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

Sponsor
European Society for Blood and Marrow Transplantation (Other)
Overall Status
Enrolling by invitation
CT.gov ID
NCT05049668
Collaborator
(none)
197
1
158
1.2

Study Details

Study Description

Brief Summary

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

Condition or Disease Intervention/Treatment Phase
  • 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 Design

Study Type:
Observational [Patient Registry]
Anticipated Enrollment :
197 participants
Observational Model:
Cohort
Time Perspective:
Prospective
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
Anticipated Study Start Date :
Oct 1, 2021
Anticipated Primary Completion Date :
Dec 1, 2033
Anticipated Study Completion Date :
Dec 1, 2034

Arms and Interventions

Arm 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
  • Outcome Measures

    Primary Outcome Measures

    1. 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

    1. Response Rate: number of patients who reach a hematological response [15 years]

      Response Rate

    2. Overall Survival [15 years]

      Overall Survival

    3. Cumulative incidence of relapse after response [15 years]

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

    4. 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)

    5. 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

    6. Cumulative incidence of Solid Tumors [15 years]

      Cumulative incidence of solid tumours

    7. Number of patients who need a Human Stem Cell Transplantation (HSCT) [15 years]

      Need for HSCT

    8. 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)

    9. 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)

    10. Number of patients who need additional Eltrombopag (EPAG) [15 years]

      Need for additional EPAG

    11. Number of patients who need any other approved Aplastic Anemia (AA) treatment [15 years]

      Need for any other approved Aplastic Anemia (AA) treatment

    12. Monitoring of Clonal Hematopoiesis of Indetermined Potential (CHIP) [15 years]

      As tracked by somatic mutations in genes associated with myeloid disorders

    Eligibility Criteria

    Criteria

    Ages Eligible for Study:
    N/A and Older
    Sexes Eligible for Study:
    All
    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

    Contacts and Locations

    Locations

    Site City State Country Postal Code
    1 Hospital St. Louis Paris France

    Sponsors and Collaborators

    • 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 Documents (Full-Text)

    None provided.

    More Information

    Publications

    None provided.
    Responsible Party:
    European Society for Blood and Marrow Transplantation
    ClinicalTrials.gov Identifier:
    NCT05049668
    Other Study ID Numbers:
    • EBMT-RACE 2
    First Posted:
    Sep 20, 2021
    Last Update Posted:
    Sep 27, 2021
    Last Verified:
    Sep 1, 2021
    Individual Participant Data (IPD) Sharing Statement:
    No
    Plan to Share IPD:
    No
    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
    Additional relevant MeSH terms:

    Study Results

    No Results Posted as of Sep 27, 2021