Nonmyeloablative Peripheral Blood Mobilized Hematopoietic Precursor Cell Transplantation for Sickle Cell Disease and Beta-thalassemia in People With Higher Risk of Transplant Failure

Summary

Background:

\- Some sickle cell disease or beta-thalassemia can be cured with transplant. Researchers want to test a variation of transplant that uses low dose radiation and a combination of immunosuppressive drugs. They want to know if it helps a body to better accept donor stem cells.

Objectives:

\- To see if low dose radiation (300 rads), oral cyclophosphamide, pentostatin, and sirolimus help a body to better accept donor stem cells.

Eligibility:

\- People 4 and older with beta-thalassemia or sickle cell disease that can be cured with transplant, and their donors.

Design:

* Participants and donors will be screened with medical history, physical exam, blood test, tissue and blood typing, and bone marrow sampling. They will visit a social worker.
* Donors:
* may receive an intravenous (IV) tube in their groin vein.
* will receive a drug injection daily for 5 or 6 days to move the blood stem cells from the bone marrow into general blood circulation.
* will undergo apheresis: an IV is put into a vein in each arm. Blood is taken from one arm, a machine removes the white blood cells that contain blood stem cells, and the rest is returned through the other arm.
* Participants:
* may undergo red cell exchange procedure.
* will remain in the hospital for about 30 days.
* will receive a large IV line that can stay in their body from transplant through recovery.
* will receive a dose of radiation, and transplant related drugs by mouth or IV.
* will receive blood stem cells over 8 hours by IV.
* will take neuropsychological tests and may complete questionnaires throughout the transplant process.
* must stay near NIH for 4 months. They will visit the outpatient clinic weekly.

Conditions

• Sickle Cell Disease
• Thalassemia
• Stem Cell Transplantation
• Graft vs Host Disease

Interventions

DRUG

Alemtuzumab

Immunosuppressant

DRUG

Sirolimus

Immunosuppressant

DRUG

Cyclophosphamide

Immunosuppressant

DRUG

Pentostatin

Immunosuppressant

PROCEDURE

Radiotherapy

Immunosuppressant and myelosuppressant

DRUG

Filgrastim

mobilize peripheral blood stem cells for apheresis collection

Sponsors & Collaborators

• National Cancer Institute (NCI)

  collaborator NIH

• National Institutes of Health Clinical Center (CC)

  collaborator NIH

• National Heart, Lung, and Blood Institute (NHLBI)

  lead NIH

Principal Investigators

• Matthew M Hsieh, M.D. · National Heart, Lung, and Blood Institute (NHLBI)

Study Design

Allocation

NON_RANDOMIZED

Purpose

TREATMENT

Masking

NONE

Model

PARALLEL

Eligibility

Min Age

4 Years

Max Age

80 Years

Sex

ALL

Healthy Volunteers

Yes

Timeline & Regulatory

Start

2014-04-21

Primary Completion

2023-12-31

Completion

2027-12-31

FDA Drug

Yes

Countries

• United States

Study Locations