Trial · NCT04965597
Treosulfan-Based Conditioning Regimen Before a Blood or Bone Marrow Transplant for the Treatment of Bone Marrow Failure Diseases (BMT CTN 1904)
NCT04965597, Phase 2, Completed, Fred Hutchinson Cancer Center
3 sourcestrials-2026-11-12-0.5.0
Cite this record
ConductScience Library. Treosulfan-Based Conditioning Regimen Before a Blood or Bone Marrow Transplant for the Treatment of Bone Marrow Failure Diseases (BMT CTN 1904). Trials, record NCT04965597. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT04965597
On this page
Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Treosulfan-Based Conditioning Regimen Before a Blood or Bone Marrow Transplant for the Treatment of Bone Marrow Failure Diseases (BMT CTN 1904)
- NCT number
- NCT04965597
- Status
- Completed
- Phases
- Phase 2
- Enrollment
- 40 participants, actual
- Lead sponsor
- Fred Hutchinson Cancer Center
- 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, Fludarabine Phosphate, Tacrolimus, Methotrexate, Lapine T-Lymphocyte Immune Globulin, Peripheral Blood Stem Cell Transplantation, Allogeneic Bone Marrow Transplantation, Quality-of-Life Assessment, Echocardiography, Multigated Acquisition Scan, Bone Marrow Biopsy, Bone Marrow Aspiration, Biospecimen Collection, X-Ray Imaging, Computed Tomography
- First posted
- 2021-07-16
- Last updated
- 2026-03-09
Source ClinicalTrials.gov
Registered outcomes21
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Graft-Versus Host-Disease (GVHD)-Free Event-Free Survival (EFS) | 1 year post-HCT | The primary endpoint is the incidence of 1-year GVHD free, EFS (GEFS). An event is defined as death due to any cause, primary or secondary graft failure/rejection, or 2nd HCT whichever occurs first. Grade III-IV acute GVHD and chronic GVHD (using National Institutes of Health \[NIH\] consensus criteria) requiring systemic immune suppression will be considered in this estimate. |
| Secondary | Overall Survival | Day 100 post-HCT | Overall survival at day 100 after HCT |
| Secondary | Overall Survival | 6 months post-HCT | Overall survival at 6 months after HCT |
| Secondary | Overall Survival | 1 year post-HCT | Overall survival at 1 year after HCT |
| Secondary | Event-Free Survival | 1 year post-HCT | Event-free survival will be estimated at 12 months after HCT. An event is defined as death due to any cause, primary or secondary graft failure/rejection, or 2nd HCT, whichever occurs first |
| Secondary | Hematologic Recovery: Neutrophil recovery | Assessed up to 1 year post-HCT | Hematologic recovery will be assessed according to neutrophil recovery after transplant. Neutrophil recovery is defined as achieving an absolute neutrophil count (ANC) ≥ to 500/mm3 for 3 consecutive measurements on 3 different days. The first of the three days will be designated the day of neutrophil recovery. The competing event is death without neutrophil recovery. |
| Secondary | Hematologic Recovery: Platelet recovery | Day 100 post-HCT | Hematologic recovery will be assessed according to platelet counts recovery after transplant. Platelet recovery is defined as the first day of a minimum of 3 days that the patient has a sustained platelet count ≥ 20,000/mm3 with no platelet transfusions in the preceding 7 days. The first day of sustained platelet count above these thresholds will be designated the day of platelet engraftment. |
| Secondary | Donor Chimerism (CD3 and Myeloid) | Day 28 post-HCT | Proportions of patients with full (\>95%), mixed (5-95%), or rejection (\<5%) myeloid |
| Secondary | Donor Chimerism (CD3 and Myeloid) | Day 100 post-HCT | Proportions of patients with full (\>95%), mixed (5-95%), or rejection (\<5%) myeloid |
| Secondary | Donor Chimerism (CD3 and Myeloid) | 1 year post-HCT | Proportions of patients with full (\>95%), mixed (5-95%), or rejection (\<5%) myeloid |
1 to 10 of 21Source ClinicalTrials.gov
See all 21 registered outcomesOutcomes0
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
None in this release.
Results by group0
Each measurement the trial posted, group by group, in the words and numbers it posted.
None in this release.
Participant flow0
How many started, completed or left each group, period by period.
None in this release.
Adverse events0
Each adverse event the trial posted, group by group: the participants affected out of those at risk.
None in this release.
Adverse event totals0
The totals the trial posted for each group.
None in this release.
Papers0
The papers the trial lists on ClinicalTrials.gov.
None in this release.
Grants1
The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.
| Project | Title | Principal investigators | Organization | Funder | Fiscal years |
|---|---|---|---|---|---|
| U10HL069294 | Blood & Marrow Transplant Clinical Trials Network Data Coordinating Center | HOROWITZ, MARY MARESCA | MEDICAL COLLEGE OF WISCONSIN | NATIONAL HEART, LUNG, AND BLOOD INSTITUTE | 2011 to 2016 |
Source NIH RePORTER
Trial comparisons0
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
None in this release.
Tests0
The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.
None in this release.
Data dates
The day each source was copied for this release.
Sources 3
- ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
- ClinicalTrials.govNo license stated
- NIH RePORTER's clinical studies and project filesPublic domain
Each source's terms
- ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
- ClinicalTrials.govnone stated
- NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)
ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.
For software
Read this trial from software.
The record and each of its lists are JSON, twenty rows a page. No key.
GET https://conductscience.org/api/trials/v1/NCT04965597
GET https://conductscience.org/api/trials/v1/NCT04965597/registered?page=2