Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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COMPLETED
PHASE3
202 participants
INTERVENTIONAL
2015-07-31
2020-12-31
Brief Summary
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Detailed Description
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Conditions
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Keywords
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Study Design
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RANDOMIZED
PARALLEL
TREATMENT
NONE
Study Groups
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hATG + CsA
Control Arm
hATG
CsA
hATG + CsA + Eltrombopag
Experimental
hATG
CsA
Eltrombopag
Interventions
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hATG
CsA
Eltrombopag
Other Intervention Names
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Eligibility Criteria
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Inclusion Criteria
* 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
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.
15 Years
ALL
No
Sponsors
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Novartis
INDUSTRY
Pfizer
INDUSTRY
European Society for Blood and Marrow Transplantation
NETWORK
Responsible Party
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Principal Investigators
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Antonio Risitano, MD, PhD
Role: PRINCIPAL_INVESTIGATOR
Federico II Medical School, Haematology Division, Napels
Regis Peffault de Latour, MD, PhD
Role: PRINCIPAL_INVESTIGATOR
St. Louis Hospital, Haematology Division, Paris
Locations
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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
Countries
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References
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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.
Peffault 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.
Other Identifiers
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2014-000363-40
Identifier Type: EUDRACT_NUMBER
Identifier Source: secondary_id
EBMT-RACE
Identifier Type: -
Identifier Source: org_study_id