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

Sponsor

Baylor College of Medicine (Other)

Overall Status

Terminated

CT.gov ID

NCT00040469

Collaborator

The Methodist Hospital Research Institute (Other), Center for Cell and Gene Therapy, Baylor College of Medicine (Other)

Enrollment

Locations

39.7

Duration (Months)

7.5

Patients Per Site

0.2

Patients Per Site Per Month

Study Details

Study Description

Brief Summary

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.

Condition or Disease	Intervention/Treatment	Phase
Sickle Cell Anemia Hemoglobinopathy Thalassemia	Drug: Campath -1H Drug: Dilantin Drug: Busulfan Drug: Cyclophosphamide	Phase 2

Detailed 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.

Study Design

Study Type:

Interventional

Allocation:

Non-Randomized

Intervention Model:

Single Group Assignment

Masking:

None (Open Label)

Primary Purpose:

Treatment

Official Title:

Allogeneic Bone Marrow Transplant From HLA Identical Related Donors for Patients With High Risk Hemoglobinopathies: Hemoglobin SS, Hemoglobin SC, Hemoglobin SB0/+ Thalassemia, or Homozygous B0/+ Thalassemia or Severe Variants of B0/+ Thalassemia

Study Start Date :

Aug 1, 2000

Actual Primary Completion Date :

Nov 21, 2003

Actual Study Completion Date :

Nov 21, 2003

Outcome Measures

Primary Outcome Measures

Eligibility Criteria

Criteria

Ages Eligible for Study:

1 Day to 64 Years

Sexes Eligible for Study:

All

Accepts Healthy Volunteers:

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.