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Three drugs, treosulfan, fludarabine and lapine T-lymphocyte immune globulin (rATG), before allogeneic (cells from a donor) blood or marrow transplant (BMT) to treat bone marrow failure (BMF) disorders in children, teens and adults (BMT CTN 1904)


1 - 49

Phase 2

24 Locations


Clinical Trial Goal

To find out if the combination of treosulfan, fludarabine and rATG before allogeneic BMT is safe and works well to treat BMF disorders in children, teens and adults

You may be able to join this trial if you:

  • Are 1 - 49 years old
  • Have a bone marrow failure disorder that can be treated with allogeneic BMT. Some examples are:
    • Congenital amegakaryocytic thrombocytopenia (CAMT)
    • Congenital sideroblastic anemia (CSA)
    • Diamond Blackfan anemia (DFA) 
    • GATA2 deficiency
    • Paroxysmal nocturnal hemoglobinuria (PNH)
    • SAMD9 disorder
    • SAMD9L disorder
    • Shwachman-Diamond syndrome (SDS)
    • Other bone marrow failure disorder, even if the cause is unknown
  • Do not have idiopathic aplastic anemia
  • Do not have Fanconi anemia
  • Do not have dyskeratosis congenita (telomere biology disorder (TBD))
  • Do not have congenital neutropenia
  • Do not have myelodysplastic syndrome (MDS)
  • Do not have leukemia 
  • Have not had an allogeneic BMT
  • Agree to have other standard tests done to see if you can be in the clinical trial 

Trial Details

rATG and tacrolimus are standard drugs used to prevent graft-versus-host disease (GVHD).
Fludarabine, methotrexate and treosulfan are chemotherapy (chemo) drugs that block the growth of cancer.

Before transplant, you'll get:
  • Fludarabine – Given as intravenous (IV) infusions 1 time each day for 5 days. Each infusion takes 1 hour
  • rATG – Given as IV infusions 1 time each day for 3 days. Each infusion takes up to 6 hours
  • Tacrolimus – Given as IV infusions starting 2 days before transplant day
  • Treosulfan – Given as IV infusions 1 time each day for 3 days. Each infusion takes 2 hours

On transplant day, the blood-forming cells from your donor are given to you through an IV infusion.

After transplant, you'll get:
  • Methotrexate – Given as IV infusions 4 times
  • Tacrolimus – Given as IV infusions or as a pill that you take by mouth until at least 6 months after transplant

You'll have have biopsies to see how well the treatment is working. The clinical trial doctors will check your health for 1 year. 

The Food and Drug Administration (FDA) has approved all of the drugs used in this trial. 


Adam Mendizabal, PhD,

Megan Scott, 240-599-5648,


Children's Hospital Los AngelesRecruiting

Los Angeles, California
Kimberly Arieli,

Rady Children's Hospital/UCSDRecruiting

San Diego, California

University of California San FranciscoRecruiting

San Francisco, California
Kevin Magruder,

Children's Hospital ColoradoRecruiting

Aurora, Colorado
Courtney Newbold,

Children's Healthcare of AtlantaRecruiting

Atlanta, Georgia
Judson Russell,

Johns Hopkins UniversityRecruiting

Baltimore, Maryland
Jennifer Courneya,

Boston Children's HospitalRecruiting

Boston, Massachusetts
Brandi Bratrude,

University of Michigan Medical CenterRecruiting

Ann Arbor, Michigan
Connie Varner,

University of MinnesotaRecruiting

Minneapolis, Minnesota
Merve Tekmen,

St. Louis Children's HospitalRecruiting

Saint Louis, Missouri
Lisa Murray,

Cohen Children's Hospital of NYRecruiting

Queens, New York
Amelia Halac,

Memorial Sloan Kettering Cancer CenterRecruiting

New York, New York
Kirsten Fuller

Roswell Park Comprehensive Cancer CenterRecruiting

Buffalo, New York
Rachel Carter,

Duke University Medical CenterRecruiting

Durham, North Carolina
Erin Arbuckle,

Cincinnati Children's HospitalRecruiting

Cincinnati, Ohio
Samantha (Sam) McBride,

Nationwide Children's HospitalRecruiting

Columbus, Ohio
Lori Jewell,

Oregon Health & Science UniversityRecruiting

Portland, Oregon
Rebecca Hulme,

Children's Hospital of PhiladelphiaRecruiting

Philadelphia, Pennsylvania
Patricia Hankins,

Vanderbilt University Medical CenterRecruiting

Nashville, Tennessee
Delia Darst,

MD Anderson Cancer CenterRecruiting

Houston, Texas
Marissa Rose,

Texas Children's HospitalRecruiting

Houston, Texas
Emily Jobe,

Primary Children's/University of UtahRecruiting

Salt Lake City, Utah
Rebecca Stoffel,

Fred Hutch/University of Washington Cancer ConsortiumRecruiting

Seattle, Washington
Courtney Vandervlugt,

Medical College of Wisconsin/Children's Hospital of WisconsinRecruiting

Milwaukee, Wisconsin
Emily Ruskiewicz, record

NCT04965597. First posted on 7/16/21

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