Sickle Cell Disease Clinical Trial
— SCALLOPOfficial title:
Allogeneic Bone Marrow Transplantation From HLA Identical Related Donors for Patients With Hemoglobinopathies: Hemoglobin SS, Hemoglobin SC, or Hemoglobin SB0/+ Thalassemia
Verified date | July 2020 |
Source | Baylor College of Medicine |
Contact | n/a |
Is FDA regulated | No |
Health authority | |
Study type | Interventional |
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.
Status | Terminated |
Enrollment | 8 |
Est. completion date | July 2012 |
Est. primary completion date | July 2012 |
Accepts healthy volunteers | No |
Gender | All |
Age group | N/A to 40 Years |
Eligibility |
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. |
Country | Name | City | State |
---|---|---|---|
United States | Methodist Hospital | Houston | Texas |
United States | Texas Children's Hospital | Houston | Texas |
Lead Sponsor | Collaborator |
---|---|
Tami D. John | Baylor College of Medicine, The Methodist Hospital System |
United States,
Type | Measure | Description | Time frame | Safety issue |
---|---|---|---|---|
Primary | Number of Participants Evaluated for Evidence of Recovery of Organ Function Measured Via MRI or PET Scan. | Assess Number of participants that recovered organ function diagnosed with sickle cell disease (SCD) or sickle hemoglobin variants after undergoing allogeneic SCT/BMT from HLA genotype identical donors. | One year | |
Primary | Number of Participants With Pre and Post Transplant PET Scan to Assess Organ Recovery Based on Rate of Acquisition. | Number of participants that did or did not have pre- and post- PET scans. | One year | |
Primary | Number of Participants With Immune Response to Immunization After BMT in Participants With SCD, Hemoglobin SC, or Hemoglobin Sb0/+. | Vaccine response will be measured via a humoral immunity panel evaluating streptococcus pneumonia IgG antibodies (microgram/mL). Panels are obtained pre and 1+ month post vaccination. Standard recommendations define a normal responder as having >4 fold increase in antibody level in 50-70% of the serotypes found in the vaccine. | up to 12 months |
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