An Open-Label Pilot Study to Evaluate the Safety, Tolerability, and Efficacy of Tofacitinib in Patients With Stevens-Johnson Syndrome (SJS) and Toxic Epidermal Necrolysis (TEN)
Phase: NA
Conditions: Stevens-Johnson Syndrome, Toxic Epidermal Necrolysis
Sponsor: Chang Gung Memorial Hospital

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Study To Evaluate The Efficacy Of Tofacitinib In  
Patients With SJS/TEN 
ClinicalTrials.gov ID NCT06474078 
Sponsor Chang Gung Memorial Hospital 
Information provided by Chun Bing Chen, Chang Gung Memorial Hospital (Responsible Party) 
 
Study Overview 
Brief Summary 
The goal of this study is to evaluate the effect of tofacitinib in patients with Stevens-Johnson Syndrome 
(SJS) and Toxic Epidermal Necrolysis (TEN). The primary outcome of the study is the time to 
complete re-epithelialization. The secondary outcomes are to determine mortality, length of 
hospitalization, adverse events, the time to beginning of epithelization, the time to halting of 
progression of SJS/TEN, ocular complications, and infections. 
 
Detailed Description 
Stevens-Johnson syndrome (SJS) and toxic epidermal necrolysis (TEN) belong to life-threatening 
severe cutaneous adverse drug reactions. SJS/TEN is classified by the extent of the detachment over 
the total body-surface area: SJS (<10%), SJS-TEN overlap (10%-30%), and TEN (>30%). TEN has 
the highest mortality (30-35%); SJS and transitional forms correspond to the same syndrome, but with 
less extensive skin detachment and a lower mortality (5-15%). Currently, there is still no conclusive 
effective immunomodulator treatment for SJS/TEN. And, there is still a clinical unmet need for the 
treatment of SJS/TEN. 
According to our past research reports, interleukin-15 (IL-15) plays an important role in SJS/TEN, 
which is related to disease severity and mortality. Janus kinase (JAK) inhibitors can inhibit the 
downstream JAK to inhibit the production and transmission of inflammatory cytokines, as a treatment 
for severe skin drug hypersensitivity reactions. Tofacitinib, a JAK1/3 inhibitor, is an intervention 
known to effectively treat several inflammatory diseases including rheumatoid arthritis, psoriatic 
arthritis, and ulcerative colitis. Notably, a recent study identified a potential therapeutic target with 
JAK-STAT pathway in a patient with recalcitrant and refractory drug rash with eosinophilia and 
systemic symptoms (DRESS). Based the current evidence, a targeting therapy to IL-15 by tofacitinib 
treatment are suggesting likely to be effective in treating SJS/TEN. 

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This is an "open label" study, all patients enrolled in the study will receive the active medication; 
meaning that there will not be a placebo or control group. The aims of this project are (1) to investigate 
the effect of tofacitinib treatment for SJS/TEN patients, including healing time, mortality rate, adverse 
events, beginning of re-epithelialization time, internal organ recovery time, and ocular complications, 
and (2) to investigate the molecular mechanism of SJS/TEN after tofacitinib treatment through 
collection of timed samples to include DNA, RNA, PBMCs, blister cells and supernatant and skin 
tissue. 
The primary outcome of the study is the time to complete re-epithelialization as defined by complete 
absence of erosion on the skin. The secondary outcomes are to determine the time to beginning of 
epithelization (defined as the time to start re-epithelialization of the erosions on the skin and mucosa), 
the time to halting of progression of SJS/TEN (considered significant progression if there are any new 
blistering lesions or any new detached or detachable skin), mortality, length of hospitalization, ocular 
complications, infections, and adverse events. The investigators also determine the molecular and 
cellular mechanisms of SJS/TEN through collection of timed samples to include DNA, RNA, PBMCs, 
blister cells and supernatant and skin tissue. 
Official Title 
An Open-Label Pilot Study to Evaluate the Safety, Tolerability, 
and Efficacy of Tofacitinib in Patients With Stevens-Johnson Syndrome (SJS) and Toxic Epidermal 
Necrolysis (TEN) 
 
Intervention / Treatment  
Drug: Tofacitinib 
 
Other Study ID Numbers  
IRB No. 202102411A3 
 
Contacts and Locations 
This section provides the contact details for those conducting the study, and information on where 
this study is being conducted. 
Study Contact  (Project Leader) 
Name: Chun Bing Chen 
Phone Number:03-3281200 ext 8494 
Email: chunbing.chen@gmail.com 
 
Study Contact Backup (Co-Investigator) 

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Name: Wen-Hung Chung                      Name:  Chuang-Wei Wang 
Phone Number:03-3281200 ext 8495            Phone Number: 03-3281200 ext 7784 
Email: wenhungchung@yahoo.com             Email: kiruamairo@gmail.com 
 
 
Participation Criteria 
Researchers look for people who fit a certain description, called eligibility criteria. Some examples of 
these criteria are a person's general health condition or prior treatments. 
 
Eligibility Criteria 
Description 
Inclusion Criteria: 
1. Willing to sign inform consent form 
2. Subject has been diagnosed with Stevens-Johnson syndrome or toxic epidermal necrolysis by 
at least two dermatologists. 
3. Male or female aged over 20 years old and under 90 years old. 
Exclusion Criteria: 
1. Subject or legally authorized representative is not willing to provide informed consent. 
2. Women who are pregnant or breastfeeding 
3. Subject has an active, untreated, or serious infectious disease that is ineffective in treatment, 
such as sepsis. 
4. Subject suffers from severe life-threatening cardiac arrhythmia, such as ventricular tachycardia, 
have had myocardial infarction (myocardial infarction), severe hypertension that has not 
responded to treatment within the past week, or other cardiologist diagnosed severe 
cardiovascular disease 
5. Subject has active viral hepatitis 
6. Subject has active tuberculosis 
7. Subject received live vaccination during the illness 
 
 

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Study Protocol  
This section provides details of the study plan, including how the study is designed and what the study 
is measuring. 
 
How is the study designed? 
Design Details 
Primary Purpose  : Treatment 
Allocation  : N/A 
Interventional Model  : Single Group Assignment 
Masking  : None (Open Label) 
Arms and Interventions 
 
 
 
 
 

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What is the study measuring? 
Primary Outcome Measures  
 
Secondary Outcome Measures  
 
Study endpoint 
The endpoint of this clinical trial was defined as the absence of serious or treatment-related adverse 
events within three weeks after the final tofacitinib dose. Most SJS/TEN patients were monitored for 
3–4 weeks post-treatment, with the total observation period spanning 1–2 months. Patients without 
AEs during this window were considered to have completed the trial and met the safety endpoint. 
 
Study duration  
Start Date from 2022-08-01 to 2025-07-31 
 
Statistical Analysis Plan 
For evaluating the time taken to heal skin erosion, the Kaplan-Meier product-limit estimates method 
was performed.  
 
Collaborators and Investigators 
This is where you will find people and organizations involved with this study. 
Sponsor  
Chang Gung Memorial Hospital 
Investigators  

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 
Principal Investigator: Chun Bing Chen, Chang Gung Memorial Hospital