Open-label Trial of Tofacitinib in Cutaneous Sarcoidosis and Granuloma Annulare

• Register: ClinicalTrials.gov
• Last refreshed on: 26 July 2021
• Main ID: NCT03910543
• Date of registration: 04/04/2019
• Prospective Registration: Yes
• Primary sponsor: Yale University
• Public title: Open-label Trial of Tofacitinib in Cutaneous Sarcoidosis and Granuloma Annulare
• Scientific title: Open-label Trial of Tofacitinib in Cutaneous Sarcoidosis and Granuloma Annulare
• Date of first enrolment: April 11, 2019
• Target sample size: 15
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT03910543
• Study type: Interventional
• Study design: Allocation: Non-Randomized. Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).
• Phase: Phase 1

Countries of recruitment

United States

Contacts

• Name: William Damsky, MD, PhD
• Affiliation: Yale University

Key inclusion & exclusion criteria

Inclusion Criteria:

- 18 years old or older

- Diagnosis of cutaneous sarcoidosis or granuloma annulare with supportive skin biopsies
in which other causes of granulomas (infectious, foreign body) have been ruled out

- Patients with either: Cutaneous Sarcoidosis Activity and Morphology (CSAMI) activity
score greater than or equal to 10 (patients with a CSAMI greater than or equal to 10
have active cutaneous sarcoidosis involving several distinct cutaneous sites and would
otherwise be considered candidates for systemic therapy), or any CSAMI score and
sarcoidosis involvement causing functional impairment (i.e. nasal or visual field
obstruction).

- For patients with granuloma annulare, patients with 5% or greater Body Surface Area
(BSA) will be enrolled.

- If patients are on other systemic therapies for their sarcoidosis or granuloma
annulare, they must be taking a stable dose of the other medication(s) for at least 3
months with no plans to change the regimen in the next 6 months. With the exception of
methotrexate and/or low dose prednisone, use of concomitant immunosuppressants, e.g.
infliximab, azathioprine, etc., will not be permitted.

- Females of childbearing potential must agree to use birth control during the study and
there must be a negative pregnancy test documented prior to starting the medication.

- Patients must be willing to undergo skin biopsies, blood collection, and total body
photography and comply with clinic visits

Exclusion Criteria:

- Age <18 years old

- Patients with a history of malignancy (except history of successfully treated basal
cell or squamous cell carcinoma of the skin)

- Patients known to be HIV or hepatitis B (HBV) or C (HCV) positive (prior exposure to
but clearance of HBV and HCV is acceptable for study entry as long as patient is being
monitored by hepatology)

- Patients with active tuberculosis or untreated latent tuberculosis as determined by
positive tuberculin skin test or positive QuantiFERON® Tuberculosis (TB) test and, as
necessary, chest X-ray

- Patients with significant hepatic impairment

- Patients with untreated peptic ulcer disease

- Patients taking immunosuppressive medications, with the exception of methotrexate
and/or low- dose prednisone, including but not limited to mycophenolate mofetil,
azathioprine, tacrolimus, cyclosporine, or Tumor Necrosis Factor (TNF-a) inhibitors

- Women of childbearing potential who are unable or unwilling to use birth control while
taking the medication

- Women who are pregnant or nursing. If a woman becomes pregnant during the study, she
will stop study medication and be removed from the study. She will be urged to follow
up with her Primary Care Physician or OB/GYN. The study doctors will ask to follow the
pregnancy to its outcome.



Age minimum: 18 Years
Age maximum: N/A
Gender: All

Health Condition(s) or Problem(s) studied

Cutaneous Sarcoidosis

Granuloma Annulare

Intervention(s)

Drug: Tofacitinib 5 mg twice daily

Primary Outcome(s)

Change in Cutaneous Sarcoidosis Activity and Morphology Instrument (CSAMI) [Time Frame: 6 - 12 months]

Percent Change in Body Surface Area (BSA) involvement by GA lesions [Time Frame: 6 - 12 months]

Secondary Outcome(s)

Change in Histologic Findings [Time Frame: 6 -12 months]

Change in Skindex-16: a skin-related quality of life metric [Time Frame: 6 - 12 months]

Change in cytokine biomarkers [Time Frame: 6 - 12 months]

Change in activity of internal organ sarcoidosis [Time Frame: 6 - 12 months]

Change in RNA sequencing markers (gene expression analysis) [Time Frame: 6 - 12 months]

Secondary ID(s)

2000023910

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Secondary Sponsor(s)

Pfizer