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Hematopoietic Cell Transplantation in the Treatment of Infant Leukemia Using Double Umbilical Cord Transplantation


Phase 2
N/A
2 Years
Open (Enrolling)
Both
Leukemia, Myelodysplastic Syndromes

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Trial Information

Hematopoietic Cell Transplantation in the Treatment of Infant Leukemia Using Double Umbilical Cord Transplantation


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


Inclusion Criteria:



- Patients on this trial will receive two partially HLA matched umbilical cord blood
(UCB) units, if available (treatment arm 1). If two units are not available, then
single UCB unit transplantation will be allowed (on treatment arm 2).

- UCB units will be selected according to the University Of Minnesota UCB Graft
Selection Algorithm. The unrelated cord blood donor(s) must be 4-6/6 HLA-A, B, DRB1
matched with the recipient (HLA matching using molecular techniques: A and B to
antigen level resolution and DR to allele level resolution).

- Patients aged ≤ 2 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.

- Minimal Residual Disease (MRD): 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).

- Recipients must have a Lansky score > or = 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

- Prior myeloablative transplant within the last 6 months.

- Evidence of active extramedullary disease (including central nervous system
leukemia).

Type of Study:

Interventional

Study Design:

Allocation: Non-Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Parallel Assignment, Masking: Open Label, Primary Purpose: Treatment

Outcome Measure:

Incidence of Engraftment

Outcome Description:

Defined as achieving donor derived neutrophil count >500/uL by day 42 in young children with leukemia or myelodysplastic syndrome treated with busulfan, melphalan and fludarabine followed by umbilical cord blood transplantation (UCBT) with two partially HLA matched units.

Outcome Time Frame:

Day 42 After Transplant

Safety Issue:

Yes

Principal Investigator

Michael R. Verneris, MD

Investigator Role:

Principal Investigator

Investigator Affiliation:

Masonic Cancer Center, University of Minnesota

Authority:

United States: Institutional Review Board

Study ID:

2005LS075

NCT ID:

NCT00357565

Start Date:

December 2005

Completion Date:

August 2015

Related Keywords:

  • 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 syndromes
  • secondary myelodysplastic syndromes
  • refractory anemia with excess blasts in transformation
  • refractory anemia with excess blasts
  • refractory anemia
  • de novo myelodysplastic syndromes
  • childhood myelodysplastic syndromes
  • Leukemia
  • Myelodysplastic Syndromes
  • Preleukemia

Name

Location

Masonic Cancer Center, University of MinnesotaMinneapolis, Minnesota  55455