CompletedPhase 3

hATG+CsA vs hATG+CsA+Eltrombopag for SAA

France202 participantsStarted 2015-07

Plain-language 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.

Who can participate

Age range15 Years

SexALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

Inclusion criteria

✓. Diagnosis of severe or very severe aplastic anemia, defined by \[29\]:
✓. 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.

Exclusion criteria

✕. 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
✕. CMV viremia, as defined by positive PCR or pp65 test

What they're measuring

CR rate

Timeframe: 3 months

Trial details

NCT IDNCT02099747

SponsorEuropean Society for Blood and Marrow Transplantation

Sponsor typeNETWORK

Study typeINTERVENTIONAL

Primary completion2020-12

View on ClinicalTrials.gov More Severe Aplastic Anemia trials