Phase 3
N=197
ALXN1210 Versus Eculizumab in Adult Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH) Currently Treated With Eculizumab
Paroxysmal Nocturnal Hemoglobinuria (PNH)
Bottom Line
View on ClinicalTrials.gov: NCT03056040 ↗Enrolled (actual)
197
Serious AEs
18.4%
Results posted
Mar 2019
Primary outcome: Primary: Percent Change In Lactate Dehydrogenase Levels From Baseline To Day 183 — -0.82; 8.39 percent change
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 3
- Interventions
- Ravulizumab (Biological); Eculizumab (Biological)
- Age
- Adult, Older Adult · 18+ yrs
- Sex
- All
- Sponsor
- Alexion Pharmaceuticals, Inc.
- Primary completion
- Feb 2022
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 | — |
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.
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). 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.