Ignite Creation Date:
2025-12-24 @ 5:36 PM
Ignite Modification Date:
2026-02-23 @ 11:49 AM
Study NCT ID:
NCT05049668
Status:
ENROLLING_BY_INVITATION
Last Update Posted:
2021-09-27
First Post:
2021-09-13
Is Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
RACE 2: a Long Term Follow-up of Patients Participating in the RACE Trial
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': 'D000961', 'term': 'Antilymphocyte Serum'}, {'id': 'C520809', 'term': 'eltrombopag'}], 'ancestors': [{'id': 'D007106', 'term': 'Immune Sera'}, {'id': 'D000906', 'term': 'Antibodies'}, {'id': 'D007136', 'term': 'Immunoglobulins'}, {'id': 'D007162', 'term': 'Immunoproteins'}, {'id': 'D001798', 'term': 'Blood Proteins'}, {'id': 'D011506', 'term': 'Proteins'}, {'id': 'D000602', 'term': 'Amino Acids, Peptides, and Proteins'}, {'id': 'D012712', 'term': 'Serum Globulins'}, {'id': 'D005916', 'term': 'Globulins'}, {'id': 'D001688', 'term': 'Biological Products'}, {'id': 'D045424', 'term': 'Complex Mixtures'}]}}, 'protocolSection': {'designModule': {'studyType': 'OBSERVATIONAL', 'designInfo': {'timePerspective': 'PROSPECTIVE', 'observationalModel': 'COHORT'}, 'enrollmentInfo': {'type': 'ESTIMATED', 'count': 197}, 'targetDuration': '15 Years', 'patientRegistry': True}, 'statusModule': {'overallStatus': 'ENROLLING_BY_INVITATION', 'startDateStruct': {'date': '2021-10', 'type': 'ESTIMATED'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2021-09', 'completionDateStruct': {'date': '2034-12', 'type': 'ESTIMATED'}, 'lastUpdateSubmitDate': '2021-09-21', 'studyFirstSubmitDate': '2021-09-13', 'studyFirstSubmitQcDate': '2021-09-17', 'lastUpdatePostDateStruct': {'date': '2021-09-27', 'type': 'ACTUAL'}, 'studyFirstPostDateStruct': {'date': '2021-09-20', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2033-12', 'type': 'ESTIMATED'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Failure Free Survival', 'timeFrame': '15 years', 'description': 'Failure Free Survival, where treatment failure is defined as one or more of the following: death, relapse, malignant clonal evolution, need for further (e.g. transplant)'}], 'secondaryOutcomes': [{'measure': 'Response Rate: number of patients who reach a hematological response', 'timeFrame': '15 years', 'description': 'Response Rate'}, {'measure': 'Overall Survival', 'timeFrame': '15 years', 'description': 'Overall Survival'}, {'measure': 'Cumulative incidence of relapse after response', 'timeFrame': '15 years', 'description': 'Cumulative incidence of relapse after initial hematological response (complete or partial)'}, {'measure': 'Cumulative incidence of clonal evolution', 'timeFrame': '15 years', 'description': 'Cumulative incidence of clonal evolution: Acute Myeloid Leukemia (AML), Myelodysplastic Syndrome (MDS) or karyotypic abnormalities qualifying for the diagnosis of MDS (see World Health Organization (WHO) 2016)'}, {'measure': 'Cumulative incidence of clinical Paroxysmal nocturnal hemoglobinuria (PNH)', 'timeFrame': '15 years', 'description': 'Cumulative incidence of clinical PNH (hemolysis and/or thromboembolism), and of need of anti-complement treatment'}, {'measure': 'Cumulative incidence of Solid Tumors', 'timeFrame': '15 years', 'description': 'Cumulative incidence of solid tumours'}, {'measure': 'Number of patients who need a Human Stem Cell Transplantation (HSCT)', 'timeFrame': '15 years', 'description': 'Need for HSCT'}, {'measure': 'Number of patients who need additional IST', 'timeFrame': '15 years', 'description': 'Need for additional intensive Imune Suppressive Therapy (IST) (e.g. ATG, alemtuzumab or cyclophosphamide-based, or any other lymphocyte-depleting agent)'}, {'measure': 'Number of patients who need Maintenance IST (e.g. CsA beyond 2 years)', 'timeFrame': '15 years', 'description': 'Need for maintenance intensive IST (e.g. CsA beyond 2 years)'}, {'measure': 'Number of patients who need additional Eltrombopag (EPAG)', 'timeFrame': '15 years', 'description': 'Need for additional EPAG'}, {'measure': 'Number of patients who need any other approved Aplastic Anemia (AA) treatment', 'timeFrame': '15 years', 'description': 'Need for any other approved Aplastic Anemia (AA) treatment'}, {'measure': 'Monitoring of Clonal Hematopoiesis of Indetermined Potential (CHIP)', 'timeFrame': '15 years', 'description': 'As tracked by somatic mutations in genes associated with myeloid disorders'}]}, 'oversightModule': {'isUsExport': False, 'oversightHasDmc': False, 'isFdaRegulatedDrug': True, 'isFdaRegulatedDevice': False}, 'conditionsModule': {'conditions': ['Severe Aplastic Anemia']}, 'descriptionModule': {'briefSummary': 'After exiting the RACE trial (NCT02099747) patients will be invited to participate in this long term follow-up study', 'detailedDescription': 'Patients will be followed up annually, according to standard of care.\n\nAll diagnostic and therapeutic intervention will be performed according to standard of care, at discretion of the treating physician. In particular, during the study no extra Peripheral blood or Bone Marrow sampling will be performed, in addition to routine sampling for morphology and karyotype surveillance.\n\nMolecular analysis by Next Generation Sequencing (NGS) will also be collected if the centre is doing this on a routine basis.\n\nNo Investigational Medicinal Product (IMP) or Non-Investigational Medicinal Product (NIMP) will be given to the patients.'}, 'eligibilityModule': {'stdAges': ['CHILD', 'ADULT', 'OLDER_ADULT'], 'samplingMethod': 'NON_PROBABILITY_SAMPLE', 'studyPopulation': 'Patients who participated in the initial RACE 1 trial', 'eligibilityCriteria': 'Inclusion Criteria:\n\n1. Subject participated in the RACE trial (NCT02099747, EudraCT number: 2014-000363-40) during which patient received ATGAM, Cyclosporine A with or without Eltrombopag.\n2. Subject has provided informed consent to participate in long-term data collection\n\nExclusion Criteria:\n\nNone'}, 'identificationModule': {'nctId': 'NCT05049668', 'acronym': 'RACE2', 'briefTitle': 'RACE 2: a Long Term Follow-up of Patients Participating in the RACE Trial', 'organization': {'class': 'NETWORK', 'fullName': 'European Society for Blood and Marrow Transplantation'}, 'officialTitle': 'Long-term Follow-up of Patients Participating in RACE: the Prospective Randomized Multicenter Study Comparing Horse Antithymocyte Globuline (hATG) + Cyclosporine A (CsA) With or Without Eltrombopag as Front-line Therapy for Severe Aplastic Anemia Patients', 'orgStudyIdInfo': {'id': 'EBMT-RACE 2'}}, 'armsInterventionsModule': {'armGroups': [{'label': 'RACE 1 patients', 'description': 'After exiting the RACE trial (NCT02099747) patient will be invited to participate in this study', 'interventionNames': ['Drug: ATGAM plus CsA with or without Eltrombopag']}], 'interventions': [{'name': 'ATGAM plus CsA with or without Eltrombopag', 'type': 'DRUG', 'otherNames': ['hATG+CsA +/- Revolade'], 'description': 'Standard treatment with or without Eltrombopag', 'armGroupLabels': ['RACE 1 patients']}]}, 'contactsLocationsModule': {'locations': [{'city': 'Paris', 'country': 'France', 'facility': 'Hospital St. Louis', 'geoPoint': {'lat': 48.85341, 'lon': 2.3488}}], 'overallOfficials': [{'name': 'Regis Peffault de Latour, Prof, MD', 'role': 'PRINCIPAL_INVESTIGATOR', 'affiliation': 'Hospital St. Louis, Paris, France'}, {'name': 'Antonio M Risitano, MD, PhD', 'role': 'PRINCIPAL_INVESTIGATOR', 'affiliation': 'Hospital Avellino, Napels, Italy'}]}, 'ipdSharingStatementModule': {'ipdSharing': 'NO'}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'European Society for Blood and Marrow Transplantation', 'class': 'NETWORK'}, 'responsibleParty': {'type': 'SPONSOR'}}}}