Trial · NCT00448357
Allogeneic Hematopoietic Cell Transplantation for Patients With Busulfex-based Regimen
NCT00448357, Phase 1, Phase 2, Completed, UNC Lineberger Comprehensive Cancer Center; results first posted 2017-07-17
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. Allogeneic Hematopoietic Cell Transplantation for Patients With Busulfex-based Regimen. Trials, record NCT00448357. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT00448357
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Allogeneic Hematopoietic Cell Transplantation for Patients With Busulfex-based Regimen
- NCT number
- NCT00448357
- Status
- Completed
- Phases
- Phase 1, Phase 2
- Enrollment
- 54 participants, actual
- Lead sponsor
- UNC Lineberger Comprehensive Cancer Center
- Conditions
- Chronic Myeloproliferative Disorders, Leukemia, Lymphoma, Multiple Myeloma and Plasma Cell Neoplasm, Myelodysplastic Syndromes
- Interventions
rabbit anti-thymocyte globulin (ATG), therapeutic allogeneic lymphocytes, busulfan, fludarabine phosphate, tacrolimus, allogeneic hematopoietic stem cell transplantation, peripheral blood stem cell transplantation, methotrexate
- First posted
- 2007-03-16
- Last updated
- 2017-07-17
- Results first posted
- 2017-07-17
Source ClinicalTrials.gov
Registered outcomes6
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Three-year Relapse-free Survival (RFS) Rate at the Maximum Tolerated Dose Identified During Phase I of the Trial (Target AUC 6912) | Three years post-transplant | Relapse is defined as new or increased sites of disease or positive one marrow after a complete response (CR). The RFS was calculated as the percentage of patients who were alive and without relapse at 3 years |
| Primary | Number of Participants With Dose Limiting Toxicities (DLTs) | first 6 weeks or 42 days following stem cell infusion | Dose limiting toxicity will be defined as any irreversible grade 3 or any grade 4 non-hematologic toxicity that is related to busulfan infusion and not graft vs host disease or late infection after recovery from the initial period of myelosuppression. The maximum tolerated dose (MTD) is defined as the dose with probability of dose limiting toxicity (DLT) of 0.25. The dose of continuous infusion IV busulfan based on blood levels derived from a test dose in conjunction with fludarabine and alemtuzumab plus tacrolimus for GVHD prophylaxis. |
| Secondary | Capacity of Test Dosing of Busulfan That Would Result in the Desired Area Under the Curve Concentration Exposure of Patients Receiving a Full-dose Busulfan Regimen | Day -15 to Day -11 | Test doses of busulfan were administered and plasma levels were measured to determine a targeted AUC dosing estimate. The capacity is reported as the precision with which these test dose goals predicted the actual 90-hour mean AUC levels.Dose targeting precision was estimated by root mean squared error. |
| Secondary | Incidence of Graft vs Host Disease in Patients Between One Month and Two Years Post Transplant | 100 days post transplant | GVHD can be mild, moderate or severe depending on the differences in tissue type between patient and donor. GVHD can be acute or chronic. Its symptoms can include: * Rashes, which include burning and redness, that erupt on the palms or soles and may spread to the trunk and eventually to the entire body * Blistering, causing the exposed skin surface to flake off in severe cases * Nausea, vomiting, abdominal cramps, diarrhea and loss of appetite, which can indicate that the gastrointestinal (digestive) tract is affected * Jaundice, or a yellowing of the skin, which can indicate liver damage * Excessive dryness of the mouth and throat, leading to ulcers * Dryness of the lungs, vagina and other surfaces |
| Secondary | Incidence of DNA Chimerism in Patients Between One Month Post Transplant | 30 days post transplant | Deoxyribonucleic acid (DNA) chimerism is a measure identifying the genetic profiles of the transplant recipient and of the donor and then evaluating the extent of mixture in the recipient's blood, bone marrow, or other tissue. |
| Secondary | Overall Survival | Three years post-transplant | Percentage of participants alive at 3 years post transplant |
Source ClinicalTrials.gov
Outcomes6
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Three-year Relapse-free Survival (RFS) Rate at the Maximum Tolerated Dose Identified During Phase I of the Trial (Target AUC 6912) | Three years post-transplant | Because AUC levels varied between the groups and the goal was to identify an actual achieved AUC-based dose, additional outcome analyses were undertaken by dividing patients into thirds according to the actual AUCs delivered rather than the original planned AUCs. | percentage of participants | NUMBER |
| PRIMARY | Number of Participants With Dose Limiting Toxicities (DLTs) | first 6 weeks or 42 days following stem cell infusion | DLTs | NUMBER | |
| SECONDARY | Capacity of Test Dosing of Busulfan That Would Result in the Desired Area Under the Curve Concentration Exposure of Patients Receiving a Full-dose Busulfan Regimen | Day -15 to Day -11 | percentage of error | NUMBER | |
| SECONDARY | Incidence of Graft vs Host Disease in Patients Between One Month and Two Years Post Transplant | 100 days post transplant | Because AUC levels varied between the groups and the goal was to identify an actual achieved AUC-based dose, additional outcome analyses were undertaken by dividing patients into thirds according to the actual AUCs delivered rather than the original planned AUCs. | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Incidence of DNA Chimerism in Patients Between One Month Post Transplant | 30 days post transplant | Because there were no differences in chimerism results as a function of either the immunosuppression used or busulfan dose received, the results are being reported only for the total population. | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Overall Survival | Three years post-transplant | Because AUC levels varied between the groups and the goal was to identify an actual achieved AUC-based dose, additional outcome analyses were undertaken by dividing patients into thirds according to the actual AUCs delivered rather than the original planned AUCs. | percentage of participants | NUMBER |
Source ClinicalTrials.gov
Results by group29
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Three-year Relapse-free Survival (RFS) Rate at the Maximum Tolerated Dose Identified During Phase I of the Trial (Target AUC 6912) | Low Busulfan AUC Tertile | 22 | percentage of participants | ||
| Three-year Relapse-free Survival (RFS) Rate at the Maximum Tolerated Dose Identified During Phase I of the Trial (Target AUC 6912) | Intermediate Busulfan AUC Tertile | 39 | percentage of participants | ||
| Three-year Relapse-free Survival (RFS) Rate at the Maximum Tolerated Dose Identified During Phase I of the Trial (Target AUC 6912) | High Busulfan AUC Tertile | 43 | percentage of participants | ||
| Number of Participants With Dose Limiting Toxicities (DLTs) | Dose Level 1 | 1 | DLTs | ||
| Number of Participants With Dose Limiting Toxicities (DLTs) | Dose Level 2 | 1 | DLTs | ||
| Number of Participants With Dose Limiting Toxicities (DLTs) | Dose Level 3 | 1 | DLTs | ||
| Number of Participants With Dose Limiting Toxicities (DLTs) | Dose Level 4 | 2 | DLTs | ||
| Number of Participants With Dose Limiting Toxicities (DLTs) | Dose Level 5 | 2 | DLTs | ||
| Capacity of Test Dosing of Busulfan That Would Result in the Desired Area Under the Curve Concentration Exposure of Patients Receiving a Full-dose Busulfan Regimen | Dose Level 1 | 11.7 | percentage of error | ||
| Capacity of Test Dosing of Busulfan That Would Result in the Desired Area Under the Curve Concentration Exposure of Patients Receiving a Full-dose Busulfan Regimen | Dose Level 2 | 4.9 | percentage of error |
1 to 10 of 29Source ClinicalTrials.gov
See all 29 results by groupParticipant flow3
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | Experimental: GVHD Prophylaxis | 54 | |
| Overall Study | COMPLETED | Experimental: GVHD Prophylaxis | 54 | |
| Overall Study | NOT COMPLETED | Experimental: GVHD Prophylaxis | 0 |
Source ClinicalTrials.gov
Adverse events19
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 | grade 3 or 4 hematologic toxicities (expected) | Blood and lymphatic system disorders | Experimental: GVHD Prophylaxis | 54 | 54 | |
| other | Alopecia | Skin and subcutaneous tissue disorders | Experimental: GVHD Prophylaxis | 54 | 54 | |
| other | grade 3 GI toxicity | Gastrointestinal disorders | Experimental: GVHD Prophylaxis | 21 | 54 | |
| serious | BK cystitis | Infections and infestations | Experimental: GVHD Prophylaxis | 1 | 54 | |
| serious | Liver Failiure | Hepatobiliary disorders | Experimental: GVHD Prophylaxis | 1 | 54 | |
| serious | Sepsis | Infections and infestations | Experimental: GVHD Prophylaxis | 4 | 54 | |
| serious | Gr 4 GVHD | Gastrointestinal disorders | Experimental: GVHD Prophylaxis | 1 | 54 | |
| serious | Gr 4 mucositis | Gastrointestinal disorders | Experimental: GVHD Prophylaxis | 5 | 54 | |
| serious | Veno-occlusive disease | Hepatobiliary disorders | Experimental: GVHD Prophylaxis | 3 | 54 | |
| serious | Aspergillus pneumonia | Infections and infestations | Experimental: GVHD Prophylaxis | 2 | 54 |
1 to 10 of 19Source ClinicalTrials.gov
See all 19 adverse eventsAdverse event totals3
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Experimental: GVHD Prophylaxis | 15 | 54 |
| Total, other adverse events | Experimental: GVHD Prophylaxis | 54 | 54 |
| Total, serious adverse events | Experimental: GVHD Prophylaxis | 26 | 54 |
Source ClinicalTrials.gov
Papers1
The papers the trial lists on ClinicalTrials.gov.
| PMID | Listed as |
|---|---|
| 26210442 | RESULT |
Source PubMed
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 |
|---|---|---|---|---|---|
| P30CA016086 | Cancer Center Support Grant | FERRIS, ROBERT L. | UNIV OF NORTH CAROLINA CHAPEL HILL | NATIONAL CANCER INSTITUTE | 1985 to 2025 |
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/NCT00448357
GET https://conductscience.org/api/trials/v1/NCT00448357/results?page=2