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NCT05600426

PHASE3

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

Sponsor: Boston Children's Hospital

View on ClinicalTrials.gov

Summary

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.

Official title: A Phase III Randomized Trial Comparing Unrelated Donor Bone Marrow Transplantation With Immune Suppressive Therapy for Newly Diagnosed Pediatric and Young Adult Patients With Severe Aplastic Anemia (TransIT, BMT CTN 2202)

Key Details

Gender

All

Age Range

0 Years - 25 Years

Study Type

INTERVENTIONAL

Enrollment

Start Date

2023-01-25

Completion Date

2029-12

Last Updated

2026-02-17

Healthy Volunteers

Conditions

Severe Aplastic Anemia

Interventions

DRUG

cyclosporine

PROCEDURE

Matched Unrelated Donor Hematopoetic Stem Cell Transplant

Matched Unrelated Donor (MUD) Hematopoietic Stem Cell Transplantation (HSCT)

DRUG

horse anti-thymocyte globulin (ATG)

DRUG

rabbit anti-thymocyte globulin (ATG)

DRUG

Methotrexate

methotrexate

DRUG

Fludarabine

fludarabine

DRUG

Cyclophosphamide

cyclophosphamide

RADIATION

low-dose total body irradiation (TBI)

PROCEDURE

Immunosuppressive Therapy (IST)

Inclusion Criteria: To be eligible to participate in the randomized trial, an individual must meet all the following criteria: 1. Provision of signed and dated informed consent form for the randomized trial by patient and/or legal guardian. 2. Age ≤25 years old at time of randomized trial consent. 3. Confirmed diagnosis of idiopathic SAA, defined as: 1. Bone marrow cellularity \<25%, or \<30% hematopoietic cells. 2. Two of three of the following (in peripheral blood): neutrophils \<0.5 x 10\^9/L, platelets \<20 x 10\^9/L, absolute reticulocyte count \<60 x 10\^9/L or hemoglobin \<8 g/dL. 4. No suitable fully matched related donor available (minimum 6/6 match for HLA-A and B at intermediate or high resolution and DRB1 at high resolution using DNA based typing). 5. At least 2 unrelated donors noted on NMDP search who are well matched (9/10 or 10/10 for HLA-A, B, C, DRB1, and DQB1 using high resolution). 6. In the treating physician's opinion, no obvious contraindications precluding them from BMT or IST. Exclusion Criteria: 1. Presence of Inherited bone marrow failure syndromes (IBMFS). The diagnosis of Fanconi anemia must be excluded by diepoxybutane (DEB) or equivalent testing on peripheral blood or marrow. Telomere length testing should be sent on all patients to exclude Dyskeratosis Congenita (DC), but if results are delayed or unavailable and there are no clinical manifestations of DC, patients may enroll. If patients have clinical characteristics suspicious for Shwachman-Diamond syndrome, this disorder should be excluded by pancreatic isoamylase testing or gene mutation analysis (note: pancreatic isoamylase testing is not useful in children \<3). Other testing per center may be performed to exclude IBMFS. 2. Clonal cytogenetic abnormalities or Fluorescence In-Situ Hybridization (FISH) pattern consistent with pre- myelodysplastic syndrome (pre-MDS) or MDS on marrow examination. 3. Known severe allergy to ATG. 4. Prior allogeneic or autologous stem cell transplant. 5. Prior solid organ transplant. 6. Infection with human immunodeficiency virus (HIV). 7. Active Hepatitis B or C. This only needs to be excluded in patients where there is clinical suspicion of hepatitis (e.g., elevated LFTs). 8. Female patients who are pregnant or breast-feeding. 9. Prior malignancies except resected basal cell carcinoma or treated cervical carcinoma in situ. 10. Disease modifying treatment prior to study enrollment, including but not limited to use of androgens, eltrombopag, romiplostim, or immune suppression. Note: Supportive care measures such as G-CSF, blood transfusion support and antibiotics are allowable

Locations (52)

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

University of Miami

Miami, Florida, United States

Nicklaus Children's Hospital

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

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

St. Jude Children's Research Hospital

Memphis, Tennessee, United States

Vanderbilt University Medical Center

Nashville, Tennessee, United States

Children's Medical Center Dallas

Dallas, Texas, United States

Texas Children's Hospital

Houston, Texas, United States

Methodist Healthcare

San Antonio, Texas, United States

University of Utah/Primary Children's Hospital

Salt Lake City, Utah, United States

Children's Hospital of the King's Daughter

Norfolk, Virginia, United States

Fred Hutchinson Cancer Center

Seattle, Washington, United States

University of Wisconsin

Madison, Wisconsin, United States

British Columbia Children's

Vancouver, British Columbia, Canada

Winnipeg CancerCare Manitoba

Winnipeg, Manitoba, Canada

Clinical Research Directory

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

Summary

Key Details

Conditions

Interventions

Eligibility Criteria

Locations (52)