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Immunochemotherapy and AlloSCT in Patients With High Risk CD33+ AML/MDS

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details. Identifier: NCT02221310
Recruitment Status : Recruiting
First Posted : August 20, 2014
Last Update Posted : September 2, 2020
Information provided by (Responsible Party):
Mitchell Cairo, New York Medical College

Brief Summary:
Targeted immune therapy with gemtuzumab ozogamicin (Mylotarg) in combination with chemotherapy followed by allogeneic stem cell transplantation will be given to patients with high risk acute myelogenous leukemia (AML) or myelodysplastic syndrome (MDS).

Condition or disease Intervention/treatment Phase
Acute Myelogenous Leukemia Myelodysplastic Syndrome Drug: Gemtuzumab Ozogamicin Phase 2

Detailed Description:
Gemtuzumab ozogamicin in combination with busulfan and cyclophosphamide (immunochemotherapy) conditioning followed by allogeneic stem cell transplantation + anti-thymocyte globulin (unrelated donors only) in patients with high risk CD33+AML or MDS meeting eligibility criteria.

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 25 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: A Pilot Study of Gemtuzumab Ozogamicin in Combination With Busulfan and Cyclophosphamide (Immunochemotherapy) and Allogeneic Stem Cell Transplantation in Patients With High Risk Acute Myelogenous Leukemia and Myelodysplastic Syndrome
Study Start Date : November 2011
Estimated Primary Completion Date : June 2021
Estimated Study Completion Date : June 2022

Arm Intervention/treatment
Experimental: Gemtuzumab Ozogamicin
Conditioning therapy with Gemtuzumab Ozogamicin in combination with busulfan and cyclophosphamide chemotherapy followed by allogeneic stem cell transplantation.
Drug: Gemtuzumab Ozogamicin
Gemtuzumab Ozogamicin 7.5 mg/m^2/dose given IV over 2 hours once during conditioning
Other Name: Mylotarg

Primary Outcome Measures :
  1. Response rate [ Time Frame: 1 year ]
    overall response rate (CR + PR) in patients receiving GO, busulfan and cyclophosphamide and AlloSCT in patients with measurable disease (relapse/refractory) with high risk CD33+ AML/MDS

Information from the National Library of Medicine

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Ages Eligible for Study:   up to 25 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

Inclusion Criteria:

  • Disease Status:

    • History of AML Induction/Reinduction Failure
    • AML 1st Complete Remission (CR) with poor cytogenetics
    • AML 2nd CR with minimal residual disease (MRD)
    • AML 3rd CR
    • AML in refractory relapse but ≤25% bone marrow leukemia blasts
    • MDS with >6% bone marrow blasts at diagnosis
    • Secondary MDS with ≤5% bone marrow myeloblasts at diagnosis

Disease Immunophenotype:

• Disease must express a minimum of >/= 10% CD33+ for patients with AML

Organ Function:

• Adequate renal function, adequate liver function, adequate cardiac function, adequate pulmonary function Age: ≤25 years of age Donor: matched family donor, unrelated cord blood donor, unrelated adult donor

Exclusion Criteria:

  • Patients with active central nervous system (CNS) AML/MDS disease at time of conditioning therapy
  • Female patients who are pregnant
  • Karnofsky <50% or Lansky <50% if 10 years or less
  • Age >25 years
  • Has received gemtuzumab in the previous 30 days or has not recovered from prior gemtuzumab therapy.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT02221310

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Contact: Mitchell S Cairo, MD 914-594-2150
Contact: Erin Morris, RN 714-964-5359

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United States, New York
New York Medical College Recruiting
Valhalla, New York, United States, 10595
Contact: Sandra Fabricatore, RN, PNP    914-594-2152   
Contact: Erin Morris, RN    714-964-5359   
Principal Investigator: Mitchell S Cairo, MD         
Sponsors and Collaborators
New York Medical College
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Principal Investigator: Mitchell S Cairo, MD New York Medical College
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Responsible Party: Mitchell Cairo, Principal Investigator, New York Medical College Identifier: NCT02221310    
Other Study ID Numbers: NYMC-515
L-10,349 ( Other Identifier: New York Medical College )
First Posted: August 20, 2014    Key Record Dates
Last Update Posted: September 2, 2020
Last Verified: September 2020
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Undecided
Keywords provided by Mitchell Cairo, New York Medical College:
targeted therapy
immune therapy
Additional relevant MeSH terms:
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Leukemia, Myeloid
Leukemia, Myeloid, Acute
Myelodysplastic Syndromes
Neoplasms by Histologic Type
Bone Marrow Diseases
Hematologic Diseases
Precancerous Conditions
Antineoplastic Agents, Immunological
Antineoplastic Agents