A Study of PRN1008 in Adult Patients With Pemphigus Vulgaris

• Status: Completed
• Trial end date: 2020-01-10
• Target enrollment: 0
• Participant gender: All

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.

• Phase: Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Principia Biopharma, a Sanofi Company; Principia Biopharma, Inc.
• Collaborator: Principia Biopharma Australia Pty Ltd.

Criteria

Inclusion Criteria:

• Male or female patients, aged 18 to 80 years old, with biopsy-proven, mild-moderate PV (PDAI 8 to 45) in Part A and mild to severe PV in Part B (PDAI 8 to 60) that are either:

• newly diagnosed patients (i.e. naïve to an effective induction treatment regimen) for whom an initial period of PRN1008 monotherapy is judged clinically acceptable, or

• relapsing patients, for whom an initial period of PRN1008 monotherapy, or combination therapy with any of low dose corticosteroid (≤ 10 mg/day),

Exclusion Criteria:

• Pregnant or lactating women

• A history of malignancy of any type, other than surgically excised non-melanoma skin cancers or in situ cervical cancer within 5 years before the day of dosing

• Use of immunologic response modifiers with the following periods prior to Day 1: 1 week: cyclophosphamide; 4 weeks: intravenous immunoglobulin, Kinaret (anakinra) and Enbrel (etanercept); 12 weeks: Remicade (infliximab), Humira (adalimumab), Simponi (golimumab), Orencia (abatacept), Actemra (tocilizumab), Cimzia (certolizumab), Cosentyx (secukinumab), plasmapheresis; 6 months: Rituxan/MabThera (rituximab), ofatumumab, any other anti-CD20 antibody, other long acting biologics

• Use of >10 mg per day of oral prednisolone per day within 2 weeks prior to Day 1 (inhaled and mucosal [for symptomatic treatment of oral lesions] corticosteroids are allowed)

• Use of proton pump inhibitor drugs such as omeprazole and esomeprazole

• Has received any investigational drug (or is currently using an investigational device) within the 30 days before receiving the first dose of study medication, or at least 5 times the respective elimination half-life time (whichever is longer)

• History of drug abuse within the precious 12 months

• Alcoholism or excessive alcohol use, defined as regular consumption of more than approximately 3 standard drinks per day

• Refractory nausea and vomiting, malabsorption, external biliary shunt, significant bowel resection that would preclude adequate study drug absorption

• History of anorexia nervosa or periods of there months or more of low body weight (BMI<17.5)

• Donation of a unit or more of blood or blood products within 4 weeks prior to Day 1

• History of solid organ transplant

• History of epilepsy or other forms of seizures in the last 5 years

• Positive for screening for human immunodeficiency virus, hepatitis B (surface and core antibodies unrelated to vaccination), or hepatitis C (anti-HCV antibody confirmed with Hep C RNA)

• History of active or latent tuberculosis (TB) infection (must test negative using the QuantiFERON test to be eligible)

• History of serious infections requiring intravenous (by catheter that delivers antibiotics into your blood) treatment

• Live vaccine within 28 days prior to baseline or plan to receive one during the study