RecruitingPhase 2NCT05736419

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)


Sponsor

Memorial Sloan Kettering Cancer Center

Enrollment

24 participants

Start Date

Feb 9, 2023

Study Type

INTERVENTIONAL

Conditions

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.


Eligibility

Min Age: 2 YearsMax Age: 50 Years

Plain Language Summary

Simplified for easier understanding

This clinical trial is studying a drug called Bortezomib, a drug called Cyclophosphamide, and others for people with sickle cell disease, thalassemia, and other related conditions. The study is currently recruiting participants at 6 locations. People eligible for this study include aged 2 Years to 50 Years.

This summary was AI-generated to explain the trial in plain language. It is not medical advice. Always discuss eligibility with your doctor before enrolling in a clinical trial.

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Interventions

DRUGFludarabine

PK-guided fludarabine dosing will be used for each of the 2 cycles, using the InsightRx DoseMeRx platform.

DRUGCyclophosphamide

Cyclophosphamide will be administered Post-Transplant

DRUGTacrolimus

Tacrolimus will be administered beginning on day +5

DRUGMycophenolate Mofetil

Mycophenolate mofetil (MMF) will be administered three times daily starting on day +5.

BIOLOGICALRabbit ATG

The dose and schedule of ATG will be determined according to the nomogram in Appendix A

DRUGDexamethasone

Standard Regimen: Dexamethasone on days -68 to -64 and days -40 to -36.

DRUGBortezomib

Bortezomib on days -71, -68, -65, -61, -43, -40, -37, and -33

DRUGRituximab

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

View Full Details on ClinicalTrials.gov

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NCT05736419


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