Trial Summary
What is the purpose of this trial?RATIONALE: Giving chemotherapy, such as busulfan, fludarabine, and melphalan, before a donor umbilical cord blood stem cell transplant helps stop the growth of abnormal or cancer cells and prepares the patient's bone marrow for the stem cells. When the healthy stem cells from a donor are infused into the patient they may help the patient's bone marrow make stem cells, red blood cells, white blood cells, and platelets. Sometimes the transplanted cells from a donor can make an immune response against the body's normal cells. Giving cyclosporine and mycophenolate mofetil may stop this from happening.
PURPOSE: This phase II trial is studying how well combination chemotherapy followed by a donor umbilical cord blood transplant works in treating infants with high-risk acute leukemia or myelodysplastic syndromes.
Eligibility Criteria
This trial is for infants under 3 years old with high-risk acute leukemia or myelodysplastic syndromes. They must be in complete remission or early relapse, have a matched donor for stem cell transplant, and good organ function. Infants can't join if they have an active infection, HIV, extramedullary disease, or had a myeloablative transplant within the last 6 months.Inclusion Criteria
My leukemia has high-risk features and is either in remission or early relapse.
My leukemia is high risk, in remission, and my bone marrow shows less than 5% blasts.
My liver tests are within 5 times the normal range.
My kidney function is good, with a filtration rate over 60.
My blood disorder is considered high risk but with less than 10% immature blood cells.
My transplant match is almost perfect and the cell dose is high enough.
I can do most activities and my organs work well.
My transplant match is perfect and the cell dose is high enough.
My heart pumps well, with an ejection fraction over 45%.
My treatment requires a specific match and cell dose.
My acute myeloid leukemia is considered high risk.
My cancer remains or is increasing despite having standard chemotherapy.
I was diagnosed with a blood cancer before I turned 4 years old.
Exclusion Criteria
I have a history of HIV or tested positive for it.
My leukemia has spread outside the bone marrow.
Treatment Details
The study tests how well chemotherapy (busulfan, fludarabine, melphalan) followed by umbilical cord blood stem cell transplant works in treating infant leukemia. It also looks at preventing immune reactions against normal cells using cyclosporine and mycophenolate mofetil after the transplant.
2Treatment groups
Experimental Treatment
Group I: Single Unit UCB TransplantationExperimental Treatment7 Interventions
Patients that receive one unit of umbilical cord blood transplantation (only if 2 adequate size and matched units are not available).
Group II: Double Unit UCB TransplantationExperimental Treatment7 Interventions
Patients that receive 2 units of umbilical cord blood transplantation (UCBT).
Busulfan is already approved in United States, European Union, Canada, Japan for the following indications:
🇺🇸 Approved in United States as Busulfex for:
- Chronic myeloid leukemia
- Acute myeloid leukemia
- Malignant lymphoma
- Bone marrow transplantation conditioning
🇪🇺 Approved in European Union as Busulfan for:
- Chronic myeloid leukemia
- Acute myeloid leukemia
- Bone marrow transplantation conditioning
🇨🇦 Approved in Canada as Busulfex for:
- Chronic myeloid leukemia
- Acute myeloid leukemia
- Bone marrow transplantation conditioning
🇯🇵 Approved in Japan as Busulfan for:
- Chronic myeloid leukemia
- Acute myeloid leukemia
- Bone marrow transplantation conditioning
Find a clinic near you
Research locations nearbySelect from list below to view details:
Masonic Cancer Center, University of MinnesotaMinneapolis, MN
Loading ...
Who is running the clinical trial?
Masonic Cancer Center, University of MinnesotaLead Sponsor