A Study of PRN1008 in Patients With Pemphigus

• Status: Active, not recruiting
• Trial end date: 2021-12-08
• Target enrollment: 0
• Participant gender: All

Summary

This is a Phase 3 randomized, parallel-group, double-blind, placebo-controlled trial (Blinded Treatment Period) followed by an Open-Label Extension Period intended to evaluate the efficacy and safety of oral PRN1008 in moderate to severe pemphigus. After completing the Open-Label Extension Period, eligible patients may continue in a Long Term Extension Period of 48 weeks.

• Phase: Phase 3
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Principia Biopharma, a Sanofi Company; Principia Biopharma, Inc.

Criteria

Inclusion Criteria:

• Male or female participants, aged 18 to 80 years old with moderate to severe, newly diagnosed or relapsing PV (pemphigus vulgaris) or PF (pemphigus foliaceus), with a clinical presentation and histopathology consistent with PV or PF.

• Positive circulating anti-dsg1 or 3 autoantibody titer.

• At Screening, Pemphigus disease area index (PDAI) score of at least 9 points for relapsing participants or at least 15 points for newly diagnosed participants.

• Adequate hematologic, hepatic, and renal function.

• Effective means of contraception.

Exclusion Criteria:

• Suspected paraneoplastic pemphigus and other forms of pemphigus that are not pemphigus vulgaris or pemphigus foliaceus.

• Previous use of a Bruton tyrosine kinase (BTK) inhibitor.

• Pregnant or lactating women.

• Electrocardiogram (ECG) clinically significant abnormalities.

• A history of malignancy of any type within 5 years before Day 1, other than surgically excised non-melanoma skin cancers or in situ cervical cancer.

• Use of immunologic response modifiers as concomitant medication and with the washout period.

• Use of proton pump inhibitor drugs such as omeprazole and esomeprazole within 3 days of Day 1.

• Concomitant use of known strong-to-moderate inducers or inhibitors of CYP3A within 3 days or 5 half-lives (whichever is longer) of Day 1

• Use of CYP3A-sensitive substrate drugs.

• Has received any investigational drug within the 30 days before Day 1.

• History of drug abuse within the previous 12 months.

• Alcoholism or excessive alcohol use.

• Any other clinically significant disease, condition or medical history that, in the opinion of the Investigator, would interfere with participant safety, trial evaluations, and/or trial procedures.