Non-Myeloablative Bone Marrow Transplant for Patients With Sickle Cell Anemia and Other Blood Disorders

Summary

RATIONALE: Giving low doses of chemotherapy, such as fludarabine and cyclophosphamide, and total-body irradiation before a donor bone marrow transplant helps stop the growth of abnormal cells. It also helps stop the patient's immune system from rejecting the donor's stem cells. When the healthy stem cells from a donor are infused into the patient they may help the patient's bone marrow make stem cells, red blood cells, white blood cells, and platelets. Sometimes the transplanted cells from a donor can make an immune response against the body's normal cells. Giving sirolimus and mycophenolate mofetil after transplant may stop this from happening.

PURPOSE: This phase II trial is studying how well giving fludarabine and cyclophosphamide together with total-body irradiation followed by a donor bone marrow transplant works in treating patients with sickle cell anemia and other blood disorders.

Conditions

• Sickle Cell Disease

Interventions

DRUG

Cyclophosphamide

Cyclophosphamide (Cy) 14.5 mg/kg/day intravenously (IV) on Days -6 and -5 and 50 mg/kg/day IV on Days +3 and +4.

DRUG

Fludarabine

Fludarabine 30 mg/m\^2/day IV on Days -6, -5, -4, -3, and -2.

DRUG

Mycophenolate mofetil

Mycophenolate mofetil 15 mg/kg by mouth (PO) three times a day from Day +5 to Day +35.

DRUG

Sirolimus

The first dose of Sirolimus is 6 mg PO on Day +5. Further dosing is adjusted according to drug levels. Sirolimus is continued through Day +365.

PROCEDURE

Allogeneic bone marrow transplant

An allogeneic bone marrow transplant is a procedure that involves taking bone marrow from a donor and giving it to a recipient.

RADIATION

Total body irradiation - 200

200 centigray (cGy) in one fraction on Day -1.

DRUG

Levetiracetam

Given at 500 mg PO twice daily from Day -6 to Day +365.

BIOLOGICAL

Anti-thymocyte globulin

Test dose of 0.5 mg/kg IV given on Day -9, then 2 mg/kg/day IV on Day -8 and -7.

RADIATION

Total body irradiation - 400

400 centigray (cGy) in one fraction on Day -1.

Sponsors & Collaborators

• National Cancer Institute (NCI)

  collaborator NIH

• Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins

  lead OTHER

Principal Investigators

• Javier Bolanos-Meade, MD · Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins

Study Design

Allocation

NON_RANDOMIZED

Purpose

TREATMENT

Masking

NONE

Model

SINGLE_GROUP

Eligibility

Min Age

2 Years

Max Age

70 Years

Sex

ALL

Healthy Volunteers

Yes

Timeline & Regulatory

Start

2008-06-23

Primary Completion

2018-12-29

Completion

2018-12-29

Countries

• United States

Study Locations