The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been endorsed by the ANZCTR. Before participating in a study, talk to your health care provider and refer to this information for consumers
Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/show/NCT03762265




Registration number
NCT03762265
Ethics application status
Date submitted
29/11/2018
Date registered
3/12/2018
Date last updated
1/08/2019

Titles & IDs
Public title
A Study of PRN1008 in Patients With Pemphigus
Scientific title
A Randomized, Double-Blind, Placebo-Controlled, Multicenter Trial to Evaluate the Efficacy and Safety of Oral BTK Inhibitor PRN1008 in Moderate to Severe Pemphigus
Secondary ID [1] 0 0
PRN1008-012
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Pemphigus 0 0
Condition category
Condition code
Skin 0 0 0 0
Dermatological conditions
Skin 0 0 0 0
Other skin conditions
Inflammatory and Immune System 0 0 0 0
Autoimmune diseases

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - PRN1008 Oral Tablet
Treatment: Drugs - Placebo Oral Tablet

Experimental: Experimental -

Placebo Comparator: Placebo -


Treatment: Drugs: PRN1008 Oral Tablet
Bruton's agammaglobulinemia tyrosine kinase (BTK) inhibitor

Treatment: Drugs: Placebo Oral Tablet
Placebo

Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
The proportion of patients who are in Complete Remission (CR) from Week = 29 to Week 37 with prednisone dose of =5 mg/day - Complete Remission (CR) defined as the absence of new and established lesions
Timepoint [1] 0 0
37 Weeks
Secondary outcome [1] 0 0
Cumulative Corticosteroids (CS) dose over first 36 weeks (to Week 37) - Cumulative CS dose over first 36 weeks (to Week 37)
Timepoint [1] 0 0
37 Weeks
Secondary outcome [2] 0 0
Change in EuroQOL-5 Dimension 5 Level (EQ-5D-5L) score from baseline to Weeks 5, 13, 25, and 61 - EQ-5D-5L questionnaire has 5 dimensions: "Mobility", "Self-Care," "Usual Activities", "Pain/Discomfort", "Anxiety/Depression", and all dimensions are described by 5 problem levels.
Timepoint [2] 0 0
61 Weeks
Secondary outcome [3] 0 0
Change in Autoimmune Bullous Disease Quality of Life (ABQOL) from baseline to Weeks 5, 13, 25, and 61 - The ABQOL questionnaire is a 17 item questionnaire assessing quality of life in patients with autoimmune blistering diseases
Timepoint [3] 0 0
61 Weeks

Eligibility
Key inclusion criteria
- Male or female aged 18-80 years old with moderate to severe pemphigus, newly diagnosed
or relapsing pemphigus vulgaris (PD) or pemphigus foliaceus (PF); positive anti-dsg1
or 3 autoantibody titer

- Laboratory clearance with adequate hematologic, hepatic, and renal function, body mass
index (BMI) >17.5

- Female patients who are of reproductive potential must agree for the duration of
active treatment to use an effective means of contraception

- Able to provide written informed consent
Minimum age
18 Years
Maximum age
80 Years
Gender
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
- Previous use of a Bruton's tyrosine kinase (BTK) inhibitor

- Pregnant or lactating women

- Clinically significant abnormalities, ECG findings or poorly controlled atrial
fibrillation

- A history of malignancy of any type, other than surgically excised non-melanoma skin
cancers or in situ cervical cancer within 5 years before Day 1

- Use of excluded medications within specified timeframes (proton pump inhibitors,
immunologic response modifiers, investigational drugs, CYP3A inducers or inhibitors,
CYP3A-sensitive substrates)

- History of drug abuse or excessive alcohol use

- Blood or blood product donation of a unit or more within 4 weeks

- Live vaccine within 28 days prior to Day 1 or plan to receive one during the trial

- Clinically significant diseases, positive at Screening of HIV and hepatitis A and B,
history of serious infections and active latent tuberculosis (TB)

Study design
Purpose of the study
Treatment
Allocation to intervention
Randomised controlled trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Blinded (masking used)
Who is / are masked / blinded?
The people receiving the treatment/s
The people administering the treatment/s
The people assessing the outcomes
The people analysing the results/data
Intervention assignment
Parallel
Other design features
Phase
Phase 3
Type of endpoint(s)
Statistical methods / analysis

Recruitment
Recruitment status
Recruiting
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
Sample size
Target
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
WA
Recruitment hospital [1] 0 0
Central Recruiting - Perth
Recruitment hospital [2] 0 0
Central Recruiting (Principia Biopharma) - Melbourne
Recruitment hospital [3] 0 0
Central Recruiting (Principia Biopharma) - Sydney
Recruitment postcode(s) [1] 0 0
- Perth
Recruitment postcode(s) [2] 0 0
- Melbourne
Recruitment postcode(s) [3] 0 0
- Sydney
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
Florida
Country [2] 0 0
United States of America
State/province [2] 0 0
Georgia
Country [3] 0 0
United States of America
State/province [3] 0 0
Michigan
Country [4] 0 0
United States of America
State/province [4] 0 0
Minnesota
Country [5] 0 0
United States of America
State/province [5] 0 0
New York
Country [6] 0 0
United States of America
State/province [6] 0 0
North Carolina
Country [7] 0 0
United States of America
State/province [7] 0 0
Ohio
Country [8] 0 0
United States of America
State/province [8] 0 0
Texas
Country [9] 0 0
United States of America
State/province [9] 0 0
Utah
Country [10] 0 0
Bulgaria
State/province [10] 0 0
Pleven
Country [11] 0 0
Bulgaria
State/province [11] 0 0
Sofia
Country [12] 0 0
France
State/province [12] 0 0
Lille
Country [13] 0 0
France
State/province [13] 0 0
Paris
Country [14] 0 0
France
State/province [14] 0 0
Rouen
Country [15] 0 0
Greece
State/province [15] 0 0
Thessaloníki
Country [16] 0 0
Israel
State/province [16] 0 0
Haifa
Country [17] 0 0
Italy
State/province [17] 0 0
Brescia
Country [18] 0 0
Poland
State/province [18] 0 0
Gdansk
Country [19] 0 0
Poland
State/province [19] 0 0
Lublin
Country [20] 0 0
Poland
State/province [20] 0 0
Warsaw
Country [21] 0 0
Poland
State/province [21] 0 0
Wroclaw
Country [22] 0 0
Spain
State/province [22] 0 0
Barcelona
Country [23] 0 0
Spain
State/province [23] 0 0
Pamplona
Country [24] 0 0
Taiwan
State/province [24] 0 0
Taipei
Country [25] 0 0
Turkey
State/province [25] 0 0
Istanbul
Country [26] 0 0
Turkey
State/province [26] 0 0
Konyaalti
Country [27] 0 0
Turkey
State/province [27] 0 0
Merkez
Country [28] 0 0
Ukraine
State/province [28] 0 0
Lviv
Country [29] 0 0
Ukraine
State/province [29] 0 0
Zaporizhzhya

Funding & Sponsors
Primary sponsor type
Commercial sector/Industry
Name
Principia Biopharma Inc.
Address
Country

Ethics approval
Ethics application status

Summary
Brief summary
This is a Phase 3 randomized, parallel-group, double-blind, placebo-controlled trial (Blinded
Treatment Period) followed by an Open-Label Extension Period intended to evaluate the
efficacy and safety of oral PRN1008 in moderate to severe pemphigus.
Trial website
https://clinicaltrials.gov/show/NCT03762265
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Dolca Thomas, M.D.
Address 0 0
Principia Biopharma Inc.
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Ann Neale
Address 0 0
Country 0 0
Phone 0 0
1-833-477-6700
Fax 0 0
Email 0 0
clinicaltrials@principiabio.com
Contact person for scientific queries

Summary results
For IPD and results data, please see https://clinicaltrials.gov/show/NCT03762265