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Trial details imported from ClinicalTrials.gov
For full trial details, please see the original record at
https://clinicaltrials.gov/ct2/show/NCT02704429
Registration number
NCT02704429
Ethics application status
Date submitted
24/02/2016
Date registered
10/03/2016
Date last updated
13/02/2023
Titles & IDs
Public title
A Study of PRN1008 in Adult Patients With Pemphigus Vulgaris
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Scientific 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
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Secondary ID [1]
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2015-003564-37
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Secondary ID [2]
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PRN1008-005
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Universal Trial Number (UTN)
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Trial acronym
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Linked study record
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Health condition
Health condition(s) or problem(s) studied:
Pemphigus Vulgaris
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Condition category
Condition code
Skin
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Dermatological conditions
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Skin
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Other skin conditions
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Inflammatory and Immune System
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Autoimmune diseases
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Intervention/exposure
Study type
Interventional
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Description of intervention(s) / exposure
Treatment: Drugs - PRN1008
Experimental: PRN1008 - Part A: Open-label PRN1008, 12 weeks; 12 weeks follow-up; Part B: Open-label PRN1008, 24 weeks; 4 weeks follow-up
Treatment: Drugs: PRN1008
Part A dosing was initiated administering 400 mg BID. Intrapatient dose adjustments (reductions and increases) were permitted based upon tolerability and clinical response with the maximum dose allowed up to 600 mg BID for 12 weeks.
Part B initial dosing was 400 mg QD for 2 weeks with dose escalation to 400 mg BID at the discretion of the Investigator for the purposes of investigating dose response and identifying the minimal efficacious dose of rilzabrutinib for 24 weeks.
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Intervention code [1]
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Treatment: Drugs
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Comparator / control treatment
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Control group
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Outcomes
Primary outcome [1]
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Percentage of Participants With Treatment-emergent Adverse Events
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Assessment method [1]
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Treatment-emergent adverse events (TEAEs) including clinically significant changes in physical examination, laboratory tests, and vital signs. An AE was defined as any untoward medical occurrence in a participant who received study drug and did not necessarily had to have a causal relationship with the treatment. TEAEs were defined as AEs that developed or worsened or became serious during on-treatment phase that was defined as the time from the start of study drug up to study completion.
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Timepoint [1]
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Part A: until 24 weeks and Part B: until 28 weeks
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Primary outcome [2]
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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
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Assessment method [2]
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CDA was defined as the time at which new lesions cease to form and established lesions begin to heal.
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Timepoint [2]
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4 weeks
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Secondary outcome [1]
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Percentage of Participants Able to Achieve Control of Disease Activity (CDA) Without Corticosteroids Within 4 Weeks
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Assessment method [1]
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CDA was defined as the time at which new lesions cease to form and established lesions begin to heal.
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Timepoint [1]
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4 weeks
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Secondary outcome [2]
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Percentage of Participants Able to Achieve a Complete Response (CR) Without Corticosteroids
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Assessment method [2]
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CR was defined as complete healing of all lesions and the absence of new lesions.
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Timepoint [2]
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Part A: 12 weeks treatment and Part B: 24 weeks treatment
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Secondary outcome [3]
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Percentage of Participants Able to Achieve Complete Remission (CR) Without the Need for Doses of Prednisone or Prednisolone of Greater Than 0.5mg/kg
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Assessment method [3]
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CR was defined as complete healing of all lesions and the absence of new lesions.
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Timepoint [3]
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Part A: 12 weeks treatment and Part B: 24 weeks treatment
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Secondary outcome [4]
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Time to Control of Disease Activity (CDA)
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Assessment method [4]
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CDA was defined as the time at which new lesions cease to form and established lesions begin to heal. Kaplan-Meier estimate median time is reported.
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Timepoint [4]
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Part A: until 24 weeks and Part B: until 28 weeks
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Secondary outcome [5]
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Time to End of Consolidation Phase (ECP)
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Assessment method [5]
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ECP was defined as the time at which no new lesions have developed for minimum of 2 weeks, and approximately 80% of existing lesions have healed. The median time to ECP was based on Kaplan-Meier estimate which considered censoring at end of follow-up for patients who didn't have an event. A +/-3 days window for visits at Week 3 and 5, and +/-7 days window for visits at Week 9, 13, 17, 21, 25.
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Timepoint [5]
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Part A: until 24 weeks and Part B: until 28 weeks
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Secondary outcome [6]
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Time to Complete Remission (CR)
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Assessment method [6]
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CR was defined as complete healing of all lesions and the absence of new lesions.
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Timepoint [6]
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Part A: until 24 weeks and Part B: until 28 weeks
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Secondary outcome [7]
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Time to Relapse After PRN1008 Treatment Discontinuation
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Assessment method [7]
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Relapse was defined as appearance of =3 new lesions/month that do not heal spontaneously within 1 week, or by extension of established lesions, in a patient who has achieved disease control. The median time to relapse was based on Kaplan-Meier estimate which considered censoring at end of follow-up for patients who didn't have an event. A +/-3 days window for visits at Week 3 and 5, and +/-7 days window for visits at Week 9, 13, 17, 21, 25.
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Timepoint [7]
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Part A: until 24 weeks and Part B: until 28 weeks
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Secondary outcome [8]
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Cumulative Corticosteroid Usage
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Assessment method [8]
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Cumulative corticosteroid usage
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Timepoint [8]
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Part A: until 24 weeks and Part B: until 28 weeks
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Secondary outcome [9]
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Percentage Change From Baseline in Pemphigus Disease Area Index (PDAI) Total Activity Scores
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Assessment method [9]
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The PDAI questionnaire has 2 components including activity and damage. The activity component consists of skin, scalp and mucosa parts, and the damage component consists of skin and scalp parts. The total activity score was used for the summary of PDAI scores. PDAI total activity score = Total skin activity + Total scalp activity + Total mucosa activity. PDAI Total Activity Score ranged from 0 to 250 points representing disease activity (higher scores mean a worse outcome). Negative change in total activity score from baseline indicates improvement in pemphigus activity.
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Timepoint [9]
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Part A: until 24 weeks and Part B: until 28 weeks
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Secondary outcome [10]
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Change From Baseline in Autoimmune Bullous Skin Disorder Intensity Score (ABSIS) Total Activity Score
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Assessment method [10]
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ABSIS Total Activity Score ranged from 0 to 206 points representing disease activity (higher scores mean a worse outcome). Negative change in total activity score from baseline indicates improvement in pemphigus activity.
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Timepoint [10]
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Part A: until 24 weeks and Part B: until 28 weeks
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Secondary outcome [11]
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Change From Baseline in Autoimmune Bullous Diseases Quality of Life (ABQOL)
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Assessment method [11]
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ABQOL score ranged from 0 to 68 points representing disease activity (higher scores mean a worse outcome).
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Timepoint [11]
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Part A: until 24 weeks and Part B: until 28 weeks
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Secondary outcome [12]
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Change From Baseline in Treatment of Autoimmune Bullous Diseases Quality of Life (TABQOL) Scores
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Assessment method [12]
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TABQOL score ranged from 0 to 68 points representing disease activity (higher scores mean a worse outcome).
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Timepoint [12]
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Part A: until 24 weeks and Part B: until 28 weeks
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Secondary outcome [13]
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Change From Baseline in Appetite (SNAQ Score)
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Assessment method [13]
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Simplified Nutritional Appetite Questionnaire (SNAQ) score ranged from 0 to 20 points representing disease activity (higher scores mean a better outcome).
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Timepoint [13]
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Part A: until 24 weeks and Part B: until 28 weeks
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Eligibility
Key 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
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Minimum age
18
Years
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Maximum age
80
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Sex
Both males and females
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Can healthy volunteers participate?
No
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Key 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
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Study design
Purpose of the study
Treatment
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Allocation to intervention
N/A
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Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
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Methods used to generate the sequence in which subjects will be randomised (sequence generation)
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Masking / blinding
Open (masking not used)
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Who is / are masked / blinded?
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Intervention assignment
Single group
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Other design features
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Phase
Phase 2
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Type of endpoint/s
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Statistical methods / analysis
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Recruitment
Recruitment status
Completed
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Data analysis
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Reason for early stopping/withdrawal
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Other reasons
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Date of first participant enrolment
Anticipated
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Actual
22/01/2016
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Date of last participant enrolment
Anticipated
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Actual
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Date of last data collection
Anticipated
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Actual
10/01/2020
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Sample size
Target
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Accrual to date
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Final
42
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Recruitment in Australia
Recruitment state(s)
NSW,VIC
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Recruitment hospital [1]
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Premier Specialists - Kogarah
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Recruitment hospital [2]
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Sinclair Dermatology - East Melbourne
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Recruitment hospital [3]
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Royal Melbourne, Dermatology Office - Melbourne
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Recruitment postcode(s) [1]
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2217 - Kogarah
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Recruitment postcode(s) [2]
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3002 - East Melbourne
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Recruitment postcode(s) [3]
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3050 - Melbourne
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Recruitment outside Australia
Country [1]
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Croatia
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State/province [1]
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Osijek
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Country [2]
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Croatia
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State/province [2]
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Zagreb
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Country [3]
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France
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State/province [3]
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Siene-Saint Denis
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Country [4]
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France
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State/province [4]
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Rouen
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Country [5]
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Greece
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State/province [5]
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Ioannina
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Country [6]
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Greece
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State/province [6]
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Thessaly
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Country [7]
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Greece
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State/province [7]
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Athens
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Country [8]
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Greece
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State/province [8]
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Thessaloniki
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Country [9]
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Israel
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State/province [9]
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Ramat-Gan
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Country [10]
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Israel
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State/province [10]
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Tel Aviv
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Funding & Sponsors
Primary sponsor type
Commercial sector/Industry
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Name
Principia Biopharma, a Sanofi Company
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Address
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Other collaborator category [1]
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Other
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Name [1]
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Principia Biopharma Australia Pty Ltd.
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Address [1]
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Country [1]
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Ethics approval
Ethics application status
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Summary
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.
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Trial website
https://clinicaltrials.gov/ct2/show/NCT02704429
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Trial related presentations / publications
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Public notes
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Contacts
Principal investigator
Name
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Clinical Sciences & Operations
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Address
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Sanofi
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Country
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Phone
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Fax
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Email
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Contact person for public queries
Name
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Address
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Country
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Phone
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Fax
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Email
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Contact person for scientific queries
Summary Results
For IPD and results data, please see
https://clinicaltrials.gov/ct2/show/NCT02704429
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