Tofacitinib Hypothesis-generating, Pilot Study for Corticosteroid-Dependent Sarcoidosis

• Register: ClinicalTrials.gov
• Last refreshed on: 12 December 2020
• Main ID: NCT03793439
• Date of registration: 02/01/2019
• Prospective Registration: Yes
• Primary sponsor: Oregon Health and Science University
• Public title: Tofacitinib Hypothesis-generating, Pilot Study for Corticosteroid-Dependent Sarcoidosis
• Scientific title: Tofacitinib Hypothesis-generating, Pilot Study for Corticosteroid-Dependent Sarcoidosis
• Date of first enrolment: May 15, 2019
• Target sample size: 5
• Recruitment status: Recruiting
• URL: https://clinicaltrials.gov/show/NCT03793439
• 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: Marcia A Friedman, MD

• Name: Jim Rosenbaum, MD
• Affiliation: Oregon Health and Science University

• Name: Marcia A Friedman, MD

Key inclusion & exclusion criteria

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).



Age minimum: 18 Years
Age maximum: 89 Years
Gender: All

Health Condition(s) or Problem(s) studied

Sarcoidosis Lung

Sarcoidosis

Sarcoidosis, Pulmonary

Intervention(s)

Drug: Tofacitinib 5mg Oral Tablet [Xeljanz] 16 week trial

Genetic: RNA Sequencing

Diagnostic Test: Spirometry

Drug: Corticosteroid

Drug: Tofacitinib 5mg [Xeljanz] 1 year open-label extension

Diagnostic Test: Laboratory testing

Primary Outcome(s)

50% reduction in corticosteroid requirement in at least 60% of subjects. [Time Frame: 16 weeks]

Secondary Outcome(s)

Significant decreases in of peripheral markers of STAT pathway activity [Time Frame: 16 weeks]

Secondary ID(s)

STUDY00017902

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Secondary Sponsor(s)

Pfizer