The purpose of this study is to investigate the role of the oral JAK1 inhibitor, abrocitinib 200 mg once daily, for the treatment of patients with moderate to severe cutaneous sarcoidosis.
To determine if JAK1 specific inhibition is effective in treating sarcoidosis, an inflammatory condition that can cause disfiguring skin lesions. The only FDA approved therapy is prednisone, a nonspecific immunosuppressant. The primary outcome will be the percent change in the Cutaneous Sarcoidosis Activity and Morphology Instrument (CSAMI) after 6 months of abrocitinib 200 mg daily in 10 patients with moderate to severe cutaneous sarcoidosis.The primary objective of this study is to determine whether oral abrocitinib reduces the CSAMI (a cutaneous sarcoid clinical scoring tool) in patients with moderate to severe cutaneous sarcoidosis. The CSAMI tool was selected over other scoring metrics given the highest interrater reliability of this metric in cutaneous sarcoidosis and the ability to generate subscores for active disease versus scarring. Secondary outcomes will include percent changes in organ involvement on whole body PET-CT imaging, Patients reported outcomes 1) Sarcoidosis related quality-of-life (QoL) metric (King's Sarcoidosis Questionnaire), 2) Skin related quality of life (Skindex-16) metric, 3) sarcoidosis Fatigue Assessment Scale (FAS), and 4) Rhinosinusitis Disability Index (RSDI). Secondary outcomes will also include evaluation of molecular signatures before and during treatment. Soluble IL-2 receptor levels in plasma are the most reliable known biomarker for sarcoidosis and will be assessed at 0 and 6 months. Additional secondary outcomes will include correlative immunologic changes in skin tissue and blood as a result of treatment. Documentation of any spontaneously reported adverse events will be completed throughout the study.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
10
Abrocitinib (Cibinqo) is FDA approved at 200 mg dose once daily for the treatment of atopic dermatitis. It is not currently FDA approved for the treatment of sarcoidosis.
Yale University
New Haven, Connecticut, United States
Changes in Cutaneous Sarcoidosis Activity and Morphology Instrument (CASMI) score
Improvement in the Cutaneous Sarcoidosis Activity and Morphology Instrument (CSAMI) after 6 months of abrocitinib therapy. CSAMI is a validated clinical scoring tool for cutaneous sarcoidosis activity and severity. Scores range from 0 to 165. Higher score is worse.
Time frame: Baseline and 6 Months
Percent Changes in Improvement in Internal Organ Involvement
Internal organ involvement and activity will be determined using whole-body positron emission tomography - computed tomography (PET-CT) at baseline and after 6 months of treatment. Change in active inflammation in internal organs will be assessed by calculating total lesion glycolysis (TLG) in affected internal organs. Scores range from 0 to \[no upper limit\]. Higher score is worse.
Time frame: Baseline and 6 Months
Changes in Patient Reported Outcomes Quality of Life (King's Sarcoidosis Questionnaire)
This tool assesses overall quality of life related to sarcoidosis through a questionnaire. Scores range from 1 to 100. Higher score is better.
Time frame: Baseline and 6 Months
Changes in Skin related quality of life (Skindex-16)
Skindex-16 is a validated quality of life metric which assesses the effect of diseases involving the skin on individuals' quality of life. It is assessed using a questionnaire. Scores range from 0-96. Higher score is worse.
Time frame: Baseline and 6 Months
Changes in Fatigue Assessment Scale (FAS)
FAS is a validated quality of life metric which assesses the effect of sarcoidosis on fatigue / energy level. It is assessed using a questionnaire. Scores range from 0 to 50. Higher score is worse.
Time frame: Baseline and 6 Months
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Changes in Rhinosinustitis Disability Index (RSDI)
RSDI is a validated quality of life metric which assesses the effect sarcoidosis-related nasal sinus symptoms on quality of life. It is assessed using a questionnaire. Scores range from 0 to 120. Higher Score is worse.
Time frame: Baseline and 6 months