Vatalanib in Treating Patients With Primary or Secondary Myelodysplastic Syndromes
Recruitment status was Active, not recruiting
RATIONALE: Vatalanib may be effective in preventing the development of leukemia in patients who have myelodysplastic syndromes.
PURPOSE: This phase II trial is studying vatalanib to see how well it works in treating patients with primary or secondary myelodysplastic syndromes.
|Study Design:||Allocation: Non-Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
|Official Title:||A Phase II Study of an Oral VEGF Receptor Tyrosine Kinase Inhibitor (PTK787/ZK222584) (IND #66370, NSC #719335) in Myelodysplastic Syndrome (MDS)|
- Hematologic Response rate [ Time Frame: 5 yrs ] [ Designated as safety issue: No ]measured by International Standardized Response Criteria for myelodysplastic syndromes (MDS)
- Safety [ Time Frame: Monthly ] [ Designated as safety issue: Yes ]Measured by Common Terminology Criteria for Adverse Events (CTCAE)
- Duration of response [ Time Frame: 5 yrs ] [ Designated as safety issue: No ]Measured by International Standardized Response Criteria for MDS
- Overall survival [ Time Frame: 5 yrs ] [ Designated as safety issue: No ]
- Progression-free survival [ Time Frame: 1 yr ] [ Designated as safety issue: No ]Measured by International Standardized Response Criteria for MDS
|Study Start Date:||December 2003|
|Estimated Study Completion Date:||December 2013|
|Primary Completion Date:||November 2006 (Final data collection date for primary outcome measure)|
Adult patients with MDS receive treatment with vatalanib
Pts registered before 1/15/05 1250 mg/day PO After 1/15/05: Start w/ 750 mg/day PO; escalate q 4 wks in absence of Grade 2 or > tox (1st increase=1000mg/day; 2nd increase 1250 mg/day)
Other Name: PTK787/ZK 222584
- Determine the response rate, in terms of hematologic improvement and complete and partial remission, in patients with primary or secondary (therapy-related) myelodysplastic syndromes treated with vatalanib.
- Determine the time to transformation to acute myeloid leukemia (at least 20% blasts) or death in patients treated with this drug.
- Determine the safety of this drug in these patients.
- Determine the duration of response in patients treated with this drug.
- Determine the cytogenetic response rate in patients treated with this drug.
- Determine the overall and progression-free survival of patients treated with this drug.
- Determine the incidence of infections requiring antibiotics or hospitalization or bleeding requiring red blood cell transfusions in patients treated with this drug.
OUTLINE: This is a multicenter study. Patients are stratified* according to risk group (low grade [refractory anemia with or without ringed sideroblasts, refractory anemia with excess blasts-1, refractory cytopenia with multilineage dysplasia with or without ringed sideroblasts, myelodysplastic syndromes-unclassified, or chronic myelomonocytic leukemia-1] vs high grade [refractory anemia with excess blasts-2 or chronic myelomonocytic leukemia-2]).
NOTE: *Stratification according to risk (low vs high) does not occur after 11/30/06.
Patients receive oral vatalanib once daily on days 1-28. Courses repeat every 28 days in the absence of disease progression or unacceptable toxicity. Patients with a complete response (CR) receive 6 additional courses after documentation of a CR.
Patients are followed periodically for up to 5 years from study entry.
PROJECTED ACCRUAL: Approximately 144 patients will be accrued for this study within 2.5 years.