NCT00357565

Hematopoietic Stem Cell Transplantation in the Treatment of Infant Leukemia

Hematopoietic Cell Transplantation in the Treatment of Infant Leukemia and Myelodysplastic Syndrome

• Status: Completed
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 34 (actual)
• Sponsor: Masonic Cancer Center, University of Minnesota · Academic / Other
• Sex: All
• Age: 3 Years
• Healthy volunteers: Not accepted

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

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

Type	Name	Description
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.

Timeline

• Start date: 2005-11-01
• Primary completion: 2024-06-01
• Completion: 2025-03-11
• First posted: 2006-07-27
• Last updated: 2025-08-26
• Results posted: 2025-08-26

Locations

1 site across 1 country: United States