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Leftover bone marrow could unlock better gene therapies for sickle cell disease

NCT ID NCT04671212

Knowledge-focused Sponsor: St. Jude Children's Research Hospital Source: ClinicalTrials.gov ↗

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now This study
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Jun 27, 2026 · Last updated Jun 27, 2026

Summary

This study collects bone marrow that is normally thrown away during surgery from people with and without blood disorders like sickle cell disease. Researchers will use these samples in the lab to learn how to better manipulate stem cells for gene therapy and to study cell health. The goal is to develop more effective treatments, but this study does not provide any direct treatment to participants.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Participants

About 50 people

The number the study aims to enrol. It can still change while the study runs.

Started

Jul 2022

Expected to finish

Jan 2035

An estimate. End dates often move.

Lead sponsor

Other sponsor

The registry's catch-all category, for sponsors it does not file as a company, a government agency, or a research network.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Who is studied

Participants who meet the Eligibility Criteria.

Ages

Children (under 18), adults (18 to 64) and older adults (65 and over)

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

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

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Conditions

The condition(s) this trial relates to.

beta thalassemia hemoglobin E-beta-thalassemia syndrome hemoglobinopathy inherited hemoglobinopathy sickle cell disease

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Hemoglobinopathies

How to take part

Only the study team decides who joins. These are the ways to reach them.

  1. The study's own enquiry address

    This study publishes an address for enquiries. See it below .

  2. The places running it

    1 site. The list below names each one and where it is.

  3. The official record

    ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.

    Open the record ↗

  4. A doctor treating you

    A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.

Contacts and locations

Study contacts

  • Contact

    Email: •••••@•••••

Locations

  • St. Jude Children's Research Hospital

    RECRUITING

    Memphis, Tennessee, 38105, United States

    Contact Email: •••••@•••••

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