A Study to Assess Efficacy and Safety of Eltrombopag in Combination With a Short Course of Dexamethasone in Patients With Newly Diagnosed ITP
Condition: Immune Thrombocytopenia (ITP)
MeSH terms: Thrombocytopenia; Purpura, Thrombocytopenic, Idiopathic
Study type: Interventional
Phase: Phase 2

Summary: 
      The purpose of this study is to compare the ability of eltrombopag in combination with a
      short course of high-dose dexamethasone to induce sustained response off treatment in
      patients with newly-diagnosed ITP versus 1-3 cycles of dexamethasone monotherapy.

      The unmet clinical need and the potential for eltrombopag when added to steroids to improve
      the treatment outcome and the potential to induce sustained response off treatment serve as
      the basis for clinical investigation of eltrombopag in first-line ITP.
    

Detailed description: 
      This is a Phase II, multicenter, 1:1 randomized, open-label study to compare the efficacy and
      safety of eltrombopag in combination with a short course of high-dose dexamethasone to 1-3
      cycles of high-dose dexamethasone monotherapy, as first-line treatment in adult patients with
      newly diagnosed ITP.

      Adult patients with newly diagnosed ITP who have platelet counts < 30 × 109/L and require
      treatment will be screened, and if eligible, will be randomized to either Arm A (eltrombopag
      in combination with a short course of dexamethasone) or Arm B (1-3 cycles of dexamethasone
      monotherapy).

      The study will be conducted in the following periods:

      Screening Period: Patients will be screened for 14 days based on the inclusion and exclusion
      criteria

      Treatment Period: Arm A: Patients will be treated for 26 weeks during the treatment period.
      Patients who reach platelet counts ≥ 30 × 109/L and maintain counts ≥ 30 × 109/L during the
      tapering phase will be eligible for treatment discontinuation. Duration of tapering before
      treatment discontinuation at Week 26 will be 6 weeks. Arm B: Patients will be treated up to
      12 weeks during the treatment period. Patients who reach platelet counts ≥ 30 × 109/L and
      maintain counts ≥ 30 × 109/L after 1-3 cycles of dexamethasone treatment will be eligible for
      treatment discontinuation. Patients with platelet counts < 30 × 109/L after 3 cycles of
      dexamethasone treatment will be offered a course of eltrombopag treatment within the study
      and will discontinue from study at week 52.

      Observation period: After completion of treatment period, all patients will be observed for
      sustained response off treatment until week 52. Only patients with sustained response at week
      52 will be followed for another 26 weeks. Patients who relapse between Week 52 and Week 78
      will discontinue the study.
    

Intervention name(s): Eltrombopag; Dexamethasone
Intervention type(s): Drug; Drug
Intervention description(s): Eltrombopag is for oral use and comes in 25, 50 and 75 mg tablets. Prescribed dose is taken once daily.; Dexamethasone is for oral use and comes in 8 mg tablets. Prescribed dose is taken once daily.
Other intervention name(s): ETB115
Intervention MeSH terms: Dexamethasone
Allocation: Randomized
Intervention model: Parallel Assignment
Masking: None (Open Label)
Primary purpose: Treatment
Keywords: ITP; Eltrombopag; Dexamethasone; Sustained response off treatment; TFR; Adult; ETB115; Platelets; Thrombocytopenic; Thrombopoietin receptor Agonist; Newly-diagnosed; Blood bleeding disorder