Bone Marrow Transplant From Related Donor for Patients With High Risk Hemoglobinopathies

Baylor College of Medicine

Status and phase

Terminated

Phase 2

Conditions

Thalassemia

Hemoglobinopathy

Sickle Cell Anemia

Treatments

Drug: Campath -1H

Drug: Dilantin

Drug: Busulfan

Drug: Cyclophosphamide

Study type

Interventional

Funder types

Other

Identifiers

NCT00040469

H6847

Scallo2

Details and patient eligibility

About

The major goal of this study is to determine the risks and benefits of bone marrow transplants in patients with severe thalassemia or sickle cell disease. Participation in this project will be for two years.

Full description

To do the bone marrow transplant, we must first kill the cells in the bone marrow that make the abnormal red blood cells that are found in patients with severe thalassemia or sickle cell disease.

We will do this by using three drugs: busulfan, cyclophosphamide, and CAMPATH-1H. CAMPATH-IH is an investigational drug. CAMPATH-1H is used to prevent participants from rejecting or refusing to let the donor blood cells grow in the body. After the drug treatment, participants will be given bone marrow from a brother or sister who has healthy bone marrow that matches.

Sex

All

Ages

1 day to 64 years old

Volunteers

No Healthy Volunteers

Inclusion and exclusion criteria

Inclusion:

Patients with homozygous B0/+ thalassemia or severe variants of B0/+ thalassemia with an HLA genotypically identical donor.
Patients with an HLA genotype identical donor and hemoglobin SS, hemoglobin SC, or hemoglobin Sb 0/+ and at least one of the following:

Previous central nervous system vaso-occlusive episode with or without residual neurologic findings; Frequent painful vaso-occlusive episodes which significantly interfere with normal life activities and which necessitate chronic transfusion therapy; Recurrent SCD chest syndrome events which necessitate chronic transfusion therapy.

Severe anemia which prevents acceptable quality of life and necessitates chronic transfusion therapy.
The patient must have an HLA genotype identical donor.
Between the ages of birth and 65 years.
Women of childbearing potential must have a negative pregnancy test.

Exclusion:

Biopsy proven chronic active hepatitis or fibrosis with portal bridging.
SCD chronic lung disease >/= stage 3.
Severe renal dysfunction defined as creatinine clearance <40 ml/min/1.73 M2
Severe cardiac dysfunction defined as shortening fraction <25%.
HIV infection.
Severe but unspecified chronic toxicity serious enough to detrimentally affect the patient's capacity to tolerate bone marrow transplant (BMT).
Inadequate intellectual capacity to understand the nature and risk inherent in the BMT process and give informed consent (in the case of minors, this criteria must be fulfilled by the legal guardian).
Pregnant, lactating or unwilling to use appropriate birth control.