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

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ClinicalTrials.gov Identifier: NCT02099747
Recruitment Status : Completed
First Posted : March 31, 2014
Last Update Posted : December 22, 2020
Information provided by (Responsible Party):
European Society for Blood and Marrow Transplantation

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.

Condition or disease Intervention/treatment Phase
Severe Aplastic Anemia Drug: hATG Drug: CsA Drug: Eltrombopag Phase 3

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.

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 202 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
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.
Actual Study Start Date : July 2015
Actual Primary Completion Date : December 2020
Actual Study Completion Date : December 2020

Resource links provided by the National Library of Medicine

Drug Information available for: Eltrombopag

Arm Intervention/treatment
Active Comparator: hATG + CsA
Control Arm
Drug: hATG
Other Name: ATGAM

Drug: CsA
Experimental: hATG + CsA + Eltrombopag
Drug: hATG
Other Name: ATGAM

Drug: CsA
Drug: Eltrombopag

Primary Outcome Measures :
  1. 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.

Secondary Outcome Measures :
  1. Time to best heamatological response [ Time Frame: 2 year ]
  2. Heamatological Response at 6, 12, 18 and 24 months [ Time Frame: 2 year ]
  3. Cumulative incidence of response [ Time Frame: 2 year ]
  4. Overall survival [ Time Frame: 2 year ]
  5. Event-free survival [ Time Frame: 2 year ]
  6. Cumulative incidence of relapse rate [ Time Frame: 2 year ]
  7. Cumulative incidences of clonal evolution [ Time Frame: 2 year ]
  8. Cumulative incidence of PNH population occurrence and clinical hemolytic PNH occurrence [ Time Frame: 2 year ]
  9. Cumulative incidence of discontinuation of immunosuppressive therapy [ Time Frame: 2 year ]
  10. Rate of CsA-independent hematological response at 24 months [ Time Frame: 2 year ]
  11. Need for transfusions and number of transfusions required from treatment [ Time Frame: 2 year ]
  12. Need for any supportive care [ Time Frame: 2 year ]
  13. Comparison of number of SAEs between the two arms [ Time Frame: 2 year ]
    To look for the safety and tolerability of the investigational treatment

Information from the National Library of Medicine

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Ages Eligible for Study:   15 Years and older   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

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.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02099747

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Sponsors and Collaborators
European Society for Blood and Marrow Transplantation
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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 automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
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Responsible Party: European Society for Blood and Marrow Transplantation
ClinicalTrials.gov Identifier: NCT02099747    
Other Study ID Numbers: EBMT-RACE
2014-000363-40 ( EudraCT Number )
First Posted: March 31, 2014    Key Record Dates
Last Update Posted: December 22, 2020
Last Verified: December 2020
Keywords provided by European Society for Blood and Marrow Transplantation:
Aplastic Anaemia
Additional relevant MeSH terms:
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Anemia, Aplastic
Hematologic Diseases
Bone Marrow Failure Disorders
Bone Marrow Diseases