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A Study of PRN1008 in Adult Patients With Pemphigus Vulgaris

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ClinicalTrials.gov Identifier: NCT02704429
Recruitment Status : Recruiting
First Posted : March 10, 2016
Last Update Posted : December 7, 2017
Sponsor:
Collaborator:
Information provided by (Responsible Party):

Study Description
Brief 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 will be 12 weeks, followed by 12 weeks of follow up.

Condition or disease Intervention/treatment Phase
Pemphigus Vulgaris Drug: PRN1008 Phase 2

Detailed Description:

Primary Objectives:

To evaluate the safety of PRN1008 in patients with pemphigus vulgaris (PV) To evaluate the clinical activity of PRN1008 in patients with PV, per criteria in the European Academy of Dermatology and Venereology (EADV) 2014 Pemphigus S2 Guideline (Hertl et al. 2015)

Secondary Objectives To evaluate the pharmacokinetics (PK) and the pharmacodynamics (PD) of multiple doses of PRN1008 in patients with PV To evaluate the relationship of PK and PD to each other and to efficacy and safety in this patient population


Study Design

Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 25 participants
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Masking Description: Open Label
Primary Purpose: Treatment
Official 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
Study Start Date : February 2016
Estimated Primary Completion Date : December 2019
Estimated Study Completion Date : December 2019

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Pemphigus
U.S. FDA Resources

Arms and Interventions

Arm Intervention/treatment
Experimental: PRN1008
Open-label PRN1008, 12 weeks; 12 week follow up
Drug: PRN1008
PRN1008, oral dose, 12 weeks
Other Name: BTK inhibitor


Outcome Measures

Primary Outcome Measures :
  1. Incidence of treatment-emergent adverse events at 12 weeks [ Time Frame: 12 weeks treatment ]
    The incidence of treatment-emergent adverse events (TEAEs), including clinically significant changes in physical examination, laboratory tests, and vital signs.

  2. Control of disease activity at 4 weeks [ Time Frame: 4 weeks treatment ]
    The proportion of subjects 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.


Secondary Outcome Measures :
  1. Time to control of disease activity (CDA) [ Time Frame: 12 week treatment; 12 week follow up ]
    Time to control of disease activity (CDA) as defined in European Academy of Dermatology and Venereology (EADV) 2015 Pemphigus S2 Guideline

  2. Time to end of consolidation phase [ Time Frame: 12 week treatment; 12 week follow up ]
    Time to end of consolidation phase as defined in European Academy of Dermatology and Venereology (EADV) 2015 Pemphigus S2 Guideline

  3. Time to complete remission [ Time Frame: 12 week treatment; 12 week follow up ]
    Time to complete remission as defined in European Academy of Dermatology2015 Pemphigus S2 Guideline

  4. Time to relapse after PRN1008 treatment discontinuation [ Time Frame: 12 week treatment; 12 week follow up ]
    Time to relapse after PRN1008 treatment discontinuation as defined in European Academy of Dermatology and Venereology (EADV) 2015 Pemphigus S2 Guideline


Eligibility Criteria

Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years to 80 Years   (Adult, Senior)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Male or female patients, aged 18 to 80 years old, with biopsy-proven, mild-moderate PV (PDAI 8 to 45) 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), azathioprine, mycophenolate mofetil, sulfasalazine and dapsone, is judged clinically acceptable, provided cessation of azathioprine, mycophenolate mofetil, sulfasalazine, and dapsone within two to four weeks is anticipated

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: Kinaret (anakinra) and Enbrel (etanercept); 12 weeks: Remicade (infliximab), Humira (adalimumab), Simponi (golimumab), Orencia (abatacept), Actemra (tocilizumab), Cimzia (certolizumab), Cosentyx (secukinumab), intravenous immunoglobulin, plasmapheresis; 6 months: Rituxan/MabThera (rituximab), ofatumumab, any other anti-CD20 antibody
  • Use of >10 mg per day of oral prednisolone for more than 1 week within 4 weeks prior to Day 1 (inhaled and mucosal [for symptomatic treatment of oral lesions] corticosteroids are allowed)
  • 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)
  • Donation of a unit or more of blood or blood products within 4 weeks prior to Day 1
  • History of solid organ transplant
  • Positive for screening for HIV, 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 (subjects must also test negative using the QuantiFERON® test to be eligible for the study)
Contacts and Locations

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02704429


Contacts
Contact: Steven G Gourlay, MBBS PhD 650-416-7700
Contact: Robert C Quesenberry, BS PMP CRP 650-416-7700

Locations
Australia, New South Wales
Premier Specialists Recruiting
Kogarah, New South Wales, Australia, 2217
Contact: Charmaine Peras, RN    +61295985800    murrell.patients@ori.org.au   
Principal Investigator: Dedee F. Murrell, BMBCh FRCP         
Australia, Victoria
Sinclair Dermatology Recruiting
East Melbourne, Victoria, Australia, 3002
Contact: Carol Robinson    +61 3 9654 2426    Carol.Robinson@sinclairdermatology.com.au   
Principal Investigator: Rodney D Sinclair, MBBS MD FACD         
Royal Melbourne, Dermatology Office Recruiting
Melbourne, Victoria, Australia, 3050
Contact: Annette Phemister, RN    +61 3 9342 4531    Annette.phemister@mh.org.au   
Principal Investigator: George A Varigos, MBBS PhD         
Croatia
Clinical Hospital Osijek Recruiting
Osijek, Croatia, 31000
Contact: Jasmina Knezevic    +385915460138    knezevic.jasmina@kbo.hr   
Principal Investigator: Marija Sola, MD         
Klinichki Bolnicki Centar Zagreb Recruiting
Zagreb, Croatia, 10 000
Contact: Tanja Vrcic, MD    +38516051935    tanja.vrcic@gmail.com   
Principal Investigator: Branka Marinovic, MD, PhD         
France
Hôpital Avicenne Recruiting
Bobigny, Siene-Saint Denis, France, 93009
Contact: Frederic Caux    +33148955189      
Principal Investigator: Frederic Caux         
Rouen University Hospital Recruiting
Rouen, France, 76038
Contact: Samir Stiti    +33243434351    samir.stiti@chu-rouen.fr   
Principal Investigator: Pascal Joly, MD         
Greece
University General Hospital of Ioannina Recruiting
Ioánnina, Ioannina, Greece, 45500
Contact: Chrisanthi Tziortzioti    +306949082918    sissitziortzioti@gmail.com   
Contact: George Gaitanis    +306974508561    ggaitan@cc.uoi.gr   
Principal Investigator: Ioannis Bassukas, MD         
University Hospital of Larissa Recruiting
Larissa, Thessaly, Greece, 41110
Contact: Angeliki-Viktoria Roussaki-Schulze, MD       roussaki@otenet.gr   
Principal Investigator: Angeliki-Viktoria Roussaki-Schulze, MD         
Hospital of Venereal and Skin Diseases A.Syggros Recruiting
Athens, Greece, 16121
Contact: Ioanna Kalkounou    +306945860027    ikalkounou@yahoo.gr   
Principal Investigator: Panagiotis Stavropoulos, MD         
Papageorgiou General Hospital of Thessaloniki Recruiting
Thessaloniki, Greece, 56429
Contact: Aikaterini Kyriakou    +302313323291    docmouli@gmail.com   
Principal Investigator: Aikaterini Patsatsi, MD         
Israel
Chaim Sheba Medical Center Recruiting
Ramat-Gan, Israel, 52621
Contact: Yaron Ben-Mordechai    +97236666666    jaronbm@gmail.com   
Principal Investigator: Sharon Baum, MD         
Tel Aviv Sourasky Medical Center Recruiting
Tel Aviv, Israel, 64239
Contact: Dvora Cohen    +97236973768    dvoray@tlvmc.gov.il   
Principal Investigator: Tal Zeeli, MD         
Sponsors and Collaborators
Principia Biopharma, Inc.
Principia Biopharma Australia Pty Ltd.
Investigators
Study Director: Steven G Gourlay, MBBS, PhD Principia Biopharma
More Information

Responsible Party: Principia Biopharma, Inc.
ClinicalTrials.gov Identifier: NCT02704429     History of Changes
Other Study ID Numbers: PRN1008-005
First Posted: March 10, 2016    Key Record Dates
Last Update Posted: December 7, 2017
Last Verified: December 2017
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Undecided

Additional relevant MeSH terms:
Pemphigus
Skin Diseases, Vesiculobullous
Skin Diseases
Autoimmune Diseases
Immune System Diseases