Clinical Trial: Pilot Study of Apremilast (CC-10004) in the Treatment of Moderate to Severe Lichen Planus

Study Status: Completed
Recruit Status: Unknown status
Study Type: Interventional

Official Title: An Open-Label Pilot Study to Evaluate the Safety and Efficacy of Apremilast (CC-10004) in the Treatment of Moderate to Severe Lichen Planus

Brief Summary: This study is designed to demonstrate to efficacy and safety of Apremilast 20mg oral administration twice daily over 12 weeks in subjects with moderate to severe lichen planus. The hypothesis is that the subjects will achieve a significant clinical improvement in their skin disease according to a specialized physician grading scale.

Detailed Summary:

This is a phase 2, single center, non-randomized, open label efficacy and safety study designed to characterize the response of Apremilast 20 mg oral administered twice daily over 12 weeks in subjects with moderate to severe lichen planus. The hypothesis and ideal primary end point will be that subjects achieve significant clinical response in cutaneous disease defined as a 2 or more grade improvement of the physician global assessment (PGA) after 12 weeks of treatment.

Many various therapies have been used to treat LP including topical and oral corticosteroids, retinoids, cyclosporine, griseofulvin, dapsone and phototherapy, but often with disappointing response.4 It is an inflammatory condition whose pathogenesis involves damage to basal keratinocytes by alloreactive T cells through the release proinflammatory cytokines, such as TNF-α and IFN-γ.1 Significantly elevated levels of such inflammatory mediators are present in tissue from LP lesions compared to normal controls.5 Based on these observations, the investigation of Apremilast, due to its ability to inhibit multiple inflammatory cytokines, for the treatment of moderate to severe LP is warranted.

The primary objective of this study is to evaluate the clinical efficacy of Apremilast in subjects with moderate to severe lichen planus after 12 weeks of treatment. Other objectives are to evaluate the safety and tolerability of Apremilast, effects on quality of life, and efficacy for mucosal disease if present.


Sponsor: Virginia Clinical Research, Inc.

Current Primary Outcome: Proportion of subjects achieving significant clinical response in cutaneous disease defined as a 2 or more grade improvement of the physician global assessment (PGA) after 12 weeks of treatment. [ Time Frame: 12 weeks ]

Original Primary Outcome: Same as current

Current Secondary Outcome:

  • Proportion of subjects with mucosal involvement who achieve a significant clinical response in mucosal disease defined as physician global assessment of mucosal disease (PGAMD) of "complete resolution" or "marked improvement" after 12 weeks of treatment. [ Time Frame: 12 weeks ]
  • Change in the subjects' target area lesion count after 12 weeks of treatment relative to baseline. [ Time Frame: 12 weeks ]
  • Change in the subjects' target area lesion severity score (TALSS) after 12 weeks of treatment relative to baseline. [ Time Frame: 12 weeks ]
  • Proportion of subjects who achieve a subject global assessment of "complete resolution" or "marked improvement" after 12 weeks of treatment. [ Time Frame: 12 weeks ]
  • Change in the subjects' dermatology life quality index (DLQI) score after 12 weeks of treatment relative to baseline. [ Time Frame: 12 weeks ]
  • Change in the subjects' assessment of itching on a visual analogue scale (VAS) after 12 weeks of treatment relative to baseline. [ Time Frame: 12 weeks ]
  • Safety and tolerability of Apremilast (type, frequency, severity, and relationship of adverse events to study treatment) [ Time Frame: 16 weeks total (12 weeks treatment, 4 weeks observation) ]


Original Secondary Outcome: Same as current

Information By: Virginia Clinical Research, Inc.

Dates:
Date Received: December 30, 2009
Date Started: February 2010
Date Completion: February 2012
Last Updated: December 31, 2009
Last Verified: December 2009