Arsenic Trioxide in Treating Patients With Myelodysplastic Syndromes

CTI BioPharma

Status and phase: Terminated; Phase 2

Conditions: Leukemia; Myelodysplastic Syndromes; Myelodysplastic/Myeloproliferative Neoplasms

Treatments: Drug: arsenic trioxide

Study type: Interventional

Funder types: Industry; NIH

Identifiers

NCT00020969

NCI-G01-1971

UCLA-HSPC-010104701

CTI-1058

CDR0000068734 (Registry Identifier)

Details and patient eligibility

About

RATIONALE: Drugs used in chemotherapy use different ways to stop cancer cells from dividing so they stop growing or die.

PURPOSE: Phase II trial to study the effectiveness of arsenic trioxide in treating patients who have myelodysplastic syndromes.

Full description

OBJECTIVES:

• Determine the percentage of patients with low-risk myelodysplastic syndromes (MDS) who achieve a major hematologic improvement after treatment with arsenic trioxide.
• Determine the percentage of patients with high-risk MDS who achieve complete or partial remission or major hematological improvement after treatment with this drug.
• Determine the durability of responses in patients treated with this drug.
• Determine the duration of overall and progression-free survival of patients treated with this drug.
• Assess the quality of life of patients treated with this drug.
• Assess the toxicity profile of this drug in these patients.

OUTLINE: This is a multicenter study. Patients are stratified according to risk score (low risk vs high risk).

Patients receive arsenic trioxide IV 5 days a week on weeks 1-2. Treatment repeats every 4 weeks for up to 6 courses in the absence of disease progression or unacceptable toxicity. Patients with stable disease or better may receive additional courses of treatment. Patients who achieve a complete remission (CR) should receive 2 additional courses of treatment after documentation of CR.

Quality of life is assessed at baseline, every 8 weeks during study, and then at 4 weeks after study completion.

Patients are followed at 4 weeks, every 3 months until completion of study treatment, and then annually thereafter.

PROJECTED ACCRUAL: A total of 30-110 patients (15-55 per stratum) will be accrued for this study.

Sex: All

Ages: 18 to 120 years old

Volunteers: No Healthy Volunteers

Inclusion and exclusion criteria

DISEASE CHARACTERISTICS:

• Diagnosis of myelodysplastic syndromes (MDS)

  • Refractory anemia (RA)
  • RA with ringed sideroblasts
  • RA with excess blasts (RAEB)
  • RAEB in transformation
  • Chronic myelomonocytic leukemia

• Low-risk MDS patients:

  • If serum erythropoietin less than 200 IU/mL, must have failed prior recombinant epoetin alfa (EPO) trial

• No prior acute myeloid leukemia

PATIENT CHARACTERISTICS:

Age:

• 18 and over

Performance status:

• Karnofsky 70-100%

Life expectancy:

• More than 3 months

Hematopoietic:

• Not specified

Hepatic:

• Bilirubin no greater than 2.5 times upper limit of normal (ULN)
• SGPT and SGOT no greater than 2.5 times ULN

Renal:

• Creatinine no greater than 1.5 times ULN

Cardiovascular:

• Absolute QT interval less than 460 msec in the presence of magnesium greater than 1.8 mg/dL and potassium greater than 4.0 mEq/L

Other:

• Not pregnant or nursing
• Negative pregnancy test
• Fertile patients must use effective contraception

PRIOR CONCURRENT THERAPY:

Biologic therapy:

• See Disease Characteristics
• At least 30 days since prior biologic therapy (except recombinant EPO in low-risk MDS patients)

Chemotherapy:

• Not specified

Endocrine therapy:

• Not specified

Radiotherapy:

• At least 30 days since prior radiotherapy

Surgery:

• Not specified

Other:

• At least 30 days since prior cytotoxic agents
• At least 30 days since prior investigational agents
• No prior arsenic trioxide