NCT03793439

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

• Status: Completed
• Phase: Phase 1
• Study type: Interventional
• Enrollment: 5 (actual)
• Sponsor: Oregon Health and Science University · Academic / Other
• Sex: All
• Age: 18 Years – 89 Years
• Healthy volunteers: Not accepted

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.

Detailed 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.

Conditions

• Sarcoidosis, Pulmonary
• Sarcoidosis Lung
• Sarcoidosis

Interventions

Type	Name	Description
DRUG	Tofacitinib 5mg Oral Tablet [Xeljanz] 16 week trial	Tofacitinib 5mg oral table twice daily for 16 weeks
DIAGNOSTIC_TEST	Spirometry	Spirometry testing at baseline, week 4, week 8, week 12, and week 16
GENETIC	RNA Sequencing	RNA sequencing test at baseline and week 16
DIAGNOSTIC_TEST	Laboratory testing	Laboratory testing at baseline and weeks 2, 4, 8, 12 and 16
DRUG	Corticosteroid	Taper corticosteroids starting at week 4
DRUG	Tofacitinib 5mg [Xeljanz] 1 year open-label extension	After 16 weeks, subjects who meet the primary end-point will be permitted an optional one year open-label extension.

Timeline

• Start date: 2019-05-15
• Primary completion: 2020-06-24
• Completion: 2021-06-24
• First posted: 2019-01-04
• Last updated: 2022-02-18
• Results posted: 2022-02-18

Locations

1 site across 1 country: United States

Regulatory

• FDA-regulated drug study