ClinicalTrials.gg
CompletedNCT03056040Updated Mar 12, 2025Results posted

ALXN1210 Versus Eculizumab in Adult Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH) Currently Treated With Eculizumab

A Phase 3 interventional study of Ravulizumab and Eculizumab in Paroxysmal Nocturnal Hemoglobinuria (PNH), sponsored by Alexion Pharmaceuticals, Inc.. Completed at 51 sites in 11 countries. Open to participants aged 18 Years and older. Per ClinicalTrials.gov, last updated 2025-03-12.

Sponsored by Alexion Pharmaceuticals, Inc. (part of AstraZeneca) · Phase 3, Interventional, and Treatment

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Phase
Phase 3
Study type
Interventional
Enrollment
202
Allocation
Not applicable
Ages
18 Years and older
Sex
All
01

Where this study stands

Counted across the records on this site · 2026-11-14

Among 35 studies in Hemoglobinuria, Paroxysmal by AstraZeneca — 3 open to participants now, 7 under way, 2 of them phase 3. Hemoglobinuria, Paroxysmal · AstraZeneca.

Enrollment
2nd of 23
202 · median 26
Started
2017
with 2 others
Phase 3
12 of 35
1 open now
Registered
16th of 35
by date

Among the 35 studies in Hemoglobinuria, Paroxysmal by AstraZeneca, this one is the 2nd largest by enrollment of the 23 interventional ones that give a count, is one of 3 that started in 2017, is one of 12 in phase 3, and was the 16th registered.

Hemoglobinuria, Paroxysmal

191 studies on the registry are indexed under Hemoglobinuria, Paroxysmal; 49 are open to participants now.

Browse Hemoglobinuria, Paroxysmal studies →

Open to participants now

3 of the 35 studies in Hemoglobinuria, Paroxysmal by AstraZeneca are open now, in 8 countries. Recruiting first, the newest first:

By country, each count listing its studies:

All 3 open studies →

Who these studies take

Of the 30 Hemoglobinuria, Paroxysmal studies recruiting now, 14 (47%) set no upper age limit and 12 (40%) stop at 75 or younger; 12 take children under 18; 4 take healthy volunteers.

This study: 18 Years and older · all sexes · no healthy volunteers.

The full table on the hub →

This tab as a report to print or save as PDF → With an account.

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Watch this landscapeDownload CSVNarrow it in search →With an account: a weekly mail of what changed among 35 studies in Hemoglobinuria, Paroxysmal by AstraZeneca, or all of them as a file.
02

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.

Read the detailed description

The study consisted of a 4-week Screening Period and a 26-week Randomized Treatment Period (Primary Evaluation Period). After completion of the Primary Evaluation Period, all participants had the opportunity to enter the Extension Period, wherein participants will receive ravulizumab for up to 4 years.

03

Conditions studied

  • Paroxysmal Nocturnal Hemoglobinuria (PNH)
04

Who can participate

Ages eligible
18 Years and older
Sexes eligible
All
Accepts healthy volunteers
No

Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.

Inclusion criteria

  1. Male or female ≥18 years of age.
  2. Treated with eculizumab for PNH for at least 6 months prior to Day 1.
  3. Lactate dehydrogenase level ≤1.5 times the (ULN) at screening.
  4. PNH diagnosis confirmed by documented by high-sensitivity flow cytometry.
  5. Documented meningococcal vaccination not more than 3 years prior to, or at the time of, initiating study treatment.
  6. Female participants of must use highly effective starting at screening and continuing until at least 8 months after the last dose of ravulizumab.
  7. Willing and able to give written and comply with study visit schedule.

Exclusion criteria

Exclusion Criteria:

  1. History of bone marrow transplantation.
  2. Body weight \<40 kilograms at screening.
  3. History of or ongoing major cardiac, pulmonary, renal, endocrine, or hepatic disease that, in the opinion of the investigator or sponsor, would preclude participation.
  4. 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).
  5. Female participants who are pregnant, breastfeeding, or who have a positive pregnancy test at screening or Day 1.
  6. 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 product, whichever is greater.
Terms in these criteria, in plain language(5)

Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.

ULN (upper limit of normal)

The top of the normal range for a blood test, as set by the laboratory that runs it. Each lab prints its own range next to your result.

“≤ 2.5 × ULN” means the result can be up to two and a half times that top value.

Childbearing potential

Able to become pregnant: from the first period until menopause, unless permanently unable to through surgery (removal of the womb, or of both ovaries or both fallopian tubes).

Criteria for people of childbearing potential usually ask for a negative pregnancy test and for contraception during the study and for a time after it.

Contraception

Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.

Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.

Informed consent

Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.

You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.

Investigational drug or device

A drug or device being studied in a clinical trial.

It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.

05

Study design

Phase
Phase 3
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Parallel assignment
Masking
None (open label)
Enrollment
202 participants (actual)

Study arms

  • Experimental
    Ravulizumab

    On Day 1, participants received weight-based doses of ravulizumab ranging from 2400 to 3000 milligrams (mg). Thereafter, weight-based doses of ravulizumab ranging from 3000 to 3600 mg were administered on Days 15, 71, and 127. After completion of the 26-week Primary Evaluation Period, participants had the opportunity to enter the Extension Period, wherein participants will receive weight-based doses of ravulizumab for up to 4 years.

    Biological: Ravulizumab

  • Active comparator
    Eculizumab

    Participants received 900 mg of eculizumab every 2 weeks (q2w) for 26 weeks. After completion of the 26-week Primary Evaluation Period, participants had the opportunity to enter the Extension Period, wherein participants will receive weight-based doses of ravulizumab for up to 4 years.

    Biological: Ravulizumab · Biological: Eculizumab

Interventions

  • BiologicalRavulizumab

    All treatments were given as intravenous (IV) infusions. For participants weighing ≥40 to \<60 kilograms (kg): 2400 mg was given as a single loading dose, followed by 3000 mg as maintenance dose. For participants weighing ≥60 to \<100 kg: 2700 mg was given as a loading dose, followed by 3300 mg as maintenance dose. For participants weighing ≥100 kg: 3000 mg was given as a loading dose, followed by 3600 mg as maintenance dose.

    Also known as: ALXN1210, ULTOMIRIS

  • BiologicalEculizumab

    All treatments were given as IV infusions. Participants received 900 mg of eculizumab q2w.

06

What researchers measure

Primary outcomes

  1. Percent Change In Lactate Dehydrogenase Levels From Baseline To 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).

    Time frame: Baseline, Day 183

Secondary outcomes

  1. Number Of Participants With Breakthrough Hemolysis 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).

    Time frame: Baseline through Day 183

  2. Change From Baseline To Day 183 In Functional Assessment Of Chronic Illness Therapy (FACIT)-Fatigue Scores

    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.

    Time frame: Baseline, Day 183

  3. Percentage Of Participants Who Achieved Transfusion Avoidance 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.

    Time frame: Baseline through Day 183

  4. Percentage Of Participants With Stabilized Hemoglobin Levels 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.

    Time frame: Baseline through Day 183

  5. Number Of Participants With Breakthrough Hemolysis Through End of Study

    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.

    Time frame: Baseline through end of study (up to 4 years)

  6. Change From Baseline To End of Study In FACIT-Fatigue Scores Through End of Study

    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.

    Time frame: Baseline, End of Study (up to 4 years)

  7. Percentage Of Participants Who Achieved Transfusion Avoidance Through End of Study

    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.

    Time frame: Baseline through end of study (up to 4 years)

  8. Percentage Of Participants With Stabilized Hemoglobin Levels Through End of Study

    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.

    Time frame: Baseline through end of study (up to 4 years)

07

Results

Posted Mar 21, 2019

Participant flow

Primary Evaluation Period
Participant flow — Primary Evaluation Period
MilestoneRavulizumab/RavulizumabEculizumab/Ravulizumab
Started9899
Received at least 1 dose of study drug9798
Participants in the full analysis set (fas)9798
Completed9695
Not completed24
Withdrew: Withdrawal by subject11
Withdrew: Lack of efficacy01
Withdrew: Pregnancy01
Withdrew: Randomized, not treated11
Extension Period
Participant flow — Extension Period
MilestoneRavulizumab/RavulizumabEculizumab/Ravulizumab
Started9695
Completed9288
Not completed47
Withdrew: Physician decision33
Withdrew: Lost to follow-up01
Withdrew: Death03
Withdrew: Withdrawal by subject10

Outcome measures

PrimaryPercent Change In Lactate Dehydrogenase Levels From Baseline To 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).

Time frame:
Baseline, Day 183
Reported as:
Least squares mean · percent change
Percent Change In Lactate Dehydrogenase Levels From Baseline To Day 183
percent changeRavulizumab/RavulizumabEculizumab/Ravulizumab
Percent Change In Lactate Dehydrogenase Levels From Baseline To Day 183-0.82 (-7.75 to 6.11)8.39 (1.47 to 15.32)
Statistical analysis
  • Ravulizumab/Ravulizumab vs Eculizumab/Ravulizumab · Treatment difference: -9.21 · 95% CI -18.84 to 0.42Treatment difference was estimated for ravulizumab - eculizumab.
SecondaryNumber Of Participants With Breakthrough Hemolysis 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).

Time frame:
Baseline through Day 183
Reported as:
Number · number of participants
Number Of Participants With Breakthrough Hemolysis Through Day 183
number of participantsRavulizumab/RavulizumabEculizumab/Ravulizumab
Number Of Participants With Breakthrough Hemolysis Through Day 1830 (0.00 to 3.73)5 (1.68 to 11.51)
Statistical analysis
  • Ravulizumab/Ravulizumab vs Eculizumab/Ravulizumab · Treatment difference: -5.1 · 95% CI -18.99 to 8.89Treatment difference was estimated for ravulizumab - eculizumab.
SecondaryChange From Baseline To Day 183 In Functional Assessment Of Chronic Illness Therapy (FACIT)-Fatigue Scores

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.

Time frame:
Baseline, Day 183
Reported as:
Least squares mean · units on a scale
Change From Baseline To Day 183 In Functional Assessment Of Chronic Illness Therapy (FACIT)-Fatigue Scores
units on a scaleRavulizumab/RavulizumabEculizumab/Ravulizumab
Change From Baseline To Day 183 In Functional Assessment Of Chronic Illness Therapy (FACIT)-Fatigue Scores2.01 (0.64 to 3.39)0.54 (-0.84 to 1.93)
Statistical analysis
  • Ravulizumab/Ravulizumab vs Eculizumab/Ravulizumab · Treatment difference: 1.47 · 95% CI -0.21 to 3.15Treatment difference was estimated for ravulizumab - eculizumab.
SecondaryPercentage Of Participants Who Achieved Transfusion Avoidance 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.

Time frame:
Baseline through Day 183
Reported as:
Number · percentage of participants
Percentage Of Participants Who Achieved Transfusion Avoidance Through Day 183
percentage of participantsRavulizumab/RavulizumabEculizumab/Ravulizumab
Percentage Of Participants Who Achieved Transfusion Avoidance Through Day 18387.6 (81.08 to 94.18)82.7 (75.16 to 90.15)
Statistical analysis
  • Ravulizumab/Ravulizumab vs Eculizumab/Ravulizumab · Treatment difference: 5.5 · 95% CI -4.27 to 15.68Treatment difference was estimated for ravulizumab - eculizumab.
SecondaryPercentage Of Participants With Stabilized Hemoglobin Levels 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.

Time frame:
Baseline through Day 183
Reported as:
Number · percentage of participants
Percentage Of Participants With Stabilized Hemoglobin Levels Through Day 183
percentage of participantsRavulizumab/RavulizumabEculizumab/Ravulizumab
Percentage Of Participants With Stabilized Hemoglobin Levels Through Day 18376.3 (67.82 to 84.75)75.5 (67.00 to 84.02)
Statistical analysis
  • Ravulizumab/Ravulizumab vs Eculizumab/Ravulizumab · Treatment difference: 1.4 · 95% CI -10.41 to 13.31Treatment difference was estimated for ravulizumab - eculizumab.
SecondaryNumber Of Participants With Breakthrough Hemolysis Through End of Study

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.

Time frame:
Baseline through end of study (up to 4 years)
Reported as:
Number · number of participants
Number Of Participants With Breakthrough Hemolysis Through End of Study
number of participantsRavulizumab/RavulizumabEculizumab/Ravulizumab
Number Of Participants With Breakthrough Hemolysis Through End of Study9 (4.38 to 17.05)6 (2.35 to 13.24)
SecondaryChange From Baseline To End of Study In FACIT-Fatigue Scores Through End of Study

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.

Time frame:
Baseline, End of Study (up to 4 years)
Reported as:
Mean · units on a scale
Change From Baseline To End of Study In FACIT-Fatigue Scores Through End of Study
units on a scaleRavulizumab/RavulizumabEculizumab/Ravulizumab
Change From Baseline To End of Study In FACIT-Fatigue Scores Through End of Study-1.43 ± 5.6940.00 ± 5.944
SecondaryPercentage Of Participants Who Achieved Transfusion Avoidance Through End of Study

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.

Time frame:
Baseline through end of study (up to 4 years)
Reported as:
Number · percentage of participants
Percentage Of Participants Who Achieved Transfusion Avoidance Through End of Study
percentage of participantsRavulizumab/RavulizumabEculizumab/Ravulizumab
Percentage Of Participants Who Achieved Transfusion Avoidance Through End of Study70.83 (60.67 to 79.67)70.53 (60.29 to 79.44)
SecondaryPercentage Of Participants With Stabilized Hemoglobin Levels Through End of Study

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.

Time frame:
Baseline through end of study (up to 4 years)
Reported as:
Number · percentage of participants
Percentage Of Participants With Stabilized Hemoglobin Levels Through End of Study
percentage of participantsRavulizumab/RavulizumabEculizumab/Ravulizumab
Percentage Of Participants With Stabilized Hemoglobin Levels Through End of Study58.33 (47.82 to 68.32)67.37 (59.68 to 76.64)

Adverse events

Collected over From Baseline (after first dose) to end of study (up to 4 years). Non-serious events are listed at a 5% frequency threshold.

Adverse event summary by group
GroupDeathsSeriousOther
Primary Evaluation Period (26 Weeks) Ravulizumab Treatment0/97 (0%)4/97 (4.1%)72/97 (74.2%)
Primary Evaluation Period (26 Weeks) Eculizumab Treatment0/98 (0%)8/98 (8.2%)85/98 (86.7%)
Extension Period (Ravulizumab To Ravulizumab)0/96 (0%)26/96 (27.1%)83/96 (86.5%)
Extension Period (Eculizumab to Ravulizumab)3/95 (3.2%)33/95 (34.7%)85/95 (89.5%)
Most frequent serious events
Showing 10 of 65
Most frequent serious events
EventPrimary Evaluation Period (26 Weeks) Ravulizumab TreatmentPrimary Evaluation Period (26 Weeks) Eculizumab TreatmentExtension Period (Ravulizumab To Ravulizumab)Extension Period (Eculizumab to Ravulizumab)
PyrexiaGeneral disorders0/973/985/960/95
HaemolysisBlood and lymphatic system disorders0/972/981/964/95
InfluenzaInfections and infestations1/970/981/963/95
InfectionInfections and infestations0/970/980/962/95
Haemolytic anaemiaBlood and lymphatic system disorders0/970/982/961/95
CholelithiasisHepatobiliary disorders0/971/980/961/95
Lower respiratory tract infectionInfections and infestations1/970/981/961/95
AnaemiaBlood and lymphatic system disorders0/970/980/961/95
PneumoperitoneumGastrointestinal disorders0/970/980/961/95
ToothacheGastrointestinal disorders0/970/980/961/95
Most frequent other events
Showing 10 of 45
Most frequent other events
EventPrimary Evaluation Period (26 Weeks) Ravulizumab TreatmentPrimary Evaluation Period (26 Weeks) Eculizumab TreatmentExtension Period (Ravulizumab To Ravulizumab)Extension Period (Eculizumab to Ravulizumab)
NasopharyngitisInfections and infestations20/9720/9827/9621/95
HeadacheNervous system disorders27/9717/9821/9621/95
Upper respiratory tract infectionInfections and infestations19/9710/9824/9619/95
FatigueGeneral disorders7/976/9818/9619/95
NauseaGastrointestinal disorders8/979/9813/9616/95
PyrexiaGeneral disorders9/972/9815/9616/95
CoughRespiratory, thoracic and mediastinal disorders5/9710/9816/9612/95
DizzinessNervous system disorders3/977/988/9615/95
DiarrhoeaGastrointestinal disorders9/977/9815/9614/95
Back painMusculoskeletal and connective tissue disorders4/970/9810/9613/95

Baseline characteristics

All participants who received at least 1 dose of study treatment (ravulizumab or eculizumab).

Age, Continuous
Age, Continuous(years)Ravulizumab/RavulizumabEculizumab/RavulizumabTotal
Mean46.6 ± 14.4148.8 ± 13.9747.7 ± 14.19
Sex: Female, Male
Sex: Female, Male(Participants)Ravulizumab/RavulizumabEculizumab/RavulizumabTotal
Female475097
Male504898
Ethnicity (NIH/OMB)
Ethnicity (NIH/OMB)(Participants)Ravulizumab/RavulizumabEculizumab/RavulizumabTotal
Hispanic or Latino347
Not Hispanic or Latino7677153
Unknown or Not Reported181735
Race/Ethnicity, Customized
Race/Ethnicity, Customized(participants)Ravulizumab/RavulizumabEculizumab/RavulizumabTotal
White5061111
Asian231942
Not Reported131326
Black or African American538
Unknown314
Other213
Multiple101
08

Study locations

51 sites
  • Research Site
    Duarte, California 91010, United States
  • Research Site
    Los Angeles, California 90089, United States
  • Research Site
    Baltimore, Maryland 21205, United States
  • Research Site
    Detroit, Michigan 48202, United States
  • Research Site
    Bronx, New York 10467, United States
  • Research Site
    Canberra, 2605, Australia
  • Research Site
    Kogarah, 2217, Australia
  • Research Site
    Liverpool, 2170, Australia
  • Research Site
    Melbourne, 3000, Australia
  • Research Site
    Woolloongabba, 4102, Australia
  • Research Site
    Toronto, Ontario M4N 3M5, Canada
  • Research Site
    Toronto, Ontario M5G 2C4, Canada
  • Research Site
    Montreal, Quebec H1T 2M4, Canada
  • Research Site
    Amiens, 80054, France
  • Research Site
    La Tronche, 38043, France
  • Research Site
    Marseille, 13273, France
  • Research Site
    Nice, 06200, France
  • Research Site
    Paris, 75475, France
  • Research Site
    Pierre-Benite, 69495, France
  • Research Site
    Saint-Priest-En-Jarez, 42270, France
  • Research Site
    Strasbourg, 67091, France
  • Research Site
    Aachen, 52074, Germany
  • Research Site
    Essen, 45147, Germany
  • Research Site
    Ulm, 89081, Germany
  • Research Site
    Firenze, 50134, Italy
  • Research Site
    Milano, 20122, Italy
  • Research Site
    Napoli, 80131, Italy
  • Research Site
    Torino, 10126, Italy
  • Research Site
    Vicenza, 36100, Italy
  • Research Site
    Fukushima-shi, 960-1295, Japan
  • Research Site
    Kanazawa-shi, 920-8641, Japan
  • Research Site
    Shinagawa-ku, 141-0022, Japan
  • Research Site
    Suita-shi, 565-0871, Japan
  • Research Site
    Suwa-shi, 392-8510, Japan
  • Research Site
    Bucheon-si, 14584, Korea, Republic of
  • Research Site
    Daegu, 42415, Korea, Republic of
  • Research Site
    Daejeon, 35015, Korea, Republic of
  • Research Site
    Incheon, 21565, Korea, Republic of
  • Research Site
    Seoul, 03080, Korea, Republic of
  • Research Site
    Seoul, 06351, Korea, Republic of
  • Research Site
    Seoul, 3722, Korea, Republic of
  • Research Site
    Seoul, 6591, Korea, Republic of
  • Research Site
    Suwon, 16247, Korea, Republic of
  • Research Site
    Maastricht, 6202 AZ, Netherlands
  • Research Site
    Nijmegen, 6525 GA, Netherlands
  • Research Site
    Barcelona, 8036, Spain
  • Research Site
    Madrid, 28040, Spain
  • Research Site
    Majadahonda, 28220, Spain
  • Research Site
    Airdrie, ML6 0JS, United Kingdom
  • Research Site
    Leeds, LS9 7TF, United Kingdom
  • Research Site
    London, SE5 9NU, United Kingdom
09

References and documents

Publications

  • Kulasekararaj AG, Hill A, Rottinghaus ST, Langemeijer S, Wells R, Gonzalez-Fernandez FA, Gaya A, Lee JW, Gutierrez EO, Piatek CI, Szer J, Risitano A, Nakao S, Bachman E, Shafner L, Damokosh AI, Ortiz S, Roth A, Peffault de Latour R. Ravulizumab (ALXN1210) vs eculizumab in C5-inhibitor-experienced adult patients with PNH: the 302 study. Blood. 2019 Feb 7;133(6):540-549. doi: 10.1182/blood-2018-09-876805. Epub 2018 Dec 3. PubMed 30510079 ↗
  • Brodsky RA, Peffault de Latour R, Rottinghaus ST, Roth A, Risitano AM, Weitz IC, Hillmen P, Maciejewski JP, Szer J, Lee JW, Kulasekararaj AG, Volles L, Damokosh AI, Ortiz S, Shafner L, Liu P, Hill A, Schrezenmeier H. Characterization of breakthrough hemolysis events observed in the phase 3 randomized studies of ravulizumab versus eculizumab in adults with paroxysmal nocturnal hemoglobinuria. Haematologica. 2021 Jan 1;106(1):230-237. doi: 10.3324/haematol.2019.236877. PubMed 31949012 ↗
  • Ishiyama K, Nakao S, Usuki K, Yonemura Y, Ikezoe T, Uchiyama M, Mori Y, Fukuda T, Okada M, Fujiwara SI, Noji H, Rottinghaus S, Aguzzi R, Yokosawa J, Nishimura JI, Kanakura Y, Okamoto S. Results from multinational phase 3 studies of ravulizumab (ALXN1210) versus eculizumab in adults with paroxysmal nocturnal hemoglobinuria: subgroup analysis of Japanese patients. Int J Hematol. 2020 Oct;112(4):466-476. doi: 10.1007/s12185-020-02934-6. Epub 2020 Aug 31. PubMed 32869125 ↗
  • Kulasekararaj AG, Griffin M, Langemeijer S, Usuki K, Kulagin A, Ogawa M, Yu J, Mujeebuddin A, Nishimura JI, Lee JW, Peffault de Latour R; 301/302 Study Group. Long-term safety and efficacy of ravulizumab in patients with paroxysmal nocturnal hemoglobinuria: 2-year results from two pivotal phase 3 studies. Eur J Haematol. 2022 Sep;109(3):205-214. doi: 10.1111/ejh.13783. Epub 2022 Jun 16. PubMed 35502600 ↗
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Related links

Study documents

PDFs hosted by the registry, ClinicalTrials.gov.

10

Timeline

Tracking since Sep 25, 2026
ElapsedEstimatedEstimated, not yet reachedSimilar studies: half by the dot; the band is the middle halfHalf of similar studies reached it by the dot; the band is the middle half, from a quarter to three quarters

Ran from May 2017 to primary completion in Feb 2022, 57 months.

Of 35 industry-sponsored phase 3 interventional studies under Hemoglobinuria, Paroxysmal, half reached primary completion within 23 months of starting (the median), counting those still running; a quarter within 15 and three quarters within 41. This study's 57 months is above that.

Counting every sponsor, half of 35 studies reached it within 23 months; a quarter within 15 and three quarters within 41.

How these figures are worked out, and which studies are left out of them.

Registered
Feb 14, 2017
First posted
Feb 16, 2017
StartActual
May 17, 2017
Primary completionActual
Feb 21, 2022
CompletionActual
Feb 21, 2022
Results first posted
Mar 21, 2019
Last update
Mar 12, 2025
What these dates mean
Start.
When the first participant enrolled; while estimated, when the study expects to open to recruitment.
Primary completion.
When the last participant was examined or treated to collect data for the study's main question, its primary outcome. A study that stopped early gives the date its data collection stopped.
Completion.
When the last participant's last visit took place, for every outcome, secondary ones and side effects included: the end of the study. Often later than primary completion, sometimes the same day.
Actual and Est.
Actual means the registry says the date has happened. Est. (estimated) is a date the sponsor plans or expects. It can still move, and the sponsor is expected to change it to actual once it has passed.
No changes since tracking began. The registry record was last updated on Mar 12, 2025, before this site started recording changes on Sep 25, 2026. Its history is on ClinicalTrials.gov ↗
11

Registry details

Key details

Study ID
NCT03056040
Responsible party
Sponsor
First posted
Feb 16, 2017
Start date
May 17, 2017
Primary completion
Feb 21, 2022
Completion
Feb 21, 2022
Results posted
Mar 21, 2019
Last update
Mar 12, 2025

Oversight

Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No
View the source record on ClinicalTrials.gov ↗

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Discussion

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Studies of the drug tested here: Ravulizumab

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