Purpose of this Study
This study is not testing a new treatment. People will continue to receive their usual medical care, such as a stem cell transplant or gene therapy. If they join the study, they will give blood samples during their regular visits over about five years. They may also choose to give other samples, such as stool, skin, hair, nails, or bone marrow. These samples will help researchers learn more about how the body responds to these treatments over time.
Who Can Participate?
Eligibility
People can join this study if they have a blood disease that is not cancer, such as sickle cell disease, thalassemia, or aplastic anemia. They must be planning to receive a stem cell transplant or gene therapy. They need to be able to come to study visits and follow study steps. They must be willing to give blood samples and agree to be followed for up to five years so researchers can learn how treatments work over time.
Age Range
7-100
Sex/Genders
Male (cisgender)
Female (cisgender)
Non-binary or gender fluid
Transgender male
Transgender female
Looking for Healthy Participants
No
What is Involved?
Description
This study collects samples from people who have serious blood diseases and are getting treatments like a stem cell transplant or gene therapy. The goal is to learn why these treatments work better for some people and not for others. Researchers also want to understand why some people have problems after treatment. By studying these samples over time, doctors hope to improve care and make treatments safer and more effective for future patients.
Locations
Duke University Hospital
Visit Timing
Weekdays
Compensation
No
Spanish Materials Available
No
Study Details
Full Title
Hematopoietic Cell Transplant and Gene Therapy for Non-Malignant Blood Disorders Biobank Resource
Principal Investigator
Mitchell
Horwitz
Protocol Number
PRO00119726
NCT ID
NCT07227155
Phase
N/A
Enrollment Status
Pending Open to Enrollment