Combination 5-azacitidine and Gemtuzumab Ozogamicin Therapy for Treatment of Relapsed Acute Myeloid Leukemia (AML)

This study is currently recruiting participants. (see Contacts and Locations)
Verified May 2014 by University of California, San Diego
Celgene Corporation
Information provided by (Responsible Party):
Edward Ball, University of California, San Diego Identifier:
First received: October 2, 2008
Last updated: May 27, 2014
Last verified: May 2014

This study will test an experimental combination of the drugs Mylotarg and 5-azacitidine in the hopes of finding a treatment that may be effective against Acute Myeloid Leukemia that has come back after treatment.

Condition Intervention Phase
Acute Myeloid Leukemia
Drug: 5-Azacitidine
Drug: Gemtuzumab ozogamicin
Phase 1
Phase 2

Study Type: Interventional
Study Design: Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: A Phase I/II Trial of the Combination 5-azacitidine and Gemtuzumab Ozogamicin Therapy for Treatment of Relapsed AML

Resource links provided by NLM:

Further study details as provided by University of California, San Diego:

Primary Outcome Measures:
  • In Phase I the Maximum Tolerated Dose will be the maximum dose at which fewer than one-third of patients experience Dose Limiting Toxicity [ Time Frame: approximately 4 years ] [ Designated as safety issue: Yes ]

Secondary Outcome Measures:
  • In Phase II response to the combination treatment of Mylotarg with 5-azacitidine [ Time Frame: Response will be assessed when evaluated at the time the ANC has reached 1000/mm3 for three consecutive days ] [ Designated as safety issue: Yes ]

Estimated Enrollment: 36
Study Start Date: July 2005
Estimated Study Completion Date: February 2015
Estimated Primary Completion Date: August 2014 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Treatment
5-Azacitidine, Gemtuzumab ozogamicin
Drug: 5-Azacitidine
A = given in cohorts of 3 starting with 2 doses of 5-azacitidine. The doses of 5-azacitidine will be escalated to 4 and then 6 doses if the dose escalation rules permit
Other Name: Vidaz
Drug: Gemtuzumab ozogamicin
M = Mylotarg given 2 times over 2 weeks
Other Name: Mylotarg

Detailed Description:

In the Phase I portion of the study patients with a diagnosis of AML who have relapsed disease will be treated with an assigned number of doses of 5 azacitidine followed by Mylotarg administered two times over two weeks

In the Phase II portion of the study patients will be treated with the dose of 5azacitidine determined to be safe in the Phase I portion of the study followed by Mylotarg.

A sample of blood or bone marrow will be obtained prior to initiation of treatment and another sample obtained after treatment with 5-azacitidine but before Mylotarg and the samples will be tested in the laboratory to determine response to treatment.


Ages Eligible for Study:   18 Years to 90 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No

Inclusion Criteria:

  • Diagnosis of Relapsed AML
  • Eastern Cooperative Oncology Group (ECOG) performance status 0-2, and life expectancy > 3 months
  • ≥ 18 years old
  • Previously untreated for current AML relapse
  • Adequate organ function
  • Written informed consent

Exclusion Criteria:

  • Pregnant or breast-feeding women
  • Growth factors that support platelet or white cell number or function must not have been administered within the past 7 days
  • Currently receiving another investigational drug
  • Currently receiving other anti-cancer agents
  • Uncontrolled infection
  • HIV positive
  • Received previous therapy with either Mylotarg or 5-azacitidine
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its identifier: NCT00766116

Contact: Edward D Ball, MD 858-822-6600
Contact: Jesika Reiner 858-822-5364

United States, California
UCSD Moores Cancer Center Recruiting
La Jolla, California, United States, 92093
Contact: Jesika Reiner    858-822-5364   
Contact: Jenny Lin    858-822-6396   
Stanford University Completed
Stanford, California, United States, 94305
United States, Georgia
Northside Hospital/BMTGA Completed
Atlanta, Georgia, United States, 30342
Sponsors and Collaborators
University of California, San Diego
Celgene Corporation
Principal Investigator: Edward D Ball, MD UCSD
  More Information

No publications provided

Responsible Party: Edward Ball, Professor of medicine, University of California, San Diego Identifier: NCT00766116     History of Changes
Other Study ID Numbers: 090516
Study First Received: October 2, 2008
Last Updated: May 27, 2014
Health Authority: United States: Institutional Review Board
United States: Food and Drug Administration

Keywords provided by University of California, San Diego:

Additional relevant MeSH terms:
Leukemia, Myeloid
Leukemia, Myeloid, Acute
Neoplasms by Histologic Type
Antimetabolites, Antineoplastic
Molecular Mechanisms of Pharmacological Action
Pharmacologic Actions
Antineoplastic Agents
Therapeutic Uses
Enzyme Inhibitors processed this record on September 30, 2014