hATG+CsA vs hATG+CsA+Eltrombopag for SAA (RACE)

April 21, 2026 updated by: European Society for Blood and Marrow Transplantation

A 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.

The null hypothesis of no difference in CR% at 3 months between the arms will be tested against the alternative of a difference in CR% at an alpha level of .05 by assessing the odds ratio for arm yielded by this model.

Study Overview

Status

Status

Indicates the current recruitment status or the expanded access status.
Completed

Conditions

Conditions

The disease, disorder, syndrome, illness, or injury that is being studied. On ClinicalTrials.gov, conditions may also include other health-related issues, such as lifespan, quality of life, and health risks.

Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.

Detailed Description

This is a superiority trial aiming to increase the 3 month complete response rate. The sample size is calculated on the hypothesis that the experimental treatment will increase the 3 months response rate up to 21% (by 3 folds, based on the 7% reported in Scheinberg et al [17]). Under these assumptions, the sample size to reject the null hypothesis is n=96 patients for each treatment arm, increased by 4% for possibly not evaluable patients (total number of 200 patients, 100 each treatment arm). Statistical design for sample size calculation: increase from 7% (control arm) to 21% (investigational arm) in 3 month complete response rate (two-sided binomial test); alpha-error 0.05; power 0.8.

Study Type

Study Type

Describes the nature of a clinical study. Study types include interventional studies (also called clinical trials), observational studies (including patient registries), and expanded access.
Interventional

Enrollment (Actual)

Enrollment

The number of participants in a clinical study. The "anticipated" enrollment is the target number of participants that the researchers need for the study.
202

Phase

Phase

The stage of a clinical trial studying a drug or biological product, based on definitions developed by the U.S. Food and Drug Administration (FDA). The phase is based on the study's objective, the number of participants, and other characteristics. There are five phases: Early Phase 1 (formerly listed as Phase 0), Phase 1, Phase 2, Phase 3, and Phase 4. Not Applicable is used to describe trials without FDA-defined phases, including trials of devices or behavioral interventions.
  • Phase 3

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • France
      • Besançon, France
        • Hôpital Jean Minjoz
      • Bordeaux, France
        • Hôpital Haut-Lévêque
      • Lille, France
        • Hôpital Huriez
      • Lyon, France
        • Centre Hospitalier Lyon-Sud
      • Paris, France
        • St. Louis Hospital
      • Rennes, France
        • Pontchaillou Hospital
      • Toulouse, France
        • Hopital Purpan
  • Italy
      • Bergamo, Italy
        • Azienda Ospedaliera Papa Giovanni XXIII
      • Genova, Italy
        • San Martino Hospital
      • Genova, Italy
        • Istituto G. Gaslini children's Hospital
      • Milan, Italy
        • Fondazione IRCCS ca Granda Ospedale
      • Naples, Italy
        • 'Federico II' Medical School
      • Rome, Italy
        • La Sapienza University Hospital
      • Turin, Italy
        • AOU Città della Salute e della Scienza di Torino
  • Netherlands
      • Amsterdam, Netherlands
        • AMC
      • Groningen, Netherlands
        • UMCG
      • Leiden, Netherlands
        • Leiden University Medical Center
      • Utrecht, Netherlands
        • UMCU
  • Spain
      • Badalona, Spain
        • Hospital Universitari Germans Trias i Pujol
      • Barcelona, Spain
        • Institut Català d'Oncologia - Hospital Duran i Reynals
      • Donostia / San Sebastian, Spain
        • Donostia Hospital
      • Valencia, Spain
        • Hospital La Fe
  • Switzerland
      • Basel, Switzerland
        • University Hospital Basel
      • Bern, Switzerland
        • University Hospital Bern
      • Zurich, Switzerland
        • University Hospital Zürich
  • United Kingdom
      • Leeds, United Kingdom
        • St. James Hospital
      • London, United Kingdom
        • King's College Hospital
      • London, United Kingdom
        • St. Bartholomew's Hospital
      • Nottingham, United Kingdom
        • City Hospital

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Eligibility Criteria

The key requirements that people who want to participate in a clinical study must meet or the characteristics they must have. Eligibility criteria consist of both inclusion criteria (which are required for a person to participate in the study) and exclusion criteria (which prevent a person from participating). Types of eligibility criteria include whether a study accepts healthy volunteers, has age or age group requirements, or is limited by sex.

Ages Eligible for Study

15 years and older (Child, Adult, Older Adult)

Accepts Healthy Volunteers

No

Description

Inclusion Criteria:

  1. Diagnosis of severe or very severe aplastic anemia, defined by [29]:

    • At least two of the following:

      • Absolute neutrophil counts <0.5 x 109/L (severe) or <0.2 x 109/L (very severe)
      • Platelet counts <20 x 109/L
      • Reticulocyte counts <60 x 109/L
    • Hypocellular bone marrow (<30% cellularity), without evidences of fibrosis or malignant cells
  2. Male or female age > 14 years;
  3. Written informed consent
  4. Willing and able to comply with all of the requirements and visits in the protocol
  5. Understands that they can be randomised to either treatment arm
  6. Negative pregnancy test for women of child bearing age
  7. Written acceptance to use contraception (hormonal or barrier method of birth control; abstinence) for the entire duration of study participation.

Exclusion Criteria:

  1. Prior immunosuppressive therapy with ATG (horse of rabbit) or any other lymphocyte depleting agent (i.e., alemtuzumab)
  2. Eligibility to a sibling allogeneic stem cell transplantation
  3. Evidence of a myelodysplastic syndrome, defined by the presence of myelodysplastic features, excess of blasts or karyotypic abnormalities typical of MDS (according to revised WHO 2008 criteria) [30],, as well as other primitive marrow disease. Patients with diagnosis of AA with cytogenetic abnormalities which are recurrent in MDS (according to revised WHO 2008 criteria) [30] should be included in this category, and are not eligible for the study; patients with del(20q), +8 and -Y are not included in this category, and thus are eligible for this study. The list of karyotypic abnormalities which qualifies for the diagnosis of MDS are listed in the Appendix.
  4. History or clinical suspect of constitutional aplastic anemia (i.e. Fanconi Anemia with positive DEB/MMC test or Dyskeratosis Congenita)
  5. History of malignant tumors with active disease within 5 years from enrollment, and/or previous chemo-radiotherapy
  6. Previous history of stem cell transplantation

  7. Treatment with cyclosporin A unless

    • <4 weeks of cyclosporin A treatment before enrolement and
    • wash out period of 2 weeks before enrollment
  8. CMV viremia, as defined by positive PCR or pp65 test
  9. WHO performance status ≥3
  10. Pregnant or breast feeding patients
  11. Patients with hepatic, renal or cardiac failure, or any other life- threatening concurrent disease
  12. Patients with HIV infection
  13. Patients without social health care assistance
  14. Participation in another clinical trial within 1 month before the start of this trial
  15. Patients and/or female partners of male patients not using highly effective method of birth control i.e. intrauterine device (IUD), hormonal (oral pill, injection, implants), tubal ligation or partner's vasectomy
  16. subjects with known hypersensitivity to any of the component medications

The presence of a Paroxysmal Nocturnal Hemoglobinuria clone is not an exclusion criterion.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Randomized
  • Interventional Model: Parallel Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm

Participant Group / Arm

A group or subgroup of participants in a clinical trial that receives a specific intervention/treatment, or no intervention, according to the trial's protocol.
Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.
Active Comparator: hATG + CsA
Control Arm
Drug: hATG
Other Names:
  • ATGAM
Drug: CsA
Experimental: hATG + CsA + Eltrombopag
Experimental
Drug: Eltrombopag
Drug: hATG
Other Names:
  • ATGAM
Drug: CsA

What is the study measuring?

Primary Outcome Measures

Primary Outcome Measures

In a clinical study's protocol, the planned outcome measure that is the most important for evaluating the effect of an intervention/treatment. Most clinical studies have one primary outcome measure, but some have more than one.
Outcome Measure
Measure Description
Time Frame
CR rate
Time Frame: 3 months
The primary objective of this trial is to investigate whether Eltrombopag added to standard immunosuppressive treatment increases the rate of early (at three months) complete response in untreated AA patient.
3 months

Secondary Outcome Measures

Secondary Outcome Measures

In a clinical study's protocol, a planned outcome measure that is not as important as the primary outcome measure for evaluating the effect of an intervention but is still of interest. Most clinical studies have more than one secondary outcome measure.
Outcome Measure
Measure Description
Time Frame
Overall survival
Time Frame: 2 year
2 year
Time to best heamatological response
Time Frame: 2 year
2 year
Heamatological Response at 6, 12, 18 and 24 months
Time Frame: 2 year
2 year
Cumulative incidence of response
Time Frame: 2 year
2 year
Event-free survival
Time Frame: 2 year
2 year
Cumulative incidence of relapse rate
Time Frame: 2 year
2 year
Cumulative incidences of clonal evolution
Time Frame: 2 year
2 year
Cumulative incidence of PNH population occurrence and clinical hemolytic PNH occurrence
Time Frame: 2 year
2 year
Cumulative incidence of discontinuation of immunosuppressive therapy
Time Frame: 2 year
2 year
Rate of CsA-independent hematological response at 24 months
Time Frame: 2 year
2 year
Need for transfusions and number of transfusions required from treatment
Time Frame: 2 year
2 year
Need for any supportive care
Time Frame: 2 year
2 year
Comparison of number of SAEs between the two arms
Time Frame: 2 year
To look for the safety and tolerability of the investigational treatment
2 year

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Sponsor

The organization or person who initiates the study and who has authority and control over the study.
European Society for Blood and Marrow Transplantation

Collaborators

Collaborators

An organization other than the sponsor that provides support for a clinical study. This support may include activities related to funding, design, implementation, data analysis, or reporting.
Novartis
Pfizer

Investigators

Investigators

A researcher involved in a clinical study. Related terms include site principal investigator, site sub-investigator, study chair, study director, and study principal investigator.
  • Principal Investigator: Antonio Risitano, MD, PhD, Federico II Medical School, Haematology Division, Napels
  • Principal Investigator: Regis Peffault de Latour, MD, PhD, St. Louis Hospital, Haematology Division, Paris

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

Study Start

The actual date on which the first participant was enrolled in a clinical study. The "anticipated" study start date is the date that the researchers think will be the study start date.
July 1, 2015

Primary Completion (Actual)

Primary Completion

The date on which the last participant in a clinical study was examined or received an intervention to collect final data for the primary outcome measure. Whether the clinical study ended according to the protocol or was terminated does not affect this date. For clinical studies with more than one primary outcome measure with different completion dates, this term refers to the date on which data collection is completed for all the primary outcome measures. The "anticipated" primary completion date is the date that the researchers think will be the primary completion date for the study.
December 1, 2020

Study Completion (Actual)

Study Completion

The date on which the last participant in a clinical study was examined or received an intervention/treatment to collect final data for the primary outcome measures, secondary outcome measures, and adverse events (that is, the last participant's last visit). The "anticipated" study completion date is the date that the researchers think will be the study completion date.
December 1, 2020

Study Registration Dates

First Submitted

First Submitted

The date on which the study sponsor or investigator first submitted a study record to ClinicalTrials.gov. There is typically a delay of a few days between the first submitted date and the record's availability on ClinicalTrials.gov (the first posted date).
March 6, 2014

First Submitted That Met QC Criteria

First Submitted That Met QC Criteria

The date on which the study sponsor or investigator first submits a study record that is consistent with National Library of Medicine (NLM) quality control (QC) review criteria. The sponsor or investigator may need to revise and submit a study record one or more times before NLM's QC review criteria are met. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
March 26, 2014

First Posted (Estimated)

First Posted

The date on which the study record was first available on ClinicalTrials.gov after National Library of Medicine (NLM) quality control (QC) review has concluded. There is typically a delay of a few days between the date the study sponsor or investigator submitted the study record and the first posted date.
March 31, 2014

Study Record Updates

Last Update Posted (Actual)

Last Update Posted

The most recent date on which changes to a study record were made available on ClinicalTrials.gov. There may be a delay between when the changes were submitted to ClinicalTrials.gov by the study's sponsor or investigator (the last update submitted date) and the last update posted date.
April 24, 2026

Last Update Submitted That Met QC Criteria

Last Update Submitted That Met QC Criteria

The most recent date on which the study sponsor or investigator submitted changes to a study record that are consistent with National Library of Medicine (NLM) quality control (QC) review criteria. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
April 21, 2026

Last Verified

Last Verified

The most recent date on which the study sponsor or investigator confirmed the information about a clinical study on ClinicalTrials.gov as accurate and current. If a study with a recruitment status of recruiting; not yet recruiting; or active, not recruiting has not been confirmed within the past 2 years, the study's recruitment status is shown as unknown.
April 1, 2026

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

Other Study ID Numbers

Identifiers or ID numbers other than the NCT number that are assigned to a clinical study by the study's sponsor, funders, or others. These numbers may include unique identifiers from other trial registries and National Institutes of Health grant numbers.
  • EBMT-RACE
  • 2014-000363-40 (EudraCT Number)

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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