Ignite Creation Date:
2025-12-24 @ 11:40 PM
Ignite Modification Date:
2026-03-12 @ 4:33 AM
Study NCT ID:
NCT02760251
Status:
COMPLETED
Last Update Posted:
2020-05-07
First Post:
2016-04-15
Is NOT Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
Immunomodulation With Romiplostim in Young Adults With ITP
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D016553', 'term': 'Purpura, Thrombocytopenic, Idiopathic'}], 'ancestors': [{'id': 'D011696', 'term': 'Purpura, Thrombocytopenic'}, {'id': 'D011693', 'term': 'Purpura'}, {'id': 'D001778', 'term': 'Blood Coagulation Disorders'}, {'id': 'D006402', 'term': 'Hematologic Diseases'}, {'id': 'D006425', 'term': 'Hemic and Lymphatic Diseases'}, {'id': 'D057049', 'term': 'Thrombotic Microangiopathies'}, {'id': 'D013921', 'term': 'Thrombocytopenia'}, {'id': 'D001791', 'term': 'Blood Platelet Disorders'}, {'id': 'D000095542', 'term': 'Cytopenia'}, {'id': 'D006474', 'term': 'Hemorrhagic Disorders'}, {'id': 'D001327', 'term': 'Autoimmune Diseases'}, {'id': 'D007154', 'term': 'Immune System Diseases'}, {'id': 'D006470', 'term': 'Hemorrhage'}, {'id': 'D010335', 'term': 'Pathologic Processes'}, {'id': 'D013568', 'term': 'Pathological Conditions, Signs and Symptoms'}, {'id': 'D012877', 'term': 'Skin Manifestations'}, {'id': 'D012816', 'term': 'Signs and Symptoms'}]}, 'interventionBrowseModule': {'meshes': [{'id': 'C488777', 'term': 'romiplostim'}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE4'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'NA', 'maskingInfo': {'masking': 'NONE'}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'SINGLE_GROUP'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 15}}, 'statusModule': {'overallStatus': 'COMPLETED', 'startDateStruct': {'date': '2016-04'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2020-05', 'completionDateStruct': {'date': '2020-03', 'type': 'ACTUAL'}, 'lastUpdateSubmitDate': '2020-05-06', 'studyFirstSubmitDate': '2016-04-15', 'studyFirstSubmitQcDate': '2016-04-28', 'lastUpdatePostDateStruct': {'date': '2020-05-07', 'type': 'ACTUAL'}, 'studyFirstPostDateStruct': {'date': '2016-05-03', 'type': 'ESTIMATED'}, 'primaryCompletionDateStruct': {'date': '2019-07', 'type': 'ACTUAL'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Change in Interleukin (IL)-4 concentrations (pg/ml) from baseline to week 22', 'timeFrame': 'baseline and 22 weeks', 'description': '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.\n\nThe 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).'}], 'secondaryOutcomes': [{'measure': 'Change in immunomodulation as assessed by immune cell characteristics between baseline and week 22', 'timeFrame': 'baseline and 22 weeks', 'description': 'Immunologic parameters will be investigated between baseline and week 22 of the study: immune cell characteristics\n\nfluorescence-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+'}, {'measure': 'Change in immunomodulation as assessed by immune cell characteristics between baseline and week 10', 'timeFrame': 'baseline and 10 weeks', 'description': 'Immunologic parameters will be investigated between baseline and week 10 of the study: immune cell characteristics\n\nFACS:\n\nB 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+'}, {'measure': 'Change in immunomodulation as assessed by messenger ribonucleic acid (mRNA) of cytokines between baseline and week 22', 'timeFrame': 'baseline and 22 weeks', 'description': 'mRNA of cytokines will be investigated between baseline and week 22\n\nmRNA 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)'}, {'measure': 'Change in immunomodulation as assessed by mRNA of immune cells between baseline and week 22', 'timeFrame': 'baseline and 22 weeks', 'description': 'mRNA of immune cells will be investigated between baseline and week 22\n\nmRNA essays (real-time quantitative PCR): mRNA Th1 (T-bet), Th2 (GATA-3), Th17 (RORγt), Tregs (Foxp3)'}, {'measure': 'Change in immunomodulation as assessed by mRNA of cytokines between baseline and week 10', 'timeFrame': 'baseline and 10 weeks', 'description': 'mRNA of cytokines will be investigated between baseline and week 10\n\nmRNA essays (real-time quantitative PCR): cytokine mRNA (IL2, IL4, IL6, IL10, IL17, IL35, IFNgamma, TNFalpha, TGF-β)'}, {'measure': 'Change in immunomodulation as assessed by mRNA of immune cells between baseline and week 10', 'timeFrame': 'baseline and 10 weeks', 'description': 'mRNA of immune cells will be investigated between baseline and week 10\n\nmRNA essays (real-time quantitative PCR): mRNA Th1 (T-bet), Th2 (GATA-3), Th17 (RORγt), Tregs (Foxp3)'}, {'measure': 'Change in immunomodulation as assessed by cytokine concentrations between baseline and week 22', 'timeFrame': 'baseline and 22 weeks', 'description': 'cytokine concentration will be investigated between baseline and week 22\n\nELISA Cytokines: IL2, IL4, IL6, IL10, IL17, IL35, IFNgamma, TNFalpha, TGF- β'}, {'measure': 'Change in immunomodulation as assessed by cytokine concentrations between baseline and week 10', 'timeFrame': 'baseline and 10 weeks', 'description': 'cytokine concentration will be investigated between baseline and week 10\n\nELISA Cytokines: IL2, IL4, IL6, IL10, IL17, IL35, IFNgamma, TNFalpha, TGF- β'}, {'measure': 'Clinical response between baseline and week 52: number of severe bleeding', 'timeFrame': 'baseline and 52 weeks', 'description': 'Clinical characterization of response to romiplostim therapy will be assessed additionally : measurement of severe bleeding (score Bolton-Maggs)'}, {'measure': 'Clinical response between baseline and week 52: number of days in hospital', 'timeFrame': 'baseline and 52 weeks', 'description': 'Clinical characterization of response to romiplostim therapy will be assessed additionally : need of inpatient daycare'}, {'measure': 'Clinical response between baseline and week 52: platelet more than >100G/l', 'timeFrame': 'baseline and 52 weeks', 'description': '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).'}, {'measure': 'Change in immunomodulation as assessed by immune cell characteristics between baseline and week 52', 'timeFrame': 'baseline and 52 weeks', 'description': 'Immunologic parameters will be investigated between baseline and week 52 of the study: immune cell characteristics\n\nFACS:\n\nB 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+'}, {'measure': 'Change in immunomodulation as assessed by mRNA of immune cells between baseline and week 52', 'timeFrame': 'baseline and 52 weeks', 'description': 'mRNA of immune cells will be investigated between baseline and week 52\n\nmRNA essays (real-time quantitative PCR): mRNA Th1 (T-bet), Th2 (GATA-3), Th17 (RORγt), Tregs (Foxp3)'}, {'measure': 'Change of immunomodulation as assessed by mRNA of cytokines between baseline and week 52', 'timeFrame': 'baseline and 52 weeks', 'description': 'mRNA of cytokines will be investigated between baseline and week 52\n\nmRNA essays (real-time quantitative PCR): cytokine mRNA (IL2, IL4, IL6, IL10, IL17, IL35, IFNgamma, TNFalpha, TGF-β)'}, {'measure': 'Change in immunomodulation as assessed by cytokine concentrations between baseline and week 52', 'timeFrame': 'baseline and 52 weeks', 'description': 'cytokine concentration will be investigated between baseline and week 52\n\nELISA Cytokines: IL2, IL4, IL6, IL10, IL17, IL35, IFNgamma, TNFalpha, TGF- β'}, {'measure': 'Clinical response between baseline and week 52: frequency of use of rescue treatment', 'timeFrame': 'baseline and week 52'}]}, 'oversightModule': {'oversightHasDmc': False}, 'conditionsModule': {'keywords': ['immunomodulation'], 'conditions': ['Immune Thrombocytopenia']}, 'referencesModule': {'references': [{'pmid': '37735543', 'type': 'DERIVED', 'citation': '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.'}]}, 'descriptionModule': {'briefSummary': '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.\n\nRomiplostim (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).'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['ADULT'], 'maximumAge': '45 Years', 'minimumAge': '18 Years', 'healthyVolunteers': False, 'eligibilityCriteria': 'Inclusion Criteria:\n\n* Informed consent as documented by signature (see informed consent form)\n* Primary ITP according to the definition of Rodeghiero et al. (52) and a platelet count of \\<30x109/l\n* Age range: 18-45 years\n* 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\n\nExclusion Criteria:\n\n* Adults older than 45 and children younger than 18 years\n* Platelet count higher than 30x109/l at time of screening\n* Suspicion of secondary ITP\n* Positive family history for ITP\n* Presence or history of autoimmune disease as judged by the investigator\n* Hepatosplenomegaly\n* Presence or history of relevant hepatic disease as judged by the investigator\n* Presence or history of thromboembolic disease as judged by the investigator\n* Patients with splenectomy\n* Women who are pregnant or breast feeding\n* Intention to become pregnant during the course of the study\n* Lack of safe double contraception (see 7.1)\n* Any vaccination 2 weeks prior start of the study\n* 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\n* Known or suspected non-compliance, drug or alcohol abuse\n* Inability to follow the procedures of the study, e.g. due to language problems, psychological disorders, dementia of the study subject\n* Participation in another study with investigational drug within the 30 days preceding and during the present study\n* Previous enrolment into the current study\n* Previous treatment with romiplostim or eltrombopag\n* Hypersensitivity to the active substance or to any of the excipients or to E. coli derived proteins\n* Enrolment of the investigator, his/her family members, employees and other dependent persons'}, 'identificationModule': {'nctId': 'NCT02760251', 'acronym': 'iROM', 'briefTitle': 'Immunomodulation With Romiplostim in Young Adults With ITP', 'organization': {'class': 'OTHER', 'fullName': 'University Hospital, Basel, Switzerland'}, 'officialTitle': 'Thrombopoietin-receptor Agonist-immunomodulation in Young Adult Primary Immune Thrombocytopenia (ITP): A Multi-center Open Label Trial With Romiplostim', 'orgStudyIdInfo': {'id': '20149180'}}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'Romiplostim', 'description': '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.', 'interventionNames': ['Drug: romiplostim']}], 'interventions': [{'name': 'romiplostim', 'type': 'DRUG', 'otherNames': ['Nplate'], 'armGroupLabels': ['Romiplostim']}]}, 'contactsLocationsModule': {'locations': [{'city': 'Liestal', 'state': 'Basel-Landschaft', 'country': 'Switzerland', 'facility': 'Liestal Cantonal Hospital', 'geoPoint': {'lat': 47.48455, 'lon': 7.73446}}, {'city': 'Lucerne', 'state': 'Canton of Lucerne', 'country': 'Switzerland', 'facility': 'Lucerne Cantonal Hospital', 'geoPoint': {'lat': 47.05048, 'lon': 8.30635}}, {'city': 'Aarau', 'country': 'Switzerland', 'facility': 'Aarau Cantonal Hospital', 'geoPoint': {'lat': 47.39254, 'lon': 8.04422}}, {'zip': '4000', 'city': 'Basel', 'country': 'Switzerland', 'facility': 'University Hospital Basel', 'geoPoint': {'lat': 47.55839, 'lon': 7.57327}}, {'city': 'Bern', 'country': 'Switzerland', 'facility': 'University Hospital Bern', 'geoPoint': {'lat': 46.94809, 'lon': 7.44744}}], 'overallOfficials': [{'name': 'Thomas Kühne, Prof.Dr.med', 'role': 'PRINCIPAL_INVESTIGATOR', 'affiliation': 'UKBB'}]}, 'ipdSharingStatementModule': {'ipdSharing': 'NO'}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'University Hospital, Basel, Switzerland', 'class': 'OTHER'}, 'collaborators': [{'name': "University Children's Hospital Basel", 'class': 'OTHER'}], 'responsibleParty': {'type': 'SPONSOR'}}}}