LeukemiaPhase 2NCT00357565
Hematopoietic Stem Cell Transplantation in the Treatment of Infant Leukemia
A Phase 2 study testing filgrastim, busulfan and cyclosporine in people with Leukemia and Myelodysplastic Syndromes, run by Masonic Cancer Center, University of Minnesota at 1 site in United States.
Trial summary
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. Read more about this study
- Who is it for?
- Ages Up to 3 years
- Conditions the study is for
- Leukemia, Myelodysplastic Syndromes, Childhood Acute Myeloid Leukemia in Remission
- What is being tested?
- filgrastim, busulfan and cyclosporine
- Type of treatment
- Pill or oral medicine, Injection or infusion, Surgery or procedure
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 34 participants
- Where
- 1 siteUnited States
- Study dates
- Nov 2005to 11 Mar 2025
- Phase
- Phase 2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Up to 3 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- Matched sibling donor (HLA 8/8), if available, or a unrelated partially HLA matched single unit based on the following priority:
- 1st priority: 4/6 matched unit, cell dose >5 x 10-7 nucleated cells/kg
- 2nd priority: 5/6 matched unit, cell dose > 4 x 10-7 nucleated cells/kg
- 3rd priority: 6/6 matched unit, cell dose > 3 x 10-7 nucleated cells/kg
- Patients aged ≤ 3 years at diagnosis (not age of transplant) with hematological malignancy as detailed below:
- Acute myeloid leukemia: high risk CR1 as evidenced by:
Show all 17Show fewer
- High risk cytogenetics t(4;11) or other MLL rearrangements; chromosome 5, 7, or 19 abnormalities; complex karyotype (>5 distinct changes); ≥ 2 cycles to obtain complete response (CR); CR2 or higher; Preceding myelodysplastic syndrome (MDS); All patients must be in CR or early relapse (i.e., <15% blasts in BM).
- Acute lymphocytic leukemia: high risk CR1 as evidenced by: High-risk cytogenetic: t(4;11) or other MLL rearrangements; hypodiploid; t(9;22); >1 cycle to obtain CR; CR2 or higher; All patients must be in CR as defined by hematological recovery, AND <5% blasts by light microscopy within the bone marrow with a cellularity of ≥15%.
- Myelodysplasia (MDS) IPSS Int-2 or High risk (i.e. RAEB, RAEBt) or refractory anemia with severe pancytopenia or high risk cytogenetics. Blasts must be < 10% by a representative bone marrow aspirate morphology.
- Persistent or rising minimal residual disease (MRD) after standard chemotherapy regimens: Patients with evidence of minimal residual disease at the completion of therapy or evidence of rising MRD while on therapy. MRD will be defined by either flow cytometry (>0.1% residual cells in the blast gate with immune phenotype of original leukemic clone), by molecular techniques (PCR or FISH) or conventional cytogenetics (g-banding).
- New Leukemia Subtypes: A major effort in the field of pediatric hematology is to identify patients who are of high risk for treatment failure so that patients can be appropriately stratified to either more (or less) intensive therapy. This effort is continually ongoing and retrospective studies identify new disease features or characteristics that are associated with treatment outcomes. Therefore, if new high risk features are identified after the writing of this protocol, patients can be enrolled with the approval of two members of the study committee.
- Recipients must have a Lansky score ≥ 50% and have acceptable organ function defined as:
- Renal: glomerial filtration rate > 60ml/min/1.73m^2
- Hepatic: bilirubin, AST/ALT, ALP < 5 x upper limit of normal,
- Pulmonary function: oxygen saturation >92%
- Cardiac: left ventricular ejection fraction > 45%.
- Voluntary written informed consent before performance of any study-related procedure not part of normal medical care.
Who cannot join
- Active infection at time of transplantation (including active infection with Aspergillus or other mold within 30 days).
- History of HIV infection or known positive serology
- Myeloablative transplant within the last 6 months.
- Evidence of active extramedullary disease (including central nervous system leukemia).
Trial locations and contacts
1 site in 1 country.
United States1 site
Masonic Cancer Center, University of Minnesota
Minneapolis, Minnesota, 55455
About this study
Official title: Hematopoietic Cell Transplantation in the Treatment of Infant Leukemia and Myelodysplastic Syndrome
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.
Read the detailed description
OBJECTIVES:
Primary
- Determine the incidence of engraftment, defined as achieving donor-derived neutrophil count > 500/mm³ by day 42, in infants with high-risk acute myeloid leukemia, acute lymphoblastic leukemia, or myelodysplastic syndromes treated with a non-irradiation containing myeloablative conditioning regimen comprising busulfan, fludarabine, and melphalan followed by double umbilical cord blood transplantation (UCBT) with two partially HLA-matched units.
Secondary Objectives
- Determine the incidence of transplant-related mortality (TRM) at 6 months after UCBT
- Evaluate pattern of chimerism after double UCBT
- Determine the incidence of platelet engraftment at 1 year after UCBT
- Determine the incidence of acute graft-versus-host disease (GVHD) grade II-IV and grade III-IV at day 100 after UCBT
- Evaluate the developmental outcome after UCBT
Transplant Related Objectives
- Determine the incidence of chronic GVHD at 1 year after UCBT
- Determine the survival and disease free survival at 1 and 2 years after UCBT
- Determine the incidence relapse at 1 and 2 years after UCBT
- Conditions
- Leukemia, Myelodysplastic Syndromes, Childhood Acute Myeloid Leukemia in Remission, Recurrent Childhood Acute Myeloid Leukemia, Secondary Acute Myeloid Leukemia, Childhood Acute Lymphoblastic Leukemia in Remission
- Being tested
- filgrastim, busulfan, cyclosporine, fludarabine phosphate, melphalan
- Keywords
- MDS, AML
- Medical subject headings
- Leukemia, Myelodysplastic Syndromes, Anemia, Refractory, with Excess of Blasts, Anemia, Refractory
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
filgrastimBiological
Also called G-CSF
All patients will receive G-CSF 5 mcg/kg/day intravenous (IV) (dose rounded to vial size) based on the actual body weight IV beginning on day +1 after umbilical cord blood (UCB) infusion. G-CSF will be administered daily until the absolute neutrophil count (ANC) exceeds 2.5 x 10^9/L for three consecutive days and then discontinued. If the ANC decreases to <1.0 x 10^9/L, G-CSF will be reinstituted.
busulfanDrug
Also called Busulfex
Administered 1.1 mg/kg if <12 kg intravenous (IV) every 6 hours (0.8 mg/kg if >12 kg IV every 6 hours on Days -8 through -5.
cyclosporineDrug
Also called CSA
Patients will receive cyclosporine (CSA) therapy beginning on day -3 maintaining a level of >200 ng/mL. For children < 40 kg the initial dose will be 2.5 mg/kg intravenous (IV) over 2 hours every 8 hours.
Also called Fludara
Administered 25 mg/m^2 intravenous (IV) over 60 minutes on Days -4 through -2.
melphalanDrug
Also called Alkeran
Administered 60 mg/m^2 intravenous (IV) over 30 minutes on Days -4 through -2.
Also called MMF
All patients will begin mycophenolate mofetil (MMF) on day -3. Patients <45 kilograms will receive MMF at the dose of 15 mg/kg/dose every 8 hours (max dose 1gm/dose) orally or intravenously (PO or IV).
umbilical cord blood transplantationProcedure or surgery
The product is infused via IV drip directly into the central line without a needle, pump or filter on Day 0.
| Study group | Role | What they receive |
|---|---|---|
| Double Unit UCB Transplantation | Experimental | Patients that receive 2 units of umbilical cord blood transplantation (UCBT). Receives: Biological: filgrastim; Drug: busulfan; Drug: cyclosporine; Drug: fludarabine phosphate; Drug: melphalan; Drug: mycophenolate mofetil; Procedure: umbilical cord blood transplantation |
| Single Unit UCB Transplantation | Experimental | Patients that receive one unit of umbilical cord blood transplantation (only if 2 adequate size and matched units are not available). Receives: Biological: filgrastim; Drug: busulfan; Drug: cyclosporine; Drug: fludarabine phosphate; Drug: melphalan; Drug: mycophenolate mofetil; Procedure: umbilical cord blood transplantation |
What is being measured
The primary outcome is the main question the study is designed to answer.
Incidence of Engraftment
Defined as achieving donor derived neutrophil count >500/uL by day 42 in young children with leukemia or myelodysplastic syndrome undergoing a partially matched single unit umbilical cord blood transplant (UCBT) after a myeloablative preparative regimen consisting of busulfan, melphalan and fludarabine.
Measured: Day 42 After Transplant
Secondary outcomes (7)
Incidence of Transplant-related Mortality (TRM)
defined as death due to transplant
Measured: at 6 months after transplant
Incidence of Platelet Engraftment
defined as platelet count > 50,000
Measured: at 1 year after transplant
Incidence of Acute Graft-versus-host Disease (GVHD) Grade II-IV and Grade III-IV
Graft-versus-host disease (GVHD) is a common complication of allogeneic bone marrow transplantation in which functional immune cells in the transplanted marrow recognize the recipient as "foreign" and mount an immunologic attack.
Measured: Day 100 After Transplant
Incidence of Chronic Graft-versus-host Disease (GVHD)
Graft-versus-host disease (GVHD) is a common complication of allogeneic bone marrow transplantation in which functional immune cells in the transplanted marrow recognize the recipient as "foreign" and mount an immunologic attack.
Measured: 1 Year After Transplant
Incidence of Relapse
defined using standard criteria (bone marrow blast count and cytogenetics).
Measured: 1 and 2 years after transplant
Overall Survival
Alive after transplant.
Measured: at 1 and 2 years after transplant
Disease-free Survival
defined as patients who are alive and in hematological remission.
Measured: at 1 and 2 years after transplant
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted27 Jul 2006 (estimated)
- Study startNov 2005
- Primary completionJun 2024
- Study completion11 Mar 2025
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for LeukemiaStudies of the same condition, open and under way.
- More trials run by Masonic Cancer Center, University of MinnesotaThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 34 participants (actual)
Sponsor and investigators
- Lead sponsor
- Masonic Cancer Center, University of Minnesota
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Christen Ebens, MD, Principal investigator, Masonic Cancer Center, University of Minnesota
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00357565
- Sponsor's protocol ID
- 2005LS075
- Other IDs
- UMN-MT2005-25, UMN-0511M77206
- Status last verified
- Aug 2025
- First submitted
- 26 Jul 2006
- Last updated
- 26 Aug 2025
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 26 Aug 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Hematopoietic Cell Transplantation in the Treatment of Infant Leukemia and Myelodysplastic Syndrome
- ClinicalTrials.gov ID
- NCT00357565
- Acronym / sponsor ID
- 2005LS075
- Conditions
- Leukemia, Myelodysplastic Syndromes, Childhood Acute Myeloid Leukemia in Remission, Recurrent Childhood Acute Myeloid Leukemia, Secondary Acute Myeloid Leukemia, Childhood Acute Lymphoblastic Leukemia in Remission
- Keywords
- MDS, AML
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 34 participants (actual)
- Lead sponsor
- Masonic Cancer Center, University of MinnesotaUniversity, hospital or other
- Study start
- Nov 2005 (actual)
- Primary completion
- Jun 2024 (actual)
- Study completion
- 11 Mar 2025 (actual)
- First posted
- 27 Jul 2006 (estimated)
- Results
- Posted 26 Aug 2025
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Detailed description
OBJECTIVES:
Primary
- Determine the incidence of engraftment, defined as achieving donor-derived neutrophil count > 500/mm³ by day 42, in infants with high-risk acute myeloid leukemia, acute lymphoblastic leukemia, or myelodysplastic syndromes treated with a non-irradiation containing myeloablative conditioning regimen comprising busulfan, fludarabine, and melphalan followed by double umbilical cord blood transplantation (UCBT) with two partially HLA-matched units.
Secondary Objectives
- Determine the incidence of transplant-related mortality (TRM) at 6 months after UCBT
- Evaluate pattern of chimerism after double UCBT
- Determine the incidence of platelet engraftment at 1 year after UCBT
- Determine the incidence of acute graft-versus-host disease (GVHD) grade II-IV and grade III-IV at day 100 after UCBT
- Evaluate the developmental outcome after UCBT
Transplant Related Objectives
- Determine the incidence of chronic GVHD at 1 year after UCBT
- Determine the survival and disease free survival at 1 and 2 years after UCBT
- Determine the incidence relapse at 1 and 2 years after UCBT
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Double Unit UCB Transplantation | Experimental | Biological: filgrastim, Drug: busulfan, Drug: cyclosporine, Drug: fludarabine phosphate, Drug: melphalan, Drug: mycophenolate mofetil, Procedure: umbilical cord blood transplantationPatients that receive 2 units of umbilical cord blood transplantation (UCBT). |
| Single Unit UCB Transplantation | Experimental | Biological: filgrastim, Drug: busulfan, Drug: cyclosporine, Drug: fludarabine phosphate, Drug: melphalan, Drug: mycophenolate mofetil, Procedure: umbilical cord blood transplantationPatients that receive one unit of umbilical cord blood transplantation (only if 2 adequate size and matched units are not available). |
Who may qualify
- Ages
- Up to 3 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (17 listed)
- Matched sibling donor (HLA 8/8), if available, or a unrelated partially HLA matched single unit based on the following priority:
- Patients aged ≤ 3 years at diagnosis (not age of transplant) with hematological malignancy as detailed below:
- Recipients must have a Lansky score ≥ 50% and have acceptable organ function defined as:
- Voluntary written informed consent before performance of any study-related procedure not part of normal medical care.
Key exclusion criteria (4 listed)
- Active infection at time of transplantation (including active infection with Aspergillus or other mold within 30 days).
- History of HIV infection or known positive serology
- Myeloablative transplant within the last 6 months.
- Evidence of active extramedullary disease (including central nervous system leukemia).
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| filgrastimAlso: G-CSFAll patients will receive G-CSF 5 mcg/kg/day intravenous (IV) (dose rounded to vial size) based on the actual body weight IV beginning on day +1 after umbilical cord blood (UCB) infusion. G-CSF will be administered daily until the absolute neutrophil count (ANC) exceeds 2.5 x 10^9/L for three consecutive days and then discontinued. If the ANC decreases to <1.0 x 10^9/L, G-CSF will be reinstituted. | Biological | Double Unit UCB Transplantation, Single Unit UCB Transplantation |
| busulfanAlso: BusulfexAdministered 1.1 mg/kg if <12 kg intravenous (IV) every 6 hours (0.8 mg/kg if >12 kg IV every 6 hours on Days -8 through -5. | Drug | Double Unit UCB Transplantation, Single Unit UCB Transplantation |
| cyclosporineAlso: CSAPatients will receive cyclosporine (CSA) therapy beginning on day -3 maintaining a level of >200 ng/mL. For children < 40 kg the initial dose will be 2.5 mg/kg intravenous (IV) over 2 hours every 8 hours. | Drug | Double Unit UCB Transplantation, Single Unit UCB Transplantation |
| fludarabine phosphateAlso: FludaraAdministered 25 mg/m^2 intravenous (IV) over 60 minutes on Days -4 through -2. | Drug | Double Unit UCB Transplantation, Single Unit UCB Transplantation |
| melphalanAlso: AlkeranAdministered 60 mg/m^2 intravenous (IV) over 30 minutes on Days -4 through -2. | Drug | Double Unit UCB Transplantation, Single Unit UCB Transplantation |
| mycophenolate mofetilAlso: MMFAll patients will begin mycophenolate mofetil (MMF) on day -3. Patients <45 kilograms will receive MMF at the dose of 15 mg/kg/dose every 8 hours (max dose 1gm/dose) orally or intravenously (PO or IV). | Drug | Double Unit UCB Transplantation, Single Unit UCB Transplantation |
| umbilical cord blood transplantationThe product is infused via IV drip directly into the central line without a needle, pump or filter on Day 0. | Procedure or surgery | Double Unit UCB Transplantation, Single Unit UCB Transplantation |
Primary outcome
Incidence of Engraftment
Time frame: Day 42 After Transplant
Defined as achieving donor derived neutrophil count >500/uL by day 42 in young children with leukemia or myelodysplastic syndrome undergoing a partially matched single unit umbilical cord blood transplant (UCBT) after a myeloablative preparative regimen consisting of busulfan, melphalan and fludarabine.
7 secondary outcomes
Incidence of Transplant-related Mortality (TRM)
Time frame: at 6 months after transplant
Incidence of Platelet Engraftment
Time frame: at 1 year after transplant
Incidence of Acute Graft-versus-host Disease (GVHD) Grade II-IV and Grade III-IV
Time frame: Day 100 After Transplant
Incidence of Chronic Graft-versus-host Disease (GVHD)
Time frame: 1 Year After Transplant
Incidence of Relapse
Time frame: 1 and 2 years after transplant
Overall Survival
Time frame: at 1 and 2 years after transplant
Disease-free Survival
Time frame: at 1 and 2 years after transplant
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 26 Aug 2025.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Christen Ebens, MD principal investigator
Masonic Cancer Center, University of Minnesota
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing filgrastim, busulfan and cyclosporine in people with Leukemia and Myelodysplastic Syndromes, run by Masonic Cancer Center, University of Minnesota at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 17 inclusion and 4 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 26 Aug 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 26 Aug 2025). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Incidence of Engraftment (time frame: Day 42 After Transplant). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 26 Aug 2025.