Last updated on March 2020

Eltrombopag Combined With Cyclosporine as First Line Therapy in Patients With Severe Acquired Aplastic Anemia

Brief description of study

This is a two-part study, interventional Phase II, single-arm, multicenter, open-label study to investigate the efficacy and safety of a combination regimen of eltrombopag and cyclosporine in patients with severe aplastic anemia (SAA) as first line therapy and an extension with up to 60-months follow-up. All patients will receive eltrombopag for 6 months with cyclosporine. Patients who respond (who had overall hematologic response by 6 months) will continue tapering down cyclosporine for up to 30 months and will be followed up for a post-treatment follow-up up to 60 months by regular clinic visits. Patients who do not have an overall hematologic response (non-responders) by the end of 6 month time point will be discontinued from the treatment and will be followed up for non-responder post-treatment follow-up up to 60 months.

The usage of eltrombopag and cyclosporine combines two therapies with different modes of action. Cyclosporine acts as an immunosuppressant and eltrombopag acts as a stimulator of bone marrow progenitor cells. Given that SAA is currently viewed as having an autoimmune pathogenesis resulting in bone marrow progenitor cell destruction, the combination of eltrombopag and cyclosporine is attractive. Preliminary experience with their combined use appears favorable, with no untoward toxicity observed to date.

Clinical Study Identifier: NCT02998645

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Novartis Investigative Site

Budapest, Hungary
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