A Study to Assess the Ability of Eltrombopag to Induce Sustained Response Off Treatment in Subjects With ITP
Condition: Immune Thrombocytopenic Purpura (ITP)
MeSH terms: Purpura; Purpura, Thrombocytopenic; Purpura, Thrombocytopenic, Idiopathic
Intervention: eltrombopag
Intervention description: 12.5, 25, 50 and 75 mg tablets for oral use once daily
Phase: Phase 2
Study type: Interventional
Primary purpose: Treatment
Intervention model: Single Group Assignment
Masking: None (Open Label)
Enrollment: 105.0
Sex: All
Minimum age: 18 Years
Maximum age: 99 Years
Healthy volunteers: No
Keywords: Thrombocytopenic; Immune; Platelets; Bleeding; ITP; eltrombopag; ETB115; induce sustained response off treatment; refractory or relapsed; first-line steroids

Summary:

      The purpose of this trial was to assess the ability of eltrombopag to induce sustained
      treatment-free remission in immune thrombocytopenia purpura (ITP) subjects who relapsed or
      failed to respond to an initial treatment with steroids.
    

Eligibility criteria:

        Inclusion Criteria:

          1. Signed informed consent must be obtained prior to participation in the study

          2. Patients ≥ 18 years old

          3. Patients with a confirmed diagnosis of primary ITP, who are not responsive or in
             relapse after a first line of steroid therapy ± intravenous immunoglobulin (IVIG)
             (used as a rescue therapy)

          4. Platelet count < 30×10^9/L and assessed as needing treatment (per physician's
             discretion

        Exclusion Criteria:

          1. ITP patients previously treated with any ITP second-line therapies, thrombopoietin
             receptor (TPO-R) agonists for ITP, except steroids / IVIG

          2. Patients who relapsed more than one year after the end of first-line full course of
             steroid therapy

          3. Patients with a diagnosis of secondary thrombocytopenia

          4. Patients who have life threatening bleeding complications per investigator discretion

          5. Patients who had a deep vein thrombosis or arterial thrombosis in the 6 months
             preceding enrollment

          6. Serum creatinine ≥ 1.5 mg/dL

          7. Total bilirubin > 1.5 × upper limit of normal (ULN)

          8. Aspartate transaminase (AST) > 3.0 × ULN

          9. Alanine transaminase (ALT) > 3.0 × ULN

         10. Patients who are human immune deficiency virus (HIV), hepatitis C virus (HCV),
             hepatitis B surface antigen (HBsAg) positive

         11. Patients with hepatic impairment (Child-Pugh score > 5)

         12. Patients who have active malignancy

         13. Patients with any serious and/or unstable pre-existing medical, psychiatric disorder
             or other conditions that could interfere with subject's safety, obtaining informed
             consent or compliance with the study procedures per investigator discretion

         14. History or current diagnosis of cardiac disease indicating significant risk of safety
             for Patients participating in the study

         15. Patients with known active or uncontrolled infections not responding to appropriate
             therapy

         16. Patients with evidence of current alcohol/drug abuse

         17. Women of child-bearing potential and sexually active males unwilling to use adequate
             contraception during the study

         18. Female Patients who are nursing or pregnant (positive serum or urine B-human chorionic
             gonadotrophin (B-hCG) pregnancy test) at screening or pre-dose on Day 1

        Other protocol-defined inclusion/exclusion criteria may apply.