Severe Aplastic Anemia Clinical Trial
— RACE2Official title:
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
After exiting the RACE trial (NCT02099747) patients will be invited to participate in this long term follow-up study
Status | Enrolling by invitation |
Enrollment | 197 |
Est. completion date | December 2034 |
Est. primary completion date | December 2033 |
Accepts healthy volunteers | |
Gender | All |
Age group | N/A and older |
Eligibility | Inclusion Criteria: 1. Subject participated in the RACE trial (NCT02099747, EudraCT number: 2014-000363-40) during which patient received ATGAM, Cyclosporine A with or without Eltrombopag. 2. Subject has provided informed consent to participate in long-term data collection Exclusion Criteria: None |
Country | Name | City | State |
---|---|---|---|
France | Hospital St. Louis | Paris |
Lead Sponsor | Collaborator |
---|---|
European Society for Blood and Marrow Transplantation |
France,
Type | Measure | Description | Time frame | Safety issue |
---|---|---|---|---|
Primary | Failure Free Survival | 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) | 15 years | |
Secondary | Response Rate: number of patients who reach a hematological response | Response Rate | 15 years | |
Secondary | Overall Survival | Overall Survival | 15 years | |
Secondary | Cumulative incidence of relapse after response | Cumulative incidence of relapse after initial hematological response (complete or partial) | 15 years | |
Secondary | Cumulative incidence of clonal evolution | 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) | 15 years | |
Secondary | Cumulative incidence of clinical Paroxysmal nocturnal hemoglobinuria (PNH) | Cumulative incidence of clinical PNH (hemolysis and/or thromboembolism), and of need of anti-complement treatment | 15 years | |
Secondary | Cumulative incidence of Solid Tumors | Cumulative incidence of solid tumours | 15 years | |
Secondary | Number of patients who need a Human Stem Cell Transplantation (HSCT) | Need for HSCT | 15 years | |
Secondary | Number of patients who need additional IST | Need for additional intensive Imune Suppressive Therapy (IST) (e.g. ATG, alemtuzumab or cyclophosphamide-based, or any other lymphocyte-depleting agent) | 15 years | |
Secondary | Number of patients who need Maintenance IST (e.g. CsA beyond 2 years) | Need for maintenance intensive IST (e.g. CsA beyond 2 years) | 15 years | |
Secondary | Number of patients who need additional Eltrombopag (EPAG) | Need for additional EPAG | 15 years | |
Secondary | Number of patients who need any other approved Aplastic Anemia (AA) treatment | Need for any other approved Aplastic Anemia (AA) treatment | 15 years | |
Secondary | Monitoring of Clonal Hematopoiesis of Indetermined Potential (CHIP) | As tracked by somatic mutations in genes associated with myeloid disorders | 15 years |
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