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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) 

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Phase 3

45 Locations

NCT05600426

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.

Contacts


Leah Cheng, MA, 857-218-4731, leah.cheng@childrens.harvard.edu

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

Locations

University of Alabama at BirminghamRecruiting

Birmingham, Alabama
Chibuzo Ilonze, MD, cilonze@uabmc.edu

Phoenix Children's HospitalRecruiting

Phoenix, Arizona
Holly Miller, DO, hmiller2@phoenixchildrens.com

ArkansasRecruiting

Little Rock, Arkansas
Jason Farrar, MD, JEFarrar@uams.edu

Children's Hospital Los AngelesRecruiting

Los Angeles, California
Paibel Aguayo-Hiraldo, MD, paguayohiraldo@chla.usc.edu

Children's Hospital of Orange CountyRecruiting

Orange, California
Eric Won, MD, Eric.Won@choc.org

Children's Hospital & Research Center OaklandRecruiting

Oakland, California
Nahal Lalefar, MD, Nahal.Lalefar@ucsf.edu

Rady Children's Hospital San DiegoRecruiting

San Diego, California
Nicholas Gloude, MD, ngloude@rchsd.org

StanfordRecruiting

Palo Alto, California
Jessie Alexander, MD, jlalex@stanford.edu

UCLARecruiting

Los Angeles, California
Theodore B Moore, MD, TBMoore@mednet.ucla.edu

University of California San FranciscoRecruiting

San Francisco, California
Kristin Shimano, MD, Kristin.Shimano@ucsf.edu

Children's Hospital ColoradoRecruiting

Aurora, Colorado
Taizo Nakano, MD, Taizo.Nakano@childrenscolorado.org

Yale UniversityRecruiting

New Haven, Connecticut
Lakshmanan Krishnamurti, MD, lakshmanan.krishnamurti@yale.edu

Nemours Children's Hospital, DelawareRecruiting

Wilmington, Delaware
Emi Caywood, MD, Emi.Caywood@nemours.org

Children's National HospitalRecruiting

Washington, District of Columbia
Blachy Davila Saldana, MD, BJDAVILA@childrensnational.org

Johns Hopkins All Children's HospitalRecruiting

Saint Petersburg, Florida
Deepakbabu Chellapandian, MD, dchella2@jhmi.edu

Nicklaus Children's HospitalRecruiting

Miami, Florida
Jorge Galvez Silva, MD, JorgeRicardo.GalvezSilva@Nicklaushealth.org

University of FloridaRecruiting

Gainesville, Florida
Jordan Milner, MD, jordan.milner@peds.ufl.edu

Children's Hospital of Atlanta/EmoryRecruiting

Atlanta, Georgia
Staci Arnold, MD, staci.denise.arnold@emory.edu

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

Chicago, Illinois
Sonali Chaudhury, MD, SChaudhury@luriechildrens.org

University of ChicagoRecruiting

Chicago, Illinois
James LaBelle, MD, jlabelle@bsd.uchicago.edu

Indiana University Hospital/Riley Hospital for ChildrenRecruiting

Indianapolis, Indiana
Kathleen Overholt, MD, koverho@iu.edu

Children's Hospital NOLARecruiting

New Orleans, Louisiana
Lolie Yu, MD, lyu@lsuhsc.edu

Maine HealthRecruiting

Scarborough, Maine
Sei-Gyung Sze, MD, Sei.Sze@mainehealth.org

Boston Children's HospitalRecruiting

Boston, Massachusetts
Contacts:

Helen DeVos Children's HospitalRecruiting

Grand Rapids, Michigan
Troy Quigg, DO, troy.quigg@corewellhealth.org

University of MichiganRecruiting

Ann Arbor, Michigan
Thomas Michniacki, MD, tmich@med.umich.edu

University of Mississippi Medical CenterRecruiting

Jackson, Mississippi
Dereck B Davis, MD, ddavis7@umc.edu

Washington University in St. LouisRecruiting

Saint Louis, Missouri
Shalini Shenoy, MD, shalinishenoy@wustl.edu

Hackensack University Medical CenterRecruiting

Hackensack, New Jersey
Alfred Gillio, MD, alfred.gillio@hmhn.org

Cohen Children's Medical Center of New YorkRecruiting

New Hyde Park, New York
Jonathan Fish, MD, jfish1@northwell.edu

Columbia University Medical CenterRecruiting

New York, New York
Monica Bhatia, MD, mb2476@cumc.columbia.edu

Roswell Park Comprehensive Cancer CenterRecruiting

Buffalo, New York
Nataliya Buxbaum, MD, nataliya.buxbaum@roswellpark.org

Duke UniversityRecruiting

Durham, North Carolina
Paul Martin, MD, paul.martin@duke.edu

Levine Children's HospitalRecruiting

Charlotte, North Carolina
Lyndsay Molinari, MD, lyndsay.molinari@atriumhealth.org

University of North CarolinaRecruiting

Chapel Hill, North Carolina
Kimberly Kasow-Wichlan, DO, kkasow@med.unc.edu

Nationwide Children's HospitalRecruiting

Columbus, Ohio
Rajinder Bajwa, MD, Rajinder.Bajwa@nationwidechildrens.org

Oregon Health & Science UniversityRecruiting

Portland, Oregon
Evan Shereck, MD, MEd, shereck@ohsu.edu

Children's Hospital of PhiladelphiaRecruiting

Philadelphia, Pennsylvania
Timothy Olson, MD, PhD, olsont@email.chop.edu

St. Jude Children's Research HospitalRecruiting

Memphis, Tennessee
Marcin Wlodarski, MD, PhD, Marcin.Wlodarski@stjude.org

Vanderbilt University Medical CenterRecruiting

Nashville, Tennessee
James Connelly, MD, james.a.connelly@vumc.org

Children's Medical Center DallasRecruiting

Dallas, Texas
Tiffany Simms-Waldrip, MD, tiffany.simms-waldrip@utsouthwestern.edu

Texas Children's HospitalRecruiting

Houston, Texas
Alison Bertuch, MD, aabertuc@texaschildrens.org

University of Utah/Primary Children's HospitalRecruiting

Salt Lake City, Utah
Ahmad Rayes, MD, 801-662-4700, ahmad.rayes@hsc.utah.edu

Fred Hutchinson Cancer CenterRecruiting

Seattle, Washington
Madhavi Lakkaraja, MD, mlakkara@fredhutch.org

University of Wisconsin Hospital and ClinicsRecruiting

Madison, Wisconsin
Kenneth DeSantes, MD, kbdesantes@pediatrics.wisc.edu

ClinicalTrials.gov record


NCT05600426. First posted on 10/31/22

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