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Discarded Bone Marrow for Hematology Research

NCT04671212 · Status: RECRUITING · Type: OBSERVATIONAL · Enrollment: 50

Last updated 2026-05-05

No results posted yet for this study

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

Sponsors & Collaborators

Principal Investigators

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

Eligibility

Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2022-07-26
Primary Completion
2030-01-31
Completion
2035-01-31

Countries

  • United States

Study Locations

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Entities

Companies

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT04671212 on ClinicalTrials.gov