Back to resultsALXN1210 Versus Eculizumab in Adult Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH) Currently Treated With Eculizumab
Primary Purpose
Paroxysmal Nocturnal Hemoglobinuria (PNH)
Status
Completed
Phase
Phase 3
Locations
International
Study Type
Interventional
Intervention
Ravulizumab
Eculizumab
Sponsored by
About this trial
This is an interventional treatment trial for Paroxysmal Nocturnal Hemoglobinuria (PNH)
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.
Sites / Locations
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Arms of the Study
Arm 1
Arm 2
Arm Type
Experimental
Active Comparator
Arm Label
Ravulizumab
Eculizumab
Arm Description
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 3 years.
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 3 years.
Outcomes
Primary Outcome Measures
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).
Secondary Outcome Measures
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).
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.
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.
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.
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.
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.
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.
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.
Full Information
1. Study Identification
Unique Protocol Identification Number
NCT03056040
Brief Title
ALXN1210 Versus Eculizumab in Adult Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH) Currently Treated With Eculizumab
Official Title
A Phase 3, Randomized, Open-Label, Active-Controlled Study of ALXN1210 Versus Eculizumab in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) Currently Treated With Eculizumab
Study Type
Interventional
2. Study Status
Record Verification Date
February 2023
Overall Recruitment Status
Completed
Study Start Date
June 5, 2017 (Actual)
Primary Completion Date
March 31, 2022 (Actual)
Study Completion Date
April 8, 2022 (Actual)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Alexion
4. Oversight
Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
Yes
5. Study Description
Brief 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.
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 3 years.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Paroxysmal Nocturnal Hemoglobinuria (PNH)
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 3
Interventional Study Model
Parallel Assignment
Masking
None (Open Label)
Allocation
Randomized
Enrollment
195 (Actual)
8. Arms, Groups, and Interventions
Arm Title
Ravulizumab
Arm Type
Experimental
Arm Description
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 3 years.
Arm Title
Eculizumab
Arm Type
Active Comparator
Arm Description
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 3 years.
Intervention Type
Biological
Intervention Name(s)
Ravulizumab
Other Intervention Name(s)
ALXN1210, ULTOMIRIS
Intervention Description
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.
Intervention Type
Biological
Intervention Name(s)
Eculizumab
Intervention Description
All treatments were given as IV infusions. Participants received 900 mg of eculizumab q2w.
Primary Outcome Measure Information:
Title
Percent Change In Lactate Dehydrogenase Levels From Baseline To Day 183
Description
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 Outcome Measure Information:
Title
Number Of Participants With Breakthrough Hemolysis Through Day 183
Description
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
Title
Change From Baseline To Day 183 In Functional Assessment Of Chronic Illness Therapy (FACIT)-Fatigue Scores
Description
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
Title
Percentage Of Participants Who Achieved Transfusion Avoidance Through Day 183
Description
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
Title
Percentage Of Participants With Stabilized Hemoglobin Levels Through Day 183
Description
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
Title
Number Of Participants With Breakthrough Hemolysis Through End of Study
Description
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 5 years)
Title
Change From Baseline To End of Study In FACIT-Fatigue Scores Through End of Study
Description
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 5 years)
Title
Percentage Of Participants Who Achieved Transfusion Avoidance Through End of Study
Description
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 5 years)
Title
Percentage Of Participants With Stabilized Hemoglobin Levels Through End of Study
Description
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 5 years)
10. Eligibility
Sex
All
Minimum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
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.
Facility Information:
Facility Name
Clinical Trial Site
City
Duarte
State/Province
California
ZIP/Postal Code
91010
Country
United States
Facility Name
Clinical Trial Site
City
Los Angeles
State/Province
California
ZIP/Postal Code
90033
Country
United States
Facility Name
Clinical Trial Site
City
Baltimore
State/Province
Maryland
ZIP/Postal Code
21205
Country
United States
Facility Name
Clinical Trial Site
City
Detroit
State/Province
Michigan
ZIP/Postal Code
48202
Country
United States
Facility Name
Clinical Trial Site
City
Bronx
State/Province
New York
ZIP/Postal Code
10467
Country
United States
Facility Name
Clinical Trial Site
City
Canberra
State/Province
Australian Capital Territory
ZIP/Postal Code
2605
Country
Australia
Facility Name
Clinical Trial Site
City
Kogarah
State/Province
New South Wales
ZIP/Postal Code
2217
Country
Australia
Facility Name
Clinical Trial Site
City
Liverpool
State/Province
New South Wales
ZIP/Postal Code
2170
Country
Australia
Facility Name
Clinical Trial Site
City
Woolloongabba
State/Province
Queensland
ZIP/Postal Code
4102
Country
Australia
Facility Name
Clinical Trial Site
City
Parkville
State/Province
Victoria
ZIP/Postal Code
3050
Country
Australia
Facility Name
Clinical Trial Site
City
Toronto
State/Province
Ontario
ZIP/Postal Code
M4N 3M5
Country
Canada
Facility Name
Clinical Trial Site
City
Toronto
State/Province
Ontario
ZIP/Postal Code
M5G 2C4
Country
Canada
Facility Name
Clinical Trial Site
City
Montréal
State/Province
Quebec
ZIP/Postal Code
H1T 2M4
Country
Canada
Facility Name
Clinical Trial Site
City
Amiens
ZIP/Postal Code
80054
Country
France
Facility Name
Clinical Trial Site
City
Marseille
ZIP/Postal Code
13273
Country
France
Facility Name
Clinical Trial Site
City
Paris
ZIP/Postal Code
75475
Country
France
Facility Name
Clinical Trial Site
City
Pierre-Bénite
ZIP/Postal Code
69495
Country
France
Facility Name
Clinical Trial Site
City
Saint-Priest-en-Jarez
ZIP/Postal Code
42271
Country
France
Facility Name
Clinical Trial Site
City
Strasbourg
ZIP/Postal Code
67091
Country
France
Facility Name
Clinical Trial Site
City
Ulm
State/Province
Baden Wuerttemberg
ZIP/Postal Code
89081
Country
Germany
Facility Name
Clinical Trial Site
City
Aachen
State/Province
Nordrhein Westfalen
ZIP/Postal Code
52074
Country
Germany
Facility Name
Clinical Trial Site
City
Essen
State/Province
Nordrhein Westfalen
ZIP/Postal Code
45147
Country
Germany
Facility Name
Clinical Trial Site
City
Firenze
ZIP/Postal Code
50134
Country
Italy
Facility Name
Clinical Trial Site
City
Milano
ZIP/Postal Code
20122
Country
Italy
Facility Name
Clinical Trial Site
City
Napoli
ZIP/Postal Code
80131
Country
Italy
Facility Name
Clinical Trial Site
City
Torino
ZIP/Postal Code
10126
Country
Italy
Facility Name
Clinical Trial Site
City
Vicenza
ZIP/Postal Code
36100
Country
Italy
Facility Name
Clinical Trial Site
City
Kanazawa-shi
State/Province
Ishikawa
ZIP/Postal Code
920-8641
Country
Japan
Facility Name
Clinical Trial Site
City
Suwa
State/Province
Nagano
ZIP/Postal Code
392-8510
Country
Japan
Facility Name
Clinical Trial Site
City
Suita-shi
State/Province
Osaka
ZIP/Postal Code
565-0871
Country
Japan
Facility Name
Clinical Trial Site
City
Shinagawa-Ku
State/Province
Tokyo
ZIP/Postal Code
141-8625
Country
Japan
Facility Name
Clinical Trial Site
City
Fukushima
ZIP/Postal Code
960-1295
Country
Japan
Facility Name
Clinical Trial Site
City
Daegu
ZIP/Postal Code
42415
Country
Korea, Republic of
Facility Name
Clinical Trial Site
City
Daejeon
ZIP/Postal Code
35015
Country
Korea, Republic of
Facility Name
Clinical Trial Site
City
Gyeonggi-do
ZIP/Postal Code
14584
Country
Korea, Republic of
Facility Name
Clinical Trial Site
City
Gyeonggi-do
ZIP/Postal Code
16247
Country
Korea, Republic of
Facility Name
Clinical Trial Site
City
Incheon
ZIP/Postal Code
21565
Country
Korea, Republic of
Facility Name
Clinical Trial Site
City
Seoul
ZIP/Postal Code
03080
Country
Korea, Republic of
Facility Name
Clinical Trial Site
City
Seoul
ZIP/Postal Code
03722
Country
Korea, Republic of
Facility Name
Clinical Trial Site
City
Seoul
ZIP/Postal Code
06351
Country
Korea, Republic of
Facility Name
Clinical Trial Site
City
Seoul
ZIP/Postal Code
06591
Country
Korea, Republic of
Facility Name
Clinical Trial Site
City
Maastricht
ZIP/Postal Code
6229
Country
Netherlands
Facility Name
Clinical Trial Site
City
Nijmegen
ZIP/Postal Code
6525
Country
Netherlands
Facility Name
Clinical Trial Site
City
Barcelona
ZIP/Postal Code
08036
Country
Spain
Facility Name
Clinical Trial Site
City
Madrid
ZIP/Postal Code
28040
Country
Spain
Facility Name
Clinical Trial Site
City
Majadahonda
ZIP/Postal Code
28222
Country
Spain
Facility Name
Clinical Trial Site
City
Airdrie
ZIP/Postal Code
ML6 9JS
Country
United Kingdom
Facility Name
Clinical Trial Site
City
Leeds
ZIP/Postal Code
LS9 7TF
Country
United Kingdom
Facility Name
Clinical Trial Site
City
London
ZIP/Postal Code
SE5 9RS
Country
United Kingdom
12. IPD Sharing Statement
Citations:
PubMed Identifier
30510079
Citation
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.
Results Reference
background
PubMed Identifier
31949012
Citation
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.
Results Reference
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PubMed Identifier
32869125
Citation
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.
Results Reference
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PubMed Identifier
35502600
Citation
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.
Results Reference
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PubMed Identifier
33301613
Citation
Kulasekararaj AG, Hill A, Langemeijer S, Wells R, Gonzalez Fernandez FA, Gaya A, Ojeda Gutierrez E, Piatek CI, Mitchell L, Usuki K, Bosi A, Brodsky RA, Ogawa M, Yu J, Ortiz S, Roth A, Lee JW, Peffault de Latour R. One-year outcomes from a phase 3 randomized trial of ravulizumab in adults with paroxysmal nocturnal hemoglobinuria who received prior eculizumab. Eur J Haematol. 2021 Mar;106(3):389-397. doi: 10.1111/ejh.13564. Epub 2021 Jan 3.
Results Reference
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PubMed Identifier
32449174
Citation
Peffault de Latour R, Brodsky RA, Ortiz S, Risitano AM, Jang JH, Hillmen P, Kulagin AD, Kulasekararaj AG, Rottinghaus ST, Aguzzi R, Gao X, Wells RA, Szer J. Pharmacokinetic and pharmacodynamic effects of ravulizumab and eculizumab on complement component 5 in adults with paroxysmal nocturnal haemoglobinuria: results of two phase 3 randomised, multicentre studies. Br J Haematol. 2020 Nov;191(3):476-485. doi: 10.1111/bjh.16711. Epub 2020 May 24.
Results Reference
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PubMed Identifier
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Citation
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.
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Citation
Wietz IC, Kulagin A, Nakao S, Piatek CI, Szer J, Rottinghaus ST, Volles L, Damokosh AI, Aguzzi R, Larratt L, Risitano AM. A Phase 3 study of ravulizumab (ALXN1210) versus eculizumab in adults with paroxysmal nocturnal hemoglobinuria naive to complement inhibitors: results of a subgroup analysis with patients stratified by baseline hemolysis level, transfusion history, and demographics. Blood. 2018;132 (Supplement 1):627. doi: 10.1182/blood-2018-99-110623
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Citation
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.
Results Reference
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Learn more about this trial
ALXN1210 Versus Eculizumab in Adult Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH) Currently Treated With Eculizumab
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