A Phase I/II Study to Evaluate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Efficacy of NXT007 in Persons with Severe or Moderate Hemophilia A - WP44714

F. Hoffmann-La Roche AG0 个研究点目标入组 20 人开始时间: 2023年5月30日最近更新: 2024年8月21日

研究概览

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•Body weight >= 40 kg at screening, 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%

•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 investigator’s 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, History of severe allergic or anaphylactic reactions to monoclonal antibody therapy and to chimeric or humanized antibodies or fusion proteins

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