Inducible Regulatory T Cells (iTregs) in Non-Myeloablative Sibling Donor Peripheral Blood Stem Cell Transplantation | Aplastic Anemia and MDS International Foundation

Clinical Trial: NCT01634217

Inducible Regulatory T Cells (iTregs) in Non-Myeloablative Sibling Donor Peripheral Blood Stem Cell Transplantation
For more details on this clinical trial, including contact information, please see this trial’s listing on clinicaltrials.gov:
Purpose: 

This is a phase I single center dose escalation study with an extension at the best available dose to determine the tolerability of inducible regulatory T cells (iTregs) when given to adult patients undergoing non-myeloablative HLA-identical sibling donor peripheral blood stem cell (PBSC) transplantation for the treatment of a high risk malignancy. Up to 5 dose cohorts will be tested. Once the tolerable dose is determined for iTregs, enrollment will continue with an additional 10 patients using sirolimus/Mycophenolate mofetil (MMF) graft-versus-host disease (GVHD) prophylaxis to gain further safety information and to provide pilot data in this treatment setting.

Status: 
Recruiting
Study Date: 
Fri, 11/01/2013 to Thu, 12/01/2016
Bone Marrow Disease(s): 
myelodysplastic syndromes (MDS)
Intervention: 
Biological: iTreg The iTregs will be infused at the assigned dose without a filter or pump slowly by gravity over 15-60 minutes. The iTregs should be given at least 4 hours before the peripheral blood stem cell (PBSC) infusion (MT2001-10). Study Arm (s) Experimental: Cohort 1 Administered 3 x 10^6 iTregs/kg infusion Intervention: Biological: iTreg Experimental: Cohort 2 Administered 3 x 10^7 iTregs/kg infusion Intervention: Biological: iTreg Experimental: Cohort 3 Administered 3 x 10^8 iTregs/kg infusion Intervention: Biological: iTreg Experimental: Cohort 4 Administered 10 x 10^8 iTregs/kg infusion Intervention: Biological: iTreg Experimental: Cohort 5 Extension Administered 10 x 10^8 iTregs/kg or best available dose using sirolimus/MMF as graft-versus-host disease (GVHD) prophylaxis. Immunosuppression will consist of a combination of sirolimus and mycophenolate mofetil (MMF). Sirolimus will be administered starting at day -3 with 8mg-12mg oral loading dose followed by single dose 4 mg/day. MMF will be administered starting on day -3 at a dose of 3 gram/day divided in 2 or 3 doses. Intravenous (IV) route between days -3 and +5, then may change to PO between days +6 and +30. Stop MMF at day +30 or 7 days after engraftment, whichever day is later, if no acute GVHD. Intervention: Biological: iTreg