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Phase 3 Trial of Concizumab in Hemophilia with Inhibitors

by Hald Jacobsen, Sanja , Stasyshyn, Oleksandra , Nogami, Keiji , Mahlangu, Johnny , Frei-Jones, Melissa , Vilchevska, Kateryna , Zozulya, Nadezhda , Jiménez-Yuste, Victor , Castaman, Giancarlo , Goh, Ai-Sim , Haaning, Jesper , Matsushita, Tadashi , Villarreal Martinez, Laura , You, Chur Woo , Angchaisuksiri, Pantep , Skovgaard Rasmussen, Josephine , Zulfikar, Bulent , Shapiro, Amy , Abraham, Aby , Cepo, Katarina , d’Oiron, Roseline , Mathias, Mary , Tran, Huyen , Windyga, Jerzy

in Adolescent Medicine / Antibodies, Monoclonal, Humanized - adverse effects / Antibodies, Monoclonal, Humanized - therapeutic use / Bleeding / Body Weight / Clinical outcomes / Coagulation / Disease prevention / Drug therapy / FDA approval / Genetics / Genetics General / Hematology / Hemophilia / Hemophilia A - complications / Hemophilia A - drug therapy / Hemostasis / Human health and pathology / Humans / Injections, Subcutaneous / Life Sciences / Monoclonal antibodies / Oncology / Patient safety / Pediatrics / Pediatrics General / Pharmacodynamics / Pharmacokinetics / Teenagers / Thromboembolism / Thromboembolism - prevention & control / Tissue factor / Venous access

2023

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2023

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2023

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Journal Article

Phase 3 Trial of Concizumab in Hemophilia with Inhibitors

Hald Jacobsen, Sanja,

Stasyshyn, Oleksandra,

Nogami, Keiji,

Mahlangu, Johnny,

Frei-Jones, Melissa,

Vilchevska, Kateryna,

Zozulya, Nadezhda,

Jiménez-Yuste, Victor,

Castaman, Giancarlo,

Goh, Ai-Sim,

Haaning, Jesper,

Matsushita, Tadashi,

Villarreal Martinez, Laura,

You, Chur Woo,

Angchaisuksiri, Pantep,

Skovgaard Rasmussen, Josephine,

Zulfikar, Bulent,

Shapiro, Amy,

Abraham, Aby,

Cepo, Katarina,

d’Oiron, Roseline,

Mathias, Mary,

Tran, Huyen,

Windyga, Jerzy

2023

Overview

By inactivating the tissue factor pathway inhibitor, concizumab can lead to sufficient thrombin generation for clotting. In this trial, the median annualized bleeding rate was 9.8 with no prophylaxis and 0 with concizumab prophylaxis.

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Publisher

Massachusetts Medical Society

Subject

Adolescent Medicine

/ Antibodies, Monoclonal, Humanized - adverse effects

/ Antibodies, Monoclonal, Humanized - therapeutic use