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A Dose Finding Study of the Safety of Gemtuzumab Ozogamicin Combined With Conventional Chemotherapy for Patients With Relapsed or Refractory Acute Myeloid Leukemia

Phase 1
21 Years
Not Enrolling
Leukemia, Myelodysplastic Syndromes

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Trial Information

A Dose Finding Study of the Safety of Gemtuzumab Ozogamicin Combined With Conventional Chemotherapy for Patients With Relapsed or Refractory Acute Myeloid Leukemia


- Determine the safety and maximum tolerated dose of gemtuzumab ozogamicin in combination
with conventional chemotherapy in patients with relapsed or refractory acute myeloid
leukemia or myelodysplastic syndromes.

- Determine the efficacy of this regimen in these patients.

- Correlate the likelihood of leukemic blast cells to undergo apoptosis in vitro with the
efficacy of this regimen in these patients.

- Correlate drug resistance as manifested by dye efflux or multiple drug resistance-1
expression by leukemic blast cells with the efficacy of this regimen in these patients.

OUTLINE: This is a dose-escalation, multicenter study of gemtuzumab ozogamicin. Patients are
assigned by cohort to 1 of 2 treatment regimens.

- Regimen A: Patients receive cytarabine IV over 2 hours every 12 hours on days 1-4,
mitoxantrone IV over 1 hour on days 3-6, and gemtuzumab ozogamicin IV over 2 hours on
day 7.

- Regimen B: Patients receive cytarabine IV over 3 hours every 12 hours on days 1, 2, 8,
and 9, asparaginase intramuscularly on days 2 and 9, and gemtuzumab ozogamicin IV over
2 hours on day 3.

Cohorts of 3-6 patients receive de-escalating doses of gemtuzumab ozogamicin until the
maximum tolerated dose (MTD) is determined. The MTD is defined as the dose below that at
which 2 of 3 or 2 of 6 patients experience dose-limiting toxicity.

Patients are followed monthly for 6 months, every 2 months for 6 months, every 6 months for
2 years, and then annually thereafter.

PROJECTED ACCRUAL: A total of 52 patients will be accrued for this study within 1.5 years.

Inclusion Criteria


- Diagnosis of primary acute myeloid leukemia (AML) or myelodysplastic syndromes

- Relapsed (remission duration less than 1 year) OR

- Failed induction (failed to achieve an initial complete response)

- Patients with AML as a second malignant neoplasm allowed provided no other prior
therapy for AML

- M2 or M3 bone marrow aspirate at time of study entry

- No Fanconi's anemia

- No known CNS leukemia



- 21 and under

Performance status:

- ECOG 0-2

Life expectancy:

- Not specified


- Not specified


- Bilirubin no greater than 1.5 times normal

- AST or ALT less than 2.5 times upper limit of normal

- No history of veno-occlusive disease of the liver defined as weight increase of more
than 5% over baseline and serum bilirubin greater than 5 mg/dL within 20 days after
receipt of chemotherapy


- Creatinine no greater than 1.5 times normal OR

- Creatinine clearance or radioisotope glomerular filtration rate (GFR) at least 70
mL/min OR

- Equivalent GFR by institutional normal range


- Shortening fraction more than 27% by echocardiogram or normal for institution OR

- Ejection fraction more than 50% by MUGA


- Not pregnant or nursing


Biologic therapy:

- At least 180 days since prior hematopoietic stem cell transplantation


- Not specified

Endocrine therapy:

- Not specified


- Not specified


- Not specified

Type of Study:


Study Design:

Primary Purpose: Treatment

Principal Investigator

Richard Aplenc, MD, MSCE

Investigator Role:

Study Chair

Investigator Affiliation:

Children's Hospital of Philadelphia


United States: Federal Government

Study ID:




Start Date:

July 2002

Completion Date:

Related Keywords:

  • Leukemia
  • Myelodysplastic Syndromes
  • recurrent childhood acute myeloid leukemia
  • secondary acute myeloid leukemia
  • previously treated myelodysplastic syndromes
  • Leukemia
  • Leukemia, Myeloid, Acute
  • Leukemia, Myeloid
  • Myelodysplastic Syndromes
  • Preleukemia



Children's Hospital of Philadelphia Philadelphia, Pennsylvania  19104
Mayo Clinic Cancer Center Rochester, Minnesota  55905
Emory University Hospital - Atlanta Atlanta, Georgia  30322
Indiana University Cancer Center Indianapolis, Indiana  46202-5265
Barbara Ann Karmanos Cancer Institute Detroit, Michigan  48201
University of Mississippi Medical Center Jackson, Mississippi  39216-4505
Duke Comprehensive Cancer Center Durham, North Carolina  27710
University of Texas Health Science Center at San Antonio San Antonio, Texas  78284-7811
Midwest Children's Cancer Center Milwaukee, Wisconsin  53226
Van Elslander Cancer Center at St. John Hospital and Medical Center Grosse Pointe Woods, Michigan  48236
Vanderbilt-Ingram Cancer Center Nashville, Tennessee  37232-6838
Marshfield Clinic - Marshfield Center Marshfield, Wisconsin  54449
Newark Beth Israel Medical Center Newark, New Jersey  07112
University of Wisconsin Comprehensive Cancer Center Madison, Wisconsin  53792
Siteman Cancer Center at Barnes-Jewish Hospital Saint Louis, Missouri  63110
Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins Baltimore, Maryland  21231-2410
Cancer Research Center of Hawaii Honolulu, Hawaii  96813
Cleveland Clinic Taussig Cancer Center Cleveland, Ohio  44195
Children's Hospital Los Angeles Los Angeles, California  90027-0700
Children's Hospital of Orange County Orange, California  92668
Children's National Medical Center Washington, District of Columbia  20010-2970
Children's Mercy Hospital Kansas City, Missouri  64108
Children's Hospital of Pittsburgh Pittsburgh, Pennsylvania  15213
Children's Hospital and Regional Medical Center - Seattle Seattle, Washington  98105
All Children's Hospital St. Petersburg, Florida  33701
Children's Memorial Hospital - Chicago Chicago, Illinois  60614
Dana-Farber/Harvard Cancer Center at Dana Farber Cancer Institute Boston, Massachusetts  02115
St. Jude Children's Research Hospital Memphis, Tennessee  38105-2794
Cook Children's Medical Center - Fort Worth Fort Worth, Texas  76104
Phoenix Children's Hospital Phoenix, Arizona  85016-7710
Children's Hospital Medical Center of Akron Akron, Ohio  44308
Children's Medical Center - Dayton Dayton, Ohio  45404
Texas Tech University Health Sciences Center School of Medicine Amarillo, Texas  79106
Children's Hospital of Austin Austin, Texas  78701
Arkansas Cancer Research Center at University of Arkansas for Medical Sciences Little Rock, Arkansas  72205
Markey Cancer Center at University of Kentucky Chandler Medical Center Lexington, Kentucky  40536-0084
Herbert Irving Comprehensive Cancer Center at Columbia University New York, New York  10032
Blumenthal Cancer Center at Carolinas Medical Center Charlotte, North Carolina  28232-2861
Fairview University Medical Center - University Campus Minneapolis, Minnesota  55455
Oklahoma University Medical Center Oklahoma City, Oklahoma  73104
M.D. Anderson Cancer Center at University of Texas Houston, Texas  77030
Stanford Cancer Center at Stanford University Medical Center Stanford, California  94305
Comprehensive Cancer Center at University of Alabama at Birmingham Birmingham, Alabama  35294
Jonathan Jaques Children's Cancer Center at Miller Children's Hospital Long Beach, California  90801
Children's Hospital and Health Center - San Diego San Diego, California  92123-4282
University of Florida Shands Cancer Center Gainesville, Florida  32610-0232
Florida Hospital Cancer Institute at Florida Hospital Orlando Orlando, Florida  32803-1273
St. Joseph's Cancer Institute at St. Joseph's Hospital Tampa, Florida  33607
Kaplan Cancer Center at St. Mary's Medical Center West Palm Beach, Florida  33407
St. Vincent Indianapolis Hospital Indianapolis, Indiana  46260
CancerCare of Maine at Eastern Maine Medial Center Bangor, Maine  04401
Alvin and Lois Lapidus Cancer Institute at Sinai Hospital Baltimore, Maryland  21215
C.S. Mott Children's Hospital at University of Michigan Ann Arbor, Michigan  48109-0238
SUNY Upstate Medical University Hospital Syracuse, New York  13210
Cincinnati Children's Hospital Medical Center Cincinnati, Ohio  45229-3039
Rainbow Babies and Children's Hospital Cleveland, Ohio  44106-5000
Tod Children's Hospital - Forum Health Youngstown, Ohio  44501
Hollings Cancer Center at Medical University of South Carolina Charleston, South Carolina  29425
Greenville Hospital System Cancer Center Greenville, South Carolina  29605
Simmons Comprehensive Cancer Center at University of Texas Southwestern Medical Center - Dallas Dallas, Texas  75390
Methodist Children's Hospital of South Texas San Antonio, Texas  78229-3993
Massey Cancer Center at Virginia Commonwealth University Richmond, Virginia  23298-0037
Providence Cancer Center at Sacred Heart Medical Center Spokane, Washington  99220-2555
Edwards Comprehensive Cancer Center at Cabell Huntington Hospital Huntington, West Virginia  25701
James P. Wilmot Cancer Center at University of Rochester Medical Center Rochester, New York  14642
Cancer Institute of New Jersey at UMDNJ - Robert Wood Johnson Medical School New Brunswick, New Jersey  08903
Cancer Center at Hackensack University Medical Center Hackensack, New Jersey  07601
Children's Hospital Cancer Center Denver, Colorado  80218
Columbus Children's Hospital Columbus, Ohio  43205-2696
Children's Hospital of Minnesota - Minneapolis Minneapolis, Minnesota  55404