Page 123 - MEMORIA ANUAL 2020

GRUPO ESPAÑOL DE ERITROPATOLOGÍA (GEE)
MEMORIA ANUAL 2020
SOCIEDAD ESPAÑOLA FUNDACIÓN ESPAÑOLA
DE HEMATOLOGÍA Y HEMOTERAPIA DE HEMATOLOGÍA Y HEMOTERAPIA
Talasemia
• AG348-C-018. A phase 3 double blind, randomized, placebo controlled, multicenter, study evalu- ating the efficacy and safety of Mitapivat in subjects with transfusion dependent alpha or beta thalas- semia (ENERGIZE-T).
• AG348-C-017. A phase 3 double blind, randomized, placebo controlled, multicenter, study evalu- ating the efficacy and safety of Mitapivat in subjects with non-transfusion dependent alpha or beta thalassemia (ENERGIZE).
Anemia hemolítica autoinmune
• A Phase 2, Multicenter, Non-Randomized, Open-Label Study of RVT-1401 (Batoclimab) for the Treatment of Patients with Warm Autoimmune Hemolytic Anemia.
• A Phase 3, Multi-Center, Randomized, Double-Blind, Placebo-Controlled, Study of Fostamatinib Disodium in the Treatment of Warm Antibody Autoimmune Hemolytic Anemia.
• MOM-M281-006: Efficacy and safety of M281 (Nipocalimab) in adults with warm autoimmune he- molytic anemia: a multicenter, randomized, double-blind, placebo-controlled, study with a long-term open-label extension.
• A Multicenter, Phase 1b, Open Label, Non-randomized, Single Dose Study Evaluating the Safety, Tolerability and Activity of BIVV020 in Adults With Cold Agglutinin Disease.
Hemoglobinuria paroxística nocturna
• ALXN1210-PNH-301: a phase 3, randomized, open-label, active-controlled study of alxn1210 versus eculizumab in complement inhibitor-naïve adult patients with paroxysmal nocturnal hemoglobinuria (PNH).
• ALXN1210-PNH-302: A Phase 3, randomized, open-label, active-controlled study of alxn1210 versus eculizumab in adult patients with paroxysmal nocturnal hemoglobinuria (PNH) currently treat- ed with eculizumab.
• ALXN1210-PNH-201: A Phase 2 open-label multiple ascending dose study to evaluate the ef- ficacy, safety, tolerability, inmunogenicity, pharmacokinetics, and pharmacodynamics of ALXN-1210 administered intravenously to patients with paroxysmal nocturnal hemoglobinuria.
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