Ignite Creation Date:
2025-12-25 @ 1:39 AM
Ignite Modification Date:
2026-01-07 @ 1:20 AM
Study NCT ID:
NCT03957694
Status:
COMPLETED
Last Update Posted:
2022-05-26
First Post:
2019-05-09
Is NOT Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
Study of AMG531(Romiplostim) in Patients With Aplastic Anemia
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D000741', 'term': 'Anemia, Aplastic'}], 'ancestors': [{'id': 'D000740', 'term': 'Anemia'}, {'id': 'D006402', 'term': 'Hematologic Diseases'}, {'id': 'D006425', 'term': 'Hemic and Lymphatic Diseases'}, {'id': 'D000080983', 'term': 'Bone Marrow Failure Disorders'}, {'id': 'D001855', 'term': 'Bone Marrow Diseases'}]}, 'interventionBrowseModule': {'meshes': [{'id': 'C488777', 'term': 'romiplostim'}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE2', 'PHASE3'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'NA', 'maskingInfo': {'masking': 'NONE'}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'SINGLE_GROUP', 'interventionModelDescription': 'multi-national, open-label, phase 2/3 study'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 17}}, 'statusModule': {'overallStatus': 'COMPLETED', 'startDateStruct': {'date': '2019-04-25', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2022-05', 'completionDateStruct': {'date': '2021-05-26', 'type': 'ACTUAL'}, 'lastUpdateSubmitDate': '2022-05-25', 'studyFirstSubmitDate': '2019-05-09', 'studyFirstSubmitQcDate': '2019-05-19', 'lastUpdatePostDateStruct': {'date': '2022-05-26', 'type': 'ACTUAL'}, 'studyFirstPostDateStruct': {'date': '2019-05-21', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2020-11-05', 'type': 'ACTUAL'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Achievement of complete response (CR) or partial response (PR)', 'timeFrame': '27 weeks post-dose'}], 'secondaryOutcomes': [{'measure': 'Achievement of CR or PR', 'timeFrame': 'Week 14'}, {'measure': 'Achievement of CR', 'timeFrame': 'Weeks 14 and 27'}, {'measure': 'The time to CR or PR', 'timeFrame': 'Each time point evaluated weekly until Week 27'}, {'measure': 'Reduction or independence of platelet and/or erythrocyte transfusion', 'timeFrame': 'Week 27'}, {'measure': 'Change from baseline in platelet count (/µL)', 'timeFrame': 'Each time point evaluated weekly until Week 27'}, {'measure': 'Change from baseline in hemoglobin (Hb) concentration (g/dL)', 'timeFrame': 'Each time point evaluated weekly until Week 27'}, {'measure': 'Change from baseline in neutrophil count (/µL)', 'timeFrame': 'Each time point evaluated weekly until Week 27'}, {'measure': 'Change from baseline in reticulocyte count (/µL)', 'timeFrame': 'Each time point evaluated weekly until Week 27'}]}, 'oversightModule': {'oversightHasDmc': False, 'isFdaRegulatedDrug': False, 'isFdaRegulatedDevice': False}, 'conditionsModule': {'keywords': ['aplastic anemia', 'romiplostim'], 'conditions': ['Aplastic Anemia']}, 'descriptionModule': {'briefSummary': 'To evaluate the hematological responses based on the response assessment criteria when AMG531 is subcutaneous (SC)-administered with anti-human thymocyte immunoglobulin (ATG) + ciclosporin A (CsA) therapy for 6 months in patients with aplastic anemia (AA) who were previously untreated with immunosuppressive therapy.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['ADULT', 'OLDER_ADULT'], 'minimumAge': '20 Years', 'healthyVolunteers': False, 'eligibilityCriteria': 'Inclusion Criteria:\n\n1. Voluntary signed informed consent to participate in the study;\n2. A diagnosis of AA confirmed by blood and bone-marrow examinations, etc.;\n3. Considered to require new treatment with ATG and CsA provided that NSAA must be platelet or erythrocyte transfusion-dependent.\n4. An Eastern Cooperative Oncology Group (ECOG) performance status (PS) score of 0 to 1at screening\n\nExclusion Criteria:\n\n1. Previously treated with ATG, CsA, or Alemtuzumab;\n2. Diagnosed as having congenital AA (Fanconi anemia, congenital dyskeratosis, etc.);\n3. Diagnosed as having AML or chronic myelomonocytic leukemia;\n4. Concurrent thrombocytopenia of other etiologies (e.g., MDS, ITP, cirrhosis);\n5. Concurrent active infection not adequately responding to appropriate therapy;\n6. Concurrent clinically significant illness(es) items which are deemed by the Investigator to be likely to affect the study conduct and assessments.\n7. Having active malignancies, or having a history of treatment of malignancies within 5 years prior to informed consent.\n8. Concurrent PNH\n9. Having Grade 2 or higher bone marrow reticulin based on Bone Marrow Pathology (2nd edition) ;\n10. History of chromosome aberrations discovered in bone marrow cells.\n11. Having blast cells \\> 2% in bone marrow;\n12. Positive for anti-human immunodeficiency virus (HIV) antibody;\n13. Receiving prophylactic or therapeutic treatment for hepatitis type B\n14. Positive for hepatitis C virus (HCV) antibody, and HCV infection being confirmed\n15. Planned hematopoietic stem cell transplantation during the study;\n16. Systemic treatment with any of the following medication for the treatment of AA within 4 weeks before Day 1, however, excluding their use as premedication:\n\n * Anabolic steroids\n * Corticosteroids;\n17. Pregnant or breastfeeding women, or women willing to become pregnant;\n18. Other conditions unsuitable for participation in the study in the opinion of the Investigator.'}, 'identificationModule': {'nctId': 'NCT03957694', 'briefTitle': 'Study of AMG531(Romiplostim) in Patients With Aplastic Anemia', 'organization': {'class': 'INDUSTRY', 'fullName': 'Kyowa Kirin Co., Ltd.'}, 'officialTitle': 'A Phase 2/3 Study of AMG531 in Patients With Aplastic Anemia PreviouslyUntreated With Immunosuppressive Therapy', 'orgStudyIdInfo': {'id': '531-003'}}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'AMG531', 'interventionNames': ['Drug: Romiplostim']}], 'interventions': [{'name': 'Romiplostim', 'type': 'DRUG', 'description': 'Romiplostim SC. Initial dose is 10 ug/kg/. Maximum dose is 20 ug/kg. Original duration is 6 months from first administration but if investigator decide that the patient is needed more treatment, they move to extention period and take treatment up to 1 year.', 'armGroupLabels': ['AMG531']}]}, 'contactsLocationsModule': {'locations': [{'city': 'Ehime', 'country': 'Japan', 'facility': 'Matsuyama Red Cross Hospital'}]}, 'ipdSharingStatementModule': {'ipdSharing': 'UNDECIDED'}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Kyowa Kirin Co., Ltd.', 'class': 'INDUSTRY'}, 'responsibleParty': {'type': 'SPONSOR'}}}}