Clinical trialNCT05600426Active, not recruitingPhase 3Interventional

A Trial Comparing Unrelated Donor BMT With IST for Pediatric and Young Adult Patients With Severe Aplastic Anemia (TransIT, BMT CTN 2202)

SponsorBoston Children's HospitalEnrollment53Severe Aplastic Anemia

View the registry record on ClinicalTrials.gov

Summary

The lay summary the sponsor registered on ClinicalTrials.gov.

Severe Aplastic Anemia (SAA) is a rare condition in which the body stops producing enough new blood cells. SAA can be cured with immune suppressive therapy or a bone marrow transplant. Regular treatment for patients with aplastic anemia who have a matched sibling (brother or sister), or family donor is a bone marrow transplant. Patients without a matched family donor normally are treated with immune suppressive therapy (IST). Match unrelated donor (URD) bone marrow transplant (BMT) is used as a secondary treatment in patients who did not get better with IST, had their disease come back, or a new worse disease replaced it (like leukemia). This trial will compare time from randomization to failure of treatment or death from any cause of IST versus URD BMT when used as initial therapy to treat SAA. The trial will also assess whether health-related quality of life and early markers of fertility differ between those randomized to URD BMT or IST, as well as assess the presence of marrow failure-related genes and presence of gene mutations associated with MDS or leukemia and the change in gene signatures after treatment in both study arms. This study treatment does not include any investigational drugs. The medicines and procedures in this study are standard for treatment of SAA.

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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.

Bronwen Shaw, MD
CIBMTR/Medical College of Wisconsin (MCW)
Principal Investigator
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David Williams, MD
Boston Children's Hospital
Principal Investigator
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Michael Pulsipher, MD
University of Utah
Principal Investigator
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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

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

Canada

British Columbia Children'S
Vancouver, British Columbia, Canada
Winnipeg Cancercare Manitoba
Winnipeg, Manitoba, Canada

United States

University of Alabama at Birmingham
Birmingham, Alabama, United States
Phoenix Children'S Hospital
Phoenix, Arizona, United States
Arkansas
Little Rock, Arkansas, United States
Loma Linda
Loma Linda, California, United States
Children'S Hospital Los Angeles
Los Angeles, California, United States
Ucla
Los Angeles, California, United States
Children'S Hospital & Research Center Oakland
Oakland, California, United States
Children'S Hospital of Orange County
Orange, California, United States
Stanford
Palo Alto, California, United States
Rady Children'S Hospital San Diego
San Diego, California, United States
University of California San Francisco
San Francisco, California, United States
Children'S Hospital Colorado
Aurora, Colorado, United States
Yale University
New Haven, Connecticut, United States
Nemours Children'S Hospital, Delaware
Wilmington, Delaware, United States
Children'S National Hospital
Washington D.C., District of Columbia, United States
University of Florida
Gainesville, Florida, United States
Nicklaus Children'S Hospital
Miami, Florida, United States
University of Miami
Miami, Florida, United States
Johns Hopkins All Children'S Hospital
St. Petersburg, Florida, United States
Children'S Hospital of Atlanta/emory
Atlanta, Georgia, United States
Ann & Robert H. Lurie Children'S Hospital of Chicago
Chicago, Illinois, United States
University of Chicago
Chicago, Illinois, United States
Indiana University Hospital/riley Hospital for Children
Indianapolis, Indiana, United States
Children'S Hospital Nola
New Orleans, Louisiana, United States
Maine Health
Scarborough, Maine, United States
Boston Children'S Hospital
Boston, Massachusetts, United States
University of Michigan
Ann Arbor, Michigan, United States
Helen Devos Children'S Hospital
Grand Rapids, Michigan, United States
Mayo Clinic Rochestser
Rochester, Minnesota, United States
University of Mississippi Medical Center
Jackson, Mississippi, United States
Washington University in St. Louis
St Louis, Missouri, United States
Hackensack University Medical Center
Hackensack, New Jersey, United States
Roswell Park Comprehensive Cancer Center
Buffalo, New York, United States
Cohen Children'S Medical Center of New York
New Hyde Park, New York, United States
Columbia University Medical Center
New York, New York, United States
University of North Carolina
Chapel Hill, North Carolina, United States
Levine Children'S Hospital
Charlotte, North Carolina, United States
Duke University
Durham, North Carolina, United States

12 more sites on the registry record.ClinicalTrials.gov ↗

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.

Cyclophosphamide →Fludarabine →Methotrexate →Cyclosporine →Lymphocyte immune globulin, anti-thymocyte globulin →

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: 25 Jan 2023
Primary completion: 31 Dec 2029
Completion: 31 Dec 2029
Last update posted: 30 Jul 2026
Registered enrollment: 53
Registry id: NCT05600426

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.