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

Running, not enrolling · Phase 3

Conditions studied: Severe Aplastic Anemia

In brief

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.

Key facts

Study ID

NCT05600426

Run by

Boston Children's Hospital

People needed

53

Starts

2023-01-25

Expected to finish

2029-12-01

Last updated by the study team

2026-07-30

Who can join

Age: any, up to 25. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• To be eligible to participate in the randomized trial, an individual must meet all the following criteria:
• Provision of signed and dated informed consent form for the randomized trial by patient and/or legal guardian.
• Age ≤25 years old at time of randomized trial consent.
• Confirmed diagnosis of idiopathic SAA, defined as:
• Bone marrow cellularity <25%, or <30% hematopoietic cells.
• 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.
• 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).
• 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).
• In the treating physician's opinion, no obvious contraindications precluding them from BMT or IST.

You may not qualify if…

• 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.
• Clonal cytogenetic abnormalities or Fluorescence In-Situ Hybridization (FISH) pattern consistent with pre- myelodysplastic syndrome (pre-MDS) or MDS on marrow examination.
• Known severe allergy to ATG.
• Prior allogeneic or autologous stem cell transplant.
• Prior solid organ transplant.
• Infection with human immunodeficiency virus (HIV).
• Active Hepatitis B or C. This only needs to be excluded in patients where there is clinical suspicion of hepatitis (e.g., elevated LFTs).
• Female patients who are pregnant or breast-feeding.
• Prior malignancies except resected basal cell carcinoma or treated cervical carcinoma in situ.
• 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

Where it is running

• 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
• University of Alabama at Birmingham — Birmingham, Alabama, United States

Full record on ClinicalTrials.gov

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