RACE 2: a Long Term Follow-up of Patients Participating in the RACE Trial
For patients and families
In plain language
An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.
- What is being studied
- The protocol lists: ATGAM plus CsA with or without Eltrombopag.
- Who it may be relevant to
- Registry conditions: Severe Aplastic Anemia. Basic parameters: No limits · All.
- What needs checking
- Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
- Where it takes place
- France
- Next step
- Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →
Unsure about the terms? Read our patient guide →
Official 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
Overview
After exiting the RACE trial (NCT02099747) patients will be invited to participate in this long term follow-up study
Detailed description
Patients will be followed up annually, according to standard of care.
All 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.
Molecular analysis by Next Generation Sequencing (NGS) will also be collected if the centre is doing this on a routine basis.
No Investigational Medicinal Product (IMP) or Non-Investigational Medicinal Product (NIMP) will be given to the patients.
Interventions
- Drug ATGAM plus CsA with or without Eltrombopag
Standard treatment with or without Eltrombopag
Primary outcome measures
- Failure Free Survival [Time frame: 15 years]
Secondary outcome measures (12)
- Response Rate: number of patients who reach a hematological response [Time frame: 15 years]
- Overall Survival [Time frame: 15 years]
- Cumulative incidence of relapse after response [Time frame: 15 years]
- Cumulative incidence of clonal evolution [Time frame: 15 years]
- Cumulative incidence of clinical Paroxysmal nocturnal hemoglobinuria (PNH) [Time frame: 15 years]
- Cumulative incidence of Solid Tumors [Time frame: 15 years]
- Number of patients who need a Human Stem Cell Transplantation (HSCT) [Time frame: 15 years]
- Number of patients who need additional IST [Time frame: 15 years]
- Number of patients who need Maintenance IST (e.g. CsA beyond 2 years) [Time frame: 15 years]
- Number of patients who need additional Eltrombopag (EPAG) [Time frame: 15 years]
- Number of patients who need any other approved Aplastic Anemia (AA) treatment [Time frame: 15 years]
- Monitoring of Clonal Hematopoiesis of Indetermined Potential (CHIP) [Time frame: 15 years]
Eligibility criteria
Inclusion criteria
- Subject participated in the RACE trial (NCT02099747, EudraCT number: 2014-000363-40) during which patient received ATGAM, Cyclosporine A with or without Eltrombopag.
- Subject has provided informed consent to participate in long-term data collection
Exclusion criteria
None
Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.
Study design
- Observational model
- Cohort
Study locations
France · 1 center
- Hospital St. Louis — Paris
Identifiers
NCT: NCT05049668 · EBMT-RACE 2