Discarded Bone Marrow for Hematology Research

NCT04671212 | Recruiting

• 1 other identifier: BMHR
• Study Type: observational
• Target: 50
• Locations: 1 country
• Sites: 1

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.

Conditions

Hemoglobinopathies

Keywords

Bone samples; Bone marrow; Discarded; Hemoglobinopathies; Non-hemoglobinopathies

Outcome Measures

Primary Outcomes (1)

• Collection, processing and storage of discarded bone marrow-containing bone samples from hemoglobinopathy, as well as non-hemoglobinopathy individuals.

  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.

  After completion of sample collection, approximately 10 years

Study Arms (1)

Orthopedic Surgery Patients

Participants receiving orthopedic surgery for clinical management that involve bone marrow containing bone discard.

Eligibility Criteria

• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

Study Population

Participants who meet the Eligibility Criteria.

You may qualify if:

• 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.

You may not qualify if:

• 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

Sponsors & Collaborators

• St. Jude Children's Research Hospital: lead

Study Sites (1)

St. Jude Children's Research Hospital

Memphis, Tennessee, 38105, United States

RECRUITING

Related Links

• St. Jude Children's Research Hospital
• Clinical Trials Open at St. Jude

Biospecimen

Retention: SAMPLES WITH DNA

Bone marrow-containing bone samples

MeSH Terms

Conditions

Hemoglobinopathies

Condition Hierarchy (Ancestors)

Hematologic Diseases; Hemic and Lymphatic Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Officials

• Shannon McKinney-Freeman, Ph.D.

  St. Jude Children's Research Hospital

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Shannon McKinney-Freeman, Ph.D.

CONTACT

888-226-4343; referralinfo@stjude.org

Study Design

• Study Type: observational
• Observational Model: OTHER
• Time Perspective: PROSPECTIVE
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: December 2, 2020
• First Posted: December 17, 2020
• Study Start: July 26, 2022
• Primary Completion (Estimated): January 1, 2030
• Study Completion (Estimated): January 1, 2035
• Last Updated: May 5, 2026

Record last verified: 2026-04

Locations

US (1)