Clinical Trial Details

A Phase 3 Study of Eltrombopag vs. Standard Front-Line Management for Newly Diagnosed Immune Thrombocytopenia (ITP) in Children

Categories (click each to see list of all clinical trials associated with that category): Pediatric (PEDONC)

Current Status: Open

Phase: III

Principal Investigator: Bies, Joshua

Eligibility: https://clinicaltrials.gov/study/NCT03939637?term=NCT03939637&rank=1

Summary
Primary Objective To determine if the proportion of patients with a platelet response is significantly greater in patients with newly diagnosed ITP treated with eltrombopag than those treated with standard first-line treatments Secondary Objectives 3.2.1 To compare the proportion of patients with poor bleeding scores (WHO Bleeding Scale≥2 or Modified Buchanan Score≥3) at 1, 2, 3, 4, 12 weeks and 1 year in patients with newly diagnosed ITP treated with eltrombopag vs. standard first-line agents 3.2.2 To compare the cumulative number of rescue therapies (defined in section 8.4) needed during the first 12 weeks of treatment for patients with newly diagnosed ITP treated with eltrombopag vs. those treated with standard first-line agents 3.2.3 To compare platelet response during weeks 5-12 of study in patients treated with eltrombopag vs standard first line agents who required a rescue treatment during weeks 1-2 of study 3.2.4 To compare the proportion of patients who do not need ongoing treatment at 12 weeks and 6 months for patients with newly diagnosed ITP treated with eltrombopag vs. those treated with standard first-line agents 3.2.5 To compare the proportion of patients with a treatment response (4 binary endpoints (section 5.2.5)) at one year after study enrollment in patients with newly diagnosed ITP treated with eltrombopag vs. those treated with standard first-line agents 3.2.6 To compare the number of 2nd-line therapies (defined in section 7.2.1) used in weeks 13-52 for patients with newly diagnosed with ITP treated with eltrombopag vs. those treated with standard first-line agents 3.2.7 To compare the change in percentage of CD4+25+Foxp3+ regulatory T cells (Tregs) a) from baseline to 12 weeks; and b) from baseline to 1 year, in patients with newly diagnosed ITP treated with eltrombopag vs. those treated with standard first-line agents 3.2.8 To compare change in Health Related Quality of Life (HRQoL) from a) baseline to 1 week, b) baseline to 4 weeks, c) baseline to 12 weeks, and, d) baseline to 1 year, as measured by the parent-proxy report of the Kids ITP tools (KIT) and Global Change Scale for patients with newly diagnosed ITP treated with eltrombopag vs. those treated with standard first-line agents 3.2.9 To compare fatigue at 1 week, 4 weeks, 12 weeks, and 1 year as measured by the parent-proxy report of the Hockenberry Fatigue Scale-Parent (FS-P) for patients with newly diagnosed ITP treated with eltrombopag vs. those treated with standard first-line agents 3.2.10 To compare iron indices at 12 weeks, 6 months, and 1 year in patients with newly diagnosed ITP treated with eltrombopag vs. those treated with standard first-line agents 3.2.11 Safety Evaluations: To describe the proportion of patients with abnormal liver function tests (LFTs) in patients with newly diagnosed ITP treated with eltrombopag. To describe the proportion of patients with adverse events and serious adverse events in patients with newly diagnosed ITP treated with eltrombopag and standard first-line agents 3.3 Exploratory Objectives 3.3.1 To compare additional platelet-defined responses in patients with newly diagnosed ITP treated with eltrombopag versus those treated with standard therapy. 3.3.2 To compare additional changes in HRQoL (measured by KIT) and fatigue (measured by Hockenberry Fatigue scale) at 1 week, 4 weeks, 12 weeks, and 1 year in patients with newly diagnosed ITP treated with eltrombopag versus those treated with standard therapy. 3.3.3 To compare cost of therapy (including medications, hospitalizations, the PROMIS survey, etc.) between patients treated with eltrombopag versus those treated with standard therapy.