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RECRUITING
NCT05736419
PHASE2

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

View on ClinicalTrials.gov

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

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.

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