Phase II study of oral panobinostat (LBH589), a potent pan-deacetylase inhibitor, in patients with refractory Cutaneous T-cell Lymphoma (CTCL)

8555 Background: Panobinostat (LBH589) is a potent pan-deacetylase inhibitor (DACi) which has demonstrated activity in patients (pts) with CTCL in a phase I study (Prince et al, ASCO 2007). This phase II study aims to confirm that activity. Methods: This open-label study enrolled pts with CTCL (stage IB-IVA), including Mycosis fungoides (MF) and Sezary syndrome (SS), who have failed ≥2 prior systemic therapies, have adequate organ function and ECOG PS ≤2. Pts with significant cardiovascular abnormalities or QTcF >450 msec on screening ECG were excluded. Pts were accrued to Group 1 (Grp 1, previously treated with oral bexarotene) or Group 2 (Grp 2, bexarotene naive); both following a Simon 2-stage design. Panobinostat (20 mg) was administered orally on days 1, 3, and 5 weekly until disease progression or intolerance. Primary endpoint is the response rate measured using a composite score derived from assessment of skin disease using modified Severity-Weighted Assessment Tool [mSWAT], evaluation of visceral ...