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Gentler transplant offers hope for blood disorders without cancer

NCT ID NCT00920972

Disease control Sponsor: Washington University School of Medicine 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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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 Sep 17, 2026 · Updated 2 times

Summary

This study is testing a milder type of stem cell transplant for people with non-cancerous blood, immune, or metabolic disorders. The goal is to safely get donor cells to grow in the patient's body while reducing harsh side effects. About 220 participants will receive a reduced-intensity treatment before transplant, and researchers will monitor engraftment and toxicities for 100 days.

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.

Phase

Phase 1/2

Runs two stages together: safety and dose first, then whether the treatment works.

Participants

About 220 people

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

Started

Dec 2001

Expected to finish

Dec 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.

Ages

Up to 20 years

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: Stratum 1: Patient must have non-malignant disorder, excluding thalassemia. Must be receiving a 8/8 HLA-matched bone marrow, related or unrelated Stratum 2: Patient must have thalassemia receiving 8/8 HLA-matched bone marrow or 5-8/8 HLA-matched UCB. Related or unrelated. Stratum 3: Patient must have a hemoglobinopathy receiving 7/8 HLA-matched bone marrow or 5-8/8 HLA-matched UCB. Related or unrelated. Stratum 4: Patient must have a non-malignant disorder (excluding hemoglobinopathy) receiving 7/8 HLA-matched bone marrow or 5-8/8 HLA-matched UCB. Related or unrelated. All strata: * Recipient age \< 21 years * Lansky/Karnofsky \>/= 40 * Adequate pulmonary, renal, liver, and other organ function as defined in protocol * Negative pregnancy test * Adequate total nucleated cell or CD34+ dose of product as defined in protocol * If sickle cell, Hemoglobin S \<30% Exclusion Criteria: * HIV positive * Invasive infection * Pregnancy/lactating

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Conditions

The condition(s) this trial relates to.

disease hemoglobinopathy inherited hemoglobinopathy metabolic disease sickle cell disease thalassemia

As listed by the trial registrant

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

Hematologic, immune, or bone marrow disorders Hemoglobinopathies Metabolic disorders Non-malignant disorders

How to take part

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

  1. The places running it

    1 site. 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

  • All Children's Hospital

    COMPLETED

    St. Petersburg, Florida, 33701, United States

  • BC Children's Hospital

    COMPLETED

    Vancouver, British Columbia, V6H 3N1, Canada

  • Carolinas Medical Center

    COMPLETED

    Charlotte, North Carolina, 28232, United States

  • Children's Hospital of New Orleans

    COMPLETED

    New Orleans, Louisiana, 70118, United States

  • Children's Hospital of Orange County

    ACTIVE_NOT_RECRUITING

    Orange, California, 92868, United States

  • Children's Memorial Hospital

    COMPLETED

    Chicago, Illinois, 60614, United States

  • Children's Mercy

    COMPLETED

    Kansas City, Missouri, 64108, United States

  • Columbia University Medical Center

    ACTIVE_NOT_RECRUITING

    New York, New York, 10032, United States

  • Cook Children's Hospital

    COMPLETED

    Forth Worth, Texas, 76104, United States

  • Duke Children's Hospital

    COMPLETED

    Durham, North Carolina, 27705, United States

  • George Washington University School of Medicine

    ACTIVE_NOT_RECRUITING

    Washington D.C., District of Columbia, 20010, United States

  • Hackensack University Medical Center

    COMPLETED

    Hackensack, New Jersey, 07601, United States

  • Indiana University School of Medicine

    ACTIVE_NOT_RECRUITING

    Indianapolis, Indiana, 46202, United States

  • Miami Children's Hospital

    COMPLETED

    Miami, Florida, 33155, United States

  • Nemours Children's Health

    COMPLETED

    Jacksonville, Florida, 32207, United States

  • Phoenix Children's Hospital

    ACTIVE_NOT_RECRUITING

    Phoenix, Arizona, 85016, United States

  • St. Louis University

    ACTIVE_NOT_RECRUITING

    St Louis, Missouri, 63104, United States

  • Texas Transplant Institute

    COMPLETED

    San Antonio, Texas, 78229, United States

  • The University of Oklahoma

    COMPLETED

    Oklahoma City, Oklahoma, 73104, United States

  • University of Calgary

    COMPLETED

    Calgary, Canada

  • University of California

    ACTIVE_NOT_RECRUITING

    San Diego, California, 92123, United States

  • University of Manitoba

    COMPLETED

    Winnipeg, Manitoba, MB R3E 0T4, Canada

  • University of Miami

    ACTIVE_NOT_RECRUITING

    Miami, Florida, 33136, United States

  • University of North Carolina

    ACTIVE_NOT_RECRUITING

    Chapel Hill, North Carolina, 27514, United States

  • University of Pittsburg

    COMPLETED

    Pittsburgh, Pennsylvania, 15224, United States

  • University of Utah

    ACTIVE_NOT_RECRUITING

    Salt Lake City, Utah, 84132, United States

  • Washington University School of Medicine (in St. Louis)

    RECRUITING

    St Louis, Missouri, 63110, United States

  • Yale School of Medicine

    ACTIVE_NOT_RECRUITING

    New Haven, Connecticut, 06510, United States

More trials for these conditions

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