Page 136 - Memoria SEHH 2022

MEMORIA ANUAL 2022 SOCIEDAD ESPAÑOLA DE
HEMATOLOGÍA Y HEMOTERAPIA
GEE
FUNDACIÓN ESPAÑOLA DE HEMATOLOGÍA Y HEMOTERAPIA
Ensayos clínicos
Abiertos en 2022
• AG348-C-022 . A phase 3, multicenter, randomized, double-blind, placebo-controlled study to evaluate the efficacy and safety of mitapivat in pediatric subjects with pyruvate kinase deficiency who are regularly transfused, followed by a 5-year open-label-extension period . Eduardo José Salido Fiérrez (Hospital Universitario Virgen de la Arrixaca, Murcia) .
• A study to assess the safety, tolerability, pharmacokinetics, and pharmacodynam- ics of AG-946 in healthy volunteers and in participants with sickle cell disease: Agios (2022) . Fase 1 . Marta Morado (Hospital Universitario La Paz, Madrid) .
• A multicentre trial evaluating the efficacy and safety of oral decitabine tetrahydrouridine (NDec) in patients with sickle cell disease ASCENT NN7533-4470, NovoNordisk (2022) . Fase 2-3 . Marta Morado (Hospital Universitario La Paz, Madrid) .
• Efficacy, safety and pharmacokinetics of rilzabrutinib in patients with warm autoim- mune hemolytic anemia (wAIHA) . Fase 2 . Rafael del Orbe Barreto (Hospital Universitario de Cruces, Barakaldo) .
• A study of TAK-755 in participants with congenital thrombotic thrombocytopenic purpura . Fase 3 . Rafael del Orbe Barreto (Hospital Universitario de Cruces, Barakaldo) .
Cerrados en 2022
• A study of BAX 930 in children, teenagers, and adults born with thrombotic throm- bocytopenic purpura (TTP) . Fase 2 . Rafael del Orbe Barreto (Hospital Universitario de Cruces, Barakaldo) .
En marcha en 2022
• AG348-C-011 . An open-label, multicenter, extension study of AG-348 in adult subjects with pyruvate kinase deficiency previously enrolled in AG-348 studies . Fase 3 . Eduardo José Salido Fiérrez (Hospital Universitario Virgen de la Arrixaca, Murcia), Marta Morado Arias (Hospital Universitario La Paz, Madrid) .
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