Ravulizumab Subcutaneous (SC) Versus Ravulizumab Intravenous (IV) in Adults With Paroxysmal Nocturnal Hemoglobinuria (PNH) Currently Treated With Eculizumab
NCT ID: NCT03748823
Last Updated: 2024-09-19
Study Results
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View full resultsBasic Information
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COMPLETED
PHASE3
139 participants
INTERVENTIONAL
2019-02-19
2023-08-31
Brief Summary
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Detailed Description
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Conditions
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Study Design
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RANDOMIZED
PARALLEL
TREATMENT
NONE
Study Groups
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Ravulizumab SC Treatment Group
In the Randomized Treatment Period, participants will receive an IV loading dose of ravulizumab on Day 1 followed by SC maintenance doses of ravulizumab administered via the ravulizumab OBDS on Day 15 and every week (qw) thereafter for a total of 10 weeks of study treatment.
In the Extension Period, ravulizumab SC will be administered via the ravulizumab OBDS from Day 71 qw through Day 1274.
Ravulizumab OBDS
The ravulizumab OBDS is a biological-device combination product consisting of a prefilled cartridge containing ravulizumab SC and an on-body injector.
Ravulizumab
Administered by IV infusion. Ravulizumab IV doses will be based on participant body weight.
Ravulizumab IV Treatment Group
In the Randomized Treatment Period, participants will receive an IV loading dose of ravulizumab on Day 1 followed by IV maintenance doses of ravulizumab on Day 15.
In the Extension Period, ravulizumab SC will be administered via the ravulizumab OBDS from Day 71 qw through Day 1274.
Ravulizumab OBDS
The ravulizumab OBDS is a biological-device combination product consisting of a prefilled cartridge containing ravulizumab SC and an on-body injector.
Ravulizumab
Administered by IV infusion. Ravulizumab IV doses will be based on participant body weight.
Interventions
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Ravulizumab OBDS
The ravulizumab OBDS is a biological-device combination product consisting of a prefilled cartridge containing ravulizumab SC and an on-body injector.
Ravulizumab
Administered by IV infusion. Ravulizumab IV doses will be based on participant body weight.
Other Intervention Names
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Eligibility Criteria
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Inclusion Criteria
* Treated with eculizumab for PNH for at least 3 months prior to Day 1
* LDH level ≤1.5 × upper limit of normal (ULN) at screening
* PNH diagnosis confirmed by documented high-sensitivity flow cytometry.
* Documented meningococcal vaccination not more than 3 years prior to, or at the time of, initiating study treatment.
* Body weight ≥40 to \<100 kilogram (kg)
* 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.
* 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).
* 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.
18 Years
ALL
No
Sponsors
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Alexion Pharmaceuticals, Inc.
INDUSTRY
Responsible Party
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Locations
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Research Site
Los Angeles, California, United States
Research Site
Liverpool, , Australia
Research Site
Parkville, , Australia
Research Site
Vienna, , Austria
Research Site
Antwerp, , Belgium
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Brussels, , Belgium
Research Site
Hasselt, , Belgium
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Leuven, , Belgium
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Botucatu, , Brazil
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Ribeirão Preto, , Brazil
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Rio de de Janeiro, , Brazil
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Salvador, , Brazil
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São Paulo, , Brazil
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São Paulo, , Brazil
Research Site
Toronto, Ontario, Canada
Research Site
Helsinki, , Finland
Research Site
Amiens, , France
Research Site
Brest, , France
Research Site
Lille, , France
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Montpellier, , France
Research Site
Nantes, , France
Research Site
Nice, , France
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Paris, , France
Research Site
Pessac, , France
Research Site
Pierre-Bénite, , France
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Poitiers, , France
Research Site
Rennes, , France
Research Site
Strasbourg, , France
Research Site
Tours, , France
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Catania, , Italy
Research Site
Milan, , Italy
Research Site
Roma, , Italy
Research Site
Rome, , Italy
Research Site
Maastricht, , Netherlands
Research Site
Moscow, , Russia
Research Site
Moscow, , Russia
Research Site
Saint Petersburg, , Russia
Research Site
Yekaterinburg, , Russia
Research Site
Badalona, , Spain
Research Site
Barcelona, , Spain
Research Site
Donostia / San Sebastian, , Spain
Research Site
Las Palmas de Gran Canaria, , Spain
Research Site
Madrid, , Spain
Research Site
Majadahonda, , Spain
Research Site
Seville, , Spain
Research Site
Uppsala, , Sweden
Research Site
Adana, , Turkey (Türkiye)
Research Site
Istanbul, , Turkey (Türkiye)
Research Site
Istanbul, , Turkey (Türkiye)
Research Site
Istanbul, , Turkey (Türkiye)
Research Site
Izmir, , Turkey (Türkiye)
Countries
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References
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Yenerel MN, Sicre de Fontbrune F, Piatek C, Sahin F, Fureder W, Ortiz S, Ogawa M, Ozol-Godfrey A, Sierra JR, Szer J. Phase 3 Study of Subcutaneous Versus Intravenous Ravulizumab in Eculizumab-Experienced Adult Patients with PNH: Primary Analysis and 1-Year Follow-Up. Adv Ther. 2023 Jan;40(1):211-232. doi: 10.1007/s12325-022-02339-3. Epub 2022 Oct 22.
Provided Documents
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Document Type: Study Protocol
Document Type: Statistical Analysis Plan
Related Links
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Other Identifiers
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2017-002370-39
Identifier Type: EUDRACT_NUMBER
Identifier Source: secondary_id
ALXN1210-PNH-303
Identifier Type: -
Identifier Source: org_study_id
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