Description Module
The Description Module contains narrative descriptions of the clinical trial, including a brief summary and detailed description. These descriptions provide important information about the study's purpose, methodology, and key details in language accessible to both researchers and the general public.
Description Module path is as follows:
Study -> Protocol Section -> Description Module
Description Module
Ignite Creation Date:
2025-12-25 @ 1:32 AM
Ignite Modification Date:
2025-12-25 @ 1:32 AM
NCT ID:
NCT03279094
Brief Summary:
This is a study to evaluate the safety and toxicity of a treatment regimen consisting of 2 cycles of pre-transplant immunosuppressive therapy followed by myeloablative preparative regimen and allogeneic hematopoietic stem cell transplantation from a haploidentical donor in patients with sickle cell disease.
The overall goal of this study is to expand the donor pool for hematopoietic stem cell transplantation in sickle cell disease using haploidentical donors, and to develop a non-toxic, myeloablative regimen, with the goal of achieving a consistent donor chimerism utilizing pre-transplant immunosuppressive therapy.
Detailed Description:
All patients will receive an haploidentical hematopoietic stem cell transplant with the following conditioning and GvHD prevention:
Pre-transplant immunosuppressive therapy:
2 cycles of Fludarabine and Dexamethasone x 5 days each cycle
Conditioning regimen:
rATG daily x 3 days, Fludarabine daily x 6 days and Busulfan daily x 4 days
GVHD prophylaxis:
Cyclophosphamide day +3 and +4, Tacrolimus and Mycophenolate mofetil
Study:
NCT03279094
Study Brief:
Protocol Section:
NCT03279094