Bone marrow transplant or drugs? landmark trial for rare blood disease

NCT ID NCT05600426

Ongoing Disease control Sponsor: Boston Children's Hospital Source: ClinicalTrials.gov ↗

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

Summary

This phase 3 trial compares two standard treatments for severe aplastic anemia (SAA) in patients up to age 25 who lack a matched sibling donor: unrelated donor bone marrow transplant (URD BMT) versus immune suppressive therapy (IST). The study measures which approach better prevents treatment failure or death. It also looks at quality of life and fertility. Both treatments are already used in practice, but this is the first head-to-head test as initial therapy.

What this could mean

Our plain-language read of the trial. This is informational only — not medical advice or a prediction.

Active substance

bone marrow transplant or immune suppressive drugs (cyclosporine, anti-thymocyte globulin, methotrexate, fludarabine, cyclophosphamide, low-dose radiation)

What this could lead to

If this trial succeeds, it could show that upfront unrelated donor bone marrow transplant is better than immune therapy for curing severe aplastic anemia in young patients without a matched sibling donor.

What could go wrong

This is a relatively small phase 3 trial (53 participants), and both treatments have serious risks like infection, graft-versus-host disease, or treatment failure. Results may not apply to all patients.

Disclaimer Read more

This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.

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Conditions

The condition(s) this trial relates to.

acquired aplastic anemia aplastic anemia

As listed by the trial registrant

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

SEVERE APLASTIC ANEMIA

Contacts and locations

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Locations

Ann & Robert H. Lurie Children's Hospital of Chicago

Chicago, Illinois, 60611, United States
Arkansas

Little Rock, Arkansas, 72202, United States
Boston Children's Hospital

Boston, Massachusetts, 02115, United States
British Columbia Children's

Vancouver, British Columbia, V6H 3V4, Canada
Children's Hospital & Research Center Oakland

Oakland, California, 94609, United States
Children's Hospital Colorado

Aurora, Colorado, 80045, United States
Children's Hospital Los Angeles

Los Angeles, California, 90027, United States
Children's Hospital NOLA

New Orleans, Louisiana, 70118, United States
Children's Hospital of Atlanta/Emory

Atlanta, Georgia, 30322, United States
Children's Hospital of Orange County

Orange, California, 92868, United States
Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, 19104, United States
Children's Hospital of the King's Daughter

Norfolk, Virginia, 23507, United States
Children's Medical Center Dallas

Dallas, Texas, 75235, United States
Children's National Hospital

Washington D.C., District of Columbia, 20010, United States
Cohen Children's Medical Center of New York

New Hyde Park, New York, 11040, United States
Columbia University Medical Center

New York, New York, 10032, United States
Duke University

Durham, North Carolina, 27705, United States
Fred Hutchinson Cancer Center

Seattle, Washington, 98109, United States
Hackensack University Medical Center

Hackensack, New Jersey, 07601, United States
Helen DeVos Children's Hospital

Grand Rapids, Michigan, 49503, United States
Indiana University Hospital/Riley Hospital for Children

Indianapolis, Indiana, 46202, United States
Johns Hopkins All Children's Hospital

St. Petersburg, Florida, 33701, United States
Levine Children's Hospital

Charlotte, North Carolina, 28203, United States
Loma Linda

Loma Linda, California, 92354, United States
Maine Health

Scarborough, Maine, 04074, United States
Mayo Clinic Rochestser

Rochester, Minnesota, 55902, United States
Methodist Healthcare

San Antonio, Texas, 78229, United States
Nationwide Children's Hospital

Columbus, Ohio, 43205, United States
Nemours Children's Hospital, Delaware

Wilmington, Delaware, 19803, United States
Nicklaus Children's Hospital

Miami, Florida, 33155, United States
Oregon Health & Science University

Portland, Oregon, 97239, United States
Phoenix Children's Hospital

Phoenix, Arizona, 85016, United States
Rady Children's Hospital San Diego

San Diego, California, 92123, United States
Roswell Park Comprehensive Cancer Center

Buffalo, New York, 14263, United States
St. Jude Children's Research Hospital

Memphis, Tennessee, 38105, United States
Stanford

Palo Alto, California, 94304, United States
Texas Children's Hospital

Houston, Texas, 77030, United States
UCLA

Los Angeles, California, 90095, United States
University of Alabama at Birmingham

Birmingham, Alabama, 35249, United States
University of California San Francisco

San Francisco, California, 94158, United States
University of Chicago

Chicago, Illinois, 60637, United States
University of Florida

Gainesville, Florida, 32610, United States
University of Miami

Miami, Florida, 33136, United States
University of Michigan

Ann Arbor, Michigan, 48109, United States
University of Mississippi Medical Center

Jackson, Mississippi, 39216, United States
University of North Carolina

Chapel Hill, North Carolina, 27599, United States
University of Utah/Primary Children's Hospital

Salt Lake City, Utah, 84112, United States
University of Wisconsin

Madison, Wisconsin, 53792, United States
Vanderbilt University Medical Center

Nashville, Tennessee, 37203, United States
Washington University in St. Louis

St Louis, Missouri, 63110, United States
Winnipeg CancerCare Manitoba

Winnipeg, Manitoba, R3E 0V9, Canada
Yale University

New Haven, Connecticut, 06520, United States