A Study of PRN1008 in Patients With Pemphigus
Condition: Pemphigus
MeSH terms: Pemphigus
Intervention: Rilzabrutinib; Placebo
Intervention description: Pharmaceutical form: Tablet Route of administration: Oral; Pharmaceutical form: Tablet Route of administration: Oral
Phase: Phase 3
Study type: Interventional
Primary purpose: Treatment
Allocation: Randomized
Intervention model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Enrollment: 131.0
Sex: All
Minimum age: 18 Years
Maximum age: 80 Years
Healthy volunteers: No
Keywords: Pemphigus Vulgaris (PV); Pemphigus Foliaceus (PF)

Summary:

      This was a Phase 3 randomized, parallel-group, double-blind, placebo-controlled trial
      (blinded treatment [BT] period) followed by an open-label extension [OLE] period intended to
      evaluate the efficacy and safety of oral PRN1008 in moderate to severe pemphigus. After
      completing the open-label extension period, eligible participants might continue in a long
      term extension (LTE) Period of 48 weeks.
    

Eligibility criteria:

        Inclusion Criteria:

          -  Male or female participants, aged 18 to 80 years old with moderate to severe, newly
             diagnosed or relapsing PV or PF, with a clinical presentation and histopathology
             consistent with PV or PF.

          -  Positive circulating anti-desmoglein 1 (anti-dsg1) or 3 autoantibody titer.

          -  At screening, pemphigus disease area index 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 were not PV or
             PF.

          -  Previous use of a Bruton tyrosine kinase inhibitor.

          -  Pregnant or lactating women.

          -  Electrocardiogram 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 cytochrome P450
             3A (CYP3A) within 3 days or 5 half-lives (whichever is longer) of Day 1

          -  Use of CYP3A-sensitive substrate drugs.

          -  Had 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.