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TerminatedNCT00669890High RiskUpdated Apr 22, 2015

Allogenic Stem Cell Transplantation in Patients With High Risk CD33+ AML/MDS/JMML

A Phase 1 interventional study of Gemtuzumab Ozogamicin and Busulfan in Acute Myeloid Leukemia, Juvenile Myelomonocytic Leukemia and Myelodysplastic Syndrome, sponsored by New York Medical College. Terminated at 1 site in United States. Open to participants aged Up to 30 Years. Per ClinicalTrials.gov, last updated 2015-04-22.

Sponsored by New York Medical College · Phase 1, Interventional, and Treatment

Why this study was terminated
PI left institution
Phase
Phase 1
Study type
Interventional
Enrollment
12
Allocation
Not applicable
Ages
Up to 30 Years
Sex
All
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Study summary

The addition of gemtuzumab ozogamicin (GO) in combination with Busulfan/Cyclophosphamide followed by AlloSCT in patients with high risk CD33+ AML/JMML/MDS will be safe and well tolerated.

This study will attempt to determine the maximum tolerated dose of the immune therapy (gemtuzumab) when given in combination with the myeloablative (high dose) drugs used in this study for allogeneic stem cell transplant. (Part A)

Read the detailed description

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.

02

Conditions studied

  • Acute Myeloid Leukemia
  • Juvenile Myelomonocytic Leukemia
  • Myelodysplastic Syndrome

Keywords

  • AML
  • JMML
  • MDS
  • Allogenic Atem Cell Transplant
  • Gemtuzumab Ozogamicin
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In context

Leukemia

5,441 studies on the registry are indexed under Leukemia; 636 are open to participants now.

This study's enrollment of 12 is below the median of 38 across 4,247 interventional studies indexed under Leukemia.

Browse Leukemia studies →

Lead sponsor

New York Medical College is the lead sponsor of 69 studies on the registry; 23 are open to participants now.

Of its 5 completed or terminated interventional studies of FDA-regulated products, 0 (0%) have results posted.

Counted across the registry records on this site, refreshed daily.

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Who can participate

Ages eligible
Up to 30 Years
Sexes eligible
All
Accepts healthy volunteers
No

Eligibility

Inclusion criteria

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

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.
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Study design

Phase
Phase 1
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrollment
12 participants (actual)

Study arms

  • Experimental
    study 515

    Drug: Gemtuzumab Ozogamicin · Drug: Busulfan · Drug: Cyclophosphamide · Drug: Thymoglobulin · Drug: Tacrolimus · Drug: Mycophenolate Mofetil · Drug: Methotrexate

Interventions

  • DrugGemtuzumab Ozogamicin

    Dose Escalation

    Also known as: Gemtuzumab

  • DrugBusulfan

    Conditioning Regimen

    Also known as: Busulfex

  • DrugCyclophosphamide

    Conditioning Regimen

    Also known as: Endoxan, Cytoxan

  • DrugThymoglobulin

    (Unrelated Donors only)

    Also known as: ATG

  • DrugTacrolimus

    GVHD Prophylaxis

    Also known as: FK506

  • DrugMycophenolate Mofetil

    GVHD Prophylaxis

    Also known as: MMF

  • DrugMethotrexate

    GVHD Prophylaxis

    Also known as: MTX

06

What researchers measure

Primary outcomes

  1. 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

    Time frame: 1 year

Secondary outcomes

  1. Changes, if applicable, of minimal residual disease (cytogenetics, FISH, RT-PCR) in patients with high risk CD33+ AML/JMML/MDS after AlloSCT.

    Time frame: 1 year

  2. Progression Free Survival (PFS), overall survival (OS), and disease free survival (DFS), (if applicable), following GO, Bu/CY and AlloSCT in patients with high risk CD33+ AML/JMML/MDS.

    Time frame: 1 year

  3. Quality of life before and after GO, Bu/CY conditioning and AlloSCT in patients with high risk CD33+ AML/JMML/MDS

    Time frame: 1 year

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Study locations

1 site
  • Morgan Stanley Children's Hospital of NYP
    New York City, New York 10032, United States
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References and documents

09

Updates

Tracking since Sep 25, 2026
No changes since tracking began. The registry record was last updated on Apr 22, 2015, before this site started recording changes on Sep 25, 2026. Its history is on ClinicalTrials.gov ↗
10

Registry details

Key details

Study ID
NCT00669890
Lead sponsor
New York Medical College
Responsible party
Mitchell Cairo (Professor of Pediatrics, New York Medical College) — Principal investigator
First posted
May 1, 2008
Start date
May 2004
Primary completion
Dec 2012
Completion
Dec 2013
Last update
Apr 22, 2015

Study contacts

Mitchell S Cairo, MD
study chair · Columbia University

Oversight

Data monitoring committee
Yes
View the source record on ClinicalTrials.gov ↗

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