Purpose of this Study
We are doing this study to find out if patients with SAA who have not received previous treatment for this diagnosis can be treated safely and effectively with transplant as their first therapy.
Who Can Participate?
Eligibility
Children and Adults ages 3-75 who:
- Are diagnosed with SAA
- Do not have a suitable, fully matched related donor
- Have a left ventricular ejection fraction > 40% with no signs of heart failure, or an ejection fraction > 26% if less than 13 years old
Age Range
3-75
Sex/Genders
Male (cisgender)
Female (cisgender)
Non-binary or gender fluid
Transgender male
Transgender female
Looking for Healthy Participants
No
What is Involved?
Description
If you or your child choose to join the study, you/they will:
- Receive a combination of chemotherapy drugs (fludarabine and cyclophosphamide) before you/they get your/their donor cells
- Get bone marrow donor cells as an IV (intravenous) infusion
Locations
Duke University Hospital
Visit Timing
Weekends
Compensation
No
Spanish Materials Available
No
Study Details
Full Title
A Phase II Trial of Non-Myeloablative Conditioning and Transplantation of Haploidentical Related, Partially HLA-Mismatched, or Matched Unrelated Bone Marrow for Newly Diagnosed Patients with Severe Aplastic Anemia
Principal Investigator
Mitchell
Horwitz
Protocol Number
PRO00116329
NCT ID
NCT06517641
Phase
II
Enrollment Status
Pending Open to Enrollment