A Study of Immune Suppression Treatment for People With Sickle Cell Disease or β-Thalassemia Who Are Going to Receive an Allogeneic Hematopoietic Cell Transplantation (HCT)
Pre-Transplant Immune Suppression With Hematopoietic Cell Transplantation From Haploidentical Donors for Adults and Children With Sickle Cell Disease or ß-Thalassemia (Haplo PTCy)
About This Trial
Hematopoietic Cell Transplantation/HCT involves receiving healthy blood-forming cells (stem cells) from a donor to replace the diseased or damaged cells in participants' bone marrow. The researchers think giving participants treatment with fludarabine and dexamethasone, drugs that lower the activity of the body's immune system (immune suppression), before standard conditioning therapy and HCT may help prevent serious side effects, including graft failure and GvHD. In this study, depending on how participants' body responds to the fludarabine and dexamethasone, the study doctor may decide participants should receive another drug, called cyclophosphamide, instead of fludarabine. In addition, depending on the results of participants' routine blood tests, participants may receive the drugs bortezomib and rituximab, which also help with immune suppression.
Who May Be Eligible (Plain English)
Original Eligibility Criteria
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Treatments Being Tested
Fludarabine
PK-guided fludarabine dosing will be used for each of the 2 cycles, using the InsightRx DoseMeRx platform.
Cyclophosphamide
Cyclophosphamide will be administered Post-Transplant
Tacrolimus
Tacrolimus will be administered beginning on day +5
Mycophenolate Mofetil
Mycophenolate mofetil (MMF) will be administered three times daily starting on day +5.
Rabbit ATG
The dose and schedule of ATG will be determined according to the nomogram in Appendix A
Dexamethasone
Standard Regimen: Dexamethasone on days -68 to -64 and days -40 to -36.
Bortezomib
Bortezomib on days -71, -68, -65, -61, -43, -40, -37, and -33
Rituximab
Rituximab on days -71, -58, -43, and -30.