Clinical Trial: A Study of PRN1008 in Adult Patients With Pemphigus Vulgaris

Study Status: Recruiting
Recruit Status: Recruiting
Study Type: Interventional

Official Title: 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 Newly Diagnose

Brief Summary: Open-label cohort study in adult patients with newly diagnosed or relapsing pemphigus vulgaris, with intra-patient dose-adjustment based on clinical response and BTK occupancy, and with conventional immunosuppressive "rescue treatment", if indicated. The duration of therapy will be 12 weeks, followed by 12 weeks of follow up.

Detailed Summary:

Primary Objectives:

To evaluate the safety of PRN1008 in patients with pemphigus vulgaris (PV) To evaluate the clinical activity of PRN1008 in patients with PV, per criteria in the European Academy of Dermatology and Venereology (EADV) 2014 Pemphigus S2 Guideline (Hertl et al. 2015)

Secondary Objectives To evaluate the pharmacokinetics (PK) and the pharmacodynamics (PD) of multiple doses of PRN1008 in patients with PV To evaluate the relationship of PK and PD to each other and to efficacy and safety in this patient population


Sponsor: Principia Biopharma, Inc.

Current Primary Outcome:

  • Incidence of treatment-emergent adverse events at 12 weeks [ Time Frame: 12 weeks treatment ]
    The incidence of treatment-emergent adverse events (TEAEs), including clinically significant changes in physical examination, laboratory tests, and vital signs.
  • Control of disease activity at 4 weeks [ Time Frame: 4 weeks treatment ]
    The proportion of subjects who are able to achieve control of disease activity (CDA) within 4 weeks of starting PRN1008 treatment without the need for doses of prednisone or prednisolone >0.5 mg/kg.


Original Primary Outcome: Same as current

Current Secondary Outcome:

  • Time to control of disease activity (CDA) [ Time Frame: 12 week treatment; 12 week follow up ]
    Time to control of disease activity (CDA) as defined in European Academy of Dermatology and Venereology (EADV) 2015 Pemphigus S2 Guideline
  • Time to end of consolidation phase [ Time Frame: 12 week treatment; 12 week follow up ]
    Time to end of consolidation phase as defined in European Academy of Dermatology and Venereology (EADV) 2015 Pemphigus S2 Guideline
  • Time to complete remission [ Time Frame: 12 week treatment; 12 week follow up ]
    Time to complete remission as defined in European Academy of Dermatology2015 Pemphigus S2 Guideline
  • Time to relapse after PRN1008 treatment discontinuation [ Time Frame: 12 week treatment; 12 week follow up ]
    Time to relapse after PRN1008 treatment discontinuation as defined in European Academy of Dermatology and Venereology (EADV) 2015 Pemphigus S2 Guideline


Original Secondary Outcome: Same as current

Information By: Principia Biopharma, Inc.

Dates:
Date Received: February 24, 2016
Date Started: February 2016
Date Completion: December 2017
Last Updated: February 28, 2017
Last Verified: February 2016