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

Severe Aplastic Anemia Clinical Trial

— RACE2  

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

Clinical Trial Details — Status: Enrolling by invitation

Administrative data

• NCT number: NCT05049668
• Other study ID #: EBMT-RACE 2
• Status: Enrolling by invitation
• Start date: October 2021
• Est. completion date: December 2034

Study information

• Verified date: September 2021
• Source: European Society for Blood and Marrow Transplantation
• Contact: n/a
• Is FDA regulated: No
• Study type: Observational [Patient Registry]

Clinical Trial Summary

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

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.

Recruitment information / eligibility

• Status: Enrolling by invitation
• Enrollment: 197
• Est. completion date: December 2034
• Est. primary completion date: December 2033
• 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

Related Conditions & MeSH terms

• Anemia
• Anemia, Aplastic
• Severe Aplastic Anemia

Intervention

Drug:
• ATGAM plus CsA with or without Eltrombopag
Standard treatment with or without Eltrombopag

Locations

Country	Name	City	State
France	Hospital St. Louis	Paris

Sponsors (1)

Lead Sponsor	Collaborator
European Society for Blood and Marrow Transplantation

Country where clinical trial is conducted

France, 

Outcome

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