18 and older, any sex, with Paroxysmal Nocturnal Hemoglobinuria (PNH). Patients with the condition only — healthy volunteers not accepted.
Results — posted to ClinicalTrials.gov
Per-arm endpoint measurements with 95% confidence intervals where reported. Source: trial results section.
Percent Change In Lactate Dehydrogenase Levels From Baseline To Day 183Primary· 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 strati
Group
Value
95% CI
Ravulizumab/Ravulizumab
-0.82
-7.75 – 6.11
Eculizumab/Ravulizumab
8.39
1.47 – 15.32
Number Of Participants With Breakthrough Hemolysis Through Day 183Secondary· 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).
Group
Value
95% CI
Ravulizumab/Ravulizumab
0
0.00 – 3.73
Eculizumab/Ravulizumab
5
1.68 – 11.51
Change From Baseline To Day 183 In Functional Assessment Of Chronic Illness Therapy (FACIT)-Fatigue ScoresSecondary· 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.
Group
Value
95% CI
Ravulizumab/Ravulizumab
2.01
0.64 – 3.39
Eculizumab/Ravulizumab
0.54
-0.84 – 1.93
Percentage Of Participants Who Achieved Transfusion Avoidance Through Day 183Secondary· 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.
Group
Value
95% CI
Ravulizumab/Ravulizumab
87.6
81.08 – 94.18
Eculizumab/Ravulizumab
82.7
75.16 – 90.15
Percentage Of Participants With Stabilized Hemoglobin Levels Through Day 183Secondary· 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.
Group
Value
95% CI
Ravulizumab/Ravulizumab
76.3
67.82 – 84.75
Eculizumab/Ravulizumab
75.5
67.00 – 84.02
Number Of Participants With Breakthrough Hemolysis Through End of StudySecondary· 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.
Group
Value
95% CI
Ravulizumab/Ravulizumab
9
4.38 – 17.05
Eculizumab/Ravulizumab
6
2.35 – 13.24
Change From Baseline To End of Study In FACIT-Fatigue Scores Through End of StudySecondary· 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.
Group
Value
95% CI
Ravulizumab/Ravulizumab
-1.43
± 5.694
Eculizumab/Ravulizumab
0.00
± 5.944
Percentage Of Participants Who Achieved Transfusion Avoidance Through End of StudySecondary· 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.
Group
Value
95% CI
Ravulizumab/Ravulizumab
70.83
60.67 – 79.67
Eculizumab/Ravulizumab
70.53
60.29 – 79.44
Percentage Of Participants With Stabilized Hemoglobin Levels Through End of StudySecondary· 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.
Group
Value
95% CI
Ravulizumab/Ravulizumab
58.33
47.82 – 68.32
Eculizumab/Ravulizumab
67.37
59.68 – 76.64
Adverse events — posted to ClinicalTrials.gov
Time frame: From Baseline (after first dose) to end of study (up to 4 years).
Reporting threshold: 5%.
Adverse-event reports describe events observed during the trial — not all are caused by the drug.
Primary Evaluation Period (26 Weeks) Ravulizumab Treatment
Serious: 4/97 (4%)
Deaths: 0/97
Primary Evaluation Period (26 Weeks) Eculizumab Treatment
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.
Publications & conference data
8 peer-reviewed publications reference this trial (live from Europe PMC):
NCT07399730 — Ravulizumab Outcomes in Polish Patients With aHUS
· not yet recruiting
NCT07221838 — A Study to Investigate OCS Tapering in Adult Participants With Generalized Myasthenia Gravis Treated With Ravulizumab
· Phase 4
· recruiting
NCT06333652 — Ravulizumab in Pregnancies Complicated by Severe Hypertensive Disorders
· Phase 2
· recruiting
NCT07308574 — Post-Marketing Clinical Study of Ravulizumab in Participants With Clinical aHUS
· Phase 4
· recruiting
NCT06967480 — Early Ravulizumab Treatment Of Anti- AChR Antibody-Positive Generalized Myasthenia Gravis
· recruiting
Other recruiting trials for Paroxysmal Nocturnal Hemoglobinuria (PNH)
Currently open trials in the same condition.
NCT07187401 — A First-in-Human Safety and Efficacy Study of ALN-CFB, a Small Interfering RNA (siRNA) Targeting Complement Factor B, in
· Phase 1, PHASE2
· recruiting
NCT07229235 — REAL-CARE: Real-world Effectiveness of Iptacopan in Italian Patients With Paroxysmal Nocturnal Hemoglobinuria
· recruiting
NCT06934967 — Study to Assess the Pharmacokinetics, Safety, and Tolerability of Iptacopan in Pediatric PNH Patients
· Phase 3
· recruiting
NCT06312644 — Study of Ultomiris® (Ravulizumab) Safety in Pregnancy
· recruiting
NCT06933914 — Long-term Safety and Tolerability of MY008211A Tablets in Patients With Paroxysmal Nocturnal Hemoglobinuria
· Phase 2, PHASE3
· recruiting
Other Alexion Pharmaceuticals, Inc. trials
Trials by the same sponsor.
NCT06015750 — Mitigate Immune-Mediated Loss of Therapeutic Response to Asfotase Alfa (STRENSIQ®) for Hypophosphatasia
· Phase 4
· withdrawn
NCT07352423 — Safety, Tolerability, Pharmacokinetic, and Pharmacodynamic Study of ALXN2230 in Healthy Participants
· Phase 1
· recruiting
NCT07221838 — A Study to Investigate OCS Tapering in Adult Participants With Generalized Myasthenia Gravis Treated With Ravulizumab
· Phase 4
· recruiting
NCT07413250 — Assess Long-Term Safety of Danicopan Add-on Therapy in Participants With Paroxysmal Nocturnal Hemoglobinuria: Analysis o
· active not recruiting
NCT07308574 — Post-Marketing Clinical Study of Ravulizumab in Participants With Clinical aHUS
· Phase 4
· recruiting
Publications: Europe PMC API search by NCT ID, retrieved 10 June 2026
Drug + disease cross-links: matched in real time against Drug Landscape's normalised drug + company + condition tables
Sponsor: as reported to ClinicalTrials.gov by Alexion Pharmaceuticals, Inc.
Last refreshed: 12 March 2025
Drug Landscape aggregates and links these public records for informational use only. Always verify against the primary source before clinical or regulatory decisions. Canonical URL: https://druglandscape.com/trial/NCT03056040.