About this study
This study will look at how gene therapy (replacing or repairing broken genes) and bone marrow (stem cell) transplant used to treat sickle cell disease may affect patients’ brain function.
Patients enrolled in the gene therapy studies already get MRI scans as part of treatment. If they are enrolled in FUNCOGSC also, we will get more information from their scans, which may take longer than usual. Patients will also have surveys and tests to help us look at brain functions such as:
- IQ (intelligence)
- Attention
- Flexible thinking
- Doing several tasks at once
- Self-control
- How well patients complete tasks they learned earlier
- Learning and memory
- Academic performance
Eligibility overview
- Sickle cell disease
- Enrolled in a bone marrow transplant or gene therapy trial such as SCDHCT or SAGES1
- 8–25 years old