Efficacy and Safety of Oral E5501 Plus Standard of Care for the Treatment of Thrombocytopenia in Adults With Chronic Immune Thrombocytopenia (Amendment 02)

Immune Thrombocytopenia Clinical Trial

Official title: A Phase 3, Multicenter, Randomized, Double-blind, Placebo-controlled, Parallel-group Trial With an Open-label Extension Phase to Evaluate the Efficacy and Safety of Oral E5501 Plus Standard of Care for the Treatment of Thrombocytopenia in Adults With Chronic Immune Thrombocytopenia (Idiopathic Thrombocytopenia Purpura)

Status Completed

Clinical Trial Summary

Core Study:

To demonstrate that the efficacy of avatrombopag (in addition to standard of care) is superior to placebo (in addition to standard of care) for the treatment of adult participants with chronic immune thrombocytopenia (idiopathic thrombocytopenic purpura) (ITP) as measured by cumulative number of weeks of platelet response over 6 months of once daily treatment in adults participants who received at least 1 prior ITP therapy.

Extension Phase:

To evaluate the safety and tolerability of long-term therapy with avatrombopag in participants with chronic ITP (cITP).

Clinical Trial Description

This study consists three phases: Prerandomization, Randomization (Core Study) and Extension study. The overall duration of treatment (Core and Extension) is approximately 104 weeks with the Core study being 26 weeks and the Extension study being 76 weeks. Approximately 45 participants 18 years of age and over who meet all the eligibility requirements will be randomized. No single platelet count should be greater than 35x10^9/L (liter). Participants will be centrally stratified at randomization by splenectomy status, baseline platelet count, and use of concomitant ITP medication at baseline, and randomized to receive either double-blind avatrombopag or placebo in a 2:1 ratio. Participants will receive blinded therapy at a starting dose of 20 mg avatrombopag or placebo once daily. Participants will be allowed to have their dose titrated up (maximum dose 40 mg avatrombopag or matching placebo) or down (minimum dose 5 mg for avatrombopag or matching placebo) depending on their response to study drug. The goal of dose modification is to maintain the platelet count at levels greater than or equal to 50x10^9/L and less than or equal to 150x10^9/L, and to decrease the need for ITP-directed concomitant medications. ;

Related Conditions & MeSH terms

• Chronic Thrombocytopenia
• Immune Thrombocytopenia
• Purpura, Thrombocytopenic, Idiopathic
• Thrombocytopenia

• NCT number: NCT01438840
• Study type: Interventional
• Source: Eisai Inc.
• Status: Completed
• Phase: Phase 3
• Start date: February 16, 2012
• Completion date: March 2014