Study of ALXN1210 in Complement Inhibitor Treatment-Naïve Adult and Adolescent Participants With Atypical Hemolytic Uremic Syndrome (aHUS)
NCT ID: NCT02949128
Last Updated: 2024-02-20
Study Results
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View full resultsBasic Information
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COMPLETED
PHASE3
58 participants
INTERVENTIONAL
2017-01-11
2023-01-24
Brief Summary
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Detailed Description
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Conditions
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Study Design
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NON_RANDOMIZED
SINGLE_GROUP
TREATMENT
NONE
Study Groups
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Ravulizumab
Participants were administered weight-based doses of ravulizumab every 8 weeks for 26 weeks in the Initial Evaluation Period.
After the Initial Evaluation Period, participants could enter an Extension Period and receive ravulizumab until the product registration or approval (in accordance with country-specific regulations) or for up to 4.5 years, whichever occurs first.
Ravulizumab
Single loading dose on Day 1, followed by regular maintenance dosing beginning on Day 15, based on weight: ≥ 40 to \< 60 kilograms (kg), 2400 milligrams (mg) loading, then 3000 mg every 8 weeks; ≥ 60 to \< 100 kg, 2700 mg loading, then 3300 mg every 8 weeks; ≥ 100 kg, 3000 mg loading, then 3600 mg every 8 weeks.
Interventions
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Ravulizumab
Single loading dose on Day 1, followed by regular maintenance dosing beginning on Day 15, based on weight: ≥ 40 to \< 60 kilograms (kg), 2400 milligrams (mg) loading, then 3000 mg every 8 weeks; ≥ 60 to \< 100 kg, 2700 mg loading, then 3300 mg every 8 weeks; ≥ 100 kg, 3000 mg loading, then 3600 mg every 8 weeks.
Other Intervention Names
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Eligibility Criteria
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Inclusion Criteria
2. Evidence of thrombotic microangiopathy, including low platelet count, hemolysis (breaking of red blood cells inside of blood vessels), and decreased kidney function.
3. Documented meningococcal vaccination not more than 3 years prior to, or at the time of, initiating study drug. Participants who received a meningococcal vaccine less than 2 weeks before initiating ravulizumab treatment must have received treatment with appropriate prophylactic antibiotics until 2 weeks after vaccination. Participants who had not been vaccinated prior to initiating ravulizumab treatment should have received prophylactic antibiotics prior to and for at least 2 weeks after meningococcal vaccination. Participants \< 18 years of age must have been vaccinated against haemophilus influenzae type b and streptococcus pneumoniae according to national and local vaccination schedule guidelines.
4. Female participants of childbearing potential and male participants with female partners of childbearing potential had to use highly effective contraception starting at screening and continuing until at least 8 months after the last dose of ravulizumab.
Exclusion Criteria
2. Shiga toxin-related hemolytic uremic syndrome.
3. Positive direct Coombs test.
4. Pregnancy or breastfeeding.
5. Identified drug exposure-related hemolytic uremic syndrome (HUS).
6. Bone marrow transplant/hematopoietic stem cell transplant within last 6 months prior to start of Screening.
7. HUS related to known genetic defects of cobalamin C metabolism.
8. Systemic sclerosis (scleroderma), systemic lupus erythematosus, or antiphospholipid antibody positivity or syndrome.
9. Chronic dialysis (defined as dialysis on a regular basis as renal replacement therapy for end-stage kidney disease).
ALL
No
Sponsors
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Alexion Pharmaceuticals, Inc.
INDUSTRY
Responsible Party
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Locations
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Clinical Trial Site
Fort Wayne, Indiana, United States
Clinical Trial Site
Fort Wayne, Indiana, United States
Clinical Trial Site
Durham, North Carolina, United States
Clinical Trial Site
Winston-Salem, North Carolina, United States
Clinical Trial Site
Columbus, Ohio, United States
Clinical Trial Site
Clayton, , Australia
Clinical Trial Site
Geelong, , Australia
Clinical Trial Site
Parkville, , Australia
Clinical Trial Site
Vienna, , Austria
Clinical Trial Site
Brussels, , Belgium
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London, , Canada
Clinical Trial Site
Bordeaux, , France
Clinical Trial Site
Clermont-Ferrand, , France
Clinical Trial Site
Lille, , France
Clinical Trial Site
Montpellier, , France
Clinical Trial Site
Nice, , France
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Paris, , France
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Aachen, , Germany
Clinical Trial Site
Essen, , Germany
Clinical Trial Site
Hanover, , Germany
Clinical Trial Site
München, , Germany
Clinical Trial Site
Tübingen, , Germany
Clinical Trial Site
Bologna, , Italy
Clinical Trial Site
Florence, , Italy
Clinical Trial Site
Saitama, , Japan
Clinical Trial Site
Tokyo, , Japan
Clinical Trial Site
Moscow, , Russia
Clinical Trial Site
Saint Petersburg, , Russia
Clinical Trial Site
Gyeonggi-do, , South Korea
Clinical Trial Site
Seoul, , South Korea
Clinical Trial Site
Barcelona, , Spain
Clinical Trial Site
Madrid, , Spain
Clinical Trial Site
Valencia, , Spain
Clinical Trial Site
Taichung, , Taiwan
Clinical Trial Site
Taipei, , Taiwan
Clinical Trial Site
Cardiff, , United Kingdom
Clinical Trial Site
London, , United Kingdom
Countries
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References
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Barbour T, Scully M, Ariceta G, Cataland S, Garlo K, Heyne N, Luque Y, Menne J, Miyakawa Y, Yoon SS, Kavanagh D; 311 Study Group Members. Long-Term Efficacy and Safety of the Long-Acting Complement C5 Inhibitor Ravulizumab for the Treatment of Atypical Hemolytic Uremic Syndrome in Adults. Kidney Int Rep. 2021 Mar 24;6(6):1603-1613. doi: 10.1016/j.ekir.2021.03.884. eCollection 2021 Jun.
Matsumoto M, Shimono A, Yokosawa J, Hirose K, Wang E, Maruyama S. Correlation between a 2-week change in platelet count and clinical outcomes after the initiation of ravulizumab treatment in adult patients with atypical hemolytic uremic syndrome: post-hoc analysis of the phase III trial. Thromb J. 2024 Oct 28;22(1):93. doi: 10.1186/s12959-024-00652-1.
Cammett TJ, Garlo K, Millman EE, Rice K, Toste CM, Faas SJ. Exploratory Prognostic Biomarkers of Complement-Mediated Thrombotic Microangiopathy (CM-TMA) in Adults with Atypical Hemolytic Uremic Syndrome (aHUS): Analysis of a Phase III Study of Ravulizumab. Mol Diagn Ther. 2023 Jan;27(1):61-74. doi: 10.1007/s40291-022-00620-3. Epub 2022 Nov 4.
Pugh D, O'Sullivan ED, Duthie FA, Masson P, Kavanagh D. Interventions for atypical haemolytic uraemic syndrome. Cochrane Database Syst Rev. 2021 Mar 23;3(3):CD012862. doi: 10.1002/14651858.CD012862.pub2.
Gackler A, Schonermarck U, Dobronravov V, La Manna G, Denker A, Liu P, Vinogradova M, Yoon SS, Praga M. Efficacy and safety of the long-acting C5 inhibitor ravulizumab in patients with atypical hemolytic uremic syndrome triggered by pregnancy: a subgroup analysis. BMC Nephrol. 2021 Jan 6;22(1):5. doi: 10.1186/s12882-020-02190-0.
Provided Documents
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Document Type: Study Protocol
Document Type: Statistical Analysis Plan
Other Identifiers
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2016-002027-29
Identifier Type: EUDRACT_NUMBER
Identifier Source: secondary_id
ALXN1210-aHUS-311
Identifier Type: -
Identifier Source: org_study_id
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