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)

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

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.

Sponsors & Collaborators

• Memorial Sloan Kettering Cancer Center

  lead OTHER

Principal Investigators

• Maria Cancio, MD · Memorial Sloan Kettering Cancer Center

Study Design

Allocation

NA

Purpose

TREATMENT

Masking

NONE

Model

SINGLE_GROUP

Eligibility

Min Age

2 Years

Max Age

50 Years

Sex

ALL

Healthy Volunteers

No

Timeline & Regulatory

Start

2023-02-09

Primary Completion

2027-02-09

Completion

2027-02-09

FDA Drug

Yes

Countries

• United States

Study Locations