Multicenter phase II trial of enzastaurin in patients with relapsed or refractory advanced cutaneous T-cell lymphoma

Abstract

This multicenter, single-arm, open-label non-randomized phase II trial (NCT00744991) was conducted in patients with recurrent/refractory mycosis fungoides (MF), stage IB–IVB, or Sézary syndrome (SS). A Simon two-stage design required 25 patients enrolled in stage 1 with ≥7 confirmed objective responses for expansion into stage 2. Patients were treated with oral enzastaurin (250 mg twice daily) until disease progression or intolerable toxicity. The primary endpoint was investigator-assessed response rate; secondary endpoints were time to objective response, response duration, time-to-progression, patient-reported pruritus, and safety/tolerability. Twenty-five patients were enrolled. A partial response was observed in one patient with MF. Median time-to-progression was 78 and 44 days in MF and SS, respectively. Self-reported pruritus relief and improved composite pruritus-specific symptom scores were documented in six and four patients, respectively. Enzastaurin was well tolerated with mostly grade 1–2 adverse events, mainly diarrhea and fatigue. There were two adverse event-related drug discontinuations with one possibly treatment-related.

Keywords:

• Enzastaurin
• relapsed/refractory cutaneous T-cell lymphoma
• phase II trial
• patient-reported outcome
• pruritus

Acknowledgements

This study was sponsored by Eli Lilly and Company and its subsidiaries. Medical writing support was provided by Lori Kornberg of i3 Statprobe (Ann Arbor, MI), with whom Eli Lilly and Company contracted for technical writing. The authors wish to acknowledge the patients, their families, and the study personnel who participated in this clinical trial.

Potential conflict of interest:

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