Trial Outcomes & Findings for Musculoskeletal Ultrasound Assessment of Therapeutic Response of Tofacitinib in Rheumatoid Arthritis Patients (NCT NCT02321930)

NCT ID: NCT02321930

Last Updated: 2019-03-19

Results Overview

30 joints will be evaluated using a 0 to 3 point scale for each joint and the sum of these represents PDUS. The Power Doppler Synovitis Score (PDUS) ranges from 0 to 90. Scores of 0 indicate the least amount of inflammation of the joint while scores of 3 indicate the most amount of inflammation. A higher value of the total score for PDUS represents more severe disease level.

• Recruitment status: COMPLETED
• Study phase: PHASE4
• Target enrollment: 37 participants
• Primary outcome timeframe: Baseline, Week 2, Month 3
• Results posted on: 2019-03-19

Participant Flow

Patients will be recruited from the UCLA Rheumatology Clinics. The patients meeting inclusion criteria were started on tofacitinib 5mg po bid. Patients were recruited starting on 2/16/2016. The recruitment continued through 5/17/2017.

12 did not pass inclusion criteria, these patients were not able to be assigned to a study arm

Participant milestones

Measure	Tofacitinib 5mg po Bid All subjects will receive tofacitinib. Tofacitinib: Subjects will receive tofacitinib 5mg po bid
Overall Study STARTED	25
Overall Study COMPLETED	24
Overall Study NOT COMPLETED	1

Reasons for withdrawal

Measure	Tofacitinib 5mg po Bid All subjects will receive tofacitinib. Tofacitinib: Subjects will receive tofacitinib 5mg po bid
Overall Study Lost to Follow-up	1

Baseline Characteristics

Musculoskeletal Ultrasound Assessment of Therapeutic Response of Tofacitinib in Rheumatoid Arthritis Patients

Baseline characteristics by cohort

Measure	Tofacitinib 5mg po Bid n=25 Participants All subjects will receive tofacitinib. Tofacitinib: Subjects will receive tofacitinib 5mg po bid
Age, Continuous	52 years STANDARD_DEVIATION 9.93 • n=93 Participants
Sex: Female, Male Female	22 Participants n=93 Participants
Sex: Female, Male Male	3 Participants n=93 Participants
Race/Ethnicity, Customized Caucasian	10 Participants n=93 Participants
Race/Ethnicity, Customized Black	4 Participants n=93 Participants
Race/Ethnicity, Customized Hispanic	5 Participants n=93 Participants
Race/Ethnicity, Customized Asian	4 Participants n=93 Participants
Race/Ethnicity, Customized Other	2 Participants n=93 Participants
Region of Enrollment United States	25 participants n=93 Participants
Disease duration	10.36 years STANDARD_DEVIATION 9.73 • n=93 Participants
Health Assessment Questionnaire- Disability Index (HAQ-DI)	1.37 units on a scale STANDARD_DEVIATION 0.70 • n=93 Participants
Seropositive	22 Participants n=93 Participants
BMI	31.02 Kg/m^2 STANDARD_DEVIATION 10.76 • n=93 Participants

PRIMARY outcome

Timeframe: Baseline, Week 2, Month 3

30 joints will be evaluated using a 0 to 3 point scale for each joint and the sum of these represents PDUS. The Power Doppler Synovitis Score (PDUS) ranges from 0 to 90. Scores of 0 indicate the least amount of inflammation of the joint while scores of 3 indicate the most amount of inflammation. A higher value of the total score for PDUS represents more severe disease level.

Outcome measures

Measure	Tofacitinib 5mg po Bid n=25 Participants One arm only. All subjects will receive tofacitinib. Tofacitinib: Subjects will receive tofacitinib 5mg po bid
PDUS Baseline	28.68 units on a scale Standard Deviation 17.75
PDUS Week 2	19.52 units on a scale Standard Deviation 15.38
PDUS Week 12	12.17 units on a scale Standard Deviation 10.64

PRIMARY outcome

Timeframe: Baseline, Week 2, Month 3

30 joints will be evaluated using a 0 to 3 point scale for each joint and the sum of these represents GSUS. The grey scale synovial hypertrophy score (GSUS) ranges from 0 to 90. Scores of 0 indicate the least amount of inflammation of the joint while scores of 3 indicate the most amount of inflammation. A higher value of the total score for GSUS represents more severe disease level.

Outcome measures

Measure	Tofacitinib 5mg po Bid n=25 Participants One arm only. All subjects will receive tofacitinib. Tofacitinib: Subjects will receive tofacitinib 5mg po bid
GSUS Baseline	48.44 units on a scale Standard Deviation 16.53
GSUS Week 2	44.88 units on a scale Standard Deviation 16.36
GSUS Week 12	37.92 units on a scale Standard Deviation 15.32

PRIMARY outcome

Timeframe: Baseline, Week 2, Month 3

CDAI was derived as the sum of the following: tender joint count (TJC), swollen joint count (SJC), participant global assessment (PGA) of disease activity, and physician assessment of disease activity. TJC and SJC were taken as the number of tender and swollen joints, respectively, out of 28 assessed joints. PGA and physician assessment of disease activity were scored 0-100 millimeters (mm) and rounded to the nearest centimeter (cm) on a visual analog scale (VAS), where higher scores indicate greater perceived disease activity. The total CDAI score range was 0-76, where higher scores indicate increased disease activity.

Outcome measures

Measure	Tofacitinib 5mg po Bid n=25 Participants One arm only. All subjects will receive tofacitinib. Tofacitinib: Subjects will receive tofacitinib 5mg po bid
CDAI Baseline	39.88 units on a scale Standard Deviation 13.21
CDAI Week 2	28.60 units on a scale Standard Deviation 13.23
CDAI Week 12	21.63 units on a scale Standard Deviation 13.02

PRIMARY outcome

Timeframe: Baseline, Week 2, Month 3

TJC and SJC were taken as the number of tender and swollen joints, respectively, out of 28 assessed joints. Patient Global Assessment of disease activity was scored 0-100 millimeters (mm) and rounded to the nearest centimeter (cm) on a visual analog scale (VAS), where higher scores indicate greater perceived disease activity. ESR lab value were included in the total calculation. The scale being used is called the Disease Activity Score for 28 Joints (DAS28) using the Erythrocyte Sedimentation Rate (ESR) in the calculation.The scale ranges from 0 to 9.4, where higher values represent a higher disease activity.The complete formula to calculate DAS28/ESR is 0.56*sqrt(TJC) + (0.28*sqrt(SJC)) + (0.7*ln(ESR)) + (0.014*Patient Global*10).

Outcome measures

Measure	Tofacitinib 5mg po Bid n=25 Participants One arm only. All subjects will receive tofacitinib. Tofacitinib: Subjects will receive tofacitinib 5mg po bid
DAS28/ESR Baseline	6.26 units on a scale Standard Deviation 1.19
DAS28/ESR Week 2	5.23 units on a scale Standard Deviation 1.31
DAS28/ESR Week 12	4.56 units on a scale Standard Deviation 1.36

SECONDARY outcome

Timeframe: Baseline, Week 2, Month 3

The multi-biomarker disease activity (MBDA) blood test assesses RA disease activity. An algorithm of 12 markers is used to characterize RA disease activity on a scale of 1-100, where a score of 100 represents the highest level of disease activity present.

Outcome measures

Measure	Tofacitinib 5mg po Bid n=25 Participants One arm only. All subjects will receive tofacitinib. Tofacitinib: Subjects will receive tofacitinib 5mg po bid
MBDA Baseline	50.56 units on a scale Standard Deviation 17.50
MBDA Week 2	40.96 units on a scale Standard Deviation 15.10
MBDA Week 12	39.63 units on a scale Standard Deviation 15.28

Adverse Events

Tofacitinib 5mg po Bid

Serious events: 1 serious events

Other events: 24 other events

Deaths: 0 deaths

Serious adverse events

Measure	Tofacitinib 5mg po Bid n=25 participants at risk All subjects will receive tofacitinib. Tofacitinib: Subjects will receive tofacitinib 5mg po bid
Blood and lymphatic system disorders Lymphoma	4.0% 1/25 • Number of events 1 • 1.5 years

Other adverse events

Measure	Tofacitinib 5mg po Bid n=25 participants at risk All subjects will receive tofacitinib. Tofacitinib: Subjects will receive tofacitinib 5mg po bid
Respiratory, thoracic and mediastinal disorders Upper Respiratory Infection	40.0% 10/25 • 1.5 years
Musculoskeletal and connective tissue disorders Rheumatoid Arthritis Flare	28.0% 7/25 • 1.5 years
Blood and lymphatic system disorders Dyslipidemia	28.0% 7/25 • 1.5 years
Gastrointestinal disorders Heartburn	12.0% 3/25 • 1.5 years
Gastrointestinal disorders Constipation	12.0% 3/25 • 1.5 years
Gastrointestinal disorders Diarrhea	12.0% 3/25 • 1.5 years
General disorders Headaches	12.0% 3/25 • 1.5 years
Gastrointestinal disorders Loose Stool	12.0% 3/25 • 1.5 years
Respiratory, thoracic and mediastinal disorders Sore Throat	12.0% 3/25 • 1.5 years
Gastrointestinal disorders Abdominal Discomfort	8.0% 2/25 • 1.5 years
Vascular disorders Bruises on Different Body Sites	8.0% 2/25 • 1.5 years
General disorders Dizziness/Nausea	8.0% 2/25 • 1.5 years
Respiratory, thoracic and mediastinal disorders Dry Cough	8.0% 2/25 • 1.5 years
General disorders Hair Loss	8.0% 2/25 • 1.5 years

Additional Information

Veena K. Ranganath

UCLA

Phone: 310-825-4321

Email: vranganath@mednet.ucla.edu

Results disclosure agreements

• Principal investigator is a sponsor employee
• Publication restrictions are in place