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.