of Care | 0.27 | Standard Error 0.759 | g/dL |
1 to 10 of 32Source ClinicalTrials.gov
See all 32 results by groupParticipant flow8
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | Pegcetacoplan | 35 | |
| Overall Study | STARTED | Standard of Care | 18 | |
| Overall Study | SoC to Pegcetacoplan (Escape Therapy) | Pegcetacoplan | 0 | |
| Overall Study | SoC to Pegcetacoplan (Escape Therapy) | Standard of Care | 11 | |
| Overall Study | COMPLETED | Pegcetacoplan | 33 | |
| Overall Study | COMPLETED | Standard of Care | 17 | |
| Overall Study | NOT COMPLETED | Pegcetacoplan | 2 | |
| Overall Study | NOT COMPLETED | Standard of Care | 1 |
Source ClinicalTrials.gov
Adverse events100
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 | Pyrexia | General disorders | Overall Pegcetacoplan | 4 | 46 | 4 |
| other | Pyrexia | General disorders | Standard of Care | 0 | 18 | 0 |
| other | Fatigue | General disorders | Overall Pegcetacoplan | 1 | 46 | 1 |
| other | Fatigue | General disorders | Standard of Care | 1 | 18 | 2 |
| other | Malaise | General disorders | Overall Pegcetacoplan | 0 | 46 | 0 |
| other | Malaise | General disorders | Standard of Care | 1 | 18 | 1 |
| other | Ecchymosis | Skin and subcutaneous tissue disorders | Overall Pegcetacoplan | 3 | 46 | 4 |
| other | Ecchymosis | Skin and subcutaneous tissue disorders | Standard of Care | 0 | 18 | 0 |
| other | Erythema | Skin and subcutaneous tissue disorders | Overall Pegcetacoplan | 3 | 46 | 4 |
| other | Erythema | Skin and subcutaneous tissue disorders | Standard of Care | 0 | 18 | 0 |
1 to 10 of 100Source ClinicalTrials.gov
See all 100 adverse eventsAdverse event totals6
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Overall Pegcetacoplan | 1 | 46 |
| Total, all-cause mortality | Standard of Care | 1 | 18 |
| Total, other adverse events | Overall Pegcetacoplan | 35 | 46 |
| Total, other adverse events | Standard of Care | 12 | 18 |
| Total, serious adverse events | Overall Pegcetacoplan | 6 | 46 |
| Total, serious adverse events | Standard of Care | 3 | 18 |
Source ClinicalTrials.gov
Papers8
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 comparisons5
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
| Comparison | Source |
|---|---|
| Pegcetacoplan vs Standard of Care: Number of Subjects With an Hb Response in the Absence of Transfusions | Trial comparisons |
| Pegcetacoplan vs Standard of Care: Number of Subjects With Failure of Hb Stabilization | Trial comparisons |
| Overall Pegcetacoplan vs Standard of Care: deaths from any cause | Trial comparisons |
| Pegcetacoplan vs Standard of Care: Number of Subjects Who Achieved Hemoglobin (Hb) Stabilization | Trial comparisons |
| Overall Pegcetacoplan vs Standard of Care: 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/NCT04085601
GET https://conductscience.org/api/trials/v1/NCT04085601/results?page=2