a study to investigate the efficacy and safety of lusutrombopag (s-888711) tablets administered to adults with immune thrombocytopenia (itp)

Conditions: Immune Thrombocytopenia (ITP)

Summary: the primary objective of this study was to assess the efficacy of 3 dose levels of lusutrombopag (0.5 mg, 0.75 mg, and 1.0 mg) and placebo on platelet count.


Eligibility criteria:
inclusion criteria: * a signed and dated written informed consent * males and females ≥ 18 years of age * all subjects must agree to use barrier contraception * diagnosis of itp * subjects \> 60 years must have had a diagnostic bone marrow aspiration * relapsed persistent or chronic itp status, with or without prior splenectomy (exception: in hungary only splenectomized subjects will be enrolled), after having failed at least 1 prior itp therapy (excluding tpo agonists) and have a platelet count \< 30,000/μl if not taking medications or \< 50,000/μl despite concomitant steroids or other itp therapies, such as danazol or immunosuppressive drugs * subjects receiving steroid therapy must be on a stable dose * prothrombin time (pt) and activated partial thromboplastin time (aptt) within 20% of the upper limit of normal (uln) * subjects receiving stable dosages of cyclosporine a, mycophenolate mofetil, azathioprine, or danazol are allowed. the dosages of all these medications must be stable for at least 4 weeks prior to visit 1 (day 1) exclusion criteria: * history of clinically important hemorrhagic clotting disorder * females who are pregnant, lactating, or taking oral contraceptives * history of alcohol/drug abuse or dependence within 1 year * use of the following drugs or treatment prior to visit 1 (day 1): * within 12 weeks - alemtuzumab, multi-drug systemic chemotherapy, stem cell therapy; * within 8 weeks - rituximab * within 2 weeks - platelet transfusions or plasmapheresis treatment * within 4 weeks - use of anti-platelet or anti-coagulant drugs * within 1 week - rho(d) immune globulin or intravenous immunoglobulin * history of clinically significant cardiovascular or thromboembolic disease within 26 weeks prior to screening * splenectomy within 4 weeks prior to screening * clinically significant laboratory abnormalities * hemoglobin \< 10.0 g/dl for men or women, not clearly related to itp * absolute neutrophil count \< 1000/mm\^3 * abnormal peripheral blood smear * total bilirubin \> 1.5 x upper limit of normal * alanine aminotransferase (alt) \> 1.5 x upper limit of normal * aspartate aminotransferase (ast) \> 1.5 x upper limit of normal * creatinine \> 1.5 x upper limit of normal * human immunodeficiency virus (hiv) positive * hepatitis a immunoglobulin m antibody (igm hav) positive, hepatitis b surface antigen (hbsag) or hepatitis c antibody (hcv) positive * thyroid stimulating hormone (tsh) \> 1.5 x upper limit of normal * free thyroxine (t4) \> 1.5 x upper limit of normal * exposure to previous thrombopoietin (tpo) mimetics/agonists (e.g., eltrombopag,romiplostim, e5501 \[akr-501\] or lgd-4665) within 4 weeks prior to screening * subjects unresponsive to previous tpo mimetics/agonists (e.g., eltrombopag, romiplostim, e5501 \[akr-501\] or lgd-4665) * exposure to an investigative medication within the past 30 days

Keywords: blood platelet disorders immune thrombocytopenia (itp) low platelet count thrombocytopaenia s-888711 splenectomy thrombopoiesis hematologic disease auto-immune thrombocytopenic purpura relapsed persistent or chronic itp idiopathic thrombocytopenic purpura thrombotic thrombocytopenic purpura (ttp)