RecruitingNCT04671212

Discarded Bone Marrow for Hematology Research


Sponsor

St. Jude Children's Research Hospital

Enrollment

50 participants

Start Date

Jul 26, 2022

Study Type

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

Plain Language Summary

Simplified for easier understanding

This study is collecting discarded bone marrow from orthopedic surgeries in patients with sickle cell disease or severe thalassemia. During certain bone surgeries, marrow-containing bone material is typically discarded as medical waste. This study collects and stores that material for research into new treatments for these blood disorders — particularly gene therapy and bone marrow transplantation approaches. No additional procedures are required beyond the patient's planned surgery. The research aims to better understand how stem cells from these patients behave, which could eventually lead to improved therapies for sickle cell disease and thalassemia. You may be eligible if: - You have sickle cell disease (SS or S-beta thalassemia) and are 2 years or older, OR - You have HbE-beta-thalassemia or severe beta-thalassemia (transfusion-dependent or severe) - You are already scheduled for orthopedic surgery that will involve discarded bone You may NOT be eligible if: - You are currently experiencing a sickle cell crisis, acute chest syndrome, stroke, or active infection - You are pregnant - Your platelet or white blood cell count is too low, or your clotting times are abnormal - You have a history of excessive bleeding during previous procedures Talk to your doctor to see if this trial is right for you.

This summary was AI-generated to explain the trial in plain language. It is not medical advice. Always discuss eligibility with your doctor before enrolling in a clinical trial.

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Locations(1)

St. Jude Children's Research Hospital

Memphis, Tennessee, United States

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NCT04671212


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