Sorafenib in Myelodysplastic Syndrome
The purpose of this study is to evaluate the efficacy of sorafenib in patients with Myelodysplastic Syndrome (MDS). Eligible subjects will receive Sorafenib administered at 400mg orally twice a day, given on days 1-28 of a 28-day cycle. Patients will be evaluated for hematological response after 2 cycles and then every 3 cycles thereafter for a maximum of 5 years from study entry. If a patient achieves a complete response they may receive an additional 6 cycles of therapy beyond documentation of complete response unless unacceptable toxicity occurs. For patients with partial response, hematological improvement or stable disease they will continue treatment until relapse, progression of disease, or unacceptable toxicity occurs.
Leukemia, Myelomonocytic, Chronic
|Study Design:||Endpoint Classification: Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
|Official Title:||Phase II Trial of Sorafenib in Patients With Myelodysplastic Syndrome|
- Number of Subjects Achieving Hematological Response [ Time Frame: During treatment - up to a maximum of 5 years ] [ Designated as safety issue: No ]Hematological response is defined as the number of subjects who achieve either a complete response (CR), Partial response (PR) or Hematologic improvement.(HI). HI is defined as peripheral blood counts with hemoglobin ≥11 g/dL, absolute neutrophil count ≥1x10(9)/L and platelet count ≥100x10(9)/L, and normal bone marrow morphology with no evidence of dysplasia or blasts. CR is defined as the disappearance of all signs and symptoms related to disease, along with HI. PR is defined as fulfilling the criteria for CR in the peripheral blood but blasts decreasing by 50% or more in the bone marrow or to a less advanced WHO classification pretreatment.
- Number of Subjects Requiring Dose Reductions [ Time Frame: While on study drug, a maximum of 5 years ] [ Designated as safety issue: Yes ]The number of subjects who took study drug for more than 1 cycle and required a dose reduction down to the next dose level.
- Time to Progression [ Time Frame: 5 years ] [ Designated as safety issue: No ]Time to progression will be defined as the number of months between on-study and the date of progression or death, whichever comes first, in subjects who took study drug for at least cycle 1.
- Overall Survival [ Time Frame: 1 year from the last dose of study drug ] [ Designated as safety issue: No ]Overall Survival is defined as the number of months from enrollment onto the study until death from any cause in subjects who took study drug for at least cycle 1.
- Change in Microvessel Density [ Time Frame: Measured before and after treatment ] [ Designated as safety issue: No ]Microvessel density will be measured before and after treatment, and the distribution of change across time will be summarized with descriptive statistics.
|Study Start Date:||July 2006|
|Study Completion Date:||July 2011|
|Primary Completion Date:||July 2011 (Final data collection date for primary outcome measure)|
Experimental: all patients
400 mg twice a day until progression or unacceptable toxicity develops.
Other Name: Nexavar
|United States, North Carolina|
|Duke University Medical Center|
|Durham, North Carolina, United States, 27710|
|Principal Investigator:||David A Rizzieri, MD||Duke University|