Trial · NCT03056040
ALXN1210 Versus Eculizumab in Adult Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH) Currently Treated With Eculizumab
NCT03056040, Phase 3, Completed, Alexion Pharmaceuticals, Inc.; results first posted 2019-03-21
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. ALXN1210 Versus Eculizumab in Adult Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH) Currently Treated With Eculizumab. Trials, record NCT03056040. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT03056040
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- ALXN1210 Versus Eculizumab in Adult Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH) Currently Treated With Eculizumab
- NCT number
- NCT03056040
- Status
- Completed
- Phases
- Phase 3
- Enrollment
- 202 participants, actual
- Lead sponsor
- Alexion Pharmaceuticals, Inc.
- Conditions
- Paroxysmal Nocturnal Hemoglobinuria (PNH)
- Interventions
- Ravulizumab, Eculizumab
- First posted
- 2017-02-16
- Last updated
- 2025-03-12
- Other registries' IDs
- eudract:2016-002026-36
- Results first posted
- 2019-03-21
Source ClinicalTrials.gov
Registered outcomes9
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Percent Change In Lactate Dehydrogenase Levels From Baseline To Day 183 | Baseline, Day 183 | Lactate dehydrogenase (LDH) is an indicator of intravascular hemolysis that occurs in participants with paroxysmal nocturnal hemoglobinuria. A decrease in LDH indicates reduction (improvement) in hemolysis. Baseline was defined as the average of all available on-study assessments prior to the first study drug infusion. The percent change in LDH was analyzed using a mixed-effect model for repeated measures (MMRM) with the fixed, categorical effects of treatment, study visit, and study visit by treatment group interaction, as well as the continuous, fixed covariate of baseline LDH and the stratification randomization indicator of packed red blood cells transfusion history (yes/no within 12 months prior to Day 1). |
| Secondary | Number Of Participants With Breakthrough Hemolysis Through Day 183 | Baseline through Day 183 | Breakthrough hemolysis (BTH) was defined as at least one new or worsening symptom or sign of intravascular hemolysis (fatigue, hemoglobinuria, abdominal pain, dyspnea, anemia \[hemoglobin \<10 grams (g)/deciliter (dL)\], major adverse vascular event \[including thrombosis\], dysphagia, or erectile dysfunction) in the presence of elevated LDH ≥2 times the upper limit of normal (ULN). |
| Secondary | Change From Baseline To Day 183 In Functional Assessment Of Chronic Illness Therapy (FACIT)-Fatigue Scores | Baseline, Day 183 | FACIT-Fatigue score ranges from 0 to 52, with a higher score indicating less fatigue. Baseline was defined as the last non-missing assessment value prior to first study drug dose. Change in FACIT-Fatigue score from Baseline to Day 183 was analyzed using an MMRM with the fixed, categorical effects of treatment, the stratification randomization indicator of packed red blood cells transfusion history (yes/no within 12 months prior to Day 1), study visit, and study visit by treatment group interaction, as well as the continuous fixed covariate of Baseline FACIT-Fatigue score. |
| Secondary | Percentage Of Participants Who Achieved Transfusion Avoidance Through Day 183 | Baseline through Day 183 | Transfusion avoidance was defined as the percentage of participants who remained transfusion free and did not require a transfusion per protocol-specified guidelines (hemoglobin value of ≤9 g/dL with signs or symptoms of sufficient severity to warrant a transfusion, or a hemoglobin value of ≤7 g/dL regardless of presence of clinical signs or symptoms) through Day 183. |
| Secondary | Percentage Of Participants With Stabilized Hemoglobin Levels Through Day 183 | Baseline through Day 183 | Stabilized hemoglobin was defined as avoidance of a ≥2 g/dL decrease in hemoglobin level from Baseline in the absence of transfusion through Day 183. |
| Secondary | Number Of Participants With Breakthrough Hemolysis Through End of Study | Baseline through end of study (up to 4 years) | BTH was defined as at least one new or worsening symptom or sign of intravascular hemolysis (fatigue, hemoglobinuria, abdominal pain, dyspnea, anemia \[hemoglobin \<10 g/dL\], major adverse vascular event \[including thrombosis\], dysphagia, or erectile dysfunction) in the presence of elevated LDH ≥2 times the ULN. |
| Secondary | Change From Baseline To End of Study In FACIT-Fatigue Scores Through End of Study | Baseline, End of Study (up to 4 years) | FACIT-Fatigue score ranges from 0 to 52, with a higher score indicating less fatigue. Baseline was defined as the last non-missing assessment value prior to first study drug dose. Change in FACIT-Fatigue score from Baseline to Day 183 was analyzed using an MMRM with the fixed, categorical effects of treatment, the stratification randomization indicator of packed red blood cells transfusion history (yes/no within 12 months prior to Day 1), study visit, and study visit by treatment group interaction, as well as the continuous fixed covariate of Baseline FACIT-Fatigue score. |
| Secondary | Percentage Of Participants Who Achieved Transfusion Avoidance Through End of Study | Baseline through end of study (up to 4 years) | Transfusion avoidance was defined as the percentage of participants who remained transfusion free and did not require a transfusion per protocol-specified guidelines (hemoglobin value of ≤9 g/dL with signs or symptoms of sufficient severity to warrant a transfusion, or a hemoglobin value of ≤7 g/dL regardless of presence of clinical signs or symptoms) through the end of study. |
| Secondary | Percentage Of Participants With Stabilized Hemoglobin Levels Through End of Study | Baseline through end of study (up to 4 years) | Stabilized hemoglobin was defined as avoidance of a ≥2 g/dL decrease in hemoglobin level from Baseline in the absence of transfusion through end of study. |
Source ClinicalTrials.gov
Outcomes9
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Percent Change In Lactate Dehydrogenase Levels From Baseline To Day 183 | Baseline, Day 183 | Full Analysis Set: All participants who received at least 1 dose of randomized treatment (ravulizumab or eculizumab). | percent change | LEAST_SQUARES_MEAN |
| SECONDARY | Number Of Participants With Breakthrough Hemolysis Through Day 183 | Baseline through Day 183 | Full Analysis Set: All participants who received at least 1 dose of randomized treatment (ravulizumab or eculizumab). | number of participants | NUMBER |
| SECONDARY | Change From Baseline To Day 183 In Functional Assessment Of Chronic Illness Therapy (FACIT)-Fatigue Scores | Baseline, Day 183 | Full Analysis Set: All participants who received at least 1 dose of randomized treatment (ravulizumab or eculizumab). | units on a scale | LEAST_SQUARES_MEAN |
| SECONDARY | Percentage Of Participants Who Achieved Transfusion Avoidance Through Day 183 | Baseline through Day 183 | Full Analysis Set: All participants who received at least 1 dose of randomized treatment (ravulizumab or eculizumab). | percentage of participants | NUMBER |
| SECONDARY | Percentage Of Participants With Stabilized Hemoglobin Levels Through Day 183 | Baseline through Day 183 | Full Analysis Set: All participants who received at least 1 dose of randomized treatment (ravulizumab or eculizumab). | percentage of participants | NUMBER |
| SECONDARY | Number Of Participants With Breakthrough Hemolysis Through End of Study | Baseline through end of study (up to 4 years) | Full Analysis Set: All participants who received at least 1 dose of randomized treatment (ravulizumab or eculizumab). Here, 'Overall number of participants analyzed' = participants evaluable for this outcome measure. | number of participants | NUMBER |
| SECONDARY | Change From Baseline To End of Study In FACIT-Fatigue Scores Through End of Study | Baseline, End of Study (up to 4 years) | Full Analysis Set: All participants who received at least 1 dose of randomized treatment (ravulizumab or eculizumab). Here, 'Overall number of participants analyzed' = participants evaluable for this outcome measure. | units on a scale | MEAN |
| SECONDARY | Percentage Of Participants Who Achieved Transfusion Avoidance Through End of Study | Baseline through end of study (up to 4 years) | Full Analysis Set: All participants who received at least 1 dose of randomized treatment (ravulizumab or eculizumab). Here, 'Overall number of participants analyzed' = participants evaluable for this outcome measure. | percentage of participants | NUMBER |
| SECONDARY | Percentage Of Participants With Stabilized Hemoglobin Levels Through End of Study | Baseline through end of study (up to 4 years) | Full Analysis Set: All participants who received at least 1 dose of randomized treatment (ravulizumab or eculizumab). Here, 'Overall number of participants analyzed' = participants evaluable for this outcome measure. | percentage of participants | NUMBER |
Source ClinicalTrials.gov
Results by group18
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Percent Change In Lactate Dehydrogenase Levels From Baseline To Day 183 | Ravulizumab/Ravulizumab | -0.82 | 95% Confidence Interval -7.75 to 6.11 | percent change | |
| Percent Change In Lactate Dehydrogenase Levels From Baseline To Day 183 | Eculizumab/Ravulizumab | 8.39 | 95% Confidence Interval 1.47 to 15.32 | percent change | |
| Number Of Participants With Breakthrough Hemolysis Through Day 183 | Ravulizumab/Ravulizumab | 0 | 95% Confidence Interval 0.00 to 3.73 | number of participants | |
| Number Of Participants With Breakthrough Hemolysis Through Day 183 | Eculizumab/Ravulizumab | 5 | 95% Confidence Interval 1.68 to 11.51 | number of participants | |
| Change From Baseline To Day 183 In Functional Assessment Of Chronic Illness Therapy (FACIT)-Fatigue Scores | Ravulizumab/Ravulizumab | 2.01 | 95% Confidence Interval 0.64 to 3.39 | units on a scale | |
| Change From Baseline To Day 183 In Functional Assessment Of Chronic Illness Therapy (FACIT)-Fatigue Scores | Eculizumab/Ravulizumab | 0.54 | 95% Confidence Interval -0.84 to 1.93 | units on a scale | |
| Percentage Of Participants Who Achieved Transfusion Avoidance Through Day 183 | Ravulizumab/Ravulizumab | 87.6 | 95% Confidence Interval 81.08 to 94.18 | percentage of participants | |
| Percentage Of Participants Who Achieved Transfusion Avoidance Through Day 183 | Eculizumab/Ravulizumab | 82.7 | 95% Confidence Interval 75.16 to 90.15 | percentage of participants | |
| Percentage Of Participants With Stabilized Hemoglobin Levels Through Day 183 | Ravulizumab/Ravulizumab | 76.3 | 95% Confidence Interval 67.82 to 84.75 | percentage of participants | |
| Percentage Of Participants With Stabilized Hemoglobin Levels Through Day 183 | Eculizumab/Ravulizumab | 75.5 | 95% Confidence Interval 67.00 to 84.02 | percentage of participants |
1 to 10 of 18Source ClinicalTrials.gov
See all 18 results by groupParticipant flow16
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Primary Evaluation Period | STARTED | Ravulizumab/Ravulizumab | 98 | |
| Primary Evaluation Period | STARTED | Eculizumab/Ravulizumab | 99 | |
| Primary Evaluation Period | Received at Least 1 Dose of Study Drug | Ravulizumab/Ravulizumab | 97 | |
| Primary Evaluation Period | Received at Least 1 Dose of Study Drug | Eculizumab/Ravulizumab | 98 | |
| Primary Evaluation Period | Participants in the Full Analysis Set (FAS) | Ravulizumab/Ravulizumab | 97 | |
| Primary Evaluation Period | Participants in the Full Analysis Set (FAS) | Eculizumab/Ravulizumab | 98 | |
| Primary Evaluation Period | COMPLETED | Ravulizumab/Ravulizumab | 96 | |
| Primary Evaluation Period | COMPLETED | Eculizumab/Ravulizumab | 95 | |
| Primary Evaluation Period | NOT COMPLETED | Ravulizumab/Ravulizumab | 2 | |
| Primary Evaluation Period | NOT COMPLETED | Eculizumab/Ravulizumab | 4 |
1 to 10 of 16Source ClinicalTrials.gov
See all 16 participant flowAdverse events440
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 | Primary Evaluation Period (26 Weeks) Ravulizumab Treatment | 5 | 97 | |
| other | Anaemia | Blood and lymphatic system disorders | Primary Evaluation Period (26 Weeks) Eculizumab Treatment | 3 | 98 | |
| other | Anaemia | Blood and lymphatic system disorders | Extension Period (Ravulizumab To Ravulizumab) | 4 | 96 | |
| other | Anaemia | Blood and lymphatic system disorders | Extension Period (Eculizumab to Ravulizumab) | 7 | 95 | |
| other | Abdominal pain | Gastrointestinal disorders | Primary Evaluation Period (26 Weeks) Ravulizumab Treatment | 7 | 97 | |
| other | Abdominal pain | Gastrointestinal disorders | Primary Evaluation Period (26 Weeks) Eculizumab Treatment | 9 | 98 | |
| other | Abdominal pain | Gastrointestinal disorders | Extension Period (Ravulizumab To Ravulizumab) | 12 | 96 | |
| other | Abdominal pain | Gastrointestinal disorders | Extension Period (Eculizumab to Ravulizumab) | 11 | 95 | |
| other | Constipation | Gastrointestinal disorders | Primary Evaluation Period (26 Weeks) Ravulizumab Treatment | 6 | 97 | |
| other | Constipation | Gastrointestinal disorders | Primary Evaluation Period (26 Weeks) Eculizumab Treatment | 5 | 98 |
1 to 10 of 440Source ClinicalTrials.gov
See all 440 adverse eventsAdverse event totals12
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Primary Evaluation Period (26 Weeks) Ravulizumab Treatment | 0 | 97 |
| Total, all-cause mortality | Primary Evaluation Period (26 Weeks) Eculizumab Treatment | 0 | 98 |
| Total, all-cause mortality | Extension Period (Ravulizumab To Ravulizumab) | 0 | 96 |
| Total, all-cause mortality | Extension Period (Eculizumab to Ravulizumab) | 3 | 95 |
| Total, other adverse events | Primary Evaluation Period (26 Weeks) Ravulizumab Treatment | 72 | 97 |
| Total, other adverse events | Primary Evaluation Period (26 Weeks) Eculizumab Treatment | 85 | 98 |
| Total, other adverse events | Extension Period (Ravulizumab To Ravulizumab) | 83 | 96 |
| Total, other adverse events | Extension Period (Eculizumab to Ravulizumab) | 85 | 95 |
| Total, serious adverse events | Primary Evaluation Period (26 Weeks) Ravulizumab Treatment | 4 | 97 |
| Total, serious adverse events | Primary Evaluation Period (26 Weeks) Eculizumab Treatment | 8 | 98 |
1 to 10 of 12Source ClinicalTrials.gov
See all 12 adverse event totalsPapers9
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/NCT03056040
GET https://conductscience.org/api/trials/v1/NCT03056040/events?page=2