NCT04797000

Study of Efficacy and Safety of Eltrombopag in Lower-risk MDS Patients With Platelet Transfusion Dependence

A Randomized, Double-blind, Placebo-controlled, Japan Local Phase II Clinical Study Comparing Eltrombopag Monotherapy Versus Placebo in Adult Lower-risk Myelodysplastic Syndromes (MDS) Patients With Platelet Transfusion Dependence

• Status: Active Not Recruiting
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 36 (actual)
• Sponsor: Novartis Pharmaceuticals · Industry
• Sex: All
• Age: 20 Years – 100 Years
• Healthy volunteers: Not accepted

Summary

This study is designed to evaluate the efficacy and safety of eltrombopag monotherapy in Japanese adult patients with platelet transfusion-dependent lower-risk Myelodysplastic syndromes (LR-MDS).

Detailed description

This is a randomized, double-blind, placebo-controlled, Japanese local phase II study to evaluate the efficacy and safety of eltrombopag monotherapy in Japanese adult patients with platelet transfusion-dependent lower-risk MDS (IPSS-R very low, low, intermediate risk with bone marrow blast count \< 5% and cytogenetic very good, good or intermediate risk). Platelet transfusion dependence at baseline is defined as receiving platelet transfusion regularly with a frequency of 2 or more times within 4 weeks prior to randomization. Platelet transfusion should be performed for a patient with platelet counts \< 20 X 10\^9/L, or with hemorrhagic symptoms and platelet counts \< 30 X 10\^9/L. The primary objective is to demonstrate superiority of eltrombopag versus placebo in terms of the proportion of participants who achieve platelet transfusion independence at Week 24.

Conditions

• Myelodysplastic Syndromes

Interventions

Type	Name	Description
DRUG	Eltrombopag	Eltrombopag comes in 12.5 mg \& 25 mg tablets and is taken orally once per day (QD)
DRUG	Placebo	Placebo comes in 12.5 mg \& 25 mg tablets and is taken orally once per day (QD)

Timeline

• Start date: 2021-05-25
• Primary completion: 2025-04-26
• Completion: 2026-12-09
• First posted: 2021-03-15
• Last updated: 2026-03-19

Locations

20 sites across 1 country: Japan