A Study of PRN1008 in Adult Patients With Pemphigus Vulgaris

• Register: ClinicalTrials.gov
• Last refreshed on: 11 January 2021
• Main ID: NCT02704429
• Date of registration: 24/02/2016
• Prospective Registration: No
• Primary sponsor: Principia Biopharma Inc.
• Public title: A Study of PRN1008 in Adult Patients With Pemphigus Vulgaris
• Scientific 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 Diagnosed or Relapsing Pemphigus Vulgaris
• Date of first enrolment: January 22, 2016
• Target sample size: 42
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT02704429
• Study type: Interventional
• Study design: Allocation: N/A. Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).
• Phase: Phase 2

Countries of recruitment

Australia, Croatia, France, Greece, Israel

Contacts

• Name: Dolca Thomas, MD
• Affiliation: Principia Biopharma

Key inclusion & exclusion 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



Age minimum: 18 Years
Age maximum: 80 Years
Gender: All

Health Condition(s) or Problem(s) studied

Pemphigus Vulgaris

Intervention(s)

Drug: PRN1008

Primary Outcome(s)

Control of disease activity at 4 weeks [Time Frame: 4 weeks treatment]

Incidence of treatment-emergent adverse events [Time Frame: Part A: 24 week treatment]

Secondary Outcome(s)

Time to end of consolidation phase [Time Frame: Part A: 12 week treatment; 12 week follow up and Part B: 24 week treatment, 4 week follow-up]

Time to control of disease activity (CDA) [Time Frame: Part A: 12 week treatment; 12 week follow up and Part B: 24 week treatment, 4 week follow-up]

Time to complete response [Time Frame: Part A: 12 week treatment; 12 week follow up and Part B: 24 week treatment, 4 week follow-up]

Cumulative corticosteroid usage [Time Frame: Part A: 12 week treatment; 12 week follow up and Part B: 24 week treatment, 4 week follow-up]

Time to relapse after PRN1008 treatment discontinuation [Time Frame: Part A: 12 week follow up and Part B: 4 week follow-up]

Secondary ID(s)

PRN1008-005

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Secondary Sponsor(s)

Principia Biopharma Australia Pty Ltd.