Trial Outcomes & Findings for Safety of Tofacitinib, an Oral Janus Kinase Inhibitor, in Primary Sjogren's Syndrome (NCT NCT04496960)
NCT ID: NCT04496960
Last Updated: 2025-12-18
Results Overview
Count of adverse events by grade was assessed using the National Cancer Institute (NCI), Common Terminology Criteria for Adverse Events (CTCAE), Version 5.0. Grade 1: Mild; asymptomatic or mild symptoms; clinical or diagnostic observations only; intervention not indicated. Grade 2: Moderate; minimal, local or noninvasive intervention indicated; limiting age appropriate instrumental ADL. Grade 3: Severe or medically significant but not immediately life-threatening; hospitalization or prolongation of hospitalization indicated; disabling; limiting self care ADL. Grade 4: Life-threatening consequences; urgent intervention indicated. Grade 5: Death related to adverse event. Serious is defined as any grade 3 or higher adverse event. Toxicity is defined as any study drug-related Grade 3 or higher adverse event.
ACTIVE_NOT_RECRUITING
PHASE1/PHASE2
23 participants
Up to day 196
2025-12-18
Participant Flow
23 Participants were consented * 12 participants started the study * 11 participants were screen failure
Participant milestones
| Measure |
Drug: Tofacitinib
Sjogren's Disease (SjD) patients with mild to moderate disease activity receive tofacitinib 5 mg orally twice daily for 168 days.
|
Placebo
Sjogren's Disease (SjD) patients with mild to moderate disease activity receive placebo orally twice daily for 168 days.
|
|---|---|---|
|
Overall Study
STARTED
|
8
|
4
|
|
Overall Study
COMPLETED
|
8
|
3
|
|
Overall Study
NOT COMPLETED
|
0
|
1
|
Reasons for withdrawal
| Measure |
Drug: Tofacitinib
Sjogren's Disease (SjD) patients with mild to moderate disease activity receive tofacitinib 5 mg orally twice daily for 168 days.
|
Placebo
Sjogren's Disease (SjD) patients with mild to moderate disease activity receive placebo orally twice daily for 168 days.
|
|---|---|---|
|
Overall Study
Physician Decision
|
0
|
1
|
Baseline Characteristics
Safety of Tofacitinib, an Oral Janus Kinase Inhibitor, in Primary Sjogren's Syndrome
Baseline characteristics by cohort
| Measure |
Drug: Tofacitinib
n=8 Participants
Sjogren's Disease (SjD) patients with mild to moderate disease activity receive tofacitinib 5 mg orally twice daily for 168 days.
|
Placebo
n=4 Participants
Sjogren's Disease (SjD) patients with mild to moderate disease activity receive placebo orally twice daily for 168 days.
|
Total
n=12 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Categorical
<=18 years
|
0 Participants
n=47 Participants
|
0 Participants
n=41 Participants
|
0 Participants
n=88 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
7 Participants
n=47 Participants
|
3 Participants
n=41 Participants
|
10 Participants
n=88 Participants
|
|
Age, Categorical
>=65 years
|
1 Participants
n=47 Participants
|
1 Participants
n=41 Participants
|
2 Participants
n=88 Participants
|
|
Sex: Female, Male
Female
|
8 Participants
n=47 Participants
|
4 Participants
n=41 Participants
|
12 Participants
n=88 Participants
|
|
Sex: Female, Male
Male
|
0 Participants
n=47 Participants
|
0 Participants
n=41 Participants
|
0 Participants
n=88 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
2 Participants
n=47 Participants
|
0 Participants
n=41 Participants
|
2 Participants
n=88 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
6 Participants
n=47 Participants
|
4 Participants
n=41 Participants
|
10 Participants
n=88 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=47 Participants
|
0 Participants
n=41 Participants
|
0 Participants
n=88 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=47 Participants
|
0 Participants
n=41 Participants
|
0 Participants
n=88 Participants
|
|
Race (NIH/OMB)
Asian
|
1 Participants
n=47 Participants
|
0 Participants
n=41 Participants
|
1 Participants
n=88 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=47 Participants
|
0 Participants
n=41 Participants
|
0 Participants
n=88 Participants
|
|
Race (NIH/OMB)
Black or African American
|
0 Participants
n=47 Participants
|
0 Participants
n=41 Participants
|
0 Participants
n=88 Participants
|
|
Race (NIH/OMB)
White
|
6 Participants
n=47 Participants
|
4 Participants
n=41 Participants
|
10 Participants
n=88 Participants
|
|
Race (NIH/OMB)
More than one race
|
1 Participants
n=47 Participants
|
0 Participants
n=41 Participants
|
1 Participants
n=88 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=47 Participants
|
0 Participants
n=41 Participants
|
0 Participants
n=88 Participants
|
PRIMARY outcome
Timeframe: Up to day 196Population: Intent to treat population
Count of adverse events by grade was assessed using the National Cancer Institute (NCI), Common Terminology Criteria for Adverse Events (CTCAE), Version 5.0. Grade 1: Mild; asymptomatic or mild symptoms; clinical or diagnostic observations only; intervention not indicated. Grade 2: Moderate; minimal, local or noninvasive intervention indicated; limiting age appropriate instrumental ADL. Grade 3: Severe or medically significant but not immediately life-threatening; hospitalization or prolongation of hospitalization indicated; disabling; limiting self care ADL. Grade 4: Life-threatening consequences; urgent intervention indicated. Grade 5: Death related to adverse event. Serious is defined as any grade 3 or higher adverse event. Toxicity is defined as any study drug-related Grade 3 or higher adverse event.
Outcome measures
| Measure |
Drug: Tofacitinib
n=33 Count of adverse events
Sjogren's Disease (SjD) patients with mild to moderate disease activity receive tofacitinib 5 mg orally twice daily for 168 days.
|
Placebo
n=12 Count of adverse events
Sjogren's Disease (SjD) patients with mild to moderate disease activity receive placebo orally twice daily for 168 days.
|
|---|---|---|
|
Number of Adverse Events by Grade/Category
Grade 1
|
19 Count of adverse events
|
3 Count of adverse events
|
|
Number of Adverse Events by Grade/Category
Grade 2
|
10 Count of adverse events
|
7 Count of adverse events
|
|
Number of Adverse Events by Grade/Category
Grade 3
|
4 Count of adverse events
|
2 Count of adverse events
|
|
Number of Adverse Events by Grade/Category
Grade 4
|
0 Count of adverse events
|
0 Count of adverse events
|
|
Number of Adverse Events by Grade/Category
Grade 5
|
0 Count of adverse events
|
0 Count of adverse events
|
|
Number of Adverse Events by Grade/Category
Serious
|
4 Count of adverse events
|
1 Count of adverse events
|
|
Number of Adverse Events by Grade/Category
Toxicity
|
4 Count of adverse events
|
0 Count of adverse events
|
PRIMARY outcome
Timeframe: Up to day 196Population: Intent to treat population
Number participants with any adverse events by grade and severity was assessed using the National Cancer Institute (NCI), Common Terminology Criteria for Adverse Events (CTCAE), Version 5.0. Grade 1 Mild; asymptomatic or mild symptoms; clinical or diagnostic observations only; intervention not indicated. Grade 2 Moderate; minimal, local or noninvasive intervention indicated; limiting age appropriate instrumental activity of daily living (ADL). Grade 3 Severe or medically significant but not immediately life-threatening; hospitalization or prolongation of hospitalization indicated; disabling; limiting self care ADL. Grade 4 Life-threatening consequences; urgent intervention indicated. Grade 5 Death related to adverse event. Serious is defined as any grade 3 or higher adverse event. Toxicity is defined as any study drug-related Grade 3 or higher adverse event.
Outcome measures
| Measure |
Drug: Tofacitinib
n=8 Participants
Sjogren's Disease (SjD) patients with mild to moderate disease activity receive tofacitinib 5 mg orally twice daily for 168 days.
|
Placebo
n=4 Participants
Sjogren's Disease (SjD) patients with mild to moderate disease activity receive placebo orally twice daily for 168 days.
|
|---|---|---|
|
Participants With Adverse Events
Subjects with at least one adverse event
|
7 Participants
|
3 Participants
|
|
Participants With Adverse Events
Subjects with at least one adverse event with grade 3 and above
|
1 Participants
|
1 Participants
|
|
Participants With Adverse Events
Subjects with at least one Severe adverse event (SAE)
|
1 Participants
|
1 Participants
|
|
Participants With Adverse Events
Subjects with at least one toxicity
|
1 Participants
|
0 Participants
|
SECONDARY outcome
Timeframe: Day 168 minus day 1Population: Intent to treat population
The Physician Global Activity (PGA) is a subjective physician reported disease activity index that uses a 10-cm Visual Analog Scale (VAS) to score a patient's disease activity. The 10-cm visual analogue scale (VAS) was scored by a physician with a vertical line on the scale marking disease activity where 0 cm indicates no evidence of disease activity, and 10 cm indicates severe disease activity, with score reported on a scale from 0-10. Higher score indicates more disease activity. Change in disease activity index was measured as the mean difference in disease activity scores between time points. Data was analyzed as the change in score between day 1 (baseline) and study day 168 (Change = day 168 - day 1) for the treatment/placebo groups as repeated measures.
Outcome measures
| Measure |
Drug: Tofacitinib
n=8 Participants
Sjogren's Disease (SjD) patients with mild to moderate disease activity receive tofacitinib 5 mg orally twice daily for 168 days.
|
Placebo
n=3 Participants
Sjogren's Disease (SjD) patients with mild to moderate disease activity receive placebo orally twice daily for 168 days.
|
|---|---|---|
|
Change in Physicians Global Assessment (PGA) Score
|
0 Units on a scale
Standard Deviation 3.07
|
-1.67 Units on a scale
Standard Deviation 2.89
|
SECONDARY outcome
Timeframe: Day 168 minus day 1Population: Intent to treat population
The EULAR Sjögren's syndrome (SS) disease activity index (ESSDAI) is a systemic disease activity index designed to measure disease activity in patients with primary SS. It includes 12 organ domains (e.g., constitutional, glandular, neurological) for disease activity, each with a 4-point scale (0 = No activity; 3=High activity). These scores are then multiplied by domain-specific weights (1-6) and summed to produce a total score ranging from 0 to 123, with higher scores indicating greater disease activity. Data was analyzed as the change in score between day 1 (baseline) and study day 168 (Change = day 168 - day 1) for the treatment/placebo groups as repeated measures.
Outcome measures
| Measure |
Drug: Tofacitinib
n=8 Participants
Sjogren's Disease (SjD) patients with mild to moderate disease activity receive tofacitinib 5 mg orally twice daily for 168 days.
|
Placebo
n=3 Participants
Sjogren's Disease (SjD) patients with mild to moderate disease activity receive placebo orally twice daily for 168 days.
|
|---|---|---|
|
Change in EULAR Sjögren's Syndrome (SS) Disease Activity Index (ESSDAI) Score
|
-0.13 Units on a scale
Standard Deviation 1.13
|
-1.33 Units on a scale
Standard Deviation 2.31
|
SECONDARY outcome
Timeframe: Day 168 minus day 1Population: Intent to treat population
Changes in salivary flow was assessed by measuring whole unstimulated saliva flow (WUS) and glandular parotid and submandibular/sublingual unstimulated and 0.2% citric acid stimulated flow rates using ultrasonograph. Validated scoring criteria was used to assess ultrasonographic feature parameters at baseline and at the study day 168. Data was analyzed as the change in score between day 1 (baseline) and study day 168 (Change = day 168 - day 1) for the treatment/placebo groups as repeated measures.
Outcome measures
| Measure |
Drug: Tofacitinib
n=8 Participants
Sjogren's Disease (SjD) patients with mild to moderate disease activity receive tofacitinib 5 mg orally twice daily for 168 days.
|
Placebo
n=3 Participants
Sjogren's Disease (SjD) patients with mild to moderate disease activity receive placebo orally twice daily for 168 days.
|
|---|---|---|
|
Change in Whole Unstimulated Saliva Flow
|
-0.13 ml/min/gland
Standard Deviation 1.13
|
-1.33 ml/min/gland
Standard Deviation 2.31
|
SECONDARY outcome
Timeframe: Day 168 minus day 1Population: Intent to treat population
Changes in salivary flow was assessed by measuring whole stimulated saliva flow and glandular parotid and submandibular/sublingual stimulated and 0.2% citric acid stimulated flow rates using ultrasonograph. Validated scoring criteria was used to assess ultrasonographic feature parameters at baseline and at the study day 168. Data was analyzed as the change in score between day 1 (baseline) and study day 168 (Change = day 168 - day 1) for the treatment/placebo groups as repeated measures.
Outcome measures
| Measure |
Drug: Tofacitinib
n=8 Participants
Sjogren's Disease (SjD) patients with mild to moderate disease activity receive tofacitinib 5 mg orally twice daily for 168 days.
|
Placebo
n=3 Participants
Sjogren's Disease (SjD) patients with mild to moderate disease activity receive placebo orally twice daily for 168 days.
|
|---|---|---|
|
Change in Whole Stimulated Saliva Flow
|
10.6 ml/min/gland
Standard Deviation 1.13
|
6.21 ml/min/gland
Standard Deviation 2.31
|
SECONDARY outcome
Timeframe: Day 168 minus day 1Population: Intent to treat population
The EULAR Sjögren's Syndrome (SS) Patient Reported Index (ESSPRI) is a patient-reported outcome measure (PROM) that focuses on the subjective experience of symptoms in SS. The tool assesses the key symptoms of dryness, pain and fatigue. A single 0-10 numerical scale is used to assess each of these symptoms. Final score is the average of the scores from the three domains. A final ESSPRI score of less than 5 is considered indicative of acceptable disease status, while a score of 5 or higher suggests high disease activity. Questionnaire was completed at baseline and day 168. Data was analyzed as
Outcome measures
| Measure |
Drug: Tofacitinib
n=8 Participants
Sjogren's Disease (SjD) patients with mild to moderate disease activity receive tofacitinib 5 mg orally twice daily for 168 days.
|
Placebo
n=3 Participants
Sjogren's Disease (SjD) patients with mild to moderate disease activity receive placebo orally twice daily for 168 days.
|
|---|---|---|
|
Change in EULAR Sjögren's Syndrome (SS) Patient Reported Index (ESSPRI)
|
-1.33 Units on a scale
Standard Deviation 2.04
|
0.78 Units on a scale
Standard Deviation 0.51
|
Adverse Events
Drug: Tofacitinib
Placebo
Serious adverse events
| Measure |
Drug: Tofacitinib
n=8 participants at risk
Sjogren's Disease (SjD) patients with mild to moderate disease activity receive tofacitinib 5 mg orally twice daily for 168 days.
|
Placebo
n=3 participants at risk
Sjogren's Disease (SjD) patients with mild to moderate disease activity receive placebo orally twice daily for 168 days.
|
|---|---|---|
|
Investigations
Neutrophil count decreased
|
12.5%
1/8 • Up to day 196
|
33.3%
1/3 • Up to day 196
|
Other adverse events
| Measure |
Drug: Tofacitinib
n=8 participants at risk
Sjogren's Disease (SjD) patients with mild to moderate disease activity receive tofacitinib 5 mg orally twice daily for 168 days.
|
Placebo
n=3 participants at risk
Sjogren's Disease (SjD) patients with mild to moderate disease activity receive placebo orally twice daily for 168 days.
|
|---|---|---|
|
Surgical and medical procedures
Spinal fusion surgery
|
0.00%
0/8 • Up to day 196
|
33.3%
1/3 • Up to day 196
|
|
Infections and infestations
Vaginal infection
|
0.00%
0/8 • Up to day 196
|
33.3%
1/3 • Up to day 196
|
|
Injury, poisoning and procedural complications
Barotrauma
|
12.5%
1/8 • Up to day 196
|
0.00%
0/3 • Up to day 196
|
|
Investigations
Neutrophil count decreased
|
12.5%
1/8 • Up to day 196
|
0.00%
0/3 • Up to day 196
|
|
Musculoskeletal and connective tissue disorders
Arthralgia
|
12.5%
1/8 • Up to day 196
|
0.00%
0/3 • Up to day 196
|
|
Musculoskeletal and connective tissue disorders
Back pain
|
12.5%
1/8 • Up to day 196
|
0.00%
0/3 • Up to day 196
|
|
Musculoskeletal and connective tissue disorders
Myalgia
|
12.5%
1/8 • Up to day 196
|
0.00%
0/3 • Up to day 196
|
|
Nervous system disorders
Headache
|
0.00%
0/8 • Up to day 196
|
33.3%
1/3 • Up to day 196
|
|
Respiratory, thoracic and mediastinal disorders
Nasal congestion
|
0.00%
0/8 • Up to day 196
|
33.3%
1/3 • Up to day 196
|
|
Respiratory, thoracic and mediastinal disorders
Reflux laryngitis
|
12.5%
1/8 • Up to day 196
|
0.00%
0/3 • Up to day 196
|
|
Respiratory, thoracic and mediastinal disorders
Upper respiratory tract infection
|
62.5%
5/8 • Up to day 196
|
33.3%
1/3 • Up to day 196
|
|
Skin and subcutaneous tissue disorders
Rash maculo-papular
|
12.5%
1/8 • Up to day 196
|
0.00%
0/3 • Up to day 196
|
|
Gastrointestinal disorders
Abdominal pain
|
12.5%
1/8 • Up to day 196
|
0.00%
0/3 • Up to day 196
|
|
Gastrointestinal disorders
Diarrhoea
|
12.5%
1/8 • Up to day 196
|
0.00%
0/3 • Up to day 196
|
|
Gastrointestinal disorders
Nausea
|
0.00%
0/8 • Up to day 196
|
33.3%
1/3 • Up to day 196
|
|
Gastrointestinal disorders
Salivary gland calculus
|
12.5%
1/8 • Up to day 196
|
0.00%
0/3 • Up to day 196
|
|
Gastrointestinal disorders
Vomiting
|
12.5%
1/8 • Up to day 196
|
0.00%
0/3 • Up to day 196
|
|
Immune system disorders
Allergy to arthropod sting
|
12.5%
1/8 • Up to day 196
|
0.00%
0/3 • Up to day 196
|
|
Infections and infestations
Herpes simplex reactivation
|
0.00%
0/8 • Up to day 196
|
33.3%
1/3 • Up to day 196
|
|
Infections and infestations
BK virus infection
|
12.5%
1/8 • Up to day 196
|
0.00%
0/3 • Up to day 196
|
|
Infections and infestations
Conjunctivitis
|
0.00%
0/8 • Up to day 196
|
33.3%
1/3 • Up to day 196
|
|
Infections and infestations
Oral candidiasis
|
12.5%
1/8 • Up to day 196
|
33.3%
1/3 • Up to day 196
|
|
Infections and infestations
Otitis media
|
12.5%
1/8 • Up to day 196
|
0.00%
0/3 • Up to day 196
|
|
Infections and infestations
Urinary tract infection
|
25.0%
2/8 • Up to day 196
|
0.00%
0/3 • Up to day 196
|
Additional Information
Dr. Blake Warner
National Institute of Dental and Craniofacial Research
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place