NCT03524612

A Study to Assess the Ability of Eltrombopag to Induce Sustained Response Off Treatment in Subjects With ITP

A Phase II, Open-label, Prospective, Single-arm, Study to Assess Ability of Eltrombopag to Induce Sustained Remission in Subjects With ITP Who Are Refractory or Relapsed After First-line Steroids

• Status: Completed
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 105 (actual)
• Sponsor: Novartis Pharmaceuticals · Industry
• Sex: All
• Age: 18 Years – 99 Years
• Healthy volunteers: Not accepted

Summary

The purpose of this trial was to assess the ability of eltrombopag to induce sustained treatment-free remission in immune thrombocytopenia purpura (ITP) subjects who relapsed or failed to respond to an initial treatment with steroids.

Detailed description

Protocol Amendment 01 added a follow-up period of 12 months for patients with sustained response off treatment at month 12 to obtain further data on response duration. The starting dose was eltrombopag 50 mg daily (25 mg daily for Asian patients and 12.5 mg daily for Japanese patients in Japan). The starting dose for this study was consistent with the dosing guidelines approved for eltrombopag use in ITP. An increase of eltrombopag dose up to 75 mg was allowed for patients who did not respond to standard-dosage treatment and to reduce the risk of bleeding. The rationale for this increased dose was to use the minimal efficacious dosage of eltrombopag in order to achieve a platelet count ≥ 100×10\^9/L and maintain it around 100×10\^9/L (no counts below 70×10\^9/L) for 2 months in order to allow patients to start the eltrombopag tapering and withdrawal process. Patients who reached a platelet count of \>= 100 × 10\^9/L and maintained counts around 100×10\^9/L for 2 months with no platelet count \< 70 × 10\^9/L were eligible for tapering-off and treatment discontinuation, which occurred via 25 mg reduction every 2 weeks up to 25 mg on alternate days for 2 weeks until treatment discontinuation. Patients who successfully discontinue eltrombopag and maintained platelet count \>= 30 × 10\^9/L in the absence of bleeding or use of rescue therapy were followed to month 12. If a relapse (defined as platelet count \<30 × 10\^9/L) occurred during the 12-month treatment period, they were offered a new course of eltrombopag treatment within this timeframe at the appropriate starting dose. If a patient relapsed in the post 12-month follow-up period, no further attempts for tapering and achieving sustained response off treatment were made. The taper-off scheme followed recommendations within the current established dosing regimen for when to consider dose adjustment.

Conditions

• Immune Thrombocytopenic Purpura (ITP)

Interventions

Type	Name	Description
DRUG	eltrombopag	12.5, 25, 50 and 75 mg tablets for oral use once daily

Timeline

• Start date: 2018-11-02
• Primary completion: 2021-10-22
• Completion: 2022-10-03
• First posted: 2018-05-15
• Last updated: 2025-01-07
• Results posted: 2023-12-27

Locations

32 sites across 15 countries: United States, Austria, Brazil, Chile, France, Greece, Italy, Japan, Mexico, Oman, Russia, Spain, Switzerland, Turkey (Türkiye), United Kingdom

Regulatory

• FDA-regulated drug study