A trial in which will be studied if the addition of a medicine that is used for improving platelet counts (eltrombopag) to the regular treatment for aplastic anemia (hATG + CsA) also improves the numbers of other cell lines and therefore the overall bone marrow function.

• Conditions: Idiopathic (Severe) Aplastic Anemia, a bone marrow failure syndrome which results in the normal hematopoietic tissue completely missing from the bone marrow, accounting for the subsequent pancytopenia.; MedDRA version: 18.0Level: PTClassification code 10002967Term: Aplastic anaemiaSystem Organ Class: 10005329 - Blood and lymphatic system disorders; Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]

• Registration Number: EUCTR2014-000363-40-ES
• Lead Sponsor: European Society for Blood and Marrow Transplantation

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2015-09-07
• Last Update Posted: 16 November 2015

Recruitment & Eligibility

• Status: Authorised-recruitment may be ongoing or finished
• Sex: All
• Target Recruitment: 200

Inclusion Criteria

1.Diagnosis of severe or very severe aplastic anemia, defined by
?At least two of the following:
?Absolute neutrophil counts <0.5 x 10(9)/L (severe) or
<0.2 x 10(9)/L (very severe)
?Platelet counts <20 x 10(9)/L
?Reticulocyte counts <60 x 10(9)/L
?Hypocellular bone marrow (<30% cellularity), without evidences of fibrosis or malignant cells
2.Age ? 15 years;
3.Written informed consent
Are the trial subjects under 18? yes
Number of subjects for this age range: 15
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 160
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 25

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) , 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) 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 Appendix 1 of the protocol

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-rediotherapy

6.Previous history of stem cell transplantation

7.Treatment with cyclosporin A <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.Subjects with known hypersensitivity to any of the component medications

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Main Objective: To investigate whether Eltrombopag added to standard immunosuppressive treatment increases the rate of early (at three months) complete response in untreated AA patient.;Secondary Objective: To investigate the impact of Eltrombopag added to standard treatment on all heamatology outcome measures, Quality of Life and safety/tolerability in untreated AA patients.;Primary end point(s): Rate of Complete response (defined as Hb >10 g/dL, ANC > 1,000/?L and Plt >100,000 ?L) at 3 months since start of treatment in untreated severe AA patients.;Timepoint(s) of evaluation of this end point: For the main endpoint a valid response has to be measured between month 3 plus or minus 10 days; in case of more than one measurement available in this time period, the best response will be taken.