Topical Ruxolitinib Cream for Refractory Cutaneous Dermatomyositis
Phase: PHASE2
Conditions: Dermatomyositis
Sponsor: The Cleveland Clinic

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Topical Ruxolitinib cream for refractory cutaneous Dermatomyositis:  
PART 1: Prospective, Single-Arm, Open-Label Pilot study 
 
Anthony P. Fernandez, MD, PhD 
Cleveland Clinic, Cleveland, Ohio 
 
Dermatomyositis (DM) is an immune-mediated disease that most commonly affects the skin and 
the muscles.  Cutaneous involvement of DM can lead to ulceration with secondary infection, in 
addition to permanent skin damage in the form of atrophy, scarring, calcinosis, decreased range 
of motion or lipoatrophy.  Furthermore, persistent cutaneous DM is associated with adverse 
psychological effects and physical symptoms such as pain, burning, and pruritus.  The 
combination of these contribute to a significant negative impact on DM patients’ quality of life 
(1).  Thus, effective treatment of cutaneous DM represents an important therapeutic goal (2).  
 
Unfortunately, cutaneous DM manifestations often persist despite myositis resolution and can 
be particularly difficult to control. Studies in patients with juvenile DM have found that up to 
60% have persistent rash, and the percentage with persistent rash is often more than double 
that of patients with persistent myositis (3-5).   In a recent study of adult DM patients who were 
prospectively observed, only 28 of 74 (38%) achieved clinical remission of their cutaneous 
disease over a 3-year period despite aggressive systemic therapy treatment regimens (6).   
Given its refractory nature, combination systemic therapy is often prescribed in attempts to 
control DM skin involvement, including systemic glucocorticoids, antimalarials and various 
steroid-sparing immunosuppressive medications. Despite such combinations, though, cutaneous 
manifestations of DM still often remain refractory and symptomatic. In these patients, in 
particular, lack of adequate skin improvement coupled with increased risk of serious infection or 
malignancy from systemic immunosuppression becomes concerning.  Therefore, safe and 
effective treatments for cutaneous disease represents an unmet need for patients with DM.    
  
Janus kinase inhibitors (JAK inhibitors) are a relatively new class of medications that have been 
hypothesized to have utility in treating various autoimmune diseases, including DM (7, 8).  
Excitement for the emerging use of JAK inhibitors in patients with DM has been underscored by 
their efficacy in case reports and small trials.  Specifically, oral ruxolitinib and tofacitinib have 
recently been reported to effectively treat DM patients, all of whom had refractory cutaneous 
disease despite previous or current exposure to numerous systemic medications (9-11).  
However, adding further systemic immunosuppression to DM patients who are otherwise 
controlled except for refractory skin disease is unattractive due to risk of infection, and other 
possible adverse effects of oral JAK inhibitors may preclude their use in some DM patients. 
The addition of topical corticosteroids and calcineurin inhibitors for refractory DM skin 
involvement as adjunct treatment in patients already taking systemic medications is attractive 
because they do not lead to further immunosuppression.  However, cutaneous DM symptoms 
and inflammation are also often refractory to these topical medications (12, 13). Additionally, 

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continued topical corticosteroid use is associated with well-known adverse effects, including 
potential worsening of DM-related skin atrophy and adrenal insufficiency (14, 15).   
Recently, topical ruxolitinib has been developed and studied in several dermatologic conditions.  
In phase 2 and 3 trials in atopic dermatitis, topical ruxolitinib 1.5% cream resulted in significant 
efficacy in terms of improving both disease activity and itching, without significant side effects 
(16-18).  We propose to study use of topical ruxolitinib 1.5% cream as an adjunct for treatment 
of refractory cutaneous DM lesions in patients who have previously failed other topical 
medication options. 
 
Study Design (PART 1) 
 
Prospective, Single-Arm, Open-Label Pilot study 
 
Patient population and estimated sample size: 
 
I. 
Approximately 15-30 patients with refractory cutaneous symptoms (CDASI activity 
score >6) related to either classic dermatomyositis, juvenile dermatomyositis, or 
amyopathic dermatomyositis will be enrolled. 
a. 15 patients are need to detect a 35% decrease in the mean CDASI activity score 
or CDASI total score with 80% power and significance level of 0.05 at 8 weeks. 
b. 20 patients are need to detect a 30% decrease in the mean CDASI activity score 
or CDASI total score with 80% power and significance level of 0.05 at 8 weeks.   
c. 29 patients are need to detect a 25% decrease in the mean CDASI activity score 
or CDASI total score with 80% power and significance level of 0.05 at 8 weeks.   
  
Inclusion Criteria: 
• Patients 18 years and older with refractory cutaneous symptoms related to either 
classic dermatomyositis (CD), juvenile dermatomyositis (JD), or amyopathic 
dermatomyositis (AD).  Diagnosis will be based on either Bohan and Peter criteria (CD 
and JD) or Sontheimer’s criteria (AD) (19-22). 
 
• Patients must have had a skin biopsy with histologic features consistent with 
dermatomyositis and current cutaneous manifestations consistent with 
dermatomyositis. 
 

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• Patients will be considered to have refractory disease if cutaneous manifestations 
exist despite treatment with systemic corticosteroids and at least one steroid-sparing 
systemic treatment commonly found to be useful in patients with dermatomyositis.  
These may include azathioprine, cyclosporine, mycophenolate mofetil, IVIG, 
methotrexate, hydroxychloroquine, cyclophosphamide, chlorambucil, sirolimus, 
tacrolimus, and rituximab.   
 
• Patients must have sufficiently active cutaneous involvement of dermatomyositis 
(BSA>1% to <20%, CDASI activity score > 6, and Physician Global Assessment (PGA) 
activity score >2). 
 
• Patients must have tried and failed at least one commonly prescribed topical 
medication in the past, with the last application of a topical medication to active skin 
lesions occurring greater than 2 weeks prior to enrollment.   
o Commonly prescribed topical medications for dermatomyositis include 
corticosteroids or calcineurin inhibitors (tacrolimus or pimecrolimus). 
 
• Patients must have been on a stable systemic medication regimen for at least 2 
months (60 days) and must agree to keep the regimen stable throughout the study 
period.  As patients with dermatomyositis are commonly treated with combination 
regimens that include both topical and systemic immunosuppressive medications, 
any added risk of adverse effects related to ruxolitinib 1.5% cream is considered 
negligible. 
 
• Patients must be agreeable to use appropriate contraceptive measures while enrolled 
in the study.   
o Women of childbearing potential must be willing to practice abstinence or use 
either an oral contraceptive medication or IUD if sexually active. 
o Women of childbearing potential must be willing to have monthly urine 
pregnancy tests while enrolled in the study 
o Men of childbearing potential must be willing to practice abstinence or use 
condoms if sexually active. 
 
 

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Exclusion criteria: 
• Patients with dermatomyositis who have minimal-to-no active cutaneous disease 
(mild involvement with < 1% total body surface area involved and/or CDASI activity 
score of < 6). 
 
• Patients who have > 20% total BSA involvement of cutaneous dermatomyositis. 
 
• Patients who have used a common prescription topical medication within the 
previous 2 weeks. 
 
• Patients whose cutaneous findings are not consistent with dermatomyositis and/or 
have previous biopsy results suggestive of an alternative diagnosis 
 
• Patients not on stable systemic medication regimens for at least 2 months and/or 
who will not agree to keep the regimen stable throughout the study period. 
 
• Patients who have previously taken a systemic Janus kinase inhibitor but had a poor 
response, patients who are currently taking systemic Janus kinase inhibitors, or 
patients who have used a topical Janus kinase inhibitor for their dermatomyositis or 
any other condition and had poor responses. 
 
• Patients with inflammatory myositis other than dermatomyositis, such as 
polymyositis or inclusion body myositis. 
 
• Patients with clear features of an overlap autoimmune myositis or with an 
inflammatory myositis not consistent with dermatomyositis, such as polymyositis or 
inclusion body myositis. 
 
• Patients with an active malignancy other than non-melanoma skin cancer, or with 
malignancy-associated dermatomyositis. 
 
• Patients younger than 18 years old  

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• Participants with concurrent conditions and history of other diseases: 
a. Immunocompromised (eg, lymphoma, acquired immunodeficiency 
syndrome, Wiskott-Aldrich syndrome). 
b. Chronic or acute infection requiring treatment with systemic antibiotics, 
antivirals, antiparasitics, antiprotozoals, or antifungals within 2 weeks before 
baseline. 
c. Active acute bacterial, fungal, or viral skin infection (eg, herpes simplex, 
herpes zoster, chicken pox) within 1 week before baseline. 
d. Any other concomitant skin disorder (eg, generalized erythroderma such as 
Netherton syndrome), pigmentation, or extensive scarring that, in the 
opinion of the investigator, may interfere with the evaluation of AD lesions 
or compromise participant safety. 
e. Current or history of hepatitis B or C virus infection 
 
• Pregnant or lactating females, or female patients planning to get pregnant within 
the study period timeline. 
 
• Patients who are sexually active and/or of child-bearing potential who are unwilling 
to use appropriate contraceptive measures during the study period. 
 
• Patients with a history of myocardial infarction or major thromboembolic events, 
including pulmonary embolism, deep venous thrombosis, or ischemic stroke. 
 
• Patients with a history of significant cytopenias. 
 
• Patients with any medical condition that is felt by the primary investigator to place 
the patient at unreasonable risk for adverse effects during treatment with 
Ruxolitinib cream.   
 
• Hypersensitivity to ruxolitinib cream or any of its components. 
 
 

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Methods: 
 
I. 
Patients will be prescribed 1.5% ruxolitinib cream and will be instructed to apply a thin 
film twice daily to all areas with active cutaneous disease (up to 20% BSA total).  All 
other topical medications used up to this point will be discontinued.  
II. 
Patients will require follow-up visits in person at 4 weeks, 8 weeks (primary endpoint), 
and 12 weeks (+/- 5 days) after enrollment, at which time examinations and clinical 
scoring tool assessments will be repeated.   
III. 
Objective cutaneous DM scoring by the primary investigator at each visit will include the 
Cutaneous Dermatomyositis Disease Area and Severity Index (CDASI) score, Physician 
Global Assessment for (a) skin activity (b) skin damage, and estimate of body surface 
area (BSA) involvement with active dermatomyositis (23-28). 
IV. 
Objective scoring tools to be completed by the patient at each visit will include:  Patient 
Global Skin Score, Patient Global Itch Score, and the Dermatology Life Quality Index 
(23-28). 
V. 
Photographs of cutaneous lesions will be performed at baseline and at each follow-up 
visit for all enrolled patients. 
Lesional (4mm) biopsies of a representative active area to be treated at study initiation and 
again at 8 weeks will be optional, but a goal of obtaining biopsies from > 4 patients will be 
established.   Biopsies will be evaluated for histologic features of DM by routine light 
microscopy.  Additionally Nanostring PCR will be performed to assess for changes in RNA 
transcript levels and inflammatory cytokines related to DM and JAK-STAT pathways. 
Research Procedures  
I. 
Upon enrollment patients will undergo a thorough history (including assessing time of 
diagnosis), review of systems (including inquiry about photosensitivity), and a complete 
physical examination. 
II. 
Patients will be prescribed 1.5% ruxolitinib cream to be used twice daily on affected 
areas (up to 20% BSA total) throughout the study period.  All other previous/current 
topical medications will be required to be discontinued at least 2 weeks prior to this 

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point. 
III. 
Patients will apply the first dose in clinic after all clinical assessments under the 
supervision of the PI to ensure they apply the correct amount to affected areas.  
Instructions to wait 8-12 hours between applications will be relayed.  Patients will be 
provided with a drug diary to document number and times of applications on a day-to-
day basis.  If patients miss a dose, this should be documented but patients should not 
try to “make up” this dose by applying the medication more than twice on any given 
day.    Adequate supplies of 1.5% ruxolitinib cream will be dispensed at each clinic visit 
during the study. 
IV. 
Patients will be instructed to continue their current topical moisturizers, and sunscreen 
based on their current practice during the study period. 
V. 
A detailed examination of the skin and mucous membranes will be performed, noting 
for presence/absence of the following: scalp erythema and atrophy, alopecia, heliotrope 
rash, typical erythema and atrophy of sun-exposed areas on the neck, upper chest and 
upper back (shawl sign), erythema +/- scaling of the elbows and/or knees (Gottron’s 
sign), gottron’s papules, periungual erythema, ragged cuticles, proximal nailfold capillary 
loops, palmar scaling and hyperkeratosis (mechanic’s hands), a rash involving the hips 
(holster sign), oral ulcerations, cutaneous ulcerations, purpura. 
VI. 
Objective cutaneous dermatomyositis scoring by the primary investigator using the 
Cutaneous Dermatomyositis Disease Area and Severity Index (CDASI) score Physician 
Global Assessment for (a) skin activity (b) skin damage, and estimate of body surface 
area (BSA) involvement (handprint method) with active dermatomyositis will be 
performed at each visit (23-28). 
VII. 
Objective scoring tools to be completed by the patient at each visit will include:  Patient 
Global Skin Score (0-10 visual analog scale), Patient Global Itch Score (0-10 visual 
analog scale), and the Dermatology Life Quality Index (validated quality of life 
assessment) (23-28).  
 
VIII. 
Photographs of all cutaneous lesions will be performed at baseline and at each follow-
up visit 
IX. 
All patients will have a baseline comprehensive myositis autoantibody panel test 

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(Oklahoma Medical Research Foundation; https://omrf.org/research-faculty/core-
facilities/myositis-testing/ ) to assess for presence of myositis-associated and myositis-
specific autoantibodies.   
X. 
Patients will be expected to continue their current systemic immunosuppressive 
treatment regimens throughout the study.  If it is felt that patients should taper off of 
medications they will require a washout period prior to starting the study: 
- 
corticosteroids- 4 weeks  
- 
methotrexate/azathioprine/mycophenolate/cyclosporine/tacrolimus/adrenocort
icotropic hormone analogs/chlorambucil/cyclophosphamide/other systemic 
immunomodulatory agents- 4 weeks 
- 
Biologic medications (rituximab, etc.) and IVIG: - 5 half-lives or 12 weeks.  
XI. 
 If it is felt that patients should start a new systemic medication they will require a 
period of 8 weeks (60 days) on the medication prior to starting this study. 
XII. 
Patients requiring live-attenuated vaccinations will need a 2 week “washout” period 
prior to enrolling in the study, and live-attenuated vaccines are not recommended 
during the treatment period. 
XIII. 
Patients will require follow-up visits in person at 4 weeks (+/- 5 days), 8 weeks (+/- 5 
days) and  12 weeks (+/- 5 days) after initiation of ruxolitinib 1.5% cream, at which time 
examinations and clinical scoring tool assessments will be repeated.   
XIV. 
Ruxolitinib 1.5% cream will be discontinued in any given patient at the primary 
investigator’s discretion if deemed necessary for the patient’s overall health and well-
being at any point during the study.  
XV. 
To monitor safety and tolerability of 1.5% ruxolitinib cream, patients will be asked about 
any adverse symptoms at each clinic visit and instructed to call the investigators with 
any adverse symptoms that occur in between visits. Additionally, routine laboratory 
results (CBC and CMP) will be obtained and analyzed per routine intervals based on 
specific systemic medication regimens.   
 
 
 

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Primary end-point: 
 
I.     
Improvement in CDASI activity by > 25% (n=30) / 30% (n=20) / 35% (n=15) at 8 weeks. 
 
**primary end-point goal will depend on number of patients enrolled 
 
 
Secondary end-points: 
 
I.           Improvement in CDASI activity by > 25% (n=30) / 30% (n=20) / 35% (n=15) at 12 weeks. 
**primary end-point goal will depend on number of patients enrolled 
 
II. 
Change in patient Global Itch Score by at least 2 points at 8 and 12 weeks.  
 
III. 
Improvement in Physician’s Global Assessment by at least 2 points at 8 and 12 weeks. 
 
IV. 
Improvement in Global Patient Skin Score by at least 2 points at 8 and 12 weeks. 
 
V.          Improvement in the Dermatology Life Quality Index score by at least 2 points at 8 and   
              12 weeks. 
 
VI.         Improvement in % BSA of active DM rash by at least 1% at 8 and 12 weeks. 
 
VII. 
Safety and tolerability of 1.5% ruxolitinib cream. 
 
 
 
 
Exploratory end-points: 
 
I.   Changes in RNA transcript levels of inflammatory cytokines related to DM and JAK-STAT 
pathways at 8 weeks. 
 
 
 
 

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Study Assessments 
Study 
Assessments 
Baseline 
4 weeks +/- 5d 
8 weeks +/- 5d 
12 weeks  +/- 5d 
Informed 
consent 
X 
 
 
 
Medical history 
X 
 
 
 
Physical Exam 
X 
X 
X 
X 
CDASI 
X 
X 
X 
X 
% BSA active 
skin disease 
(handprint 
method) (28) 
X 
X 
X 
X 
PGA skin activity 
(VAS 10-point 
scale) 
X 
X 
X 
X 
Patient Global 
Assessment Skin 
Activity Score  
(VAS 10-point 
scale) 
X 
X 
X 
X 
Patient Global 
Itch Score  
(VAS 10-point 
scale) 
X 
X 
X 
X 
DLQI 
X 
X 
X 
X 
Photography 
X 
X 
X 
X 
Skin Biopsy 
(subset of pts) 
X 
 
X 
 

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Comprehensive 
Myositis 
Autoantibody 
Panel 
X 
 
 
 
Pregnancy 
testing 
X 
X 
X 
X 
IP application 
training 
X 
 
 
 
IP 
administration 
in clinic 
X 
 
 
 
Dispense IP 
X 
X 
X 
 
Collect IP 
 
X 
X 
X 
Assess 
Compliance 
 
X 
X 
X 
 
Length of each test/procedure: 
Modified CDASI: 5 min 
% BSA:  5 min 
PGA (VAS scoring): < 1 minute 
Patient Global Skin Score (VAS scoring): < 1 min 
Patient Global Itch Score (VAS scoring): < 1 min  
DLQI: 5 min 
**see references above associated with each individual test for details concerning validity 
and reliability 
Investigational Product materials and management 
Information about drug  
The IP is 1.5% ruxolitinib topical cream.  
 

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Packaging and dispensation of Investigational Product 
The IP is a white-to-off-white cream containing 1.5% ruxolitinib and is supplied in 60g aluminum 
tubes. IP will be provided at each visit for a total of 3 months of IP application. 
 
Storage and Handling of Investigational Product 
The IP should be stored at 20°C to 25°C (68°F to 77°F), with excursions allowed from 15°C to 
30°C (59°F to 86°F) 
 
Application  
The IP should be applied in a thin layer twice daily to affected areas of up to 20% body surface 
area.   The Investigator will instruct the subject on how to apply the IP at the Baseline visit and 
the initial application of the IP will take place in clinic under observation.  No other IP 
applications at subsequent visits will be required to be observed in the clinic setting.   
 
In addition to the verbal instructions given during this visit, written instructions may be 
provided to the subjects.  Subjects should apply twice daily applications of IP approximately 12 
hours apart (between 8 and 12 hours).  If an IP application has been missed or delayed such 
that there would be < 6 hours between applications, the subject should not apply that dose of 
IP and just wait to apply the next dose. The importance of IP compliance should be discussed 
with the subject during each site visit.   
 
Subjects will be instructed to store their IP in a secure location away from children.  WOCBP 
should not come in contact with the IP unless using contraception as specified in this protocol.   
 
If it is suspected that inappropriate amounts of IP are being applied by the subject further 
review of IP application in the clinic may be warranted.   
 
Investigational Product Accountability and Disposal 
Upon receipt of the IP, the Investigator (or designee, e.g., study center pharmacist) will 
acknowledge receipt of the IP after reviewing the shipment’s content and condition. The 
Investigator (or designee) is responsible for ensuring that the designated study site staff 
conduct a complete inventory of study materials and assume responsibility for their storage 
and dispensing. The Investigator (or designee) must agree to keep all study materials in a 
secure location with restricted access. The Investigator (or designee) will keep a record of the 
inventory and dispensing of all IPs. This record will be made available to the Study Monitor for 
the purpose of accounting for all clinical supplies. Any significant discrepancy and/or deficiency 
must be recorded with an explanation.  
All supplies sent to the investigators will be accounted for and, in no case, used in any 
unauthorized situation. Each tube of IP administered at the study center will be administered 
by qualified study center staff. At each site visit, IP compliance will be discussed with the 
subject.  

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At the end of the study, following final IP inventory reconciliation by the monitor, the study site 
will dispose of and/or destroy all supplies (including used, partially used, and/or unused tubes 
of IP) where possible, in compliance with the site’s SOPs for IP disposal/destruction. In the 
event that the site is unable to dispose of and/or destroy IP supplies, the Sponsor will be 
notified and a third-party vendor may be contracted to manage destruction of IP supplies. 
 
Data Analysis 
Access to Data will be limited to the P.I., research coordinator, and research nurse. Approval 
from the IRB to allow access to data will be sought via an IRB amendment application if any 
additional research personnel are added to the study. 
Confidentiality of patients will be protected by assigning numbers to patients and avoiding use 
of PHI. Results of the study will be reported in aggregate and anonymously 
Statistical analyses will be conducted by the section of Biostatistics in the Quantitative Health 
Sciences department at the Cleveland Clinic using the methods described below: 
Statistical Methods 
Patient and disease characteristics will be summarized using means, standard deviations, 
medians and other percentiles of interest for continuous measures and frequencies and 
percentages for categorical factors.  Changes in scoring tools and body surface area measures 
from baseline to 8 weeks will be calculated and compared using paired t-tests.  If necessary, 
transformations of the data will be performed prior to analysis to meet distributional 
assumptions.   If proper transformations cannot be found, then nonparametric Wilcoxon signed 
rank tests will be performed.  To assess changes scores over time, linear mixed effect models 
will be fit and estimates of the average change per month will be calculated.  Specific contrasts 
will also be created to estimate changes at 12 and 16 weeks relative to baseline and 8 weeks.    
Analysis will be performed using SAS software (The SAS Institute; Cary, NC).  A significance level 
of 0.05 will be assumed for all tests.  Correction for multiple testing is not planned, since the 
purpose of the present study is estimation. 
Sample Size Considerations 

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The primary assessment tool will be the CDASI.  Scores for the CDASI range from 0 to132 for the 
total score.  In the paper by Yassaee (29), investigators found a mean (SD) total score of 20.10 
(10.25).  Given the apparent skewed distributions for this score, a log-normal distribution for 
the scores was assumed.  Based on this estimate, a coefficient of variation (CV = Standard 
Deviation/Mean) of 0.6 appears appropriate.   
Table 1 shows the minimum detectable decreases in CDASI scores, based on the assumptions 
above, and assuming use of two-sided paired t-tests on log-transformed values.  These 
calculations further assume that a moderate positive correlation (r=0.5) between baseline and 
follow-up scores within person, and use of a 0.05 significance level.  Under these assumptions, 
15 patients are need to detect a 35% decrease in the mean CDASI activity score or CDASI total 
score with 80% power.  Using the study by Yassaee as a reference, if the mean total score was 
20, then there would be adequate power to detect decreases of 7 points in the mean total 
score.  To detect smaller differences, the sample size increases rapidly.  For example, to detect 
a 25% mean decrease (5 points) in CDASI score 29 patients would be required, while to see a 2 
point mean difference (10% decrease), 203 patients would be needed. 
These calculations do not incorporate drop out, so additional subjects may need to be enrolled 
if some patients fail to reach the 8 week follow-up period.  Note that if variability is larger in the 
present study or correlation differs between group, the detectable differences may vary from 
the measures shown below.   
 
Table 1.  Minimum detectable differences are shown to detect specified differences in the 
mean CDASI scales at 8 weeks with 80% power and significance level of 0.05. 
 
Sample Size 
Scale 
10 
15 
20 
CDASI Total Score (CV = 0.6) 
40% 
35% 
30% 
 
Adverse Events and Data Monitoring Committee (DMC) 
Patients will be instructed to call immediately with any adverse events during the 
administration of 1.5% ruxolitinib cream.  The Principal Investigator (PI) must report all Serious 

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Adverse Events (SAEs) to Incyte within 24 hours of learning of an event, regardless of the PI’s 
causality assessment. This notification should be provided on a completed Serious Adverse 
Event (SAE) form.  SAE reporting for each subject begins the day the informed consent is signed 
by the patient and within 30 days after subject has completed or discontinued from the study 
or has taken last dose of the study drug, or as described in the protocol. 
SAEs, occurring using Incyte study drug, are reported in accordance with the effective protocol.  
SAEs occurring with any other commercial drug are reported to the manufacturer of that drug 
in accordance with regulations and protocol.  
Initial SAEs and/or subsequent follow-up reports should be reported via email to 
SafetyReporting@Incyte.com  or fax (+) 1-866-981-2057.  SAE reports should be for a single 
subject.  SAE forms should be sent with a cover sheet and any additional attachments. 
All adverse event information is reported to Incyte on the Principal Investigator’s/Institution’s 
Adverse Event Report Form, or a CIOMS-I or MedWatch Form FDA 3500A, or on an Adverse 
Event.    Any serious adverse events will also be reported to the IRB in accordance with their 
policy. 
• An interim analysis is not definitively planned, but will be considered for 
presentation purposes. 
Consent 
Consent will be obtained in a clinical setting at the baseline clinical appointment by a member 
of the study team at the time of planned enrollment.  Vulnerable subjects will not be enrolled 
in this clinical study. 
 
 
References 
 
1. Bailey EE, Fiorentino DF. Amyopathic dermatomyositis: definitions, diagnosis, and 
management. Curr Rheumatol Rep. 2014 Dec;16(12):465.  
 

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2.  Robinson ES, Feng R, Okawa J, et al. Improvement in the cutaneous disease activity of 
patients with dermatomyositis is associated with a better quality of life. Br J Dermatol 
2015;172:169–74. 
 
3 Huber AM, Kim S, Reed AM et al. Childhood Arthritis and Rheumatology Research Alliance 
Consensus Clinical treatment plans for juvenile dermatomyositis with persistent skin rash. J 
Rheumatol 2017; 44: 110–16. 
 
4 Sanner H, Sjaastad I, Flatø B. Disease activity and prognostic factors in juvenile 
dermatomyositis: a long-term follow-up study applying the Paediatric Rheumatology 
International Trials Organization criteria for inactive disease and the myositis disease activity 
assessment tool. Rheumatol Oxf Engl 2014; 53: 1578–85. 
 
5 Seshadri R, Feldman BM, Ilowite N et al. The role of aggressive corticosteroid therapy in 
patients with juvenile dermatomyositis: a propensity score analysis. Arthritis Rheum 2008; 59: 
989–95. 
 
6. Wolstencroft PW, Chung L, Li S, et al. Factors associated with clinical remission of skin disease 
in dermatomyositis. JAMA Dermatol 2018;154:44–51. 
 
7. Ladislau L, Suárez-Calvet X, Toquet S, Landon-Cardinal O, Amelin D, Depp M, Rodero MP, 
Hathazi D, Duffy D, Bondet V, Preusse C, Bienvenu B, Rozenberg F, Roos A, Benjamim CF, 
Gallardo E, Illa I, Mouly V, Stenzel W, Butler-Browne G, Benveniste O, Allenbach Y. JAK inhibitor 
improves type I interferon induced damage: proof of concept in dermatomyositis. Brain. 2018 
Jun 1;141(6):1609-1621. 
 
8. You H, Xu D, Zhao J, Li J, Wang Q, Tian X, Li M, Zeng X. JAK Inhibitors: Prospects in Connective 
Tissue Diseases. Clin Rev Allergy Immunol. 2020 Dec;59(3):334-351. 
 
9.  Hornung T, Janzen V, Heidgen FJ, et al. Remission of recalcitrant dermatomyositis treated 
with ruxolitinib. N Engl J Med 2014;371:2537–8. 
 
10. Kurtzman DJ, Wright NA, Lin J, et al. Tofacitinib citrate for refractory cutaneous 
dermatomyositis: an alternative treatment. JAMA Dermatol 2016;152: 944–5. 
 
11.  Paik JJ, Casciola-Rosen L, Shin JY, Albayda J, Tiniakou E, Leung DG, Gutierrez-Alamillo L, 
Perin J, Florea L, Antonescu C, Leung SG, Purwin G, Koenig A, Christopher-Stine L. Study of 
Tofacitinib In Refractory Dermatomyositis (STIR): An open label pilot study of 10 patients. 
Arthritis Rheumatol. 2020 Dec 1. Epub ahead of print. 
 
12.  Dawkins MA, Jorizzo JL, Walker FO, Albertson D, Sinal SH, Hinds A. Dermatomyositis: a 
dermatology-based case series. J Am Acad Dermatol. 1998 Mar;38(3):397-404. 
 

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13. García-Doval I, Cruces M. Topical tacrolimus in cutaneous lesions of dermatomyositis: lack of 
effect in side-by-side comparison in five patients. Dermatology. 2004;209(3):247-8. 
 
14. Barnes L, Kaya G, Rollason V. Topical corticosteroid-induced skin atrophy: a comprehensive 
review. Drug Saf. 2015 May;38(5):493-509. 
 
15. Hengge UR, Ruzicka T, Schwartz RA, Cork MJ. Adverse effects of topical glucocorticosteroids. 
J Am Acad Dermatol. 2006 Jan;54(1):1-15. 
 
16. Kim BS, Howell MD, Sun K, Papp K, Nasir A, Kuligowski ME; INCB 18424-206 Study 
Investigators. Treatment of atopic dermatitis with ruxolitinib cream (JAK1/JAK2 inhibitor) or 
triamcinolone cream. J Allergy Clin Immunol. 2020 Feb;145(2):572-582 
 
17.  Kim BS, Sun K, Papp K, Venturanza M, Nasir A, Kuligowski ME. Effects of ruxolitinib cream 
on pruritus and quality of life in atopic dermatitis: Results from a phase 2, randomized, dose-
ranging, vehicle- and active-controlled study. J Am Acad Dermatol. 2020 Jun;82(6):1305-1313. 
 
18.  Papp, K., Szepietowski, J. C., Kircik, L., Toth, D., Kuligowski, M. E., Venturanza, M. E., Sun, K., 
& Simpson, E. L. (2020). Efficacy and Safety of Ruxolitinib Cream for the Treatment of Atopic 
Dermatitis: Results From Two Phase 3, Randomized, Double-Blind Studies. SKIN The Journal of 
Cutaneous Medicine, 4(6), s95. 
 
19. Bohan A, Peter JB.  Polymyositis and dermatomyositis.  N Engl J Med 1975; 292(7): 344-7. 
 
20.  Bohan A, Peter JB.  Polymyositis and dermatomyositis.  N Engl J Med 1975; 292(8): 403-7. 
 
21.  Gerami P, Schope JM, McDonald L, Walling HW, Sontheimer RD.  A systematic review of 
adult-onset clinically amyopathic dermatomyositis (dermatomyositis sine myositis): a missing 
link within the spectrum of the idiopathic inflammatory myopathies.  J Am Acad Dermatol 2006; 
54: 597-613. 
 
22.  Euwer RL, Sontheimer RD.  Amyopathic dermatomyositis: a review.  J Invest Dermatol 
1993; 100: 124S-127S. 
 
23.  Klein RQ, Bangert CA, Costner M, Connolly MK, et al.  Comparison of the reliability and 
validity of outcome instruments for cutaneous dermatomyositis.  Br J Dermatol 2008; 159: 887-
894. 
 
24.  Goreshi R, Okawa J, Rose M, Feng R, et al.  Evaluation of reliability, validity, and 
responsiveness of the CDASI and CAT-BM.  J Invest Dermatol 2012; 132: 1117-1124. 
 
25.  Carroll CL, Lang W, Snively B, Feldman SR et al.  Development and validation of the 
dermatomyositis skin severity index.  Br J Dermatol 2008; 158: 345-350. 
 

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Version date 3/29/2023 
26.  Rider LG, Werth VP, Huber AM, Alexanderson H et al.  Measures of adult and juvenile 
dermatomyositis, polymyositis, and inclusion body myositis.  Arth Care Res 2011; 63(S11): S118-
S157. 
 
27.  Rider LG, Koziol D, Giannini EH, Jain MS et al.  Validation of manual muscle testing and a 
subset of eight muscles for adult and juvenile idiopathic inflammatory myopathies.  Arth Care 
and Res 2010; 465-472. 
 
28. Thomas CL, Finlay AY. The 'handprint' approximates to 1% of the total body surface area 
whereas the 'palm minus the fingers' does not. Br J Dermatol. 2007 Nov; 157(5): 1080-1. 
 
29.  Yassaee M, Fiorentino D, Okawa J, Taylor L, Coley AB. Troxel AB, Werth VP.  Modificiation of 
the Cutaneous Dermatomyositis Disease Area and Severity Index, an outcome instrument.  Br J 
Dermatol 2010; 162:  669-673.