New transplant cocktail aims to cut complications in bone marrow failure

NCT ID NCT04965597

Completed Disease control Sponsor: Fred Hutchinson Cancer Center Source: ClinicalTrials.gov ↗

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

Summary

This phase 2 trial tested a combination of treosulfan, fludarabine, and rabbit antithymocyte globulin before a bone marrow transplant in 40 people with bone marrow failure diseases like Diamond-Blackfan anemia and Shwachman-Diamond syndrome. The goal was to see if this conditioning regimen could reduce severe complications such as graft-versus-host disease and transplant failure. The study has completed, and results will show whether this approach improves outcomes compared to standard treatments.

What this could mean

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

Active substance: Treosulfan, fludarabine, rabbit antithymocyte globulin, tacrolimus, methotrexate
What this could lead to: If successful, this approach could reduce severe complications like graft-versus-host disease after bone marrow transplant for bone marrow failure diseases.
What could go wrong: This is a small, completed phase 2 trial with only 40 participants, so results may not apply to everyone. There are still risks of transplant failure, rejection, or serious side effects from the drugs.

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

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Conditions

The condition(s) this trial relates to.

Anemia, Hypochromic Bone Marrow Failure Disorders bone marrow failure syndrome congenital amegakaryocytic thrombocytopenia congenital amegakaryocytic thrombocytopenia 1 Diamond-Blackfan anemia GATA2 deficiency with susceptibility to MDS/AML Hemoglobinuria, Paroxysmal inherited sideroblastic anemia paroxysmal nocturnal hemoglobinuria SAMD9-related spectrum and myeloid neoplasm risk SAMD9L-related spectrum and myeloid neoplasm risk Shwachman-Diamond syndrome

As listed by the trial registrant

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

BONE MARROW FAILURE SYNDROME CONGENITAL AMEGAKARYOCYTIC THROMBOCYTOPENIA DIAMOND-BLACKFAN ANEMIA HEMATOLOGIC NEOPLASM WITH GERMLINE GATA2 MUTATION HEMATOLOGIC NEOPLASM WITH GERMLINE SAMD9 MUTATION HEMATOLOGIC NEOPLASM WITH GERMLINE SAMD9L MUTATION HEREDITARY SIDEROBLASTIC ANEMIA PAROXYSMAL NOCTURNAL HEMOGLOBINURIA SHWACHMAN DIAMOND SYNDROME

Contacts and locations

Locations

Boston Children's Hospital

Boston, Massachusetts, 02115, United States
Children's Healthcare of Atlanta

Atlanta, Georgia, 30329, 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 of Philadelphia

Philadelphia, Pennsylvania, 19104, United States
Cincinnati Children's Hospital

Cincinnati, Ohio, 45229, United States
Cohen Children's Hospital of NY

Queens, New York, 11040, United States
Duke University Medical Center

Durham, North Carolina, 27705, United States
Fred Hutch/University of Washington Cancer Consortium

Seattle, Washington, 98109, United States
Johns Hopkins University

Baltimore, Maryland, 21231, United States
MD Anderson Cancer Center

Houston, Texas, 77030, United States
Medical College of Wisconsin/Children's Hospital of Wisconsin

Milwaukee, Wisconsin, 53226, United States
Memorial Sloan Kettering Cancer Center

New York, New York, 10065, United States
Nationwide Children's Hospital

Columbus, Ohio, 43205, United States
Oregon Health & Science University

Portland, Oregon, 97239, United States
Primary Children's/University of Utah

Salt Lake City, Utah, 84113, United States
Rady Children's Hospital/UCSD

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

Buffalo, New York, 14203, United States
St. Louis Children's Hospital

St Louis, Missouri, 63110, United States
Texas Children's Hospital

Houston, Texas, 77030, United States
University of California San Francisco

San Francisco, California, 94143, United States
University of Michigan Medical Center

Ann Arbor, Michigan, 48109, United States
University of Minnesota

Minneapolis, Minnesota, 55455, United States
Vanderbilt University Medical Center

Nashville, Tennessee, 37232, United States

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