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COMPLETED
NCT04543591
PHASE3

Ravulizumab in Thrombotic Microangiopathy After Hematopoietic Stem Cell Transplant

Sponsor: Alexion Pharmaceuticals, Inc.

View on ClinicalTrials.gov

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

Interventions

BIOLOGICAL

Ravulizumab

Weight-based doses of ravulizumab will be administered intravenously as loading dose regimen followed by maintenance dosing every 8 weeks.

OTHER

Placebo

Matching placebo

OTHER

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