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Observational Study of Discarded Bone Marrow for Hemoglobinopathy Research

This study aims to collect and store discarded bone marrow-containing bone samples from individuals with hemoglobinopathies (like sickle cell disease and beta-thalassemia) and those without these conditions. Researchers want to understand how to best work with hematopoietic stem and progenitor cells (HSPCs) from patients with hemoglobinopathies using gene therapy and gene editing technologies. The goal is to develop better treatments for these inherited anemias. You may be able to participate if you are having orthopedic surgery that involves discarding bone marrow-containing bone, and if you have certain types of hemoglobinopathy (homozygous S/S disease or doubly heterozygous for S and β thalassemia) and are at least two years old. The study is collecting samples for about 10 years to build a resource for future research.

Study design
This is an observational study, meaning researchers will collect samples and information without giving any new treatments. It plans to include about 50 participants.
What's involved
Not specified in the trial record.
Compensation
Not stated in the trial record.
Follow-up
Samples will be collected and processed, with the primary endpoint measured after approximately 10 years of sample collection.

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NCT04671212

Discarded Bone Marrow for Hematology Research

Recruiting
Not specifiedAll AgesObservational
St. Jude Children's Research Hospital
~50 participants
Updated 2026-05-05 on ClinicalTrials.gov

At a glance

Recruiting sites
1 of 1 listed site is recruiting right now
RecruitingSuspended, closed, or not yet open
What they're measuring
Collection, processing and storage of discarded bone marrow-containing bone samples from hemoglobinopathy, as well as non-hemoglobinopathy individuals.
Measured over After completion of sample collection, approximately 10 years

Conditions

Hemoglobinopathies

Explore related trials

Beta ThalassemiaHemoglobinopathySickle Cell Disease

Where it's being run

1 sites across 1 states
Tennessee1

Study leadership

  • Shannon McKinney-Freeman, Ph.D. · PRINCIPAL_INVESTIGATOR · St. Jude Children's Research Hospital

Who to contact

Shannon McKinney-Freeman, Ph.D.
[email protected]
888-226-4343
Email the study team

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Do you actually qualify for this trial?

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Eligibility criteria

Inclusion

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

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

What this trial measures

  • Collection, processing and storage of discarded bone marrow-containing bone samples from hemoglobinopathy, as well as non-hemoglobinopathy individuals.After completion of sample collection, approximately 10 years

    Discarded bone marrow-containing bone samples will be collected for use in research; 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. Samples may be subject to comprehensive assessment of hematopoietic activity using tissue culture based assays, as well as molecular profiling studies of global transcriptome and epigenomes.