Bone Marrow Transplant From Donor Using Less Toxic Conditioning for Patient With High Risk Hemoglobinopathies

• Status: Terminated
• Trial end date: 2003-11-21
• Target enrollment: 0
• Participant gender: All

Summary

The major goal of this study is to determine the risks and benefits of stem cell transplants in combination with a newer, less toxic conditioning chemotherapy treatment in patients with severe sickle cell disease (SCD) or sickle hemoglobin variants (hemoglobin SC or hemoglobin SB0/+), or homozygous b0/+ thalassemia or severe B0/+ thalassemia variants. Participation in this project will be for one year, with follow up evaluations done every 6 months thereafter for 10 years or until participants are 18 years old.

• Phase: Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Baylor College of Medicine
• Collaborators: Texas Children's Hospital; The Methodist Hospital Research Institute; The Methodist Hospital System
• Treatments: Alemtuzumab; Fludarabine; Lenograstim; Sargramostim

Criteria

Inclusion:

• Patients with a haploidentical related HLA donor and hemoglobin SS, hemoglobin SC, or hemoglobin Sb0/+ thalassemia and at least one of the following conditions:

1. previous central nervous system vaso-occlusive episode with or without residual neurologic findings;

2. frequent painful vaso-occlusive episodes which significantly interfere with normal life activities and which necessitate chronic transfusion therapy;

3. recurrent SCD chest syndrome events, which necessitate chronic transfusion therapy;

4. severe anemia, which prevents acceptable quality of life and necessitates chronic transfusion therapy.

• Patients with a haploidentical related HLA donor and homozygous b0/+ thalassemia or severe variants of b0/+ thalassemia and require chronic transfusion therapy.

• Women of childbearing potential must have a negative pregnancy test.

• Between the ages of birth and 65 years.

Exclusion:

• HLA identical or 5/6 HLA matched sibling donor

• Biopsy proven chronic active hepatitis or portal fibrosis.

• 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.

• Unspecified chronic toxicity serious enough to detrimentally affect the patient's capacity to tolerate Stem Cell Transplant.

• Patient or guardian(s) unable to understand the nature and risks inherent in the stem cell transplant process.

• Pregnant or lactating females and those unwilling to use acceptable contraception.