Trial · NCT00919503
Treosulfan and Fludarabine Phosphate Before Donor Stem Cell Transplant in Treating Patients With Nonmalignant Inherited Disorders
NCT00919503, Phase 2, Completed, Fred Hutchinson Cancer Center; results first posted 2021-08-13
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. Treosulfan and Fludarabine Phosphate Before Donor Stem Cell Transplant in Treating Patients With Nonmalignant Inherited Disorders. Trials, record NCT00919503. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT00919503
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Treosulfan and Fludarabine Phosphate Before Donor Stem Cell Transplant in Treating Patients With Nonmalignant Inherited Disorders
- NCT number
- NCT00919503
- Status
- Completed
- Phases
- Phase 2
- Enrollment
- 98 participants, actual
- Lead sponsor
- Fred Hutchinson Cancer Center
- Conditions
- Non-Neoplastic Hematologic and Lymphocytic Disorder
- Interventions
Allogeneic Bone Marrow Transplantation, Anti-Thymocyte Globulin, Cyclosporine, Fludarabine Phosphate, Laboratory Biomarker Analysis, Methotrexate, Mycophenolate Mofetil, Peripheral Blood Stem Cell Transplantation, Tacrolimus, Total-Body Irradiation, Treosulfan, Umbilical Cord Blood Transplantation
- First posted
- 2009-06-12
- Last updated
- 2021-08-13
- Results first posted
- 2021-08-13
Source ClinicalTrials.gov
Registered outcomes10
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Preliminary Efficacy | 1 year following transplant | Number of patients engrafted (\>5% donor CD3+ peripheral blood chimerisms) at 1 year following transplant |
| Secondary | Non-relapse Mortality | 1 year following transplant | Number of patients who experienced non-relapse mortality by 1 year following transplant |
| Secondary | Number of Patients With Grade II-IV Acute Graft-versus-host Disease | Day 100 post transplant | Number of patients diagnosed with overall grade II-IV acute GVHD by Day 100 post transplant |
| Secondary | Number of Patients With of Chronic Graft-versus-host Disease | 1 year following transplant | Number of patients diagnosed with chronic GVHD and requiring systemic immunosuppression within 1 year following transplant |
| Secondary | Donor Chimerism CD3 at 100 Days Post Transplant | Day 100 post transplant | Number of patients with peripheral blood donor chimerism for CD3 less than 5%, 5-49%, 50-94% and greater than or equal to 95% at 100 days post transplant. |
| Secondary | Disease Response at One Year Following Hematopoietic Cell Transplantation | 1 year following transplant | Number of patients with no evidence of disease at one year following transplant |
| Secondary | Immune Reconstitution Following Hematopoietic Cell Transplantation | 1 year following transplant | Number of patients with immune reconstitution (defined by a normal range CD3) at 1 year post transplant |
| Secondary | Number of Participants With Infections | 100 days post transplant | Number of participants with clinically significant infections (bacterial, fungal, viral) requiring treatment within 100 days following transplant |
| Secondary | Overall Survival | 1 year following transplant | Number of patients alive at 1 year following transplant |
| Secondary | Donor Chimerism CD33 at Day 100 Post Transplant | 100 days post transplant | Number of patients with peripheral blood donor chimerism for CD33 less than 5%, 5-49%, 50-94% and greater than or equal to 95% at 100 days post transplant. |
Source ClinicalTrials.gov
Outcomes10
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Preliminary Efficacy | 1 year following transplant | Regimen B: Three patients expired without CD3+ chimerisms being performed, therefore could not be analyzed for primary efficacy | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Non-relapse Mortality | 1 year following transplant | Participants | COUNT_OF_PARTICIPANTS | |
| SECONDARY | Number of Patients With Grade II-IV Acute Graft-versus-host Disease | Day 100 post transplant | Regimen B: 2 patients expired too early for evaluation for acute GVHD and could not be evaluated for this outcome | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Number of Patients With of Chronic Graft-versus-host Disease | 1 year following transplant | Regimen B: 3 patients expired too early for evaluation for chronic GVHD and could not be evaluated for this outcome | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Donor Chimerism CD3 at 100 Days Post Transplant | Day 100 post transplant | Regimen B: 3 patients expired without CD3+ chimerisms being performed and could not be evaluated for this outcome | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Disease Response at One Year Following Hematopoietic Cell Transplantation | 1 year following transplant | Regimen B: 3 patients expired prior to disease response being evaluated and could not be evaluated for this outcome | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Immune Reconstitution Following Hematopoietic Cell Transplantation | 1 year following transplant | Regimen A: 11 patients did not have lymphocyte subsets drawn post-transplant and could not be evaluated for outcome; Regimen B: 3 patients expired prior to lymphocyte subsets being evaluated and 2 patients did not have lymphocyte subsets drawn post-transplant and could not be evaluated for outcome | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Number of Participants With Infections | 100 days post transplant | Participants | COUNT_OF_PARTICIPANTS | |
| SECONDARY | Overall Survival | 1 year following transplant | Participants | COUNT_OF_PARTICIPANTS | |
| SECONDARY | Donor Chimerism CD33 at Day 100 Post Transplant | 100 days post transplant | Regimen B: 2 patients expired without CD33+ chimerisms being performed and could not be evaluated for this outcome | Participants | COUNT_OF_PARTICIPANTS |
Source ClinicalTrials.gov
Results by group32
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Preliminary Efficacy | Regimen A (PBSCT and BMT) | 83 | Participants | ||
| Preliminary Efficacy | Regimen B (UBCT) | 10 | Participants | ||
| Non-relapse Mortality | Regimen A (PBSCT and BMT) | 2 | Participants | ||
| Non-relapse Mortality | Regimen B (UBCT) | 5 | Participants | ||
| Number of Patients With Grade II-IV Acute Graft-versus-host Disease | Regimen A (PBSCT and BMT) | 44 | Participants | ||
| Number of Patients With Grade II-IV Acute Graft-versus-host Disease | Regimen B (UBCT) | 8 | Participants | ||
| Number of Patients With of Chronic Graft-versus-host Disease | Regimen A (PBSCT and BMT) | 29 | Participants | ||
| Number of Patients With of Chronic Graft-versus-host Disease | Regimen B (UBCT) | 5 | Participants | ||
| Donor Chimerism CD3 at 100 Days Post Transplant | Regimen A (PBSCT and BMT) | Greater than equal to 95% | 49 | Participants | |
| Donor Chimerism CD3 at 100 Days Post Transplant | Regimen B (UBCT) | Greater than equal to 95% | 7 | Participants |
1 to 10 of 32Source ClinicalTrials.gov
See all 32 results by groupParticipant flow6
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | Regimen A (PBSCT and BMT) | 84 | |
| Overall Study | STARTED | Regimen B (UBCT) | 14 | |
| Overall Study | COMPLETED | Regimen A (PBSCT and BMT) | 84 | |
| Overall Study | COMPLETED | Regimen B (UBCT) | 14 | |
| Overall Study | NOT COMPLETED | Regimen A (PBSCT and BMT) | 0 | |
| Overall Study | NOT COMPLETED | Regimen B (UBCT) | 0 |
Source ClinicalTrials.gov
Adverse events118
Each adverse event the trial posted, group by group: the participants affected out of those at risk.
| Type | Event | Organ system | Group | Affected | At risk | Events |
|---|---|---|---|---|---|---|
| other | Anaphylaxis reaction: Possible reaction to rATG | Immune system disorders | Regimen A (PBSCT and BMT) | 1 | 84 | 1 |
| other | Anaphylaxis reaction: Possible reaction to rATG | Immune system disorders | Regimen B (UBCT) | 1 | 14 | 1 |
| other | Pericardial effusion: Tamponade | Cardiac disorders | Regimen A (PBSCT and BMT) | 0 | 84 | 0 |
| other | Pericardial effusion: Tamponade | Cardiac disorders | Regimen B (UBCT) | 2 | 14 | 3 |
| other | Thrombotic microangiopathy (requiring intervention) with acute renal fail: Hemolytic uremic syndrome | Blood and lymphatic system disorders | Regimen A (PBSCT and BMT) | 0 | 84 | 0 |
| other | Thrombotic microangiopathy (requiring intervention) with acute renal fail: Hemolytic uremic syndrome | Blood and lymphatic system disorders | Regimen B (UBCT) | 1 | 14 | 1 |
| other | Fluid retention - evidence of ascites | Gastrointestinal disorders | Regimen A (PBSCT and BMT) | 0 | 84 | 0 |
| other | Fluid retention - evidence of ascites | Gastrointestinal disorders | Regimen B (UBCT) | 1 | 14 | 1 |
| other | Diarrhea (associated with GVHD): >15ml/kg per day | Gastrointestinal disorders | Regimen A (PBSCT and BMT) | 1 | 84 | 1 |
| other | Diarrhea (associated with GVHD): >15ml/kg per day | Gastrointestinal disorders | Regimen B (UBCT) | 1 | 14 | 1 |
1 to 10 of 118Source ClinicalTrials.gov
See all 118 adverse eventsAdverse event totals6
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Regimen A (PBSCT and BMT) | 7 | 84 |
| Total, all-cause mortality | Regimen B (UBCT) | 6 | 14 |
| Total, other adverse events | Regimen A (PBSCT and BMT) | 53 | 84 |
| Total, other adverse events | Regimen B (UBCT) | 10 | 14 |
| Total, serious adverse events | Regimen A (PBSCT and BMT) | 17 | 84 |
| Total, serious adverse events | Regimen B (UBCT) | 5 | 14 |
Source ClinicalTrials.gov
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 |
|---|---|---|---|---|---|
| P30CA015704 | Cancer Center Support Grant | LYNCH, THOMAS JAMES | FRED HUTCHINSON CANCER CENTER | NATIONAL CANCER INSTITUTE | 1985 to 2025 |
Source NIH RePORTER
Trial comparisons10
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
| Comparison | Source |
|---|---|
| Regimen A (PBSCT and BMT) vs Regimen B (UBCT): Disease Response at One Year Following Hematopoietic Cell Transplantation | Trial comparisons |
| Regimen A (PBSCT and BMT) vs Regimen B (UBCT): Preliminary Efficacy | Trial comparisons |
| Regimen A (PBSCT and BMT) vs Regimen B (UBCT): Number of Patients With of Chronic Graft-versus-host Disease | Trial comparisons |
| Regimen A (PBSCT and BMT) vs Regimen B (UBCT): Number of Participants With Infections | Trial comparisons |
| Regimen A (PBSCT and BMT) vs Regimen B (UBCT): Number of Patients With Grade II-IV Acute Graft-versus-host Disease | Trial comparisons |
| Regimen A (PBSCT and BMT) vs Regimen B (UBCT): deaths from any cause | Trial comparisons |
| Regimen A (PBSCT and BMT) vs Regimen B (UBCT): Overall Survival | Trial comparisons |
| Regimen A (PBSCT and BMT) vs Regimen B (UBCT): Immune Reconstitution Following Hematopoietic Cell Transplantation | Trial comparisons |
| Regimen A (PBSCT and BMT) vs Regimen B (UBCT): Non-relapse Mortality | Trial comparisons |
| Regimen A (PBSCT and BMT) vs Regimen B (UBCT): serious adverse events | Trial comparisons |
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/NCT00919503
GET https://conductscience.org/api/trials/v1/NCT00919503/results?page=2