Trial · NCT01824693
Busulfan, Cyclophosphamide, and Melphalan or Busulfan and Fludarabine Phosphate Before Donor Hematopoietic Cell Transplant in Treating Younger Patients With Juvenile Myelomonocytic Leukemia
NCT01824693, Phase 2, Completed, Children's Oncology Group; results first posted 2018-12-05
3 sourcestrials-2026-11-12-0.5.0
Cite this record
ConductScience Library. Busulfan, Cyclophosphamide, and Melphalan or Busulfan and Fludarabine Phosphate Before Donor Hematopoietic Cell Transplant in Treating Younger Patients With Juvenile Myelomonocytic Leukemia. Trials, record NCT01824693. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT01824693
On this page
Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Busulfan, Cyclophosphamide, and Melphalan or Busulfan and Fludarabine Phosphate Before Donor Hematopoietic Cell Transplant in Treating Younger Patients With Juvenile Myelomonocytic Leukemia
- NCT number
- NCT01824693
- Status
- Completed
- Phases
- Phase 2
- Enrollment
- 30 participants, actual
- Lead sponsor
- Children's Oncology Group
- Conditions
- Juvenile Myelomonocytic Leukemia
- Interventions
Allogeneic Hematopoietic Stem Cell Transplantation, Busulfan, Cyclophosphamide, Fludarabine Phosphate, Laboratory Biomarker Analysis, Melphalan, Mycophenolate Mofetil, Pharmacological Study, Tacrolimus
- First posted
- 2013-04-05
- Last updated
- 2018-12-05
- Results first posted
- 2018-12-05
Source ClinicalTrials.gov
Registered outcomes4
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Percent Probability of Event-free Survival (EFS) | From transplant up to 18 months | Probability of Event-free Survival (EFS) for Patients after 18 months. An event is either treatment related mortality (TRM), primary or secondary graft failure, or relapse/non-response (as defined in protocol section 10). Time to event is time from transplant with patients who die between the start of the conditioning regimen and transplant given a time to event of zero. |
| Primary | Number of Participants Who Experience Treatment-Related Mortality (TRM) by Day 100 | From transplant up to 100 days | The number of patients who experience TRM on day 100. Treatment-Related Mortality (TRM) an event defined as a death prior to relapse or non-response. Time to TRM is defined as time from transplants to TRM. Patients who die between the start of the conditioning regimen and transplant will be considered a TRM with time to TRM of zero. |
| Secondary | Percentage of Participants Who Experience Primary Graft Failure Event Between Arms | Day 0 - day 540 (18 months) following completion of stem cell transplant | Primary Graft failure is defined as the failure to achieve an ANC \>= 500/uL after 42 days, determined by 3 consecutive measurements on different days; OR \< 5% donor cells in blood or bone marrow by day +42 (as demonstrated by a chimerism assay), without evidence of Juvenile Myelomonocytic Leukemia (JMML). |
| Secondary | Percent Probability of 18 Months-relapse Event Between Arms | From transplant up to 18 months | Probability of patients relapsing at 18 months. A relapse event is defined in protocol section 10.2.3. Time to relapse/non-response is defined as time from transplant to when all criteria of section 10.2.3 are met. |
Source ClinicalTrials.gov
Outcomes4
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Percent Probability of Event-free Survival (EFS) | From transplant up to 18 months | All eligible randomized patients | percent probability | NUMBER |
| PRIMARY | Number of Participants Who Experience Treatment-Related Mortality (TRM) by Day 100 | From transplant up to 100 days | All eligible randomized patients | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Percentage of Participants Who Experience Primary Graft Failure Event Between Arms | Day 0 - day 540 (18 months) following completion of stem cell transplant | All Eligible randomized patients | percentage of patients | NUMBER |
| SECONDARY | Percent Probability of 18 Months-relapse Event Between Arms | From transplant up to 18 months | All eligible randomized patients | percent probability | NUMBER |
Source ClinicalTrials.gov
Results by group8
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Percent Probability of Event-free Survival (EFS) | Arm I (Busulfan, Cyclophosphamide, Melphalan) | 83 | 95% Confidence Interval 27 to 97 | percent probability | |
| Percent Probability of Event-free Survival (EFS) | Arm II (Busulfan, Fludarabine Phosphate) | 22 | 95% Confidence Interval 3 to 51 | percent probability | |
| Number of Participants Who Experience Treatment-Related Mortality (TRM) by Day 100 | Arm I (Busulfan, Cyclophosphamide, Melphalan) | 0 | Participants | ||
| Number of Participants Who Experience Treatment-Related Mortality (TRM) by Day 100 | Arm II (Busulfan, Fludarabine Phosphate) | 1 | Participants | ||
| Percentage of Participants Who Experience Primary Graft Failure Event Between Arms | Arm I (Busulfan, Cyclophosphamide, Melphalan) | 0 | percentage of patients | ||
| Percentage of Participants Who Experience Primary Graft Failure Event Between Arms | Arm II (Busulfan, Fludarabine Phosphate) | 0 | percentage of patients | ||
| Percent Probability of 18 Months-relapse Event Between Arms | Arm I (Busulfan, Cyclophosphamide, Melphalan) | 17 | 95% Confidence Interval 0.4 to 55 | percent probability | |
| Percent Probability of 18 Months-relapse Event Between Arms | Arm II (Busulfan, Fludarabine Phosphate) | 55 | 95% Confidence Interval 16 to 83 | percent probability |
Source ClinicalTrials.gov
Participant flow9
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | Arm I (Busulfan, Cyclophosphamide, Melphalan) | 6 | |
| Overall Study | STARTED | Arm II (Busulfan, Fludarabine Phosphate) | 9 | |
| Overall Study | STARTED | ARM III (Non-Randomized) | 15 | |
| Overall Study | COMPLETED | Arm I (Busulfan, Cyclophosphamide, Melphalan) | 5 | |
| Overall Study | COMPLETED | Arm II (Busulfan, Fludarabine Phosphate) | 2 | |
| Overall Study | COMPLETED | ARM III (Non-Randomized) | 0 | |
| Overall Study | NOT COMPLETED | Arm I (Busulfan, Cyclophosphamide, Melphalan) | 1 | |
| Overall Study | NOT COMPLETED | Arm II (Busulfan, Fludarabine Phosphate) | 7 | |
| Overall Study | NOT COMPLETED | ARM III (Non-Randomized) | 15 |
Source ClinicalTrials.gov
Adverse events135
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 | Abdominal distension | Gastrointestinal disorders | Arm I (Busulfan, Cyclophosphamide, Melphalan) | 2 | 6 | 2 |
| other | Abdominal distension | Gastrointestinal disorders | Arm II (Busulfan, Fludarabine Phosphate) | 0 | 9 | 0 |
| other | Abdominal distension | Gastrointestinal disorders | ARM III (Non-Randomized) | 0 | 12 | 0 |
| other | Abdominal pain | Gastrointestinal disorders | Arm I (Busulfan, Cyclophosphamide, Melphalan) | 2 | 6 | 2 |
| other | Abdominal pain | Gastrointestinal disorders | Arm II (Busulfan, Fludarabine Phosphate) | 1 | 9 | 1 |
| other | Abdominal pain | Gastrointestinal disorders | ARM III (Non-Randomized) | 0 | 12 | 0 |
| other | Alanine aminotransferase increased | Investigations | Arm I (Busulfan, Cyclophosphamide, Melphalan) | 1 | 6 | 1 |
| other | Alanine aminotransferase increased | Investigations | Arm II (Busulfan, Fludarabine Phosphate) | 2 | 9 | 2 |
| other | Alanine aminotransferase increased | Investigations | ARM III (Non-Randomized) | 0 | 12 | 0 |
| other | Anemia | Blood and lymphatic system disorders | Arm I (Busulfan, Cyclophosphamide, Melphalan) | 0 | 6 | 0 |
1 to 10 of 135Source ClinicalTrials.gov
See all 135 adverse eventsAdverse event totals9
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Arm I (Busulfan, Cyclophosphamide, Melphalan) | 1 | 6 |
| Total, all-cause mortality | Arm II (Busulfan, Fludarabine Phosphate) | 5 | 9 |
| Total, all-cause mortality | ARM III (Non-Randomized) | 1 | 12 |
| Total, other adverse events | Arm I (Busulfan, Cyclophosphamide, Melphalan) | 5 | 6 |
| Total, other adverse events | Arm II (Busulfan, Fludarabine Phosphate) | 7 | 9 |
| Total, other adverse events | ARM III (Non-Randomized) | 0 | 12 |
| Total, serious adverse events | Arm I (Busulfan, Cyclophosphamide, Melphalan) | 1 | 6 |
| Total, serious adverse events | Arm II (Busulfan, Fludarabine Phosphate) | 1 | 9 |
| Total, serious adverse events | ARM III (Non-Randomized) | 0 | 12 |
Source ClinicalTrials.gov
Papers0
The papers the trial lists on ClinicalTrials.gov.
None in this release.
Grants2
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 | Source |
|---|---|---|---|---|---|---|
| U10CA180886 | COG NCTN Network Group Operations Center | Hawkins, Douglas S. | COG RESEARCH FOUNDATION, LLC | NATIONAL CANCER INSTITUTE | 2014 to 2026 | NIH RePORTER |
| U10CA098543 | Children's Oncology Group Chair's Grant | ADAMSON, PETER C. | CHILDREN'S HOSP OF PHILADELPHIA | NATIONAL CANCER INSTITUTE | 2003 to 2013 | NIH RePORTER |
Trial comparisons1
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
| Comparison |
|---|
| Arm I (Busulfan, Cyclophosphamide, Melphalan) vs Arm II (Busulfan, Fludarabine Phosphate): Number of Participants Who Experience Treatment-Related Mortality (TRM) by Day 100 |
Source 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/NCT01824693
GET https://conductscience.org/api/trials/v1/NCT01824693/events?page=2