NCT05736419

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)

• Status: Recruiting
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 24 (estimated)
• Sponsor: Memorial Sloan Kettering Cancer Center · Academic / Other
• Sex: All
• Age: 2 Years – 50 Years
• Healthy volunteers: Not accepted

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.

Conditions

• Sickle Cell Disease
• Thalassemia, Beta
• Thalassemia

Interventions

Type	Name	Description
DRUG	Fludarabine	PK-guided fludarabine dosing will be used for each of the 2 cycles, using the InsightRx DoseMeRx platform.
DRUG	Cyclophosphamide	Cyclophosphamide will be administered Post-Transplant
DRUG	Tacrolimus	Tacrolimus will be administered beginning on day +5
DRUG	Mycophenolate Mofetil	Mycophenolate mofetil (MMF) will be administered three times daily starting on day +5.
BIOLOGICAL	Rabbit ATG	The dose and schedule of ATG will be determined according to the nomogram in Appendix A
DRUG	Dexamethasone	Standard Regimen: Dexamethasone on days -68 to -64 and days -40 to -36.
DRUG	Bortezomib	Bortezomib on days -71, -68, -65, -61, -43, -40, -37, and -33
DRUG	Rituximab	Rituximab on days -71, -58, -43, and -30.

Timeline

• Start date: 2023-02-09
• Primary completion: 2027-02-09
• Completion: 2027-02-09
• First posted: 2023-02-21
• Last updated: 2026-04-08

Locations

6 sites across 1 country: United States

Regulatory

• FDA-regulated drug study