Bone Marrow Transplantation, Hemoglobinopathies, SCALLOP

• Status: Terminated
• Trial end date: 2012-07-01
• Target enrollment: 0
• Participant gender: All

Summary

Patients are being asked to participate in this study because they have severe sickle cell anemia (SCD) with or without the beta thalassemia trait. Sickle cell anemia is an illness where the red blood cells change shape and can clog up blood vessels. This keeps the body from getting the oxygen it needs. Thalassemia is when the body does not make enough hemoglobin, something that helps the oxygen get to the places it needs to go in the body. The patient may or may not need to get regular blood transfusions (getting more blood) to improve their quality of life (feel better) and prevent organ damage (problems with the brain, heart, lung, kidney, and gonad, for example.). The transfusions can also cause problems, including iron overload (too much iron in the blood), which can be fatal (patients can die) without regular deferoxamine shots. Even with the best usual treatments, people with thalassemia or SCD die sooner. There is no proven cure.

We would like to treat patients using bone marrow transplantation, a treatment that has been used for people with SCD. The transplant uses healthy "matched" bone marrow. This comes from a brother or sister who does not have sickle cell disease or severe thalassemia. If the treatment works, the sickle cell disease or thalassemia may be cured. This treatment has been used to treat patients with sickle cell disease or thalassemia. It has worked in most cases. We hope, but cannot promise, that the transplanted marrow will make healthy cells, and patients will not have sickle cell disease or severe thalassemia anymore.

We do not know what effect this treatment will have on the damage that has already been done by the disease. Finding that out is the main reason for this study. Currently, very little has been reported about organ function after bone marrow transplants in patients with sickle cell anemia.

• Phase: N/A
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Baylor College of Medicine; Tami D. John
• Collaborators: Baylor College of Medicine; Texas Children's Hospital; The Methodist Hospital Research Institute; The Methodist Hospital System
• Treatments: Alemtuzumab; Busulfan; Cyclophosphamide; Mesna

Criteria

Inclusion Criteria:

1. Patients with a related HLA genotype identical donor and hemoglobin SS, hemoglobin SC, or hemoglobin Sb0/+ and at least one of the following conditions:

1. Previous central nervous system vaso-occlusive episode with or without residual neurologic findings, or has an abnormal transcranial doppler exam without neurologic findings, or abnormal MRI/MRA of the brain with or without 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;

5. Any of the above symptoms in which the patient is not undergoing chronic transfusion therapy;

6. The patient is undergoing chronic transfusion therapy for symptoms other than those listed and which significantly interferes with normal life activities;

7. Failed hydroxyurea therapy;

8. Indication of pulmonary hypertension on 2 separate echocardiogram examinations;

9. Patients who plan to return to resource poor areas/countries.

2. Between the ages of birth and 40 years.

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

EXCLUSION CRITERIA:

1. Patient with biopsy proven chronic active hepatitis or fibrosis with portal bridging.

2. Patient with SCD chronic lung disease > stage 3 (see Appendix 1).

3. Patient with severe renal dysfunction defined as creatinine clearance < 40 mL/min/1.73 M^2.

4. Patient with severe cardiac dysfunction defined as echocardiogram shortening fraction < 25% or NYHA class III or IV.

5. Patient with HIV infection.

6. Patient with unspecified chronic toxicity serious enough to detrimentally affect the patient's capacity to tolerate bone marrow transplantation.

7. Patient or patient's guardian(s) unable to understand the nature and risks inherent in the BMT process.

8. Pregnant/lactating women and those unwilling to use acceptable contraception will be excluded.

9. Patient or patient's guardian who have not signed an informed consent.

NOTE: Patients who would be excluded from the protocol strictly for laboratory abnormalities can be included at the investigator's discretion after approval by the CAGT Protocol Review Committee and the FDA reviewer.