Clinical trial NCT03793439
Conditions: Sarcoidosis, Pulmonary Sarcoidosis Lung Sarcoidosis
Keywords: Sarcoidosis Corticosteroid dependent sarcoidosis
Sponsor: Oregon Health and Science University
Start date: 2019-05-15; Completion date: 2021-06-24
Enrollment: 5 participants; Allocation: NA; Intervention model: SINGLE_GROUP

Brief summary:
This is a pilot study to determine whether further research is warranted to assess whether tofacitinib is an effective steroid sparing treatment for pulmonary sarcoidosis. The primary endpoint for this study is a 50% or greater reduction in corticosteroid requirement.

Detailed description:
Primary Objectives:

Objective 1: Test the hypothesis that the addition of tofacitinib will allow patients with sarcoidosis to have 50% or greater reduction in their corticosteroid requirement without a significant decrease in pulmonary function testing, and with a similar quality of life as measured by a validated questionnaire (1).

Objective 2: Test the hypothesis that the addition of tofacitinib will result in significantly decreased expression of signal transducer and activator of transcription (STAT)-1 dependent gene expression.

Outline:

This is a 16-week open-label, interventional, proof of concept, hypothesis-generating study. All subjects will receive Tofacitinib 5mg twice daily for 16 weeks. After four weeks on Tofacitinib, the corticosteroid will be tapered per a pre-defined protocol; once a reduction of 50% has been achieved, any further taper will be per physician discretion. After 16 weeks, subjects who meet the primary end-point will be permitted an optional one year open-label extension.

Arms: arm 1: All subjects will receive tofacitinib 5mg twice daily from week 0 to week 16, and a corticosteroid taper starting at week 16. Participants also undergo spirometry, RNA sequence testing, and laboratory evaluations.
Interventions: intervention 1: Tofacitinib 5mg Oral Tablet [Xeljanz] 16 week trial intervention 2: Spirometry intervention 3: RNA Sequencing intervention 4: Laboratory testing intervention 5: Corticosteroid intervention 6: Tofacitinib 5mg [Xeljanz] 1 year open-label extension
Intervention descriptions: intervention 1: Tofacitinib 5mg oral table twice daily for 16 weeks intervention 2: Spirometry testing at baseline, week 4, week 8, week 12, and week 16 intervention 3: RNA sequencing test at baseline and week 16 intervention 4: Laboratory testing at baseline and weeks 2, 4, 8, 12 and 16 intervention 5: Taper corticosteroids starting at week 4 intervention 6: After 16 weeks, subjects who meet the primary end-point will be permitted an optional one year open-label extension.

Protocol documents:
Prot_SAP_000.pdf: 
 
Page 1 of 13 
Revised: 7/15/2019 
1. Protocol Title 
Tofacitinib Hypothesis-generating, Pilot Study for Corticosteroid-Dependent Sarcoidosis 
 
2. Objectives 
This study will enroll 5 subjects with corticosteroid dependent pulmonary sarcoidosis. 
 
The purpose of this pilot study is to determine if further research is warranted to assess if tofacitinib is an 
effective steroid sparing treatment for pulmonary sarcoidosis. The primary end point for this study is a 50% or 
greater reduction in corticosteroid requirement. We hypothesize that 60% of the patients will have a 50% or 
greater reduction in their steroid requirement without a significant decrease in pulmonary function testing and 
with a similar quality of life. We further hypothesize that STAT-1 dependent gene expression studies will show 
a significant decrease after tofacitinib treatment.  
 
Objective 1: Test the hypothesis that the addition of tofacitinib will allow patients with sarcoidosis to have 50% 
or greater reduction in their corticosteroid requirement without a significant decrease in pulmonary function 
testing, and with a similar quality of life as measured by a validated questionnaire (1).   
 
Objective 2: Test the hypothesis that the addition of tofacitinib will result in significantly decreased expression 
of STAT-1 dependent gene expression.  
 
3. Background 
Sarcoidosis is an inflammatory disorder of unclear etiology with a prevalence that has been estimated to be as 
high as 300/100,000. (2) The mainstay of treatment is oral corticosteroids. However, this therapy has 
numerous side effects and has been linked to increased relapses and poor quality of life, particularly at high 
doses. (3, 4)  Furthermore, there is a subset of patients with persistent disease despite corticosteroid 
treatment. This has led to multiple trials evaluating various immunomodulatory agents for steroid sparing 
treatment in sarcoidosis.  Our group and others have previously shown upregulation of the STAT1 pathway 
including the downstream cytokines in tissue, whole blood and lymph nodes of patients with sarcoidosis. (5-7) 
 
Tofacitinib is a janus kinase (JAK) inhibitor with a predominant effect on JAK1 and JAK3 which in turn decreases 
STAT1 activation. (8)  Tofacitinib is currently FDA approved for treatment of psoriatic arthritis and rheumatoid 
arthritis and has recently shown superiority when compared to methotrexate as front-line therapy. It is similar 
to TNF-alpha inhibition when added as an adjunctive therapy. (9, 10)  Other trials have shown efficacy in a 
number of other inflammatory disorders including ulcerative colitis. (11, 12)  
 
Given the need for improved therapeutics, and the role of STAT1 in the pathogenesis of sarcoidosis, we 
propose a study examining the reduction in steroid use in patients who are dependent on a stable dose 
(defined as same dose for 4 weeks) of 15 mg/day to 30 mg/day of prednisone or its equivalent to control their 
disease. A recent query of OHSU’s electronic medical record identified over 800 adult patients with sarcoidosis, 
generally 10-30% of patients with sarcoid require chronic treatment for an expected population of 80-240 
eligible patients in our system. (13) We intend to enroll 5 patients with pulmonary sarcoidosis, age 18 or older 
of either gender in an open label proof-of-concept study design and treat with 5 mg of Tofacitinib BID for 16 
weeks as a steroid sparing agent. We hypothesize that 60% of patients will have a 50% or greater reduction in 
their steroid requirements without a significant decrease (defined as less than 15% reduction in forced 
expiratory volume in 1 second (FEV1), forced vital capacity (FVC) or diffusing capacity for carbon monoxide 
(DLCO)) in pulmonary function testing and a similar quality of life as measured by the St. George’s Respiratory 
Questionnaire (SGRQ).(1)  A clinically significant change is defined as >4 units in the SGRQ. (14) Patients who 
respond by these criteria will be allowed to continue on the study drug for additional time up to 52 weeks. This 
small study will serve as a pilot and safety study; larger trials will ultimately be necessary to validate these 
results. 


 
Page 2 of 13 
Revised: 7/15/2019 
 
 
4. Study Design 
This is a 16-week open-label, interventional, proof of concept, hypothesis-generating study. This study will 
enroll five subjects over the course of two years. Patients recruited for this study must have histologically 
proven sarcoidosis, evidence of pulmonary disease on chest imaging, and an FVC >50%.  They must require a 
stable dose (defined by 4 weeks or more) of prednisone at least 15 mg/day or equivalent corticosteroid prior 
to enrollment in the study. The prednisone dose cannot exceed 30 mg/day.  The above criteria should insure 
that this is a relatively uniform group of subjects. All subjects will receive Tofacitinib 5mg twice daily for 16 
weeks. After four weeks on Tofacitinib, the corticosteroid will be tapered per a pre-defined protocol (attached); 
once a reduction of 50% has been achieved, any further taper will be per physician discretion. The schedule 
for corticosteroid taper is based on 30 plus years of experience and customary practice at the Oregon Health 
& Science University. The experience of the investigators has consistently demonstrated an inability to taper 
corticosteroid dosage in many patients with sarcoidosis unless an effective, alternative immunosuppressive 
has been added to the patient’s regimen. Furthermore, higher doses of corticosteroids have consistently been 
correlated with the risk of infection. (15) Throughout the trial we will be monitoring blood counts, liver function 
tests, lipids, renal function, and pulmonary function testing.  We will also evaluate STAT-1 activity through 
measurement of CXCL10 levels and RNA-Seq measurement of selected transcripts (see below) before and after 
Tofacitinib therapy. The primary endpoint is a 50% or greater reduction in steroids with no significant decrease 
in pulmonary function or quality of life. After 16 weeks, subjects who meet the primary end-point will be 
permitted an optional one year open-label extension.   
 
5. Study Population 
 
a. Number of Subjects 
Five subjects with histologically proven sarcoidosis with pulmonary involvement and corticosteroid 
dependence will be enrolled in this trial.  
 
b. Inclusion and Exclusion Criteria 
Inclusion Criteria: Subjects age 18 and older of either sex will be included. Subjects must meet the WASOG 
definition of pulmonary sarcoidosis. (16) They must have histologically proven sarcoidosis, evidence of 
Pulmonary sarcoidosis on chest radiograph (Stage I, II, III or IV) (17), an FVC >50%, and can have concomitant 
extrapulmonary disease. Subjects must require 15 mg to 30 mg/day prednisone or equivalent corticosteroid 
to control their sarcoidosis. Dose of prednisone must be stable for 4 weeks prior to enrollment in the study. 
Subjects may be taking methotrexate, but may not be taking other immunosuppressive or immunomodulatory 
treatments (see below).  
 
Exclusion Criteria:  
• 
Patients taking immunomodulatory medications other than methotrexate in the two months prior to the 
study period will not be included. This includes but is not limited to azathioprine, cyclophosphamide, 
leflunomide, mycophenolate mofetil, cyclosporine, tacrolimus, and biologic medications.  
• 
Patients requiring >30 mg/day prednisone or its equivalent.  
• 
Pregnant or lactating women will not be included. 
• 
Subjects with the following blood dyscrasias will not be included: 
o Hemoglobin <9g/dL or Hematocrit <30% 
o White blood cell count <3.0 K/cu mm 
o Absolute neutrophil count <1.2 K/cu mm 
o Platelet count <100 K/cu mm 
• 
Subjects with an estimated GFR ≤40 ml/min 
• 
Subjects with a total bilirubin, AST, or ALT more than 1.5 times the upper limit of normal at screening.  


 
Page 3 of 13 
Revised: 7/15/2019 
• 
Severe, progressive, or uncontrolled chronic liver disease including fibrosis, cirrhosis, or recent or active 
hepatitis.  
• 
History of any lymphoproliferative disorder such as Epstein Barr virus (EBV) related lymphoproliferative 
disorder, history of lymphoma, leukemia, or signs and symptoms suggest of current lymphatic disease. 
• 
Current malignancy or history of malignancy, with the exception of adequately treated or excised non-
metastatic basal cell or squamous cell cancer of the skin, or cervical carcinoma in situ.  
• 
Have or have had an opportunistic infection (e.g., herpes zoster [shingles], cytomegalovirus, Pneumocystis 
carinii, aspergillosis and aspergilloma, histoplasmosis, or mycobacteria other than TB) within 6 months prior 
to screening. 
• 
Have a known infection with human immunodeficiency virus (HIV) 
• 
Have current signs and symptoms of systemic lupus erythematosus, or severe, progressive, or uncontrolled 
renal, hepatic, hematologic, endocrine, pulmonary, cardiac (New York Heart Association class III or IV), 
neurologic, or cerebral diseases (with the exception of sarcoidosis). 
 
Screening: Subjects will be recruited through the Oregon Health and Science University (OHSU) pulmonary, 
ophthalmology, rheumatology, and internal medicine clinics in addition to local community pulmonology 
clinics by referral. Review of OHSU medical records and Epic query will also be used to identify potential 
subjects. Pre-screening will take place first through chart review and then telephone interview. Subjects who 
are eligible for the trial after pre-screening will then be screened in person. At the screening visit subjects will 
receive informed consent as below. Health history and medications will be reviewed. Blood and/or urine 
samples will be collected, and pulmonary function testing will be done as per the baseline visit plan detailed 
below. Data from subjects who fail screening will be destroyed at the end of the study.  
 
c. Vulnerable Populations 
This study will not include children, pregnant women, neonates, prisoners, or decisionally impaired adults.  
 
d. Setting 
Subjects will be consented and samples will be drawn at the OHSU specialty clinics on the 3rd and 4th floors of 
the Physician’s Pavilion. Spirometry will be performed in the pulmonary function lab in Multnomah Pavilion or 
Physician’s Pavilion. Samples will be stored in a central repository as part of Dr. Jim Rosenbaum’s laboratory.  
 
RNA extraction and gene expression analyses will be performed by the OHSU Gene Profiling Shared Resource 
and the Massively Parallel Sequencing Shared Resource under the guidance of Dr. Christina Harrington.  
 
e. Recruitment Methods 
Subjects will be recruited through the OHSU rheumatology, pulmonary, ophthalmology, and internal medicine 
clinics. Subjects referred from outside clinicians/scientists or self-referral will be allowed, but is expected to 
account for a minority of subjects. Postings on internet support groups for people living with sarcoidosis will 
also be used for recruitment. An Epic and medical record query will be used to identify potential subjects. 
Subjects will be contacted by phone. A copy of the phone script is attached. 
  
f. Consent Process 
The consent process will take place in the OHSU pulmonary clinic or other appropriate OHSU clinic prior to any 
study procedures.  Dr. Marcia Friedman, Dr. Janelle Stevens, or a member of their research team will be 
responsible for discussing the study and consent documents with study participants.  Ample time will be 
provided to ask and answer questions.  It will be emphasized that participation is entirely voluntary and that 
this will not affect ongoing clinical care.  If needed, an interpreter will be provided for the consent process and 
subsequent study visits.  There are no current plans to provide a translated consent. Written consent will be 
verified by study staff prior to any research procedures.  The signed form will be kept in study binders. 
 


 
Page 4 of 13 
Revised: 7/15/2019 
6. Procedures Involved 
• 
Blood draw: A blood draw will be done at baseline and at weeks 2, 4, 8, 12, 16 (end of treatment). We 
estimate that the blood draw at baseline and week 16 will be approximately 15ml. We estimate that the 
blood draw at weeks 2, 4, 8, and 12 will be approximately 7.5ml.  
• 
Urinalysis: Women will have a baseline urine pregnancy test. 
• 
RNA Seq: at baseline and week 16 blood will be drawn for blood levels of CXCL10 and RNA-Seq to assess 
change in STAT1-dependent gene expression using a known STAT1 pathway analysis. This is genetic 
testing. Consent will be obtained using the attached consent from.  We have chosen RNA-Seq because this 
is a discovery protocol and because we have expertise in the implementation of this technology with two 
funded, NIH RO1 proposals dependent on it. However, because this study is based on just 5 paired samples 
and we wish to minimize multiple statistical comparisons, we will perform a paired T test on 14 transcripts 
which we have previously shown to be up regulated in the blood of patients with sarcoidosis and which 
are downstream of STAT-1 (including STAT-1 itself).7 These transcripts are A2M, WARS, CCND1, STAT-1, 
MT-1, TRH, CD64, IRF1, PSMB9, MHC2TA, OPRM1, Pim-1, CXCL9, and CDKN1A. The thousands of additional 
transcripts detected by RNA Seq will be analyzed, but in all cases, changes in expression will be considered 
as a preliminary observation which requires validation. 
• 
Pulmonary function testing: At baseline and at the end of treatment (week 16), we will measure 
spirometry with DLCO. At weeks 4, 8, and 12 we will measure spirometry only.  
• 
Study drug: Tofacitinib 5 mg twice daily will be given from week 0 to week 16 to treat sarcoidosis. 
Tofacitinib is FDA approved for the treatment of rheumatoid arthritis but is not FDA approved for the 
treatment of sarcoidosis.  
• 
Questionnaires: At each visit, quality of life will be measured with the Saint George Respiratory 
Questionnaire (SGRQ), and assessment of involved organ systems will be measured via the WASOG 
sarcoidosis organ assessment instrument. 
• 
Subjects should have had a chest x-ray within one year of enrollment and will have a repeat chest x-ray 
after the 16 weeks of treatment.  
 
Procedures 
Basel
ine 
 
Week 
0* 
We
ek 2 
Wee
k 4 
We
ek 8 
Week 
12 
Week 
16 
Informed Consent 
X 
 
 
 
 
 
 
Screening 
labs**: 
Quantiferon, HIV, HBsAg, 
HBcAB, HCV ab,  
X 
 
 
 
 
 
 
Urine 
pregnancy 
test 
(women only) 
X 
 
 
 
 
 
 
Start study drug 
 
X 
 
 
 
 
 
End study drug 
 
 
 
 
 
 
X 
Start steroid taper 
 
 
 
X 
 
 
 
Physical Exam 
X 
 
X 
X 
X 
X 
X 
Spirometry with DLCO 
X*** 
 
 
 
 
 
X 
Spirometry 
 
 
 
X 
X 
X 
 
Labs: 
CBC, 
LFT, 
Lipid 
measurement, BMP.  
X*** 
 
X 
X 
X 
X 
X 
Labs: CRP 
X 
 
 
 
 
 
X 
Labs: CXCL10 level 
X 
 
 
 
 
 
X 
RNA Seq  
X 
 
 
 
 
 
X 
Questionnaires: 
SGRQ, 
WASOG 
X 
 
X 
X 
X 
X 
X 


 
Page 5 of 13 
Revised: 7/15/2019 
Adverse event reporting 
 
 
X 
X 
X 
X 
X 
Medication Reconciliation  
 
 
X 
X 
X 
X 
X 
Chest Xray 
X** 
 
 
 
 
 
X 
* There should be no more than 4 weeks between baseline and start of study drug (week 0) 
**If screening labs or Chest Xray have been done within the past one year, they do not need to be repeated 
here. 
***If these tests have been done within 4 weeks of baseline visit, they do not need to be repeated. HIV: 
human immunodeficiency virus, HBsAG: hepatitis B surface antigen, HBcAB: hepatitis B core antibody, HCV: 
hepatitis C virus, PFT: pulmonary function testing, CBC: complete blood count, LFT: liver function test, BMP: 
basic metabolic panel, CPR: C-reactive protein, SCGQ: Saint George Respiratory Questionnaire  
 
All of the above procedures, testing, and study drug will be billed to the study. Patients and their insurance 
will not be billed for any of the above procedures during the 16 week study. Patients continuing on to long-
term follow up may have laboratory testing billed to insurance as below under “Long term follow-up”. 
 
Long term follow-up: Patients who do not meet the primary end point will not be followed long term. Patients 
who meet the primary endpoint will be continued on study drug for up to one year. During that one year they 
will be followed with labs (CBC, BMP, liver function panel, lipid panel) every two months, and the above 
questionnaires and adverse event reporting by phone every three months. A clinic visit will be required every 
6 months. Labs done during the one-year extension period will be billed to the patient’s insurance; study drug 
will be covered by Pfizer.   
 
Voluntary withdrawal from the study: Subjects may withdraw from the intervention (study drug Tofacitinib) 
at any time. For subjects who choose to withdraw from the intervention we will collect the following data two 
weeks after withdrawal from the intervention: CBC, lipid measurement, LFT, BMP, adverse event reporting.  
 
Treatment with Tofacitanib to be discontinued and the patient withdrawn from this study for: 
• 
Serious infections (those requiring parenteral antimicrobial therapy or hospitalization) 
• 
A decline in glomerular filtration rate of 33% or greater 
• 
An increase in liver function tests (LFTs) >2.5x the upper limit of normal 
• 
Neutrophil counts <1000 neutrophils/mm3 
• 
Platelet counts <100,000 platelets/mm3 
• 
Any single hemoglobin value <8.0 g/dL or one that drops ≥2 gm/dL below baseline 
• 
Other serious or severe AEs that the investigator believes are sufficient to discontinue the medication 
• 
Extrapulmonary disease such as uveitis with substantial worsening despite tofacitinib therapy as 
determined by another health care provider 
 
7. Data and Specimens 
a. Handling of Data and Specimens 
Clinical case report forms and signed consent forms will be kept on paper in locked filing cabinets in restricted 
access offices at OHSU. Electronic data is stored on restricted drives on the OHSU network through encrypted 
computers, and using OCTRI’s installation of REDCap.  Samples will be stored in Dr. Jim Rosenbaum’s laboratory 
on the second floor of the Biomedical Research Building. Dr. Friedman, Dr. Stevens or their study staff will 
verify that consent has been given to participate in genetic research prior to collecting any samples for genetic 
studies (see study consent). The PAXGene Blood tubes will be transported by the study coordinator to the 
Gene Profiling Shared Resource in Richard Jones Hall where the RNA extraction will be performed.  Case report 
forms will be kept for three years after study completion and then destroyed. After three years, electronic data 
and samples will be de-identified and encrypted and stored in a repository as discussed below under “Data and 


 
Page 6 of 13 
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Specimen Banking”. Dr. Friedman or Dr. Stevens will be responsible for receipt and transmission of data and 
specimens.  
 
Data for this project will be stored in OCTRI's installation of REDCap, a highly secure and robust web-based 
research data collection and management system. 
  
Features of REDCap that protect participants' privacy and data security include: 
·        Physical Security: OCTRI's REDCap software is housed on servers located in ITG's Advanced Computing 
Center providing locked physical security 
·        Electronic Security: The REDCap servers are housed behind both the OHSU firewall and a second ACC 
firewall.  All web-based data transmissions are encrypted with industry-standard SSL methods.   
·        Controlled User Access: REDCap is employs a robust multi-level security system that enables researchers 
to easily implement "minimum necessary" data access for their research staff, including specification of data 
fields that are identifiers. This feature includes “single click” ability to provide completely deidentified 
(removing all identified data fields and shifting dates) for analysis or other purposes.  User activities are logged 
to enable auditing of all data access.   Access is integrated with OHSU's network such that users who are also 
OHSU employees are authenticated against their OHSU network credentials.   
·        Data Integrity: REDCap is jointly managed in accordance with OHSU Information Security Directives by 
ACC staff and members of OCTRI's Biomedical Informatics Program, ensuring fidelity of database configuration 
and back-ups.  User activities are logged to enable auditing of all data changes. 
 
b. Sharing of Results with Subjects 
Results of standard tests (CBC, LFT, Lipids, BMP, CRP, pregnancy testing, quantiferon, HIV, Hepatitis B, Hepatitis 
C, and PFT/spirometry) will be shared with subjects at their request or if deemed clinically necessary. Lab tests 
evaluating RNA expression and CXCL10, as well as scores on the SGRQ, WASOG-SOAI will not be shared as they 
are for research purposes only and the RNA-expression and CXCL10 laboratory tests are not CLIA certified. 
 
c. Data and Specimen Banking 
After three years, all clinical data and laboratory specimens will be de-identified with an assigned numerical 
code that will not contain any personal identifiers. The key to associating codes and subjects’ personally 
identifying information will be restricted to the PI and study staff. De-identified data will be stored indefinitely 
in an encrypted databank. De-identified samples will be stored indefinitely in a repository in Dr. Rosenbaum’s 
laboratory on the second floor of the Biomedical Research Building.  De-identified samples may be used for 
future research including genetic research.  
 
d. Repository 
Blood will be obtained from the patient in PAXGene Blood tubes via veinipuncture and be transported by the 
study coordinator to the Gene Profiling Shared Resource in Richard Jones Hall where the RNA extraction will 
be performed.  Specimens will be accepted only if in appropriate containers with appropriate labeling.  Dr. 
Friedman, Dr. Stevens or their study staff will verify that consent has been given to participate in genetic 
research and repository prior to collecting any samples for genetic studies (see study consent). Participating in 
the repository is mandatory for inclusion into the study.  Subjects will be assigned a numerical code that will 
not contain any personal identifiers. The key associating the codes and the subjects personally identifying 
information will be restricted to the PI and study staff. This code will be used on all clinical case report forms, 
electronic files for data analysis, and for tracking of specimens.  De-identified samples will be stored indefinitely 
in a locked repository in Dr. Rosenbaum’s laboratory on the second floor of the Biomedical Research Building. 
The Repository Guardian is Dr. James Rosenbaum. His responsibilities include: 
  
• 
Ensuring that data/specimens are received and released according to OHSU policy and the IRB approved 
repository protocol. 


 
Page 7 of 13 
Revised: 7/15/2019 
• 
Executing a repository sharing agreement each time data or specimens are released for research purposes. 
• 
Ensuring the security and confidentiality of stored data and specimens. 
• 
Ensuring the security and confidentiality of data and specimens during transfer. 
• 
Tracking acquisitions and releases of data and specimens. 
• 
Maintaining methods for identifying data/samples for which consent has been withdrawn and ensuring no 
future use. 
• 
Identifying data/samples that have limitations on future uses and ensuring that future uses are not contrary 
to those limits. 
 
A separate IRB approval/determination will be required for each specific human subject research activity that 
uses identifiable data/specimens from the repository. Material transfer agreements will be used when 
necessary for the transfer of biological materials. Only de-identified samples will be released to ensure security 
and confidentiality during the release of data and specimens. 
 
8. Data Analysis 
Objective 1: 
The primary endpoint will be a 50% reduction in corticosteroid requirement in at least 60% subjects (or 3/5 of 
the subjects) by week 16, without significant decline in their pulmonary function—defined as a >15% decline 
in FVC, FEV1, or DLCO relative to the baseline value.  
 
 
Objective 2: 
The secondary endpoints include significant decreases in of peripheral markers of STAT pathway activity using 
RNA sequencing and CXCL10 levels, both measured before and after 16 weeks of tofacitinib treatment.  
 
RNA sequencing will be done using before the study drug is started, and at week 16. We will evaluate STAT1-
dependent gene expression as detected in our prior publication. (18) In our previous study, we found that of 
the 13/18 genes known to be downstream of STAT1 were upregulated in the peripheral blood of sarcoidosis 
patients (WARS, A2M, CCND1, CD64, MT-1, TRH, IRF-1, PSMB9, MHC2TA, OPRM1, Pim-1, CXCL9, and CDKN1A). 
We expect these 13 genes to be upregulated in the blood of pre-treatment subjects, and expect this expression 
level to significantly decrease in post-treatment subjects.  An additional 8 genes in the JAK-STAT pathway were 
also found to be upregulated (IFNAR1, IFNAR2, IFNAR3, JAK1, JAK2, STAT1, STAT2, and STAT3). In a rheumatoid 
arthritis study of synovial biopsies before and after tofacitinib, STAT1 and STAT3 were found to be significantly 
decreased after tofacitinib treatment. (19) Interferon alpha receptors (IFNAR) are located upstream of the JAK-
STAT pathway, and whether these expression profiles are expected to change is less clear. Expression of JAK 1, 
JAK2, STAT1, STAT2, and STAT3 are expected to significantly decrease with tofacitinib therapy.  A paired T test 
will be used to compare expression levels at baseline and after 16 weeks of treatment, where a p<0.05 will be 
considered significant. 
 
Finally, we will be measuring peripheral CXCL10 levels. STAT1 activation leads to CXCL10 transcription, making 
CXCL10 a valuable peripheral marker of JAK-STAT pathway suppression. (20) Serum levels of CXCL10 have been 
found to be elevated in the peripheral blood of patients with sarcoidosis, and were also be found to be 
associated with severity and chronicity of disease. (21) Peripheral expression of CXCL10 in the rheumatoid 
arthritis has been found to decrease after treatment with tofacitinib. (19) A pair T-test will be used to compare 
peripheral CXCL10 levels before and after 16 weeks of treatment, where a p<0.05 will be considered significant.  
 
9. Privacy, Confidentiality, and Data Security 
Standard institutional practices will be followed as described in the OHSU Information Security and Research 
Data Resource Guide (http://ozone.ohsu.edu/cc/sec/isg/res_sec.pdf) to maintain the confidentiality and 
security of data collected in this study. Study staff will be trained with regard to these procedures. Paper files 


 
Page 8 of 13 
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will be stored in locked filing cabinets in restricted access offices at OHSU. Electronic data is stored on restricted 
drives on the OHSU network. Access to data/specimens is restricted to study personnel.  
 
Upon completion of the trial, data will be de-identified and stored in a repository. Upon completion of the trial, 
subjects 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 subject code. Codes 
will not contain any part of the 18 HIPAA identifiers (initials, DOB, MRN). The key associating the codes and the 
subjects personally identifying information will be restricted to the PI and study staff. The key will be kept 
secure on a restricted OHSU network drive in a limited access folder. 
 
10. Provisions to Monitor the Data to Ensure the Safety of Subjects 
See uploaded Data Safety Monitoring Plan form.  
 
11. Risks and Benefits 
a. Risks to Subjects 
Blood draw: A blood draw may cause bleeding, a bruise, an infection, or fainting. Subjects may feel some pain 
when blood is drawn.  
 
Pulmonary function testing or spirometry: Pulmonary function testing or spirometry may cause subjects to feel 
short of breath or dizzy for a few moments after the test.  
Genetic testing:  Although we will make efforts to protect subjects’ identity, there is a small risk of loss of 
confidentiality.  If the results of these studies of genetic makeup were to be accidentally released, it might be 
possible that the information we will gather could become available to an insurer or an employer, or a relative, 
or someone else outside the study. Even though there are certain genetic discrimination and confidentiality 
protections in both Oregon law and federal law, there is still a small chance that a subject could be harmed if 
a release occurred. 
 
Study drug Tofacitinib:  
Tofacitinib has been FDA approved for the treatment of rheumatoid arthritis, but not for the treatment of 
sarcoidosis. It is possible the risks of this drug in sarcoidosis are different from the risks in rheumatoid arthritis. 
In rheumatoid arthritis, this drug has been found to have the following risks: (22)  
 
• 
Infections: Serious and sometimes fatal infections due to bacterial, mycobacterial, invasive fungal, viral, or 
other opportunistic pathogens have been reported in rheumatoid arthritis patients receiving Tofacitinib. 
The most common serious infections reported with Tofacitinib included pneumonia, cellulitis, herpes 
zoster, urinary tract infection, and diverticulitis. Among opportunistic infections, tuberculosis and other 
mycobacterial infections, cryptococcosis, esophageal candidiasis, pneumocystosis, multi-dermatomal 
herpes zoster, cytomegalovirus, and BK virus were reported with Tofacitinib. Some patients have presented 
with disseminated rather than localized disease, and were often taking concomitant immunomodulating 
agents such as methotrexate or corticosteroids. Other serious infections that were not reported in clinical 
studies may also occur (e.g., histoplasmosis, coccidioidomycosis, and listeriosis). 
 
o Overall Infections: In the seven controlled trials, during the 0 to 3 months exposure, the overall 
frequency of infections was 20% and 22% in the 5 mg twice daily and 10 mg twice daily groups, 
respectively, and 18% in the placebo group. The most commonly reported infections with 
Tofacitinib were upper respiratory tract infections, nasopharyngitis, and urinary tract infections 
(4%, 3%, and 2% of patients, respectively). 
 
o Serious Infections: In the seven controlled trials, during the 0 to 3 months exposure, serious 
infections were reported in 1 patient (0.5 events per 100 patient-years) who received placebo and 


 
Page 9 of 13 
Revised: 7/15/2019 
11 patients (1.7 events per 100 patient-years) who received Tofacitinib 5 mg or 10 mg twice daily. 
The rate difference between treatment groups (and the corresponding 95% confidence interval) 
was 1.1 (-0.4, 2.5) events per 100 patient-years for the combined 5 mg twice daily and 10 mg twice 
daily Tofacitinib group minus placebo. In the seven controlled trials, during the 0 to 12 months 
exposure, serious infections were reported in 34 patients (2.7 events per 100 patient-years) who 
received 5 mg twice daily of Tofacitinib and 33 patients (2.7 events per 100 patient-years) who 
received 10 mg twice daily of Tofacitinib. The rate difference between Tofacitinib doses (and the 
corresponding 95% confidence interval) was -0.1 (-1.3, 1.2) events per 100 patient-years for 10 mg 
twice daily Tofacitinib minus 5 mg twice daily Tofacitinib. The most common serious infections 
included pneumonia, cellulitis, herpes zoster, and urinary tract infection. 
 
o Tuberculosis: In the seven controlled trials, during the 0 to 3 months exposure, tuberculosis was 
not reported in patients who received placebo, 5 mg twice daily of Tofacitinib, or 10 mg twice daily 
of Tofacitinib. In the seven controlled trials, during the 0 to 12 months exposure, tuberculosis was 
reported in 0 patients who received 5 mg twice daily of Tofacitinib and 6 patients (0.5 events per 
100 patient-years) who received 10 mg twice daily of Tofacitinib. The rate difference between 
Tofacitinib doses (and the corresponding 95% confidence interval) was 0.5 (0.1, 0.9) events per 100 
patient-years for 10 mg twice daily Tofacitinib minus 5 mg twice daily Tofacitinib.  Cases of 
disseminated tuberculosis were also reported. The median Tofacitinib exposure prior to diagnosis 
of tuberculosis was 10 months (range from 152 to 960 days). 
 
o Opportunistic Infections (excluding tuberculosis): In the seven controlled trials, during the 0 to 3 
months exposure, opportunistic infections were not reported in patients who received placebo, 5 
mg twice daily of Tofacitinib, or 10 mg twice daily of Tofacitinib. In the seven controlled trials, 
during the 0 to 12 months exposure, opportunistic infections were reported in 4 patients (0.3 
events per 100 patient-years) who received 5 mg twice daily of Tofacitinib and 4 patients (0.3 
events per 100 patient-years) who received 10 mg twice daily of Tofacitinib. The rate difference 
between Tofacitinib doses (and the corresponding 95% confidence interval) was 0 (-0.5, 0.5) events 
per 100 patient-years for 10 mg twice daily Tofacitinib minus 5 mg twice daily Tofacitinib.  The 
median Tofacitinib exposure prior to diagnosis of an opportunistic infection was 8 months (range 
from 41 to 698 days) 
 
• 
Malignancies and Lymphoproliferative Disorders:  In the seven controlled rheumatoid arthritis clinical 
studies, 11 solid cancers and one lymphoma were diagnosed in 3328 patients receiving Tofacitinib with or 
without DMARD, compared to 0 solid cancers and 0 lymphomas in 809 patients in the placebo with or 
without DMARD group during the first 12 months of exposure. Lymphomas and solid cancers have also 
been observed in the long-term extension studies in rheumatoid arthritis patients treated with Tofacitinib. 
In Phase 2B, controlled dose-ranging studies in de-novo renal transplant patients, all of whom received 
induction therapy with basiliximab, high dose corticosteroids, and mycophenolic acid products, Epstein Barr 
Virus-associated post-transplant lymphoproliferative disorder was observed in 5 out of 218 patients treated 
with Tofacitinib (2.3%) compared to 0 out of 111 patients treated with cyclosporine. Non-melanoma skin 
cancers have been reported in patients treated with Tofacitinib and identified as an adverse drug reaction.  
 
o Malignancy: In the seven controlled trials, during the 0 to 3 months exposure, malignancies 
excluding NMSC were reported in 0 patients who received placebo and 2 patients (0.3 events per 
100 patient-years) who received either Tofacitinib 5 mg or 10 mg twice daily. The rate difference 
between treatment groups (and the corresponding 95% confidence interval) was 0.3 (-0.1, 0.7) 
events per 100 patient-years for the combined 5 mg and 10 mg twice daily Tofacitinib group minus 
placebo. In the seven controlled trials, during the 0 to 12 months exposure, malignancies excluding 
NMSC were reported in 5 patients (0.4 events per 100 patient-years) who received 5 mg twice daily 


 
Page 10 of 13 
Revised: 7/15/2019 
of Tofacitinib and 7 patients (0.6 events per 100 patient-years) who received 10 mg twice daily of 
Tofacitinib. The rate difference between Tofacitinib doses (and the corresponding 95% confidence 
interval) was 0.2 (-0.4, 0.7) events per 100 patient-years for 10 mg twice daily Tofacitinib minus 5 
mg twice daily Tofacitinib. One of these malignancies was a case of lymphoma that occurred during 
the 0 to 12 month period in a patient treated with Tofacitinib 10 mg twice daily. The most common 
types of malignancy, including malignancies observed during the long-term extension, were lung 
and breast cancer, followed by gastric, colorectal, renal cell, prostate cancer, lymphoma, and 
malignant melanoma 
 
• 
Gastrointestinal Perforation: Events of gastrointestinal perforation have been reported in clinical studies 
with Tofacitinib in rheumatoid arthritis patients, although the role of JAK inhibition in these events is not 
known. 
 
• 
Laboratory Abnormalities: Treatment with Tofacitinib was associated with initial lymphocytosis at one 
month of exposure followed by a gradual decrease in mean absolute lymphocyte counts below the baseline 
of approximately 10% during 12 months of therapy. Lymphocyte counts less than 500 cells/mm3 were 
associated with an increased incidence of treated and serious infections. Treatment with Tofacitinib was 
associated with an increased incidence of neutropenia (less than 2000 cells/mm3) compared to placebo. 
Treatment with Tofacitinib was associated with an increased incidence of liver enzyme elevation compared 
to placebo. Most of these abnormalities occurred in studies with background DMARD (primarily 
methotrexate) therapy.  
 
• 
Treatment with Tofacitinib was associated with increases in lipid parameters including total cholesterol, 
low-density lipoprotein (LDL) cholesterol, and high-density lipoprotein (HDL) cholesterol. Maximum effects 
were generally observed within 6 weeks. The effect of these lipid parameter elevations on cardiovascular 
morbidity and mortality has not been determined. 
 
o Lymphopenia: In the controlled clinical trials, confirmed decreases in absolute lymphocyte counts 
below 500 cells/mm3 occurred in 0.04% of patients for the 5 mg twice daily and 10 mg twice daily 
Tofacitinib groups combined during the first 3 months of exposure. Confirmed lymphocyte counts 
less than 500 cells/mm3 were associated with an increased incidence of treated and serious 
infections. 
 
o Neutropenia: In the controlled clinical trials, confirmed decreases in ANC below 1000 cells/mm3 
occurred in 0.07% of patients for the 5 mg twice daily and 10 mg twice daily Tofacitinib groups 
combined during the first 3 months of exposure. There were no confirmed decreases in ANC below 
500 cells/mm3 observed in any treatment group. There was no clear relationship between 
neutropenia and the occurrence of serious infections. In the long-term safety population, the 
pattern and incidence of confirmed decreases in ANC remained consistent with what was seen in 
the controlled clinical trials. 
 
o Liver Enzyme Elevations: Confirmed increases in liver enzymes greater than 3 times the upper limit 
of normal (3x ULN) were observed in patients treated with Tofacitinib. In patients experiencing liver 
enzyme elevation, modification of treatment regimen, such as reduction in the dose of concomitant 
DMARD, interruption of Tofacitinib, or reduction in Tofacitinib dose, resulted in decrease or 
normalization of liver enzymes. In the controlled monotherapy trials (0-3 months), no differences 
in the incidence of ALT or AST elevations were observed between the placebo, and Tofacitinib 5 
mg, and 10 mg twice daily groups. In the controlled background DMARD trials (0-3 months), ALT 
elevations greater than 3x ULN were observed in 1.0%, 1.3% and 1.2% of patients receiving placebo, 
5 mg, and 10 mg twice daily, respectively. In these trials, AST elevations greater than 3x ULN were 


 
Page 11 of 13 
Revised: 7/15/2019 
observed in 0.6%, 0.5% and 0.4% of patients receiving placebo, 5 mg, and 10 mg twice daily, 
respectively. One case of drug-induced liver injury was reported in a patient treated with Tofacitinib 
10 mg twice daily for approximately 2.5 months. The patient developed symptomatic elevations of 
AST and ALT greater than 3x ULN and bilirubin elevations greater than 2x ULN, which required 
hospitalizations and a liver biopsy. 
 
o Lipid elevations: In the controlled clinical trials, dose-related elevations in lipid parameters (total 
cholesterol, LDL cholesterol, HDL cholesterol, triglycerides) were observed at one month of 
exposure and remained stable thereafter. Changes in lipid parameters during the first 3 months of 
exposure in the controlled clinical trials are summarized: Mean LDL cholesterol increased by 15% 
in the Tofacitinib 5 mg twice daily arm and 19% in the Tofacitinib 10 mg twice daily arm. Mean HDL 
cholesterol increased by 10% in the Tofacitinib 5 mg twice daily arm and 12% in the Tofacitinib 10 
mg twice daily arm. Mean LDL/HDL ratios were essentially unchanged in Tofacitinib-treated 
patients. In a controlled clinical trial, elevations in LDL cholesterol and ApoB decreased to 
pretreatment levels in response to statin therapy. In the long-term safety population, elevations in 
lipid parameters remained consistent with what was seen in the controlled clinical trials. 
 
o Serum creatinine elevations: In the controlled clinical trials, dose-related elevations in serum 
creatinine were observed with Tofacitinib treatment. The mean increase in serum creatinine was 
<0.1 mg/dL in the 12-month pooled safety analysis; however, with increasing duration of exposure 
in the long-term extensions, up to 2% of patients were discontinued from Tofacitinib treatment 
due to the protocol-specified discontinuation criterion of an increase in creatinine by more than 
50% of baseline. The clinical significance of the observed serum creatinine elevations is unknown. 
 
• 
Other adverse reactions:  The following occurred in at least 2% or more of patients and at least 1% greater 
than that observed in patients on placebo:  
 
 
Tofacitinib 
5 
mg 
Twice 
Daily 
Tofacitinib 10 
mg 
Twice 
Daily* 
Placebo 
Preferred Term  
N = 1336 (%)  
N = 1349 (%)  
N = 809 (%)  
Diarrhea  
4.0  
2.9  
2.3  
Nasopharyngitis  
3.8  
2.8  
2.8  
Upper respiratory tract 
infection  
4.5  
3.8  
3.3  
Headache  
4.3  
3.4  
2.1  
Hypertension  
1.6  
2.3  
1.1  
N reflects randomized and treated patients from the seven clinical trials 
 
• 
Adverse reactions occurring in controlled and open-label extension studies include:  
o Blood and lymphatic system disorders: Anemia  
o Infections and infestations: Diverticulitis  
o Metabolism and nutrition disorders: Dehydration  
o Psychiatric disorders: Insomnia  
o Nervous system disorders: Paresthesia  
o Respiratory, thoracic and mediastinal disorders: Dyspnea, cough, sinus congestion  
o Gastrointestinal disorders: Abdominal pain, dyspepsia, vomiting, gastritis, nausea 
o Hepatobiliary disorders: Hepatic steatosis  
o Skin and subcutaneous tissue disorders: Rash, erythema, pruritus  
o Musculoskeletal, connective tissue and bone disorders: Musculoskeletal pain, arthralgia, 
tendonitis, joint swelling  


 
Page 12 of 13 
Revised: 7/15/2019 
o Neoplasms benign, malignant and unspecified (including cysts and polyps): Non-melanoma skin 
cancers  
o General disorders and administration site conditions: Pyrexia, fatigue, peripheral edema 
 
• 
Pregnancy and Teratogenic effects: Pregnancy Category C. Pregnant and breastfeeding women will not be 
included in this trial.  
 
b. Potential Benefits to Subjects 
The purpose of this trial is to determine if Tofacitinib is an effective therapy in sarcoidosis. If this therapy is 
effective, subjects may see a symptomatic improvement and a reduction in corticosteroid requirement. 
Subjects who meet the primary endpoint will go on to a one-year extension where they will be given the study 
drug free of cost.   
 
12. Resources Available  
This study involves RNA extraction and RNA sequencing, which requires special resources through the Gene 
Profiling Shared Resource (GPSR). Dr. Christina Harrington is the director of the GPSR and will be assisting with 
this study.  
 
13. Drugs or Devices  
We will follow the applicable Research Pharmacy policies and procedures and provide Research Pharmacy with 
a pharmacy manual for the study.  
 
References 
 
1. 
Barr JT, Schumacher GE, Freeman S, LeMoine M, Bakst AW, Jones PW. American translation, 
modification, and validation of the St. George's Respiratory Questionnaire. Clin Ther. 
2000;22(9):1121-45. 
2. 
Erdal BS, Clymer BD, Yildiz VO, Julian MW, Crouser ED. Unexpectedly high prevalence of 
sarcoidosis in a representative U.S. Metropolitan population. Respir Med. 2012;106(6):893-9. 
3. 
Judson MA, Chaudhry H, Louis A, Lee K, Yucel R. The effect of corticosteroids on quality of 
life in a sarcoidosis clinic: the results of a propensity analysis. Respir Med. 2015;109(4):526-31. 
4. 
Gottlieb JE, Israel HL, Steiner RM, Triolo J, Patrick H. Outcome in sarcoidosis. The relationship 
of relapse to corticosteroid therapy. Chest. 1997;111(3):623-31. 
5. 
Koth LL, Solberg OD, Peng JC, Bhakta NR, Nguyen CP, Woodruff PG. Sarcoidosis blood 
transcriptome reflects lung inflammation and overlaps with tuberculosis. Am J Respir Crit Care Med. 
2011;184(10):1153-63. 
6. 
Rosenbaum JT, Pasadhika S, Crouser ED, Choi D, Harrington CA, Lewis JA, et al. Hypothesis: 
sarcoidosis is a STAT1-mediated disease. Clin Immunol. 2009;132(2):174-83. 
7. 
Zhou T, Casanova N, Pouladi N, Wang T, Lussier Y, Knox KS, et al. Identification of Jak-STAT 
signaling involvement in sarcoidosis severity via a novel microRNA-regulated peripheral blood 
mononuclear cell gene signature. Sci Rep. 2017;7(1):4237. 
8. 
Ghoreschi K, Jesson MI, Li X, Lee JL, Ghosh S, Alsup JW, et al. Modulation of innate and 
adaptive immune responses by tofacitinib (CP-690,550). J Immunol. 2011;186(7):4234-43. 
9. 
Lee EB, Fleischmann R, Hall S, Wilkinson B, Bradley JD, Gruben D, et al. Tofacitinib versus 
methotrexate in rheumatoid arthritis. N Engl J Med. 2014;370(25):2377-86. 
10. 
van Vollenhoven RF, Fleischmann R, Cohen S, Lee EB, Garcia Meijide JA, Wagner S, et al. 
Tofacitinib or adalimumab versus placebo in rheumatoid arthritis. N Engl J Med. 2012;367(6):508-19. 


 
Page 13 of 13 
Revised: 7/15/2019 
11. 
Mamolo C, Harness J, Tan H, Menter A. Tofacitinib (CP-690,550), an oral Janus kinase inhibitor, 
improves patient-reported outcomes in a phase 2b, randomized, double-blind, placebo-controlled study 
in patients with moderate-to-severe psoriasis. J Eur Acad Dermatol Venereol. 2014;28(2):192-203. 
12. 
Sandborn WJ, Ghosh S, Panes J, Vranic I, Su C, Rousell S, et al. Tofacitinib, an oral Janus kinase 
inhibitor, in active ulcerative colitis. N Engl J Med. 2012;367(7):616-24. 
13. 
Statement on sarcoidosis. Joint Statement of the American Thoracic Society (ATS), the European 
Respiratory Society (ERS) and the World Association of Sarcoidosis and Other Granulomatous 
Disorders (WASOG) adopted by the ATS Board of Directors and by the ERS Executive Committee, 
February 1999. Am J Respir Crit Care Med. 1999;160(2):736-55. 
14. 
Jones PW. Interpreting thresholds for a clinically significant change in health status in asthma 
and COPD. Eur Respir J. 2002;19(3):398-404. 
15. 
Miloslavsky EM, Naden RP, Bijlsma JW, Brogan PA, Brown ES, Brunetta P, et al. Development 
of a Glucocorticoid Toxicity Index (GTI) using multicriteria decision analysis. Ann Rheum Dis. 
2017;76(3):543-6. 
16. 
Judson MA, Costabel U, Drent M, Wells A, Maier L, Koth L, et al. The WASOG Sarcoidosis 
Organ Assessment Instrument: An update of a previous clinical tool. Sarcoidosis Vasc Diffuse Lung 
Dis. 2014;31(1):19-27. 
17. 
Siltzbach LE, James DG, Neville E, Turiaf J, Battesti JP, Sharma OP, et al. Course and prognosis 
of sarcoidosis around the world. Am J Med. 1974;57(6):847-52. 
18. 
Satoh J, Tabunoki H. A Comprehensive Profile of ChIP-Seq-Based STAT1 Target Genes 
Suggests the Complexity of STAT1-Mediated Gene Regulatory Mechanisms. Gene Regul Syst Bio. 
2013;7:41-56. 
19. 
Boyle DL, Soma K, Hodge J, Kavanaugh A, Mandel D, Mease P, et al. The JAK inhibitor 
tofacitinib suppresses synovial JAK1-STAT signalling in rheumatoid arthritis. Ann Rheum Dis. 
2015;74(6):1311-6. 
20. 
Rashighi M, Harris JE. Interfering with the IFN-gamma/CXCL10 pathway to develop new 
targeted treatments for vitiligo. Ann Transl Med. 2015;3(21):343. 
21. 
Su R, Nguyen ML, Agarwal MR, Kirby C, Nguyen CP, Ramstein J, et al. Interferon-inducible 
chemokines reflect severity and progression in sarcoidosis. Respir Res. 2013;14:121. 
22. 
Administration USFaD. Xeljaz Full Prescribing Information; Reference ID: 3891137. FDA 
Maryland; 2016. 
 

ICF_001.pdf: 
Updated 3/29/2019 
 
Page 1 of 2 
 
 
IRB#:17902 
 
 
MED. REC. NO. _____ 
NAME 
_____ 
BIRTHDATE 
_____ 
 
 
 
Clinical Research Consent Summary 
 
 
TITLE: Tofacitinib Hypothesis-generating, Pilot Study for Corticosteroid-Dependent Sarcoidosis 
 
PRINCIPAL INVESTIGATOR: 
James Rosenbaum, MD  (503) 494-8637 
 
You are being asked to join a research study. You do not have to join the study.  Even if you 
decide to join now, you can change your mind later. 
 
1. The purpose of this study is to learn more about the treatment of sarcoidosis. 
 
2. In this study, we will learn about a drug called Tofacitinib.  Tofacitinib will be called 
“the study drug” throughout this form.  We want to learn: 
 
a. We want to learn whether this study drug can be used to treat sarcoidosis in 
patients who are requiring treatment with corticosteroids (like prednisone).  
 
b. We want to learn whether this study drug allows us to lower your dose of 
steroids. 
 
c. We want to learn whether markers in the blood that are related to sarcoidosis can 
be reduced with the use of the study drug.  
 
d. We want to learn if this drug is safe in patients with sarcoidosis.  
 
3. The study drug is produced by Pfizer. Pfizer is paying for the research study.   
 
4. We do not know if the study drug works in sarcoidosis. 
 
5. This study drug is approved by the Food and Drug Administration (FDA) for rheumatoid 
arthritis and psoriatic arthritis. It has not been approved by the FDA for sarcoidosis  
 
6. The study drug is a tablet taken by mouth twice daily. 
 
7. This is an open label study, meaning that every patient who enrolls gets the study drug. 
There is no placebo pill.  
 
8. If you join the study, you will receive the study drug for 16 weeks. The study duration is 
16 weeks. You will have 7 visits to OHSU which includes a baseline visit.   
 


 
 
Updated 3/29/2019 
 
Page 2 of 2 
9. If by the end of 16 weeks your steroids are able to be decreased by more than half your 
original dose and you are not experiencing any side effects, you will have the option to 
continue on study drug for up to one year. This will include 2 additional visits to OHSU. 
 
10. There are risks involved in participating in the study, some of which may be very serious. 
 
11. Samples and information collected during the study will be saved for future research. 
 
12. Samples collected during the study will be used for genetic research. 


Updated 3/29/2019 
 
Page 1 of 12 
 
 
 
IRB#: 17902 
 
 
MED. REC. NO. _____ 
NAME 
_____ 
BIRTHDATE 
_____ 
 
 
Clinical Research Consent and Authorization Form 
 
TITLE: Tofacitinib Hypothesis-generating, Pilot Study for Corticosteroid-Dependent Sarcoidosis 
 
 
PRINCIPAL INVESTIGATOR: 
James Rosenbaum, MD  (503) 494-8637 
 
CO-INVESTIGATORS: 
 
Marcia Friedman, MD  (503) 494-8637 
 
Janelle Stevens, DO  (503) 494-8637 
FUNDED BY:  Pfizer Inc. 
 
OHSU is being compensated by the funder to conduct this study.  This is to pay for tests 
performed for study purposes, and for the time involved on the part of the investigator(s) and 
study staff. You may freely discuss this with your physician and the investigator if you have 
concerns. 
 
PURPOSE: 
 
You have been invited to be in this research study because you have sarcoidosis that requires 
continued steroids. The purpose of this study is to determine whether an investigational drug may 
help treat sarcoidosis.  
 
The study drug may allow us to treat sarcoidosis more effectively and be able to lower your 
prednisone dose.  
 
The use of the study drug in sarcoidosis is experimental.  It has been approved by the FDA for 
use in rheumatoid arthritis and psoriatic arthritis, but not for sarcoidosis. 
 
Following the enrollment visit, this study requires 6 visits to the clinic and will take 16 weeks to 
complete. 
 
Genes are the units of DNA--the chemical structure carrying your genetic information--that 
determine many human characteristics such as the color of your eyes, your height, and whether 
you are male or female. 
 
The blood samples provided by you will be analyzed in the laboratory to determine whether certain 
genes that show increased activity in sarcoidosis, reduce their activity after treatment with the 
study drug.  
 
We are asking you to provide blood and information for a blood and data bank, also called a 
repository.  These samples will be stored indefinitely and may be used and disclosed in the future 
for research, which will include genetic research. 
 
This study will be conducted at OHSU and aims to enroll five patients.  
 
PROCEDURES:   


 
 
 
 
Updated 3/29/2019 
 
Page 2 of 12 
 
This is an open label study, meaning that there is no placebo group. If you enroll in this study you 
will receive the study drug.  
 
This study will take place over 16 weeks. If you enroll in this study you will receive the study drug 
for 16 weeks. The study will involve 7 total visits. At the initial visit you will be asked to fill out 
questionnaires, have blood drawn, undergo a physical exam, and have breathing tests done 
(called pulmonary function tests). The second, third, fourth, fifth, and sixth visits will involve 
physical exams, questionnaires, brief breathing tests (called spirometry), and blood draws. At 
your 6th study visit you will also repeat your full pulmonary function tests. This information is 
detailed in the table below.  
 
During this study we will be decreasing your steroid (also called prednisone) dose. For the first 
month your steroid dose will be kept the same. After the first month your steroids will be gradually 
decreased. If at any time during the steroid taper your disease worsens beyond the three days 
after the dose reduction, we will increase you to the previous dose for 2 weeks. After 2 weeks on 
that dose we will try to lower your dose more gradually. If you are unable to tolerate the steroid 
dose reduction, you will be maintained on the effective steroid dose for the duration of the study.  
 
If by the end of 16 weeks, your steroids are able to be decreased by more than half the original 
dose at the start of the study and you are not experiencing any side effects, you will have the 
option to continue on study drug for up to one year. This will include labs every 2 months at OHSU, 
a phone call check in every 3 months and a clinic visit every 6 months until the end of the extension 
period.  
 
Procedures 
Baseline 
 
Week 
0* 
Week 
2 
Week 
4 
Week 
8 
Week 
12 
Week 
16 
Informed Consent 
X 
 
 
 
 
 
 
Screening Blood draw 
(2 teaspoons ) 
X 
 
 
 
 
 
 
Urine pregnancy test 
(women only) 
X 
 
 
 
 
 
 
Start study drug 
 
X 
 
 
 
 
 
End study drug 
 
 
 
 
 
 
X 
Start steroid taper 
 
 
 
X 
 
 
 
Physical Exam 
X 
 
X 
X 
X 
X 
X 
Pulmonary Function Tests 
X*** 
 
 
 
 
 
X 
 Pulmonary Function Tests 
 
 
 
X 
X 
X 
 
Routine Blood Draw 
(2-4 teaspoons)  
X*** 
 
X 
X 
X 
X 
X 
Genetic Blood Draw   
(2 teaspoons) 
X 
 
 
 
 
 
X 
Questionnaires: Symptom 
assessment  
X 
 
X 
X 
X 
X 
X 
Medication Reconciliation  
 
 
X 
X 
X 
X 
X 
Chest Xray 
X** 
 
 
 
 
 
X 
Length of Visit 
2-4 
hours 
0-2 
hours 
1-2 
hours 
1-2 
hours 
1-2 
hours 
1-2 
hours 
1-2 
hours 


 
 
 
 
Updated 3/29/2019 
 
Page 3 of 12 
* There should be no more than 4 weeks between baseline and start of study drug (week 0) 
**If screening labs or a Chest Xray have been done within the past one year, they do not need to be 
repeated here. 
***If these tests have been done within 4 weeks of baseline visit, they do not need to be repeated. If 
done at another institution records will be requested.  
 
All of the above procedures, testing, and study drug will be billed to the study. Patients and their 
insurance will not be billed for any of the above procedures during the 16 week study. Patients 
continuing on to long-term follow up may have laboratory testing billed to insurance as below under 
“Long term follow-up”. 
 
Women who can become pregnant must have a negative pregnancy test before enrolling in the 
trial. For females, adequate birth control methods will be defined as: hormonal contraceptives, 
intrauterine device or double barrier contraception, i.e., condom + diaphragm, condom or 
diaphragm + spermicidal gel or foam. For males, Adequate birth control methods is defined as: 
use of a condom and/or female birth control methods including: hormonal contraceptives, 
intrauterine device or double barrier contraception, i.e., condom + diaphragm, condom or 
diaphragm + spermicidal gel or foam. 
 
Spirometry or pulmonary function testing evaluates how your lungs work by measuring how much 
and how fast air moves out of your lungs. You will wear a nose clip and forcefully blow into a tube 
hooked to a machine. 
 
Questionnaires and surveys will be used to see how you are doing. We will be asking you 
questions about how you have been feeling and how your illness is affecting your ability to 
function, questions about your physical symptoms, questions about how your medications are 
affecting you, and questions about medication side effects. These questionnaires should take you 
no more than 15 minutes to complete.  
 
If you enroll in this study, the investigators will be reviewing your OHSU medical record. If outside 
records are necessary, you will be asked for written permission prior to collecting outside records.  
 
In the future, your blood samples may be shared with other researchers for other research studies. 
These studies will include genetic research.  The samples and information will be labeled as 
described in the CONFIDENTIALITY section. 
 
RESEARCH REPOSITORY 
 
Generally, a research repository collects, stores and distributes human specimens (samples of 
blood, tissue, or body fluids) and/or data for use in future research projects. Storing and gathering 
lots of specimens and data together can help to conduct future research and avoid re-collecting 
specimens and data over and over again.  With this stored information and samples, researchers 
may understand better how the human body works, develop new tests to find diseases, find new 
ways to treat diseases, or develop new products, such as drugs.   
 
The purpose of this repository is to learn more about sarcoidosis, and to learn more about how 
patients with sarcoidosis respond to tofacitinib. We will be looking at the relationship between 
genes, sarcoidosis, and tofacitinib.  
 
We will collect your blood sample and information about your medical history. 
 
At the start of the study, we will collect about 1 tablespoon of blood which will be stored in the 
repository. After completion of the study, we will collect another 1 tablespoon of blood which will 


 
 
 
 
Updated 3/29/2019 
 
Page 4 of 12 
be stored in the repository. We will also review your medical record and collect information about 
your sarcoidosis (how it has affected you, how long you have had it, and what you have tried to 
treat it), your other medical conditions, medications you have taken, allergies, and results of your 
laboratory tests and imaging studies.  
 
The blood work that we collect will be used to study genes in sarcoidosis. These genetic tests are 
experimental.  
 
ACCESS TO YOUR TEST RESULTS 
 
We will share with you the results of your non-genetic blood tests and breathing tests.  The results 
will be placed in your medical record. 
 
The results of the genetic tests will not be made available to you because the research is still in 
an early phase and the reliability of the results is unknown.  
 
If we discover information that is important for your health care, either in this study or in the future, 
we will contact you and ask if you want to know the results.  If you choose to receive the results, 
you may need to have the test repeated in a non-research laboratory.  You may learn information 
about your health that is upsetting or that impacts your family planning, family relationships, and 
ability to get insurance. Because genetic information is complex and sensitive, the results should 
be discussed with a genetic counselor or your primary care giver who can answer your questions 
or discuss your concerns.  You would be responsible for all costs associated with having the test 
repeated and visiting a doctor or genetic counselor to discuss the results. 
 
The blood tests and breathing tests are being done to answer research questions. If we find an 
abnormality that requires urgent follow-up, we will contact you and your doctor (with your 
permission) to help answer questions and get the right follow-up care for you.  It is possible that 
you could be unnecessarily worried if a problem were suspected, but not actually found. 
 
RISKS AND DISCOMFORTS:  
 
SIDE EFFECTS OF TOFACITINIB 
 
Common side effects 
 
(these may affect up to 
1 in 10 people, or up to 
10%) 
• 
Upper respiratory tract infection (nasopharyngitis, common 
cold)  
• 
lung infection (pneumonia and bronchitis) 
• 
shingles (herpes zoster) 
• 
influenza (flu) 
• 
sinusitis 
• 
urinary bladder infection (cystitis) 
• 
sore throat (pharyngitis) 
• 
increased muscle enzymes or cholesterol 
• 
weight gain 
• 
stomach (belly) pain (which may be from inflammation of the 
stomach lining) 
• 
vomiting, diarrhea, nausea, indigestion 
• 
pain in the joints 
• 
low red blood cell count (anemia) 
• 
fever 
• 
fatigue (tiredness) 
• 
swelling of the feet and hands 
• 
headache 


 
 
 
 
Updated 3/29/2019 
 
Page 5 of 12 
• 
high blood pressure (hypertension) 
• 
cough 
• 
rash 
Uncommon side effects  
 
(these may affect up to 
1 in 100 people, or up 
to 1%) 
• Tuberculosis 
• kidney infection 
• viral infections 
• skin infection 
• herpes simplex or cold sores (oral herpes) 
• viral infections affecting the gut 
• increased liver enzymes 
• low white blood cell count 
• blood creatinine increased (a possible sign of decreased 
kidney function) 
• dehydration 
• 
pain in the musclesmuscle strain, tendonitis, joint swelling, 
ligament sprain, abnormal sensations, poor sleep 
• 
shortness of breath or difficulty breathing 
• 
sinus congestion 
• 
skin redness, itching 
• 
fatty liver 
• 
inflammation of outpouchings of your intestine (diverticulitis), 
tears in your stomach or intestines 
• 
some types of skin cancers (nonmelanoma-types). 
Rare side effects  
 
(these may affect up to 
1 in 1,000 people, or 
up to 0.1%) 
• 
Blood infection (sepsis) 
• 
joint infection 
• 
tuberculosis involving the brain and spinal cord, bones and 
other organs 
• 
other unusual infections. 
 
1. SERIOUS INFECTIONS 
Tofacitinib is a medicine that affects your immune system. Tofacitinib can lower the ability of 
your immune system to fight infections. Some people have serious infections while taking oral 
tofacitinib, including tuberculosis, and infections caused by bacteria, fungi, or viruses that can 
spread throughout the body.  Some people have died from these infections. 
 
You should not start taking tofacitinib if you have any kind of infection. People who are older 
than 65 years of age, people with diabetes, and people with chronic lung disease may have 
a higher risk of infection. The risk of shingles (herpes zoster) in Asian people may be higher 
than for non-Asian people. 
 
After starting tofacitinib, call your study doctor right away if you have any symptoms of an 
infection. Tofacitinib can make you more likely to get infections or make worse any infection 
that you have. 
 
2. CANCER AND IMMUNE SYSTEM PROBLEMS 
Tofacitinib may increase your risk of certain cancers by changing the way your immune system 
works. 
 
Your risk of some skin cancers may be increased with tofacitinib.  Your skin may be examined 
periodically by your doctor. 
 


 
 
 
 
Updated 3/29/2019 
 
Page 6 of 12 
Lymphoma and other cancers can happen in patients taking tofacitinib. Some of the other 
cancers that have happened include lung cancer, breast cancer, melanoma, prostate cancer 
and pancreatic cancer. 
 
Some people who have taken oral tofacitinib with certain other medicines to prevent kidney 
transplant rejection have had a problem with certain white blood cells growing out of control 
(Epstein Barr Virus-associated post-transplant lymphoproliferative disorder). 
 
Tell your study doctor if you have ever had any type of cancer or if you are diagnosed with any 
type of cancer during the study. 
 
3. TEARS OR HOLES (PERFORATION) IN THE STOMACH OR INTESTINES 
Tell your study doctor if you have had diverticulitis (inflammation of outpouchings in parts of 
the large intestine) or ulcers in your stomach or intestines. Some people taking oral tofacitinib 
get tears or holes in their stomach or intestine. 
 
Tell your study doctor right away if you have fever and stomach-area pain that does not go 
away, and a change in your bowel habits. 
 
4. CHANGES IN CERTAIN LABORATORY TEST RESULTS 
Your study doctor will do blood tests before you start receiving tofacitinib and while you take 
tofacitinib. Some changes in blood tests that can occur with tofacitinib include: 
 
• 
Changes in lymphocyte counts. Lymphocytes are white blood cells that help the body 
fight off infections. 
 
• 
Low neutrophil counts. Neutrophils are white blood cells that help the body fight off 
infections. 
 
• 
Low red blood cell count. This may mean that you have anemia, which may make you 
feel weak and tired. 
 
Your study doctor will routinely check certain liver tests as these can increase with tofacitinib 
treatment. You may also have changes in other laboratory tests, such as your blood 
cholesterol levels. Small changes in a test for muscle injury and a test for kidney function may 
also occur. 
 
5. EFFECTS ON PREGNANCY 
In animals, tofacitinib has been shown to have effects on fertility (the ability to become 
pregnant or maintain pregnancy), birth defects and delivery (delay, stillborn births or acute 
newborn death). 
 
You should not take tofacitinib if: 
 
• 
You plan to become pregnant during the study or are pregnant. It is not known if 
tofacitinib will harm an unborn baby. 
 
• 
Plan to breastfeed or are breastfeeding. 
 
• 
If you think you are pregnant, tell the study doctor immediately. Pregnancy will be a 
reason to stop study treatment. 
 
 


 
 
 
 
Updated 3/29/2019 
 
Page 7 of 12 
If you are nursing an infant or you are pregnant now, you must not be in the study.  This study 
may involve risks to an embryo, fetus, or nursing infant that are currently unknown.  If you are 
sexually active and could become pregnant, you and your male partner(s) must use birth 
control that works well. or you must not have sex. For females, adequate birth control methods 
will be defined as: hormonal contraceptives, intrauterine device or double barrier 
contraception, i.e., condom + diaphragm, condom or diaphragm + spermicidal gel or foam. 
You will have to do this the whole time you are in this study. If you become pregnant during 
the research study, please tell the investigator and your doctor immediately. 
 
The drugs in this study may damage sperm or be present in seminal fluid. You should not 
father a child or donate sperm while you are in this study. If you are a sexually active male 
and could cause a pregnancy, you must be sure that your female partner(s) are using birth 
control that works well or you must not have sex. This study may involve risks to an embryo 
or fetus that are currently unknown.  The investigator will talk to you about the types of birth 
control that are acceptable.  If a sexual partner becomes pregnant during the research study, 
please tell the investigator and ask your partner to tell her doctor immediately that you are 
involved in a clinical study. 
 
GENERAL RISKS 
 
• 
You may have some side effects we do not expect because we are still learning about 
how the study drug works in people who have sarcoidosis. You may not have symptoms 
for some of these side effects, such as abnormal lab results, but you will be monitored by 
the investigator to check for any changes throughout the study. 
 
• 
One risk to taking part in this study is that the study drug or the dose you receive may not 
be effective in helping to treat your disease. This means you may spend time in the study 
and experience side effects taking a drug that may not provide you with any health-related 
benefits but may benefit others with the disease. 
 
• 
There are several drugs (prescription and non-prescription) that may cause problems 
when taken with the study drug.  The investigator will carefully review all of the drugs you 
are taking before giving you the study drug.  If any other health care provider prescribes 
any new drug(s) for you while you are in this study, please tell the investigator before you 
take the new drug.  You could also have that provider talk to the investigator before 
prescribing the new drug.  Do not take any new over-the-counter drugs while you are in 
this study unless you first check with the investigator  
 
• 
A federal law, called the Genetic Information Nondiscrimination Act (GINA), generally 
makes it illegal for health insurance companies, group health plans, and most employers 
to discriminate against you based on your genetic information.  Be aware that this federal 
law does not protect you against genetic discrimination by companies that sell life 
insurance, disability insurance, or long-term care insurance.  GINA also does not protect 
you against discrimination if you have already been diagnosed with the genetic disease 
being tested. 
 
• 
Although we have made efforts to protect your identity, there is a small risk of loss of 
confidentiality.  If the results of these studies of your genetic makeup were to be 
accidentally released, it might be possible that the information we will gather about you as 
part of this study could become available to an insurer or an employer, or a relative, or 
someone else outside the study. Even though there are certain genetic discrimination and 
confidentiality protections in both Oregon law and federal law, there is still a small chance 
that you could be harmed if a release occurred. 
 


 
 
 
 
Updated 3/29/2019 
 
Page 8 of 12 
PROCEDURE RISKS 
 
• 
We will draw blood from your arm or hand. You may feel some pain when your blood is 
drawn.  There is a small chance the needle will cause bleeding, a bruise, an infection, or 
fainting. 
 
• 
Spirometry or pulmonary function testing may make you cough or feel lightheaded. This 
will go away shortly after the test is finished. 
 
• 
Some of these questions asked in the questionnaires may seem very personal or 
embarrassing.  They may upset you.  You may refuse to answer any of the questions that 
you do not wish to answer.  If the questions make you very upset, we will help you to find 
a counselor.   
 
BENEFITS: 
 
You may or may not personally benefit from being in this study.  However, by serving as a subject, 
you may help us learn how to benefit patients in the future. 
 
ALTERNATIVES:  
You may choose not to be in this study. If you do not choose to be in this study, there are other 
drugs for sarcoidosis that your doctor may use to treat you.  
 
CONFIDENTIALITY  
We will take steps to keep your personal information confidential, but we cannot guarantee total 
privacy.   
 
We will create and collect health information about you as described in the Purpose and 
Procedures sections of this form.  Health information is private and is protected under federal law 
and Oregon law.  By agreeing to be in this study, you are giving permission (also called 
authorization) for us to use and disclose your health information as described in this form. 
 
The investigators, study staff, and others at OHSU will use the information we collect and create 
about you in order to conduct and oversee this research study. They will also store your 
information and blood specimens in a repository to conduct future research.   
 
Your blood sample will be stored in a freezer in Dr. Jim Rosenbaum’s laboratory. Once the study 
is finished, your sample will be assigned a code that does not contain any personally identifying 
information. Only Dr. Rosenbaum and his team will have access to the code to re-identify your 
sample. Your medical information will be stored on a password protected document on an 
encrypted OHSU drive. Only Dr. Rosenbaum and his team will have access to this information. 
The samples and data will be stored indefinitely.   
 
Data/specimens from this study may be shared with other investigators for future research 
studies.  A code number will be assigned to you, your cells and genetic information, as well as to 
information about you.  Only the investigators and people involved in the conduct of the study will 
be authorized to link the code number to you.  Other investigators who may receive samples of 
your blood/ genetic information/medical information for research will be given only the code 
number which will not identify you.  
 
 
 
 
We will release this information to others outside of OHSU who are involved in conducting or 
overseeing research, including: 


 
 
 
 
Updated 3/29/2019 
 
Page 9 of 12 
 
• 
The funder of this study, Pfizer Inc., and the funder’s representatives  
• 
The Food and Drug Administration 
• 
The Office for Human Research Protections, a federal agency that oversees research 
involving humans 
 
Those listed above may also be permitted to review and copy your records, including your medical 
records. 
 
We will not release information about you to others not listed above, unless required or permitted 
by law.  We will not use your name or your identity for publication or publicity purposes, unless 
we have your special permission. 
 
When we send specimens or information outside of OHSU, they may no longer be protected 
under federal or Oregon law.  In this case, your specimens or information could be used and re-
released without your permission. 
 
Data and specimens from this study may be shared with other investigators for future research 
studies.  All identifying information about you will be removed from the samples before they are 
released to any other investigators.  
 
Your genetic information may be shared in a public online database for future research.  The 
database will not contain any information that directly identifies you, such as your name, address, 
or birth date, so it is unlikely that someone would know the genetic information came from you.  
In the future, people may develop ways to identify you or your blood relatives from this information, 
but currently, there is not a way to identify you without having additional information to compare 
to it, such as information from your DNA sample. 
 
We may continue to use and disclose your information as described above indefinitely.  
 
Some of the information collected and created in this study may be placed in your OHSU medical 
record.  While the research is in progress, you may or may not have access to this information.  
After the study is complete, you will be able to access any study information that was added to 
your OHSU medical record.  If you have questions about what study information you will be able 
to access, and when, ask the investigator. 
 
COMMERCIAL DEVELOPMENT: 
 
Samples and information about you or obtained from you in this research may be used for 
commercial purposes, such as making a discovery that could, in the future, be patented or 
licensed to a company, which could result in a possible financial benefit to that company, OHSU, 
and its researchers.  There are no plans to pay you if this happens.  You will not have any property 
rights or ownership or financial interest in or arising from products or data that may result from 
your participation in this study.  Further, you will have no responsibility or liability for any use that 
may be made of your samples or information. 
 
COSTS:   
 
Some of the services or items in this study are part of the regular treatment for your condition.  
These would be performed or used even if you were not in this study.  The costs for these services 
or items will be billed to your insurance.  You will be responsible for any costs your insurance 
does not cover.  If you have any questions about these costs, or what out-of-pocket expenses 
you may be responsible for, contact your insurance company.  If you are uninsured, you will be 
responsible for these costs.   


 
 
 
 
Updated 3/29/2019 
 
Page 10 of 12 
 
You will not be billed for the costs of any services or procedures that are required by the study 
but are not considered part of your regular treatment. 
 
If enrolled in this study, you will be compensated for participation in the study. You will receive 
$50 per visit that you complete via a debit card up to $400 total over the course of the study. If 
you withdraw before completing the study you will not receive the full amount. 
 
You may receive payment via a debit card.  There may be fees (for example, if the card is inactive 
for more than six months), which will be deducted from the balance on your card.  Details on how 
to use the card and any fees are included in the separate card member agreement and FAQ 
sheet. 
 
LIABILITY:   
 
If you believe you have been injured or harmed as a result of participating in this research and 
require treatment, contact Marcia Friedman, MD at (503) 494-8637. In the case of an emergency 
you should contact 911.  
 
If you are injured or harmed by the study drug you will be treated.  OHSU and the funder do not 
offer any financial compensation or payment for the cost of treatment if you are injured or harmed 
as a result of participating in this research.  Therefore, any medical treatment you need may be 
billed to you or your insurance.  However, you are not prevented from seeking to collect 
compensation for injury related to negligence on the part of those involved in the research.  
Oregon law (Oregon Tort Claims Act [ORS 30.260 through 30.300]) may limit the dollar amount 
that you may recover from OHSU or its caregivers and researchers for a claim relating to care or 
research at OHSU, and the time you have to bring a claim. 
 
If you have questions on this subject, please call the OHSU Research Integrity Office at (503) 
494-7887. 
 
PARTICIPATION: 
 
If you have any questions, concerns, or complaints regarding this study now or in the future, 
contact James Rosenbaum, MD (503) 494-8637 or other members of the study team at (503) 
494-8637 
 
This research is being overseen by an Institutional Review Board (“IRB”). You may talk to the IRB 
at (503) 494-7887 or irb@ohsu.edu if: 
 
• 
Your questions, concerns, or complaints are not being answered by the research team. 
• 
You want to talk to someone besides the research team. 
• 
You have questions about your rights as a research subject. 
• 
You want to get more information or provide input about this research. 
 
You may also submit a report to the OHSU Integrity Hotline online at 
https://secure.ethicspoint.com/domain/media/en/gui/18915/index.html or by calling toll-free (877) 
733-8313 (anonymous and available 24 hours a day, 7 days a week).   
 
Your participation in this study is voluntary. You do not have to join this or any research study.  
You do not have to allow the use and disclosure of your health information in the study, but if you 
do not, you cannot be in the study.  
 


 
 
 
 
Updated 3/29/2019 
 
Page 11 of 12 
If you do join the study and later change your mind, you have the right to quit at any time.  This 
includes the right to withdraw your authorization to use and disclose your health information. If 
you choose not to join any or all parts of this study, or if you withdraw early from any or all parts 
of the study, there will be no penalty or loss of benefits to which you are otherwise entitled, 
including being able to receive health care services or insurance coverage for services.  Talk to 
the investigator if you want to withdraw from the study. 
 
If you no longer want your health information to be used and disclosed as described in this form, 
you must send a written request or email stating that you are revoking your authorization to: 
 
Marcia Friedman, MD 
3181 SW Sam Jackson Park Rd.  
Mail Code OP09 
Portland, OR 97239 
friedmam@ohsu.edu 
 
Your request will be effective as of the date we receive it.  However, health information collected 
before your request is received may continue to be used and disclosed to the extent that we have 
already acted based on your authorization.   
 
If you do chose to withdraw from the study, you will be requested to answer questions regarding 
side-effects you may have had, and we will ask to draw your blood two weeks after you withdraw 
from the study to evaluate for any side effects from the study drug.  
 
The samples and information we will collect from you will be provided to the funder (Phizer). It will 
be stored with a coded identifier to protect your privacy.  Once provided to the funder, we will not 
be able to destroy your samples or data if you decide in the future you do not wish to participate 
in the research. 
 
You may be removed from the study if the investigator or funder stops the study, you become 
pregnancy, you develop serious side effects, your disease gets worse, or you do not follow study 
instructions.  
 
We will give you any new information during the course of this research study that might change 
the way you feel about being in the study. 
 
Your health care provider may be one of the investigators of this research study and, as an 
investigator, is interested in both your clinical welfare and in the conduct of this study.  Before 
entering this study or at any time during the research, you may ask for a second opinion about 
your care from another doctor who is in no way involved in this project.  You do not have to be in 
any research study offered by your physician.   
 
 
 
 
 
 
 
 
 
 
 
 
 


 
 
 
 
Updated 3/29/2019 
 
Page 12 of 12 
 
 
 
 
 
 
 
SIGNATURES: 
 
Your signature below indicates that you have read this entire form and that you agree to be in 
this study.   
 
We will give you a copy of this signed form. 
 
 
 
Subject Printed Name 
 
Subject Signature 
 
Date 
Person Obtaining Consent Printed Name 
 
Person Obtaining Consent Signature 
 
Date 
 
 
 
Complete if the participant is not fluent in English and an interpreter was used to obtain 
consent.  Participants who do not read or understand English must not sign this full consent 
form, but instead sign the short form translated into their native language.  This form should 
be signed by the investigator and interpreter only.  If the interpreter is affiliated with the study 
team, the signature of an impartial witness is also required. 
 
Print name of interpreter: ______________________________________ 
 
Signature of interpreter: ___________________________________  Date: _________ 
 
An oral translation of this document was administered to the subject in _____________ 
(state language) by an individual proficient in English and ____________ (state language).  
 
If applicable: 
Print name of impartial witness: __________________________________ 
 
Signature of impartial witness: ________________________________Date: _________ 
 
See the attached short form for documentation.