| ID | Type | Description | Link |
|---|---|---|---|
| MT1999-20 | Other Identifier | Blood and Marrow Transplant Program | |
| 9911M24961 | Other Identifier | IRB, University of Minnesota |
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
The investigators hypothesize that long-term disease-free survival (DFS) in patients with JMML can be achieved with a treatment of busulfan (BU), cyclophosphamide (CY) and melphalan (L-PAM) followed by hematopoietic cell transplantation (HCT).
Prior to transplantation, subjects will receive BUSULFAN via the central venous line, six times a day for four days, CYCLOPHOSPHAMIDE via the central venous line once a day for two days, and MELPHALAN via the central venous line for one day. Busulfan, cyclophosphamide, and melphalan are given to destroy the subject's leukemia. As well, these drugs will destroy the subject's own immune system to help ensure the new bone marrow takes and grows after transplantation.
On the day of transplantation, bone marrow or umbilical cord blood from the donor will arrive to the bone marrow transplant unit and be transfused via venous line. These new cells will replace the subject's bone marrow.
Not provided
Not provided
Not provided
Not provided
| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Intent-to-Treat | Experimental | Patients receiving study regimen. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Stem Cell Transplant | Biological | Transplantation on Day 0. |
|
| Measure | Description | Time Frame |
|---|---|---|
| Determine Probability of Long-term Disease Free Survival in JMML | at 1 year after transplant |
| Measure | Description | Time Frame |
|---|---|---|
| Combined Incidence of Neutrophil Engraftment, Graft-versus-host Disease (GVHD), Regimen-related Toxicity, and Relapse. | Measured by AGVHD Grades 2 - 4. | at 1 year after transplant |
Not provided
Inclusion Criteria:
Patients must have a diagnosis of JMML and fulfill these minimal criteria (International diagnostic criteria for JMML):
Leukocytosis (> 13,000) with absolute monocytosis (> 1,000)
The presence of immature myeloid cells in the peripheral blood
Less than 30% marrow blasts
Absence of t(9:22) or BCR-ABL transcript
Adequate major organ function including:
Written informed consent.
Exclusion Criteria:
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
| Name | Affiliation | Role |
|---|---|---|
| Margaret MacMillan, MD | Masonic Cancer Center, University of Minnesota | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Masonic Cancer Center, University of Minnesota | Minneapolis | Minnesota | 55455 | United States |
Not provided
Not provided
Not provided
| ID | Title | Description |
|---|---|---|
| FG000 | Intent-to-Treat | Patients receiving study regimen. Stem Cell Transplant: Transplantation on Day 0. Preparative Regimen: - Busulfan
|
| Title | Milestones | Reasons Not Completed | ||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Overall Study |
|
Not provided
Not provided
| ID | Title | Description |
|---|---|---|
| BG000 | Intent-to-Treat | Patients receiving study regimen. Stem Cell Transplant: Transplantation on Day 0. Preparative Regimen: - Busulfan
|
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes |
|---|---|---|---|---|---|---|---|---|---|
| Age, Categorical | Count of Participants |
| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | ||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | Determine Probability of Long-term Disease Free Survival in JMML | Posted | Number | 95% Confidence Interval | Percentage of participants | at 1 year after transplant |
|
|
1 year
Not provided
Not provided
| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | Intent-to-Treat | Patients receiving study regimen. Stem Cell Transplant: Transplantation on Day 0. Preparative Regimen: - Busulfan
|
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Aspergillus Fumigatus | Infections and infestations | Systematic Assessment |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Hypertension | Vascular disorders | Systematic Assessment |
Not provided
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Dr. Margaret MacMillan, MD | University of Minnesota, Masonic Cancer Center | +1 612-626-2961 | macmi002@umn.edu |
Not provided
| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| Prot_SAP | Yes | Yes | No | Study Protocol and Statistical Analysis Plan | Jan 3, 2022 | Aug 29, 2023 | Prot_SAP_000.pdf |
| ICF | No | No | Yes | Informed Consent Form | Jan 12, 2022 | Aug 29, 2023 | ICF_001.pdf |
Not provided
| ID | Term |
|---|---|
| D054429 | Leukemia, Myelomonocytic, Juvenile |
| ID | Term |
|---|---|
| D007951 | Leukemia, Myeloid |
| D007938 | Leukemia |
| D009370 | Neoplasms by Histologic Type |
| D009369 | Neoplasms |
Not provided
Not provided
| ID | Term |
|---|---|
| D033581 | Stem Cell Transplantation |
| D016026 | Bone Marrow Transplantation |
| ID | Term |
|---|---|
| D017690 | Cell Transplantation |
| D064987 | Cell- and Tissue-Based Therapy |
| D001691 | Biological Therapy |
| D013812 | Therapeutics |
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
| Preparative Regimen | Drug |
|
|
| Participants |
|
| Sex: Female, Male | Count of Participants | Participants |
|
| Ethnicity (NIH/OMB) | Count of Participants | Participants |
|
| Race (NIH/OMB) | Count of Participants | Participants |
|
| Region of Enrollment | Number | participants |
|
|
| Secondary | Combined Incidence of Neutrophil Engraftment, Graft-versus-host Disease (GVHD), Regimen-related Toxicity, and Relapse. | Measured by AGVHD Grades 2 - 4. | Posted | Number | 95% Confidence Interval | Percentage of participants | at 1 year after transplant |
|
|
|
| 4 |
| 15 |
| 5 |
| 15 |
| 15 |
| 15 |
| Acute Renal Failure | Renal and urinary disorders | Systematic Assessment |
|
| Sepsis | Infections and infestations | Systematic Assessment |
|
| Hepatomegaly | General disorders | Systematic Assessment |
|
| Seizure | General disorders | Systematic Assessment |
|
| Pericardial Effusion | Cardiac disorders | Systematic Assessment |
|
| Blood in Stool | Gastrointestinal disorders | Systematic Assessment |
|
| Blood in Urine | Gastrointestinal disorders | Systematic Assessment |
|
| Rectal Bleed | Gastrointestinal disorders | Systematic Assessment |
|
| Bowel Perferation | Gastrointestinal disorders | Systematic Assessment |
|
| Colitis | Gastrointestinal disorders | Systematic Assessment |
|
| Ischemia | Gastrointestinal disorders | Systematic Assessment |
|
| Ascites | Gastrointestinal disorders | Systematic Assessment |
|
| Vomiting | Gastrointestinal disorders | Systematic Assessment |
|
| Platelet count decreased | Investigations | Systematic Assessment |
|
| Blood bilirubin increased | Investigations | Systematic Assessment |
|
| Hickman Line Infection | Infections and infestations | Systematic Assessment |
|
| Ear Infection | Ear and labyrinth disorders | Systematic Assessment |
|
| Upper respiratory infection | Infections and infestations | Systematic Assessment |
|
| Lung Infection | Infections and infestations | Systematic Assessment |
|
| Pneumonia | Reproductive system and breast disorders | Systematic Assessment |
|
| Dermititis | Skin and subcutaneous tissue disorders | Systematic Assessment |
|
Not provided
Not provided
Not provided
| D054437 |
| Myelodysplastic-Myeloproliferative Diseases |
| D001855 | Bone Marrow Diseases |
| D006402 | Hematologic Diseases |
| D006425 | Hemic and Lymphatic Diseases |
| D014180 |
| Transplantation |
| D013514 | Surgical Procedures, Operative |
| D016378 | Tissue Transplantation |