Actively Recruiting
Discarded Bone Marrow for Hematology Research to Study Hemoglobinopathies Using Gene Therapy and Drug Strategies
Led by St. Jude Children's Research Hospital · Updated on 2026-05-05
50
Participants Needed
1
Research Sites
260 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
This research aims to collect and study discarded bone marrow-containing bone samples from patients with hemoglobinopathies, such as sickle cell disease (SCD) and thalassemia, as well as individuals without these conditions. The goal is to better understand how to manipulate hematopoietic stem and progenitor cells (HSPCs) from hemoglobinopathy patients using gene therapy, gene editing, and drug treatments to potentially develop curative therapies. The study also seeks to characterize the biology and function of bone marrow cells isolated from these patients. Bone marrow-containing bone samples that are normally discarded during orthopedic surgeries will be collected from patients undergoing these procedures, including those with hemoglobinopathies and those without. The samples will be processed, stored, and shipped to the research center for experimental studies aimed at developing gene transfer, gene editing, and drug treatment strategies. This study does not involve administering treatments to participants but focuses on laboratory research using the collected samples. Participants will be patients undergoing orthopedic surgery where bone marrow-containing bone is discarded. Researchers will collect and process these samples over approximately ten years to build a repository for future studies. The study includes detailed laboratory assessments to analyze cellular function and potential for transplantation. Safety monitoring and direct patient interventions are not part of this observational study, and participants' usual medical care will continue uninterrupted.
CONDITIONS
Brief Title
Discarded Bone Marrow for Hematology Research
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Patients of any age receiving orthopedic surgery for clinical management involving bone marrow-containing bone discard
- Patients with homozygous S/S sickle cell disease or doubly heterozygous for S and beta thalassemia who are 2 years or older
- Patients with HbE-beta thalassemia or severe beta-thalassemia, including transfusion dependent or severely anemic but relatively transfusion independent individuals
- Diagnosis based on standard hematological parameters, red cell indices, hemoglobin electrophoresis, and quantitative determination of HbF and HbA2
You will not qualify if you...
- Active, acute sickle cell disease symptoms including painful crisis, acute chest syndrome, cerebrovascular events, or active infection
- Pregnant women
- Inability or unwillingness to give written informed consent
- Platelet count less than 150,000/mm^3
- Neutrophil count less than 2000/mm^3, or less than 1000/mm^3 for patients on hydroxyurea therapy
- Prothrombin time greater than 17 seconds
- Partial thromboplastin time greater than 43 seconds
- History of excessive bleeding during previous procedures including surgery and dental extractions
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person) during preoperative evaluation
Duration - Day of surgery
Participants undergo orthopedic surgery during which bone marrow-containing bone samples that would normally be discarded are collected for research.
1 surgery visit
Duration - Up to 10 years after sample collection
Participants who undergo orthopedic surgery are observed and their discarded bone marrow samples are processed and studied to better understand hemoglobinopathies and test gene therapy and drug strategies.
No additional visits required for research purposes
Trial Site Locations
Total: 1 location
1
St. Jude Children's Research Hospital
Memphis, Tennessee, United States, 38105
Actively Recruiting
Research Team
S
Shannon McKinney-Freeman, Ph.D.
How is the study designed?
Study Type
OBSERVATIONAL
Masking
N/A
Allocation
N/A
Model
N/A
Primary Purpose
N/A
Number of Arms
1
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