TriangleNCT00448357 · STUDY IN CONTEXT
Allogeneic Hematopoietic Cell Transplantation for Patients With Busulfex-based Regimen
Draft scope assessment: narrower. Linked by condition-string match at the cancer-entity level. The study's eligibility - stage, biomarker, treatment line and prior therapy - has not been reconciled against this page's definition, so its population is narrower than this page in ways not yet recorded. The condition was transformed or resolved through the terminology hierarchy to reach this cancer type, so the link itself is a candidate a reviewer must confirm. Found by searching ClinicalTrials.gov for this cancer type, restricted to studies that have posted results to the registry. No FDA notice, FDA label or NCI summary held by this site cites this study. Because the search asked for posted results, studies without posted results were never looked for, so their absence from this page means nothing.
Read the full study record and all posted results →
Linked outcomes
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
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.
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.
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
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.
Percentage of participants alive at 3 years post transplant
Reported safety
Quality of life and patient-reported measures
No linked quality-of-life measure is recorded for this setting and study.
Full draft applicability and source-link record
- trial
- release id
- clinical-indications-draft-1
- disease state id
- ds-myeloproliferative-neoplasms
- nct id
- NCT00448357
- trial label
- Allogeneic Hematopoietic Cell Transplantation for Patients With Busulfex-based Regimen
- applicability
- narrower
- applicability note
- Linked by condition-string match at the cancer-entity level. The study's eligibility - stage, biomarker, treatment line and prior therapy - has not been reconciled against this page's definition, so its population is narrower than this page in ways not yet recorded. The condition was transformed or resolved through the terminology hierarchy to reach this cancer type, so the link itself is a candidate a reviewer must confirm.
- curation status
- draft
- reviewed by
- Source null
- reviewed at
- Source null
- created at
- 2026-09-10T00:00:00Z
- provenance class
- registry-condition-search
- discovery note
- Found by searching ClinicalTrials.gov for this cancer type, restricted to studies that have posted results to the registry. No FDA notice, FDA label or NCI summary held by this site cites this study. Because the search asked for posted results, studies without posted results were never looked for, so their absence from this page means nothing.
- outcome Count
- 6
- harms Count
- 1
- qol Count
- 0
- outcomes
- release id
- clinical-indications-draft-1
- disease state id
- ds-myeloproliferative-neoplasms
- nct id
- NCT00448357
- outcome key
- om-0000
- source section id
- NCT00448357-om-0000
- source outcome index
- 0
- endpoint
- Three-year Relapse-free Survival (RFS) Rate at the Maximum Tolerated Dose Identified During Phase I of the Trial (Target AUC 6912)
- endpoint definition
- 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
- time origin
- Three years post-transplant
- analysis role
- primary
- reporting status
- POSTED
- data cutoff
- Source null
- population note
- 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.
- curation status
- draft
- reviewed by
- Source null
- reviewed at
- Source null
- created at
- 2026-09-10T00:00:00Z
- release id
- clinical-indications-draft-1
- disease state id
- ds-myeloproliferative-neoplasms
- nct id
- NCT00448357
- outcome key
- om-0001
- source section id
- NCT00448357-om-0001
- source outcome index
- 1
- endpoint
- Number of Participants With Dose Limiting Toxicities (DLTs)
- endpoint definition
- 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.
- time origin
- first 6 weeks or 42 days following stem cell infusion
- analysis role
- primary
- reporting status
- POSTED
- data cutoff
- Source null
- population note
- Source null
- curation status
- draft
- reviewed by
- Source null
- reviewed at
- Source null
- created at
- 2026-09-10T00:00:00Z
- release id
- clinical-indications-draft-1
- disease state id
- ds-myeloproliferative-neoplasms
- nct id
- NCT00448357
- outcome key
- om-0002
- source section id
- NCT00448357-om-0002
- source outcome index
- 2
- endpoint
- 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
- endpoint definition
- 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.
- time origin
- Day -15 to Day -11
- analysis role
- secondary
- reporting status
- POSTED
- data cutoff
- Source null
- population note
- Source null
- curation status
- draft
- reviewed by
- Source null
- reviewed at
- Source null
- created at
- 2026-09-10T00:00:00Z
- release id
- clinical-indications-draft-1
- disease state id
- ds-myeloproliferative-neoplasms
- nct id
- NCT00448357
- outcome key
- om-0003
- source section id
- NCT00448357-om-0003
- source outcome index
- 3
- endpoint
- Incidence of Graft vs Host Disease in Patients Between One Month and Two Years Post Transplant
- endpoint definition
- 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
- time origin
- 100 days post transplant
- analysis role
- secondary
- reporting status
- POSTED
- data cutoff
- Source null
- population note
- 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.
- curation status
- draft
- reviewed by
- Source null
- reviewed at
- Source null
- created at
- 2026-09-10T00:00:00Z
- release id
- clinical-indications-draft-1
- disease state id
- ds-myeloproliferative-neoplasms
- nct id
- NCT00448357
- outcome key
- om-0004
- source section id
- NCT00448357-om-0004
- source outcome index
- 4
- endpoint
- Incidence of DNA Chimerism in Patients Between One Month Post Transplant
- endpoint definition
- 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.
- time origin
- 30 days post transplant
- analysis role
- secondary
- reporting status
- POSTED
- data cutoff
- Source null
- population note
- 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.
- curation status
- draft
- reviewed by
- Source null
- reviewed at
- Source null
- created at
- 2026-09-10T00:00:00Z
- release id
- clinical-indications-draft-1
- disease state id
- ds-myeloproliferative-neoplasms
- nct id
- NCT00448357
- outcome key
- om-0005
- source section id
- NCT00448357-om-0005
- source outcome index
- 5
- endpoint
- Overall Survival
- endpoint definition
- Percentage of participants alive at 3 years post transplant
- time origin
- Three years post-transplant
- analysis role
- secondary
- reporting status
- POSTED
- data cutoff
- Source null
- population note
- 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.
- curation status
- draft
- reviewed by
- Source null
- reviewed at
- Source null
- created at
- 2026-09-10T00:00:00Z
- arms
- release id
- clinical-indications-draft-1
- disease state id
- ds-myeloproliferative-neoplasms
- nct id
- NCT00448357
- outcome key
- om-0000
- arm key
- OG000
- arm label
- Low Busulfan AUC Tertile
- regimen
- Participants with the lowest tertile of measured busulfan Area under the curve (AUC); with a mean value of 5078 (full range 3933-5615) microM/min
- analyzed count
- 18
- analyzed unit
- Participants
- is comparator
- Source null
- source pointer
- NCT00448357-om-0000#groups/OG000
- release id
- clinical-indications-draft-1
- disease state id
- ds-myeloproliferative-neoplasms
- nct id
- NCT00448357
- outcome key
- om-0000
- arm key
- OG001
- arm label
- Intermediate Busulfan AUC Tertile
- regimen
- Participants with the lowest tertile of measured busulfan Area under the curve (AUC); with a mean value of 6372 (full range 5639-6965) microM/min
- analyzed count
- 18
- analyzed unit
- Participants
- is comparator
- Source null
- source pointer
- NCT00448357-om-0000#groups/OG001
- release id
- clinical-indications-draft-1
- disease state id
- ds-myeloproliferative-neoplasms
- nct id
- NCT00448357
- outcome key
- om-0000
- arm key
- OG002
- arm label
- High Busulfan AUC Tertile
- regimen
- Participants with the lowest tertile of measured busulfan Area under the curve (AUC); with a mean value of 7605 (full range 7054-8863) microM/min
- analyzed count
- 18
- analyzed unit
- Participants
- is comparator
- Source null
- source pointer
- NCT00448357-om-0000#groups/OG002
- release id
- clinical-indications-draft-1
- disease state id
- ds-myeloproliferative-neoplasms
- nct id
- NCT00448357
- outcome key
- om-0001
- arm key
- OG000
- arm label
- Dose Level 1
- regimen
- Target AUC/24 hrs of 4800 micrometer (uM)-min +/-15%
- analyzed count
- 14
- analyzed unit
- Participants
- is comparator
- Source null
- source pointer
- NCT00448357-om-0001#groups/OG000
- release id
- clinical-indications-draft-1
- disease state id
- ds-myeloproliferative-neoplasms
- nct id
- NCT00448357
- outcome key
- om-0001
- arm key
- OG001
- arm label
- Dose Level 2
- regimen
- Target AUC/24 hrs of 5760 uM-min +/- 15%
- analyzed count
- 7
- analyzed unit
- Participants
- is comparator
- Source null
- source pointer
- NCT00448357-om-0001#groups/OG001
- release id
- clinical-indications-draft-1
- disease state id
- ds-myeloproliferative-neoplasms
- nct id
- NCT00448357
- outcome key
- om-0001
- arm key
- OG002
- arm label
- Dose Level 3
- regimen
- Target AUC/24 hrs of 6912 uM-min +/- 15%
- analyzed count
- 24
- analyzed unit
- Participants
- is comparator
- Source null
- source pointer
- NCT00448357-om-0001#groups/OG002
- release id
- clinical-indications-draft-1
- disease state id
- ds-myeloproliferative-neoplasms
- nct id
- NCT00448357
- outcome key
- om-0001
- arm key
- OG003
- arm label
- Dose Level 4
- regimen
- Target AUC/24 hrs of 7603 uM-min+/- 15%
- analyzed count
- 7
- analyzed unit
- Participants
- is comparator
- Source null
- source pointer
- NCT00448357-om-0001#groups/OG003
- release id
- clinical-indications-draft-1
- disease state id
- ds-myeloproliferative-neoplasms
- nct id
- NCT00448357
- outcome key
- om-0001
- arm key
- OG004
- arm label
- Dose Level 5
- regimen
- Target AUC/24 hrs of 8363 uM-min+/- 15%
- analyzed count
- 2
- analyzed unit
- Participants
- is comparator
- Source null
- source pointer
- NCT00448357-om-0001#groups/OG004
- release id
- clinical-indications-draft-1
- disease state id
- ds-myeloproliferative-neoplasms
- nct id
- NCT00448357
- outcome key
- om-0002
- arm key
- OG000
- arm label
- Dose Level 1
- regimen
- Target AUC/24 hrs of 4800 uM-min +/-15%
- analyzed count
- 14
- analyzed unit
- Participants
- is comparator
- Source null
- source pointer
- NCT00448357-om-0002#groups/OG000
- release id
- clinical-indications-draft-1
- disease state id
- ds-myeloproliferative-neoplasms
- nct id
- NCT00448357
- outcome key
- om-0002
- arm key
- OG001
- arm label
- Dose Level 2
- regimen
- Target AUC/24 hrs of 5760 uM-min +/- 15%
- analyzed count
- 7
- analyzed unit
- Participants
- is comparator
- Source null
- source pointer
- NCT00448357-om-0002#groups/OG001
- release id
- clinical-indications-draft-1
- disease state id
- ds-myeloproliferative-neoplasms
- nct id
- NCT00448357
- outcome key
- om-0002
- arm key
- OG002
- arm label
- Dose Level 3
- regimen
- Target AUC/24 hrs of 6912 uM-min +/- 15%
- analyzed count
- 24
- analyzed unit
- Participants
- is comparator
- Source null
- source pointer
- NCT00448357-om-0002#groups/OG002
- release id
- clinical-indications-draft-1
- disease state id
- ds-myeloproliferative-neoplasms
- nct id
- NCT00448357
- outcome key
- om-0002
- arm key
- OG003
- arm label
- Dose Level 4
- regimen
- Target AUC/24 hrs of 7603 uM-min+/- 15%
- analyzed count
- 7
- analyzed unit
- Participants
- is comparator
- Source null
- source pointer
- NCT00448357-om-0002#groups/OG003
- harms
- release id
- clinical-indications-draft-1
- disease state id
- ds-myeloproliferative-neoplasms
- nct id
- NCT00448357
- harm key
- EG000
- source section id
- NCT00448357-ae
- event group key
- EG000
- event group label
- Experimental: GVHD Prophylaxis
- serious affected
- 26
- serious at risk
- 54
- other affected
- 54
- other at risk
- 54
- frequency threshold
- 5
- collection period
- Mortality was assessed at one year
- event definition
- Additional toxicities were not captured as this was a high-dose transplant study and grades 1, 2 and 3 AEs were generally expected and no unexpected toxicities were seen beyond those reported as SAEs
- undenominated note
- Source null
- curation status
- draft
- reviewed by
- Source null
- reviewed at
- Source null
- created at
- 2026-09-10T00:00:00Z
- quality of life
- setting source links
Preserved source evidence · Not medical advice