Clinical trial NCT05049668: RACE 2: a Long Term Follow-up of Patients Participating in the RACE Trial
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
Study type: OBSERVATIONAL
Overall status: ENROLLING_BY_INVITATION
Conditions: Severe Aplastic Anemia
Lead sponsor: European Society for Blood and Marrow Transplantation
Sponsor class: NETWORK
First posted: 2021-09-20
Enrollment: 197
Standard ages: CHILD
ADULT
OLDER_ADULT
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Patients who participated in the initial RACE 1 trial

Brief summary: 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.

Eligibility criteria: 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

Primary outcomes:
measure: Failure Free Survival
timeFrame: 15 years
Secondary outcomes:
measure: Response Rate: number of patients who reach a hematological response
timeFrame: 15 years
measure: Overall Survival
timeFrame: 15 years
measure: Cumulative incidence of relapse after response
timeFrame: 15 years
measure: Cumulative incidence of clonal evolution
timeFrame: 15 years
measure: Cumulative incidence of clinical Paroxysmal nocturnal hemoglobinuria (PNH)
timeFrame: 15 years
measure: Cumulative incidence of Solid Tumors
timeFrame: 15 years
measure: Number of patients who need a Human Stem Cell Transplantation (HSCT)
timeFrame: 15 years
measure: Number of patients who need additional IST
timeFrame: 15 years
measure: Number of patients who need Maintenance IST (e.g. CsA beyond 2 years)
timeFrame: 15 years
measure: Number of patients who need additional Eltrombopag (EPAG)
timeFrame: 15 years
measure: Number of patients who need any other approved Aplastic Anemia (AA) treatment
timeFrame: 15 years
measure: Monitoring of Clonal Hematopoiesis of Indetermined Potential (CHIP)
timeFrame: 15 years
Other outcomes:
Locations:
city: Paris
country: France
facility: Hospital St. Louis
Study officials:
affiliation: Hospital St. Louis, Paris, France
name: Regis Peffault de Latour, Prof, MD
role: PRINCIPAL_INVESTIGATOR
affiliation: Hospital Avellino, Napels, Italy
name: Antonio M Risitano, MD, PhD
role: PRINCIPAL_INVESTIGATOR
References: