RACE 2: a Long Term Follow-up of Patients Participating in the RACE Trial (RACE2)

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

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

Study Overview

• Status: Enrolling by invitation
• Conditions: Severe Aplastic Anemia
• Intervention / Treatment: Drug: ATGAM plus CsA with or without Eltrombopag

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 Type: Observational
• Enrollment (Anticipated): 197

Contacts and Locations

Study Locations

• France
  • Paris, France
    • Hôspital St. Louis

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: ADULT; OLDER_ADULT; CHILD
• Accepts Healthy Volunteers: N/A

• Sampling Method: Non-Probability Sample

Study Population

Patients who participated in the initial RACE 1 trial

Description

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 Plan

How is the study designed?

Number of groups / cohorts

1

Cohorts and Interventions

Group / Cohort	Intervention / Treatment
RACE 1 patients; After exiting the RACE trial (NCT02099747) patient will be invited to participate in this study	Drug: ATGAM plus CsA with or without Eltrombopag; Standard treatment with or without Eltrombopag; Other Names: hATG+CsA +/- Revolade

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
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 Outcome Measures

Outcome Measure	Measure Description	Time Frame
Response Rate: number of patients who reach a hematological response	Response Rate	15 years
Overall Survival	Overall Survival	15 years
Cumulative incidence of relapse after response	Cumulative incidence of relapse after initial hematological response (complete or partial)	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)	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	15 years
Cumulative incidence of Solid Tumors	Cumulative incidence of solid tumours	15 years
Number of patients who need a Human Stem Cell Transplantation (HSCT)	Need for HSCT	15 years
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
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
Number of patients who need additional Eltrombopag (EPAG)	Need for additional EPAG	15 years
Number of patients who need any other approved Aplastic Anemia (AA) treatment	Need for any other approved Aplastic Anemia (AA) treatment	15 years
Monitoring of Clonal Hematopoiesis of Indetermined Potential (CHIP)	As tracked by somatic mutations in genes associated with myeloid disorders	15 years

Collaborators and Investigators

• Sponsor: European Society for Blood and Marrow Transplantation
• Investigators: Principal Investigator: Regis Peffault de Latour, Prof, MD, Hospital St. Louis, Paris, France; Principal Investigator: Antonio M Risitano, MD, PhD, Hospital Avellino, Napels, Italy

Study record dates

Study Major Dates

• Study Start (ANTICIPATED): October 1, 2021
• Primary Completion (ANTICIPATED): December 1, 2033
• Study Completion (ANTICIPATED): December 1, 2034

Study Registration Dates

• First Submitted: September 13, 2021
• First Submitted That Met QC Criteria: September 17, 2021
• First Posted (ACTUAL): September 20, 2021

Study Record Updates

• Last Update Posted (ACTUAL): September 27, 2021
• Last Update Submitted That Met QC Criteria: September 21, 2021
• Last Verified: September 1, 2021

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Bone Marrow Diseases; Hematologic Diseases; Bone Marrow Failure Disorders; Anemia; Anemia, Aplastic
• Other Study ID Numbers: EBMT-RACE 2

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: No