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NCT05600426 · ClinicalTrials.gov registry record · Phase 3
A Trial Comparing Unrelated Donor BMT With IST for Pediatric and Young Adult Patients With Severe Aplastic Anemia (TransIT, BMT CTN 2202)
A Phase 3 study of Severe Aplastic Anemia, sponsored by Boston Children's Hospital.
- Active
- Registry status
- Phase 3
- Development phase
- 53
- Enrollment target
- 20
- Study locations
NCT05600426: Active Phase 3 study of Severe Aplastic Anemia, sponsored by Boston Children's Hospital.
NCT05600426 is a Phase 3 study of Severe Aplastic Anemia that is active but no longer recruiting, run by Boston Children's Hospital. The registered enrollment target is 53 participants, below the 123-participant average among 16 other Severe Aplastic Anemia trials with a reported enrollment target (57% lower). The trial reports 20 study locations across 10 states. According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT05600426, a Phase 3 study of Severe Aplastic Anemia, is active but no longer recruiting, sponsored by Boston Children's Hospital.
- ACTIVE NOT RECRUITING
- Registry status
- Phase 3
- Development phase
- 53 participants
- Enrollment target
- 20
- Study locations
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.
Primary Outcome
The median time to failure or death will be compared on the two arms using the log-rank test. Failure of IST is defined as the date that a second definitive therapy was recommended (BMT, second course of ATG) and failure of BMT defined as the date that a second definitive therapy was recommended (second BMT, course of ATG).
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 |
What the registry record for NCT05600426 still lists
NCT05600426 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 53 participants, a relatively small participant target, below the 123-participant average among 16 other Severe Aplastic Anemia trials with a reported enrollment target (57% lower).
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.
NCT05600426 names 20 study sites across 10 states, led by California, Florida, Alabama.
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.
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).
Who is sponsoring clinical trial NCT05600426?
This trial is sponsored by Boston Children's Hospital, which has 462 total clinical trials registered on ClinicalTrials.gov.
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.
Learn More About Clinical Trials
Similar trials for Severe Aplastic Anemia
Matched on the same primary condition, ranked to surface studies in the same phase first, then by recruiting status, no relevance scoring or editorial curation.
Where NCT05600426's enrollment target sits among peer trials
53 9th of 16 higher than 8 of 16 other Severe Aplastic Anemia trials
participants (enrollment target), bucketed by value
Each bar is a band; taller bars hold more other Severe Aplastic Anemia trials. The dashed line + filled bar mark this entry. Hover or tap any bar for its full count and share, and where it sits relative to this entry.
Source ClinicalTrials.gov registry export · 2026-08-08
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