Using discarded bone marrow to improve treatments for blood disorders

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

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.

Why it matters

Who can join: the full rules

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 it's running

• 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

Similar studies

Other studies for the same condition or treatment. Recruiting studies are listed first.

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• Donor transplant for high risk blood disorders

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• Investigating genetic factors in blood disorders

  This study is trying to find out how genes affect blood disorders like sickle cell disease and thalassemia by looking at the DNA of many people with these conditions.

  Recruiting at 17 sites Cyprus Institute of Neurology and Genetics · Boston, Massachusetts and 25 other locations · NCT05799118

• Evaluating a single dose of CTX001 for treating transfusion-dependent beta-thalassemia and severe sickle cell disease

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• Cord blood transplant for children and young adults with blood cancers and disorders

  This study is testing a new treatment using chemotherapy followed by a cord blood transplant to see if it can help children and young adults with serious blood cancers and disorders who don't have a matched donor.

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How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.