Trial · NCT03226678
Study to Assess the Safety, Tolerability, Efficacy and PK of APL-2 in Patients With Warm Type Autoimmune Hemolytic Anemia (wAIHA) or Cold Agglutinin Disease (CAD)
NCT03226678, Phase 2, Completed, Apellis Pharmaceuticals, Inc.; results first posted 2024-12-12
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. Study to Assess the Safety, Tolerability, Efficacy and PK of APL-2 in Patients With Warm Type Autoimmune Hemolytic Anemia (wAIHA) or Cold Agglutinin Disease (CAD). Trials, record NCT03226678. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT03226678
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Study to Assess the Safety, Tolerability, Efficacy and PK of APL-2 in Patients With Warm Type Autoimmune Hemolytic Anemia (wAIHA) or Cold Agglutinin Disease (CAD)
- NCT number
- NCT03226678
- Status
- Completed
- Phases
- Phase 2
- Enrollment
- 24 participants, actual
- Lead sponsor
- Apellis Pharmaceuticals, Inc.
- Conditions
- Warm Autoimmune Hemolytic Anemia, Cold Agglutinin Disease
- Interventions
- APL-2
- First posted
- 2017-07-24
- Last updated
- 2024-12-12
- Results first posted
- 2024-12-12
Source ClinicalTrials.gov
Registered outcomes10
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 | Part A:From first dose of study drug (Part A Day 1) up to 30days after last dose of study drug in Part A,approximately 366days Part B:From first dose of study drug (Part B Day 1) up to 56days after last dose of study drug in Part B,approximately 980days | TEAEs were defined as adverse events (AEs) that occurred after dosing on Day 1 and up to 56 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. |
| Secondary | Mean Change From Baseline in Hemoglobin at Weeks 48 and 132 | Part A: Baseline (Day 1 of Part A) and Week 48; Part B: Baseline (Day 1 of Part B) and Week 132 | 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. Mean change from baseline in hemoglobin results were summarized from Part A baseline for Part A reporting groups and from Part B baseline for Part B reporting groups. |
| Secondary | Number of Subjects Who Received Red Blood Cell (RBC) Transfusions | From Day 1 up to Week 132 | The number of on-study RBC transfusions were monitored throughout the treatment period. |
| Secondary | Mean Change From Baseline in Absolute Reticulocyte Count (ARC) at Weeks 48 and 132 | Part A: Baseline (Day 1 of Part A) and Week 48; Part B: Baseline (Day 1 of Part B) and Week 132 | 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. Mean change from baseline in ARC results were summarized from Part A baseline for Part A reporting groups and from Part B baseline for Part B reporting groups. |
| Secondary | Mean Change From Baseline in Lactate Dehydrogenase (LDH) at Weeks 48 and 132 | Part A: Baseline (Day 1 of Part A) and Week 48; Part B: Baseline (Day 1 of Part B) and Week 132 | 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. Mean change from baseline in LDH results were summarized from Part A baseline for Part A reporting groups and from Part B baseline for Part B reporting groups. |
| Secondary | Mean Change From Baseline in Haptoglobin at Weeks 48 and 132 | Part A: Baseline (Day 1 of Part A) and Week 48; Part B: Baseline (Day 1 of Part B) and Week 132 | 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. Mean change from baseline in haptoglobin results were summarized from Part A baseline for Part A reporting groups and from Part B baseline for Part B reporting groups. |
| Secondary | Mean Change From Baseline in Indirect Bilirubin at Weeks 48 and 132 | Part A: Baseline (Day 1 of Part A) and Week 48; Part B: Baseline (Day 1 of Part B) and Week 132 | Serum chemistry assessments of indirect bilirubin were made at the last measurement prior to the first dose of pegcetacoplan (baseline) and periodically throughout the different parts of the treatment period. Mean change from baseline in indirect bilirubin results were summarized from Part A baseline for Part A reporting groups and from Part B baseline for Part B reporting groups. |
| Secondary | Maximum Observed Trough Serum Concentration (Ctrough,Max) of Pegcetacoplan | Pre-dose and 4 hours postdose on Day 1; pre-dose on Weeks 1, 2, 4, 8, 12, 16, 20, 24, 32, 36, 40, 48, 60, 72, 84, 96, 108, 120 and 132 | Blood samples were collected for the assessment of Ctrough,max of pegcetacoplan. |
| Secondary | Mean Change From Baseline in Functional Assessment of Chronic Illness Therapy (FACIT)-Fatigue Score at Weeks 48 and 132 | Part A: Baseline (Day 1 of Part A) and Week 48; Part B: Baseline (Day 1 of Part B) and Week 132 | 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. Mean change from baseline in FACIT-Fatigue score results were summarized from Part A baseline for Part A reporting groups and from Part B baseline for Part B reporting groups. |
| Secondary | Mean Change From Baseline in Linear Analog Scale Assessment (LASA) Score at Weeks 48 and 132 | Part A: Baseline (Day 1 of Part A) and Week 48; Part B: Baseline (Day 1 of Part B) and Week 132 | The LASA consists of 5 single statements asking subjects to rate their perceived level of functioning. Specific domains include physical well-being (fatigue, activity level), emotional well-being (depression, anxiety, stress), spiritual well-being (sense of meaning), and intellectual well-being (ability to think clearly and concentrate). An item for overall quality of life is also included. Each domain scale range from 0 to 10. Where, 0 = as bad as it can be and 10 = as good as it can be. The total score ranging from 0 to 50 and higher scores indicating better quality of life. Mean change from baseline in LASA score results were summarized from Part A baseline for Part A reporting groups and from Part B baseline for Part B reporting groups. |
Source ClinicalTrials.gov
Outcomes10
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 | Part A:From first dose of study drug (Part A Day 1) up to 30days after last dose of study drug in Part A,approximately 366days Part B:From first dose of study drug (Part B Day 1) up to 56days after last dose of study drug in Part B,approximately 980days | The Safety set included all subjects who received at least 1 dose of study drug. | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Mean Change From Baseline in Hemoglobin at Weeks 48 and 132 | Part A: Baseline (Day 1 of Part A) and Week 48; Part B: Baseline (Day 1 of Part B) and Week 132 | The intent-to-treat (ITT) analysis set included all subjects who received at least 1 dose of study drug. Only subjects analyzed at specific timepoint are reported. | gram per deciliter (g/dL) | MEAN |
| SECONDARY | Number of Subjects Who Received Red Blood Cell (RBC) Transfusions | From Day 1 up to Week 132 | The ITT analysis set included all subjects who received at least 1 dose of study drug. | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Mean Change From Baseline in Absolute Reticulocyte Count (ARC) at Weeks 48 and 132 | Part A: Baseline (Day 1 of Part A) and Week 48; Part B: Baseline (Day 1 of Part B) and Week 132 | The ITT analysis set included all subjects who received at least 1 dose of study drug. Only subjects analyzed at specific timepoint are reported. | 10^9 cells/liter (L) | MEAN |
| SECONDARY | Mean Change From Baseline in Lactate Dehydrogenase (LDH) at Weeks 48 and 132 | Part A: Baseline (Day 1 of Part A) and Week 48; Part B: Baseline (Day 1 of Part B) and Week 132 | The ITT analysis set included all subjects who received at least 1 dose of study drug. Only subjects analyzed at specific timepoint are reported. | international units/L | MEAN |
| SECONDARY | Mean Change From Baseline in Haptoglobin at Weeks 48 and 132 | Part A: Baseline (Day 1 of Part A) and Week 48; Part B: Baseline (Day 1 of Part B) and Week 132 | The ITT analysis set included all subjects who received at least 1 dose of study drug. Only subjects analyzed at specific timepoint are reported. | g/L | MEAN |
| SECONDARY | Mean Change From Baseline in Indirect Bilirubin at Weeks 48 and 132 | Part A: Baseline (Day 1 of Part A) and Week 48; Part B: Baseline (Day 1 of Part B) and Week 132 | The ITT analysis set included all subjects who received at least 1 dose of study drug. Only subjects analyzed at specific timepoint are reported. | micromole/L | MEAN |
| SECONDARY | Maximum Observed Trough Serum Concentration (Ctrough,Max) of Pegcetacoplan | Pre-dose and 4 hours postdose on Day 1; pre-dose on Weeks 1, 2, 4, 8, 12, 16, 20, 24, 32, 36, 40, 48, 60, 72, 84, 96, 108, 120 and 132 | The Pharmacokinetic analysis set included all subjects in the Safety set who had at least 1 evaluable post-dose pharmacokinetic measurement that was not below the limit of quantification. | microgram per milliliter | MEAN |
| SECONDARY | Mean Change From Baseline in Functional Assessment of Chronic Illness Therapy (FACIT)-Fatigue Score at Weeks 48 and 132 | Part A: Baseline (Day 1 of Part A) and Week 48; Part B: Baseline (Day 1 of Part B) and Week 132 | The ITT analysis set included all subjects who received at least 1 dose of study drug. Only subjects analyzed at specific timepoint are reported. | score on a scale | MEAN |
| SECONDARY | Mean Change From Baseline in Linear Analog Scale Assessment (LASA) Score at Weeks 48 and 132 | Part A: Baseline (Day 1 of Part A) and Week 48; Part B: Baseline (Day 1 of Part B) and Week 132 | The ITT analysis set included all subjects who received at least 1 dose of study drug. Only subjects analyzed at specific timepoint are reported. | score on a scale | MEAN |
Source ClinicalTrials.gov
Results by group108
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 | Part A: Cohort 1 wAIHA - 270 mg | All TEAEs | 5 | Participants | |
| Number of Subjects With Treatment Emergent Adverse Events (TEAEs) Including by Severity | Part A: Cohort 1 wAIHA - 360 mg | All TEAEs | 5 | Participants | |
| Number of Subjects With Treatment Emergent Adverse Events (TEAEs) Including by Severity | Part A: Cohort 2 CAD - 270 mg | All TEAEs | 7 | Participants | |
| Number of Subjects With Treatment Emergent Adverse Events (TEAEs) Including by Severity | Part A: Cohort 2 CAD - 360 mg | All TEAEs | 6 | Participants | |
| Number of Subjects With Treatment Emergent Adverse Events (TEAEs) Including by Severity | Part B: Cohort 1 wAIHA - 1080 mg | All TEAEs | 5 | Participants | |
| Number of Subjects With Treatment Emergent Adverse Events (TEAEs) Including by Severity | Part B: Cohort 2 CAD - 1080 mg | All TEAEs | 9 | Participants | |
| Number of Subjects With Treatment Emergent Adverse Events (TEAEs) Including by Severity | Part A: Cohort 1 wAIHA - 270 mg | Treatment related TEAEs | 4 | Participants | |
| Number of Subjects With Treatment Emergent Adverse Events (TEAEs) Including by Severity | Part A: Cohort 1 wAIHA - 360 mg | Treatment related TEAEs | 4 | Participants | |
| Number of Subjects With Treatment Emergent Adverse Events (TEAEs) Including by Severity | Part A: Cohort 2 CAD - 270 mg | Treatment related TEAEs | 5 | Participants | |
| Number of Subjects With Treatment Emergent Adverse Events (TEAEs) Including by Severity | Part A: Cohort 2 CAD - 360 mg | Treatment related TEAEs | 4 | Participants |
1 to 10 of 108Source ClinicalTrials.gov
See all 108 results by groupParticipant flow36
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Part A (Core Study Phase) | STARTED | Part A: Cohort 1 wAIHA - 270 mg | 5 | |
| Part A (Core Study Phase) | STARTED | Part A: Cohort 1 wAIHA - 360 mg | 6 | |
| Part A (Core Study Phase) | STARTED | Part A: Cohort 2 CAD - 270 mg | 7 | |
| Part A (Core Study Phase) | STARTED | Part A: Cohort 2 CAD - 360 mg | 6 | |
| Part A (Core Study Phase) | STARTED | Part B: Cohort 1 wAIHA - 1080 mg | 0 | |
| Part A (Core Study Phase) | STARTED | Part B: Cohort 2 CAD - 1080 mg | 0 | |
| Part A (Core Study Phase) | COMPLETED | Part A: Cohort 1 wAIHA - 270 mg | 3 | |
| Part A (Core Study Phase) | COMPLETED | Part A: Cohort 1 wAIHA - 360 mg | 3 | |
| Part A (Core Study Phase) | COMPLETED | Part A: Cohort 2 CAD - 270 mg | 5 | |
| Part A (Core Study Phase) | COMPLETED | Part A: Cohort 2 CAD - 360 mg | 5 |
1 to 10 of 36Source ClinicalTrials.gov
See all 36 participant flowAdverse events1,236
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 | Anaemia | Blood and lymphatic system disorders | Part A: Cohort 1 wAIHA - 270 mg | 0 | 5 | |
| other | Anaemia | Blood and lymphatic system disorders | Part A: Cohort 1 wAIHA - 360 mg | 2 | 6 | |
| other | Anaemia | Blood and lymphatic system disorders | Part A: Cohort 2 CAD - 270 mg | 0 | 7 | |
| other | Anaemia | Blood and lymphatic system disorders | Part A: Cohort 2 CAD - 360 mg | 0 | 6 | |
| other | Anaemia | Blood and lymphatic system disorders | Part B: Cohort 1 wAIHA - 1080 mg | 1 | 5 | |
| other | Anaemia | Blood and lymphatic system disorders | Part B: Cohort 2 CAD - 1080 mg | 2 | 9 | |
| other | Autoimmune haemolytic anaemia | Blood and lymphatic system disorders | Part A: Cohort 1 wAIHA - 270 mg | 0 | 5 | |
| other | Autoimmune haemolytic anaemia | Blood and lymphatic system disorders | Part A: Cohort 1 wAIHA - 360 mg | 0 | 6 | |
| other | Autoimmune haemolytic anaemia | Blood and lymphatic system disorders | Part A: Cohort 2 CAD - 270 mg | 0 | 7 | |
| other | Autoimmune haemolytic anaemia | Blood and lymphatic system disorders | Part A: Cohort 2 CAD - 360 mg | 0 | 6 |
1 to 10 of 1,236Source ClinicalTrials.gov
See all 1,236 adverse eventsAdverse event totals18
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Part A: Cohort 1 wAIHA - 270 mg | 0 | 5 |
| Total, all-cause mortality | Part A: Cohort 1 wAIHA - 360 mg | 0 | 6 |
| Total, all-cause mortality | Part A: Cohort 2 CAD - 270 mg | 1 | 7 |
| Total, all-cause mortality | Part A: Cohort 2 CAD - 360 mg | 0 | 6 |
| Total, all-cause mortality | Part B: Cohort 1 wAIHA - 1080 mg | 0 | 5 |
| Total, all-cause mortality | Part B: Cohort 2 CAD - 1080 mg | 1 | 9 |
| Total, other adverse events | Part A: Cohort 1 wAIHA - 270 mg | 5 | 5 |
| Total, other adverse events | Part A: Cohort 1 wAIHA - 360 mg | 5 | 6 |
| Total, other adverse events | Part A: Cohort 2 CAD - 270 mg | 7 | 7 |
| Total, other adverse events | Part A: Cohort 2 CAD - 360 mg | 6 | 6 |
1 to 10 of 18Source ClinicalTrials.gov
See all 18 adverse event totalsPapers1
The papers the trial lists on ClinicalTrials.gov.
| PMID | Listed as |
|---|---|
| 39486046 | DERIVED |
Source PubMed
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/NCT03226678
GET https://conductscience.org/api/trials/v1/NCT03226678/results?page=2