Immune ThrombocytopeniaPhase 4iROMNCT02760251
Immunomodulation With Romiplostim in Young Adults With ITP
A Phase 4 study testing romiplostim in people with Immune Thrombocytopenia, run by University Hospital, Basel, Switzerland at 5 sites in Switzerland.
Trial summary
The study aims to investigate immunomodulatory effects of thrombopoietin-receptor Agonist (TPO-RA) in patients with primary ITP, who failed first-line therapy or who became intolerant to it. Read more about this study
- Who is it for?
- Ages 18 to 45 years
- Conditions the study is for
- Immune Thrombocytopenia
- What is being tested?
- romiplostim
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 15 participants
- Where
- 5 sitesSwitzerland
- Study dates
- Apr 2016to Mar 2020
- Phase
- Phase 4
- 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 45 years
- All sexes
- No healthy volunteers
- Adult
Who may be able to join
- Informed consent as documented by signature (see informed consent form)
- Primary ITP according to the definition of Rodeghiero et al. (52) and a platelet count of <30x109/l
- Age range: 18-45 years
- Previously treated patients, with failure or intolerance to first-line therapy, or relapse after first-line therapy, i.e. corticosteroids, intravenous immunoglobulin (IVIG), or anti-D immunoglobulins
Who cannot join
- Adults older than 45 and children younger than 18 years
- Platelet count higher than 30x109/l at time of screening
- Suspicion of secondary ITP
- Positive family history for ITP
- Presence or history of autoimmune disease as judged by the investigator
- Hepatosplenomegaly
Show all 21Show fewer
- Presence or history of relevant hepatic disease as judged by the investigator
- Presence or history of thromboembolic disease as judged by the investigator
- 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 (see 7.1)
- Any vaccination 2 weeks prior start of the study
- Drugs with a known impact on the immune system or on platelet function must be recorded and an exclusion of the study should be discussed with the study center
- 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, dementia of the study subject
- Participation in another study with investigational drug within the 30 days preceding and during the present study
- Previous enrolment into the current study
- Previous treatment with romiplostim or eltrombopag
- Hypersensitivity to the active substance or to any of the excipients or to E. coli derived proteins
- Enrolment of the investigator, his/her family members, employees and other dependent persons
Trial locations and contacts
5 sites in 1 country.
5 sites in 1 country
Switzerland5 sites
Aarau Cantonal Hospital
Aarau
University Hospital Basel
Basel, 4000
University Hospital Bern
Bern
Liestal Cantonal Hospital
Liestal, Basel-Landschaft
Lucerne Cantonal Hospital
Lucerne, Canton of Lucerne
About this study
Official title: Thrombopoietin-receptor Agonist-immunomodulation in Young Adult Primary Immune Thrombocytopenia (ITP): A Multi-center Open Label Trial With Romiplostim
The study aims to investigate immunomodulatory effects of thrombopoietin-receptor Agonist (TPO-RA) in patients with primary ITP, who failed first-line therapy or who became intolerant to it. It is hypothesized that the early phase of this autoimmune disease may exhibit a stronger immunomodulatory potential in response to a stimulus, such as romiplostim. Such a process may subsequently be capable to induce regulatory mechanisms or tolerance.
Romiplostim (a thrombopoietin-receptor agonist, TPO-RA) will be administered subcutaneously once weekly over 22 weeks with a starting dose of 1mcg/kg body weight. The dose will be adjusted based on platelet counts as described in the summary of Product Characteristics (SmPC).
- Conditions
- Immune Thrombocytopenia
- Being tested
- romiplostim
- Keywords
- immunomodulation
- 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.
romiplostimDrug
Also called Nplate
| Study group | Role | What they receive |
|---|---|---|
| Romiplostim | Experimental | Romiplostim (a thrombopoietin-receptor agonist, TPO-RA) will be administered subcutaneously once weekly over 22 weeks with a starting dose of 1mcg/kg body weight. The dose will be adjusted based on platelet counts as described in the summary of Product Characteristics (SmPC). Followup examination at week 52. Receives: Drug: romiplostim |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change in Interleukin (IL)-4 concentrations (pg/ml) from baseline to week 22
The primary aim of the study is to demonstrate an immunomodulatory effect of the study drug. Investigators expect a shift in the Th1/Th2 balance towards Th2.
The primary outcome is to compare the pre- and post-treatment IL-4 concentrations (pg/ml) of all included patients (Th2 profile). Assessment of change in pre- and post-treatment IL-4 concentrations (pg/ml).
Measured: baseline and 22 weeks
Secondary outcomes (16)
Change in immunomodulation as assessed by immune cell characteristics between baseline and week 22
Immunologic parameters will be investigated between baseline and week 22 of the study: immune cell characteristics
fluorescence-activated cell sorting (FACS): B cells: cluster of Differentiation Antigen (CD)3- cluster of Differentiation Antigen (CD)19+ Memory B lymphocytes CD19+cluster of Differentiation Antigen (CD)10-cluster of Differentiation Antigen (CD)27+cluster of Differentiation Antigen (CD)38- Plasma cells: CD19+ CD10- cluster of Differentiation Antigen (CD)20- CD27++ cluster of Differentiation Antigen (CD)38++ Tcells and subpopulations: CD3+/ CD3+ CD4+/ CD3+ cluster of Differentiation Antigen (CD)8+ natural killer (NK) cells CD3- cluster of Differentiation Antigen (CD)16+ cluster of Differentiation (CD)56+ Monocytes CD56- cluster of Differentiation Antigen (CD)14 (low) CD16+ cluster of Differentiation Antigen (CD)33 Tregs CD4+ cluster of Differentiation Antigen (CD)25 superhigh FoxP3+
Measured: baseline and 22 weeks
Change in immunomodulation as assessed by immune cell characteristics between baseline and week 10
Immunologic parameters will be investigated between baseline and week 10 of the study: immune cell characteristics
FACS:
B cells: CD3- CD19+ Memory B lymphocytes CD19+CD10-CD27+CD38- Plasma cells: CD19+ CD10- CD20- CD27++ CD38++ Tcells and subpopulations: CD3+/ CD3+ CD4+/ CD3+ CD8+ NK cells CD3- CD16+ CD56+ Monocytes CD56- CD14 (low) CD16+ CD33 Tregs CD4+ CD25 superhigh FoxP3+
Measured: baseline and 10 weeks
Change in immunomodulation as assessed by messenger ribonucleic acid (mRNA) of cytokines between baseline and week 22
mRNA of cytokines will be investigated between baseline and week 22
mRNA essays (real-time quantitative PCR): cytokine mRNA (interleucin (IL)2, interleucin (IL)4, interleucin (IL)6, interleucin (IL)10, IL17, interleucin (IL)35, IFNgamma, TNFalpha, transforming growth factor (TGF)-β)
Measured: baseline and 22 weeks
Change in immunomodulation as assessed by mRNA of immune cells between baseline and week 22
mRNA of immune cells will be investigated between baseline and week 22
mRNA essays (real-time quantitative PCR): mRNA Th1 (T-bet), Th2 (GATA-3), Th17 (RORγt), Tregs (Foxp3)
Measured: baseline and 22 weeks
Change in immunomodulation as assessed by mRNA of cytokines between baseline and week 10
mRNA of cytokines will be investigated between baseline and week 10
mRNA essays (real-time quantitative PCR): cytokine mRNA (IL2, IL4, IL6, IL10, IL17, IL35, IFNgamma, TNFalpha, TGF-β)
Measured: baseline and 10 weeks
Change in immunomodulation as assessed by mRNA of immune cells between baseline and week 10
mRNA of immune cells will be investigated between baseline and week 10
mRNA essays (real-time quantitative PCR): mRNA Th1 (T-bet), Th2 (GATA-3), Th17 (RORγt), Tregs (Foxp3)
Measured: baseline and 10 weeks
Change in immunomodulation as assessed by cytokine concentrations between baseline and week 22
cytokine concentration will be investigated between baseline and week 22
ELISA Cytokines: IL2, IL4, IL6, IL10, IL17, IL35, IFNgamma, TNFalpha, TGF- β
Measured: baseline and 22 weeks
Change in immunomodulation as assessed by cytokine concentrations between baseline and week 10
cytokine concentration will be investigated between baseline and week 10
ELISA Cytokines: IL2, IL4, IL6, IL10, IL17, IL35, IFNgamma, TNFalpha, TGF- β
Measured: baseline and 10 weeks
Clinical response between baseline and week 52: number of severe bleeding
Clinical characterization of response to romiplostim therapy will be assessed additionally : measurement of severe bleeding (score Bolton-Maggs)
Measured: baseline and 52 weeks
Clinical response between baseline and week 52: number of days in hospital
Clinical characterization of response to romiplostim therapy will be assessed additionally : need of inpatient daycare
Measured: baseline and 52 weeks
Clinical response between baseline and week 52: platelet more than >100G/l
Clinical characterization of response to romiplostim therapy will be assessed additionally : assessment of platelet response to romiplostim, according to the definitions of Rodeghiero et al. (49).
Measured: baseline and 52 weeks
Change in immunomodulation as assessed by immune cell characteristics between baseline and week 52
Immunologic parameters will be investigated between baseline and week 52 of the study: immune cell characteristics
FACS:
B cells: CD3- CD19+ Memory B lymphocytes CD19+CD10-CD27+CD38- Plasma cells: CD19+ CD10- CD20- CD27++ CD38++ Tcells and subpopulations: CD3+/ CD3+ CD4+/ CD3+ CD8+ NK cells CD3- CD16+ CD56+ Monocytes CD56- CD14 (low) CD16+ CD33 Tregs CD4+ CD25 superhigh FoxP3+
Measured: baseline and 52 weeks
Change in immunomodulation as assessed by mRNA of immune cells between baseline and week 52
mRNA of immune cells will be investigated between baseline and week 52
mRNA essays (real-time quantitative PCR): mRNA Th1 (T-bet), Th2 (GATA-3), Th17 (RORγt), Tregs (Foxp3)
Measured: baseline and 52 weeks
Change of immunomodulation as assessed by mRNA of cytokines between baseline and week 52
mRNA of cytokines will be investigated between baseline and week 52
mRNA essays (real-time quantitative PCR): cytokine mRNA (IL2, IL4, IL6, IL10, IL17, IL35, IFNgamma, TNFalpha, TGF-β)
Measured: baseline and 52 weeks
Change in immunomodulation as assessed by cytokine concentrations between baseline and week 52
cytokine concentration will be investigated between baseline and week 52
ELISA Cytokines: IL2, IL4, IL6, IL10, IL17, IL35, IFNgamma, TNFalpha, TGF- β
Measured: baseline and 52 weeks
Clinical response between baseline and week 52: frequency of use of rescue treatment
Measured: baseline and week 52
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted3 May 2016 (estimated)
- Study startApr 2016
- Primary completionJul 2019
- Study completionMar 2020
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Immune ThrombocytopeniaStudies of the same condition, open and under way.
- More trials run by University Hospital, Basel, SwitzerlandThe sponsor's other registered studies.
- 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 4
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 15 participants (actual)
Sponsor and investigators
- Lead sponsor
- University Hospital, Basel, Switzerland
- Sponsor type
- University, hospital or other
- Collaborators
- University Children's Hospital Basel
- Responsible party
- Sponsor
- Investigators
- Thomas Kühne, Prof.Dr.med, Principal investigator, UKBB
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02760251
- Sponsor's protocol ID
- 20149180
- Acronym
- iROM
- Status last verified
- May 2020
- First submitted
- 15 Apr 2016
- Last updated
- 7 May 2020
- Results posted
- No
- Data monitoring committee
- No
- Individual data sharing
- No
Publications
Schifferli A, Rufer A, Rovo A, Nimmerjahn F, Cantoni N, Holbro A, Favre G, Dirks J, Wieland A, Faeth H, Pereira R, Kuhne T. Immunomodulation with romiplostim as a second-line strategy in primary immune thrombocytopenia: The iROM study. Br J Haematol. 2023 Oct;203(1):119-130. doi: 10.1111/bjh.19074.
PubMed 37735543
Study at a glance
From the registry record, last updated by the sponsor on 7 May 2020. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Thrombopoietin-receptor Agonist-immunomodulation in Young Adult Primary Immune Thrombocytopenia (ITP): A Multi-center Open Label Trial With Romiplostim
- ClinicalTrials.gov ID
- NCT02760251
- Acronym / sponsor ID
- iROM / 20149180
- Conditions
- Immune Thrombocytopenia
- Keywords
- immunomodulation
- Phase
- Phase 4
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 15 participants (actual)
- Lead sponsor
- University Hospital, Basel, SwitzerlandUniversity, hospital or other
- Collaborators
- University Children's Hospital Basel
- Study start
- Apr 2016
- Primary completion
- Jul 2019 (actual)
- Study completion
- Mar 2020 (actual)
- First posted
- 3 May 2016 (estimated)
- 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 thrombopoietin-receptor Agonist (TPO-RA) in patients with primary ITP, who failed first-line therapy or who became intolerant to it. It is hypothesized that the early phase of this autoimmune disease may exhibit a stronger immunomodulatory potential in response to a stimulus, such as romiplostim. Such a process may subsequently be capable to induce regulatory mechanisms or tolerance.
Romiplostim (a thrombopoietin-receptor agonist, TPO-RA) will be administered subcutaneously once weekly over 22 weeks with a starting dose of 1mcg/kg body weight. The dose will be adjusted based on platelet counts as described in the summary of Product Characteristics (SmPC).
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Romiplostim | Experimental | Drug: romiplostimRomiplostim (a thrombopoietin-receptor agonist, TPO-RA) will be administered subcutaneously once weekly over 22 weeks with a starting dose of 1mcg/kg body weight. The dose will be adjusted based on platelet counts as described in the summary of Product Characteristics (SmPC). Followup examination at week 52. |
Who may qualify
- Ages
- 18 to 45 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult
Key inclusion criteria (4 listed)
- Informed consent as documented by signature (see informed consent form)
- Primary ITP according to the definition of Rodeghiero et al. (52) and a platelet count of <30x109/l
- Age range: 18-45 years
- Previously treated patients, with failure or intolerance to first-line therapy, or relapse after first-line therapy, i.e. corticosteroids, intravenous immunoglobulin (IVIG), or anti-D immunoglobulins
Key exclusion criteria (21 listed)
- Adults older than 45 and children younger than 18 years
- Platelet count higher than 30x109/l at time of screening
- Suspicion of secondary ITP
- Positive family history for ITP
- Presence or history of autoimmune disease as judged by the investigator
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 |
|---|---|---|
| romiplostimAlso: Nplate | Drug | Romiplostim |
Primary outcome
Change in Interleukin (IL)-4 concentrations (pg/ml) from baseline to week 22
Time frame: baseline and 22 weeks
The primary aim of the study is to demonstrate an immunomodulatory effect of the study drug. Investigators expect a shift in the Th1/Th2 balance towards Th2. The primary outcome is to compare the pre- and post-treatment IL-4 concentrations (pg/ml) of all included patients (Th2 profile). Assessment of change in pre- and post-treatment IL-4 concentrations (pg/ml).
16 secondary outcomes
Change in immunomodulation as assessed by immune cell characteristics between baseline and week 22
Time frame: baseline and 22 weeks
Change in immunomodulation as assessed by immune cell characteristics between baseline and week 10
Time frame: baseline and 10 weeks
Change in immunomodulation as assessed by messenger ribonucleic acid (mRNA) of cytokines between baseline and week 22
Time frame: baseline and 22 weeks
Change in immunomodulation as assessed by mRNA of immune cells between baseline and week 22
Time frame: baseline and 22 weeks
Change in immunomodulation as assessed by mRNA of cytokines between baseline and week 10
Time frame: baseline and 10 weeks
Change in immunomodulation as assessed by mRNA of immune cells between baseline and week 10
Time frame: baseline and 10 weeks
Change in immunomodulation as assessed by cytokine concentrations between baseline and week 22
Time frame: baseline and 22 weeks
Change in immunomodulation as assessed by cytokine concentrations between baseline and week 10
Time frame: baseline and 10 weeks
Clinical response between baseline and week 52: number of severe bleeding
Time frame: baseline and 52 weeks
Clinical response between baseline and week 52: number of days in hospital
Time frame: baseline and 52 weeks
Clinical response between baseline and week 52: platelet more than >100G/l
Time frame: baseline and 52 weeks
Change in immunomodulation as assessed by immune cell characteristics between baseline and week 52
Time frame: baseline and 52 weeks
Change in immunomodulation as assessed by mRNA of immune cells between baseline and week 52
Time frame: baseline and 52 weeks
Change of immunomodulation as assessed by mRNA of cytokines between baseline and week 52
Time frame: baseline and 52 weeks
Change in immunomodulation as assessed by cytokine concentrations between baseline and week 52
Time frame: baseline and 52 weeks
Clinical response between baseline and week 52: frequency of use of rescue treatment
Time frame: baseline and week 52
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 7 May 2020.
- Derived from this study: PubMed 37735543Schifferli A, Rufer A, Rovo A, Nimmerjahn F, Cantoni N, Holbro A, Favre G, Dirks J, Wieland A, Faeth H, Pereira R, Kuhne T. Immunomodulation with romiplostim as a second-line strategy in primary immune thrombocytopenia: The iROM study. Br J Haematol. 2023 Oct;203(1):119-130. doi: 10.1111/bjh.19074.
- Individual participant dataSharing planned: No
Related trials for Immune Thrombocytopenia
RecruitingPhase 2
A Study to Assess the Tolerability of Ianalumab (VAY736) With Investigator's Choice Thrombopoietin Receptor Agonist (IC TPO-RA) in Patients With Primary Immune Thrombocytopenia (ITP)
Primary Immune Thrombocytopenia (ITP), Primary Evans Syndrome (ES)
RecruitingPhase 1/2
A Study of Pirtobrutinib in Participants With Immune Thrombocytopenia
Immune Thrombocytopenia (ITP)
RecruitingPhase 3
A Study of Mezagitamab in Adults With Chronic Primary Immune Thrombocytopenia
Immune Thrombocytopenic Purpura (ITP)
RecruitingPhase 2
Study to Assess the Safety and Tolerability of Tafasitamab in Adult Participants With Primary Autoimmune Blood Cell Disorders
Immune Thrombocytopenia
RecruitingPhase 3
A Follow-up Study of Mezagitamab in Adults With Chronic Primary Immune Thrombocytopenia
Immune Thrombocytopenic Purpura (ITP)
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.
Thomas Kühne, Prof.Dr.med principal investigator
UKBB
5 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 4 study testing romiplostim in people with Immune Thrombocytopenia, run by University Hospital, Basel, Switzerland at 5 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 21 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 7 May 2020. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 5 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 4 studies take place after a treatment is approved, to learn more about its long-term benefits and risks in everyday use.
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 Interleukin (IL)-4 concentrations (pg/ml) from baseline to week 22 (time frame: baseline and 22 weeks). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT02760251, which also lists 1 linked publication.
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 7 May 2020.