A Reduced Toxicity Allogeneic Unrelated Donor Stem Cell Transplantation (SCT) for Severe Sickle Cell Disease
Status:
Terminated
Trial end date:
2015-01-01
Target enrollment:
Participant gender:
Summary
Majority of patients who are eligible for allogeneic HSCT for cure of severe sickle cell
disease lack a matched family donor. This study aims for cure of sickle cell disease by
performing unrelated donor (outside family) allogeneic HSCT. Donors or unrelated cord blood
units will be selected from the NMDP database. It is designed to estimate the safety of a
novel reduced toxicity, yet an immunosuppressive and myeloablative preparative regimen. This
is meant for patients <21 years old who have severe complications from sickle cell and do not
have matched sibling donors in the family to undergo stem cell transplant. Patients will
undergo transplant using unrelated donor stem cells after receiving the protocol therapy.
They will be followed for 1 year to monitor for engraftment of donor cells and complications
like graft versus host disease (GVHD), infections and death.