Phase 3
Completed N=197
ALXN1210 Versus Eculizumab in Adult Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH) Currently Treated With Eculizumab
Paroxysmal Nocturnal Hemoglobinuria (PNH)
Source: ClinicalTrials.gov NCT03056040 ↗
Enrolled (actual)
197
Serious AEs
18.4%
Results posted
Mar 2019
Primary outcomePrimary: Percent Change In Lactate Dehydrogenase Levels From Baseline To Day 183 — -0.82; 8.39 percent change
◆ Published Evidence
Highly cited
378citations · ~54 / year
Ravulizumab (ALXN1210) vs eculizumab in C5-inhibitor-experienced adult patients with PNH: the 302 study.
Summary
The primary purpose of this study was to assess the noninferiority of ravulizumab compared to eculizumab in adult participants with PNH who were clinically stable after having been treated with eculizumab for at least 6 months.
Linked Publications (5)
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Ravulizumab (ALXN1210) vs eculizumab in C5-inhibitor-experienced adult patients with PNH: the 302 study.
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Characterization of breakthrough hemolysis events observed in the phase 3 randomized studies of ravulizumab versus eculizumab in adults with paroxysmal nocturnal hemoglobinuria.
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Ravulizumab demonstrates long-term efficacy, safety and favorable patient survival in patients with paroxysmal nocturnal hemoglobinuria.
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Norm-based comparison of the quality-of-life impact of ravulizumab and eculizumab in paroxysmal nocturnal hemoglobinuria.
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Results from multinational phase 3 studies of ravulizumab (ALXN1210) versus eculizumab in adults with paroxysmal nocturnal hemoglobinuria: subgroup analysis of Japanese patients.
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Percent Change In Lactate Dehydrogenase Levels From Baseline To Day 183 |
-0.82; 8.39 | — |
| SECONDARY Number Of Participants With Breakthrough Hemolysis Through Day 183 |
0; 5 | — |
| SECONDARY Change From Baseline To Day 183 In Functional Assessment Of Chronic Illness Therapy (FACIT)-Fatigue Scores |
2.01; 0.54 | — |
| SECONDARY Percentage Of Participants Who Achieved Transfusion Avoidance Through Day 183 |
87.6; 82.7 | — |
| SECONDARY Percentage Of Participants With Stabilized Hemoglobin Levels Through Day 183 |
76.3; 75.5 | — |
| SECONDARY Number Of Participants With Breakthrough Hemolysis Through End of Study |
9; 6 | — |
| SECONDARY Change From Baseline To End of Study In FACIT-Fatigue Scores Through End of Study |
-1.43; 0.00 | — |
| SECONDARY Percentage Of Participants Who Achieved Transfusion Avoidance Through End of Study |
70.83; 70.53 | — |
| SECONDARY Percentage Of Participants With Stabilized Hemoglobin Levels Through End of Study |
58.33; 67.37 | — |
Eligibility Criteria
Inclusion Criteria
- Male or female ≥18 years of age.
- Treated with eculizumab for PNH for at least 6 months prior to Day 1.
- Lactate dehydrogenase level ≤1.5 times the upper limit of normal (ULN) at screening.
- PNH diagnosis confirmed by documented by high-sensitivity flow cytometry.
- Documented meningococcal vaccination not more than 3 years prior to, or at the time of, initiating study treatment.
- Female participants of childbearing potential must use highly effective contraception starting at screening and continuing until at least 8 months after the last dose of ravulizumab.
- Willing and able to give written informed consent and comply with study visit schedule.
Exclusion Criteria
- History of bone marrow transplantation.
- Body weight <40 kilograms at screening.
- History of or ongoing major cardiac, pulmonary, renal, endocrine, or hepatic disease that, in the opinion of the investigator or sponsor, would preclude participation.
- Unstable medical conditions (for example, myocardial ischemia, active gastrointestinal bleeding, severe congestive heart failure, anticipated need for major surgery within 6 months of randomization, or coexisting chronic anemia unrelated to PNH).
- Female participants who are pregnant, breastfeeding, or who have a positive pregnancy test at screening or Day 1.
- Participation in another interventional clinical study or use of any experimental therapy within 30 days before initiation of study treatment on Day 1 in this study or within 5 half-lives of that investigational product, whichever is greater.
Data sourced from ClinicalTrials.gov (NCT03056040) and the linked publication. Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication. Informational only — not medical advice.