Ravulizumab in Thrombotic Microangiopathy After Hematopoietic Stem Cell Transplant
NCT ID: NCT04543591
Last Updated: 2025-11-12
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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ACTIVE_NOT_RECRUITING
PHASE3
148 participants
INTERVENTIONAL
2020-12-10
2026-03-20
Brief Summary
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Detailed Description
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Conditions
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Keywords
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Study Design
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RANDOMIZED
PARALLEL
TREATMENT
QUADRUPLE
Study Groups
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Ravulizumab
In Stage 1, all participants will receive open-label ravulizumab plus Best Supportive Care (BSC).
In Stage 2, participants will receive blinded ravulizumab plus Best Supportive Care (BSC).
Ravulizumab
Weight-based doses of ravulizumab will be administered intravenously as loading dose regimen followed by maintenance dosing every 8 weeks.
Best supportive care
Participants will receive medications, therapies, and interventions per standard hospital treatment protocols (unless specifically prohibited by the protocol).
Placebo
In Stage 2, participants randomized to the placebo arm will receive matching placebo plus BSC.
Placebo
Matching placebo
Best supportive care
Participants will receive medications, therapies, and interventions per standard hospital treatment protocols (unless specifically prohibited by the protocol).
Interventions
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Ravulizumab
Weight-based doses of ravulizumab will be administered intravenously as loading dose regimen followed by maintenance dosing every 8 weeks.
Placebo
Matching placebo
Best supportive care
Participants will receive medications, therapies, and interventions per standard hospital treatment protocols (unless specifically prohibited by the protocol).
Other Intervention Names
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Eligibility Criteria
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Inclusion Criteria
2. Received HSCT within the past 12 months.
3. Diagnosis of TMA that persists for at least 72 hours after initial management of any triggering agent/condition.
4. A TMA diagnosis based on meeting the laboratory-based criteria during the Screening Period and/or ≤14 days prior to the Screening Period.
5. Body weight ≥ 30 kilograms at Screening or ≤7 days prior to the start of the Screening Period (date of consent).
6. Female participants of childbearing potential and male participants with female partners of childbearing potential must use highly effective contraception.
7. Participants must be vaccinated against meningococcal infections if clinically feasible. Participants who cannot receive meningococcal vaccine should receive antibiotic prophylaxis. Participants \<18 years of age must be re-vaccinated against Haemophilus influenzae type b (Hib) and Streptococcus pneumoniae if clinically feasible.
8. Participants or their legally authorized representative must be capable of giving signed informed consent or assent.
Exclusion Criteria
2. Known Shiga toxin-related hemolytic uremic syndrome as demonstrated by positive test.
3. Positive direct Coombs test indicative of a clinically significant immune-mediated hemolysis not due to TMA.
4. Clinical diagnosis of disseminated intravascular coagulation (DIC).
5. Known bone marrow/graft failure for the current HSCT.
6. Diagnosis of veno-occlusive disease which is unresolved at the time of Screening.
7. Human immunodeficiency virus (HIV) infection.
8. Unresolved meningococcal disease.
9. Presence of sepsis requiring vasopressor support.
10. Pregnancy or breastfeeding.
11. Hypersensitivity to murine proteins or to one of the excipients of ravulizumab.
12. Any ongoing or history of medical or psychological conditions unrelated to HSCT-TMA that could increase the risk to the participant or confound the outcome of the study.
13. Respiratory failure requiring mechanical ventilation.
14. Acute and/or chronic heart failure with an ejection fraction ≤ 40%.
15. Previously or currently treated with a complement inhibitor.
16. Participation in an interventional treatment study of any therapy for TMA.
12 Years
100 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
Tampa, Florida, United States
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Grosse Pointe Farms, Michigan, United States
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Durham, North Carolina, United States
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Pittsburgh, Pennsylvania, United States
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Seattle, Washington, United States
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Parkville, , Australia
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Bruges, , Belgium
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Brussels, , Belgium
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Chênée, , Belgium
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Yvoir, , Belgium
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Cerqueira César, , Brazil
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Florianópolis, , Brazil
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Jaú, , Brazil
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Porto Alegre, , Brazil
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Porto Alegre, , Brazil
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Rio de Janeiro, , Brazil
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São José do Rio Preto, , Brazil
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São Paulo, , Brazil
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Calgary, Alberta, Canada
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Shanghai, , China
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Suzhou, , China
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Tianjin, , China
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Angers, , France
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La Tronche, , France
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Nice, , France
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Hamburg, , Germany
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Ulm, , Germany
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Athens, , Greece
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Pátrai, , Greece
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Thessaloniki, , Greece
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Halfa, , Israel
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Ramat Gan, , Israel
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Roma, , Italy
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Udine, , Italy
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Akita, , Japan
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Anjo, , Japan
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Chiba, , Japan
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Fukushima, , Japan
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Isehara-shi, , Japan
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Kurashiki-shi, , Japan
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Minatoku, , Japan
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Okayama, , Japan
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Osaka, , Japan
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Osakasayama-shi, , Japan
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Sapporo, , Japan
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Suita-shi, , Japan
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Tsukuba, , Japan
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Wakayama, , Japan
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Groningen, , Netherlands
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Goyang-si, , South Korea
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Seoul, , South Korea
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Seoul, , South Korea
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Seoul, , South Korea
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Barcelona, , Spain
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Granada, , Spain
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L'Hospitalet de Llobregat, , Spain
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Madrid, , Spain
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Madrid, , Spain
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Madrid, , Spain
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Madrid, , Spain
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Málaga, , Spain
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Pamplona, , Spain
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Salamanca, , Spain
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Seville, , Spain
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Huddinge, , Sweden
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London, , United Kingdom
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Nottingham, , United Kingdom
Countries
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Other Identifiers
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ALXN1210-TMA-313
Identifier Type: -
Identifier Source: org_study_id