Haploidentical Hematopoietic Stem Cell Transplantation

PHASE1CompletedINTERVENTIONAL
Enrollment

7

Participants

Timeline

Start Date

January 31, 2015

Primary Completion Date

January 31, 2024

Study Completion Date

October 31, 2024

Conditions
Sickle Cell-thalassemia DiseaseThalassemia
Interventions
DRUG

peripheral blood stem cell graft that are CD34+ selected

The preparatory regimen will consist of Hydroxyurea from Days -50 to -22, Alemtuzumab from days -21 to -19 (test dose Alemtuzumab on day -22), Fludarabine days -8 to -4, Thiotepa Day -4, Melphalan day -3 to -2 (Table 4a). In patients with intolerance to or have received alemtuzumab in the prior 6 months, alemtuzumab will be replaced with rabbit ATG on days -10 through -7, followed by infusion of a peripheral blood stem cell graft collected from haploidentical family donors that are CD34+ positively selected using the CliniMACS device. Sirolimus will be used for GVHD prophylaxis and given for 9 months post-transplant and then tapered off by one year.

Trial Locations (1)

20010

Childrens National Medical Center, Washington D.C.

All Listed Sponsors
collaborator

Children's National Research Institute

OTHER

lead

Catherine Bollard

OTHER