Clinical trialNCT04965597CompletedPhase 2Interventional

Treosulfan-Based Conditioning Regimen Before a Blood or Bone Marrow Transplant for the Treatment of Bone Marrow Failure Diseases (BMT CTN 1904)

SponsorFred Hutchinson Cancer CenterEnrollment40Bone Marrow Failure SyndromeCongenital Amegakaryocytic ThrombocytopeniaDiamond-Blackfan AnemiaHematologic Neoplasm With Germline GATA2 MutationHematologic Neoplasm With Germline SAMD9 MutationHematologic Neoplasm With Germline SAMD9L Mutation+3 more conditions

View the registry record on ClinicalTrials.gov

Summary

The lay summary the sponsor registered on ClinicalTrials.gov.

This phase II trial tests whether treosulfan, fludarabine, and rabbit antithymocyte globulin (rATG) work when given before a blood or bone marrow transplant (conditioning regimen) to cause fewer complications for patients with bone marrow failure diseases. Chemotherapy drugs, such as treosulfan, work in different ways to stop the growth of tumor cells, either by killing the cells, by stopping them from dividing, or by stopping them from spreading. Fludarabine may stop the growth of cancer cells by blocking some of the enzymes needed for cell growth. rATG is used to decrease the body's immune response and may improve bone marrow function and increase blood cell counts. Adding treosulfan to a conditioning regimen with fludarabine and rATG may result in patients having less severe complications after a blood or bone marrow transplant.

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Investigators

The overall officials registered on this study. Names that resolve to a findmyKOL profile link to it; the rest are shown as registered.

Lauri Burroughs, MD
Fred Hutch/University of Washington Cancer Consortium
Study Chair
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Margaret MacMillan, MD
University of Minnesota
Study Chair
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A profile link is shown only when the registered name resolves to a person record with a confident, namesake-checked match; ambiguous names stay unlinked.

Sites

24 registered facilities, with the per-site recruitment status where the registry publishes one. ClinicalTrials.gov does not publish per-site enrollment counts.

United States

Children'S Hospital Los Angeles
Los Angeles, California, United States
Rady Children'S Hospital/ucsd
San Diego, California, United States
University of California San Francisco
San Francisco, California, United States
Children'S Hospital Colorado
Aurora, Colorado, United States
Children'S Healthcare of Atlanta
Atlanta, Georgia, United States
Johns Hopkins University
Baltimore, Maryland, United States
Boston Children'S Hospital
Boston, Massachusetts, United States
University of Michigan Medical Center
Ann Arbor, Michigan, United States
University of Minnesota
Minneapolis, Minnesota, United States
St. Louis Children'S Hospital
St Louis, Missouri, United States
Roswell Park Comprehensive Cancer Center
Buffalo, New York, United States
Memorial Sloan Kettering Cancer Center
New York, New York, United States
Cohen Children'S Hospital of Ny
Queens, New York, United States
Duke University Medical Center
Durham, North Carolina, United States
Cincinnati Children'S Hospital
Cincinnati, Ohio, United States
Nationwide Children'S Hospital
Columbus, Ohio, United States
Oregon Health & Science University
Portland, Oregon, United States
Children'S Hospital of Philadelphia
Philadelphia, Pennsylvania, United States
Vanderbilt University Medical Center
Nashville, Tennessee, United States
Md Anderson Cancer Center
Houston, Texas, United States
Texas Children'S Hospital
Houston, Texas, United States
Primary Children'S/university of Utah
Salt Lake City, Utah, United States
Fred Hutch/university of Washington Cancer Consortium
Seattle, Washington, United States
Medical College of Wisconsin/children'S Hospital of Wisconsin
Milwaukee, Wisconsin, United States

Site investigator names are published by ClinicalTrials.gov only while a study recruits, so completed studies list sites without them.

Drugs studied

The drug and biological interventions registered on this study, normalized to their RxNorm ingredient where possible.

Fludarabine →Lymphocyte immune globulin, anti-thymocyte globulin →Methotrexate →Tacrolimus →Treosulfan →

Linked drugs open the directory filtered to KOLs working with them.clinicaltrials.gov ↗

Registration & key dates

Registry attributes as recorded on the ClinicalTrials.gov study record.

Start: 19 Apr 2022
Primary completion: 18 Feb 2026
Completion: 18 Feb 2026
Last update posted: 9 Mar 2026
Registered enrollment: 40
Registry id: NCT04965597

clinicaltrials.gov ↗

Source: ClinicalTrials.gov, via the CTTI AACT database, data as of 24 Aug 2026. Registry facts are shown as registered by the study sponsor; findmyKOL does not interpret them.