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About the Study

Who runs this study?

The Section of Transplantation Immunotherapy within the Hematology Branch of the National Heart, Lung and Blood Institute (NHLBI) will conduct this study.

The Principal Investigator of this study is Richard Childs, MD. He is Chief of the Section of Transplantation Immunotherapy and Clinical Director of Division of Intramural Research at the NHLBI.

What is this study about?

Our goal is 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, expedite engraftment and improve transplant outcome.

The primary objective of the Phase II study is to evaluate the ability of the CordIn™ unit to achieve sustained early engraftment. Secondary endpoints will include 100 day and 200 day treatment related mortality (TRM), and standard transplant outcome variables such as non-hematologic toxicity, incidence and severity of acute and chronic GVHD, and relapse of disease. Health related quality of life will also be assessed as secondary outcome measure.

How can I ask questions or ask for help?

Email us at kristen.gunn@nih.gov (or click on Contact). We will respond within 2 business days.