Eltrombopag for Severe Aplastic Anemia - Clinical Trial
What is the Purpose of this Study?
We are doing this study to understand how the body absorbs, distributes, breaks down, and eliminates eltrombopag (also known as Promacta and Revolade) in children with severe aplastic anemia.
Severe Aplastic Anemia (SAA)
Who Can Participate in the Study?
Children ages 1-17 years who:
- Have Severe Aplastic Anemia
- Don't have congenital/inherited bone marrow failure syndromes
- Have refused or are not eligible for hematopoietic stem cell transplantation (HSCT)
What is Involved?
If you decide that you would like for your child to be part of this study, your child will:
- Get the study medicine, eltrombopag by mouth.
- Be asked to give blood and urine samples
- Be asked to come to the clinic once a week for the first 8 weeks, then once every 2 weeks for the next 6 weeks, then visit once a month for a total 18 months
- Come for visits once every 3 months for the following 3 years.