NCT04812483

Immunomodulation With Eltrombopag in ITP

Immunomodulation in Young and Midlife Adults With Newly Diagnosed Primary Immune Thrombocytopenia (ITP): A Randomized Open Label Trial With High-dose Dexamethasone Versus Eltrombopag and High-dose Dexamethasone

• Status: Completed
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 2 (actual)
• Sponsor: University Children's Hospital Basel · Academic / Other
• Sex: All
• Age: 18 Years – 55 Years
• Healthy volunteers: Not accepted

Summary

The study aims to investigate immunomodulatory effects of eltrombopag combined with dexamethasone in young and midlife adult patients with newly diagnosed primary Immune thrombocytopenia (ITP).

Detailed description

The randomized open lable study aims to investigate immunomodulatory effects of eltrombopag combined with dexamethasone in young and midlife adult patients with newly diagnosed primary ITP. Treatment protocol will be HD-DXM (40 mg PO, day 1-4) with or without eltrombopag (25-50 mg PO, day 5-140) on an outpatient basis. Immunological investigations will be performed before start of treatment and then on week 3, 20 (end of therapy) and 30. 1. Intervention phase: Medical history and physical examination including assessment of severe bleeding every week until week 4, every second week until week 20. Complete blood count every week until week 10. For the adjustment of the Thrombopoietin receptor agonist (TPO-RA) dose - every second week until week 20. Immunologic panel at the beginning and at week 3 and 20. 2. Follow-up: Three clinical visits are scheduled in the follow-up including a complete blood count: at week 22, 24 and 30. Immunologic panel will be done at week 30 (end of study). High-dose dexamethasone (HD-DXM) will be administered orally (40 mg) from day 1-4, followed by Arm 1 or 2 (1:1 randomization). Arm 1: Standard Arm No planed further treatment. = standard therapy. In case of non-response after 2 courses of HD-DXM (week 4): cross-over to Arm 2: Start Eltrombopag (Revolade®), 50 mg PO until day 140 (details see Arm 2). In case of relapse: repeat HD-DXM (40 mg day 1-4), up to a maximal of 3 courses. Time between 2 courses should be minimal 14 days. In case of re-relapse after the third course: cross-over to Arm 2: Start Eltrombopag (Revolade®), 50 mg PO until day 140 (details see Arm 2). Arm 2: Study Arm Eltrombopag (Revolade®), 50 mg per os, from day 5-140. Tapering over 1 week from day 141-148 with 50 mg every second day. In case of non-response after 4 weeks on eltrombopag: drop out

Conditions

• Primary Immune Thrombocytopenia (ITP)

Interventions

Type	Name	Description
DRUG	Eltrombopag (Revolade®)	Eltrombopag is a thrombopoietin receptor agonist (TPO-RA) indicated in patients with ITP refractory to first-line drugs or lasting more than 6 months. Administration of Eltrombopag (Revolade®), 50 mg PO, from day 5-140. Tapering over 1 week from day 141-148 with 50 mg every second day.
DRUG	standard therapy (without eltrombopag): HD-DXM	standard therapy (without eltrombopag): HD-DXM administered orally (40 mg) from day 1-4

Timeline

• Start date: 2022-09-01
• Primary completion: 2024-04-11
• Completion: 2024-04-11
• First posted: 2021-03-23
• Last updated: 2024-10-15

Locations

6 sites across 1 country: Switzerland