hATG+CsA vs hATG+CsA+Eltrombopag for SAA
RACE
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.
2 other identifiers
interventional
202
6 countries
29
Brief 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.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P25-P50 for phase_3
Started Jul 2015
Longer than P75 for phase_3
29 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
Click on a node to explore related trials.
Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
March 6, 2014
CompletedFirst Posted
Study publicly available on registry
March 31, 2014
CompletedStudy Start
First participant enrolled
July 1, 2015
CompletedPrimary Completion
Last participant's last visit for primary outcome
December 1, 2020
CompletedStudy Completion
Last participant's last visit for all outcomes
December 1, 2020
CompletedApril 24, 2026
April 1, 2026
5.4 years
March 6, 2014
April 21, 2026
Conditions
Keywords
Outcome Measures
Primary Outcomes (1)
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.
3 months
Secondary Outcomes (13)
Time to best heamatological response
2 year
Heamatological Response at 6, 12, 18 and 24 months
2 year
Cumulative incidence of response
2 year
Overall survival
2 year
Event-free survival
2 year
- +8 more secondary outcomes
Study Arms (2)
hATG + CsA
ACTIVE COMPARATORControl Arm
hATG + CsA + Eltrombopag
EXPERIMENTALExperimental
Interventions
Eligibility Criteria
You may qualify if:
- 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
- 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.
You may not qualify if:
- 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
- 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
- +3 more criteria
Contact the study team to confirm eligibility.
Sponsors & Collaborators
- Novartiscollaborator
- Pfizercollaborator
- European Society for Blood and Marrow Transplantationlead
Study Sites (29)
Hopital Jean Minjoz
Besançon, France
Hôpital Haut-Lévèque
Bordeaux, France
Hôpital Huriez
Lille, France
Centre Hospitalier Lyon-Sud
Lyon, France
St. Louis Hospital
Paris, France
Pontchaillou Hospital
Rennes, France
Hôpital Purpan
Toulouse, France
Azienda Ospedaliera Papa Giovanni XXIII
Bergamo, Italy
Istituto G. Gaslini children's Hospital
Genova, Italy
San Martino Hospital
Genova, Italy
Fondazione IRCCS ca Granda Ospedale
Milan, Italy
'Federico II' Medical School
Naples, Italy
La Sapienza University Hospital
Rome, Italy
AOU Città della Salute e della Scienza di Torino
Turin, Italy
AMC
Amsterdam, Netherlands
UMCG
Groningen, Netherlands
Leiden University Medical Center
Leiden, Netherlands
UMCU
Utrecht, Netherlands
Hospital Universitari Germans Trias I Pujol
Badalona, Spain
Institut Català d'Oncologia - Hospital Duran i Reynals
Barcelona, Spain
Donostia Hospital
Donostia / San Sebastian, Spain
Hospital La Fe
Valencia, Spain
University Hospital Basel
Basel, Switzerland
University Hospital Bern
Bern, Switzerland
University Hospital Zürich
Zurich, Switzerland
St. James Hospital
Leeds, United Kingdom
King's College Hospital
London, United Kingdom
St. Bartholomew's Hospital
London, United Kingdom
City Hospital
Nottingham, United Kingdom
Related Publications (2)
de 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.
PMID: 38088156DERIVEDPeffault 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.
PMID: 34986284DERIVED
MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
Intervention Hierarchy (Ancestors)
Study Officials
- 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
Study Design
- Study Type
- interventional
- Phase
- phase 3
- Allocation
- RANDOMIZED
- Masking
- NONE
- Purpose
- TREATMENT
- Intervention Model
- PARALLEL
- Sponsor Type
- NETWORK
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
March 6, 2014
First Posted
March 31, 2014
Study Start
July 1, 2015
Primary Completion
December 1, 2020
Study Completion
December 1, 2020
Last Updated
April 24, 2026
Record last verified: 2026-04