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

Primary Purpose

Paroxysmal Nocturnal Hemoglobinuria (PNH)

Status
Completed
Phase
Phase 3
Locations
International
Study Type
Interventional
Intervention
Ravulizumab
Eculizumab
Sponsored by
Alexion
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Paroxysmal Nocturnal Hemoglobinuria (PNH)

Eligibility Criteria

12 Years - undefined (Child, Adult, Older Adult)All SexesDoes not accept healthy volunteers

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 (hemoglobin <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 upper limit of normal 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 childbearing potential must use highly effective contraception starting at screening and continuing until at least 8 months after the last dose of ravulizumab.
  7. Willing and able to give written informed consent 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 investigational 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.

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

Participants received weight-based doses of ravulizumab ranging from 2400 to 3000 milligram (mg) on Day 1. Thereafter, weight-based doses of ravulizumab ranging from 3000 to 3600 mg were administered on Day 15 and every 8 weeks thereafter for 26 weeks. After completion of the Primary Evaluation Period, all participants had the opportunity to enter the Extension Period, wherein participants will receive weight-based doses of ravulizumab for up to 5 years.

Participants received 600 mg of eculizumab on Days 1, 8, 15, and 22, followed by 900 mg of eculizumab on Day 29 and every 2 weeks thereafter for 26 weeks. After completion of the Primary Evaluation Period, all participants had the opportunity to enter the Extension Period, wherein participants will receive weight-based doses of ravulizumab for up to 5 years.

Outcomes

Primary Outcome Measures

Proportion Of Participants With Normalization Of Lactate Dehydrogenase (LDH) Levels
LDH is an indicator of intravascular hemolysis that occurs in patients 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.
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.

Secondary Outcome Measures

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.
Percent Change From Baseline In Lactate Dehydrogenase (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.
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.
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.

Full Information

First Posted
October 25, 2016
Last Updated
April 13, 2023
Sponsor
Alexion
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1. Study Identification

Unique Protocol Identification Number
NCT02946463
Brief Title
ALXN1210 (Ravulizumab) Versus Eculizumab in Complement Inhibitor Treatment-Naïve Adult Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH)
Official Title
A Phase 3, Randomized, Open-Label, Active-Controlled Study of ALXN1210 Versus Eculizumab in Complement Inhibitor-Naïve Adult Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH)
Study Type
Interventional

2. Study Status

Record Verification Date
April 2023
Overall Recruitment Status
Completed
Study Start Date
December 20, 2016 (Actual)
Primary Completion Date
January 25, 2018 (Actual)
Study Completion Date
February 28, 2023 (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
No

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 had never been treated with a complement inhibitor (treatment-naïve).
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. This study is ongoing. The data presented is for the Primary Evaluation Period. The results for the Extension Period will be reported after study completion.

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
270 (Actual)

8. Arms, Groups, and Interventions

Arm Title
Ravulizumab
Arm Type
Experimental
Arm Description
Participants received weight-based doses of ravulizumab ranging from 2400 to 3000 milligram (mg) on Day 1. Thereafter, weight-based doses of ravulizumab ranging from 3000 to 3600 mg were administered on Day 15 and every 8 weeks thereafter for 26 weeks. After completion of the Primary Evaluation Period, all participants had the opportunity to enter the Extension Period, wherein participants will receive weight-based doses of ravulizumab for up to 5 years.
Arm Title
Eculizumab
Arm Type
Active Comparator
Arm Description
Participants received 600 mg of eculizumab on Days 1, 8, 15, and 22, followed by 900 mg of eculizumab on Day 29 and every 2 weeks thereafter for 26 weeks. After completion of the Primary Evaluation Period, all participants had the opportunity to enter the Extension Period, wherein participants will receive weight-based doses of ravulizumab for up to 5 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 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.
Intervention Type
Biological
Intervention Name(s)
Eculizumab
Intervention Description
All treatments were given as IV infusions. Participants were administered induction doses of 600 mg followed by maintenance doses of 900 mg.
Primary Outcome Measure Information:
Title
Proportion Of Participants With Normalization Of Lactate Dehydrogenase (LDH) Levels
Description
LDH is an indicator of intravascular hemolysis that occurs in patients 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
Title
Percentage Of Participants Who Achieved Transfusion Avoidance (TA)
Description
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 Outcome Measure Information:
Title
Percentage Of Participants With Breakthrough Hemolysis (BTH)
Description
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
Title
Percent Change From Baseline In Lactate Dehydrogenase (LDH) Levels
Description
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
Title
Change From Baseline In Quality Of Life As Assessed By The Functional Assessment Of Chronic Illness Therapy (FACIT)-Fatigue
Description
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
Title
Percentage Of Participants With Stabilized Hemoglobin Levels
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

10. Eligibility

Sex
All
Minimum Age & Unit of Time
12 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Criteria For Patient Cohort Originally Enrolled in ALXN1210-PNH-301 Study: Inclusion Criteria: Male or female ≥18 years of age. PNH diagnosis confirmed by documented by high-sensitivity flow cytometry. 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 (hemoglobin <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. Lactate dehydrogenase (LDH) level ≥1.5 times the upper limit of normal at screening. 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: Treatment with a complement inhibitor at any time. History of bone marrow transplantation. Body weight <40 kg. Females 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 drug on Day 1 in this study or within 5 half-lives of that investigational product, whichever is greater. 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 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: All participants regardless of age, who are currently receiving ALXN1210 IV in an ongoing ALXN1210 study in patients with PNH 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 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.
Facility Information:
Facility Name
Clinical Trial Site
City
Los Angeles
State/Province
California
ZIP/Postal Code
90033
Country
United States
Facility Name
Clinical Trial Site
City
Los Angeles
State/Province
California
ZIP/Postal Code
90048
Country
United States
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Clinical Trial Site
City
Fort Worth
State/Province
Texas
ZIP/Postal Code
76104
Country
United States
Facility Name
Clinical Trial Site
City
Buenos Aires
ZIP/Postal Code
C1015ABO
Country
Argentina
Facility Name
Clinical Trial Site
City
Buenos Aires
ZIP/Postal Code
C1425AUM
Country
Argentina
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City
Córdoba
Country
Argentina
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Clinical Trial Site
City
Perth
Country
Australia
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City
Linz
Country
Austria
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City
Vienna
Country
Austria
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Clinical Trial Site
City
Hasselt
Country
Belgium
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City
Leuven
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Belgium
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City
Belém
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Brazil
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City
Rio de Janeiro
Country
Brazil
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Clinical Trial Site
City
Salvador
Country
Brazil
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Clinical Trial Site
City
São Paulo
ZIP/Postal Code
05403-000
Country
Brazil
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Clinical Trial Site
City
São Paulo
ZIP/Postal Code
08270-070
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Brazil
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City
Edmonton
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Canada
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Toronto
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Canada
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Plzen
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Czechia
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Praha
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Czechia
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Tallinn
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Estonia
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Poitiers
State/Province
Vienne
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France
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Limoges
Country
France
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Montpellier
Country
France
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Paris
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France
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Pierre-Bénite
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France
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Rennes
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France
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Essen
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Germany
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Ulm
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Germany
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Ascoli Piceno
Country
Italy
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City
Firenze
Country
Italy
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City
Milano
Country
Italy
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City
Napoli
Country
Italy
Facility Name
Clinical Trial Site
City
Vicenza
Country
Italy
Facility Name
Clinical Trial Site
City
Bunkyō-Ku
ZIP/Postal Code
113-8431
Country
Japan
Facility Name
Clinical Trial Site
City
Bunkyō-Ku
Country
Japan
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City
Fukuoka
Country
Japan
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City
Fukushima
Country
Japan
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City
Hamamatsu-shi
Country
Japan
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City
Kanazawa-shi
Country
Japan
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City
Koshigaya-shi
Country
Japan
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City
Kumamoto
Country
Japan
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City
Nishinomiya-shi
Country
Japan
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City
Ogaki-shi
Country
Japan
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City
Okayama-city
Country
Japan
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City
Okayama-shi
Country
Japan
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City
Osakasayama-shi
Country
Japan
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City
Sapporo
Country
Japan
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City
Shimotsuke-shi
Country
Japan
Facility Name
Clinical Trial Site
City
Shinagawa-Ku
Country
Japan
Facility Name
Clinical Trial Site
City
Shinjuku-Ku
ZIP/Postal Code
160-0023
Country
Japan
Facility Name
Clinical Trial Site
City
Shinjuku-Ku
ZIP/Postal Code
160-8582
Country
Japan
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City
Suita-shi
Country
Japan
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City
Tokorozawa-shi
Country
Japan
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Toyoake-shi
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Japan
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City
Tsukuba
Country
Japan
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City
Wakayama-shi
Country
Japan
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Clinical Trial Site
City
Yokohama-City
ZIP/Postal Code
227-8501
Country
Japan
Facility Name
Clinical Trial Site
City
Yokohama-City
ZIP/Postal Code
236-0004
Country
Japan
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City
Anyang-si
Country
Korea, Republic of
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Clinical Trial Site
City
Busan
Country
Korea, Republic of
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Clinical Trial Site
City
Daegu
Country
Korea, Republic of
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Clinical Trial Site
City
Incheon
Country
Korea, Republic of
Facility Name
Clinical Trial Site
City
Jeonju
Country
Korea, Republic of
Facility Name
Clinical Trial Site
City
Jinju-si
Country
Korea, Republic of
Facility Name
Clinical Trial Site
City
Seoul
ZIP/Postal Code
02841
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
04401
Country
Korea, Republic of
Facility Name
Clinical Trial Site
City
Seoul
ZIP/Postal Code
05505
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
Seoul
ZIP/Postal Code
07985
Country
Korea, Republic of
Facility Name
Clinical Trial Site
City
Seoul
ZIP/Postal Code
08308
Country
Korea, Republic of
Facility Name
Clinical Trial Site
City
Suwon-si
ZIP/Postal Code
16247
Country
Korea, Republic of
Facility Name
Clinical Trial Site
City
Suwon-si
ZIP/Postal Code
16499
Country
Korea, Republic of
Facility Name
Clinical Trial Site
City
Ulsan
Country
Korea, Republic of
Facility Name
Clinical Trial Site
City
Kota Bharu
State/Province
Kelantan
Country
Malaysia
Facility Name
Clinical Trial Site
City
Miri
State/Province
Sarawak
Country
Malaysia
Facility Name
Clinical Trial Site
City
Sibu
State/Province
Sarawak
Country
Malaysia
Facility Name
Clinical Trial Site
City
Ampang
Country
Malaysia
Facility Name
Clinical Trial Site
City
Johor Bahru
Country
Malaysia
Facility Name
Clinical Trial Site
City
Kota Bahru
ZIP/Postal Code
16150
Country
Malaysia
Facility Name
Clinical Trial Site
City
Kota Bharu
ZIP/Postal Code
15586
Country
Malaysia
Facility Name
Clinical Trial Site
City
Kota Kinabalu
Country
Malaysia
Facility Name
Clinical Trial Site
City
Kubang Kerian
Country
Malaysia
Facility Name
Clinical Trial Site
City
Kuching
Country
Malaysia
Facility Name
Clinical Trial Site
City
Pulau Pinang
Country
Malaysia
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Clinical Trial Site
City
Monterrey
Country
Mexico
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Clinical Trial Site
City
Gdańsk
Country
Poland
Facility Name
Clinical Trial Site
City
Warsaw
Country
Poland
Facility Name
Clinical Trial Site
City
Arkhangel'sk
Country
Russian Federation
Facility Name
Clinical Trial Site
City
Barnaul
Country
Russian Federation
Facility Name
Clinical Trial Site
City
Bryansk
Country
Russian Federation
Facility Name
Clinical Trial Site
City
Irkutsk
Country
Russian Federation
Facility Name
Clinical Trial Site
City
Kaluga
Country
Russian Federation
Facility Name
Clinical Trial Site
City
Kirov
Country
Russian Federation
Facility Name
Clinical Trial Site
City
Krasnodar
ZIP/Postal Code
350007
Country
Russian Federation
Facility Name
Clinical Trial Site
City
Krasnoyarsk
ZIP/Postal Code
660003
Country
Russian Federation
Facility Name
Clinical Trial Site
City
Krasnoyarsk
ZIP/Postal Code
660022
Country
Russian Federation
Facility Name
Clinical Trial Site
City
Moscow
ZIP/Postal Code
125167
Country
Russian Federation
Facility Name
Clinical Trial Site
City
Moscow
ZIP/Postal Code
125284
Country
Russian Federation
Facility Name
Clinical Trial Site
City
Murmansk
Country
Russian Federation
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Clinical Trial Site
City
Nizhny Novgorod
Country
Russian Federation
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Clinical Trial Site
City
Novosibirsk
Country
Russian Federation
Facility Name
Clinical Trial Site
City
Petrozavodsk
Country
Russian Federation
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Clinical Trial Site
City
Rostov-na-Donu
Country
Russian Federation
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Clinical Trial Site
City
Saint Petersburg
Country
Russian Federation
Facility Name
Clinical Trial Site
City
Saratov
Country
Russian Federation
Facility Name
Clinical Trial Site
City
Ufa
Country
Russian Federation
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Clinical Trial Site
City
Singapore
Country
Singapore
Facility Name
Clinical Trial Site
City
Barcelona
Country
Spain
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Clinical Trial Site
City
Madrid
Country
Spain
Facility Name
Clinical Trial Site
City
Majadahonda
Country
Spain
Facility Name
Clinical Trial Site
City
Uppsala
Country
Sweden
Facility Name
Clinical Trial Site
City
Chang-hua
Country
Taiwan
Facility Name
Clinical Trial Site
City
Hualien City
Country
Taiwan
Facility Name
Clinical Trial Site
City
Kaohsiung
Country
Taiwan
Facility Name
Clinical Trial Site
City
Taichung
Country
Taiwan
Facility Name
Clinical Trial Site
City
Tainan
Country
Taiwan
Facility Name
Clinical Trial Site
City
Taipei
Country
Taiwan
Facility Name
Clinical Trial Site
City
Bangkok
Country
Thailand
Facility Name
Clinical Trial Site
City
Hat Yai
Country
Thailand
Facility Name
Clinical Trial Site
City
Pathum Wan
Country
Thailand
Facility Name
Clinical Trial Site
City
Eskişehir
Country
Turkey
Facility Name
Clinical Trial Site
City
Leeds
Country
United Kingdom
Facility Name
Clinical Trial Site
City
London
Country
United Kingdom

12. IPD Sharing Statement

Citations:
PubMed Identifier
30510080
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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.
Results Reference
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PubMed Identifier
34526067
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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PubMed Identifier
33178408
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.
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
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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Learn more about this trial

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

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