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Allogeneic (cells from a donor) blood or marrow transplant (BMT) or immunosuppressive therapy to treat severe aplastic anemia (SAA) that has not yet been treated in children, teens and young adults (TransIT, BMT CTN 2202) 


0 - 25

Phase 3

45 Locations


Clinical Trial Goal

To find out whether allogeneic BMT or immunosuppressive therapy is more effective in treatment of SAA that has not yet been treated in children, teens and young adults

You may be able to join this trial if you:

You or your child:
  • Is 25 years old or younger
  • Has SAA
  • Has two bone marrow donor matches on the public registry
  • Does not have a family member who is their full match and can be their stem cell donor
  • Does not have dyskeratosis congenita, Fanconi anemia, Shwachman-Diamond syndrome or other forms of inherited (genetic) marrow failure syndromes
  • Has not had a BMT
  • You agree to have other standard tests done to see if your child can be in the clinical trial 

Trial Details

Anti-thymocyte globulin (ATG) and cyclosporine are standard drugs used to prevent graft-versus-host disease (GVHD) or to slow down the immune system.
Cyclophosphamide and fludarabine are chemotherapy (chemo) drugs that slow down the immune system.
Methotrexate is a chemo drug that prevents GVHD.
In this trial, you or your child will be randomized to 1 of 2 groups. Once randomized, you or your child will be told what group you/they're in:
  • Group 1 – Standard immunosuppressive therapy
  • Group 2 – Unrelated allogeneic BMT

Randomized means doctors will use a computer to assign you or your child to either group. A computer assigns by chance, like flipping a coin or drawing a name out of a hat. You, your child's doctor or the clinical trial doctor won’t have any control over which group is assigned. This means you won’t be able to choose you o your child's group.

In Group 1, you or your child will get standard immunosuppressive therapy with:
  • Cyclosporine - A pill taken by mouth
  • ATG – Given as intravenous (IV) infusions

In Group 2, you or your child will get an allogeneic BMT.

First, you/they will get:
  • ATG - Given as IV infusions
  • Cyclophosphamide – Given as IV infusions
  • Fludarabine – Given as IV infusions
  • Total body irradiation (TBI) – A treatment done 1 time to prepare the body for transplant

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

After transplant, you or your child will get:
  • Cyclosporine – Given as IV infusions
  • Methotrexate – Given as IV infusions 

You or your child may continue treatment for as long as the clinical trial doctors think it’s best for your/their health. Biopsies will be done to see how well the treatment is working. The clinical trial doctors will check you or your child's health for up to 5 years. 

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


Leah Cheng, MA, 857-218-4731,

Kristi Wilmes, MS, (763) 406-3416, kwilmes@NMDP.ORG


University of Alabama at BirminghamRecruiting

Birmingham, Alabama
Chibuzo Ilonze, MD,

Phoenix Children's HospitalRecruiting

Phoenix, Arizona
Holly Miller, DO,


Little Rock, Arkansas
Jason Farrar, MD,

Children's Hospital Los AngelesRecruiting

Los Angeles, California
Paibel Aguayo-Hiraldo, MD,

Children's Hospital of Orange CountyRecruiting

Orange, California
Eric Won, MD,

Children's Hospital & Research Center OaklandRecruiting

Oakland, California
Nahal Lalefar, MD,

Rady Children's Hospital San DiegoRecruiting

San Diego, California
Nicholas Gloude, MD,


Palo Alto, California
Jessie Alexander, MD,


Los Angeles, California
Theodore B Moore, MD,

University of California San FranciscoRecruiting

San Francisco, California
Kristin Shimano, MD,

Children's Hospital ColoradoRecruiting

Aurora, Colorado
Taizo Nakano, MD,

Yale UniversityRecruiting

New Haven, Connecticut
Lakshmanan Krishnamurti, MD,

Nemours Children's Hospital, DelawareRecruiting

Wilmington, Delaware
Emi Caywood, MD,

Children's National HospitalRecruiting

Washington, District of Columbia
Blachy Davila Saldana, MD,

Johns Hopkins All Children's HospitalRecruiting

Saint Petersburg, Florida
Deepakbabu Chellapandian, MD,

Nicklaus Children's HospitalRecruiting

Miami, Florida
Jorge Galvez Silva, MD,

University of FloridaRecruiting

Gainesville, Florida
Jordan Milner, MD,

Children's Hospital of Atlanta/EmoryRecruiting

Atlanta, Georgia
Staci Arnold, MD,

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

Chicago, Illinois
Sonali Chaudhury, MD,

University of ChicagoRecruiting

Chicago, Illinois
James LaBelle, MD,

Indiana University Hospital/Riley Hospital for ChildrenRecruiting

Indianapolis, Indiana
Kathleen Overholt, MD,

Children's Hospital NOLARecruiting

New Orleans, Louisiana
Lolie Yu, MD,

Maine HealthRecruiting

Scarborough, Maine
Sei-Gyung Sze, MD,

Boston Children's HospitalRecruiting

Boston, Massachusetts

Helen DeVos Children's HospitalRecruiting

Grand Rapids, Michigan
Troy Quigg, DO,

University of MichiganRecruiting

Ann Arbor, Michigan
Thomas Michniacki, MD,

University of Mississippi Medical CenterRecruiting

Jackson, Mississippi
Dereck B Davis, MD,

Washington University in St. LouisRecruiting

Saint Louis, Missouri
Shalini Shenoy, MD,

Hackensack University Medical CenterRecruiting

Hackensack, New Jersey
Alfred Gillio, MD,

Cohen Children's Medical Center of New YorkRecruiting

New Hyde Park, New York
Jonathan Fish, MD,

Columbia University Medical CenterRecruiting

New York, New York
Monica Bhatia, MD,

Roswell Park Comprehensive Cancer CenterRecruiting

Buffalo, New York
Nataliya Buxbaum, MD,

Duke UniversityRecruiting

Durham, North Carolina
Paul Martin, MD,

Levine Children's HospitalRecruiting

Charlotte, North Carolina
Lyndsay Molinari, MD,

University of North CarolinaRecruiting

Chapel Hill, North Carolina
Kimberly Kasow-Wichlan, DO,

Nationwide Children's HospitalRecruiting

Columbus, Ohio
Rajinder Bajwa, MD,

Oregon Health & Science UniversityRecruiting

Portland, Oregon
Evan Shereck, MD, MEd,

Children's Hospital of PhiladelphiaRecruiting

Philadelphia, Pennsylvania
Timothy Olson, MD, PhD,

St. Jude Children's Research HospitalRecruiting

Memphis, Tennessee
Marcin Wlodarski, MD, PhD,

Vanderbilt University Medical CenterRecruiting

Nashville, Tennessee
James Connelly, MD,

Children's Medical Center DallasRecruiting

Dallas, Texas
Tiffany Simms-Waldrip, MD,

Texas Children's HospitalRecruiting

Houston, Texas
Alison Bertuch, MD,

University of Utah/Primary Children's HospitalRecruiting

Salt Lake City, Utah
Ahmad Rayes, MD, 801-662-4700,

Fred Hutchinson Cancer CenterRecruiting

Seattle, Washington
Madhavi Lakkaraja, MD,

University of Wisconsin Hospital and ClinicsRecruiting

Madison, Wisconsin
Kenneth DeSantes, MD, record

NCT05600426. First posted on 10/31/22

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