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)

PHASE2RecruitingINTERVENTIONAL
Enrollment

24

Participants

Timeline

Start Date

February 9, 2023

Primary Completion Date

February 9, 2026

Study Completion Date

February 9, 2026

Conditions
Sickle Cell DiseaseThalassemia, BetaThalassemia
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.

Trial Locations (6)

10604

RECRUITING

Memorial Sloan Kettering Westchester (Consent only), Harrison

11553

RECRUITING

Memorial Sloan Kettering Nassau (All protocol activities), Rockville Centre

11725

RECRUITING

Memorial Sloan Kettering Suffolk - Commack (Consent only), Commack

07920

RECRUITING

Memorial Sloan Kettering at Basking Ridge (Consent only), Basking Ridge

07748

RECRUITING

Memorial Sloan Kettering Monmouth (Consent only), Middletown

07645

RECRUITING

Memorial Sloan Kettering Bergen (Consent only), Montvale

All Listed Sponsors
lead

Memorial Sloan Kettering Cancer Center

OTHER