Trial Outcomes & Findings for Tofacitinib Hypothesis-generating, Pilot Study for Corticosteroid-Dependent Sarcoidosis (NCT NCT03793439)

Last Updated: 2022-02-18

Results Overview

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

Recruitment status

COMPLETED

Study phase

PHASE1

Target enrollment

5 participants

Primary outcome timeframe

16 weeks

Results posted on

2022-02-18

Participant Flow

Participant milestones

Participant milestones
Measure	Open Label Tofacitinib 5 mg twice daily by mouth
Overall Study STARTED	5
Overall Study COMPLETED	3
Overall Study NOT COMPLETED	2

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

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

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Open-label Treatment n=5 Participants 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. Tofacitinib 5mg Oral Tablet \[Xeljanz\] 16 week trial: Tofacitinib 5mg oral table twice daily for 16 weeks Spirometry: Spirometry testing at baseline, week 4, week 8, week 12, and week 16 RNA Sequencing: RNA sequencing test at baseline and week 16 Laboratory testing: Laboratory testing at baseline and weeks 2, 4, 8, 12 and 16 Corticosteroid: Taper corticosteroids starting at week 4 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.
Age, Continuous	40.8 years n=93 Participants
Sex: Female, Male Female	1 Participants n=93 Participants
Sex: Female, Male Male	4 Participants n=93 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=93 Participants
Race (NIH/OMB) Asian	0 Participants n=93 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=93 Participants
Race (NIH/OMB) Black or African American	1 Participants n=93 Participants
Race (NIH/OMB) White	4 Participants n=93 Participants
Race (NIH/OMB) More than one race	0 Participants n=93 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants n=93 Participants
Region of Enrollment United States	5 participants n=93 Participants

PRIMARY outcome

Timeframe: 16 weeks

Outcome measures

Outcome measures
Measure	Open Label n=5 Participants open label tofacitinib
Number of Participants With 50% Reduction in Corticosteroid Requirement	3 Participants

SECONDARY outcome

Timeframe: 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.

Outcome measures

Outcome measures
Measure	Open Label n=2 Participants open label tofacitinib
Number of Participants With Significantly Decreased Expression of STAT1 Mediated Genes as Determined by RNA Sequencing	0 Participants

Adverse Events

Open Label

Serious events: 2 serious events

Other events: 4 other events

Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure	Open Label n=5 participants at risk open label tofacitinib
Nervous system disorders Peripheral motor neuropathy	20.0% 1/5 • Number of events 1 • Adverse event data were collected from week 0 (start of study drug) until study drug was stopped, plus 4 weeks of post-treatment follow up. This corresponded to 72 weeks for subjects who completed the study and open-label extension: study (16 weeks), open-label extension (52 weeks), and post-treatment follow up (4 weeks). Adverse event data from subjects who terminated the study early were collected until study drug was stopped + 4 weeks.
Skin and subcutaneous tissue disorders Skin infection	20.0% 1/5 • Number of events 1 • Adverse event data were collected from week 0 (start of study drug) until study drug was stopped, plus 4 weeks of post-treatment follow up. This corresponded to 72 weeks for subjects who completed the study and open-label extension: study (16 weeks), open-label extension (52 weeks), and post-treatment follow up (4 weeks). Adverse event data from subjects who terminated the study early were collected until study drug was stopped + 4 weeks.

Other adverse events

Other adverse events
Measure	Open Label n=5 participants at risk open label tofacitinib
Skin and subcutaneous tissue disorders Rash maculo-papular	20.0% 1/5 • Number of events 1 • Adverse event data were collected from week 0 (start of study drug) until study drug was stopped, plus 4 weeks of post-treatment follow up. This corresponded to 72 weeks for subjects who completed the study and open-label extension: study (16 weeks), open-label extension (52 weeks), and post-treatment follow up (4 weeks). Adverse event data from subjects who terminated the study early were collected until study drug was stopped + 4 weeks.
Gastrointestinal disorders Vomiting	20.0% 1/5 • Number of events 1 • Adverse event data were collected from week 0 (start of study drug) until study drug was stopped, plus 4 weeks of post-treatment follow up. This corresponded to 72 weeks for subjects who completed the study and open-label extension: study (16 weeks), open-label extension (52 weeks), and post-treatment follow up (4 weeks). Adverse event data from subjects who terminated the study early were collected until study drug was stopped + 4 weeks.
Musculoskeletal and connective tissue disorders Arthralgia	40.0% 2/5 • Number of events 2 • Adverse event data were collected from week 0 (start of study drug) until study drug was stopped, plus 4 weeks of post-treatment follow up. This corresponded to 72 weeks for subjects who completed the study and open-label extension: study (16 weeks), open-label extension (52 weeks), and post-treatment follow up (4 weeks). Adverse event data from subjects who terminated the study early were collected until study drug was stopped + 4 weeks.
Gastrointestinal disorders Diarrhea	20.0% 1/5 • Number of events 1 • Adverse event data were collected from week 0 (start of study drug) until study drug was stopped, plus 4 weeks of post-treatment follow up. This corresponded to 72 weeks for subjects who completed the study and open-label extension: study (16 weeks), open-label extension (52 weeks), and post-treatment follow up (4 weeks). Adverse event data from subjects who terminated the study early were collected until study drug was stopped + 4 weeks.
Respiratory, thoracic and mediastinal disorders Allergic rhinitis	20.0% 1/5 • Number of events 1 • Adverse event data were collected from week 0 (start of study drug) until study drug was stopped, plus 4 weeks of post-treatment follow up. This corresponded to 72 weeks for subjects who completed the study and open-label extension: study (16 weeks), open-label extension (52 weeks), and post-treatment follow up (4 weeks). Adverse event data from subjects who terminated the study early were collected until study drug was stopped + 4 weeks.
Respiratory, thoracic and mediastinal disorders Cough	20.0% 1/5 • Number of events 1 • Adverse event data were collected from week 0 (start of study drug) until study drug was stopped, plus 4 weeks of post-treatment follow up. This corresponded to 72 weeks for subjects who completed the study and open-label extension: study (16 weeks), open-label extension (52 weeks), and post-treatment follow up (4 weeks). Adverse event data from subjects who terminated the study early were collected until study drug was stopped + 4 weeks.
Renal and urinary disorders Renal colic	20.0% 1/5 • Number of events 1 • Adverse event data were collected from week 0 (start of study drug) until study drug was stopped, plus 4 weeks of post-treatment follow up. This corresponded to 72 weeks for subjects who completed the study and open-label extension: study (16 weeks), open-label extension (52 weeks), and post-treatment follow up (4 weeks). Adverse event data from subjects who terminated the study early were collected until study drug was stopped + 4 weeks.
Respiratory, thoracic and mediastinal disorders Bronchial infection	20.0% 1/5 • Number of events 1 • Adverse event data were collected from week 0 (start of study drug) until study drug was stopped, plus 4 weeks of post-treatment follow up. This corresponded to 72 weeks for subjects who completed the study and open-label extension: study (16 weeks), open-label extension (52 weeks), and post-treatment follow up (4 weeks). Adverse event data from subjects who terminated the study early were collected until study drug was stopped + 4 weeks.

Additional Information

Dr. Marcia Friedman

Oregon Health & Science University

Phone: 5034948637

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place