Severe Aplastic AnemiaPhase 3RACENCT02099747
hATG+CsA vs hATG+CsA+Eltrombopag for SAA
A Phase 3 study testing hATG, CsA and Eltrombopag in people with Severe Aplastic Anemia, run by European Society for Blood and Marrow Transplantation at 29 sites in 6 countries.
Trial summary
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. Read more about this study
- Who is it for?
- Ages 15 years and older
- Conditions the study is for
- Severe Aplastic Anemia
- What is being tested?
- hATG, CsA and Eltrombopag
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 202 participants
- Where
- 29 sitesin 6 countries
- Study dates
- Jul 2015to Dec 2020
- Phase
- Phase 3
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 15 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- 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
Show all 12Show fewer
- Male or female age > 14 years;
- Written informed consent
- Willing and able to comply with all of the requirements and visits in the protocol
- Understands that they can be randomised to either treatment arm
- Negative pregnancy test for women of child bearing age
- Written acceptance to use contraception (hormonal or barrier method of birth control; abstinence) for the entire duration of study participation.
Who cannot join
- Prior immunosuppressive therapy with ATG (horse of rabbit) or any other lymphocyte depleting agent (i.e., alemtuzumab)
- Eligibility to a sibling allogeneic stem cell transplantation
- 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.
- History or clinical suspect of constitutional aplastic anemia (i.e. Fanconi Anemia with positive DEB/MMC test or Dyskeratosis Congenita)
- History of malignant tumors with active disease within 5 years from enrollment, and/or previous chemo-radiotherapy
- Previous history of stem cell transplantation
Show all 19Show fewer
- Treatment with cyclosporin A unless
- <4 weeks of cyclosporin A treatment before enrolement and
- wash out period of 2 weeks before enrollment
- CMV viremia, as defined by positive PCR or pp65 test
- WHO performance status ≥3
- Pregnant or breast feeding patients
- Patients with hepatic, renal or cardiac failure, or any other life- threatening concurrent disease
- Patients with HIV infection
- Patients without social health care assistance
- Participation in another clinical trial within 1 month before the start of this trial
- 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
- subjects with known hypersensitivity to any of the component medications
- The presence of a Paroxysmal Nocturnal Hemoglobinuria clone is not an exclusion criterion.
Trial locations and contacts
29 sites in 6 countries.
29 sites in 6 countries
France7 sites
Hopital Jean Minjoz
Besançon
Hôpital Haut-Lévèque
Bordeaux
Hôpital Huriez
Lille
Centre Hospitalier Lyon-Sud
Lyon
St. Louis Hospital
Paris
Pontchaillou Hospital
Rennes
Hôpital Purpan
Toulouse
Italy7 sites
Azienda Ospedaliera Papa Giovanni XXIII
Bergamo
Istituto G. Gaslini children's Hospital
Genova
San Martino Hospital
Genova
Fondazione IRCCS ca Granda Ospedale
Milan
'Federico II' Medical School
Naples
La Sapienza University Hospital
Rome
AOU Città della Salute e della Scienza di Torino
Turin
Netherlands4 sites
AMC
Amsterdam
UMCG
Groningen
Leiden University Medical Center
Leiden
UMCU
Utrecht
Spain4 sites
Hospital Universitari Germans Trias I Pujol
Badalona
Institut Català d'Oncologia - Hospital Duran i Reynals
Barcelona
Donostia Hospital
Donostia / San Sebastian
Hospital La Fe
Valencia
United Kingdom4 sites
St. James Hospital
Leeds
King's College Hospital
London
St. Bartholomew's Hospital
London
City Hospital
Nottingham
Switzerland3 sites
University Hospital Basel
Basel
University Hospital Bern
Bern
University Hospital Zürich
Zurich
About this study
Official title: 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.
Read the 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.
- Conditions
- Severe Aplastic Anemia
- Being tested
- hATG, CsA, Eltrombopag
- Keywords
- Aplastic Anaemia, Eltrombopag, HATG, ATGAM
- Medical subject headings
- Anemia, Aplastic
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
hATGDrug
Also called ATGAM
CsADrug
EltrombopagDrug
| Study group | Role | What they receive |
|---|---|---|
| hATG + CsA | Active comparator | Control Arm Receives: Drug: hATG; Drug: CsA |
| hATG + CsA + Eltrombopag | Experimental | Experimental Receives: Drug: hATG; Drug: CsA; Drug: Eltrombopag |
What is being measured
The primary outcome is the main question the study is designed to answer.
CR rate
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.
Measured: 3 months
Secondary outcomes (13)
Time to best heamatological response
Measured: 2 year
Heamatological Response at 6, 12, 18 and 24 months
Measured: 2 year
Cumulative incidence of response
Measured: 2 year
Overall survival
Measured: 2 year
Event-free survival
Measured: 2 year
Cumulative incidence of relapse rate
Measured: 2 year
Cumulative incidences of clonal evolution
Measured: 2 year
Cumulative incidence of PNH population occurrence and clinical hemolytic PNH occurrence
Measured: 2 year
Cumulative incidence of discontinuation of immunosuppressive therapy
Measured: 2 year
Rate of CsA-independent hematological response at 24 months
Measured: 2 year
Need for transfusions and number of transfusions required from treatment
Measured: 2 year
Need for any supportive care
Measured: 2 year
Comparison of number of SAEs between the two arms
To look for the safety and tolerability of the investigational treatment
Measured: 2 year
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted31 Mar 2014 (estimated)
- Study startJul 2015
- Primary completionDec 2020
- Study completionDec 2020
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (3)Papers the sponsor has linked to this study.
- Other trials for Severe Aplastic AnemiaStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 202 participants (actual)
Sponsor and investigators
- Lead sponsor
- European Society for Blood and Marrow Transplantation
- Sponsor type
- Research network
- Collaborators
- Novartis, Pfizer
- Responsible party
- Sponsor
- Investigators
- Antonio Risitano, MD, PhD, Principal investigator, Federico II Medical School, Haematology Division, NapelsRegis Peffault de Latour, MD, PhD, Principal investigator, St. Louis Hospital, Haematology Division, Paris
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02099747
- Sponsor's protocol ID
- EBMT-RACE
- Other IDs
- 2014-000363-40, 2014
- Acronym
- RACE
- Status last verified
- Apr 2026
- First submitted
- 6 Mar 2014
- Last updated
- 24 Apr 2026
- Results posted
- No
- Data monitoring committee
- Yes
Publications
Kaya DE, Cook R, Iacobelli S, Napolitani G, Gerlevik S, Seymen N, Sicre de Fontbrune F, Griffin M, Frieri C, Halkes CJM, Recher C, Barraco F, Forcade E, Mear JB, Laurino M, Drexler B, Daguindau E, van Os M, Terwel S, Dufour C, Karimi MM, Kulasekararaj AG, Peffault De Latour R, Risitano AM, Mufti G. Clonal Dynamics of Hematopoiesis in Aplastic Anemia after Immunosuppression and Eltrombopag. NEJM Evid. 2026 May;5(5):EVIDoa2500174. doi: 10.1056/EVIDoa2500174. Epub 2026 Apr 28.
PubMed 42047558de Latour RP, Kulasekararaj A, Iacobelli S, Griffin M, Halkes CJ, Dufour C, Risitano AM. Plain language summary of RACE study results: addition of eltrombopag to standard treatment of severe aplastic anemia. Immunotherapy. 2024 Feb;16(3):135-142. doi: 10.2217/imt-2023-0200. Epub 2023 Dec 13.
PubMed 38088156Peffault de Latour R, Kulasekararaj A, Iacobelli S, Terwel SR, Cook R, Griffin M, Halkes CJM, Recher C, Barraco F, Forcade E, Vallejo JC, Drexler B, Mear JB, Smith AE, Angelucci E, Raymakers RAP, de Groot MR, Daguindau E, Nur E, Barcellini W, Russell NH, Terriou L, Iori AP, La Rocca U, Sureda A, Sanchez-Ortega I, Xicoy B, Jarque I, Cavenagh J, Sicre de Fontbrune F, Marotta S, Munir T, Tjon JML, Tavitian S, Praire A, Clement L, Rabian F, Marano L, Hill A, Palmisani E, Muus P, Cacace F, Frieri C, van Lint MT, Passweg JR, Marsh JCW, Socie G, Mufti GJ, Dufour C, Risitano AM; Severe Aplastic Anemia Working Party of the European Society for Blood and Marrow Transplantation. Eltrombopag Added to Immunosuppression in Severe Aplastic Anemia. N Engl J Med. 2022 Jan 6;386(1):11-23. doi: 10.1056/NEJMoa2109965.
PubMed 34986284
Study at a glance
From the registry record, last updated by the sponsor on 24 Apr 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- 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.
- ClinicalTrials.gov ID
- NCT02099747
- Acronym / sponsor ID
- RACE / EBMT-RACE
- Conditions
- Severe Aplastic Anemia
- Keywords
- Aplastic Anaemia, Eltrombopag, HATG, ATGAM
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 202 participants (actual)
- Lead sponsor
- European Society for Blood and Marrow TransplantationResearch network
- Collaborators
- Novartis, Pfizer
- Study start
- Jul 2015 (actual)
- Primary completion
- Dec 2020 (actual)
- Study completion
- Dec 2020 (actual)
- First posted
- 31 Mar 2014 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
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 design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| hATG + CsA | Active comparator | Drug: hATG, Drug: CsAControl Arm |
| hATG + CsA + Eltrombopag | Experimental | Drug: hATG, Drug: CsA, Drug: EltrombopagExperimental |
Who may qualify
- Ages
- 15 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (12 listed)
- Diagnosis of severe or very severe aplastic anemia, defined by [29]:
- Male or female age > 14 years;
- Written informed consent
- Willing and able to comply with all of the requirements and visits in the protocol
- Understands that they can be randomised to either treatment arm
Key exclusion criteria (19 listed)
- Prior immunosuppressive therapy with ATG (horse of rabbit) or any other lymphocyte depleting agent (i.e., alemtuzumab)
- Eligibility to a sibling allogeneic stem cell transplantation
- 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.
- History or clinical suspect of constitutional aplastic anemia (i.e. Fanconi Anemia with positive DEB/MMC test or Dyskeratosis Congenita)
- History of malignant tumors with active disease within 5 years from enrollment, and/or previous chemo-radiotherapy
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| hATGAlso: ATGAM | Drug | hATG + CsA, hATG + CsA + Eltrombopag |
| CsA | Drug | hATG + CsA, hATG + CsA + Eltrombopag |
| Eltrombopag | Drug | hATG + CsA + Eltrombopag |
Primary outcome
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.
13 secondary outcomes
Time to best heamatological response
Time frame: 2 year
Heamatological Response at 6, 12, 18 and 24 months
Time frame: 2 year
Cumulative incidence of response
Time frame: 2 year
Overall survival
Time frame: 2 year
Event-free survival
Time frame: 2 year
Cumulative incidence of relapse rate
Time frame: 2 year
Cumulative incidences of clonal evolution
Time frame: 2 year
Cumulative incidence of PNH population occurrence and clinical hemolytic PNH occurrence
Time frame: 2 year
Cumulative incidence of discontinuation of immunosuppressive therapy
Time frame: 2 year
Rate of CsA-independent hematological response at 24 months
Time frame: 2 year
Need for transfusions and number of transfusions required from treatment
Time frame: 2 year
Need for any supportive care
Time frame: 2 year
Comparison of number of SAEs between the two arms
Time frame: 2 year
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 24 Apr 2026.
- Derived from this study: PubMed 42047558Kaya DE, Cook R, Iacobelli S, Napolitani G, Gerlevik S, Seymen N, Sicre de Fontbrune F, Griffin M, Frieri C, Halkes CJM, Recher C, Barraco F, Forcade E, Mear JB, Laurino M, Drexler B, Daguindau E, van Os M, Terwel S, Dufour C, Karimi MM, Kulasekararaj AG, Peffault De Latour R, Risitano AM, Mufti G. Clonal Dynamics of Hematopoiesis in Aplastic Anemia after Immunosuppression and Eltrombopag. NEJM Evid. 2026 May;5(5):EVIDoa2500174. doi: 10.1056/EVIDoa2500174. Epub 2026 Apr 28.
- Derived from this study: PubMed 38088156de Latour RP, Kulasekararaj A, Iacobelli S, Griffin M, Halkes CJ, Dufour C, Risitano AM. Plain language summary of RACE study results: addition of eltrombopag to standard treatment of severe aplastic anemia. Immunotherapy. 2024 Feb;16(3):135-142. doi: 10.2217/imt-2023-0200. Epub 2023 Dec 13.
- Derived from this study: PubMed 34986284Peffault de Latour R, Kulasekararaj A, Iacobelli S, Terwel SR, Cook R, Griffin M, Halkes CJM, Recher C, Barraco F, Forcade E, Vallejo JC, Drexler B, Mear JB, Smith AE, Angelucci E, Raymakers RAP, de Groot MR, Daguindau E, Nur E, Barcellini W, Russell NH, Terriou L, Iori AP, La Rocca U, Sureda A, Sanchez-Ortega I, Xicoy B, Jarque I, Cavenagh J, Sicre de Fontbrune F, Marotta S, Munir T, Tjon JML, Tavitian S, Praire A, Clement L, Rabian F, Marano L, Hill A, Palmisani E, Muus P, Cacace F, Frieri C, van Lint MT, Passweg JR, Marsh JCW, Socie G, Mufti GJ, Dufour C, Risitano AM; Severe Aplastic Anemia Working Party of the European Society for Blood and Marrow Transplantation. Eltrombopag Added to Immunosuppression in Severe Aplastic Anemia. N Engl J Med. 2022 Jan 6;386(1):11-23. doi: 10.1056/NEJMoa2109965.
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Severe Aplastic Anemia
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Antonio Risitano, MD, PhD principal investigator
Federico II Medical School, Haematology Division, Napels
Regis Peffault de Latour, MD, PhD principal investigator
St. Louis Hospital, Haematology Division, Paris
29 sites in 6 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing hATG, CsA and Eltrombopag in people with Severe Aplastic Anemia, run by European Society for Blood and Marrow Transplantation at 29 sites in 6 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 12 inclusion and 19 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 24 Apr 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 29 sites in France, Italy, Netherlands, Spain, United Kingdom and 1 more country. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
CR rate (time frame: 3 months). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT02099747, which also lists 3 linked publications.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 24 Apr 2026; AzTrials last refreshed its copy on 28 Oct 2026.