Discarded Bone Marrow for Hematology Research
St. Jude Children's Research Hospital
50 participants
Jul 26, 2022
OBSERVATIONAL
Conditions
Summary
The primary objective of this study is to establish a mechanism to obtain discarded bone marrow-containing bone samples from hemoglobinopathy, as well as non-hemoglobinopathy individuals. The processing of samples will help to understand how best to manipulate HSPC's from hemoglobinopathy patients with gene therapy and gene technologies in the laboratory environment. It will also allow us to establish a reservoir of samples that can be studied in the future to assess cellular function and fitness for transplant. Secondary objectives * To develop gene transfer and gene editing strategies as potentially curative therapy for hemoglobinopathies (e.g. sickle cell disease (SCD) and β-thalassemia). * To develop a drug treatment strategy which elevates the expression of fetal hemoglobin to a potentially curative level for hemoglobinopathies. * To examine the biology of bone marrow cells isolated from patients with hemoglobinopathies.
Eligibility
Inclusion Criteria4
- Patients of any age receiving orthopedic surgery for clinical management that involve bone marrow containing bone discard.
- Patients receiving orthopedic surgery for clinical management will be considered for this study if they have the following diagnosis and criteria:
- Homozygous S/S disease or doubly heterozygous for S and β thalassemia who are two years or older are eligible.
- HbE-β- thalassemia or homozygous (severe) β-thalassemia. including 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.
Exclusion Criteria9
- 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
- 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
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Locations(1)
View Full Details on ClinicalTrials.gov
For the most up-to-date information, visit the official listing.
NCT04671212