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 (iROM2)

  • STATUS
    Recruiting
  • End date
    Oct 22, 2024
  • participants needed
    24
  • sponsor
    University Children's Hospital Basel
Updated on 22 September 2022

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).

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

Details
Condition Primary Immune Thrombocytopenia (ITP)
Treatment Eltrombopag (Revolade®), standard therapy (without eltrombopag): HD-DXM
Clinical Study IdentifierNCT04812483
SponsorUniversity Children's Hospital Basel
Last Modified on22 September 2022

Eligibility

Yes No Not Sure

Inclusion Criteria

Informed consent as documented by signature
Newly diagnosed primary ITP according to the definition of Rodeghiero et al. and a risk of platelet count of <30x109/l or risk of severe bleeding
First-line therapy maximum for 1 week prior to enrolment
Bleeding severity and quality of life are neither an inclusion nor an exclusion criterion

Exclusion Criteria

Patients previously treated for ITP more than 7 days prior to enrolment (e.g. Steroid, intravenous immunoglobulin (IVIG), platelet infusion)
Patients treated with second-line drugs prior to enrolment
Life-threatening bleeding (and inability to sign informed consent)
Secondary ITP
Positive family history for ITP
Presence or history of autoimmune disease as judged by the investigator
Hepatosplenomegaly in the clinical examination
Relevant hepatic disease as judged by the investigator
Presence or history of thromboembolic disease
Patients with splenectomy
Women who are pregnant or breast feeding
Intention to become pregnant during the course of the study
Lack of safe double contraception
Any vaccination 2 weeks prior start of the study
Immunsuppressive and antiplatelet drugs
Known or suspected non-compliance, drug or alcohol abuse
Inability to follow the procedures of the study, e.g. due to language problems, psychological disorders, incompetence to judge
Participation in another study with investigational drug within the 30 days preceding and during the present study
Enrolment of the investigator, his/her family members, employees and other dependent persons
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