About this study
Treatments for sickle cell disease include medicines and blood transfusions to reduce or prevent pain and other side effects. The only cure is a stem cell (bone marrow) transplant.
We want to see if a drug called motixafortide is safe for adults with sickle cell disease. We also want to find out if the drug will help a person’s body increase the number of stem cells that can be collected for a possible future transplant.
In this study, patients will receive motixafortide by an injection under the skin. About 8 hours later, we will collect stem cells. The patient will return to the clinic 7–10 days after the last drug dose for another exam and blood draw.
Some of the stem cells we collect will be stored in case the patient needs a transplant or gene therapy later. If the patient later has a transplant that does not work, we could use these stored cells as a “rescue” therapy. This study will also show us how well their body might respond to a necessary step in the gene therapy process. What we learn from this study may help us improve medical care and advance cures for sickle cell disease.
Eligibility overview
- Age 18 or older
- Severe sickle cell disease
- Has a central line in place or is able to undergo apheresis without one