Treosulfan-Based Conditioning Regimen Before a Blood or Bone Marrow Transplant for the Treatment of Bone Marrow Failure Diseases (BMT CTN 1904)
Public ClinicalTrials.gov record NCT04965597. Field values are reproduced from the official study page; the official ClinicalTrials.gov record remains the source of truth for eligibility, enrollment, and contact information.
Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.
Official title
Hematopoietic Cell Transplantation Using Treosulfan-Based Conditioning for the Treatment of Bone Marrow Failure Diseases
Brief summary
Reproduced verbatim from the official ClinicalTrials.gov record. Not medical advice.
This phase II trial tests whether treosulfan, fludarabine, and rabbit antithymocyte globulin (rATG) work when given before a blood or bone marrow transplant (conditioning regimen) to cause fewer complications for patients with bone marrow failure diseases. Chemotherapy drugs, such as treosulfan, work in different ways to stop the growth of tumor cells, either by killing the cells, by stopping them from dividing, or by stopping them from spreading. Fludarabine may stop the growth of cancer cells by blocking some of the enzymes needed for cell growth. rATG is used to decrease the body's immune response and may improve bone marrow function and increase blood cell counts. Adding treosulfan to a conditioning regimen with fludarabine and rATG may result in patients having less severe complications after a blood or bone marrow transplant.
Study identification
- NCT ID
- NCT04965597
- Recruitment status
- Completed
- Study type
- Interventional
- Phase
- Phase 2
- Enrollment
- 40 participants
Conditions and interventions
Conditions
- Bone Marrow Failure Syndrome
- Congenital Amegakaryocytic Thrombocytopenia
- Diamond-Blackfan Anemia
- Hereditary Sideroblastic Anemia
- Paroxysmal Nocturnal Hemoglobinuria
- Shwachman-Diamond Syndrome
- Hematologic Neoplasm With Germline GATA2 Mutation
- Hematologic Neoplasm With Germline SAMD9 Mutation
- Hematologic Neoplasm With Germline SAMD9L Mutation
Interventions
- Treosulfan Drug
- Fludarabine Phosphate Drug
- Tacrolimus Drug
- Methotrexate Drug
- Lapine T-Lymphocyte Immune Globulin Biological
- Peripheral Blood Stem Cell Transplantation Procedure
- Allogeneic Bone Marrow Transplantation Procedure
- Quality-of-Life Assessment Other
- Echocardiography Procedure
- Multigated Acquisition Scan Procedure
- Bone Marrow Biopsy Procedure
- Bone Marrow Aspiration Procedure
- Biospecimen Collection Procedure
- X-Ray Imaging Procedure
- Computed Tomography Procedure
Drug · Biological · Procedure + 1 more
Eligibility (public fields only)
- Age range
- 1 Year to 49 Years
- Sex
- All
- Healthy volunteers
- Healthy volunteers not accepted
This page does not interpret eligibility. Detailed inclusion and exclusion criteria are on the official ClinicalTrials.gov record.
Study timeline
- Start date
- Apr 19, 2022 (Actual)
- Primary completion
- Feb 18, 2026 (Actual)
- Completion
- Feb 18, 2026 (Actual)
- Last update posted
- Mar 9, 2026
2022 – 2026
United States locations
- U.S. sites
- 24
- U.S. states
- 18
- U.S. cities
- 23
| Facility | City | State | ZIP | Site status |
|---|---|---|---|---|
| Children's Hospital Los Angeles | Los Angeles | California | 90027 | — |
| Rady Children's Hospital/UCSD | San Diego | California | 92123 | — |
| University of California San Francisco | San Francisco | California | 94143 | — |
| Children's Hospital Colorado | Aurora | Colorado | 80045 | — |
| Children's Healthcare of Atlanta | Atlanta | Georgia | 30329 | — |
| Johns Hopkins University | Baltimore | Maryland | 21231 | — |
| Boston Children's Hospital | Boston | Massachusetts | 02115 | — |
| University of Michigan Medical Center | Ann Arbor | Michigan | 48109 | — |
| University of Minnesota | Minneapolis | Minnesota | 55455 | — |
| St. Louis Children's Hospital | St Louis | Missouri | 63110 | — |
| Roswell Park Comprehensive Cancer Center | Buffalo | New York | 14203 | — |
| Memorial Sloan Kettering Cancer Center | New York | New York | 10065 | — |
| Cohen Children's Hospital of NY | Queens | New York | 11040 | — |
| Duke University Medical Center | Durham | North Carolina | 27705 | — |
| Cincinnati Children's Hospital | Cincinnati | Ohio | 45229 | — |
| Nationwide Children's Hospital | Columbus | Ohio | 43205 | — |
| Oregon Health & Science University | Portland | Oregon | 97239 | — |
| Children's Hospital of Philadelphia | Philadelphia | Pennsylvania | 19104 | — |
| Vanderbilt University Medical Center | Nashville | Tennessee | 37232 | — |
| MD Anderson Cancer Center | Houston | Texas | 77030 | — |
| Texas Children's Hospital | Houston | Texas | 77030 | — |
| Primary Children's/University of Utah | Salt Lake City | Utah | 84113 | — |
| Fred Hutch/University of Washington Cancer Consortium | Seattle | Washington | 98109 | — |
| Medical College of Wisconsin/Children's Hospital of Wisconsin | Milwaukee | Wisconsin | 53226 | — |
Site contact phone numbers, emails, and investigator names are intentionally not displayed here. Open the official ClinicalTrials.gov record for site contact information.
About this trial record page
- What this page shows
- Public field values for ClinicalTrials.gov record NCT04965597, including study identification, conditions, interventions, eligibility (age, sex, healthy volunteer), timeline, and U.S. site list.
- What this page does not do
- No medical advice, eligibility judgments, treatment recommendations, study quality scoring, or AI-generated medical summaries. No site contact phone numbers, emails, or investigator names.
- Where the data comes from
- Sourced from the official ClinicalTrials.gov public API. The official record is the source of truth.
- Last refresh
- Last update posted Mar 9, 2026 · Synced Oct 21, 2026
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Open the official record
The complete public registry record, listed eligibility criteria, and available contact information for NCT04965597 live on ClinicalTrials.gov.