NCT04671212 Discarded Bone Marrow for Hematology Research
| NCT ID | NCT04671212 |
| Status | Recruiting |
| Phase | — |
| Sponsor | St. Jude Children's Research Hospital |
| Condition | Hemoglobinopathies |
| Study Type | OBSERVATIONAL |
| Enrollment | 50 participants |
| Start Date | 2022-07-26 |
| Primary Completion | 2030-01 |
Eligibility & Interventions
Eligibility Fast-Check
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What to Expect as a Participant
This is an observational study. You will not receive an experimental treatment; researchers will collect data based on your existing condition or standard treatment.
This trial targets 50 participants in total. It began in 2022-07-26 with a primary completion date of 2030-01.
⚠ This information is for research awareness only. Always consult your physician before joining any clinical trial. Participation is voluntary and you may withdraw at any time.
Brief 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 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
Contact & Investigator
Shannon McKinney-Freeman, Ph.D.
PRINCIPAL INVESTIGATOR
St. Jude Children's Research Hospital
Frequently Asked Questions
Who can join the NCT04671212 clinical trial?
This trial is open to participants of all sexes, studying Hemoglobinopathies. Full inclusion and exclusion criteria are listed in the Eligibility Criteria section. Always confirm your eligibility with the research team before applying.
Is NCT04671212 currently recruiting?
Yes, NCT04671212 is actively recruiting participants. Contact the research team at referralinfo@stjude.org for enrollment information.
Where is the NCT04671212 trial being conducted?
This trial is being conducted at Memphis, United States.
Who is sponsoring the NCT04671212 clinical trial?
NCT04671212 is sponsored by St. Jude Children's Research Hospital. The principal investigator is Shannon McKinney-Freeman, Ph.D. at St. Jude Children's Research Hospital. The trial plans to enroll 50 participants.