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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)
Sponsor: Memorial Sloan Kettering Cancer Center
Summary
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.
Official title: Pre-Transplant Immune Suppression With Hematopoietic Cell Transplantation From Haploidentical Donors for Adults and Children With Sickle Cell Disease or ß-Thalassemia (Haplo PTCy)
Key Details
Gender
All
Age Range
2 Years - 50 Years
Study Type
INTERVENTIONAL
Enrollment
24
Start Date
2023-02-09
Completion Date
2027-02-09
Last Updated
2026-04-08
Healthy Volunteers
No
Interventions
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.
Locations (6)
Memorial Sloan Kettering at Basking Ridge (Consent only)
Basking Ridge, New Jersey, United States
Memorial Sloan Kettering Monmouth (Consent only)
Middletown, New Jersey, United States
Memorial Sloan Kettering Bergen (Consent only)
Montvale, New Jersey, United States
Memorial Sloan Kettering Suffolk - Commack (Consent only)
Commack, New York, United States
Memorial Sloan Kettering Westchester (Consent only)
Harrison, New York, United States
Memorial Sloan Kettering Nassau (All protocol activities)
Rockville Centre, New York, United States