Trial · NCT02946463
ALXN1210 (Ravulizumab) Versus Eculizumab in Complement Inhibitor Treatment-Naïve Adult Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH)
NCT02946463, Phase 3, Completed, Alexion Pharmaceuticals, Inc.; results first posted 2019-02-12
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. ALXN1210 (Ravulizumab) Versus Eculizumab in Complement Inhibitor Treatment-Naïve Adult Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH). Trials, record NCT02946463. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT02946463
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- ALXN1210 (Ravulizumab) Versus Eculizumab in Complement Inhibitor Treatment-Naïve Adult Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH)
- NCT number
- NCT02946463
- Status
- Completed
- Phases
- Phase 3
- Enrollment
- 272 participants, actual
- Lead sponsor
- Alexion Pharmaceuticals, Inc.
- Conditions
- Paroxysmal Nocturnal Hemoglobinuria (PNH)
- Interventions
- Ravulizumab, Eculizumab
- First posted
- 2016-10-27
- Last updated
- 2024-05-14
- Results first posted
- 2019-02-12
Source ClinicalTrials.gov
Registered outcomes6
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Proportion Of Participants With Normalization Of Lactate Dehydrogenase (LDH) Levels | Day 29 through Day 183 | LDH is an indicator of intravascular hemolysis that occurs in participants with paroxysmal nocturnal hemoglobinuria (PNH). A decrease in LDH from above the upper limit of normal (ULN) to below the ULN indicates reduction (improvement) in hemolysis. Normalization of LDH levels (LDH-N) was LDH levels less than or equal to 1 x ULN, from Day 29 through Day 183. The ULN for LDH is 246 U/L. |
| Primary | Percentage Of Participants Who Achieved Transfusion Avoidance (TA) | 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 through Day 183. |
| Secondary | Percentage Of Participants With Breakthrough Hemolysis (BTH) | Baseline through Day 183 | Breakthrough hemolysis was defined as at least 1 new or worsening symptom or sign of intravascular hemolysis (fatigue, hemoglobinuria, abdominal pain, shortness of breath \[dyspnea\], anemia \[hemoglobin \<10 gram/deciliter (g/dL)\], major adverse vascular event \[MAVE, including thrombosis\], dysphagia, or erectile dysfunction) in the presence of elevated LDH ≥2 × ULN, after prior LDH reduction to \<1.5 × ULN on therapy. |
| Secondary | Percent Change From Baseline In LDH Levels | Baseline, Day 183 | Baseline is defined as the average of all available assessments of LDH levels prior to first study drug dose. Estimates are based on Mixed Model for Repeated Measures (MMRM) that includes treatment group, history of transfusion (as a categorical variable based on the stratification factor levels) and baseline LDH level (as a continuous variable), study visit and study visit by treatment group interaction. An unstructured covariance structure was used. |
| Secondary | Change From Baseline In Quality Of Life As Assessed By The Functional Assessment Of Chronic Illness Therapy (FACIT)-Fatigue | Baseline, Day 183 | FACIT-Fatigue score ranges from 0 to 52, with a higher score indicating less fatigue. Baseline is defined as the last non-missing value prior to first dose of study drug. Estimates are based on MMRM that includes treatment group, the observed stratification randomization indicators (history of transfusion and LDH) and baseline FACIT-Fatigue level, study visit, and study visit by treatment group interaction. An unstructured covariance structure was used. |
| Secondary | Percentage Of Participants With Stabilized Hemoglobin Levels | 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. |
Source ClinicalTrials.gov
Outcomes6
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Proportion Of Participants With Normalization Of Lactate Dehydrogenase (LDH) Levels | Day 29 through Day 183 | Full Analysis Set: All participants who received at least 1 dose of study drug (ravulizumab or eculizumab) and had at least 1 efficacy assessment after the first infusion of study drug. | proportion of participants | NUMBER |
| PRIMARY | Percentage Of Participants Who Achieved Transfusion Avoidance (TA) | Baseline through Day 183 | Full Analysis Set: All participants who received at least 1 dose of study drug (ravulizumab or eculizumab) and had at least 1 efficacy assessment after the first infusion of study drug. | percentage of participants | NUMBER |
| SECONDARY | Percentage Of Participants With Breakthrough Hemolysis (BTH) | Baseline through Day 183 | Full Analysis Set: All participants who received at least 1 dose of study drug (ravulizumab or eculizumab) and had at least 1 efficacy assessment after the first infusion of study drug. | percentage of participants | NUMBER |
| SECONDARY | Percent Change From Baseline In LDH Levels | Baseline, Day 183 | Full Analysis Set: All participants who received at least 1 dose of study drug (ravulizumab or eculizumab) and had at least 1 efficacy assessment after the first infusion of study drug. | percent change | LEAST_SQUARES_MEAN |
| SECONDARY | Change From Baseline In Quality Of Life As Assessed By The Functional Assessment Of Chronic Illness Therapy (FACIT)-Fatigue | Baseline, Day 183 | Full Analysis Set: All participants who received at least 1 dose of study drug (ravulizumab or eculizumab) and had at least 1 efficacy assessment after the first infusion of study drug. | units on a scale | LEAST_SQUARES_MEAN |
| SECONDARY | Percentage Of Participants With Stabilized Hemoglobin Levels | Baseline through Day 183 | Full Analysis Set: All participants who received at least 1 dose of study drug (ravulizumab or eculizumab) and had at least 1 efficacy assessment after the first infusion of study drug. | percentage of participants | NUMBER |
Source ClinicalTrials.gov
Results by group12
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Proportion Of Participants With Normalization Of Lactate Dehydrogenase (LDH) Levels | Ravulizumab | 0.536 | 95% Confidence Interval 0.459 to 0.612 | proportion of participants | |
| Proportion Of Participants With Normalization Of Lactate Dehydrogenase (LDH) Levels | Eculizumab | 0.494 | 95% Confidence Interval 0.417 to 0.570 | proportion of participants | |
| Percentage Of Participants Who Achieved Transfusion Avoidance (TA) | Ravulizumab | 73.6 | 95% Confidence Interval 65.87 to 81.33 | percentage of participants | |
| Percentage Of Participants Who Achieved Transfusion Avoidance (TA) | Eculizumab | 66.1 | 95% Confidence Interval 57.68 to 74.55 | percentage of participants | |
| Percentage Of Participants With Breakthrough Hemolysis (BTH) | Ravulizumab | 4.0 | 95% Confidence Interval 0.56 to 7.44 | percentage of participants | |
| Percentage Of Participants With Breakthrough Hemolysis (BTH) | Eculizumab | 10.7 | 95% Confidence Interval 5.23 to 16.26 | percentage of participants | |
| Percent Change From Baseline In LDH Levels | Ravulizumab | -76.84 | 95% Confidence Interval -79.96 to -73.73 | percent change | |
| Percent Change From Baseline In LDH Levels | Eculizumab | -76.02 | 95% Confidence Interval -79.20 to -72.83 | percent change | |
| Change From Baseline In Quality Of Life As Assessed By The Functional Assessment Of Chronic Illness Therapy (FACIT)-Fatigue | Ravulizumab | 7.07 | 95% Confidence Interval 5.55 to 8.60 | units on a scale | |
| Change From Baseline In Quality Of Life As Assessed By The Functional Assessment Of Chronic Illness Therapy (FACIT)-Fatigue | Eculizumab | 6.40 | 95% Confidence Interval 4.85 to 7.96 | units on a scale |
1 to 10 of 12Source ClinicalTrials.gov
See all 12 results by groupParticipant flow32
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Primary Evaluation Period | STARTED | Ravulizumab/Ravulizumab | 125 | |
| Primary Evaluation Period | STARTED | Eculizumab/Ravulizumab | 121 | |
| Primary Evaluation Period | STARTED | Roll-Over Cohort: Ravulizumab - Adults | 0 | |
| Primary Evaluation Period | STARTED | Roll-Over Cohort: Ravulizumab - Pediatrics | 0 | |
| Primary Evaluation Period | Received at Least 1 Dose of Study Drug | Ravulizumab/Ravulizumab | 125 | |
| Primary Evaluation Period | Received at Least 1 Dose of Study Drug | Eculizumab/Ravulizumab | 121 | |
| Primary Evaluation Period | Received at Least 1 Dose of Study Drug | Roll-Over Cohort: Ravulizumab - Adults | 0 | |
| Primary Evaluation Period | Received at Least 1 Dose of Study Drug | Roll-Over Cohort: Ravulizumab - Pediatrics | 0 | |
| Primary Evaluation Period | COMPLETED | Ravulizumab/Ravulizumab | 125 | |
| Primary Evaluation Period | COMPLETED | Eculizumab/Ravulizumab | 119 |
1 to 10 of 32Source ClinicalTrials.gov
See all 32 participant flowAdverse events888
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 | Abdominal pain | Gastrointestinal disorders | Ravulizumab: Primary Evaluation | 7 | 125 | |
| other | Abdominal pain | Gastrointestinal disorders | Eculizumab: Primary Evaluation | 7 | 121 | |
| other | Abdominal pain | Gastrointestinal disorders | Ravulizumab/Ravulizumab: Extension Period | 10 | 124 | |
| other | Abdominal pain | Gastrointestinal disorders | Eculizumab/Ravulizumab: Extension Period | 12 | 119 | |
| other | Abdominal pain | Gastrointestinal disorders | Ravulizumab-Adults | 2 | 24 | |
| other | Abdominal pain | Gastrointestinal disorders | Ravulizumab-Pediatrics | 0 | 2 | |
| other | Diarrhoea | Gastrointestinal disorders | Ravulizumab: Primary Evaluation | 10 | 125 | |
| other | Diarrhoea | Gastrointestinal disorders | Eculizumab: Primary Evaluation | 5 | 121 | |
| other | Diarrhoea | Gastrointestinal disorders | Ravulizumab/Ravulizumab: Extension Period | 14 | 124 | |
| other | Diarrhoea | Gastrointestinal disorders | Eculizumab/Ravulizumab: Extension Period | 13 | 119 |
1 to 10 of 888Source ClinicalTrials.gov
See all 888 adverse eventsAdverse event totals18
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Ravulizumab: Primary Evaluation | 0 | 125 |
| Total, all-cause mortality | Eculizumab: Primary Evaluation | 0 | 121 |
| Total, all-cause mortality | Ravulizumab/Ravulizumab: Extension Period | 4 | 124 |
| Total, all-cause mortality | Eculizumab/Ravulizumab: Extension Period | 4 | 119 |
| Total, all-cause mortality | Ravulizumab-Adults | 0 | 24 |
| Total, all-cause mortality | Ravulizumab-Pediatrics | 0 | 2 |
| Total, other adverse events | Ravulizumab: Primary Evaluation | 87 | 125 |
| Total, other adverse events | Eculizumab: Primary Evaluation | 86 | 121 |
| Total, other adverse events | Ravulizumab/Ravulizumab: Extension Period | 102 | 124 |
| Total, other adverse events | Eculizumab/Ravulizumab: Extension Period | 100 | 119 |
1 to 10 of 18Source ClinicalTrials.gov
See all 18 adverse event totalsPapers6
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/NCT02946463
GET https://conductscience.org/api/trials/v1/NCT02946463/flow?page=2