Page 122 - MEMORIA ANUAL 2020

GRUPO ESPAÑOL DE ERITROPATOLOGÍA (GEE)
MEMORIA ANUAL 2020 SOCIEDAD ESPAÑOLA
DE HEMATOLOGÍA Y HEMOTERAPIA
FUNDACIÓN ESPAÑOLA
DE HEMATOLOGÍA Y HEMOTERAPIA
Ensayos clínicos
Anemia falciforme
• A Phase III, Multicenter, Randomized, Double-Blind Study To Assess Efficacy And Safety Of Two Doses Of Crizanlizumab Versus Placebo, With Or Without Hydroxyurea/Hydroxycarbamide Therapy, In Adolescent And Adult Sickle Cell Disease Patients With Vaso-Occlusive Crises (STAND) (>12a).
• A Phase II, multicenter, randomized, open label two arm study comparing the effect of crizanli- zumab + standard of care to standard of care alone on renal function in sickle cell disease patients ≥ 16 years with chronic kidney disease due to sickle cell nephropathy (STEADFAST).
• PRAISE: An adaptive, randomized, placebo controlled, double-blind, multicenter study of oral FT- 4202, a Pyruvate Kinase activator in patients with sickle cell disease.
• AG946-C-001: A Phase 1 Study to Assess the Safety, Tolerability, Pharmacokinetics, and Pharma- codynamics of AG-946 in Healthy Volunteers and in Subjects with Sickle Cell Disease.
• BO42452: A Phase Ib Randomized, Placebo-Controlled Study Evaluating The Safety, Pharmaco- kinetics, Pharmacodynamics, And Efficacy Of Crovalimab For The Management Of Acute Uncompli- cated Vaso-Oclusive Episodes (VOE) In Patients With Sickle Cell Disease (SCD).
• BO42451: A Randomized Double-Blind Phase 2a Study Evaluating The Efficacy, Safety, Pharma- cokinetics, And Pharmacodynamics Of Crovalimab As Adjunct Treatment In Prevention Of Acute Vaso-Occlusive Episodes (VOE) In Sickle Cell Disease (SCD).
Anemia hemolítica por déficit de piruvato cinasa
• 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.
• Pyruvate Kinase Deficiency global longitudinal registry (AG348-C-008 OBS DPK).
• Terapia génica para la deficiencia de piruvato quinasa (PKD): ensayo clínico de fase I para evaluar la seguridad de la infusión de células CD34 + autólogas transducidas con un vector lentiviral que contiene el gen de piruvato quinasa de glóbulos rojos (CoRPK) con optimización de codones en sujetos adultos y pediátricos con PKD.
122