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A Phase II Trial of Combination Therapy With Arsenic Trioxide (Trisenox) and Gemtuzumab Ozogamicin (Mylotarg) for the Treatment of Adult Patients With Advanced Myelodysplastic Syndrome

Phase 2
18 Years
Not Enrolling
Leukemia, Myelodysplastic Syndromes

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Trial Information

A Phase II Trial of Combination Therapy With Arsenic Trioxide (Trisenox) and Gemtuzumab Ozogamicin (Mylotarg) for the Treatment of Adult Patients With Advanced Myelodysplastic Syndrome



- Determine the efficacy of arsenic trioxide and gemtuzumab ozogamicin to achieve
complete and partial remissions in patients with advanced myelodysplastic syndromes.


- Determine the efficacy of this regimen, in terms of 50% decrease in RBC transfusion
requirements and change in hemoglobin concentration from baseline in patients treated
with this regimen.

- Determine the platelet, neutrophil, bone marrow, and cytogenic response in patients
treated with this regimen.

- Determine the response duration in patients treated with this regimen.

- Determine the quality of life of patients treated with this regimen.

- Determine the safety and toxicity of this regimen in these patients.

OUTLINE: This is a multicenter, open-label study.

Patients receive arsenic trioxide IV over 1 hour once daily on days 1-5 in week 1 and then
twice weekly in weeks 2-12. They also receive gemtuzumab ozogamicin IV over 2 hours on day
8. Treatment repeats every 12 weeks for up to 2 courses in the absence of disease
progression or unacceptable toxicity.

Quality of life is assessed at baseline, every 12 weeks during study treatment, and then 4
weeks after the completion of study treatment.

After completion of study treatment, patients are followed periodically.

PROJECTED ACCRUAL: A total of 40 patients will be accrued for this study.

Inclusion Criteria


- Diagnosis of one of the following hematologic malignancies:

- Myelodysplastic syndromes (MDS) of one of the following FAB classifications:

- Refractory anemia with excess blasts (RAEB) (WHO RAEB-1)

- RAEB in transformation (RAEB-t) (RAEB-2)

- Chronic myelomonocytic leukemia (CMML) with > 5% myeloblasts (WHO CMML-2)

- IPSS score of intermediate-2 or higher in the setting of > 5% myeloblasts

- Acute myeloid leukemia that has evolved from MDS

- Must not be a candidate for bone marrow transplantation as first-line therapy or must
have declined bone marrow transplantation


- ECOG performance status of 0, 1, or 2

- Life expectancy of at least 4 months

- Serum potassium ≥ 4.0 mEq/dL and serum magnesium ≥ 1.8 mg/dL (supplemental
electrolytes allowed)

- Absolute QTc interval < 460 msec

- No serious medical condition, laboratory abnormality, or psychiatric illness that, in
the view of the treating physician, would place the patient at an unacceptable risk
if he or she were to participate in the study or would prevent that person from
giving informed consent

- Not pregnant or nursing

- Fertile patients must be willing to use adequate contraception (barrier method with
spermicidal jelly, IUD, or oral contraceptives)

- Negative pregnancy test

- Creatinine > 2.5 mg/dL

- SGOT or SGPT > 1.5 times upper limit of normal

- Bilirubin > 2.0 mg/dL

- No history of malignancy within the past 3 years other than MDS except basal cell or
squamous cell carcinoma of the skin or carcinoma in situ of the cervix or breast

- Arsenic trioxide is contraindicated in patients who are hypersensitive to arsenic


- No prior bone marrow transplantation

- Must not receive another investigational or approved therapy for MDS within 4 weeks
of study enrollment, including growth factors (within 1 week of study enrollment)

- No prior arsenic trioxide or gemtuzumab ozogamicin

- No other concurrent cytotoxic drugs or investigational agents

Type of Study:


Study Design:

Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Outcome Measure:

Complete and partial remission

Outcome Time Frame:

at 12 weeks post treatment

Safety Issue:


Principal Investigator

Mikkael A. Sekeres, MD, MS

Investigator Role:

Study Chair

Investigator Affiliation:

The Cleveland Clinic


United States: Federal Government

Study ID:




Start Date:

June 2004

Completion Date:

December 2010

Related Keywords:

  • Leukemia
  • Myelodysplastic Syndromes
  • refractory anemia with excess blasts in transformation
  • refractory anemia with excess blasts
  • chronic myelomonocytic leukemia
  • secondary acute myeloid leukemia
  • previously treated myelodysplastic syndromes
  • secondary myelodysplastic syndromes
  • de novo myelodysplastic syndromes
  • Leukemia
  • Myelodysplastic Syndromes
  • Preleukemia



Cleveland Clinic Taussig Cancer CenterCleveland, Ohio  44195