12
Participants
Start Date
January 10, 2023
Primary Completion Date
October 31, 2032
Study Completion Date
October 31, 2034
Cyclophosphamide 1200 mg/Kg
Cyclophosphamide 1200 mg/Kg will be administered as part of the conditioning regimen A prior to HSCT
Fludarabine
Fludarabine (starting dose 0.5 mg/Kg and then PK guided to reach an AUC of 18-20) will be administered as part of the conditioning regimen prior to HSCT
Cyclophosphamide 100 mg/Kg
Cyclophosphamide 100 mg/Kg will be administered as part of the conditioning regimen B prior to HSCT
Total Body Irradiation
Total Body Irradiation 200 cGy will be administered as part of the conditioning regimen prior to HSCT
ATG
ATG 7.5 mg/Kg will be administered as part of the conditioning regimen prior to HSCT
Rituximab
Rituximab 200 mg/m2 will be administered within 24 hours of the HSCT
Melphalan
Melphalan 100 mg/m2 will be administered as part of the conditioning regimen prior to HSCT
CliniMACS® TCR α/β Reagent Kit and CliniMACS® CD19 System
CliniMACS® TCRαβ-Biotin and CD19 Systems will be used to create the mobilized peripheral blood stem cells (PBSC) from allogeneic donors depleted of TCRαβ+ T cells and CD19+ B cells to be infused into the patient for the HSCT. The target dose for the number of CD34+ HSC infused is \> 10 x 10\^6 cells/Kg recipient weight. The minimum dose is 2 x 10\^6 cells/Kg. There is no upper limit to the dose of CD34+ HSC infused as long as no more than 1 x 10\^5 TCRαβ+ T-cells/Kg are infused. The target dose of TCRαβ+ T cells/Kg is \< 0.50 x 10\^5.
Kidney Transplant
In the presence of donor myeloid engraftment, at least 3 months post-HSCT, with \> 95% donor CD3+ chimerism, in the absence of signs of active aGvHD or cGvHD (moderate or severe), at least 4 weeks off of immunosuppression for any previously occurring acute or chronic GvHD (except single agent treatment of mild cGvHD), and with a BMI \>18.5, ambulatory and active in addition to the eligibility for the standard of care KT criteria, patients will undergo a living donor KT using same donor as HSCT
RECRUITING
Lucile Packard Children's Hospital, Palo Alto
California Institute for Regenerative Medicine (CIRM)
OTHER
Alice Bertaina
OTHER