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Gemtuzumab Ozogamicin in Combination With Busulfan and Cyclophosphamid and Allogenic Stem Cell Transplantation in Patients With High Risk CD33+ Acute Myelogenous Leukemia/Myelodysplastic Syndrome/Juvenile Myelomonocytic Leukemia


Phase 1
N/A
30 Years
Not Enrolling
Both
Acute Myeloid Leukemia, Juvenile Myelomonocytic Leukemia, Myelodysplastic Syndrome

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Trial Information

Gemtuzumab Ozogamicin in Combination With Busulfan and Cyclophosphamid and Allogenic Stem Cell Transplantation in Patients With High Risk CD33+ Acute Myelogenous Leukemia/Myelodysplastic Syndrome/Juvenile Myelomonocytic Leukemia


Gemtuzumab Ozogamicin (CMA-676) is a chemotherapeutic agent consisting of 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 leukemia
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 the complex that is internalized, upon which calicheamicin
derivative is released with in the lysosomes of the myeloid cell. The free calicheamicin
derivative then binds to the DNA, resulting in DNA double strand breaks and consequential
cell death.


Eligibility

Inclusion Criteria:



Disease Status

- AML Induction Failure

- AML in 1st, 2nd, or 3rd Relapse (>10% bone marrow blasts)

- AML greater than or equal to 3rd CR

- MDS with >6% bone marrow blasts at diagnosis

- Secondary MDS with less than or equal to 5% bone marrow myeloblasts at diagnosis

- JMML with >6% bone marrow myeloblasts at diagnosis

Disease Immunophenotype Patients (AML only) receiving gemtuzumab ozogamicin must express
minimum of >10% or =10% CD33 positivity. Patients with <10% CD33 positivity will not
receive gemtuzumab ozogamicin.

Organ Function

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 1.5 x normal, or SGOT (AST) or SGPT (ALT) <2.0 x normal or =2.0 x
normal

- Adequate cardiac function defined as:

- Shortening fraction of >27% by echocardiogram, or

- Ejection fraction of >47% by radionuclide angiogram or echocardiogram

- Adequate pulmonary function defined as:

- DLCO >55% or =55% by PFT

- 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/MDS disease at time of conditioning therapy

- Female patients who are pregnant (positive HCG)

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

- Age >65 years

- Has received gemtuzumab in the previous 30 days or has not recovered from prior
gemtuzumab therapy.

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:

Maximal tolerated dose or tolerable dose of Gemtuzumab Ozogamicin (anti-CD33 immunotoxin) therapy combined with Busulfan/ Cyclophosphamide in the conditioning regimen prior to AlloSCT in patients with high risk CD33+ AML/JMML/MDS

Outcome Time Frame:

1 year

Safety Issue:

Yes

Principal Investigator

Mitchell S Cairo, MD

Investigator Role:

Study Chair

Investigator Affiliation:

Columbia University

Authority:

United States: Institutional Review Board

Study ID:

AAAA2533

NCT ID:

NCT00669890

Start Date:

May 2004

Completion Date:

December 2013

Related Keywords:

  • Acute Myeloid Leukemia
  • Juvenile Myelomonocytic Leukemia
  • Myelodysplastic Syndrome
  • AML
  • JMML
  • MDS
  • Allogenic Atem Cell Transplant
  • Gemtuzumab Ozogamicin
  • Leukemia
  • Leukemia, Myeloid, Acute
  • Leukemia, Myeloid
  • Myelodysplastic Syndromes
  • Preleukemia
  • Leukemia, Myelomonocytic, Acute
  • Leukemia, Myelomonocytic, Chronic
  • Leukemia, Myelomonocytic, Juvenile

Name

Location

Morgan Stanley Children's Hospital of NYPNew York, New York  10032