A Global, Open-label, Adaptive Design Study to Investigate the Efficacy and Safety of SerpinPC in Subjects With Severe Hemophilia A or Moderately Severe to Severe Hemophilia B (PRESent-2)
ESTADO CLÍNICO
Reclutando
FASE/S DEL ENSAYO
Fase II
PATOLOGÍA
Coagulopatías hemorrágicas, Leucemia mieloide aguda, --> Otros, Terapia celular y T-CARs
CENTRO INVESTIGADOR
H.U. La Paz
INVESTIGADOR PRINCIPAL
María Teresa Álvarez Román
FECHA DE APERTURA
Julio, 2023
FECHA DE CIERRE
,
CRITERIOS DE INCLUSIÓN
Male subjects 12 and 65 years of age with severe HemA, with or without inhibitors, or moderately severe to severe HemB, without high titer inhibitor (high titer inhibitor defined as 5 Bethesda Units [BU]/mL) and not requiring current treatment with bypass agents.
Subject is currently included in a prophylaxis program OR subject is undergoing an on-demand treatment regimen; if the latter, must have had 6 documented acute bleeding episodes that required treatment during the 6 months before Screening
At least 12 (Part 1) or 24 (Part 2) weeks of prospective documentation of bleeding episodes in the AP-0105 noninterventional study before dosing, or willing to complete the observation period in AP-0102
No bleeding in the 7 days before Baseline (observation period can be extended if actively bleeding)
Adequate hematologic, hepatic, and renal function, and D-dimer of 750 g/L
Subject is currently included in a prophylaxis program OR subject is undergoing an on-demand treatment regimen; if the latter, must have had 6 documented acute bleeding episodes that required treatment during the 6 months before Screening
At least 12 (Part 1) or 24 (Part 2) weeks of prospective documentation of bleeding episodes in the AP-0105 noninterventional study before dosing, or willing to complete the observation period in AP-0102
No bleeding in the 7 days before Baseline (observation period can be extended if actively bleeding)
Adequate hematologic, hepatic, and renal function, and D-dimer of 750 g/L
CRITERIOS DE EXCLUSIÓN
Known severe thrombophilia, previous deep vein thrombosis (excluding line-associated thrombosis), pulmonary embolism, myocardial infarction, stroke, uncontrolled hypertension (>160/100 mmHg) or has active cancer and/or requires therapy for cancer, except for basal cell carcinoma
Subject with previous factor VIII or factor IX inhibitor who responded to immune tolerance induction and remains on prophylactic factor concentrate
Body weight < 30 kg OR >150 kg OR body mass index >40 kg/m2
Use of emicizumab in the 24 weeks before Baseline (Day 0) / Ongoing, or planned treatment with gene therapy for hemophilia / Current or planned treatment with anticoagulant or antiplatelet drugs
Subject with previous factor VIII or factor IX inhibitor who responded to immune tolerance induction and remains on prophylactic factor concentrate
Body weight < 30 kg OR >150 kg OR body mass index >40 kg/m2
Use of emicizumab in the 24 weeks before Baseline (Day 0) / Ongoing, or planned treatment with gene therapy for hemophilia / Current or planned treatment with anticoagulant or antiplatelet drugs
DESCRIPCIÓN
To evaluate the efficacy and safety of prophylactic SerpinPC administered subcutaneously (SC) in subjects with moderately severe to severe hemophilia B (HemB)
PALABRAS CLAVE
check-point inhibitor, complement inhibitor, hemophilia, ph + acute lymphoblastic leukemia
