A Phase I/II Study to Evaluate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Efficacy of NXT007 in Persons with Severe or Moderate Hemophilia A
ESTADO CLÍNICO
Reclutando
FASE/S DEL ENSAYO
Fase I-II
PATOLOGÍA
Coagulopatías hemorrágicas
CENTRO INVESTIGADOR
H.U. La Paz
INVESTIGADOR PRINCIPAL
Victor Jiménez
FECHA DE APERTURA
Septiembre, 2023
FECHA DE CIERRE
,
CRITERIOS DE INCLUSIÓN
Male (based on sex at birth)
Diagnosis of severe (FVIII:C <1 IU/dL) or moderate [FVIII coagulant activity (FVIII:C) between >= 1 IU/dL and <= 5 IU/dL)] congenital hemophilia A (HA) with or without inhibitors against FVIII
Participants with FVIII inhibitors: participants using recombinant activated factor VII (rFVIIa) or willing to switch to rFVIIa as primary bypassing agent for the treatment of breakthrough bleeds, trauma, or procedures
Historic local FVIII inhibitor test results being available during screening to confirm any previous inhibitor history and current status
Participants who previously successfully completed immune tolerance induction (ITI) must have done so at least 5 years before screening and must have no evidence of inhibitor recurrence (permanent or temporary) since. FVIII tolerance defined as < 0.6 bethesda unit (BU)/mL (< 1.0 BU/mL only for laboratories with an historical sensitivity cutoff for inhibitor detection of 1.0 BU/mL) and in vivo recovery (IVR) > 66%
Diagnosis of severe (FVIII:C <1 IU/dL) or moderate [FVIII coagulant activity (FVIII:C) between >= 1 IU/dL and <= 5 IU/dL)] congenital hemophilia A (HA) with or without inhibitors against FVIII
Participants with FVIII inhibitors: participants using recombinant activated factor VII (rFVIIa) or willing to switch to rFVIIa as primary bypassing agent for the treatment of breakthrough bleeds, trauma, or procedures
Historic local FVIII inhibitor test results being available during screening to confirm any previous inhibitor history and current status
Participants who previously successfully completed immune tolerance induction (ITI) must have done so at least 5 years before screening and must have no evidence of inhibitor recurrence (permanent or temporary) since. FVIII tolerance defined as < 0.6 bethesda unit (BU)/mL (< 1.0 BU/mL only for laboratories with an historical sensitivity cutoff for inhibitor detection of 1.0 BU/mL) and in vivo recovery (IVR) > 66%
CRITERIOS DE EXCLUSIÓN
Inherited or acquired bleeding disorders other than congenital HA
Protein C activity, protein S free antigen, or anti-thrombin III activity levels below the lower limit of the reference range at screening
At high risk for thrombotic microangiopathy (TMA), including past personal or family history of TMA, in the investigators judgment
Previous or current treatment for thromboembolic disease (with the exception of previous catheter-associated thrombosis for which anti-thrombotic treatment is not currently ongoing) or signs of thromboembolic disease
Personal history of ischemic heart disease, cerebrovascular disease, or diabetes mellitus (for participants =12 and <60 years)
History of severe allergic or anaphylactic reactions to monoclonal antibody therapy and to chimeric or humanized antibodies or fusion proteins
Protein C activity, protein S free antigen, or anti-thrombin III activity levels below the lower limit of the reference range at screening
At high risk for thrombotic microangiopathy (TMA), including past personal or family history of TMA, in the investigators judgment
Previous or current treatment for thromboembolic disease (with the exception of previous catheter-associated thrombosis for which anti-thrombotic treatment is not currently ongoing) or signs of thromboembolic disease
Personal history of ischemic heart disease, cerebrovascular disease, or diabetes mellitus (for participants =12 and <60 years)
History of severe allergic or anaphylactic reactions to monoclonal antibody therapy and to chimeric or humanized antibodies or fusion proteins
DESCRIPCIÓN
To evaluate the safety of multiple doses of NXT007
PALABRAS CLAVE
hemophilia
