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.

What is the Condition Being Studied?

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)

Age Group

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. 

Study Details

Full Title
A phase II, open-label, non-controlled, intra-patient dose- escalation study to characterize the pharmacokinetics after oral administration of eltrombopag in pediatric patients with refractory, relapsed or treatment naïve severe aplastic anemia or recurren
Principal Investigator
Pediatric Hematology-Oncology Specialist
Protocol Number
IRB: PRO00083766
NCT: NCT03025698
Phase II
Enrollment Status
Open for Enrollment
Contact the Duke Recruitment Innovation Center