LBH589 is an oral drug that targets the myelofibrosis cells in the bone marrow and induces cell death by allowing for the expression of certain suppressed genes that are important in regulating cell survival. Based on laboratory studies, the hypothesis is that this drug will selectively kill the stem cells responsible for causing myelofibrosis and result in reduction in spleen size and ultimately restoration of normal bone marrow function.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
22
Dose escalation study starting at 20mg by mouth three times a week, given weekly for 24 weeks in the phase I portion of the study.
Icahn School of Medicine at Mount Sinai
New York, New York, United States
To assess the safety and tolerability of oral LBH589 in patients with PMF, post-PV/ET MF
Phase I
Time frame: 28 days
Evaluation of treatment response by International Working Group for Myelofibrosis Research and Treatment (IWG-MRT)
Phase II
Time frame: 6 months
Assess changes in biomarkers
JAK2V617F allele burden H3/H4 acetylation status peripheral blood CD34+ stem cell burden CXCR4 expression on CD34+ peripheral blood stem cells
Time frame: 6 months
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