Status:
COMPLETED
Bone Marrow for Hemoglobinopathy Research
Lead Sponsor:
St. Jude Children's Research Hospital
Collaborating Sponsors:
National Heart, Lung, and Blood Institute (NHLBI)
Assisi Foundation
Conditions:
Sickle Cell Anemia
Thalassemia
Eligibility:
All Genders
Brief Summary
Human participants affected with sickle cell disease or thalassemia will donate bone marrow for use in experimental laboratory models to study potential new treatments. This is an observational study ...
Detailed Description
These studies are designed to evaluate the potential of retroviral vector mediated gene transfer, gene editing, or drug treatment to correct the pathophysiology of sickle cell anemia and β-thalassemia...
Eligibility Criteria
Inclusion
- Patients with homozygous S/S disease or doubly heterozygous for S and β thalassemia who are 2 years or older are eligible. Patients with HbE- β- thalassemia or homozygous (severe) β-thalassemia are also eligible. Patients with thalassemia include those who are transfusion dependent (major) or severely anemic but relatively transfusion independent (intermedia). Diagnostic criteria include standard hematological parameters, red cell indices, hemoglobin electrophoresis and quantitative determination of HbF and HbA2.
- Patients are eligible for participation in the protocol only if they are currently clinically stable and have been free of all acute disease manifestations for a minimum of 14 days.
- Patients may participate while continuing their current therapeutic regimen including regular transfusion therapy or hydroxyurea administration.
- In general, two categories of patients will be considered as research participants in this protocol.
- Patients who are 18 years or older and therefore able to provide informed consent will be eligible. Such individuals will be recruited from among patients followed at SJCRH. In addition, individuals followed in an outside clinic who are recruited will be asked to come to the Hematology Clinic at SJCRH to enroll and have the procedure performed. Alternatively, if a patient who is 18 or older is to undergo a diagnostic or surgical procedure under general anesthesia, and they agree to participate in the study, the bone marrow aspirate will be obtained at that time.
- Patients between the ages of 2 and 17 years who are scheduled for a diagnostic or surgical procedure at SJCRH or LeBonheur Children's Medical Center for which sedation or general anesthesia is indicated will be eligible for protocol enrollment. A bone marrow aspiration will be performed during the sedation or general anesthesia for the diagnostic or surgical procedure.
Exclusion
- Active, acute manifestations of sickle cell disease including painful crisis, acute chest syndrome, cerebrovascular events or active infection.
- Pregnant women will not be eligible for study enrollment.
- Inability or unwillingness of the research participant or legal guardian/representative to give written informed consent will preclude enrollment on this research protocol.
- Platelet count \< 150,000/mm\^3
- Neutrophil count \< 2000/mm\^3 (unless on hydroxyurea therapy)
- Neutrophil count \< 1000/mm\^3 for patients on hydroxyurea therapy
- Prothrombin Time \> 17 seconds
- Partial thromboplastin Time \> 43 seconds
- History of excessive bleeding in the context of previous procedures including surgery and dental extractions
Key Trial Info
Start Date :
May 15 2008
Trial Type :
OBSERVATIONAL
Allocation :
ACTUAL
End Date :
August 29 2019
Estimated Enrollment :
26 Patients enrolled
Trial Details
Trial ID
NCT00669305
Start Date
May 15 2008
End Date
August 29 2019
Last Update
February 25 2020
Active Locations (1)
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1
St. Jude Children's Research Hospital
Memphis, Tennessee, United States, 38105