RECRUITING NOW (Victorian residents only)

An Open Label, Phase 2, Pilot Study Investigating the Safety, Clinical Activity, Pharmacokinetics and Pharmacodynamics of Oral Treatment with the BTK Inhibitor: PRN1008 in Patients with Pemphigus Vulgaris

Pemphigus vulgaris is a rare autoimmune blistering disease of the skin and mucous membranes.  Untreated the mortality is 100%.  Current treatments including high dose systemic glucocorticosteroids (often administered concomitantly with other immunosuppressive treatments) reduce the mortality to less than 20% but cause significant morbidity.

PRN 1008 is an oral small molecule drug with the specificity of an antibody.  PRN 1008 inhibits BTK (Bruton’s Tyrosine Kinase) signalling, which has a broad role in multiple inflammatory pathways, and specifically impacts on B cell function and autoantibody signalling inhibition without severe immunosuppression.

The aim of this study is to determine the efficacy and safety of PRN 1008 in subjects with pemphigus vulgaris.  During the study, patients may remain on, or be tapered off, low dose prednisolone, and steroid sparing immunosuppressive agents will be ceased early in the treatment period, assuming good clinical response.

We are seeking male and female subjects aged 18 through 80 years of age who have been diagnosed with pemphigus vulgaris.

There are 11 clinic visits over 26 weeks.  There is no placebo in this study.

Participants, including country and interstate patients are eligible to receive reimbursement for travel expenses.

If you are interested and would like to participate in this trial, please click on the following link to answer a few short questions so one of our co-ordinators can get back to you: eligibility questionnaire