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Ravulizumab in Thrombotic Microangiopathy After Hematopoietic Stem Cell Transplant
Sponsor: Alexion Pharmaceuticals, Inc.
Summary
This study will evaluate the efficacy, safety, pharmacokinetics, and pharmacodynamics of ravulizumab in adult and adolescent participants with hematopoietic stem cell transplant-associated thrombotic microangiopathy (HSCT-TMA). In Stage 1, an open-label, single-arm period, the dosing regimen will be confirmed. In Stage 2, participants will be randomized to receive either blinded ravulizumab plus best supportive care or matching placebo plus best supportive care. The treatment period is 26 weeks (open-label for Stage 1, and randomized, double-blind, and placebo-controlled for Stage 2) followed by a 26-week follow-up period.
Official title: A Phase 3, Randomized, Double-Blind, Placebo-Controlled, Multicenter Study of Ravulizumab in Adult and Adolescent Participants Who Have Thrombotic Microangiopathy (TMA) After Hematopoietic Stem Cell Transplant (HSCT)
Key Details
Gender
All
Age Range
12 Years - 100 Years
Study Type
INTERVENTIONAL
Enrollment
148
Start Date
2020-12-10
Completion Date
2026-03-20
Last Updated
2026-06-18
Healthy Volunteers
No
Conditions
Interventions
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).
Locations (66)
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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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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