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

• Conditions: Severe Aplastic Anemia
• Interventions: Drug: ATGAM plus CsA with or without Eltrombopag

• Registration Number: NCT05049668
• Lead Sponsor: European Society for Blood and Marrow Transplantation

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.

Study Timeline

• Status Verified: 2021-09
• Study First Submitted: 2021-09-13
• Study First Submitted QC: 2021-09-17
• Study First Posted: 2021-09-20
• Last Update Submitted: 2021-09-21
• Last Update Posted: 2021-09-27
• Study Start: 2021-10
• Primary Completion: 2033-12
• Study Completion: 2034-12

Recruitment & Eligibility

• Status: ENROLLING_BY_INVITATION
• Sex: Not specified
• Target Recruitment: 197

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

Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Arm && Interventions

Group	Intervention	Description
RACE 1 patients	ATGAM plus CsA with or without Eltrombopag	After exiting the RACE trial (NCT02099747) patient will be invited to participate in this study

Primary Outcome Measures

Name	Time	Method
Failure Free Survival	15 years	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)

Secondary Outcome Measures

Name	Time	Method
Monitoring of Clonal Hematopoiesis of Indetermined Potential (CHIP)	15 years	As tracked by somatic mutations in genes associated with myeloid disorders
Response Rate: number of patients who reach a hematological response	15 years	Response Rate
Overall Survival	15 years	Overall Survival
Number of patients who need additional IST	15 years	Need for additional intensive Imune Suppressive Therapy (IST) (e.g. ATG, alemtuzumab or cyclophosphamide-based, or any other lymphocyte-depleting agent)
Number of patients who need Maintenance IST (e.g. CsA beyond 2 years)	15 years	Need for maintenance intensive IST (e.g. CsA beyond 2 years)
Number of patients who need additional Eltrombopag (EPAG)	15 years	Need for additional EPAG
Cumulative incidence of relapse after response	15 years	Cumulative incidence of relapse after initial hematological response (complete or partial)
Cumulative incidence of clonal evolution	15 years	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)
Cumulative incidence of clinical Paroxysmal nocturnal hemoglobinuria (PNH)	15 years	Cumulative incidence of clinical PNH (hemolysis and/or thromboembolism), and of need of anti-complement treatment
Cumulative incidence of Solid Tumors	15 years	Cumulative incidence of solid tumours
Number of patients who need a Human Stem Cell Transplantation (HSCT)	15 years	Need for HSCT
Number of patients who need any other approved Aplastic Anemia (AA) treatment	15 years	Need for any other approved Aplastic Anemia (AA) treatment

Trial Locations

Locations (1)

Hospital St. Louis; 🇫🇷 Paris, France