Primary Immune Thrombocytopenia (ITP)Phase 2iROM2NCT04812483
Immunomodulation With Eltrombopag in ITP
A Phase 2 study testing Eltrombopag (Revolade®) and standard therapy (without eltrombopag): HD-DXM in people with Primary Immune Thrombocytopenia (ITP), run by University Children's Hospital Basel at 6 sites in Switzerland.
Trial 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). Read more about this study
- Who is it for?
- Ages 18 to 55 years
- Conditions the study is for
- Primary Immune Thrombocytopenia (ITP)
- What is being tested?
- Eltrombopag (Revolade®) and standard therapy (without eltrombopag): HD-DXM
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 2 participants
- Where
- 6 sitesSwitzerland
- Study dates
- 1 Sep 2022to 11 Apr 2024
- Phase
- Phase 2
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 55 years
- All sexes
- No healthy volunteers
- Adult
Who may be able to join
- 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.
Who cannot join
- 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
Show all 19Show fewer
- 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
Trial locations and contacts
6 sites in 1 country.
6 sites in 1 country
Switzerland6 sites
Aarau Cantonal Hospital, Division of Hematology
Aarau
University Children's Hospital Basel (UKBB)
Basel, 4031
University Hospital Basel, Division of Hematology
Basel, 4031
University Hospital Bern, Division of Hematology
Bern
Liestal Cantonal Hospital, Division of Hematology
Liestal
Lucerne Cantonal Hospital, Division of Hematology
Lucerne
About this study
Official title: 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
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).
Read the 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
- Conditions
- Primary Immune Thrombocytopenia (ITP)
- Being tested
- Eltrombopag (Revolade®), standard therapy (without eltrombopag): HD-DXM
- Keywords
- eltrombopag, thrombopoietin receptor agonist (TPO-RA), ITP in young and midlife adults
- Medical subject headings
- Purpura, Thrombocytopenic, Idiopathic
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Eltrombopag (Revolade®)Drug
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.
standard therapy (without eltrombopag): HD-DXMDrug
standard therapy (without eltrombopag): HD-DXM administered orally (40 mg) from day 1-4
| Study group | Role | What they receive |
|---|---|---|
| Standard Arm | Other | HD-DXM will be administered orally (40 mg) from day 1-4, followed by Arm 1:
No planed further treatment. = standard therapy (without eltrombopag) Receives: Drug: standard therapy (without eltrombopag): HD-DXM |
| Study Arm | Experimental | 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. Receives: Drug: Eltrombopag (Revolade®); Drug: standard therapy (without eltrombopag): HD-DXM |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change in percentual T-regulatory cells (Tregs)
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).
Measured: before (Tregs/CD4), at week 3 and at the end of the treatment (week 20)
Secondary outcomes (3)
Change in Th1/Th2 balance
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)
Measured: at baseline and weeks 3, 20 and 30
Clinical response to eltrombopag therapy
Clinical response to eltrombopag therapy (by assessing need of inpatient daycare and use of rescue treatment)
Measured: trial duration (baseline to week 30)
Platelet response to eltrombopag
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).
Measured: at baseline and weeks 6, 20 and 30
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted23 Mar 2021
- Study start1 Sep 2022
- Primary completion11 Apr 2024
- Study completion11 Apr 2024
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Primary Immune Thrombocytopenia (ITP)Studies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Model description
- Randomized study evaluating the immunomodulatory effects of eltrombopag versus standard high-dose (HD) DXM therapy (1:1) in newly diagnosed ITP.
- Masking
- None (open label)
- Enrolment
- 2 participants (actual)
Sponsor and investigators
- Lead sponsor
- University Children's Hospital Basel
- Sponsor type
- University, hospital or other
- Collaborators
- Stiftung zur Förderung medizinischer und biologischer Forschung, Novartis Pharmaceuticals, University of Erlangen-Nürnberg, Department of Biology
- Responsible party
- Sponsor
- Investigators
- Alexandra Schifferli, Dr. med., Principal investigator, University Children's Hospital Basel
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04812483
- Sponsor's protocol ID
- 2021-00044; ks19Schifferli
- Acronym
- iROM2
- Status last verified
- Oct 2024
- First submitted
- 1 Mar 2021
- Last updated
- 15 Oct 2024
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 15 Oct 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- 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
- ClinicalTrials.gov ID
- NCT04812483
- Acronym / sponsor ID
- iROM2 / 2021-00044; ks19Schifferli
- Conditions
- Primary Immune Thrombocytopenia (ITP)
- Keywords
- eltrombopag, thrombopoietin receptor agonist (TPO-RA), ITP in young and midlife adults
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 2 participants (actual)
- Lead sponsor
- University Children's Hospital BaselUniversity, hospital or other
- Collaborators
- Stiftung zur Förderung medizinischer und biologischer Forschung, Novartis Pharmaceuticals, University of Erlangen-Nürnberg, Department of Biology
- Study start
- 1 Sep 2022 (actual)
- Primary completion
- 11 Apr 2024 (actual)
- Study completion
- 11 Apr 2024 (actual)
- First posted
- 23 Mar 2021 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
- 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 design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
- Model description
- Randomized study evaluating the immunomodulatory effects of eltrombopag versus standard high-dose (HD) DXM therapy (1:1) in newly diagnosed ITP.
| Arm | Type | Receives |
|---|---|---|
| Standard Arm | Other | Drug: standard therapy (without eltrombopag): HD-DXMHD-DXM will be administered orally (40 mg) from day 1-4, followed by Arm 1: No planed further treatment. = standard therapy (without eltrombopag) |
| Study Arm | Experimental | Drug: Eltrombopag (Revolade®), Drug: standard therapy (without eltrombopag): HD-DXMHD-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. |
Who may qualify
- Ages
- 18 to 55 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult
Key inclusion criteria (4 listed)
- 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.
Key exclusion criteria (19 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| 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 | Study Arm |
| standard therapy (without eltrombopag): HD-DXMstandard therapy (without eltrombopag): HD-DXM administered orally (40 mg) from day 1-4 | Drug | Standard Arm, Study Arm |
Primary outcome
Change in percentual T-regulatory cells (Tregs)
Time frame: before (Tregs/CD4), at week 3 and at the end of the treatment (week 20)
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).
3 secondary outcomes
Change in Th1/Th2 balance
Time frame: at baseline and weeks 3, 20 and 30
Clinical response to eltrombopag therapy
Time frame: trial duration (baseline to week 30)
Platelet response to eltrombopag
Time frame: at baseline and weeks 6, 20 and 30
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 15 Oct 2024.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Alexandra Schifferli, Dr. med. principal investigator
University Children's Hospital Basel
6 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Eltrombopag (Revolade®) and standard therapy (without eltrombopag): HD-DXM in people with Primary Immune Thrombocytopenia (ITP), run by University Children's Hospital Basel at 6 sites in Switzerland.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 4 inclusion and 19 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 15 Oct 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 6 sites in Switzerland. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Change in percentual T-regulatory cells (Tregs) (time frame: before (Tregs/CD4), at week 3 and at the end of the treatment (week 20)). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 15 Oct 2024.