Hematopoietic Stem Cell Transplantation in the Treatment of Infant Leukemia

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.

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
• Previously Treated Myelodysplastic Syndrome
• Secondary Myelodysplastic Syndrome
• Refractory Anemia With Excess Blasts in Transformation
• Refractory Anemia With Excess Blasts
• Refractory Anemia
• De Novo Myelodysplastic Syndrome
• Childhood Myelodysplastic Syndrome

Interventions

BIOLOGICAL

filgrastim

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.

DRUG

busulfan

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.

DRUG

cyclosporine

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.

DRUG

fludarabine phosphate

Administered 25 mg/m\^2 intravenous (IV) over 60 minutes on Days -4 through -2.

DRUG

melphalan

Administered 60 mg/m\^2 intravenous (IV) over 30 minutes on Days -4 through -2.

DRUG

mycophenolate mofetil

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).

PROCEDURE

umbilical cord blood transplantation

The product is infused via IV drip directly into the central line without a needle, pump or filter on Day 0.

Sponsors & Collaborators

• Masonic Cancer Center, University of Minnesota

  lead OTHER

Principal Investigators

• Christen Ebens, MD · Masonic Cancer Center, University of Minnesota

Study Design

Allocation

NON_RANDOMIZED

Purpose

TREATMENT

Masking

NONE

Model

PARALLEL

Eligibility

Max Age

3 Years

Sex

ALL

Healthy Volunteers

No

Timeline & Regulatory

Start

2005-11-30

Primary Completion

2024-06-30

Completion

2025-03-11

Countries

• United States

Study Locations