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 : August 31, 2018
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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 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.
| 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 : | 50 participants |
| Intervention Model: | Single Group Assignment |
| Intervention Model Description: | Part A with 12 weeks treatment and 12 weeks follow-up. Part B with 24 weeks treatment and 4 weeks follow-up. |
| 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
Part A: Open-label PRN1008, 12 weeks; 12 week follow up; Part B: Open-label PRN1008, 24 weeks;4 weeks follow up
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Drug: PRN1008
Part A: PRN1008, oral dose, 12 weeks; Part B: PRN1008, oral dose 24 weeks
Other Name: BTK inhibitor |
Primary Outcome Measures :
- Incidence of treatment-emergent adverse events [ Time Frame: Part A: 24 week 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: Part A: 12 week treatment; 12 week follow up and Part B: 24 week treatment, 4 week follow-up ]Time to control of disease activity (CDA) defined in EADV 2015 Pemphigus S2 Guideline
- Time to end of consolidation phase [ Time Frame: Part A: 12 week treatment; 12 week follow up and Part B: 24 week treatment, 4 week follow-up ]Time to end of consolidation phase as defined in EADV 2015 Pemphigus S2 Guideline
- Time to complete response [ Time Frame: Part A: 12 week treatment; 12 week follow up and Part B: 24 week treatment, 4 week follow-up ]Time to complete response as defined in EADV 2015 Pemphigus S2 Guideline
- Time to relapse after PRN1008 treatment discontinuation [ Time Frame: Part A: 12 week follow up and Part B: 4 week follow-up ]Time to relapse after PRN1008 treatment discontinuation
- Cumulative corticosteroid usage [ Time Frame: Part A: 12 week treatment; 12 week follow up and Part B: 24 week treatment, 4 week follow-up ]Cumulative corticosteroid usage
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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) 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 (≤ 10 mg/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
- Use of >10 mg per day of oral prednisolone per day within 2 weeks prior to Day 1 (inhaled and mucosal [for symptomatic treatment of oral lesions] corticosteroids are allowed)
- 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 there months or more of low body weight (BMI<17.5)
- 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
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 | 650-416-7758 | clinicaltrials@principiabio.com | |
| Contact: Ann Neale, RN BSN | 650-416-7784 | clinicaltrials@principiabio.com |
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: | August 31, 2018 |
| Last Verified: | August 2018 |
| 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 |