Belinostat in Treating Patients With Myelodysplastic Syndromes
de Novo Myelodysplastic Syndromes
Previously Treated Myelodysplastic Syndromes
Secondary Myelodysplastic Syndromes
|Study Design:||Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
|Official Title:||Phase II Study of the Histone Deacetylase Inhibitor PXD101 for the Treatment of Myelodysplastic Syndrome|
- Number of Confirmed Responses (Complete Response, Partial Response, or Hematologic Improvement) Noted on 2 Consecutive Evaluations at Least 4 Weeks Apart [ Time Frame: 12 weeks ] [ Designated as safety issue: No ]
Complete Response (CR)
A CR is defined as a participant with bone marrow showing less than 5% myeloblasts with no evidence of dysplasia and with adequate peripheral blood counts for at least 2 months (hemoglobin > 11 g/dl, neutrophils ≥ 1500/mm3, platelets ≥ 100,000/mm3) and with no blasts in the peripheral.
Partial Response (PR)
All the CR criteria except bone marrow blasts decreased by ≥ 50% over pretreatment, or a less advanced WHO classification than pretreatment.
Hematologic Improvement (HI)
A 2g/dl increase in hemoglobin for participants with <11g/dl hemoglobin at pretreatment, or an increase of >30,000/mm^3 platelets for participants with <100,000/mm^3 at pretreatment, or a 100% increase in neutrophil counts for participants with <1500/mm^3 at pretreatment
- Time to Progression [ Time Frame: Time from registration to the date of progression or last follow-up, assessed up to 3 years ] [ Designated as safety issue: No ]Estimated using the method of Kaplan-Meier.
- Overall Survival [ Time Frame: From date of registration to the date of last follow-up or death due to any cause, assessed up to 3 years ] [ Designated as safety issue: No ]Estimated using the method of Kaplan-Meier.
- Duration of Response [ Time Frame: From the date of documented response until the date of progression or last follow-up, assessed up to 3 years ] [ Designated as safety issue: No ]Estimated using the method of Kaplan-Meier.
- Toxicity of Belinostat in Patients With Myelodysplastic Syndrome [ Time Frame: Prior to each course (every 21 days), and every 3 months for up to 3 years after completion of study treatment ] [ Designated as safety issue: Yes ]Graded using the Common Terminology Criteria for Adverse Events (CTCAE) version 3.0. Reporting events deemed at least possibly related to study treatment.
|Study Start Date:||May 2006|
|Study Completion Date:||December 2010|
|Primary Completion Date:||December 2010 (Final data collection date for primary outcome measure)|
Experimental: Treatment (enzyme inhibitor therapy)
Patients receive belinostat IV over 30 minutes on days 1-5. Treatment repeats every 21 days for 4 courses in the absence of disease progression or unacceptable toxicity.
Other Name: PXD101
I. Establish the efficacy and safety of PXD101 (belinostat) in patients with myelodysplastic syndromes that progressed after or is ineligible for azacitidine treatment.
II. Assess the biological activity of PXD101 in these patients via assays of histone acetylation, gene expression profiling, and DNA methylation.
OUTLINE: This is a multicenter study.
Patients receive belinostat intravenously (IV) over 30 minutes on days 1-5. Treatment repeats every 21 days for 4 courses in the absence of disease progression or unacceptable toxicity. Patients achieving complete response, partial response, or hematologic improvement after 4 courses receive 4 additional courses of therapy. After completion of study treatment, patients are followed every 3-6 months for up to 3 years.
Please refer to this study by its ClinicalTrials.gov identifier: NCT00357162
|United States, Minnesota|
|Rochester, Minnesota, United States, 55905|
|Principal Investigator:||Amanda Cashen||Mayo Clinic|