Using discarded bone marrow to improve treatments for blood disorders
Discarded Bone Marrow for Hematology Research
This study is trying to see if using leftover bone marrow from patients with blood disorders can help develop better gene therapies and drug treatments for conditions like sickle cell disease and β-thalassemia.
Quick facts
| Study type | Observational |
|---|---|
| Enrollment | 50 (estimated) |
| Sex | All |
| Sponsor | St. Jude Children's Research Hospital Academic / other |
| Locations | 1 site (Memphis, Tennessee) |
| Trial ID | NCT04671212 on ClinicalTrials.gov |
What this trial studies
This observational study aims to collect discarded bone marrow samples from individuals with hemoglobinopathies and non-hemoglobinopathies to enhance the understanding of hematopoietic stem and progenitor cells (HSPCs). The research focuses on developing gene therapy and gene editing strategies as potential cures for conditions like sickle cell disease and β-thalassemia. By analyzing these samples, researchers hope to optimize treatment protocols and assess cellular function for future transplant applications. The study also seeks to explore drug treatments that could elevate fetal hemoglobin levels in affected patients.
Who should consider this trial
Good fit: Ideal candidates include patients of any age undergoing orthopedic surgery who have specific diagnoses related to hemoglobinopathies.
Not a fit: Patients experiencing active complications from sickle cell disease or those who are pregnant will not benefit from this study.
Why it matters
Potential benefit: If successful, this research could lead to more effective and accessible treatments for patients with hemoglobinopathies.
How similar studies have performed: Previous studies have shown promise in using gene therapy and editing approaches for hemoglobinopathies, indicating potential success for this research.
Eligibility criteria
Show full inclusion / exclusion criteria
Inclusion Criteria: * 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 Criteria: * 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
Where this trial is running
Memphis, Tennessee
- St. Jude Children's Research Hospital — Memphis, Tennessee, United States (Recruiting)
Study contacts
- Principal investigator: Shannon McKinney-Freeman, Ph.D. — St. Jude Children's Research Hospital
- Study coordinator: Shannon McKinney-Freeman, Ph.D.
- Email: referralinfo@stjude.org
- Phone: 888-226-4343
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.