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Allogeneic Stem Cell Transplantation Followed By Targeted Immune Therapy In Average Risk Acute Myelogenous Leukemia/Myelodysplastic Syndrome/Juvenile Myelomonocytic Leukemia (Aml/Mds/Jmml)


Phase 1
1 Month
64 Years
Open (Enrolling)
Both
Acute Myelogenous Leukemia, Myelodysplastic Syndrome, Juvenile Myelomonocytic Leukemia

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Trial Information

Allogeneic Stem Cell Transplantation Followed By Targeted Immune Therapy In Average Risk Acute Myelogenous Leukemia/Myelodysplastic Syndrome/Juvenile Myelomonocytic Leukemia (Aml/Mds/Jmml)


Gemtuzumab Ozogamicin (CMA-676) is a chemotherapeutic agent consisting of a recombinant
humanized anti-CD33 antibody conjugated with calicheamicin, a highly potent cytotoxic
antitumor antibiotic. The antibody portion of Gemtuzumab binds specifically to the CD33
antigen, a sialic acid-dependent adhesion protein expressed on the surface of leukemic
blasts, normal and leukemic myeloid colony-forming cells, including leukemic clonogenic
precursors, but excluding pluripotent hematopoietic stem cells and nonhematopoietic cells.
This results in formation of a complex that is internalized, upon which the calicheamicin
derivative is released within the lysosomes of the myeloid cell. The free calicheamicin
derivative then binds to DNA, resulting in DNA double strand breaks and consequential cell
death. Over 80% of AML patients possess myeloid blast cells with CD33 surface antigen
expression.


Inclusion Criteria:



- Disease Status

- AML 1st CR with a matched family donor (excluding Downs Syndrome, APL, and
patients consented to and registered on an upfront AML COG study with a matched
family donor)

- AML 1st CR [excluding Downs Syndrome, APL, and chromosome translocation (8;21)
or inversion (16)] with unrelated donor

- AML 2nd CR

- MDS and < 5% bone marrow myeloblasts at diagnosis (de novo patients only)

- JMML and < 5% bone marrow myeloblasts at diagnosis

- Disease must express a minimum of >10% CD33 positivity for patients with AML

- Patients must have adequate organ function as defined below:

- Adequate renal function defined as:

- Serum creatinine < 1.5 x normal, or

- Creatinine clearance or radioisotope GFR 40 ml/min/m2 or > 60 ml/min/1.73 m2
or an equivalent GFR as determined by the institutional normal range

- Adequate liver function defined as:

- Total bilirubin 2.0 x ULN, or SGOT (AST) or SGPT (ALT) < 5.0 xULN

- Adequate cardiac function defined as:

- Shortening fraction of > 25% by echocardiogram, or

- Ejection fraction of > 45% by radionuclide angiogram or echocardiogram

- Adequate pulmonary function defined as:

- DLCO > 40% by PFT (Uncorrected)

- For children who are uncooperative, no evidence of dyspnea at rest, no exercise
intolerance, and a pulse oximetry > 94% on room air

Exclusion Criteria:

- Patients with active CNS AML/JMML disease at time of preparative regimen

- Secondary MDS

- Female patients who are pregnant (positive HCG)

- Karnofsky <70% or Lansky <50% if 10 years or less

- Age >65 years

- Seropositive for HIV

- Patients consented to and registered on an upfront COG AML study with a matched
family donor

Type of Study:

Interventional

Study Design:

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

Outcome Measure:

To determine the feasibility and toxicity of a Reduced Intensity (RI) regimen, AlloSCT followed by targeted immune therapy, Gemtuzumab Ozogamicin (GO) , in average risk AML/JMML/MDS.

Outcome Time Frame:

Day 60, Day 100, Day 180, 1 year, 2 years

Safety Issue:

Yes

Principal Investigator

Monica Bhatia, MD

Investigator Role:

Principal Investigator

Investigator Affiliation:

Columbia University

Authority:

United States: Institutional Review Board

Study ID:

AAAA6378

NCT ID:

NCT01020539

Start Date:

September 2002

Completion Date:

September 2013

Related Keywords:

  • Acute Myelogenous Leukemia
  • Myelodysplastic Syndrome
  • Juvenile Myelomonocytic Leukemia
  • AML
  • MDS
  • JMML
  • Leukemia
  • Leukemia, Myeloid, Acute
  • Leukemia, Myeloid
  • Myelodysplastic Syndromes
  • Preleukemia
  • Leukemia, Myelomonocytic, Acute
  • Leukemia, Myelomonocytic, Chronic
  • Leukemia, Myelomonocytic, Juvenile

Name

Location

Columbia University Medical CenterNew York, New York  10032