Tofacitinib Hypothesis-generating, Pilot Study for Corticosteroid-Dependent Sarcoidosis
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|ClinicalTrials.gov Identifier: NCT03793439|
Recruitment Status : Completed
First Posted : January 4, 2019
Results First Posted : February 18, 2022
Last Update Posted : February 18, 2022
|Condition or disease||Intervention/treatment||Phase|
|Sarcoidosis, Pulmonary Sarcoidosis Lung Sarcoidosis||Drug: Tofacitinib 5mg Oral Tablet [Xeljanz] 16 week trial Diagnostic Test: Spirometry Genetic: RNA Sequencing Diagnostic Test: Laboratory testing Drug: Corticosteroid Drug: Tofacitinib 5mg [Xeljanz] 1 year open-label extension||Phase 1|
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.
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.
|Study Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||5 participants|
|Intervention Model:||Single Group Assignment|
|Intervention Model Description:||Open-label, interventional, proof of concept, hypothesis-generating study|
|Masking:||None (Open Label)|
|Official Title:||Tofacitinib Hypothesis-generating, Pilot Study for Corticosteroid-Dependent Sarcoidosis|
|Actual Study Start Date :||May 15, 2019|
|Actual Primary Completion Date :||June 24, 2020|
|Actual Study Completion Date :||June 24, 2021|
Experimental: Open-label treatment
All subjects will receive tofacitinib 5mg twice daily from week 0 to week 16, and a corticosteroid taper starting at week 16. Participants also undergo spirometry, RNA sequence testing, and laboratory evaluations.
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
Other Name: Pulmonary function test
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
Other Name: Blood work
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.
- Number of Participants With 50% Reduction in Corticosteroid Requirement [ Time Frame: 16 weeks ]50% reduction in corticosteroid requirement by week 16, without significant decline in their pulmonary function-defined as a >15% decline in forced vital capacity (FVC), forced expiratory volume at 1 second (FEV1), or diffusing capacity of lung for carbon monoxide (DLCO) relative to the baseline value
- Number of Participants With Significantly Decreased Expression of STAT1 Mediated Genes as Determined by RNA Sequencing [ Time Frame: 16 weeks ]Peripheral blood RNA sequencing performed before and after 16 weeks of tofacitinib treatment. Significant changes are defined as at least 1.5 fold change in the expression of STAT1-mediated genes, with a false discover rate p value of < 0.05.
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03793439
|United States, Oregon|
|Oregon Health & Science University|
|Portland, Oregon, United States, 97239|
|Principal Investigator:||Jim Rosenbaum, MD||Oregon Health and Science University|