A Study of PRN1008 in Adult Patients With Pemphigus Vulgaris
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| ClinicalTrials.gov Identifier: NCT02704429 |
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Recruitment Status :
Recruiting
First Posted : March 10, 2016
Last Update Posted : December 7, 2017
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Sponsor:
Principia Biopharma, Inc.
Collaborator:
Principia Biopharma Australia Pty Ltd.
Information provided by (Responsible Party):
Principia Biopharma, Inc.
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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 |
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 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 |
| Arm | Intervention/treatment |
|---|---|
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Experimental: PRN1008
Open-label PRN1008, 12 weeks; 12 week follow up
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Drug: PRN1008
PRN1008, oral dose, 12 weeks
Other Name: BTK inhibitor |
Primary Outcome Measures :
- 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.
- 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 :
- 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
- 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
- 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
- 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
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| Ages Eligible for Study: | 18 Years to 80 Years (Adult, Older Adult) |
| Sexes Eligible for Study: | All |
| Accepts Healthy Volunteers: | No |
Criteria
Inclusion Criteria:
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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)
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 |
| 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:
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Pemphigus Skin Diseases, Vesiculobullous Skin Diseases Autoimmune Diseases Immune System Diseases |