RACE 2: a Long Term Follow-up of Patients Participating in the RACE Trial

N/AEnrolling By InvitationNCT05049668

European Society for Blood and Marrow Transplantation197 enrolled

Overview

After exiting the RACE trial (NCT02099747) patients will be invited to participate in this long term follow-up study

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.

Study Type

OBSERVATIONAL

Enrollment

197

Conditions

Severe Aplastic Anemia

Interventions

ATGAM plus CsA with or without EltrombopagDRUG

Standard treatment with or without Eltrombopag

Eligibility

Medical Language ↔ Plain English

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

Locations (1)

Hospital St. Louis

Paris, France

Outcomes

Primary Outcomes

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)

Time frame: 15 years

Secondary Outcomes

Response Rate: number of patients who reach a hematological response

Response Rate

Time frame: 15 years

Overall Survival

Time frame: 15 years

Cumulative incidence of relapse after response

Cumulative incidence of relapse after initial hematological response (complete or partial)

Time frame: 15 years

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)

Time frame: 15 years

Cumulative incidence of clinical Paroxysmal nocturnal hemoglobinuria (PNH)

Cumulative incidence of clinical PNH (hemolysis and/or thromboembolism), and of need of anti-complement treatment

Time frame: 15 years

Cumulative incidence of Solid Tumors

Cumulative incidence of solid tumours

Time frame: 15 years

Number of patients who need a Human Stem Cell Transplantation (HSCT)

Data from ClinicalTrials.gov

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