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Enrolling by Invitation

NCT Number: NCT05049668

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

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

Enrolling by Invitation

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Key information

Conditions

Severe Aplastic Anemia Anemia Anemia, Aplastic Bone Marrow Diseases Bone Marrow Failure Disorders Hematologic Diseases Hemic and Lymphatic Diseases

Study type

Observational

Primary location

Hospital St. Louis

Paris, France

About this 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.

Who can participate

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

  • Subject participated in the RACE trial (NCT02099747, EudraCT number: 2014-000363-40) during which patient received ATGAM, Cyclosporine A with or without Eltrombopag.
  • Subject has provided informed consent to participate in long-term data collection

Exclusion criteria

None

Treatment and study plan

ATGAM plus CsA with or without Eltrombopag

Drug

Standard treatment with or without Eltrombopag

Other names: hATG+CsA +/- Revolade

Primary outcomes

  1. Failure Free Survival

    Time frame: 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 outcomes

  1. Response Rate: number of patients who reach a hematological response

    Time frame: 15 years

    Response Rate

  2. Overall Survival

    Time frame: 15 years

    Overall Survival

  3. Cumulative incidence of relapse after response

    Time frame: 15 years

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

  4. Cumulative incidence of clonal evolution

    Time frame: 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)

    Time frame: 15 years

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

  6. Cumulative incidence of Solid Tumors

    Time frame: 15 years

    Cumulative incidence of solid tumours

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

    Time frame: 15 years

    Need for HSCT

  8. Number of patients who need additional IST

    Time frame: 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)

    Time frame: 15 years

    Need for maintenance intensive IST (e.g. CsA beyond 2 years)

  10. Number of patients who need additional Eltrombopag (EPAG)

    Time frame: 15 years

    Need for additional EPAG

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

    Time frame: 15 years

    Need for any other approved Aplastic Anemia (AA) treatment

  12. Monitoring of Clonal Hematopoiesis of Indetermined Potential (CHIP)

    Time frame: 15 years

    As tracked by somatic mutations in genes associated with myeloid disorders

Sponsors and collaborators

Lead sponsor

European Society for Blood and Marrow Transplantation

Network

Registry information

Official study 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

Acronym: RACE2

Important dates

Study start
2021
Primary completion
2033
Study completion
2034
First posted
Sep 20, 2021
Registry last updated
Sep 27, 2021

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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