ClinicalTrials.gg
CompletedNCT02946463Updated May 14, 2024Results posted

ALXN1210 (Ravulizumab) Versus Eculizumab in Complement Inhibitor Treatment-Naïve Adult Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH)

A Phase 3 interventional study of Ravulizumab and Eculizumab in Paroxysmal Nocturnal Hemoglobinuria (PNH), sponsored by Alexion Pharmaceuticals, Inc.. Completed at 120 sites in 26 countries. Per ClinicalTrials.gov, last updated 2024-05-14.

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

Help build ClinicalTrials.gg — short surveys, remote interviews or testing, in your language. Take part →

Phase
Phase 3
Study type
Interventional
Enrollment
272
Allocation
Randomized
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
Largest of 23
272 · median 26
Started
2016
with 1 other
Phase 3
12 of 35
1 open now
Registered
14th of 35
by date

Among the 35 studies in Hemoglobinuria, Paroxysmal by AstraZeneca, this one has the largest enrollment of the 23 interventional ones that give a count, is one of 2 that started in 2016, is one of 12 in phase 3, and was the 14th 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: 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.

Was this useful?
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 had never been treated with a complement inhibitor (treatment-naïve).

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 26-week Primary Evaluation Period, all participants had the opportunity to enter the Extension Period, wherein participants will receive ravulizumab for up to 5 years.

03

Conditions studied

  • Paroxysmal Nocturnal Hemoglobinuria (PNH)
04

Who can participate

Ages eligible
Child (0–17), Adult (18–64), Older adult (65+)
Sexes eligible
All
Accepts healthy volunteers
No

Eligibility criteria

Criteria For Patient Cohort Originally Enrolled in ALXN1210-PNH-301 Study: Inclusion Criteria: 1. Male or female ≥18 years of age. 2. PNH diagnosis confirmed by documented by high-sensitivity flow cytometry. 3. Presence of 1 or more of the following PNH-related signs or symptoms within 3 months of screening: fatigue, hemoglobinuria, abdominal pain, shortness of breath (dyspnea), anemia ( \<10 gram/deciliter), history of a major adverse vascular event (including thrombosis), dysphagia, or erectile dysfunction; or history of packed red blood cells (pRBC) transfusion due to PNH. 4. Lactate dehydrogenase (LDH) level ≥1.5 times the at screening. 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: 1. Treatment with a complement inhibitor at any time. 2. History of bone marrow transplantation. 3. Body weight \<40 kg. 4. Females who are pregnant, breastfeeding, or who have a positive pregnancy test at screening or Day 1. 5. Participation in another interventional clinical study or use of any experimental therapy within 30 days before initiation of study drug on Day 1 in this study or within 5 half-lives of that product, whichever is greater. 6. History of or ongoing major cardiac, pulmonary, renal, endocrine, or hepatic disease that, in the opinion of the investigator or sponsor, would preclude participation. 7. Unstable medical conditions (for example, myocardial ischemia, active gastrointestinal bleed, severe congestive heart failure, anticipated need for major surgery within 6 months of randomization, coexisting chronic anemia unrelated to PNH). Eligibility Criteria For Roll-over Cohort: 1. All participants regardless of age, who are currently receiving ALXN1210 IV in an ongoing ALXN1210 study in patients with PNH 2. Participants must be willing and able to give written informed consent and to comply with all Extension study visits and procedures, including the use of any data collection device(s) to directly record patient data 3. Females of childbearing potential and male patients with female partners of childbearing potential must use highly effective contraception continuing until at least 8 months after the last dose of ravulizumab.

Terms in these criteria, in plain language(6)

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.

Hemoglobin (Hb)

The protein in red blood cells that carries oxygen. A low level is anaemia, which can cause tiredness and breathlessness. Measured in g/dL or g/L (9 g/dL is 90 g/L).

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
Randomized
Intervention model
Parallel assignment
Masking
None (open label)
Enrollment
272 participants (actual)

Study arms

  • Experimental
    Ravulizumab

    Biological: Ravulizumab

  • Active comparator
    Eculizumab

    Biological: Ravulizumab · Biological: Eculizumab

Interventions

  • BiologicalRavulizumab

    All treatments were given as intravenous (IV) infusions. For participants weighing ≥40 to \<60 kilogram (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.

  • BiologicalEculizumab

    All treatments were given as IV infusions. Participants were administered induction doses of 600 mg followed by maintenance doses of 900 mg.

06

What researchers measure

Primary outcomes

  1. Proportion Of Participants With Normalization Of Lactate Dehydrogenase (LDH) Levels

    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.

    Time frame: Day 29 through Day 183

  2. Percentage Of Participants Who Achieved Transfusion Avoidance (TA)

    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.

    Time frame: Baseline through Day 183

Secondary outcomes

  1. Percentage Of Participants With Breakthrough Hemolysis (BTH)

    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.

    Time frame: Baseline through Day 183

  2. Percent Change From Baseline In LDH Levels

    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.

    Time frame: Baseline, Day 183

  3. Change From Baseline In Quality Of Life As Assessed By The Functional Assessment Of Chronic Illness Therapy (FACIT)-Fatigue

    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.

    Time frame: Baseline, Day 183

  4. Percentage Of Participants With Stabilized Hemoglobin Levels

    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

07

Results

Posted Feb 12, 2019

Participant flow

24 adult and 2 pediatric participants rolled over from other Alexion ongoing studies of ravulizumab intravenous in participants with PNH into the extension period of this study and received weight-based doses of ravulizumab until end of study.

Primary Evaluation Period
Participant flow — Primary Evaluation Period
MilestoneRavulizumab/RavulizumabEculizumab/RavulizumabRoll-Over Cohort: Ravulizumab - AdultsRoll-Over Cohort: Ravulizumab - Pediatrics
Started12512100
Received at least 1 dose of study drug12512100
Completed12511900
Not completed0200
Withdrew: Physician decision0100
Withdrew: Withdrawal by subject0100
Extension Period
Participant flow — Extension Period
MilestoneRavulizumab/RavulizumabEculizumab/RavulizumabRoll-Over Cohort: Ravulizumab - AdultsRoll-Over Cohort: Ravulizumab - Pediatrics
Started124119242
Received at least 1 dose of ravulizumab124119242
Completed108102222
Not completed161720
Withdrew: Death4400
Withdrew: Withdrawal by subject4300
Withdrew: Other than specified2320
Withdrew: Physician decision1400
Withdrew: Pregnancy3100
Withdrew: Adverse event1200
Withdrew: Lost to follow-up1000

Outcome measures

PrimaryProportion Of Participants With Normalization Of Lactate Dehydrogenase (LDH) Levels

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.

Time frame:
Day 29 through Day 183
Reported as:
Number · proportion of participants
Proportion Of Participants With Normalization Of Lactate Dehydrogenase (LDH) Levels
proportion of participantsRavulizumabEculizumab
Proportion Of Participants With Normalization Of Lactate Dehydrogenase (LDH) Levels0.536 (0.459 to 0.612)0.494 (0.417 to 0.570)
Statistical analysis
  • Ravulizumab vs Eculizumab · Odds ratio (or): 1.187 · 95% CI 0.796 to 1.769
PrimaryPercentage Of Participants Who Achieved Transfusion Avoidance (TA)

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.

Time frame:
Baseline through Day 183
Reported as:
Number · percentage of participants
Percentage Of Participants Who Achieved Transfusion Avoidance (TA)
percentage of participantsRavulizumabEculizumab
Percentage Of Participants Who Achieved Transfusion Avoidance (TA)73.6 (65.87 to 81.33)66.1 (57.68 to 74.55)
Statistical analysis
  • Ravulizumab vs Eculizumab · Treatment difference: 6.8 · 95% CI -4.66 to 18.14Treatment difference was estimated for ravulizumab - eculizumab.
SecondaryPercentage Of Participants With Breakthrough Hemolysis (BTH)

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.

Time frame:
Baseline through Day 183
Reported as:
Number · percentage of participants
Percentage Of Participants With Breakthrough Hemolysis (BTH)
percentage of participantsRavulizumabEculizumab
Percentage Of Participants With Breakthrough Hemolysis (BTH)4.0 (0.56 to 7.44)10.7 (5.23 to 16.26)
Statistical analysis
  • Ravulizumab vs Eculizumab · Treatment difference: -6.7 · 95% CI -14.21 to 0.18Treatment difference was estimated for ravulizumab - eculizumab.
SecondaryPercent Change From Baseline In LDH Levels

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.

Time frame:
Baseline, Day 183
Reported as:
Least squares mean · percent change
Percent Change From Baseline In LDH Levels
percent changeRavulizumabEculizumab
Percent Change From Baseline In LDH Levels-76.84 (-79.96 to -73.73)-76.02 (-79.20 to -72.83)
Statistical analysis
  • Ravulizumab vs Eculizumab · Treatment difference: -0.83 · 95% CI -5.21 to 3.56Treatment difference was estimated for ravulizumab - eculizumab.
SecondaryChange From Baseline In Quality Of Life As Assessed By The Functional Assessment Of Chronic Illness Therapy (FACIT)-Fatigue

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.

Time frame:
Baseline, Day 183
Reported as:
Least squares mean · units on a scale
Change From Baseline In Quality Of Life As Assessed By The Functional Assessment Of Chronic Illness Therapy (FACIT)-Fatigue
units on a scaleRavulizumabEculizumab
Change From Baseline In Quality Of Life As Assessed By The Functional Assessment Of Chronic Illness Therapy (FACIT)-Fatigue7.07 (5.55 to 8.60)6.40 (4.85 to 7.96)
Statistical analysis
  • Ravulizumab vs Eculizumab · Treatment difference: 0.67 · 95% CI -1.21 to 2.55Treatment difference was estimated for ravulizumab - eculizumab.
SecondaryPercentage Of Participants With Stabilized Hemoglobin Levels

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
percentage of participantsRavulizumabEculizumab
Percentage Of Participants With Stabilized Hemoglobin Levels68.0 (59.82 to 76.18)64.5 (55.93 to 72.99)
Statistical analysis
  • Ravulizumab vs Eculizumab · Treatment difference: 2.9 · 95% CI -8.80 to 14.64Treatment difference was estimated for ravulizumab - eculizumab.

Adverse events

Collected over Baseline up to Day 2045. Non-serious events are listed at a 5% frequency threshold.

Adverse event summary by group
GroupDeathsSeriousOther
Ravulizumab: Primary Evaluation0/125 (0%)11/125 (8.8%)87/125 (69.6%)
Eculizumab: Primary Evaluation0/121 (0%)9/121 (7.4%)86/121 (71.1%)
Ravulizumab/Ravulizumab: Extension Period4/124 (3.2%)52/124 (41.9%)102/124 (82.3%)
Eculizumab/Ravulizumab: Extension Period4/119 (3.4%)43/119 (36.1%)100/119 (84%)
Ravulizumab-Adults0/24 (0%)2/24 (8.3%)13/24 (54.2%)
Ravulizumab-Pediatrics0/2 (0%)0/2 (0%)0/2 (0%)
Most frequent serious events
Showing 10 of 106
Most frequent serious events
EventRavulizumab: Primary EvaluationEculizumab: Primary EvaluationRavulizumab/Ravulizumab: Extension PeriodEculizumab/Ravulizumab: Extension PeriodRavulizumab-AdultsRavulizumab-Pediatrics
AnaemiaBlood and lymphatic system disorders1/1250/1217/1242/1190/240/2
PneumoniaInfections and infestations0/1251/1215/1246/1190/240/2
COVID-19Infections and infestations0/1250/1216/1243/1190/240/2
Aplastic anaemiaBlood and lymphatic system disorders1/1250/1215/1241/1191/240/2
CholelithiasisHepatobiliary disorders0/1250/1212/1240/1191/240/2
Cholecystitis acuteHepatobiliary disorders0/1250/1211/1244/1190/240/2
PyrexiaGeneral disorders1/1252/1214/1243/1190/240/2
HaemolysisBlood and lymphatic system disorders0/1250/1214/1242/1190/240/2
Haemolytic anaemiaBlood and lymphatic system disorders0/1250/1212/1243/1190/240/2
PancytopeniaBlood and lymphatic system disorders0/1250/1210/1243/1190/240/2
Most frequent other events
Showing 10 of 42
Most frequent other events
EventRavulizumab: Primary EvaluationEculizumab: Primary EvaluationRavulizumab/Ravulizumab: Extension PeriodEculizumab/Ravulizumab: Extension PeriodRavulizumab-AdultsRavulizumab-Pediatrics
COVID-19Infections and infestations0/1250/12119/12416/1199/240/2
HeadacheNervous system disorders45/12540/12116/12420/1192/240/2
Upper respiratory tract infectionInfections and infestations13/1257/12130/12425/1190/240/2
NasopharyngitisInfections and infestations11/12520/12114/12426/1190/240/2
PyrexiaGeneral disorders6/12511/12122/12418/1192/240/2
AstheniaGeneral disorders0/1250/1210/1240/1194/240/2
CoughRespiratory, thoracic and mediastinal disorders4/1258/12114/12418/1190/240/2
ArthralgiaMusculoskeletal and connective tissue disorders11/12511/12117/12412/1190/240/2
DiarrhoeaGastrointestinal disorders10/1255/12114/12413/1190/240/2
Abdominal painGastrointestinal disorders7/1257/12110/12412/1192/240/2

Baseline characteristics

All participants who received at least 1 dose of study drug.

Age, Categorical
Age, Categorical(Participants)Ravulizumab/RavulizumabEculizumab/RavulizumabRoll-Over Cohort: Ravulizumab - AdultsRoll-Over Cohort: Ravulizumab - PediatricsTotal
<=18 years00022
Between 18 and 65 years111103220236
>=65 years14182034
Sex: Female, Male
Sex: Female, Male(Participants)Ravulizumab/RavulizumabEculizumab/RavulizumabRoll-Over Cohort: Ravulizumab - AdultsRoll-Over Cohort: Ravulizumab - PediatricsTotal
Female6052121125
Male6569121147
Ethnicity (NIH/OMB)
Ethnicity (NIH/OMB)(Participants)Ravulizumab/RavulizumabEculizumab/RavulizumabRoll-Over Cohort: Ravulizumab - AdultsRoll-Over Cohort: Ravulizumab - PediatricsTotal
Hispanic or Latino5136226
Not Hispanic or Latino11610200218
Unknown or Not Reported4618028
Race (NIH/OMB)
Race (NIH/OMB)(Participants)Ravulizumab/RavulizumabEculizumab/RavulizumabRoll-Over Cohort: Ravulizumab - AdultsRoll-Over Cohort: Ravulizumab - PediatricsTotal
American Indian or Alaska Native11002
Asian725710130
Native Hawaiian or Other Pacific Islander00000
Black or African American24006
White4351112107
More than one race00000
Unknown or Not Reported7812027
08

Study locations

120 sites
  • Research Site
    Los Angeles, California 90033, United States
  • Research Site
    Whittier, California 90603, United States
  • Research Site
    Fort Worth, Texas 76104, United States
  • Research Site
    Buenos Aires, C1015ABO, Argentina
  • Research Site
    Buenos Aires, C1425AUM, Argentina
  • Research Site
    Córdoba, X5004BAL, Argentina
  • Research Site
    Perth, 6000, Australia
  • Research Site
    Linz, 4020, Austria
  • Research Site
    Vienna, A-1090, Austria
  • Research Site
    Bruxelles, 1200, Belgium
  • Research Site
    Hasselt, 3500, Belgium
  • Research Site
    Leuven, 3000, Belgium
  • Research Site
    Rio De Janeiro, Brazil
  • Research Site
    Salvador, 41253-190, Brazil
  • Research Site
    Sao Paulo, 05403-000, Brazil
  • Research Site
    Sao Paulo, Brazil
  • Research Site
    Edmonton, Alberta T6G 2G3, Canada
  • Research Site
    Toronto, Ontario M4N 3M5, Canada
  • Research Site
    Plzeň, 323 00, Czechia
  • Research Site
    Prague, Czechia
  • Research Site
    Tallinn, 13419, Estonia
  • Research Site
    Limoges, 87042, France
  • Research Site
    MONTPELLIER Cedex 5, 34295, France
  • Research Site
    Paris Cedex 10, 75475, France
  • Research Site
    Pierre Benite, 69310, France
  • Research Site
    Poitiers, 86021, France
  • Research Site
    Rennes Cedex 9, 35033, France
  • Research Site
    Aachen, 52074, Germany
  • Research Site
    Essen, 45122, Germany
  • Research Site
    Ulm, 89081, Germany
  • Research Site
    Ascoli Piceno, 63100, Italy
  • Research Site
    Firenze, 50134, Italy
  • Research Site
    Milano, 20122, Italy
  • Research Site
    Napoli, 80131, Italy
  • Research Site
    Vicenza, 36100, Italy
  • Research Site
    Bunkyo-ku, 113-8431, Japan
  • Research Site
    Bunkyo-ku, 113-8519, Japan
  • Research Site
    Fukuoka-Shi, 812-8582, Japan
  • Research Site
    Fukushima-shi, 960-1295, Japan
  • Research Site
    Hamamatsu-shi, 432-8580, Japan
  • Research Site
    Kanazawa-shi, 920-8641, Japan
  • Research Site
    Kitakyusyu-shi, 806-8501, Japan
  • Research Site
    Koshigaya-shi, 343-8555, Japan
  • Research Site
    Kumamoto-shi, 860-8556, Japan
  • Research Site
    Nagoya-shi, 453-8511, Japan
  • Research Site
    Nishinomiya-shi, 663-8501, Japan
  • Research Site
    Ogaki-shi, 503-8502, Japan
  • Research Site
    Okayama-shi, 700-8558, Japan
  • Research Site
    Okayama-shi, 701-1192, Japan
  • Research Site
    Osakasayama-shi, 589-8511, Japan
  • Research Site
    Sapporo-shi, 060-8543, Japan
  • Research Site
    Shimotsuke-shi, 329-0498, Japan
  • Research Site
    Shinjuku-ku, 160-0023, Japan
  • Research Site
    Shinjuku-ku, 160-8582, Japan
  • Research Site
    Suita, 565-0871, Japan
  • Research Site
    Tokorozawa-shi, Japan
  • Research Site
    Tokyo, Japan
  • Research Site
    Toyoake-shi, 470-1192, Japan
  • Research Site
    Tsukuba-shi, 305-8576, Japan
  • Research Site
    Wakayama-shi, 641-8510, Japan
  • Research Site
    Daejeon, 35015, Korea, Republic of
  • Research Site
    Goyang-si, 10408, Korea, Republic of
  • Research Site
    Incheon, 21565, Korea, Republic of
  • Research Site
    Jeonju-si, 561-712, Korea, Republic of
  • Research Site
    JinJoo, 52727, Korea, Republic of
  • Research Site
    Jung-gu, 41944, Korea, Republic of
  • Research Site
    Seoul, 02841, Korea, Republic of
  • Research Site
    Seoul, 03080, Korea, Republic of
  • Research Site
    Seoul, 03722, Korea, Republic of
  • Research Site
    Seoul, 04401, Korea, Republic of
  • Research Site
    Seoul, 06591, Korea, Republic of
  • Research Site
    Seoul, 07985, Korea, Republic of
  • Research Site
    Seoul, 135-710, Korea, Republic of
  • Research Site
    Seoul, 152703, Korea, Republic of
  • Research Site
    Songpa-gu, 05505, Korea, Republic of
  • Research Site
    Suwon-si, 16247, Korea, Republic of
  • Research Site
    Ulsan, 44033, Korea, Republic of
  • Research Site
    George, 10990, Malaysia
  • Research Site
    Johor Bahru, 80100, Malaysia
  • Research Site
    Kota Bharu, 15586, Malaysia
  • Research Site
    Kota Bharu, 16150, Malaysia
  • Research Site
    Kota Kinabalu, 88586, Malaysia
  • Research Site
    Kuching, 93586, Malaysia
  • Research Site
    Miri, 98000, Malaysia
  • Research Site
    Sibu, 96000, Malaysia
  • Research Site
    Monterrey, 64460, Mexico
  • Research Site
    Gdańsk, 80-214, Poland
  • Research Site
    Warszawa, 02-172, Poland
  • Research Site
    Arkhangelsk, 163045, Russian Federation
  • Research Site
    Barnaul, 656024, Russian Federation
  • Research Site
    Irkutsk, 664079, Russian Federation
  • Research Site
    Kirov, 610027, Russian Federation
  • Research Site
    Moscow, 117997, Russian Federation
  • Research Site
    Moscow, 125284, Russian Federation
  • Research Site
    Murmansk, 183047, Russian Federation
  • Research Site
    Novosibirsk, 630091, Russian Federation
  • Research Site
    Omsk, 644013, Russian Federation
  • Research Site
    Petrozavodsk, 185019, Russian Federation
  • Research Site
    Rostov-on-Don, 344022, Russian Federation
  • Research Site
    Saint-Petersburg, 197022, Russian Federation

Showing the first 100 of 120 sites across 26 countries. Choose a country and city above to see the rest.

09

References and documents

Publications

  • Lee JW, Sicre de Fontbrune F, Wong Lee Lee L, Pessoa V, Gualandro S, Fureder W, Ptushkin V, Rottinghaus ST, Volles L, Shafner L, Aguzzi R, Pradhan R, Schrezenmeier H, Hill A. Ravulizumab (ALXN1210) vs eculizumab in adult patients with PNH naive to complement inhibitors: the 301 study. Blood. 2019 Feb 7;133(6):530-539. doi: 10.1182/blood-2018-09-876136. Epub 2018 Dec 3. PubMed 30510080 ↗
  • Schwartz CE, Stark RB, Borowiec K, Nolte S, Myren KJ. Norm-based comparison of the quality-of-life impact of ravulizumab and eculizumab in paroxysmal nocturnal hemoglobinuria. Orphanet J Rare Dis. 2021 Sep 15;16(1):389. doi: 10.1186/s13023-021-02016-8. PubMed 34526067 ↗
  • Schrezenmeier H, Kulasekararaj A, Mitchell L, Sicre de Fontbrune F, Devos T, Okamoto S, Wells R, Rottinghaus ST, Liu P, Ortiz S, Lee JW, Socie G. One-year efficacy and safety of ravulizumab in adults with paroxysmal nocturnal hemoglobinuria naive to complement inhibitor therapy: open-label extension of a randomized study. Ther Adv Hematol. 2020 Oct 24;11:2040620720966137. doi: 10.1177/2040620720966137. eCollection 2020. PubMed 33178408 ↗
  • 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 ↗
  • 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 ↗

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 Dec 2016 to primary completion in Feb 2023, 74 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 74 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
Oct 25, 2016
First posted
Oct 27, 2016
StartActual
Dec 12, 2016
Primary completionActual
Feb 28, 2023
CompletionActual
Feb 28, 2023
Results first posted
Feb 12, 2019
Last update
May 14, 2024
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 May 14, 2024, before this site started recording changes on Sep 25, 2026. Its history is on ClinicalTrials.gov ↗
11

Registry details

Key details

Study ID
NCT02946463
Responsible party
Sponsor
First posted
Oct 27, 2016
Start date
Dec 12, 2016
Primary completion
Feb 28, 2023
Completion
Feb 28, 2023
Results posted
Feb 12, 2019
Last update
May 14, 2024

Oversight

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

Not currently enrolling

This study is completed, as verified in Apr 2024. You cannot join it, but the record below documents what was studied.

Follow this study

Get an email when the registry record changes — status, dates, results — or when someone posts here.

Sign in to follow

Discussion

Questions and observations about this study, from anyone following it. Not medical advice, and not a channel to the study team — their contact details are on the registry record.

Sign in to join the discussion. Reading takes no account; posting does. You choose a display name, and a pseudonym is the default.

Nothing here yet. If you are running this trial, taking part in it, or weighing whether to, this is the place to say so.

Studies of the drug tested here: Ravulizumab

Start the discussion