Skip to main content
U.S. flag

An official website of the United States government

Official websites use .gov
A .gov website belongs to an official government organization in the United States.

Secure .gov websites use HTTPS
A lock ( ) or https:// means you’ve safely connected to the .gov website. Share sensitive information only on official, secure websites.

Introduction

Severe aplastic anemia (SAA) and myelodysplastic syndrome (MDS) are life-threatening bone marrow disorders. For SAA patients, long term survival can be achieved with immunosuppressive treatment. However, of those patients treated with immunosuppressive therapy, one quarter to one third will not respond, and about 50% of responders will relapse.

Combined haplo-cord transplant as an alternative to cord or haploidentical donor alone transplantation has recently been shown to be a viable transplant option for SAA patients lacking an HLA matched donor. However, engraftment patterns have varied substantially and in some patients, cord engraftment was profoundly delayed or never occurred. CordIn™ is a cryopreserved stem/progenitor cell-based product of purified CD133+ cells composed of ex vivo expanded allogeneic UCB cells. CordIn™ comprises: 1) cord blood derived ex vivo expanded CD133+ cells (CordIn™ cultured fraction (CF)); and 2) the non-cultured cell fraction (CD133-) of the same CBU (CordIn™ Non-cultured Fraction (NF)). Both fractions, i.e. CordIn™ CF and CordIn™ NF are kept frozen until they are infused on the day of transplantation.

Therefore, this EXCITE SAA study is phase II trial designed to evaluate the safety and effectiveness of transplantation with ex vivo expanded UCB (CordIn™) to overcome the high incidence of graft rejection associated with conventional UCB for aplastic anemia, where graft rejection occurs in up to 50% of subjects. We believe, based on preliminary data, that transplantation of CordIn™ will not only lead to rapid engraftment, but will also lead to sustained hematopoiesis, expedited immune recovery, and will reduce the chance of cord graft failure in this setting, potentially obviating the need for co-transplanting haploidentical CD34+ cells as a stem cell back-up.

To get started:

  • Navigate through the menu to learn more about the study.
  • Log in to the application and fill out questionnaires.

Please contact us if you have any questions or concerns.