Phase 2
N=27
A Study of PRN1008 in Adult Patients With Pemphigus Vulgaris
Pemphigus Vulgaris
Bottom Line
View on ClinicalTrials.gov: NCT02704429 ↗Enrolled (actual)
27
Serious AEs
7.1%
Results posted
Feb 2023
Primary outcome: Primary: Percentage of Participants With Treatment-emergent Adverse Events — 74.1; 86.7; 11.1; 0 percentage of participants
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 2
- Interventions
- PRN1008 (Drug)
- Age
- Adult, Older Adult · 18+ yrs
- Sex
- All
- Sponsor
- Principia Biopharma, a Sanofi Company
- Primary completion
- Dec 2019
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Percentage of Participants With Treatment-emergent Adverse Events |
74.1; 86.7; 11.1; 0; 11.1; 0 | — |
| PRIMARY Percentage of Participants Who Are Able to Achieve Control of Disease Activity (CDA) Within 4 Weeks of Starting PRN1008 Treatment Without the Need for Doses of Prednisone or Prednisolone >0.5 mg/kg |
51.9; 60.0 | — |
| SECONDARY Percentage of Participants Able to Achieve Control of Disease Activity (CDA) Without Corticosteroids Within 4 Weeks |
11.1; 6.7 | — |
| SECONDARY Percentage of Participants Able to Achieve a Complete Response (CR) Without Corticosteroids |
0; 0 | — |
| SECONDARY Percentage of Participants Able to Achieve Complete Remission (CR) Without the Need for Doses of Prednisone or Prednisolone of Greater Than 0.5mg/kg |
14.8; 33.3 | — |
| SECONDARY Time to Control of Disease Activity (CDA) |
33.0; 29.0 | — |
| SECONDARY Time to End of Consolidation Phase (ECP) |
170.0; 58.0 | — |
| SECONDARY Time to Complete Remission (CR) |
NA; NA | — |
| SECONDARY Time to Relapse After PRN1008 Treatment Discontinuation |
96.0; 198.0 | — |
| SECONDARY Cumulative Corticosteroid Usage |
983.43; 2089.600 | — |
| SECONDARY Percentage Change From Baseline in Pemphigus Disease Area Index (PDAI) Total Activity Scores |
-55.7; -78.60; -57.7; -59.68 | — |
| SECONDARY Change From Baseline in Autoimmune Bullous Skin Disorder Intensity Score (ABSIS) Total Activity Score |
-8.18; -6.591; -9.98; -5.705 | — |
| SECONDARY Change From Baseline in Autoimmune Bullous Diseases Quality of Life (ABQOL) |
-3.7; -6.36; -2.9; -3.79 | — |
| SECONDARY Change From Baseline in Treatment of Autoimmune Bullous Diseases Quality of Life (TABQOL) Scores |
-0.0; -2.00; -0.1; -2.14 | — |
| SECONDARY Change From Baseline in Appetite (SNAQ Score) |
1.1; 0.21; 1.0; 0.50 | — |
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.
Eligibility 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, ie.0.5 mg/kg of prednis(ol)one per 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
- More than 0.5 mg/kg of prednis(ol)one per day ("low dose corticosteroids") within the two weeks prior to Day 1
- 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 three months or more of low body weight in the past 5 years
- 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
Data sourced from ClinicalTrials.gov (NCT02704429). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.