QBR106641

• Research type

  Research Study

• Full title

  A Phase 1, Open label, Single Dose, Sequential, 2-Part Study to Investigate the Absolute Bioavailability and to Evaluate Novel Controlled release Formulations of VX-765 in Healthy Adult Subjects

• IRAS ID

  65846

• Contact name

  Sharan Sidhu

• Sponsor organisation

  Vertex Pharmaceuticals Incorporated

• Eudract number

  2010-023276-99

• Research summary

  There is increasing evidence that inflammation plays an important role in establishing and maintaining seizures, though to date, there are currently no anti inflammatory drugs in epilepsy clinical trials. Surgical specimens of human brain from patients with treatment refractory epilepsy show a number of inflammatory features, including elevated interleukin converting enzyme (ICE, also known as caspase 1), elevated interleukin 1ß (IL 1ß), and elevated IL 1ß receptors. These human brain studies suggested that ICE and the IL 1ß system may be involved in causing epilepsy, particularly epilepsy that is resistant to current treatments as these tissue samples were from patients with treatment-refractory epilepsy. Several nonclinical experiments support an important role for ICE/IL 1ß systems in epilepsy and indicate that inhibition of ICE/IL 1ß systems has therapeutic potential for treating epilepsy. Previous nonclinical studies strongly implicate the ICE/IL 1ß system in seizure pathophysiology and suggest that inhibiting ICE/IL 1ß might be an important avenue for treating epilepsy in humans. This is a Phase 1, open-label, single dose, sequential, 2 part study in healthy adult subjects. This study is designed to evaluate novel controlled release formulations and an immediate release core granule formulation of VX-765 and to investigate the absolute bioavailability of VX-765. The contract research organization, on behalf of Vertex, will use a RapidFACT service approach with ‘Design Space’ concepts to develop and evaluate clinical prototype modified release VX-765 multiparticulate formulations.

• REC name

  North West - Greater Manchester Central Research Ethics Committee

• REC reference

  10/IEC01/26

• Date of REC Opinion

  5 Jan 2011

• REC opinion

  Further Information Favourable Opinion