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New biobank aims to unlock better treatments for blood diseases

NCT ID NCT07227155

Knowledge-focused Sponsor: Medical College of Wisconsin 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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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 Aug 28, 2026 · Updated 2 times

Summary

This study collects blood samples and health information from 375 people with non-cancerous blood disorders like aplastic anemia, sickle cell disease, or thalassemia who are receiving a stem cell transplant or gene therapy. The goal is to create a biobank that helps researchers understand why these treatments sometimes fail and how to make them safer and more effective. Participants provide samples before and after treatment, but this study does not test a new therapy itself.

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 375 people

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

Started

May 2026

Expected to finish

Mar 2031

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

Patients undergoing HCT or GT for treatment of Non-malignant blood disorders: Aplastic Anemia, Hemoglobinopathies or Bone Marrow Failure. Related donors of enrolled participants undergoing HCT are also eligible for the study.

Ages

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

Sex

Anyone

Healthy volunteers

Accepted

You do not need to have the condition being studied to take part.

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.

HCT/GT Inclusion: 1. Patients with a diagnosis of Aplastic Anemia (AA), hemoglobinopathies or bone marrow failure from other causes except for malignant diseases will be eligible for enrollment on this protocol: 1. AA will be defined as having peripheral blood cytopenias with a hypocellular bone marrow for age and a clinical diagnosis of aplastic anemia as determined by their treating physicians. 2. Hemoglobinopathies include sickle cell disease or thalassemia. Patients receiving potentially curative therapy with HCT or GT for hemoglobinopathies will be eligible for this study. 3. Individuals with bone marrow failure due to clinical or molecularly diagnosed inherited bone marrow failure, inborn errors of immunity or other cause will be included. 2. Patients must receive an HCT or GT for management of their underlying disease. Allogeneic transplants including all conditioning regimens, donors, and GVHD prophylaxis regimens are eligible. This study does not define how the transplant or transplant-supportive care will be performed. 3. Patients or their legal guardian must consent to participate in the CIBMTR "Protocol for a Research Database for Hematopoietic Cell Transplantation and Marrow Toxic Injuries" (NCT 1166009) to allow linkage with the longitudinal clinical data collected by CIBMTR. 4. All ages minorities, sexes and genders are eligible for the study, but participants must weigh at least 10 kilograms (kg) at the time of study enrollment given the volume and number of blood draws required. 5. All participants or parent/legal guardian must sign an informed consent for this study. If there are questions regarding a patient's eligibility for the study, contact the Protocol Team for review and discussion by emailing [email protected]. HCT/GT Exclusion 1. Patients with aplastic anemia or hemoglobinopathies who are not pursuing allogeneic HCT or GT. 2. Active malignancy. 3. Hematologic malignancy or therapy for a prior hematologic malignancy in the previous five 5 years. 4. Weight ≤ 10.0 kg at time of study enrollment. 5. Prior autologous or allogeneic transplant. Related Donor Inclusion: 1\. All related donors for eligible recipients undergoing allogeneic HCT for AA, hemoglobinopathies, or bone marrow failure as defined in the recipient eligibility criteria above are eligible. Note: HCT recipient participants will remain eligible if the related donor declines to participate in the study. Related Donor Exclusion: 1\. Donor weight ≤ 10.0 kg at time of study enrollment

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Conditions

The condition(s) this trial relates to.

aplastic anemia Bone Marrow Failure Disorders bone marrow failure syndrome hemoglobinopathy inborn error of immunity inherited hemoglobinopathy sickle cell disease thalassemia

As listed by the trial registrant

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

Aplastic anemias Bone marrow failure Hemoglobinopathies

How to take part

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

  1. The places running it

    14 sites. The list below names each one and where it is.

  2. 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 ↗

  3. 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

Locations

  • Baylor College of Medicine

    RECRUITING

    Houston, Texas, 77030, United States

  • Children's Healthcare of Atlanta

    RECRUITING

    Atlanta, Georgia, 30329, United States

  • Children's Hospital of Wisconsin

    RECRUITING

    Milwaukee, Wisconsin, 53226, United States

  • City of Hope

    RECRUITING

    Duarte, California, 91010, United States

  • Duke University Medical Center

    RECRUITING

    Durham, North Carolina, 27710, United States

  • Fred Hutchinson Cancer Center

    RECRUITING

    Seattle, Washington, 98109, United States

  • Lurie Children's Hospital of Chicago

    RECRUITING

    Chicago, Illinois, 60611, United States

  • Memorial Sloan Kettering Cancer Center

    RECRUITING

    New York, New York, 10065, United States

  • Oregon Health and Science University

    RECRUITING

    Portland, Oregon, 97239, United States

  • St. Louis Children's

    RECRUITING

    St Louis, Missouri, 63110, United States

  • University of Florida

    RECRUITING

    Gainesville, Florida, 32611, United States

  • Vanderbilt University Medical Center

    RECRUITING

    Nashville, Tennessee, 37235, United States

  • Virginia Commonwealth University

    RECRUITING

    Richmond, Virginia, 23298, United States

  • Washington University

    RECRUITING

    St Louis, Missouri, 63110, United States

More trials for these conditions

Other studies related to the condition(s) this trial covers.