ABOUT THIS STUDY
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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.
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.
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Descriptive Information | |||||||
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Brief Title ICMJE | hATG+CsA vs hATG+CsA+Eltrombopag for SAA | ||||||
Official Title ICMJE | 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. | ||||||
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. | ||||||
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 ICMJE | Interventional | ||||||
Study Phase ICMJE | Phase 3 | ||||||
Study Design ICMJE | Allocation: Randomized Intervention Model: Parallel Assignment Masking: None (Open Label) Primary Purpose: Treatment | ||||||
Condition ICMJE | Severe Aplastic Anemia | ||||||
Intervention ICMJE |
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Study Arms ICMJE |
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Publications * | Not Provided | ||||||
* Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline. | |||||||
Recruitment Information | |||||||
Recruitment Status ICMJE | Completed | ||||||
Actual Enrollment ICMJE | 202 | ||||||
Original Enrollment ICMJE | Not Provided | ||||||
Actual Study Completion Date ICMJE | December 2020 | ||||||
Actual Primary Completion Date | December 2020 (Final data collection date for primary outcome measure) | ||||||
Eligibility Criteria ICMJE | Inclusion Criteria:
Exclusion Criteria:
The presence of a Paroxysmal Nocturnal Hemoglobinuria clone is not an exclusion criterion. | ||||||
Sex/Gender ICMJE |
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Ages ICMJE | 15 Years and older (Child, Adult, Older Adult) | ||||||
Accepts Healthy Volunteers ICMJE | No | ||||||
Contacts ICMJE | Contact information is only displayed when the study is recruiting subjects | ||||||
Listed Location Countries ICMJE | France, Italy, Netherlands, Spain, Switzerland, United Kingdom | ||||||
Removed Location Countries | Germany | ||||||
Administrative Information | |||||||
NCT Number ICMJE | NCT02099747 | ||||||
Other Study ID Numbers ICMJE | EBMT-RACE 2014-000363-40 ( EudraCT Number ) | ||||||
Has Data Monitoring Committee | Yes | ||||||
U.S. FDA-regulated Product | Not Provided | ||||||
IPD Sharing Statement ICMJE | Not Provided | ||||||
Responsible Party | European Group for Blood and Marrow Transplantation | ||||||
Study Sponsor ICMJE | European Group for Blood and Marrow Transplantation | ||||||
Collaborators ICMJE |
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Investigators ICMJE |
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PRS Account | European Group for Blood and Marrow Transplantation | ||||||
Verification Date | December 2020 | ||||||
ICMJE Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP |