NCT03939637

Eltrombopag vs Standard Front Line Management for Newly Diagnosed Immune Thrombocytopenia (ITP) in Children

A Phase III Study of Eltrombopag vs Standard Front Line Management for Newly Diagnosed Immune Thrombocytopenia in Children

• Status: Completed
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 122 (actual)
• Sponsor: Baylor College of Medicine · Academic / Other
• Sex: All
• Age: 1 Year – 18 Years
• Healthy volunteers: Not accepted

Summary

This is an investigator initiated, multicenter, open label, randomized phase 3 study for subjects with newly diagnosed ITP from ages 1 to less than 18 years old.

Detailed description

This is a prospective, open label, randomized, two-arm, multi-center Phase 3 trial. Patients with newly diagnosed ITP are randomized 2:1 to receive the experimental treatment, eltrombopag, or investigator's choice of 3 standard therapies. The primary objective is to determine if the proportion of patients with platelet response is significantly greater in patients treated with eltrombopag compared to those treated with standard therapies.

Conditions

• Immune Thrombocytopenia

Interventions

Type	Name	Description
DRUG	Eltrombopag	Starting dose for eltrombopag will be based on manufacturer recommendations, and drug will be titrated to effect per guidelines. * Children 1 to 5 years: Initial: 25 mg once daily * Children ≥6 years and Adolescents: Initial: 50 mg once daily (25 mg once daily for patients of East-Asian ethnicity \[e.g., Chinese, Japanese, Korean, Taiwanese\]) Dose should be titrated based on platelet response. Maximum dose: 75 mg once daily.
DRUG	Steroids	Prednisone/Prednisolone 4mg/kg/day (Max 120 mg/day) x 4 day
DRUG	IVIG	IVIG 1 g/kg x1 (no steroids for pre-medication or adjunctive therapy)
DRUG	Rho(D) Immune Globulin	Anti-D globulin 75 mcg/kg x1 (no steroids for pre-medication or adjunctive therapy)

Timeline

• Start date: 2019-05-02
• Primary completion: 2024-04-17
• Completion: 2025-02-26
• First posted: 2019-05-07
• Last updated: 2025-07-29
• Results posted: 2025-07-29

Locations

27 sites across 1 country: United States

Regulatory

• FDA-regulated drug study