Trial · NCT05096403
A Study to Evaluate the Efficacy and Safety of Pegcetacoplan in Patients With Cold Agglutinin Disease (CAD)
NCT05096403, Phase 3, Completed, Swedish Orphan Biovitrum; results first posted 2025-10-30
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 Cold Agglutinin Disease (CAD). Trials, record NCT05096403. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT05096403
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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 Cold Agglutinin Disease (CAD)
- NCT number
- NCT05096403
- Status
- Completed
- Phases
- Phase 3
- Enrollment
- 24 participants, actual
- Lead sponsor
- Swedish Orphan Biovitrum
- Conditions
- Cold Agglutinin Disease
- Interventions
- Pegcetacoplan, Placebo matching Pegcetacoplan
- First posted
- 2021-10-27
- Last updated
- 2025-10-30
- Results first posted
- 2025-10-30
Source ClinicalTrials.gov
Registered outcomes33
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Number of Patients Achieving a Response (R) at Week 24 | Week 24 | A participant was considered to have a response if the Hgb level increased greater than or equal to (\>=) 1.5 gram per deciliter (g/dL) from baseline and this increase was maintained from Week 16 through Week 24 in absence of blood transfusion from Week 5 through Week 24. |
| Secondary | Change From Baseline to Week 24 in Hemoglobin (Hgb) Level-Part A in the Absence of Intercurrent Events (ICEs). | Week 24 | The ICEs of interest were : * Withdrawal from treatment or lost to follow-up before the end of the double-blind period * Use of prohibited medications (rituximab alone or in combination, any other complement inhibitor, any other investigational drug, and plasma exchange) Mean change from Baseline to Week 24 in Hemoglobin (Hgb) level. |
| Secondary | Number of Patients Achieving Transfusion Avoidance From Week 5 to Week 24-Part A | Week 24 | Percentage of patients who did not receive a blood transfusion between Week 5 and Week 24 was assessed |
| Secondary | Change From Baseline to Week 24 in FACT-An Scale Score (Quality of Life)-Part A in the Absence of Intercurrent Events (ICEs) | Week 24 | The ICEs of interest were : * Withdrawal from treatment or lost to follow-up before the end of the double-blind period * Use of prohibited medications (rituximab alone or in combination, any other complement inhibitor, any other investigational drug, and plasma exchange). FACT-An is used to measure quality of life (QoL) in patients with anemia. Each item is rated on a 5-point Likert scale:0=Not at all, 1=A little bit ,2=Somewhat, 3=Quite a bit, 4=Very much. Some items are reverse scored. Higher scores in this scale denote a better QoL with less impact of anemia.The total FACT-An scale score ranges from 0 to 160. The total score gives a comprehensive view of a patient's well-being. It combines the FACT-G with an Anemia subscale. FACT-G (27 items):Physical Well-Being (PWB) and Social/Family Well-Being (SWB) 14 items in total, Emotional Well-Being (EWB)-6 items,Functional Well-Being (FWB)-7 items, Anemia Subscale (AnS): 13 items. Total FACT-An score=FACT-G + Anemia Subscale=40 item |
| Secondary | Number of Packed Red Blood Cell Transfusions Received by Patients From Week 5 to Week 24-Part A | Week 24 | Number of blood transfusions received between Week 5 and Week 24 were assessed. |
| Secondary | Change From Baseline to Week 24 in LDH Levels-Part A in the Absence of Intercurrent Events (ICEs) | Week 24 | The ICEs of interest were : * Withdrawal from treatment or lost to follow-up before the end of the double-blind period * Use of prohibited medications (rituximab alone or in combination, any other complement inhibitor, any other investigational drug, and plasma exchange) Mean change from baseline to Week 24 in Lactate dehydrogenase (LDH) levels |
| Secondary | Change From Baseline to Week 24 in Haptoglobin Levels-Part A in the Absence of Intercurrent Events (ICEs) | Week 24 | The ICEs of interest were : * Withdrawal from treatment or lost to follow-up before the end of the double-blind period * Use of prohibited medications (rituximab alone or in combination, any other complement inhibitor, any other investigational drug, and plasma exchange). Mean change from baseline to Week 24 in Haptoglobin level |
| Secondary | Change From Baseline to Week 24 in Indirect Bilirubin-Part A in the Absence of Intercurrent Events (ICE) | Week 24 | The ICEs of interest were : * Withdrawal from treatment or lost to follow-up before the end of the double-blind period * Use of prohibited medications (rituximab alone or in combination, any other complement inhibitor, any other investigational drug, and plasma exchange). Mean change from baseline to Week 24 in Indirect bilirubin level |
| Secondary | Change From Baseline to Week 24 in ARC-Part A in the Absence of Intercurrent Events (ICEs) | Week 24 | The ICEs of interest were : * Withdrawal from treatment or lost to follow-up before the end of the double-blind period * Use of prohibited medications (rituximab alone or in combination, any other complement inhibitor, any other investigational drug, and plasma exchange). Mean change from baseline to Week 24 in Absolute reticulocyte counts (ARC). |
| Secondary | Change From Baseline to Week 24 in D-dimer Levels-Part A in the Absence of Intercurrent Events (ICEs | Week 24 | * Withdrawal from treatment or lost to follow-up before the end of the double-blind period * Use of prohibited medications (rituximab alone or in combination, any other complement inhibitor, any other investigational drug, and plasma exchange) change from baseline to Week 24 in D-dimer levels. |
1 to 10 of 33Source ClinicalTrials.gov
See all 33 registered outcomesOutcomes33
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Number of Patients Achieving a Response (R) at Week 24 | Week 24 | Participants | COUNT_OF_PARTICIPANTS | |
| SECONDARY | Change From Baseline to Week 24 in Hemoglobin (Hgb) Level-Part A in the Absence of Intercurrent Events (ICEs). | Week 24 | grams per deciliter (g/dL) | LEAST_SQUARES_MEAN | |
| SECONDARY | Number of Patients Achieving Transfusion Avoidance From Week 5 to Week 24-Part A | Week 24 | Number of Patients Achieving Transfusion Avoidance at Week 24 | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Change From Baseline to Week 24 in FACT-An Scale Score (Quality of Life)-Part A in the Absence of Intercurrent Events (ICEs) | Week 24 | Part A | Units on a scale | MEAN |
| SECONDARY | Number of Packed Red Blood Cell Transfusions Received by Patients From Week 5 to Week 24-Part A | Week 24 | Number of transfusions | MEAN | |
| SECONDARY | Change From Baseline to Week 24 in LDH Levels-Part A in the Absence of Intercurrent Events (ICEs) | Week 24 | U/L | MEAN | |
| SECONDARY | Change From Baseline to Week 24 in Haptoglobin Levels-Part A in the Absence of Intercurrent Events (ICEs) | Week 24 | g/L | MEAN | |
| SECONDARY | Change From Baseline to Week 24 in Indirect Bilirubin-Part A in the Absence of Intercurrent Events (ICE) | Week 24 | µmol/L | MEAN | |
| SECONDARY | Change From Baseline to Week 24 in ARC-Part A in the Absence of Intercurrent Events (ICEs) | Week 24 | 10^9 cells/L | MEAN | |
| SECONDARY | Change From Baseline to Week 24 in D-dimer Levels-Part A in the Absence of Intercurrent Events (ICEs | Week 24 | µg/L FEU | MEAN |
1 to 10 of 33Source ClinicalTrials.gov
See all 33 outcomesResults by group86
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Number of Patients Achieving a Response (R) at Week 24 | Pegcetacoplan Double Blind During Part A | 8 | Participants | ||
| Number of Patients Achieving a Response (R) at Week 24 | Placebo Matching Pegcetacoplan-Double-blind During Part A | 2 | Participants | ||
| Change From Baseline to Week 24 in Hemoglobin (Hgb) Level-Part A in the Absence of Intercurrent Events (ICEs). | Pegcetacoplan Double Blind During Part A | 2.85 | Standard Error 0.306 | grams per deciliter (g/dL) | |
| Change From Baseline to Week 24 in Hemoglobin (Hgb) Level-Part A in the Absence of Intercurrent Events (ICEs). | Placebo Matching Pegcetacoplan-Double-blind During Part A | 1.36 | Standard Error 0.278 | grams per deciliter (g/dL) | |
| Number of Patients Achieving Transfusion Avoidance From Week 5 to Week 24-Part A | Pegcetacoplan Double Blind During Part A | 12 | -0.374 to 0.691 | Participants | |
| Number of Patients Achieving Transfusion Avoidance From Week 5 to Week 24-Part A | Placebo Matching Pegcetacoplan-Double-blind During Part A | 5 | 0 to 0 | Participants | |
| Change From Baseline to Week 24 in FACT-An Scale Score (Quality of Life)-Part A in the Absence of Intercurrent Events (ICEs) | Pegcetacoplan Double Blind During Part A | 20.67 | Standard Deviation 19.175 | Units on a scale | |
| Change From Baseline to Week 24 in FACT-An Scale Score (Quality of Life)-Part A in the Absence of Intercurrent Events (ICEs) | Placebo Matching Pegcetacoplan-Double-blind During Part A | 16.40 | Standard Deviation 37.246 | Units on a scale | |
| Number of Packed Red Blood Cell Transfusions Received by Patients From Week 5 to Week 24-Part A | Pegcetacoplan Double Blind During Part A | 1.5 | Standard Deviation 3.62 | Number of transfusions | |
| Number of Packed Red Blood Cell Transfusions Received by Patients From Week 5 to Week 24-Part A | Placebo Matching Pegcetacoplan-Double-blind During Part A | 1.1 | Standard Deviation 2.80 | Number of transfusions |
1 to 10 of 86Source ClinicalTrials.gov
See all 86 results by groupParticipant flow18
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Double-blind (Part A) | STARTED | Pegcetacoplan Double Blind During Part A | 16 | |
| Double-blind (Part A) | STARTED | Placebo Matching Pegcetacoplan-Double-blind During Part A | 8 | |
| Double-blind (Part A) | COMPLETED | Pegcetacoplan Double Blind During Part A | 14 | |
| Double-blind (Part A) | COMPLETED | Placebo Matching Pegcetacoplan-Double-blind During Part A | 7 | |
| Double-blind (Part A) | NOT COMPLETED | Pegcetacoplan Double Blind During Part A | 2 | |
| Double-blind (Part A) | NOT COMPLETED | Placebo Matching Pegcetacoplan-Double-blind During Part A | 1 | |
| Open-label treatment (Part B) | STARTED | Pegcetacoplan Double Blind During Part A | 14 | |
| Open-label treatment (Part B) | STARTED | Placebo Matching Pegcetacoplan-Double-blind During Part A | 7 | |
| Open-label treatment (Part B) | COMPLETED | Pegcetacoplan Double Blind During Part A | 8 | |
| Open-label treatment (Part B) | COMPLETED | Placebo Matching Pegcetacoplan-Double-blind During Part A | 3 |
1 to 10 of 18Source ClinicalTrials.gov
See all 18 participant flowAdverse events460
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 | Fatigue | General disorders | Pegcetacoplan Double Blind During Part A | 3 | 16 | |
| other | Fatigue | General disorders | Placebo Matching Pegcetacoplan-Double-blind During Part A | 0 | 8 | |
| other | Fatigue | General disorders | Open-label Pegcetacoplan During Parts B and C | 0 | 14 | |
| other | Fatigue | General disorders | Open-label Pegcetacoplan (Placebo Matching Pegcetacoplan during Part A) During Parts B&C | 0 | 7 | |
| other | Oedema peripheral | General disorders | Pegcetacoplan Double Blind During Part A | 3 | 16 | |
| other | Oedema peripheral | General disorders | Placebo Matching Pegcetacoplan-Double-blind During Part A | 3 | 8 | |
| other | Oedema peripheral | General disorders | Open-label Pegcetacoplan During Parts B and C | 0 | 14 | |
| other | Oedema peripheral | General disorders | Open-label Pegcetacoplan (Placebo Matching Pegcetacoplan during Part A) During Parts B&C | 0 | 7 | |
| other | Gait disturbance | General disorders | Pegcetacoplan Double Blind During Part A | 0 | 16 | |
| other | Gait disturbance | General disorders | Placebo Matching Pegcetacoplan-Double-blind During Part A | 0 | 8 |
1 to 10 of 460Source ClinicalTrials.gov
See all 460 adverse eventsAdverse event totals12
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Pegcetacoplan Double Blind During Part A | 1 | 16 |
| Total, all-cause mortality | Placebo Matching Pegcetacoplan-Double-blind During Part A | 0 | 8 |
| Total, all-cause mortality | Open-label Pegcetacoplan During Parts B and C | 0 | 14 |
| Total, all-cause mortality | Open-label Pegcetacoplan (Placebo Matching Pegcetacoplan during Part A) During Parts B&C | 0 | 7 |
| Total, other adverse events | Pegcetacoplan Double Blind During Part A | 16 | 16 |
| Total, other adverse events | Placebo Matching Pegcetacoplan-Double-blind During Part A | 8 | 8 |
| Total, other adverse events | Open-label Pegcetacoplan During Parts B and C | 12 | 14 |
| Total, other adverse events | Open-label Pegcetacoplan (Placebo Matching Pegcetacoplan during Part A) During Parts B&C | 4 | 7 |
| Total, serious adverse events | Pegcetacoplan Double Blind During Part A | 5 | 16 |
| Total, serious adverse events | Placebo Matching Pegcetacoplan-Double-blind During Part A | 1 | 8 |
1 to 10 of 12Source ClinicalTrials.gov
See all 12 adverse event totalsPapers0
The papers the trial lists on ClinicalTrials.gov.
None in this release.
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 comparisons11
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
| Comparison | Source |
|---|---|
| Pegcetacoplan Double Blind During Part A vs Placebo Matching Pegcetacoplan-Double-blind During Part A: Number of Patients Achieving Transfusion Avoidance From Week 5 to Week 24-Part A | Trial comparisons |
Pegcetacoplan Double Blind During Part A vs Placebo Matching Pegcetacoplan-Double-blind During Part A: Count and Percentage of Patients With a First Normalization From Baseline by Week 24 for ARC Levels-Part A | Trial comparisons |
| Pegcetacoplan Double Blind During Part A vs Placebo Matching Pegcetacoplan-Double-blind During Part A: Normalization of Markers of Hemolysis (ARC) at Week 24-Part A | Trial comparisons |
| Pegcetacoplan Double Blind During Part A vs Placebo Matching Pegcetacoplan-Double-blind During Part A: Normalization of Markers of Hemolysis (LDH) at Week 24-Part A | Trial comparisons |
Pegcetacoplan Double Blind During Part A vs Placebo Matching Pegcetacoplan-Double-blind During Part A: Count and Percentage of Patients With a First Normalization From Baseline by Week 24 for LDH Levels-Part A | Trial comparisons |
Pegcetacoplan Double Blind During Part A vs Placebo Matching Pegcetacoplan-Double-blind During Part A: Count and Percentage of Patients With a First Normalization From Baseline by Week 24 for Indirect Bilirubin Levels-Part A | Trial comparisons |
| Pegcetacoplan Double Blind During Part A vs Placebo Matching Pegcetacoplan-Double-blind During Part A: Normalization of Markers of Hemolysis (Haptoglobin) at Week 24-Part A | Trial comparisons |
Pegcetacoplan Double Blind During Part A vs Placebo Matching Pegcetacoplan-Double-blind During Part A: Count and Percentage of Patients With a First Normalization From Baseline by Week 24 for Haptoglobin Levels-Part A | Trial comparisons |
| Pegcetacoplan Double Blind During Part A vs Placebo Matching Pegcetacoplan-Double-blind During Part A: Number of Patients Achieving a Response (R) at Week 24 | Trial comparisons |
Pegcetacoplan Double Blind During Part A vs Placebo Matching Pegcetacoplan-Double-blind During Part A: Count and Percentage of Patients With a First Normalization From Baseline by Week 24 for Hemoglobin Levels-Part A | Trial comparisons |
1 to 10 of 11
See all 11 trial comparisonsTests0
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/NCT05096403
GET https://conductscience.org/api/trials/v1/NCT05096403/registered?page=2