Clinical trial NCT00020969: Arsenic Trioxide in Treating Patients With Myelodysplastic Syndromes
Official title: Phase Two Multicenter Study Of Arsenic Trioxide In Patients With Myelodysplastic Syndromes
Study type: INTERVENTIONAL
Overall status: TERMINATED
Phase: PHASE2
Conditions: Leukemia
Myelodysplastic Syndromes
Myelodysplastic/Myeloproliferative Neoplasms
Keywords: refractory anemia
refractory anemia with ringed sideroblasts
refractory anemia with excess blasts
refractory anemia with excess blasts in transformation
chronic myelomonocytic leukemia
de novo myelodysplastic syndromes
previously treated myelodysplastic syndromes
secondary myelodysplastic syndromes
atypical chronic myeloid leukemia, BCR-ABL1 negative
myelodysplastic/myeloproliferative neoplasm, unclassifiable
Lead sponsor: CTI BioPharma
Sponsor class: INDUSTRY
Collaborators: name: National Cancer Institute (NCI)
First posted: 2003-01-27
Sex: ALL
Minimum age (years): 18
Maximum age (years): 120
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Primary purpose: TREATMENT

Brief summary: 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.

Detailed 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.

Eligibility 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

Primary outcomes:
Secondary outcomes:
Other outcomes:
Locations:
city: Tucson
country: United States
facility: Arizona Cancer Center at University of Arizona Health Sciences Center
state: Arizona
city: La Jolla
country: United States
facility: Green Cancer Center at Scripps Clinic
state: California
city: Los Angeles
country: United States
facility: USC/Norris Comprehensive Cancer Center and Hospital
state: California
city: Los Angeles
country: United States
facility: Jonsson Comprehensive Cancer Center, UCLA
state: California
city: Orange
country: United States
facility: St. Joseph Hospital Regional Cancer Center - Orange
state: California
city: Boca Raton
country: United States
facility: Lynn Regional Cancer Center West
state: Florida
city: Atlanta
country: United States
facility: Georgia Cancer Specialists - Northside Office
state: Georgia
city: Corpus Christi
country: United States
facility: Corpus Christi Cancer Center
state: Texas
Study officials:
affiliation: CTI BioPharma
name: Scott C. Stromatt, MD
role: STUDY_CHAIR
References:
citation: Schiller GJ, Slack J, Hainsworth JD, Mason J, Saleh M, Rizzieri D, Douer D, List AF. Phase II multicenter study of arsenic trioxide in patients with myelodysplastic syndromes. J Clin Oncol. 2006 Jun 1;24(16):2456-64. doi: 10.1200/JCO.2005.03.7903. Epub 2006 May 1.