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A Trial Comparing Unrelated Donor BMT With IST for Pediatric and Young Adult Patients With Severe Aplastic Anemia (TransIT, BMT CTN 2202)
NCT05600426 · View on ClinicalTrials.gov ↗
Study 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.
Conditions Studied
Interventions
- DRUG Methotrexate
- DRUG cyclosporine
- PROCEDURE Matched Unrelated Donor Hematopoetic Stem Cell Transplant
- DRUG horse anti-thymocyte globulin (ATG)
- DRUG rabbit anti-thymocyte globulin (ATG)
Study Locations (20)
California
- Loma Linda — Loma Linda
- Children's Hospital Los Angeles — Los Angeles
- UCLA — Los Angeles
- Children's Hospital & Research Center Oakland — Oakland
- Children's Hospital of Orange County — Orange
- Stanford — Palo Alto
- Rady Children's Hospital San Diego — San Diego
- University of California San Francisco — San Francisco
Florida
- University of Florida — Gainesville
- University of Miami — Miami
- Nicklaus Children's Hospital — Miami
- Johns Hopkins All Children's Hospital — St. Petersburg
Alabama
- University of Alabama at Birmingham — Birmingham
Arizona
- Phoenix Children's Hospital — Phoenix
Arkansas
- Arkansas — Little Rock
Colorado
- Children's Hospital Colorado — Aurora
Connecticut
- Yale University — New Haven
Delaware
- Nemours Children's Hospital, Delaware — Wilmington
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 53 participants |
| Start Date | 2023-01-25 |
| Est. Completion | 2029-12 |
| Phase | Phase 3 |
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Full Details on ClinicalTrials.gov ↗What the Registry Record Tells You About NCT05600426
The ClinicalTrials.gov registry entry for NCT05600426 describes a study currently listed as active not recruiting. It is categorized as Phase 3, which is the standard way researchers label where a study sits along the investigational pathway from early safety work through later efficacy and post-marketing evaluation. The registered enrollment target is 53 participants, a figure that helps gauge the scale of data the investigators plan to collect. The listed sponsor is Boston Children's Hospital, which has 752 total studies on file at ClinicalTrials.gov, and sponsors are the parties responsible for study design, oversight, and regulatory filings.
The record links to 1 condition, with Severe Aplastic Anemia appearing as the primary indexed condition, and to 5 interventions — of which Methotrexate is the first listed. Interventions can include drugs, devices, procedures, behavioral programs, or observational arms, and each is tracked as a separate registry field so that downstream queries can filter accurately. When a trial lists multiple interventions, it usually reflects a multi-arm design or a comparison protocol rather than a single treatment being tested in isolation. The brief summary published in the registry is the clearest source of protocol intent and should be read before drawing conclusions from any sidebar tags.
Geographic footprint matters for practical reasons: NCT05600426 reports 20 study locations spanning 10 distinct geographic areas — top geographies include California, Florida, Alabama. A larger site network tends to correlate with broader recruitment capacity, but it does not imply anything about study quality, and site-level enrollment status can diverge from the overall registry status shown above. Every data point on this page comes from the public ClinicalTrials.gov dataset and is reproduced here for reference only; it is not a medical recommendation, an endorsement of the sponsor, or an invitation to enroll. Verify current status, eligibility criteria, and contact details directly at ClinicalTrials.gov, and discuss any participation decision with your own healthcare provider.
Frequently Asked Questions
What is clinical trial NCT05600426 about?
NCT05600426 is a clinical study titled "A Trial Comparing Unrelated Donor BMT With IST for Pediatric and Young Adult Patients With Severe Aplastic Anemia (TransIT, BMT CTN 2202)". 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...
What is the current status of trial NCT05600426?
This trial is currently active not recruiting. It is a Phase 3 study. The enrollment target is 53 participants. The study started on 2023-01-25. Estimated completion is 2029-12.
What conditions does trial NCT05600426 study?
This clinical trial studies the following conditions: Severe Aplastic Anemia. These conditions were identified from the trial registry and reflect the primary focus areas of the research.
What interventions are being tested in trial NCT05600426?
The interventions under investigation include: Methotrexate (DRUG), cyclosporine (DRUG), Matched Unrelated Donor Hematopoetic Stem Cell Transplant (PROCEDURE), horse anti-thymocyte globulin (ATG) (DRUG), rabbit anti-thymocyte globulin (ATG) (DRUG). Each intervention is being evaluated for safety and efficacy as part of this clinical study.
Who is sponsoring clinical trial NCT05600426?
This trial is sponsored by Boston Children's Hospital, which has 752 total clinical trials registered on ClinicalTrials.gov. The sponsor is responsible for the study's design, funding, and regulatory compliance.
Where is trial NCT05600426 being conducted?
This trial has 20 study locations across Alabama, Arizona, Arkansas, California, Colorado. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
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