Tofacitinib Hypothesis-generating Pilot Study for Corticosteroid-Dependent Sarcoidosis

  • STATUS
    Recruiting
  • End date
    Mar 1, 2022
  • participants needed
    5
  • sponsor
    Oregon Health and Science University
Updated on 24 January 2021

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.

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.

Details
Condition Lymphoproliferative Disorder, Sarcoidosis, Pulmonary sarcoidosis, Lymphoproliferative disorders, sarcoidosis, pulmonary, sarcoid
Treatment spirometry, Corticosteroid, RNA sequencing, Tofacitinib 5mg Oral Tablet [Xeljanz] 16 week trial, Laboratory testing, Tofacitinib 5mg [Xeljanz] 1 year open-label extension
Clinical Study IdentifierNCT03793439
SponsorOregon Health and Science University
Last Modified on24 January 2021

Eligibility

Yes No Not Sure

Inclusion Criteria

Meet World Association of Sarcoidosis and other Granulomatous Disorders (WASOG) definition of pulmonary sarcoid
Histologically proven sarcoid
Evidence of pulmonary sarcoid on chest radiograph
FVC of > 50%
Require 15-30mg/day of prednisone or equivalent corticosteroid to control sarcoidosis
Stable dose of prednisone or equivalent corticosteroid for 4 weeks prior to enrollment

Exclusion Criteria

May be taking methotrexate but not other immunosuppressive or immunomodulatory treatments in the two months prior to study period. This includes but is not limited to azathioprine, cyclophosphamide, leflunomide, mycophenolate mofetil, cyclosporine, tacrolimus, and biologic medications
Patients requiring >30mg/day prednisone or equivalent
Pregnant or lactating women
Hemoglobin < 9g/dL or hematocrit < 30%
White blood cell count <3.0 K/cu mm
Absolute neutrophil count <1.2 K/cu mm
Platelet count <100 K/cu mm
Subjects with an estimated glomerular filtration rate (GFR) 40 ml/min
Subjects with a total bilirubin, aspartate aminotransferase (AST), or alanine aminotransferase (ALT) more than 1.5 times the upper limit of normal at screening
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)
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