NCT00357565 · REGISTRY CONDITION SEARCH

Hematopoietic Stem Cell Transplantation in the Treatment of Infant Leukemia

Found through the registry condition index, not selected as a treatment recommendation. This discovery collection was restricted to studies with posted results.

Phase: PHASE2
Status at capture: COMPLETED
Registry last update: 2025-08-26

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.

Open the original ClinicalTrials.gov record → · Download preserved record

What did the study report?

Outcomes, safety and baseline populations are separate source sections. Quality-of-life measures appear under their original outcome titles.

flow

Participant flow

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baseline

Baseline characteristics

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outcome · POSTED

Incidence of Engraftment

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outcome · POSTED

Incidence of Transplant-related Mortality (TRM)

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outcome · POSTED

Incidence of Platelet Engraftment

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outcome · POSTED

Incidence of Acute Graft-versus-host Disease (GVHD) Grade II-IV and Grade III-IV

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outcome · POSTED

Incidence of Chronic Graft-versus-host Disease (GVHD)

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outcome · POSTED

Incidence of Relapse

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outcome · POSTED

Overall Survival

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outcome · POSTED

Disease-free Survival

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adverse events

Adverse events

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more info

Limitations, agreements, and source contact

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Who could take part

eligibility Criteria: Inclusion Criteria: * 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: * 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. Exclusion Criteria: * 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).
healthy Volunteers: false
maximum Age: 3 Years
sex: ALL
std Ages: CHILD

Treatment arms and interventions

arm Groups

description
Patients that receive 2 units of umbilical cord blood transplantation (UCBT).
intervention Names
Biological: filgrastim
Drug: busulfan
Drug: cyclosporine
Drug: fludarabine phosphate
Drug: melphalan
Drug: mycophenolate mofetil
Procedure: umbilical cord blood transplantation
label
Double Unit UCB Transplantation
type
EXPERIMENTAL
description
Patients that receive one unit of umbilical cord blood transplantation (only if 2 adequate size and matched units are not available).
intervention Names
Biological: filgrastim
Drug: busulfan
Drug: cyclosporine
Drug: fludarabine phosphate
Drug: melphalan
Drug: mycophenolate mofetil
Procedure: umbilical cord blood transplantation
label
Single Unit UCB Transplantation
type
EXPERIMENTAL

interventions

arm Group Labels
Double Unit UCB Transplantation
Single Unit UCB Transplantation
description
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.
name
filgrastim
other Names
G-CSF
type
BIOLOGICAL
arm Group Labels
Double Unit UCB Transplantation
Single Unit UCB Transplantation
description
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.
name
busulfan
other Names
Busulfex
type
DRUG
arm Group Labels
Double Unit UCB Transplantation
Single Unit UCB Transplantation
description
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.
name
cyclosporine
other Names
CSA
type
DRUG
arm Group Labels
Double Unit UCB Transplantation
Single Unit UCB Transplantation
description
Administered 25 mg/m\^2 intravenous (IV) over 60 minutes on Days -4 through -2.
name
fludarabine phosphate
other Names
Fludara
type
DRUG
arm Group Labels
Double Unit UCB Transplantation
Single Unit UCB Transplantation
description
Administered 60 mg/m\^2 intravenous (IV) over 30 minutes on Days -4 through -2.
name
melphalan
other Names
Alkeran
type
DRUG
arm Group Labels
Double Unit UCB Transplantation
Single Unit UCB Transplantation
description
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).
name
mycophenolate mofetil
other Names
MMF
type
DRUG
arm Group Labels
Double Unit UCB Transplantation
Single Unit UCB Transplantation
description
The product is infused via IV drip directly into the central line without a needle, pump or filter on Day 0.
name
umbilical cord blood transplantation
type
PROCEDURE

Study design

allocation

NON_RANDOMIZED

intervention Model

PARALLEL

masking Info

masking: NONE

primary Purpose

TREATMENT

Enrollment

count: 34
type: ACTUAL

Registered outcome plans (not posted results)

primary Outcomes

description
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.
measure
Incidence of Engraftment
time Frame
Day 42 After Transplant

secondary Outcomes

description
defined as death due to transplant
measure
Incidence of Transplant-related Mortality (TRM)
time Frame
at 6 months after transplant
description
defined as platelet count \> 50,000
measure
Incidence of Platelet Engraftment
time Frame
at 1 year after transplant
description
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.
measure
Incidence of Acute Graft-versus-host Disease (GVHD) Grade II-IV and Grade III-IV
time Frame
Day 100 After Transplant
description
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.
measure
Incidence of Chronic Graft-versus-host Disease (GVHD)
time Frame
1 Year After Transplant
description
defined using standard criteria (bone marrow blast count and cytogenetics).
measure
Incidence of Relapse
time Frame
1 and 2 years after transplant
description
Alive after transplant.
measure
Overall Survival
time Frame
at 1 and 2 years after transplant
description
defined as patients who are alive and in hematological remission.
measure
Disease-free Survival
time Frame
at 1 and 2 years after transplant

Full study description

brief 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. 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

Source references

Source notices and limitations

Discovery and provenance

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Preserved source evidence · Not medical advice