Conditions: Leukemia Myelodysplastic Syndromes Myelodysplastic/Myeloproliferative Neoplasms
Keywords: refractory anemia with excess blasts refractory anemia with ringed sideroblasts refractory cytopenia with multilineage dysplasia chronic myelomonocytic leukemia de novo myelodysplastic syndromes secondary myelodysplastic syndromes myelodysplastic/myeloproliferative neoplasm, unclassifiable refractory anemia previously treated myelodysplastic syndromes
Allocation: NA | Intervention model: SINGLE_GROUP
Enrollment: 155
Healthy volunteers: no

Brief summary:
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.

Detailed description:
OBJECTIVES:

Primary

* 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.

Secondary

* 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.

Arms:
arm 1: Adult patients with MDS receive treatment with vatalanib.

Interventions: intervention 1: vatalanib
Intervention descriptions: intervention 1: 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)

Protocol document: