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Comparing bone marrow transplant with immune suppression therapy for severe aplastic anemia in young patients

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)

Phase 3 Interventional Boston Children's Hospital · NCT05600426

This study is testing whether a bone marrow transplant or immune suppression therapy works better as a first treatment for severe aplastic anemia in young patients.

Quick facts

PhasePhase 3
Study typeInterventional
Enrollment234 (estimated)
Ages0 Years to 25 Years
SexAll
SponsorBoston Children's Hospital Academic / other
Drugs / interventionscyclophosphamide, fludarabine
Locations50 sites (Birmingham, Alabama and 49 other locations)
Trial IDNCT05600426 on ClinicalTrials.gov

What this trial studies

This clinical trial is a multi-center randomized phase III study that aims to compare the effectiveness of immune suppression therapy (IST) versus unrelated donor bone marrow transplantation (URD BMT) as initial treatments for severe aplastic anemia (SAA) in pediatric and young adult patients. The trial will enroll 234 participants aged 25 and younger and will assess treatment failure rates and overall survival over a period of up to 4.7 years. Additionally, it will evaluate patient-reported outcomes, quality of life, and early markers of fertility, while also exploring genetic factors associated with SAA. Participants will be randomized to receive either IST or URD BMT, with follow-up assessments conducted for late effects and survival outcomes.

Who should consider this trial

Good fit: Ideal candidates for this study are children and young adults aged 25 years or younger diagnosed with idiopathic severe aplastic anemia who do not have a suitable matched family donor.

Not a fit: Patients with a suitable matched related donor or those older than 25 years may not benefit from this study.

Why it matters

Potential benefit: If successful, this study could provide a more effective treatment option for young patients with severe aplastic anemia, potentially improving survival rates and quality of life.

How similar studies have performed: Other studies have shown promising results with similar approaches, but this trial specifically addresses a unique patient population and treatment comparison.

Eligibility criteria

Show full inclusion / exclusion criteria 
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

Where this trial is running

Birmingham, Alabama and 49 other locations

Study contacts

How to participate

  1. Review the eligibility criteria above with your treating physician.
  2. Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
  3. Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.
View on ClinicalTrials.gov → Find more trials like this →
Conditions Severe Aplastic Anemia
Last reviewed 2026-06-13 by the Find a Trial editorial team. Information on this page is for educational purposes and is not medical advice. Always consult qualified healthcare professionals about clinical trial participation.