Early Human Leukocyte Antigen (HLA) Matched Sibling Hematopoietic Stem Cell Transplantation
Status:
Recruiting
Trial end date:
2027-01-01
Target enrollment:
Participant gender:
Summary
This study aims to enroll 58 pre-adolescent (<13 years) pediatric participants with sickle
cell disease (SCD) who have a pre-adolescent sibling bone marrow donor. All participants will
go through a pre-transplant evaluation to find out if there are health problems that will
keep them from being able to receive the transplant. It usually takes 2 to 3 months to
complete the pre-transplant evaluation and make the arrangements for the transplant. Once
they are found to be eligible for transplant, participants will be admitted to the hospital
and will start transplant conditioning. Conditioning is the chemotherapy and other medicines
given to prepare them to receive donor cells. It prevents the immune system from rejecting
donor cells. Conditioning will start 21 days before transplant. Once they complete
conditioning, participants will receive the bone marrow transplant. After the transplant,
participants will stay in the hospital for 4-6 weeks. After they leave the hospital,
participants will be followed closely in the clinic. Outpatient treatment and frequent clinic
visits usually last 6 to 12 months. Routine medical care includes at least a yearly
examination for many years after transplant by doctors and nurses familiar with sickle cell
disease and transplant. The researchers will collect and study information about participants
for 5 years after transplant.