NCT02704429

A Study of PRN1008 in Adult Patients With Pemphigus Vulgaris

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 Diagnosed or Relapsing Pemphigus Vulgaris

• Status: Completed
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 42 (actual)
• Sponsor: Principia Biopharma, a Sanofi Company · Industry
• Sex: All
• Age: 18 Years – 80 Years
• Healthy volunteers: Not accepted

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 in Part A will be 12 weeks, followed by 12 weeks of follow up. The extension phase, Part B includes 24 weeks of therapy, followed by 4 weeks of follow-up.

Detailed description

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

Conditions

• Pemphigus Vulgaris

Interventions

Type	Name	Description
DRUG	PRN1008	Part A dosing was initiated administering 400 mg BID. Intrapatient dose adjustments (reductions and increases) were permitted based upon tolerability and clinical response with the maximum dose allowed up to 600 mg BID for 12 weeks. Part B initial dosing was 400 mg QD for 2 weeks with dose escalation to 400 mg BID at the discretion of the Investigator for the purposes of investigating dose response and identifying the minimal efficacious dose of rilzabrutinib for 24 weeks.

Timeline

• Start date: 2016-01-22
• Primary completion: 2019-12-18
• Completion: 2020-01-10
• First posted: 2016-03-10
• Last updated: 2023-02-13
• Results posted: 2023-02-13

Locations

13 sites across 5 countries: Australia, Croatia, France, Greece, Israel

Regulatory

• FDA-regulated drug study