An Open-label Safety Study of Lusutrombopag (S-888711) in Adults With Chronic Immune Thrombocytopenia (ITP)
Condition: Immune Thrombocytopenia
MeSH terms: Thrombocytopenia; Purpura, Thrombocytopenic, Idiopathic
Intervention: Lusutrombopag
Intervention description: tablet
Phase: Phase 2
Study type: Interventional
Primary purpose: Treatment
Intervention model: Single Group Assignment
Masking: None (Open Label)
Enrollment: 19.0
Sex: All
Minimum age: 18 Years
Healthy volunteers: No
Keywords: Splenectomy; Low Platelet Count; Thrombopoiesis; Thrombocytopaenia; Idiopathic Thrombocytopenic Purpura; Immune Thrombocytopenia (ITP); Thrombotic Thrombocytopenic Purpura (ITP); Hematologic Disease; Auto-immune Thrombocytopenic Purpura; S-888711; Blood Platelet Disorders; Relapsed Persistent or Chronic ITP; ITP

Summary:

      The primary objective of this study was to assess the long-term safety of lusutrombopag in
      the treatment of adults with relapsed persistent or chronic ITP with or without prior
      splenectomy.
    

Eligibility criteria:

        Inclusion Criteria:

          -  Subjects who previously participated in Study 0913M0621 (NCT01054443) and who
             completed treatment or discontinued treatment due to a platelet count > 400,000/μL and
             continued to meet all inclusion criteria of the previous study, listed below,
             including platelet counts < 50,000/μL were eligible for study participation. For the
             purpose of this study, initial screening visit and all prestudy time period refer to
             Study 0913M0621.

          -  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

          -  Subjects receiving steroid therapy must be on a stable dose for at least 2 weeks prior
             to Screening

          -  Prothrombin time (PT) and activated partial thromboplastin time (aPTT) within 20% of
             the upper limit of normal (ULN) at Screening

          -  Subjects receiving stable dosages of cyclosporine A, mycophenolate mofetil,
             azathioprine, or danazol are allowed

        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 1 week - Rho(D) immune globulin or intravenous immunoglobulin;

               -  Within 2 weeks - plasmaphoresis treatment;

               -  Within 4 weeks - use of anti-platelet or anti-coagulant drugs;

               -  Within 8 weeks - rituximab;

               -  Within 12 weeks - alemtuzumab, multi-drug systemic chemotherapy, stem cell
                  therapy;

          -  History of clinically significant cardiovascular or thromboembolic disease within 26
             weeks prior to Initial Screening

          -  Splenectomy within 4 weeks prior to Initial Screening

          -  Clinically significant laboratory abnormalities

               -  Hemoglobin < 10.0 g/dL for men or women, not clearly related to ITP

               -  Absolute neutrophil count < 1000/mm3

               -  Abnormal peripheral blood smear with evidence of fibrosis confirmed by bonemarrow
                  biopsy

               -  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 positive

               -  Hepatitis A IgM antibody positive, hepatitis B surface antigen or hepatitis C
                  antibody positive

          -  Exposure to previous thrombopoietin (TPO) mimetics/agonists (e.g.,
             eltrombopag,romiplostim, E5501 [AKR-501] or LGD-4665) within 4 weeks prior to Initial
             Screening

          -  Subjects unresponsive to previous TPO mimetics/agonists (e.g., eltrombopag,
             romiplostim, E5501 [AKR-501] or LGD-4665)

          -  Exposure to an investigative medication within 4 weeks prior to the initial Screening
             Visit