An Open Label Pilot Trial of Guselkumab in the Treatment of Adults With Pityriasis Rubra Pilaris (PRP)
Phase: PHASE2
Conditions: Pityriasis Rubra Pilaris
Sponsor: Oregon Health and Science University

 
Page 1 of 34 
 
 
 
 
 
 
An open label pilot trial of  
guselkumab in the  
treatment of adults with  
Pityriasis Rubra Pilaris (PRP) 
 
 
 
 
 
 
 
 
 
Protocol Version Number: 
1.1 
Protocol Version Date: 
23 January 2019 
Investigational Product: 
Guselkumab 
IND/IDE Status: 
Exempt 
Principal Investigator 
Teri Greiling, MD, PhD 
Department of Dermatology 
Oregon Health & Science University, Center for Health and Healing 
3303 SW Bond Ave, CH16D  
Portland, OR 97239  
Phone: 503-494-3376 
Fax: 503-346-8105 
Email: Greiling@ohsu.edu 
Sponsor: 
Oregon Health & Science University 
Funding Source: 
Janssen Scientific Affairs 
Trial Configuration 
Single center, open label, interventional, investigator-initiated trial 

 
Page 2 of 34 
CLINICAL PROTOCOL SYNOPSIS 
 
Title 
An open label pilot trial of guselkumab in the treatment of adults with 
pityriasis rubra pilaris (PRP) 
Principal Investigator 
Teri Greiling, MD, PhD 
Objectives 
To determine whether guselkumab provides clinical improvement for 
subjects with PRP who are candidates for systemic therapy 
Trial Configuration 
Single center, open label, interventional, investigator-initiated trial 
Setting 
The trial will recruit 15 adults with moderate-to-severe (defined by 
involved body surface area (BSA) ≥ 10%) PRP through dermatologist 
offices, recruitment letters, and advertisement.  
Study visits will occur at Screening, weeks 0, 4, 8, 12, 20, 24, and 36. 
Study visits at weeks 0, 4, and 24 will be required in-person; the 
remaining visits optionally will be performed via secure video-
conferencing using the OHSU Nexus (Cisco Meeting) app, between the 
investigator and the subject, as well as a REDCap survey sent via email. 
Sample Size Estimate 
With measurement of the mean paired difference in PASI score before 
and after treatment of a minimum of 10 subjects, we would have 80% 
power at the 0.05 level to detect a mean within-subject change of 8-
points when the standard deviation is 8-points. Due to the uncertainty 
inherent with a pilot study, the goal number of subjects with PRP who 
will be recruited for the study is 15. 
Number of Participants 
15 subjects with moderate-to-severe PRP 
Eligibility Criteria 
Inclusion criteria 
• 
Willingness to comply with study procedures/requirements. 
• 
Capable of giving informed consent. 
• 
Diagnosis of PRP by clinical assessment and biopsy. 
• 
Male age 18-99, willing to use a reliable form of birth control if 
sexually active with a woman who is able to become pregnant. 
• 
Female age 18-99; either of non-childbearing potential or of 
childbearing potential who test negative for pregnancy and 
agree to use a reliable method of birth control or remain 
abstinent during the study and for at least 12 weeks following 
the last dose of guselkumab.  
• 
Involved BSA ≥ 10% at baseline (moderate-to-severe disease).  
• 
Are a candidate for phototherapy and/or systemic therapy. 
• 
Willingness to travel to OHSU for all study visits, or willing/able 
to participate in remote videoconferencing visits with access to 
a computer with internet and webcam capabilities.  
 
Exclusion criteria 
• 
Known malignancy or lymphoproliferative disease (except 
treated basal cell skin cancer, treated squamous cell skin 
cancer, or treated cervical carcinoma in situ) for at least 5 
years. 
• 
Active, untreated, acute or chronic infection, or 

 
Page 3 of 34 
immunocompromised to an extent that such that participation 
in the study would pose an unacceptable risk to the subject.  
• 
Positive for human immunodeficiency virus, hepatitis B virus, or 
hepatitis C virus. 
• 
Have latent or active untreated tuberculosis (TB), a positive 
QuantiFERON-TB Gold test result, signs or symptoms of active 
TB on medical history or physical examination, or close contact 
with a person with active TB who have not undergone 
evaluation or treatment for TB. Those who are currently 
undergoing treatment or who have documented completed 
treatment for active or latent TB without re-exposures will not 
be excluded. 
• 
Previous treatment with any agent that targets the interleukin 
23 p19 subunit specifically. 
• 
Systemic treatment with prednisone in the last 2 weeks, or 
other systemic therapies or phototherapy for PRP within the 
past 4 weeks or 5 half-lives prior to baseline, whichever is 
longer. For biologic therapies, the specific washout periods 
used will be: etanercept <28 days; infliximab, adalimumab, 
ixekizumab, or alefacept <60 days; golimumab <90 days; 
secukinumab <5 months; ustekinumab <8 months; rituximab or 
efalizumab <12 months. 
• 
Have a known allergy or hypersensitivity to any biologic 
therapy that would pose an unacceptable risk to the subject if 
participating in this study. 
• 
Have or intend to have a live vaccine within 3 months prior to 
baseline or 12 months prior to baseline in the case of the BCG 
vaccine, or any live vaccine during the course of study or within 
3 months after the last administration of study drug. 
• 
Had any major surgery within 8 weeks prior to baseline or will 
require major surgery during the study, that in the opinion of 
the investigator would pose an unacceptable risk to the 
subject. 
• 
Presence of significant uncontrolled cerebrovascular, 
respiratory, hepatic, renal, gastrointestinal, endocrine, 
hematologic, neurologic, or neuropsychiatric disorders, or 
abnormal laboratory screening values that, in the opinion of the 
investigator, pose an unacceptable risk to the subject if 
participating in the study or of interfering with the interpretation 
of the data. 
• 
Have clinical laboratory test results at screening that are 
outside the normal reference range of the population and are 
considered clinically significant, or have any of the following 
specific abnormalities: Neutrophil count <1500 cells/µL, white 
blood cell count <3500 cells/µL, platelet count <100,000 
cells/µL, AST or ALT or alkaline phosphatase > 2 times the 
upper limit of normal, hemoglobin <10 g/dL, serum creatinine 
>1.5 mg/dL. A single retest of laboratory values is permitted if 
one or more values is out of range. 
• 
Women who are lactating or breastfeeding. 
• 
Have any other condition that precludes the subject from 
following and completing the protocol, in the opinion of the 
investigator. 
• 
Are investigator site personnel directly affiliated with this study 
and/or their immediate families (spouse, parent, child, or 

 
Page 4 of 34 
sibling). 
• 
Are currently enrolled in, or discontinued from a clinical trial 
involving an investigational product or non-approved use of a 
drug or device within the last 4 weeks or a period of at least 5 
half-lives of the last administration of the drug, whichever is 
longer, or concurrently enrolled in any other type of medical 
research judged not to be scientifically or medically compatible 
with this study. 
Description of Interventions 
Subjects with PRP will be treated with guselkumab for 20 weeks using 
the FDA-approved dosing schedule for psoriasis (100 mg subcutaneous 
injection at weeks 0, 4, 12, and 20). 
Duration of Study 
The duration of the study will be 36 weeks 
Outcome Measures 
The primary outcome will be the mean change from baseline PASI at 
week-24 after treatment with guselkumab. 
Secondary and mechanistic outcomes include measurement of 
improvement in body surface area, nail involvement, quality of life, itch, 
pain; time to improvement by 50%; sustained remission at 36 weeks; 
and correlation of improvement with germline genetic mutations and 
cutaneous and serum cytokine expression. 
Statistical Methods 
Mean improvement from baseline PASI at week-24 will be analyzed for 
statistical significance using a paired, two-tailed Student’s t-test. 
 
 
 

 
Page 5 of 34 
Table of Contents 
CLINICAL PROTOCOL SYNOPSIS ........................................................................ 2 
ABBREVIATIONS .............................................................................................. 7 
1.  BACKGROUND ............................................................................................ 8 
1.1. OVERVIEW OF PITYRIASIS RUBRA PILARIS (PRP) AND GUSELKUMAB ......................... 8 
1.2. HYPOTHESIS ................................................................................................... 8 
2. OBJECTIVES ................................................................................................. 8 
2.1. PRIMARY OBJECTIVE ........................................................................................ 8 
2.2. SECONDARY OBJECTIVES ................................................................................... 9 
2.3. EXPLORATORY OBJECTIVES ................................................................................ 9 
3. STUDY DESIGN AND ENDPOINTS .................................................................. 9 
3.1. STUDY DESIGN OVERVIEW ................................................................................ 9 
3.2. STUDY ENDPOINTS ........................................................................................... 9 
3.2.1. Primary Endpoint .................................................................................. 9 
3.2.2. Secondary Endpoints Assessed by Investigator .................................... 9 
3.2.3. Secondary Endpoints Assessed by Subject ............................................ 9 
3.2.4. Exploratory/Mechanistic Endpoints ................................................... 10 
4.  STUDY ENROLLMENT AND WITHDRAWAL ................................................. 11 
4.1. INCLUSION CRITERIA ....................................................................................... 11 
4.2. EXCLUSION CRITERIA ...................................................................................... 11 
4.3. RECRUITMENT AND IDENTIFICATION OF PARTICIPANTS ......................................... 12 
4.4. VULNERABLE POPULATIONS ............................................................................ 12 
5. STUDY PROCEDURES, EVALUATIONS, AND SCHEDULE ................................ 12 
5.1 STUDY VISIT PROCEDURES ................................................................................ 12 
5.1.1 Pre-trial ............................................................................................... 12 
5.1.2. Screening ............................................................................................ 12 
5.1.3. Enrollment .......................................................................................... 13 
5.1.4. Visit 1 / Week-0 (baseline) ................................................................. 13 
5.1.5. Visit 2 / Week-4 (28 days +/- 4 days after last visit) ........................... 14 
5.1.6. Visit 3 / Week-8 (28 days +/- 4 days after last visit) ........................... 14 
5.1.7. Visit 4 / Week-12 (28 days +/- 4 days after last visit) ......................... 15 
5.1.8. Visit 5 / Week-20 (56 days +/- 7 days after last visit) ......................... 15 
5.1.9. Visit 6 / Week-24 (primary endpoint; 28 days +/- 4 days after last 
injection) ...................................................................................................... 16 
5.1.10. Visit 7 / Week-36 (56 days +/- 7 days after last visit) ....................... 16 
5.2. EARLY WITHDRAWAL / UNSCHEDULED VISITS ..................................................... 17 
5.3. PARTICIPANT STIPENDS AND PAYMENTS ............................................................ 17 
5.4. TABLE 1: STUDY SCHEDULE OF EVENTS .............................................................. 18 
6. INVESTIGATIONAL PRODUCT AND TREATMENT PLAN ................................ 19 
6.1. INVESTIGATIONAL PRODUCT: GUSELKUMAB ....................................................... 19 
6.2. DOSING AND ADMINISTRATION ........................................................................ 19 
6.3. STORAGE ..................................................................................................... 19 
6.4. CONCOMITANT MEDICATIONS, TREATMENTS, AND PROCEDURES .......................... 19 
6.4.1. Concomitant Medication Recording ................................................... 19 

 
Page 6 of 34 
6.4.2. Prohibited Medications, Treatments, and Procedures ....................... 19 
6.4.3. Permitted Medications, Treatments, and Procedures ........................ 19 
7. STATISTICAL CONSIDERATIONS .................................................................. 20 
7.1. SAMPLE SIZE COMPUTATION AND POWER ANALYSIS ........................................... 20 
7.2. STATISTICAL ANALYSIS PLAN ............................................................................ 20 
7.2.1. Primary Endpoint Analysis .................................................................. 20 
7.2.2. Secondary Endpoint Analyses: ............................................................ 20 
7.2.3. Exploratory Endpoint Analyses ........................................................... 21 
8. SAFETY ...................................................................................................... 21 
8.1. SPECIFICATION OF SAFETY PARAMETERS ............................................................ 21 
8.2. DEFINITIONS ................................................................................................. 21 
8.2.1. Adverse Event (AE) ............................................................................. 21 
8.2.2. Serious Adverse Event (SAE) ............................................................... 22 
8.2.3. Adverse Events of Special Interest ...................................................... 23 
8.2.4. Individual Case Safety Report (ICSR) .................................................. 23 
8.2.5. Product Quality Complaint (PQC) ....................................................... 23 
8.2.6. Unanticipated Problems (UP) ............................................................. 24 
8.3. ADVERSE EVENT ASSESSMENT AND FOLLOW-UP ................................................. 24 
8.4. REPORTING PROCEDURES ............................................................................... 25 
8.4.1. OHSU IRB Reporting of Unanticipated Problems and Adverse Events 25 
8.4.2. Reporting to the Study Drug Manufacturer ....................................... 25 
8.4.3. Pregnancy ........................................................................................... 27 
8.5. DATA SAFETY MONITORING PLAN .................................................................... 27 
8.6. POTENTIAL RISKS ........................................................................................... 28 
8.7. PARTICIPANT REMOVAL FROM THE TRIAL DUE TO ADVERSE EVENTS ....................... 28 
9. DATA HANDLING & MANAGEMENT RESPONSIBILITIES ............................... 30 
9.1. PARTICIPANT & DATA CONFIDENTIALITY ............................................................ 30 
9.2. DATA COLLECTION & STORAGE: PRIVACY, CONFIDENTIALITY & SECURITY ................ 30 
9.2.1. Data Collection ................................................................................... 30 
9.2.2. Data Confidentiality ........................................................................... 30 
9.2.3. Data Storage ...................................................................................... 30 
9.2.4. Data Recording in the Electronic Medical Record (Epic) .................... 31 
9.2.5. Photography ....................................................................................... 31 
9.2.6. Telemedicine Platform ....................................................................... 31 
10. ETHICS/PROTECTION OF PARTICIPANTS ................................................... 31 
10.1. ETHICAL STANDARD ..................................................................................... 31 
10.2. INSTITUTIONAL REVIEW BOARD ..................................................................... 32 
10.3. INFORMED CONSENT ................................................................................... 32 
10.4. CONSENT PROCEDURES AND DOCUMENTATION ................................................ 32 
10.5. PROTOCOL REVIEW ...................................................................................... 32 
10.6. CHANGES TO PROTOCOL ............................................................................... 32 
11. PUBLICATION AND DISSEMINATION POLICY ............................................ 32 
12. REFERENCES ............................................................................................ 33 
 
 

 
Page 7 of 34 
ABBREVIATIONS 
 
AE 
Adverse Event 
BSA 
Body Surface Area 
CARD14 
CAspase Recruitment Domain family, member 14 
CBC 
Complete Blood Count 
CMP 
Comprehensive Metabolic Panel 
CRF 
Case Report Form 
DLQI 
Dermatology Life Quality Index 
eCRF 
Electronic Case Report Form 
EDC 
Electronic Data Capture 
HBV 
Hepatitis B Virus 
HCV 
Hepatitis C Virus 
HIV 
Human Immunodeficiency Virus 
IL23 
Interleukin-23 
IND 
Investigational New Drug 
iPRPASI 
Individual PRP Area and Severity Index 
NAPSI 
Nail Psoriasis Severity Index 
NRS 
Numeric Rating Scale 
OHRP 
Office for Human Research Protections 
OHSU 
Oregon Health and Science University 
PCQ 
Product Quality Complaint 
PASI 
Psoriasis Area and Severity Index 
PGA 
Physician’s Global Assessment 
PI 
Principal Investigator 
PPPGA 
Palmoplantar Physician’s Global Assessment 
PRP 
Pityriasis Rubra Pilaris 
SAE 
Serious Adverse Event 
SC 
Subcutaneous 
SOAP 
Subjective, Objective, Assessment, Plan 
Th17 
T Helper 17 cell 
UP 
Unanticipated Problem 
 
 
 
 

 
Page 8 of 34 
1.  BACKGROUND 
1.1. Overview of Pityriasis Rubra Pilaris (PRP) and Guselkumab  
Pityriasis rubra pilaris (PRP) is a rare and poorly understood severe inflammatory cutaneous disease 
characterized by widespread (often full-body) erythematous plaques, skin flaking and ichthyosis, painful 
thickening and cracking of the palms and soles, hair loss, crumbling nails, and severe skin pruritus and 
burning. The most common type of PRP affects otherwise-healthy adults and may be explosive in onset. 
Although PRP does not affect internal organs and no quality-of-life impact studies have been published, 
the high degree of morbidity is perhaps best illustrated by the following colorful quotes from members of a 
PRP Facebook support group (with >800 members):   
“All I want is normal skin... for my eyes to stop watering constantly, to take off my clothes one time 
and not be shrouded by a cloud of dusty skin flakes...” 
“My husband can barely walk or use his hands.” 
“My scalp is like the Sahara Desert experiencing the dust storm of the century.” 
“I want my life back!!! Please.” 
Not surprisingly, one study showed over half of patients with PRP reporting depression (1).  
 
While the pathogenesis of PRP is poorly understood, similarities with inflammatory pathways in psoriasis 
have been reported. The major effector cells present in psoriatic lesions are Th17 cells, and a recent case 
report showed similar overexpression of cytokines in the Th17 pathway in cutaneous lesions of PRP, 
including the primary effector cytokines IL-23, IL-22, and IL-17 (2). Additionally, familial cases of both 
psoriasis and PRP have been associated with germline gain-of-function mutations in the caspase 
recruitment domain family, member 14 (CARD14) gene (3, 4), and cutaneous overexpression of CARD14 
was observed in cases of sporadic PRP (5). CARD14 overexpression in vitro has also been shown to 
induce excess IL-23 (6). 
 
Guselkumab is a high affinity, humanized IgG1 lambda monoclonal antibody that selectively binds the 
p19 subunit of IL-23. Two phase III studies of guselkumab in the treatment of psoriasis showed excellent 
safety and efficacy, with “clear” or “almost clear” skin ratings in 84-85% of patients at week-16 (7, 8). 
 
There is no FDA-approved therapy for PRP, not a single published systematic therapeutic clinical trial, 
and no established guidelines for measuring disease severity. Commonly prescribed systemic 
medications include acitretin and methotrexate, but these have treatment failure rates of over 40% (1) 
and can be limited by side effects. Tumor necrosis factor inhibitors have been used for PRP but have 
even higher failure rates (1). While there are no published reports of IL-23p19 subunit-targeted therapy in 
PRP, there are case reports and case series of the use of IL-12/IL-23p40 subunit inhibition for PRP in 
over twenty patients (2, 9-23) with 90% of these showing marked improvement, often only 4 weeks after a 
single subcutaneous injection. For these reasons, guselkumab is proposed as a therapeutic intervention 
with potential for improving the lives of PRP patients. 
 
1.2. Hypothesis 
Treatment of subjects who have moderate-to-severe PRP with guselkumab will lead to clinically 
significant improvement in subjective and objective measures of disease severity. 
 
2. OBJECTIVES 
2.1. Primary Objective  
Determine whether guselkumab provides clinical improvement for subjects with PRP who are candidates 
for systemic therapy 
 

 
Page 9 of 34 
2.2. Secondary Objectives  
Use systematic measurement tools to associate the severity of cutaneous involvement of PRP with 
quality of life measures, before and after therapy with guselkumab 
 
2.3. Exploratory Objectives  
Explore the mechanisms by which guselkumab alters the cutaneous and systemic inflammatory milieu in 
PRP 
 
3. STUDY DESIGN AND ENDPOINTS 
3.1. Study Design Overview 
This will be an investigator-initiated, open-label pilot study of 15 subjects with moderate-to-severe PRP 
treated with guselkumab at the FDA-approved dosing for psoriasis for 20 weeks, examining the primary 
efficacy endpoint of mean decrease in PASI score at week-24. Sustained remission will be assessed at 
week-36. Biopsies will be obtained before the first injection and at week-4 and week-24. Blood tests will 
be performed for screening and safety and measuring levels of inflammation at Screening, week-0, week-
4, and week-24. The study schedule is outlined in Section 5.4., Table 1. 
 
3.2. Study Endpoints 
3.2.1. Primary Endpoint 
Mean change from baseline PASI at week-24 after treatment with guselkumab. 
The Psoriasis Severity Index (PASI) is a well-validated tool for measuring psoriasis, based on redness, 
thickness, scale, and body surface area assessed by the investigator, with a maximum score of 72 points. 
This is expected to be a useful tool for PRP, since PRP is characterized by widespread bright red 
erythema and scale, and is often initially misdiagnosed as severe psoriasis. The mean thickness score is 
expected to be lower in PRP than psoriasis but the mean body surface area (BSA) is expected to be 
higher than psoriasis. 
 
3.2.2. Secondary Endpoints Assessed by Investigator 
• 
Time to improvement by 50% in the PASI score (PASI 50) 
• 
Mean reduction in body surface area (BSA) at week-24. BSA is collected as part of the PASI 
assessment tool. 
• 
Proportion of subjects achieving a Physician’s Global Assessment (PGA) of 0 or 1 at week-24 
• 
Proportion of subjects achieving a Palmoplantar Physician’s Global Assessment (PPPGA) of 0 or 
1 at week-24. Palmoplantar disease will be assessed separately from overall disease since it has 
high morbidity and may respond differently to therapy. 
• 
Mean percentage improvement in nail involvement measured by Nail Psoriasis Severity Index 
(NAPSI) at week-24. The Nail Psoriasis Severity Index (NAPSI) (24) will be used for evaluating 
the fingernails and toenails of subjects with PRP. This is expected to be useful tool because nails 
in PRP have many overlapping features with psoriasis including nail plate crumbling, splinter 
hemorrhages, nail oil drop (yellow-brown) discoloration, and nail bed hyperkeratosis, although 
PRP nails are less likely to have onycholysis and red spots in the lunula (25). 
 
3.2.3. Secondary Endpoints Assessed by Subject 
• 
Proportion of subjects achieving a 4-point improvement in quality of life measured by the 
Dermatology Life Quality Index (DLQI) at week-24, proportion of patients achieving a DLQI of 0 or 
1 at week-24, and mean improvement in DLQI score at week-24. The DLQI (26) is a validated 
tool for inflammatory skin conditions. It is a 10-question survey, scored 0 – 30 points. For 

 
Page 10 of 34 
inflammatory skin conditions, a 4-point change in DLQI score is considered clinically important 
(27). 
• 
Mean percentage in improvement in Psoriasis Symptoms and Signs Diary (PSSD) at week-24, 
and proportion of patients achieving a PSSD of zero at week-24. The PSSD is an 11-question 0 – 
10-point numeric rating scale (NRS) patient-reported outcome measure assessing itch, dryness, 
cracking, skin tightness, scaling, shedding or flaking, bleeding, burning, stinging, and pain over 
the last 24 hours and 7 days respectively (28). 
• 
Mean change from baseline individual PRP Area and Severity Index (iPRPASI) at week-24 after 
treatment with guselkumab, and correlation of subject-assessed iPRPASI scores with 
investigator-assessed PASI scores. The IPRPASI is a novel subject self-assessment score of 
disease involvement and severity, based on a previously published similar tool validated for 
psoriasis (29). 
• 
Sustained remission at week-36, 16 weeks after the last guselkumab dose, as measured by the 
mean change in PASI from week-24 to week-36. Sporadic PRP is often a self-limited disease, 
resolving spontaneously after an average of 3-6 years. Other immunomodulatory medications 
have induced sustained remission of PRP (22), so there is a potential that treatment may “reset” 
the immune response and lead to long-term improvement in the disease, especially in subjects 
without genetic mutations. Subjects will be treated with guselkumab as per the FDA-approved 
psoriasis treatment guidelines, and therapy will be stopped after the 20-week dose. Subjects will 
be monitored at 24 weeks (primary study endpoint) and then at 36 weeks to assess for sustained 
remission versus relapse. 
3.2.4. Exploratory/Mechanistic Endpoints 
• 
Treatment response, as measured by PASI 50, stratified by the presence or absence of germline 
CARD14 mutations. Genetic CARD14 gain-of-function mutations were identified in 3 families with 
familial PRP as well as 12.5% of individuals with sporadic PRP (3). Genetic CARD14 mutations 
will be measured in study subjects and associated with treatment response to guselkumab. 
Additionally, de-identified samples will be stored indefinitely for future analysis of genes 
associated with PRP. 
• 
Normalization of CARD14 epithelial expression at week-24. Despite the fact that not all patients 
with PRP have genetic CARD14 mutations, immunohistochemical staining of CARD14 in 6 of 6 
biopsies from sporadic cases of PRP without germline genetic mutations showed increased 
expression of CARD14 in the keratinocyte spinous and granular layers of the epidermis (2). 
CARD14 is thought to act in a synergistic manner with IL-17, activating the transcription factor 
NF-kB. NF-kB activation was shown in vitro to stimulate IL-36gamma, which may induce an 
amplifying feedback loop from IL-23 to IL-17 and back again (6). Cutaneous biopsies will be 
obtained from subjects before and 24 weeks after treatment with guselkumab. Inhibition of IL-17 
by guselkumab is expected to lead to normalization of CARD14 expression by keratinocytes. 
• 
Normalization of cutaneous gene expression at weeks 4 and 24. Upregulation of Th17 cytokines 
(IL-17A, IL-17F, IL-22) and pro-inflammatory innate cytokines (TNF, IL-6, IL-12, IL-23, IL-1beta) 
compared to normal skin were observed in lesional skin from 3 subjects with PRP (5). In order to 
better understand the inflammatory pathways involved in PRP, lesional and non-lesional biopsies 
will be obtained from subjects at week-0 prior to the first dose of guselkumab, week-4, and week-
24. Cutaneous gene expression of the epidermis and dermis will be analyzed by RNA 
Sequencing (RNA-Seq and therapeutic response to guselkumab is expected to correlate with 
normalization of epidermal cytokine levels. De-identified tissue samples will be stored indefinitely 
for analysis. 
• 
Change in serum cytokine levels at weeks 4 and 24. Serum levels of multiple cytokines have 
been shown to correlate with disease activity in patients with psoriasis (30) but these have not 
been investigated in patients with PRP. Serum cytokine levels before and after treatment with 
guselkumab will be analyzed by multiplexed bead-based immunoassay.     
 
 

 
Page 11 of 34 
4.  STUDY ENROLLMENT AND WITHDRAWAL 
4.1. Inclusion criteria 
• 
Diagnosis of PRP by clinical assessment and biopsy. 
• 
Male subject age 18-99, willing to use a reliable form of birth control if sexually active with a 
woman who is able to become pregnant. 
• 
Female subject age 18-99; either of non-childbearing potential or of childbearing potential who 
test negative for pregnancy and agree to use a reliable method of birth control or remain 
abstinent during the study and for at least 12 weeks following the last dose of guselkumab.  
• 
Involved BSA ≥ 10% at baseline.  
• 
Are a candidate for phototherapy and/or systemic therapy. 
• 
Willingness to travel to OHSU for all study visits, or willing/able to participate in remote 
videoconferencing visits with access to a computer with internet capabilities and webcam.  
• 
Have given written informed consent approved by the OHSU Investigational Review Board. 
 
4.2. Exclusion criteria 
• 
Known malignancy or lymphoproliferative disease (except treated basal cell skin cancer, treated 
squamous cell skin cancer, or treated cervical carcinoma in situ) for at least 5 years. 
• 
Active, untreated, acute or chronic infection, or immunocompromised to an extent that such that 
participation in the study would pose an unacceptable risk to the subject. 
• 
Positive for human immunodeficiency virus, hepatitis B virus, or hepatitis C virus. 
• 
Have latent or active untreated tuberculosis (TB), a positive QuantiFERON-TB Gold test result, 
signs or symptoms of active TB on medical history or physical examination, or close contact with 
a person with active TB and have not undergone evaluation or treatment for TB. Those who are 
currently undergoing treatment or who have documented completed treatment for active or latent 
TB without re-exposures will not be excluded. 
• 
Previous treatment with any agent that targets the interleukin 23 p19 subunit specifically. 
• 
Systemic treatment with prednisone in the last 2 weeks, or other systemic therapies or 
phototherapy for PRP within the past 4 weeks or 5 half-lives prior to baseline, whichever is 
longer. For biologic therapies, the specific washout periods used will be: etanercept <28 days; 
infliximab, adalimumab, ixekizumab, or alefacept <60 days; golimumab <90 days; secukinumab 
<5 months; ustekinumab <8 months; rituximab or efalizumab <12 months. 
• 
Have a known allergy or hypersensitivity to any biologic therapy that would pose an unacceptable 
risk to the subject if participating in this study. 
• 
Have or intend to have a live vaccine within 3 months prior to baseline or 12 months prior to 
baseline in the case of the BCG vaccine, or any live vaccine during the course of study or within 3 
months after the last administration of study drug. 
• 
Had any major surgery within 8 weeks prior to baseline or will require major surgery during the 
study that, in the opinion of the investigator, would pose an unacceptable risk to the subject. 
• 
Presence of significant uncontrolled cerebrovascular, respiratory, hepatic, renal, gastrointestinal, 
endocrine, hematologic, neurologic, or neuropsychiatric disorders, or abnormal laboratory 
screening values that, in the opinion of the investigator, pose an unacceptable risk to the subject 
if participating in the study or of interfering with the interpretation of the data. 
• 
Have clinical laboratory test results at screening that are outside the normal reference range of 
the population and are considered clinically significant, or have any of the following specific 
abnormalities: Neutrophil count <1500 cells/µL, white blood cell count <3500 cells/µL, platelet 
count <100,000 cells/µL, AST or ALT or alkaline phosphatase > 2 times the upper limit of normal, 
hemoglobin <10 g/dL, serum creatinine >1.5 mg/dL. A single retest of laboratory values is 
permitted if one or more values is out of range. 
• 
Women who are lactating or breastfeeding. 
• 
Have any other condition that precludes the subject from following and completing the protocol, in 
the opinion of the investigator. 
• 
Are investigator site personnel directly affiliated with this study and/or their immediate families 
(spouse, parent, child, or sibling). 

 
Page 12 of 34 
• 
Are currently enrolled in, or discontinued from a clinical trial involving an investigational product or 
non-approved use of a drug or device within the last 4 weeks or a period of at least 5 half-lives of 
the last administration of the drug, whichever is longer, or concurrently enrolled in any other type 
of medical research judged not to be scientifically or medically compatible with this study. 
 
4.3. Recruitment and Identification of Participants 
The trial will recruit subjects with moderate-to-severe PRP, either newly diagnosed or failing other 
therapies due to side effects or lack of efficacy. A number of recruitment strategies will be utilized for this 
study proven to be effective by our group in previous studies. 
• 
OHSU and community dermatology clinics 
• 
OHSU OCTRI Cohort Discovery tool to identify patients with PRP via Epic, and a phone call to 
those patients 
• 
Publicity on the PRP Support Group Facebook page with prior approval of Facebook page 
manager 
• 
Publicity through the PRP Alliance patient advocacy organization, including an email to members 
of the PRP Alliance, following prior approval from listserv manager 
• 
Research Match 
• 
Letters mailed to physicians outside of OHSU 
 
4.4. Vulnerable Populations 
Children, neonates, pregnant women, prisoners, and decisionally impaired adults will not be enrolled in 
the study. 
 
5. STUDY PROCEDURES, EVALUATIONS, AND SCHEDULE 
5.1 Study Visit Procedures 
5.1.1 Pre-trial 
During the first contact with the study team, an overview of the trial will be given. Because of the rarity of 
PRP, subjects may be traveling from distant locations for the study; thus the phone script is intended to 
be somewhat more comprehensive for a subject to determine if they are interested and able to 
participate. If they would like to participate, a request for information will be signed by the subject and 
sent to the subject’s healthcare providers to review records pertinent to the diagnosis of PRP. Once the 
study investigator has reviewed the records and confirmed that a diagnosis of PRP has been made by a 
qualified healthcare provider, an appointment for a screening visit will be made. For subjects in whom the 
screening visit will be completed by telemedicine, the informed consent form will be emailed or mailed to 
the subject prior to the screening visit. 
 
5.1.2. Screening 
Subjects will read the informed consent form and all questions about the study will be answered by a 
study investigator and research staff.  Screening questions will be asked and eligibility will be determined 
by qualified research staff. Subjects will be asked about vaccine history to ensure that recommended 
vaccines are up to date, and subjects will be reminded that live vaccines (such as the intranasal influenza 
vaccine, BCG vaccine, or live varicella zoster vaccine (Zostavax)) are not permitted during the study. 
Contraception method will also be recorded. An investigator will perform an assessment of BSA to 
calculate disease severity. If all other criteria are met, a blood sample will be drawn to screen for 
pregnancy (in female subjects), HBV, HCV, HIV, and QuantiFERON gold for tuberculosis, and to check 
CBC and CMP. If subjects have had HBV, HCV, HIV, and tuberculosis testing within 3 months prior to 
screening, records of these results will be sufficient and the tests will not be repeated. PPD skin testing 
may be used if blood testing for tuberculosis is unavailable. 

 
Page 13 of 34 
For interested subjects, the screening visit may be performed via secure video-conferencing using the 
OHSU Nexus (Cisco Meeting) app, in which case the study consent will be reviewed during the 
telemedicine videoconference. Eligible subjects willing to proceed with treatment will be asked to 
document consent via e-consent in REDCap, or signed written consent emailed or faxed to the 
investigators. Following receipt of signed consent, a screening blood draw will be performed at the lab of 
the subject’s choice and results will be sent to the investigators.  
Upon confirmation of normal blood tests as defined in the exclusion criteriasubjects will be scheduled for 
enrollment and Visit 1 as appropriate for any medication washout period required in the exclusion criteria. 
If blood tests are abnormal, the subject will be informed and referred to her or his primary care physician 
for assessment. 
For Spanish-speaking subjects, the English-written consent form will be verbally translated with the help 
of a Spanish interpreter. All questions regarding the study will be translated to study staff and answered 
to patient satisfaction. Subjects will then be provided the adapted boilerplate consent, “Consent Form – 
Short – Spanish,” to sign. A copy of both signed consents will be provided to the patient and uploaded in 
the patient’s chart. 
 
5.1.3. Enrollment 
The inclusion/exclusion criteria will be re-reviewed and subjects may proceed with visit 1 on the same day 
if they continue to meet criteria. 
 
5.1.4. Visit 1 / Week-0 (baseline) 
Visit 1 will be performed at OHSU for all subjects.  
• 
Study personnel will gather the following information from the subjects and record the results in 
REDCap: 
§ 
Demographics (sex, gender, race, ethnicity) 
§ 
Current medications 
§ 
Drug allergies 
§ 
Use of alcohol, tobacco, and other substances 
§ 
PRP medical history 
§ 
General medical history and adverse events since screening 
§ 
Family medical history 
§ 
Review of Systems  
§ 
Height and weight 
§ 
Blood pressure 
§ 
Pulse 
• 
Subjects will enter the following information about their disease and medical history via OHSU 
REDCap survey: 
§ 
DLQI 
§ 
PSSD  
§ 
iPRPASI 
• 
The subject will be clinically examined by an investigator (clinician or research nurse) who will assess 
the following and record the results in OHSU REDCap: 
§ 
Full body skin exam with documentation of PASI, PGA, PPPGA, and NAPSI 
§ 
Examination of the oral mucosa 
§ 
Heart and lung auscultation 
§ 
Palpation of the cervical, axillary, and inguinal lymph nodes 
§ 
Presence of peripheral edema 
• 
Photographs will be taken using a high-resolution digital camera of all body regions against a dark 
backdrop to visually record extent of disease. Subjects will assume standardized poses according to 
published recommendations (31), with the addition of focused photographs of the palms and nails. 
• 
Subjects will undergo a punch biopsy (consisting of three adjacent 3 mm punch biopsies, a total 
surface area equivalent to the size of a 5 mm punch biopsy) from affected skin, performed by an 

 
Page 14 of 34 
investigator. One additional 3 mm punch biopsy will be obtained from non-lesional skin as a control if 
non-lesional skin is available. For consistency, skin of back will be chosen for biopsy if an affected 
site is available. If back skin is not available, alternate sites in order of preference will include: chest, 
arm, leg. Location of the biopsy will be carefully documented. 
• 
Blood will be drawn for measuring levels of inflammation 
• 
A saliva sample will be collected for genetic analysis 
• 
The first dose of 100 mg of subcutaneous guselkumab will be administered by trained study 
personnel while the study subject undergoes teaching for future self-administration. 
• 
Subjects who plan to complete future visits by videoconferencing will receive a scale and 
sphygmomanometer for future vital sign assessments 
 
5.1.5. Visit 2 / Week-4 (28 days +/- 4 days after last visit) 
Visit 2 will be performed at OHSU for all subjects.  
• 
Study personnel will gather the following information from the subjects and record the results in 
REDCap: 
§ 
Change in medications since the last visit 
§ 
Change in drug allergies since the last visit 
§ 
Change in personal or family medical history since the last visit, including adverse events  
§ 
Weight  
§ 
Blood pressure  
§ 
Pulse  
• 
Subjects will enter the following information about their disease and medical history via OHSU 
REDCap survey: 
§ 
DLQI 
§ 
PSSD  
§ 
iPRPASI 
• 
The subject will be clinically examined by an investigator (clinician or research nurse) who will assess 
the following: 
§ 
Full body skin exam with documentation of PASI, PGA, and PPPGA 
• 
Photographs will be taken against a dark backdrop with standardized poses according to published 
recommendations (31), with the addition of focused photographs of the palms and nails. 
• 
Subjects will undergo a punch biopsy (consisting of three adjacent 3 mm punch biopsies, a total 
surface area equivalent to the size of a 5 mm punch biopsy) in the same body region as the first 
documented biopsy (back, chest, arm, or leg) 
• 
The second dose of 100 mg of subcutaneous guselkumab will be administered by the subject with 
observation and teaching from study personnel. 
• 
Blood will be drawn for monitoring the CBC, CMP, and measuring levels of inflammation 
 
5.1.6. Visit 3 / Week-8 (28 days +/- 4 days after last visit) 
Visit 3 will be performed at OHSU or optionally by telemedicine and REDCap survey.  
• 
Study personnel will gather the following information from the subjects and record the results in 
REDCap: 
§ 
Change in medications since the last visit 
§ 
Change in drug allergies since the last visit 
§ 
Change in personal or family medical history, including adverse events, since the last visit  
§ 
Weight (performed by the subject for videoconferencing visits) 
§ 
Blood pressure (performed by the subject for videoconferencing visits) 
§ 
Pulse (performed by the subject for videoconferencing visits) 
• 
Subjects will enter the following information about their disease and medical history via OHSU 
REDCap survey: 
§ 
DLQI 
§ 
PSSD 
§ 
iPRPASI 

 
Page 15 of 34 
• 
The subject will be clinically examined by an investigator (clinician or research nurse, either in-person 
or via telemedicine) who will assess the following: 
§ 
Full body skin exam with documentation of PASI, PGA, and PPPGA 
 
5.1.7. Visit 4 / Week-12 (28 days +/- 4 days after last visit) 
Visit 4 will be performed at OHSU or optionally by telemedicine and REDCap survey for subjects who live 
>30 miles from OHSU.  
• 
Study personnel will gather the following information from the subjects and record the results in 
REDCap: 
§ 
Change in medications since the last visit 
§ 
Change in drug allergies since the last visit 
§ 
Change in personal or family medical history, including adverse events, since the last visit  
§ 
Weight (performed by the subject for videoconferencing visits) 
§ 
Blood pressure (performed by the subject for videoconferencing visits) 
§ 
Pulse (performed by the subject for videoconferencing visits) 
• 
Subjects will enter the following information about their disease and medical history via OHSU 
REDCap survey: 
§ 
DLQI 
§ 
PSSD  
§ 
iPRPASI 
• 
The subject will be clinically examined by an investigator (clinician or research nurse, either in-person 
or via telemedicine) who will assess the following: 
§ 
Full body skin exam with documentation of PASI, PGA, and PPPGA 
• 
The third dose of 100 mg of subcutaneous guselkumab will be administered by the subject with 
observation and teaching from study personnel. 
• 
Blood will be drawn for CBC, CMP (for telemedicine visits this will be done at a local lab of the 
subject’s choice within +/- 2 days of the visit) 
 
5.1.8. Visit 5 / Week-20 (56 days +/- 7 days after last visit) 
Visit 5 will be performed at OHSU or optionally by telemedicine and REDCap survey for subjects who live 
>30 miles from OHSU.  
• 
Study personnel will gather the following information from the subjects and record the results in 
REDCap: 
§ 
Change in medications since the last visit 
§ 
Change in drug allergies since the last visit 
§ 
Change in personal or family medical history, including adverse events, since the last visit  
§ 
Weight (performed by the subject for videoconferencing visits) 
§ 
Blood pressure (performed by the subject for videoconferencing visits) 
§ 
Pulse (performed by the subject for videoconferencing visits) 
• 
Subjects will enter the following information about their disease and medical history via OHSU 
REDCap survey: 
§ 
DLQI 
§ 
PSSD 
§ 
iPRPASI 
• 
The subject will be clinically examined by an investigator (clinician or research nurse, either in-person 
or via telemedicine) who will assess the following: 
§ 
Full body skin exam with documentation of PASI, PGA, and PPPGA 
• 
The fourth dose of 100 mg of subcutaneous guselkumab will be administered by the subject with 
observation and teaching from study personnel. 
 

 
Page 16 of 34 
5.1.9. Visit 6 / Week-24 (primary endpoint; 28 days +/- 4 days after last 
injection) 
Visit 6 will be performed at OHSU for all subjects.  
• 
Study personnel will gather the following information from the subjects and record the results in 
REDCap: 
§ 
Change in medications since the last visit 
§ 
Change in drug allergies since the last visit 
§ 
Change in personal or family medical history, including adverse events, since the last visit  
§ 
Weight 
§ 
Blood pressure 
§ 
Pulse 
• 
Subjects will enter the following information about their disease and medical history via OHSU 
REDCap survey: 
§ 
DLQI 
§ 
PSSD  
§ 
iPRPASI 
• 
The subject will be clinically examined by an investigator (clinician or research nurse) who will assess 
the following: 
§ 
Full body skin exam with documentation of PASI, PGA, PPPGA, and NAPSI 
§ 
Examination of the oral mucosa 
§ 
Heart and lung auscultation 
§ 
Palpation of the cervical, axillary, and inguinal lymph nodes 
§ 
Presence of peripheral edema 
• 
Photographs will be taken against a dark backdrop with standardized poses according to published 
recommendations (31), with the addition of focused photographs of the palms and nails. 
• 
Blood will be drawn for monitoring the CBC, CMP, and measuring levels of inflammation 
• 
Subjects will undergo a punch biopsy (consisting of three adjacent 3 mm punch biopsies, a total 
surface area equivalent to the size of a 5 mm punch biopsy) in the same body region as the first 
documented biopsy (back, chest, arm, or leg), preferably in an area affected by PRP at the first study 
visit and possibly cleared or improved at the endpoint visit. 
 
5.1.10. Visit 7 / Week-36 (56 days +/- 7 days after last visit) 
Visit 7 will be performed at OHSU or optionally by telemedicine and REDCap survey for subjects who live 
>30 miles from OHSU.  
• 
Study personnel will gather the following information from the subjects and record the results in 
REDCap: 
§ 
Change in medications since the last visit 
§ 
Change in drug allergies since the last visit 
§ 
Change in personal or family medical history, including adverse events, since the last visit  
§ 
Weight (performed by the subject for videoconferencing visits) 
§ 
Blood pressure (performed by the subject for videoconferencing visits) 
§ 
Pulse (performed by the subject for videoconferencing visits) 
• 
Subjects will enter the following information about their disease and medical history via OHSU 
REDCap survey: 
§ 
DLQI 
§ 
PSSD 
§ 
iPRPASI 
• 
The subject will be clinically examined by an investigator (clinician or research nurse, either in-person 
or via telemedicine) who will assess the following: 
§ 
Full body skin exam with documentation of PASI, PGA, and PPPGA 
 

 
Page 17 of 34 
5.2. Early Withdrawal / Unscheduled Visits 
If the subject wishes to withdraw from the trial before week-24, they will be asked to attend for a final trial 
(early termination) visit.  At this visit, if the subject is willing, the same procedures as for the primary 
outcome / week-24 visit will be carried out. If the subject withdraws after the week-24 visit, the final week-
36 visit will be carried out early instead. 
 
5.3. Participant Stipends and Payments 
Participants will be paid $50 for all visits that do not involve skin biopsy (Screening and visits 3, 4, 5, and 
7) and $150 for the three visits that involve skin biopsy (baseline, visit 2, and visit 6), for a total of $700. 
Other contacts are not reimbursed. For those subjects traveling >200 miles to OHSU, travel will be 
reimbursed at the federal Internal Revenue Service suggested rate of $0.545/mile up to 1000 miles 
(range $218 - $1090 for a round-trip visit to OHSU) for each of the three in-person visits (baseline, visit 2, 
and visit 6). 
 
 

 
Page 18 of 34 
5.4. Table 1: Study Schedule of Events 
 
Visit 
Pre-
trial 
Screening* Enrollment 
V1 
V2 
V3 
V4 
V5 
V6 
V7 
Week 
WK0 
WK4 
WK8* 
WK12* WK20* 
WK24 
WK36* 
Baseline Documentation 
Confirmation of dx 
X 
 
 
 
 
 
 
 
 
 
Informed consent 
 
X 
 
 
 
 
 
 
 
 
Investigator 
assessment 
 
X 
 
X 
X 
X 
X 
X 
X 
X 
Medical history 
 
X 
 
X 
X 
X 
X 
X 
X 
X 
Exclusion/Inclusion 
criteria 
 
X 
X 
 
 
 
 
 
 
 
Dosing 
Injection 
counseling/training 
 
 
 
X 
 
 
 
 
 
 
Injection review 
 
 
 
 
X 
 
X 
X 
 
 
Study drug 
dispensing 
 
 
 
X 
X 
 
 
 
 
 
Study drug 
injection/dose** 
 
 
 
X 
X 
 
X 
X 
 
 
Laboratory Studies 
Blood draw*** 
 
X 
 
X 
X 
 
X 
 
X 
 
Other Assessments and Procedures 
Skin biopsy 
 
 
 
X 
X 
 
 
 
X 
 
Adverse events 
 
 
 
X 
X 
X 
X 
X 
X 
X 
Subject REDCap 
survey 
 
 
 
X 
X 
X 
X 
X 
X 
X 
 
 
*Visit optionally to be performed via secure telemedicine videoconferencing for subjects >30 miles from Oregon 
Health & Science University.  
**Guselkumab subcutaneous dose: 100mg on W0, W4, W12, W20. 
***Laboratory studies will include: CBC and CMP at screening, W4, W12, and W20 as well as HBV, HCV, HIV, 
tuberculosis, and pregnancy (for females if childbearing potential) at screening. 
 
 
 

 
Page 19 of 34 
6. INVESTIGATIONAL PRODUCT AND TREATMENT PLAN 
6.1. Investigational Product: Guselkumab 
Guselkumab is a humanized monoclonal antibody that binds and blocks IL-23.  
 
6.2. Dosing and Administration 
Guselkumab treatment will be administered at the FDA-approved dosing schedule for psoriasis as 
follows: 
• 
100 mg SC injection at week-0, week-4, week-12, and week-20 
The 1 mL single-dose pre-filled syringe will be injected at room temperature into the subcutaneous tissue 
of the upper arms, thighs, or abdomen, avoiding areas affected by PRP when possible. Each injection will 
be administered in an anatomic location different from the previous injection, and the location will be 
recorded. Injections will be performed by qualified study personnel or by the subject after proper training 
in SC injection technique. Injections performed at home by the subject will be recorded by the subject in 
an injection diary and returned to the investigators. 
 
6.3. Storage 
The study drug will be stored in the Research Pharmacy and all applicable Research Pharmacy policies 
and procedures will be followed.  
 
6.4. Concomitant Medications, Treatments, and Procedures 
6.4.1. Concomitant Medication Recording 
All concomitant medications taken during the study must be recorded. Subjects will be asked about 
concomitant medications at each study visit. 
 
6.4.2. Prohibited Medications, Treatments, and Procedures 
The following therapies will not be permitted during the course of the study: 
• 
Treatment with systemic therapy intended to treat PRP within the washout period specified in the 
exclusion criteria, including prednisone, methotrexate, cyclosporine, acitretin, and injectable 
biologic therapies. These medications could have a negative safety impact on the subjects 
enrolled and confound the results of the study. 
• 
New use of topical steroids or increased potency of topical steroids after enrollment 
• 
Live vaccines 
• 
Phototherapy within the 4-week washout period 
 
6.4.3. Permitted Medications, Treatments, and Procedures 
The following medications will be permitted during the course of the study: 
• 
Topical emollients 
• 
Topical steroids may be maintained at levels used prior to enrollment, decreased, or 
discontinued, but potency and weekly grams used may not be increased 
• 
Non-live vaccines (such as the non-live annual influenza vaccine, non-live herpes zoster vaccine 
(Shingrix), rabies, or tetanus) and/or emergency vaccines are allowed 
• 
Acetaminophen, aspirin, or ibuprofen as needed 
• 
Subjects will maintain their usual medication regimen for other concomitant diseases throughout 
the study unless specifically excluded in the protocol. Subjects taking concomitant medications 
should be on a stable dose throughout the study, unless changes need to be made for an AE or 
for appropriate medical management. If a medication is changed, the changed and reason for it 
will be documented. Additional systemic drugs are to be avoided during the study, unless 

 
Page 20 of 34 
required to treat an AE. Other medications may be allowed, if approved by the investigator. Any 
changes in medications should be discussed with the investigator. Subjects should be instructed 
to consult the investigator or other appropriate study personnel at the site before taking any new 
medications or supplements. All therapies (prescription, over-the-counter, vitamins, herbal 
supplements, vaccines) will be recorded in the eCRF as outlined in the Study Procedures. 
7. STATISTICAL CONSIDERATIONS 
7.1. Sample Size Computation and Power Analysis 
This is a pilot study with no published baseline data; thus a power analysis was performed with the 
following rough assumptions. With measurement of the mean paired difference in PASI score before 
and after treatment of a minimum of 10 subjects, we would have 80% power at the 0.05 level to detect 
a mean within-subject change of 8-points when the standard deviation is 8-points. Due to the 
uncertainty inherent with a pilot study, the goal number of subjects with PRP who will be recruited for 
the study is 15. 
 
 
7.2. Statistical Analysis Plan 
Data analysis will be ongoing throughout the trial by the PI and is expected to be complete within 3 
months after the last-subject last-visit. 
 
7.2.1. Primary Endpoint Analysis 
• 
Mean improvement from baseline PASI at week-24 will be analyzed for statistical significance 
using a paired, two-tailed Student’s t-test. 
 
7.2.2. Secondary Endpoint Analyses: 
• 
Time (weeks) to improvement by 50% in the PASI score (PASI 50) will be reported as a mean 
with standard deviation and also represented graphically. 
• 
Mean improvement from baseline BSA at week-24 will be analyzed for statistical significance 
using a paired, two-tailed Student’s t-test. 
• 
The proportion of subjects achieving a Physician’s Global Assessment of 0 or 1 at week-24 will 
be reported as a proportion, and compared with historical estimates of treatment success with 
acitretin and methotrexate (1). 
• 
The proportion of subjects achieving a Palmoplantar Physician’s Global Assessment of 0 or 1 at 
week-24 will be reported as a proportion. 
• 
The mean improvement in nail involvement measured by NAPSI (see below) at week-24 will be 
analyzed for statistical significance using a paired, two-tailed Student’s t-test. 
• 
The proportion of subjects achieving a 4-point improvement in DLQI and the proportion of 
subjects achieving a DLQI score of 0 or 1 at week-24 will be reported as proportions. The mean 

 
Page 21 of 34 
improvement in DLQI score from week-0 to week-24 will be analyzed using a paired, two-tailed 
Student’s t-test. 
• 
The mean improvement in PSSD at week-24 will be analyzed for statistical significance using a 
paired, two-tailed Student’s t-test. The proportion of subjects achieving a PSSD of zero at week-
24 will be reported as a proportion. 
 
7.2.3. Exploratory Endpoint Analyses 
• 
Sustained remission at week-36, measured by the mean change in PASI score from week-24 to 
week-36, will be analyzed for statistical significance using a paired, two-tailed Student’s t-test. 
• 
Treatment response, as measured by PASI 50, will be stratified by CARD14 germline mutations. 
The proportion of subjects with a PASI 50 response with and without CARD14 mutations will be 
reported and compared for statistical significance using an unpaired, two-tailed Student’s t-test. 
• 
Epithelial expression of CARD14 will be assessed qualitatively by a trained dermatopathologist 
who is blinded to the biopsy collection time (before or after therapy). 
• 
The gene expression level from the tissue biopsy, before, 4 weeks, and 24 weeks after treatment, 
will be analyzed for statistical significance using a pared, two-tailed Student’s t-test and also 
compared with normal and psoriatic skin using a two-tailed, two-sample Student’s t-test with 
unequal variance adjusted for multiple comparisons. 
• 
The cytokine level from serum before, 4 weeks, and 24 weeks after treatment, will be analyzed 
for statistical significance using a pared, two-tailed Student’s t-test and also compared with 
normal and psoriatic serum using a two-tailed, two-sample Student’s t-test with unequal variance 
adjusted for multiple comparisons. 
 
8. SAFETY 
8.1. Specification of Safety Parameters 
As the sponsor of the Study, OHSU and the PI shall be solely responsible for complying, within the 
required timelines, any safety reporting obligation to competent Health Authorities, IRB and any 
participating (co or sub) investigators, as defined in applicable laws and regulations.  For the purposes of 
this protocol, safety data includes adverse events, product quality complaints (PQCs), and special 
situations including pregnancies. Safety assessments will be based on medical review of adverse events 
and the results of safety evaluations at specified time points as described in Section 5.1 Study Visit 
Procedures. Any clinically significant adverse events persisting at the end of treatment visit will be 
followed by the Investigator until resolution/stabilization or death, whichever comes first. 
OHSU and the PI will provide safety information to Janssen Scientific Affairs on adverse events, special 
situations including pregnancies and product quality complaints as defined within this protocol. 
This Study has been designated as an interventional study.  As such, all adverse events for Janssen 
Study drugs regardless of causality and special situations excluding those from subjects not exposed to a 
Janssen Study drug and product quality complaints with or without an adverse event as described in this 
Protocol will be reported from the time a subject has signed and dated an Informed Consent Form (ICF) 
until completion of the subject’s last study-related procedure (which may include contact for follow-up 
safety).  Serious adverse events will be reported up to week-36 (16 weeks after the last dose of study 
drug). 
 
8.2. Definitions 
8.2.1. Adverse Event (AE) 
An adverse event is any untoward medical occurrence in a clinical study subject administered a medicinal 
(investigational or non-investigational) product. An adverse event does not necessarily have a causal 
relationship with the treatment. An adverse event can therefore be any unfavorable and unintended sign 

 
Page 22 of 34 
(including an abnormal finding), symptom, or disease temporally associated with the use of a medicinal 
(investigational or non- investigational) product, whether or not related to that medicinal (investigational or 
non-investigational) product. (Definition per International Conference on Harmonisation [ICH]) 
This includes any occurrence that is new in onset or aggravated in severity or frequency from the 
baseline condition, or abnormal results of diagnostic procedures, including laboratory test abnormalities. 
 
8.2.2. Serious Adverse Event (SAE) 
A serious adverse event based on ICH and EU Guidelines on Pharmacovigilance for Study drugs for 
Human Use is any untoward medical occurrence that at any dose: 
• 
Results in death. (Note: death for any reason will be reported as a serious adverse event.) 
• 
Is life-threatening. (The subject was at risk of death at the time of the event. It does not refer to an 
event that hypothetically might have caused death if it were more severe.) 
• 
Requires inpatient hospitalization or prolongation of existing hospitalization 
• 
Results in persistent or significant disability/incapacity 
• 
Congenital anomaly/birth defect 
• 
Suspected transmission of any infectious agent via a study drug  
• 
Serious Infections 
• 
Serious systemic hypersensitivity reactions 
• 
Suicidality 
• 
Cardiovascular events 
• 
Medically important* 
*Medical and scientific judgment should be exercised in deciding whether expedited reporting is also 
appropriate in other situations, such as important medical events that may not be immediately life 
threatening or result in death or hospitalization but may jeopardize the subject or may require intervention 
to prevent one of the other outcomes listed in the definition above. These should usually be considered 
serious. 
 
8.2.2.1. Hospitalization 
For reports of hospitalization, it is the sign, symptom or diagnosis which led to the hospitalization that is 
the serious event for which details must be provided. 
 
Any event requiring hospitalization or prolongation of hospitalization that occurs during the study must be 
reported as a serious adverse event, except hospitalizations for the following: 
• 
Hospitalizations not intended to treat an acute illness or adverse event (e.g., social reasons such 
as pending placement in long-term care facility) 
• 
Surgery or procedure planned before entry into the study. [Note: Hospitalizations that were 
planned before the start of data collection and where the underlying condition for which the 
hospitalization was planned has not worsened will not be considered serious adverse events. Any 
adverse event that results in a prolongation of the originally planned hospitalization is to be 
reported as a new serious adverse event.] 
• 
For convenience the investigator may choose to hospitalize the subject for the duration of the 
treatment period. 
 
8.2.2.2. Life-Threatening Conditions 
The cause of death of a subject in a study within 30 days of the last dose of study drug, whether or not 
the event is expected or associated with the study drug, is considered a serious adverse event. 
 

 
Page 23 of 34 
Important medical events that may not result in death, be life-threatening, or require hospitalization may 
be considered serious when, based upon appropriate medical judgment, they may jeopardize the 
participant and/or participant may require medical or surgical intervention to prevent one of the outcomes 
listed in this definition. Examples of such medical events include: 
• 
Allergic bronchospasm requiring intensive treatment in an emergency room or at home, 
• 
Blood dyscrasias or convulsions that do not result in in-patient hospitalization, or 
• 
The development of drug dependency or drug abuse.  
 
8.2.3. Adverse Events of Special Interest 
Adverse events of special interest are events that the COMPANY is actively monitoring as a result of 
a previously identified signal (even if non-serious).  No adverse events of special interest will be 
collected for this study.  
 
8.2.4. Individual Case Safety Report (ICSR) 
A valid ICSR will contain the four minimum criteria requred to meet regulatory reporting requirements. 
• 
an identifiable subject (but not disclosing personal information such as the subject’s name, initials 
or address) 
• 
an identifiable reporter (investigational site) 
• 
a Janssen study drug 
• 
an adverse event, outcome, or certain special situations 
 
The minimum information required will be: 
• 
suspected Janssen study drug (doses, indication) 
• 
date of therapy (start and end date, if available) 
• 
batch or lot number, if available 
• 
subject details (subject ID and country) 
• 
gender 
• 
age at AE onset 
• 
reporter ID 
• 
adverse event detail (AE verbatim in English), onset date, relatedness, causality, action taken, 
outcome, (if available) 
• 
Janssen protocol ID 
 
8.2.5. Product Quality Complaint (PQC) 
A product quality compliant is defined as any suspicion of a product defect related to a potential quality 
issue during manufacturing, packaging, release testing, stability monitoring, dose preparation, storage or 
distribution of the product, or delivery system.  Not all PQCs involve a subject.  Lot and batch numbers 
are of high significance and need to be collected whenever available. 
 
Examples of PQC include but not limited to: 
• 
Functional Problem: e.g., altered delivery rate in a controlled release product 
• 
Physical Defect:  e.g. abnormal odor, broken or crushed tablets/capsules 
• 
Potential Dosing Device Malfunction: e.g., autoinjector button not working, needle detaching from 
syringe 
• 
Suspected Contamination 

 
Page 24 of 34 
• 
Suspected Counterfeit 
 
8.2.6. Unanticipated Problems (UP) 
The Office for Human Research Protections (OHRP) considers UPs involving risks to participants or 
others to include, in general, any incident, experience, or outcome that meets all of the following criteria: 
• 
Unexpected in terms of nature, severity, or frequency given (a) the research procedures that are 
described in the Tremfya package insert; and (b) the characteristics of the participant population 
being studied; 
• 
Related or possibly related to participation in the research (“possibly related” means there is a 
reasonable possibility that the incident, experience, or outcome may have been caused by the 
procedures involved in the research); and 
• 
Suggests that the research places participants or others at a greater risk of harm (including 
physical, psychological, economic, or social harm) than previously known or recognized. 
This study will use the OHRP definition of UP. 
 
8.3. Adverse Event Assessment and Follow-Up 
The occurrence of an UP, AE or SAE may come to the attention of study personnel during study visits 
and interviews of a study participant presenting for medical care, or upon review by the PI. All AEs 
including local and systemic reactions not meeting the criteria for SAEs will be captured on the 
appropriate eCRF. Information to be collected includes event description, time of onset, clinician’s 
assessment of severity, seriousness, expectedness, relationship to study product (assessed only by 
those with the training and authority to make a diagnosis), and date of resolution/stabilization of the event. 
All AEs occurring while on study must be documented appropriately regardless of relationship. All AEs 
will be followed to adequate resolution. 
 
Any medical condition that is present at the time that the participant is screened will be considered as 
baseline and not reported as an AE. However, if the study participant’s condition deteriorates at any time 
during the study, it will be recorded as an AE.  
 
Changes in the severity of an AE will be documented to allow an assessment of the duration of the event 
at each level of severity to be performed. AEs characterized as intermittent require documentation of 
onset and duration of each episode. 
 
At each study visit, the Investigator will inquire about the occurrence of AE/SAEs since the last visit. 
Events will be followed for outcome information until resolution or stabilization.  
 
The Investigator will record all reportable events with start dates occurring any time after informed 
consent is obtained until the week-36 follow-up study visit. Any SAE that occurs after treatment with 
alternative therapy will be reported only if the Investigator or current treating physician has assessed the 
SAE as related to the study treatment.  
 
All serious adverse events that have not resolved by the end of the study, or that have not resolved upon 
discontinuation of the subject's participation in the study, will be followed until any of the following occurs: 
• 
The event resolves 
• 
The event stabilizes 
• 
The event returns to baseline, if a baseline value/status is available 
• 
The event can be attributed to agents other than the study drug or to factors unrelated to study 
conduct 

 
Page 25 of 34 
• 
It becomes unlikely that any additional information can be obtained (subject or health care 
practitioner refusal to provide additional information, lost to follow-up after demonstration of due 
diligence with follow-up efforts) 
All follow-up information for serious adverse events or AEs of special interest and special situations that 
are not resolved at the end of the study or by the time of patient withdrawal will be reported directly by the 
PI, within 24 hours becoming aware, to Janssen Scientific Affairs using Janssen Scientific Affairs’ 
Serious Adverse Event Report.  
 
All available clinical information relevant to the evaluation of a related SAE, Adverse Events of Special 
Interest, serious ADR or special situation is required. 
• 
The PI is responsible for ensuring that these cases are complete and if not are promptly followed-
up. A safety report is not considered complete until all clinical details needed to interpret the case 
are received. Reporting of follow-up information should follow the same timeline as initial reports. 
• 
Copies of any and all relevant extraordinary (not including routine initial or follow-up ICSR 
submission) correspondences with regulatory authorities and ethics committees regarding any 
and all serious adverse events, irrespective of association with the study drug, will be provided to 
Janssen Scientific Affairs within 24 hours of such report or correspondence being sent to 
applicable health authorities. 
 
  
8.4. Reporting Procedures 
All adverse events and special situations, whether serious or non-serious, related or not related, following 
exposure to the study drug will be documented by the investigator and recorded in the eCRF and in the 
subject’s source records. Investigators will record in the eCRF their opinion concerning the relationship of 
the adverse event to the study drug. All (serious and non-serious) adverse events reported for the study 
drug will be followed-up in accordance with clinical practice. 
8.4.1. OHSU IRB Reporting of Unanticipated Problems and Adverse Events 
Unanticipated Problems and AEs will be reported to OHSU IRB according to the policies, procedures and 
guidelines posted on the OHSU IRB web site. 
 
Events that must be reported by the Investigator to the IRB are detailed in the OHSU IRB Investigator 
Guidance: Prompt Reporting Requirements (HRP-801). At a minimum, events requiring reporting to the 
IRB include: 
• 
Any new or increased risk related to the research, including AEs or IND safety reports that require 
a change to the protocol or consent,  
• 
New FDA black box warning,  
• 
Publications identifying new risks,  
• 
Data Safety Monitoring Board/Committee letters recommending changes or discussing new risks 
• 
Unauthorized disclosure of confidential participant information 
 
8.4.2. Reporting to the Study Drug Manufacturer 
At a minimum, on a semi-annual basis and at the end of the Study, Janssen Scientific Affairs will provide 
to the PI, a listing of all SAEs reported to Janssen Scientific Affairs. The PI will review this listing and will 
resolve any discrepancies with the data provided byJanssen Scientific Affairs. 
 
 
 
The following methods will be used for transmission of safety information to Janssen Scientific Affairs: 

 
Page 26 of 34 
• 
Electronically via Janssen SECURE Email service IIS-BIO-VIRO-GCO@its.jnj.com                         
(preferred) 
• 
For business continuity purposes, if SECURE Email is non-functional: 
o 
Facsimile (fax) at 1-866-451-0371, receipt of which is evidenced in a successful fax 
transmission report 
o 
Telephone (if fax is non-functional). 
 
The PI will prepare a final report including a complete and full summary of all adverse events, special 
situations and pregnancy reports according to the timeframe outlined in the agreement. 
 
8.4.2.1 Safety Event Reporting to the Study Drug Manufacturer 
Safety events of interest for a Janssen study drug that require expediting reporting and/or safety 
evaluation include, but are not limited to: 
• 
Drug exposure during pregnancy (maternal and paternal) 
• 
Overdose of the study drug  
• 
Exposure to the study drug from breastfeeding  
• 
Suspected abuse/misuse of the study drug 
• 
Inadvertent or accidental exposure to the study drug  
• 
Any failure of expected pharmacological action (i.e., lack of effect) of the study drug 
• 
Medication error involving the study drug (with or without patient exposure to the study drug, e.g., 
name confusion) 
• 
Suspected transmission of any infectious agent via administration of the study drug 
• 
Unexpected therapeutic or clinical benefit from use of the study drug 
 
These safety events may not meet the definition of an adverse event; however, from a Janssen Scientific 
Affairs perspective, they are treated in the same manner as adverse events. Special situations should be 
recorded on the Adverse Event section of the eCRF. 
 
8.4.2.2 Non-Serious Adverse Event Reporting to the Study Drug Manufacturer 
All non-serious adverse events will be reported to Janssen Scientific Affairs annually and at the end of the 
study.  
 
8.4.2.3 Serious Adverse Event Reporting to the Study Drug Manufacturer 
Any SAE, Adverse Event of Special Interest or special situation that meets the criteria of a serious 
adverse event should be recorded on a Serious Adverse Event Report Form and be reported to Janssen 
Scientific Affairs within 24 hours of becoming aware of the event. 
 
8.4.2.4 PQC Reporting to the Study Drug Manufacturer 
A PQC may have an impact on the safety and efficacy of the product. Timely, accurate, and complete 
reporting and analysis of PQC information from studies are crucial for the protection of patients, 
investigators, and Janssen Scientific Affairs, and are mandated by regulatory agencies worldwide. 
Janssen Scientific Affairs has established procedures in conformity with regulatory requirements 
worldwide to ensure appropriate reporting of PQC information. Lot and/or Batch #s shall be collected or 
any reports failure of expected pharmacological action (i.e., lack of effect). The product should be 
quarantined immediately and if possible, take a picture. 
 

 
Page 27 of 34 
All initial PQCs involving the study drug will be reported to Janssen Scientific Affairs by the investigators 
within 24 hours after being made aware of the event. The Janssen contact will provide additional 
information/form to be completed. 
 
If the defect for a Janssen study drug under study is combined with either a serious adverse event or non-
serious adverse event, the PI must report the PQC to Janssen Scientific Affairs according to the serious 
adverse event reporting timelines.  A sample of the suspected product should be maintained for further 
investigation if requested by Janssen Scientific Affairs. 
 
8.4.3. Pregnancy 
All initial reports of pregnancy will be reported to Janssen Scientific Affairs by the PI within 24 hours of 
becoming aware of the event using the Serious Adverse Event Form. Abnormal pregnancy outcomes 
(e.g., spontaneous abortion, fetal death, stillbirth, congenital anomaly, ectopic pregnancy) are considered 
serious adverse events and must be reported using the Serious Adverse Event Form.  
 
Any subject who becomes pregnant during the study will be promptly withdrawn from the study and 
discontinue further study treatment. 
 
Because the effect of the Janssen study drug on sperm is unknown, pregnancies in partners of male 
subjects exposed to a Janssen study drug will be reported by the PI within 24 hours of their knowledge of 
the event using the Serious Adverse Event Form.  Depending on local legislation this may require prior 
consent of the partner. Follow-up information regarding the outcome of the pregnancy and any postnatal 
sequelae in the infant will be collected. 
 
8.5. Data Safety Monitoring Plan 
Subject safety will be monitored in an ongoing way throughout the study. Concomitant medications will be 
recorded at every study visit. Vital signs including blood pressure and pulse (measured a minimum of 5 
minutes after resting), and weight/BMI will be recorded at every study visit. Laboratory testing will be 
performed at the screening visit that will include screening for HIV, HVB, HCV, and tuberculosis as 
detailed in the exclusion criteria. Baseline, week-4, week-12, and week-24 laboratory testing will include 
complete blood counts with differential and a comprehensive metabolic panel. 
 
Subjects will be encouraged to report any potential problems at any time to qualified research staff or PI. 
The occurrence of adverse events will be assessed at each study visit and details including diagnosis, 
severity, possibility of relationship to the study drug, and action taken will be recorded in the eCRF.  
 
Data safety monitoring will involve real-time review of adverse events (AE), dropouts, complaints or 
breaches of confidentiality. The review of AEs will be performed by the PI (Dr. Greiling) in real time, with 
review by a co-Investigator if Dr. Greiling is unavailable.  The study will be halted if one or more 
unexpected AEs occur that are at least possibly related and determined to be moderate in severity or 
greater. 
 
Data collected during study visits will be entered into the REDCap database by study personnel and study 
subjects as outlined in the description of visits above. The PI, who has no financial conflict of interest with 
Janssen Scientific Affairs, will review the data at least monthly. 
 
The PI will provide all adverse events, both serious and non-serious, in report format to Janssen Scientific 
Affairs. However, in certain circumstances more frequent provision of safety data may be necessary, e.g. 
to fulfill a regulatory request, and as such the data shall be made available within a reasonable time frame 
at Janssen Scientific Affairs’ request. 

 
Page 28 of 34 
 
  
8.6. Potential Risks 
The risks associated with participation in this study include the use of guselkumab, the collection of a skin 
biopsy and blood sample, genetic testing, and loss of confidentiality. 
 
Risks of taking guselkumab that were identified as higher than the placebo group in clinical trials of 
subjects with psoriasis are outlined below. The full package insert is publicly available. 
• 
Guselkumab may increase the risk of infection. In clinical trials, use of guselkumab was 
associated with a higher rate of infection than the placebo group (23% versus 21%). Upper 
respiratory tract infections, gastroenteritis, tinea infections, and herpes simplex infections 
occurred more frequently in the guselkumab group than in the placebo group. The rate of serious 
infections was <1% and the same in the guselkumab and placebo groups. 
• 
The rate of developing neutralizing antibodies against guselkumab was 6% over one year of 
treatment. It is uncertain whether this may lead to a loss of efficacy of the study drug over time. 
• 
Injection site reactions including pain, erythema, bruising, and inflammation occurred in 4.5% of 
subjects who used guselkumab (versus 2.8% of the placebo group). 
• 
Subjects taking guselkumab had a slightly higher rate of developing headache (4.6% vs 3.3% of 
placebo), arthralgias (2.7% versus 2.1% of placebo), and diarrhea (1.6% versus 0.9% of 
placebo). 
• 
Elevated liver enzymes were reported more frequently in the guselkumab group (2.6%) than in 
the placebo group (1.9%). 
• 
Adverse reactions that occurred in <1% of the study drug group but at a higher rate than place 
include migraine, candida infections, and urticaria. 
The risk to the fetus or infant in pregnant or lactating women is unknown and this is an exclusion criterion 
from the study. Women who may become pregnant and men who may father a child are asked to use a 
reliable form of birth control. There have been no reports to date of harm and no negative effects in 
laboratory animals. There may be additional drug side effects that are not yet known. 
 
Risks of blood draw include discomfort, and a small chance of bleeding, bruising, infection, or fainting. 
 
Risks of biopsy include allergic reaction to the local anesthetic (1 in 10,000 risk), infection, bleeding, and 
scar. 
 
Risks of genetic testing include the loss of confidentiality that may affect the subject’s ability to obtain life 
insurance, disability insurance, or long-term care insurance. Even though there are certain genetic 
discrimination and confidentiality protections in both Oregon law and federal law, there is still a small 
chance that the subject could be harmed if a release occurred. 
 
Although medical information and medical photographs will be encoded and carefully protected, there is a 
small risk of loss of confidentiality and release of personal information. 
 
8.7. Participant Discontinuation of Study Drug or Withdrawal from the Study 
8.7.1. Participant Discontinuation of Study Drug 
A subject who discontinues study drug administration will not be automatically withdrawn from the study if 
they discontinue study drug administrations before the end of the study drug administration period. If a 
subject discontinues study drug before week-24, the week-24 study visit will be performed early if the 
subject is willing and able to participate. 
A subject's study drug must be discontinued if: 

 
Page 29 of 34 
• 
The investigator believes that for safety reasons or tolerability reasons (e.g., AE) it is in the best 
interest of the subject to discontinue study drug 
• 
The subject becomes pregnant or plans a pregnancy within the study period.  
• 
The subject (or the subject’s representative) withdraws consent for administration of study drug. 
• 
The subject develops a systemic opportunistic infection. 
• 
The subject is deemed ineligible according to the following TB screening criteria: 
o 
A diagnosis of active or latent TB is made. 
o 
A subject has symptoms suggestive of active TB based on follow-up assessment 
questions and physical examination, or has had recent close contact with a person with 
active TB, and cannot or will not continue to undergo additional evaluation. 
• 
The subject develops an allergic reaction resulting in bronchospasm with wheezing or dyspnea 
that requires ventilatory support. In general, discontinuation of study drug administration must be 
considered for subjects who develop a severe allergic reaction. 
• 
The subject has a reaction resulting in myalgia or arthralgia with fever or rash (suggestive of 
serum sickness and not representative of signs and symptoms of other recognized clinical 
syndromes) occurring 1 to 14 days after an injection of study drug. These may be accompanied 
by other events including pruritus, facial, hand, or lip edema, dysphagia, urticaria, sore throat, and 
headache.  
• 
The subject has a malignancy, not including cutaneous squamous cell skin cancer < 2 cm in 
diameter or basal cell skin cancers that are adequately treated with no evidence of residual 
disease. 
• 
The subject has any severe infection (NCI-CTCAE Grade 3 or higher) that is considered related 
to the study drug by the investigator. 
Discontinuation of study drug will also be strongly considered if the subject develops a serious infection, 
including but not limited to sepsis or pneumonia, or any other serious AE at the direction of the 
investigator or the subject’s request..  
 
If a subject suffers any injury and/or damage from this research project through the fault of the Oregon 
Health & Science University, its officers or employees, they will have the right to bring legal action against 
the University to recover the damage done subject to the limitation and conditions of the Oregon Tort 
Claims Act.  
 
8.7.2 Subject Withdrawal from the Study 
A subject will be withdrawn from the study for any of the following reasons: 
• 
Lost to follow-up 
• 
Withdrawal of consent 
• 
Death 
• 
Investigator decision (e.g., participating in any other clinical study with an investigational agent) 
When a subject withdraws before completing the study, the reason for withdrawal will be documented in 
the eCRF. Additional subjects may be enrolled to ensure the protocol-specified number of participants 
complete the study. If a subject withdraws after the first dose of study drug and before week-24, the 
week-24 study visit will be performed early if the subject is willing and able to participate. If a subject 
withdraws after week-24, the week-36 assessment will be performed early if the subject is willing and able 
to participate. If the reason for withdrawal is withdrawal of consent then no additional assessments will be 
performed. 
 

 
Page 30 of 34 
9. DATA HANDLING & MANAGEMENT RESPONSIBILITIES 
9.1. Participant & Data Confidentiality 
The information obtained during the conduct of this clinical study is confidential, and unless otherwise 
noted, disclosure to third parties is prohibited. Information contained within this study will be maintained in 
accordance with applicable laws protecting participant privacy, including the provisions of the Health 
Insurance Portability and Accountability Act (HIPAA). 
Participant confidentiality is strictly held in trust by the participating Investigator(s) and study team. This 
confidentiality is extended to cover testing of biological samples in addition to the clinical information 
relating to participants. Therefore, the study protocol, documentation, data, and all other information 
generated will be held in strict confidence.  
Representatives of the IRB or manufacturer supplying study product may inspect all documents and 
records required to be maintained by the Investigator, including but not limited to, medical records (office, 
clinic, or hospital) and pharmacy records for the participants in this study. The clinical study site will permit 
access to such records.  
Upon enrollment, participants will be assigned a code that will be used instead of their name, medical 
record number or other personally identifying information. Electronic files for data analysis will contain 
only the participant code. Codes will not contain any part of the 18 HIPAA identifiers (e.g., initials, DOB, 
MRN). The key associating the codes and the participants’ personally identifying information will be 
restricted to the Investigator and study staff. The key will be kept secure on a restricted OHSU network 
drive in a limited access folder. 
The study participant’s contact information will be securely stored at each clinical site for internal use 
during the study. At the end of the study, all records will continue to be kept in a secure location for as 
long a period as dictated by local IRB and institutional regulations. 
 
9.2. Data Collection & Storage: Privacy, Confidentiality & 
Security  
9.2.1. Data Collection 
Data collection is the responsibility of the clinical trial staff at the site under the supervision of the site 
Investigator. The Investigator is responsible for ensuring the accuracy, completeness, legibility, and 
timeliness of the data reported. Standard institutional practices will be followed as described in the 
OHSU’s Information Security Directives to maintain the confidentiality and security of data collected in this 
study. Study staff will be trained with regard to these procedures. 
9.2.2. Data Confidentiality 
Loss of participant confidentiality is a risk of participation. Efforts will be made to keep study participant 
identities confidential except as required by law. Participants’ samples will be identified by code only. 
Specifically, each consenting participant will be assigned a unique coded identifier consisting of numbers. 
This identifier will be associated with the participant throughout the duration of their participation in the 
trial. The coded identifier will also be used to identify any participant specific samples. 
9.2.3. Data Storage 
Basic accrual tracking information (demographic, consent, visit information) will be captured in OHSU’s 
electronic clinical information research system (eCRIS), hosted on OHSU secure servers and managed 
by OHSU’s information technology group at their data center in downtown Portland, Oregon. Any 
additional printed documents containing participant identifiers, such as those from the medical record to 
confirm eligibility, will be filed in binders and kept in a locked, secure location. 
Study outcome data will be captured in electronic case report forms (eCRFs) using an electronic data 
capture (EDC) system (REDCap) on OHSU secure servers, which facilitates information being stored in a 

 
Page 31 of 34 
unified format and location. To further preserve confidentiality, PHI in the EDC system will be limited to 
just birth date and visit dates, email (to be used for REDCap surveys) and city of residence. The REDCap 
system is password protected and encrypted with role-based security, and administered by designated 
informatics staff within OHSU. All users of the database are assigned a unique ID, username, and 
password and must complete training appropriate to their role before they are authorized to enter, access, 
and store data in the database.  
Data from correlative studies will be entered into the EDC system by study personnel at OHSU. All other 
electronic data extracts will be stored only on OHSU computers and restricted drives, limited only to study 
investigators and staff with authorization to access the data.  
Individual participant medical information obtained as a result of this study is considered confidential and 
disclosure to third parties is prohibited. Research materials obtained specifically for the purposes of this 
study will include biopsies for mechanistic studies and blood tests for CBC, CMP, HIV, HBV, HCV, 
tuberculosis, and measuring levels of inflammation. Saliva will be collected for genetic testing. All 
samples used for mechanistic testing will be coded and blinded to the investigator. Data regarding 
medical history and symptom questionnaires will be obtained during the course of this study. Absolute 
confidentiality will be maintained. Only the principal investigator and study coordinator will have access to 
identifiable private information to facilitate subject recruitment and subject reimbursement. All data not 
stored in REDCap are stored in locked compartments and are not released without consent of the 
participants. If data are used in scientific presentations or publications, individuals are never identified. 
Medical information gathered during the course of the study may be given to the participant’s medical 
team and all appropriate medical personnel responsible for the participant’s welfare. 
9.2.4. Data Recording in the Electronic Medical Record (Epic) 
An Epic research encounter will be created for each in-person visit or at the time of any SAE. A brief 
S.O.A.P. note that includes study drug administration will be entered into Epic to facilitate communication 
with the subject’s other healthcare providers. 
 
9.2.5. Photography 
Photographs of the skin involvement in each subject will be captured as digital files, coded with the 
subject’s study number and visit number, and kept on the cloud file storage solution provided by Box.com. 
For purposes of publication or education (i.e., viewing by anyone other than study personnel), any 
identifying features such as the eyes, jewelry, or a tattoo will be digitally blocked out of the photo. 
 
9.2.6. Telemedicine Platform 
The study will use a HIPAA-compliant videoconferencing platform (Nexus/Cisco Meeting). A one-time-use 
unique hyperlink will be sent to the study subject for each visit, via OHSU MyChart or email. No protected 
health information will be stored unless it is deliberately captured and downloaded by one of the 
participants in the videoconference session. For convenience, we will choose a platform that works 
natively within a browser with the use of static plugins and that allows patients to access the study “room” 
via a specific URL without need for preregistration. The platform will use standard audio/video capabilities 
and will allow access through mobile devices.     
 
10. ETHICS/PROTECTION OF PARTICIPANTS 
10.1. Ethical Standard 
The Investigator will ensure that this study is conducted in adherence with the protocol and International 
Conference on Harmonization Good Clinical Practice E6 (ICH-GCP).  
 

 
Page 32 of 34 
10.2. Institutional Review Board 
The protocol, informed consent form, recruitment materials, and all participant materials will be submitted 
to the IRB for review and approval. Approval of both the protocol and the consent form must be obtained 
before any participant is enrolled. Any amendment to the protocol will require review and approval by the 
IRB before the changes are implemented to the study. All changes to the consent form will be IRB 
approved; a determination will be made regarding whether previously consented participants need to be 
re-consented. 
 
10.3. Informed Consent 
Written informed consent will be obtained from all participants participating in this trial, as stated in the 
Informed Consent section of 21 CFR Part 50. Documentation of the consent process and a copy of the 
signed consent shall be maintained in the participant’s medical record.  
 
10.4. Consent Procedures and Documentation 
Informed consent is a process that is initiated prior to the individual’s agreement to participate in the study 
and continues throughout the individual’s study participation. Extensive discussion of risks and possible 
benefits of participation will be provided to the participants and their families as appropriate. Consent 
forms will be IRB-approved and the participant will be asked to read and review the document. The 
Investigator will explain the research study to the participant and answer any questions that may arise. All 
participants will receive a verbal explanation in terms suited to their comprehension of the purposes, 
procedures, and potential risks/benefits of the study, alternatives to participation, and of their rights as 
research participants. Participants will have the opportunity to carefully review the written consent form 
and ask questions prior to signing. The participants should have the opportunity to discuss the study with 
their surrogates or think about it prior to agreeing to participate. The participant will sign the informed 
consent document prior to any procedures being done specifically for the study. The participants may 
withdraw consent at any time throughout the course of the trial. A copy of the informed consent document 
will be given to the participants for their records. The rights and welfare of the participants will be 
protected by emphasizing to them that the quality of their medical care will not be adversely affected if 
they decline to participate in this study. 
 
10.5. Protocol Review 
The protocol and informed consent form for this study must be reviewed and approved in writing by the 
OHSU IRB prior to any participant being consented on this study.   
 
10.6. Changes to Protocol  
Any modification of this protocol must be documented in the form of a protocol revision or amendment 
submitted by the Investigator and reviewed and approved by Janssen Scientific Affairs, then approved by 
the IRB, before the revision or amendment may be implemented.  The only circumstance in which the 
amendment may be initiated without regulatory approval is for a change necessary to eliminate an 
apparent and immediate hazard to the participant.  In that event, the Investigator must notify the IRB 
within 5 business days after the implementation.  
 
11. PUBLICATION AND DISSEMINATION POLICY 
The study results will be published in a peer-reviewed journal.  Participants will not be identified in any 
publications. 
This protocol will be registered in the ClinicalTrials.gov website. This registration will occur after IRB 
approval. 
 

 
Page 33 of 34 
12. REFERENCES 
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N. A. Ross et al., Epidemiologic, Clinicopathologic, Diagnostic, and 
Management Challenges of Pityriasis Rubra Pilaris: A Case Series of 
100 Patients. JAMA dermatology 152, 670-675 (2016). 
2. 
L. Feldmeyer et al., Interleukin 23-Helper T Cell 17 Axis as a Treatment 
Target for Pityriasis Rubra Pilaris. JAMA dermatology,  (2017). 
3. 
D. Fuchs-Telem et al., Familial pityriasis rubra pilaris is caused by 
mutations in CARD14. American journal of human genetics 91, 163-170 
(2012). 
4. 
C. T. Jordan et al., PSORS2 is due to mutations in CARD14. American 
journal of human genetics 90, 784-795 (2012). 
5. 
O. Eytan et al., Increased epidermal expression and absence of 
mutations in CARD14 in a series of patients with sporadic pityriasis rubra 
pilaris. The British journal of dermatology 170, 1196-1198 (2014). 
6. 
M. A. Lowes, C. B. Russell, D. A. Martin, J. E. Towne, J. G. Krueger, The 
IL-23/T17 pathogenic axis in psoriasis is amplified by keratinocyte 
responses. Trends in immunology 34, 174-181 (2013). 
7. 
A. Blauvelt et al., Efficacy and safety of guselkumab, an anti-interleukin-
23 monoclonal antibody, compared with adalimumab for the continuous 
treatment of patients with moderate to severe psoriasis: Results from the 
phase III, double-blinded, placebo- and active comparator-controlled 
VOYAGE 1 trial. Journal of the American Academy of Dermatology 76, 
405-417 (2017). 
8. 
K. Reich et al., Efficacy and safety of guselkumab, an anti-interleukin-23 
monoclonal antibody, compared with adalimumab for the treatment of 
patients with moderate to severe psoriasis with randomized withdrawal 
and retreatment: Results from the phase III, double-blind, placebo- and 
active comparator-controlled VOYAGE 2 trial. Journal of the American 
Academy of Dermatology 76, 418-431 (2017). 
9. 
M. Napolitano et al., Ustekinumab treatment of pityriasis rubra pilaris: A 
report of five cases. The Journal of dermatology 45, 202-206 (2018). 
10. 
N. J. Maloney, L. D. Hisaw, S. Worswick, Refractory pityriasis rubra 
pilaris treated with etanercept, adalimumab, or ustekinumab: A 
retrospective investigation. Dermatologic therapy 30,  (2017). 
11. 
S. M. Lwin et al., Beneficial effect of ustekinumab in familial pityriasis 
rubra pilaris with a new missense mutation in CARD14. The British 
journal of dermatology,  (2017). 
12. 
A. Paganelli, S. Ciardo, G. Odorici, G. Pellacani, A. Conti, Efficacy of 
ustekinumab after failure of infliximab CT-P13 in a HLA-Cw6-positive 
patient affected by pityriasis rubra pilaris: monitoring with reflectance 
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