- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT04812483
Immunomodulation With Eltrombopag in ITP (iROM2)
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
Study Overview
Status
Conditions
Intervention / Treatment
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.
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.
- 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
Study Type
Enrollment (Actual)
Phase
- Phase 2
Contacts and Locations
Study Contact
- Name: Alexandra Schifferli, Dr. med.
- Phone Number: +41-61-704-1212
- Email: Alexandra.Schifferli@ukbb.ch
Study Contact Backup
- Name: Thomas Kühne, Prof. Dr. med.
- Phone Number: +41-61-704-1212
Study Locations
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Aarau, Switzerland
- Aarau Cantonal Hospital, Division of Hematology
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Basel, Switzerland, 4031
- University Children's Hospital Basel (UKBB)
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Basel, Switzerland, 4031
- University Hospital Basel, Division of Hematology
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Bern, Switzerland
- University Hospital Bern, Division of Hematology
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Liestal, Switzerland
- Liestal Cantonal Hospital, Division of Hematology
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Lucerne, Switzerland
- Lucerne Cantonal Hospital, Division of Hematology
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Participation Criteria
Eligibility Criteria
Ages Eligible for Study
Accepts Healthy Volunteers
Description
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
Study Plan
How is the study designed?
Design Details
- Primary Purpose: Treatment
- Allocation: Randomized
- Interventional Model: Parallel Assignment
- Masking: None (Open Label)
Arms and Interventions
Participant Group / Arm |
Intervention / Treatment |
---|---|
Other: Standard Arm
HD-DXM will be administered orally (40 mg) from day 1-4, followed by Arm 1: No planed further treatment. = standard therapy (without eltrombopag) |
standard therapy (without eltrombopag): HD-DXM administered orally (40 mg) from day 1-4
|
Experimental: Study Arm
HD-DXM will be administered orally (40 mg) from day 1-4, followed by Arm 2: The subjects in the experimental arm will be treated with eltrombopag: Eltrombopag (Revolade®), 50 mg PO, from day 5-140. Tapering over 1 week (week 21) from day 141-148 with 50 mg every second day. Eltrombopag will be administered on a starting dose of 50mg. After the end of treatment a clinical and laboratory observation follow-up period until week 30 follows. |
standard therapy (without eltrombopag): HD-DXM administered orally (40 mg) from day 1-4
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.
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What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
Change in percentual T-regulatory cells (Tregs)
Time Frame: before (Tregs/CD4), at week 3 and at the end of the treatment (week 20)
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Assessment of the percentage of Tregs (Tregs/CD4) in the study arm compared to the standard arm.
The Tregs will be defined as CD4+CD25+ CD127+ in the fluorescence-activated cell sorting (FACS).
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before (Tregs/CD4), at week 3 and at the end of the treatment (week 20)
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Secondary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
Change in Th1/Th2 balance
Time Frame: at baseline and weeks 3, 20 and 30
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Change in Th1/Th2 balance will be performed by analysis of immunologic profile (immune cell characteristics, messenger ribonucleic acid (mRNA) of immune cells, cytokines, cytokine concentrations)
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at baseline and weeks 3, 20 and 30
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Clinical response to eltrombopag therapy
Time Frame: trial duration (baseline to week 30)
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Clinical response to eltrombopag therapy (by assessing need of inpatient daycare and use of rescue treatment)
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trial duration (baseline to week 30)
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Platelet response to eltrombopag
Time Frame: at baseline and weeks 6, 20 and 30
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Platelet response to eltrombopag: proportion of subjects achieving a platelet count of ≥50x109/l (complete response, response and no response;Response at each assessment is defined as a platelet count of ≥ 50x109/l).
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at baseline and weeks 6, 20 and 30
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Other Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
Number of Adverse Events
Time Frame: trial duration (baseline to week 30)
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Safety of eltrombopag analyzed by documentation of number of Adverse Events
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trial duration (baseline to week 30)
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Severe bleeding
Time Frame: trial duration (baseline to week 30)
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Severe bleeding is defined as bleeding requiring hospital admission and/or blood transfusion.
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trial duration (baseline to week 30)
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Collaborators and Investigators
Collaborators
Investigators
- Principal Investigator: Alexandra Schifferli, Dr. med., University Children's Hospital Basel, UKBB
Study record dates
Study Major Dates
Study Start (Actual)
Primary Completion (Estimated)
Study Completion (Estimated)
Study Registration Dates
First Submitted
First Submitted That Met QC Criteria
First Posted (Actual)
Study Record Updates
Last Update Posted (Estimated)
Last Update Submitted That Met QC Criteria
Last Verified
More Information
Terms related to this study
Additional Relevant MeSH Terms
- Pathologic Processes
- Immune System Diseases
- Autoimmune Diseases
- Hematologic Diseases
- Hemorrhage
- Hemorrhagic Disorders
- Blood Coagulation Disorders
- Skin Manifestations
- Blood Platelet Disorders
- Thrombotic Microangiopathies
- Purpura, Thrombocytopenic
- Purpura
- Purpura, Thrombocytopenic, Idiopathic
- Thrombocytopenia
Other Study ID Numbers
- 2021-00044; ks19Schifferli
Drug and device information, study documents
Studies a U.S. FDA-regulated drug product
Studies a U.S. FDA-regulated device product
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
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