Patient-Reported Outcomes In Rheumatoid Arthritis Patients Treated With Tofacitinib Or Biological Disease-Modifying Antirheumatic Drugs (DMARDs) In Real Life Conditions

• ClinicalTrials.gov Identifier: NCT03073109
• Recruitment Status: Completed
• First Posted: March 8, 2017
• Results First Posted: October 22, 2020
• Last Update Posted: October 22, 2020
• Sponsor: Pfizer
• Information provided by (Responsible Party): Pfizer

Tracking Information

• First Submitted Date: January 3, 2017
• First Posted Date: March 8, 2017
• Results First Submitted Date: September 1, 2020
• Results First Posted Date: October 22, 2020
• Last Update Posted Date: October 22, 2020
• Actual Study Start Date: March 15, 2017
• Actual Primary Completion Date: September 16, 2019 (Final data collection date for primary outcome measure)

Current Primary Outcome Measures (submitted: October 20, 2020)

Change From Baseline in Routine Assessment of Patient Index Data 3 (RAPID3) Score at Month 6 [ Time Frame: Baseline, Month 6 ]

Disease activity was assessed using RAPID3, which was based on participant-reported multi-dimensional health assessment questionnaire (MDHAQ). RAPID3 included 3 core data set measures of physical function, pain, and patient global estimate. Physical function=mean of scores from 10 individual questions on activities of daily living (each question scored from '0'=no difficulty to '3'=much difficulty), scores were transformed to give a total score=0 to 10, higher scores=greater difficulty. Pain and global estimate of health measured on Likert scale from '0'=no pain/feeling very well to '10'=pain as bad as it could be/ feeling very poorly, higher scores=greater difficulty/ worsening of condition. RAPID3 composite score: mean of physical function, pain, and global assessment scores, ranging from 0 to 10, higher values=greater disease activity. Data has been provided in terms of adjusted (consider impact of co-variables) and unadjusted mean (does not consider co-variables impact).

• Original Primary Outcome Measures (submitted: March 2, 2017): Change from baseline in RAPID3 score at approximately month 6 [ Time Frame: Baseline, Month 6 ]

Current Secondary Outcome Measures (submitted: October 20, 2020)

• Adapted Health Assessment Questionnaire - Disability Index (HAQ-DI) Score at Month 6 [ Time Frame: Month 6 ]
  Functional status of participants was assessed using adapted HAQ-DI. Adapted HAQ-DI is a participant-reported questionnaire for the assessment of ability to perform tasks in 8 categories of daily living activities due to rheumatoid arthritis: dress/groom; arise; eat; walk; reach; grip; hygiene; and common activities over past week. Each item scored on 4-point Likert scale from 0 to 3, where 0=no difficulty; 1=some difficulty; 2=much difficulty; 3=unable to do. Overall score was computed as the sum of domain scores and divided by total number of domains answered. Total possible score ranged from 0 to 3, where 0 = least difficulty and 3 = extreme difficulty, higher scores indicating worse functioning. Data has been provided in terms of adjusted (consider impact of co-variables) and unadjusted mean (does not consider co-variables impact).
• Change From Baseline in Adapted Health Assessment Questionnaire - Disability Index (HAQ-DI) Score at Month 6 [ Time Frame: Baseline, Month 6 ]
  Functional status of participants was assessed using adapted HAQ-DI. Adapted HAQ-DI is a participant-reported questionnaire for the assessment of ability to perform tasks in 8 categories of daily living activities due to rheumatoid arthritis: dress/groom; arise; eat; walk; reach; grip; hygiene; and common activities over past week. Each item scored on 4-point Likert scale from 0 to 3, where 0=no difficulty; 1=some difficulty; 2=much difficulty; 3=unable to do. Overall score was computed as the sum of domain scores and divided by total number of domains answered. Total possible score ranged from 0 to 3, where 0 = least difficulty and 3 = extreme difficulty, higher scores indicating worse functioning. Data has been provided in terms of adjusted (consider impact of co-variable) and unadjusted mean (does not consider co-variable impact).
• European Quality of Life- 5 Dimension-3 Levels (EQ-5D-3L) Index Score at Month 6 [ Time Frame: Month 6 ]
  EQ-5D-3L is a standardized, participant-administered measure of self-reported health outcomes. It consists of two parts: EQ-5D index score (Part I) and the EQ-VAS (Part II). For Part I, i.e. EQ-5D-3L index score, participants rated their current health state on 5 single-item dimensions: mobility, self-care, usual activities, pain/discomfort, and anxiety/depression with each dimension having three levels of measure: 1= no problems, 2= some problems and 3= extreme problems. Scoring formula developed by EuroQol Group assigns a utility value for each domain in the profile. Score was transformed and results in a total index score range of 0 to 1.00; higher scores indicating a better health state. Data has been provided in terms of adjusted (consider impact of co-variables) and unadjusted mean (does not consider co-variables impact).
• Change From Baseline in European Quality of Life- 5 Dimension-3 Levels (EQ-5D-3L) Index Score at Month 6 [ Time Frame: Baseline, Month 6 ]
  EQ-5D-3L is a standardized, participant-administered measure of self-reported health outcomes. It consists of two parts: EQ-5D index score (Part I) and the EQ-VAS (Part II). For Part I, i.e. EQ-5D-3L index score, participants rated their current health state on 5 single-item dimensions: mobility, self-care, usual activities, pain/discomfort, and anxiety/depression with each dimension having three levels of function:. 1=no problems, 2=some problems and 3=extreme problems. Scoring formula developed by EuroQol Group assigns a utility value for each domain in the profile. Score was transformed and results in a total index score range of 0 to 1.00; higher scores indicating a better health state. Data has been provided in terms of adjusted (consider impact of co-variables) and unadjusted mean (does not consider co-variables impact).
• Work Productivity and Activity Impairment Questionnaire for Rheumatoid Arthritis (WPAI:RA) Scores at Month 6 [ Time Frame: Month 6 ]
  WPAI-RA: 6-questions participant rated questionnaire that measures effect of participant's RA on general health and symptom severity on work productivity and regular activities. Consisted of 6 questions, a binary question on current employment, 3 questions on hours of work and work-loss, and 2 questions based on 0-10 point scale to judge how RA affected productivity at work and outside of work (0 = no effect on work and 10 = completely prevented from working). It yielded four sub-scores: percent work time missed due to health (absenteeism), percent impairment while working (presenteeism), percent overall work impairment due to health (work productivity loss) and percent activity impairment due to health (daily activity impairment/loss). Sub-scores were expressed as an impairment percentage range from 0 to 100, where 0 = no impairment, 100 = completely impaired, higher scores=greater impairment and less productivity. Data has been provided in terms of adjusted and unadjusted mean.
• Change From Baseline in Work Productivity and Activity Impairment Questionnaire for Rheumatoid Arthritis (WPAI:RA) Scores at Month 6 [ Time Frame: Baseline, Month 6 ]
  WPAI-RA: 6-questions participant rated questionnaire that measures effect of participant's RA on general health and symptom severity on work productivity and regular activities. Consisted of 6 questions, a binary question on current employment, 3 questions on hours of work and work-loss, and 2 questions based on 0-10 point scale to judge how RA affected productivity at work and outside of work (0 = no effect on work and 10 = completely prevented from working). It yielded four sub-scores: percent work time missed due to health (absenteeism), percent impairment while working (presenteeism), percent overall work impairment due to health (work productivity loss) and percent activity impairment due to health (daily activity impairment/loss). Sub-scores were expressed as an impairment percentage range from 0 to 100, where 0 = no impairment, 100 = completely impaired, higher numbers=greater impairment and less productivity. Data has been provided in terms of adjusted and unadjusted mean.
• Disease Activity Score Based on 28-joints Count (DAS28) at Month 6 [ Time Frame: Month 6 ]
  DAS28 calculated from the number of swollen joints and painful joints using the 28 joints count, erythrocyte sedimentation rate (ESR) (measured in millimeters per hour [mm/hour]) and patient's global assessment of disease activity according to 100-millimeter (mm) Visual Analog Scale (VAS) (scores ranging 0 [very well] to 100 mm [extremely bad], higher scores=worsening of condition). DAS28-ESR scores were calculated as [0.56 × square root of TJC] + [0.28 × square root of SJC] + [0.70 × natural log (ESR)] + [0.014 × VAS]. DAS28 scores ranged from 0 to 10; higher scores indicated greater affectation due to disease activity. Data has been provided in terms of adjusted and unadjusted mean.
• Change From Baseline in Disease Activity Score Based on 28-joints Count (DAS28) at Month 6 [ Time Frame: Baseline, Month 6 ]
  DAS28 calculated from the number of swollen joints and painful joints using the 28 joints count, erythrocyte sedimentation rate (ESR) (measured in millimeters per hour [mm/hour]) and patient's global assessment of disease activity according to 100-millimeter (mm) Visual Analog Scale (VAS) (scores ranging 0 [very well] to 100 mm [extremely bad], higher scores=worsening of condition). DAS28-ESR scores were calculated as [0.56 × square root of TJC] + [0.28 × square root of SJC] + [0.70 × natural log (ESR)] + [0.014 × VAS]. DAS28 scores ranged from 0 to 10; higher scores indicated greater affectation due to disease activity. Data has been provided in terms of adjusted and unadjusted mean.
• Number of Participants With Serious Adverse Event (SAE) and Non-serious Adverse Events (AEs) [ Time Frame: Baseline up to Month 6 ]
  Adverse event (AE) was any untoward medical occurrence in a participant who received study drug without regard to possibility of causal relationship. AEs included both serious and non-serious adverse event. An SAE was an AE resulting in any of the following outcomes or deemed significant for any other reason: death; initial or prolonged inpatient hospitalization; life-threatening experience (immediate risk of dying); persistent or significant disability/incapacity; congenital anomaly.
• Number of Participants With Serious Infections [ Time Frame: Baseline up to Month 6 ]
  Serious infection defined as any infection that requires hospitalization.
• Number of Participants Who Withdrew From Study Due to Adverse Events And Due to All Causes [ Time Frame: Baseline up to Month 6 ]
  Here, number of participants who withdrew from study due to AEs and due to all causes (withdrawals due to AEs, lost to follow up and unspecified reasons) have been described.

Original Secondary Outcome Measures (submitted: March 2, 2017)

• Change form baseline in Health Assessment Questionnaire-Diseability Index (HAQ-DI) score at approximately month 6 [ Time Frame: Baseline, Month 6 ]
• Change from baseline in Heatlh Assessment Questionaire EQ-5D score at approximately month 6 [ Time Frame: Baseline, Month 6 ]
• Change from baseline in Work Productivity and Activity Impairment Questionnaire for Rheumatoid Arthritis (WPAI-RA) score at approximately 6 months [ Time Frame: Baseline, Month 6 ]
• Change from baseline in Disease Activity Score (DAS28) score at month 6 [ Time Frame: Baseline, Month 6 ]

• Current Other Pre-specified Outcome Measures: Not Provided
• Original Other Pre-specified Outcome Measures: Not Provided

Descriptive Information

• Brief Title: Patient-Reported Outcomes In Rheumatoid Arthritis Patients Treated With Tofacitinib Or Biological Disease-Modifying Antirheumatic Drugs (DMARDs) In Real Life Conditions
• Official Title: PATIENT-REPORTED OUTCOMES IN RHEUMATOID ARTHRITIS PATIENTS TREATED WITH TOFACITINIB OR BIOLOGICAL DISEASE-MODIFYING ANTIRHEUMATIC DRUGS (DMARDS) IN REAL LIFE CONDITIONS
• Brief Summary: This study is aimed to describe the outcomes related to physical activity, activity of disease, quality of life, work productivity and safety in Latin-American patients with Rheumatoid Arthritis (RA) treated with tofacitinib or biological DMARDs after failure to respond to conventional DMARDs in real-life conditions. This will be a non-interventional, hybrid study (prospective and retrospective data collection) comparing tofacitinib to biologic DMARD treatments in patients with RA after failure of conventional DMARDs. The population will be composed by adult patients over 18 years of age diagnosed with RA and who have been prescribed tofacitinib or any biological DMARDs.
• Detailed Description: Not Provided
• Study Type: Observational
• Study Design: Observational Model: Cohort; Time Perspective: Prospective
• Target Follow-Up Duration: Not Provided
• Biospecimen: Not Provided
• Sampling Method: Non-Probability Sample
• Study Population: Adult patients ≥ 18 years of age diagnosed with RA and who failed to respond to conventional DMARDs, and have been prescribed tofacitinib or any biological DMARDs
• Condition: Arthritis, Rheumatoid
• Intervention: Not Provided
• Study Groups/Cohorts: Not Provided
• Publications *: Not Provided

Recruitment Information

• Recruitment Status: Completed
• Actual Enrollment (submitted: October 20, 2020): 170
• Original Estimated Enrollment (submitted: March 2, 2017): 320
• Actual Study Completion Date: September 16, 2019
• Actual Primary Completion Date: September 16, 2019 (Final data collection date for primary outcome measure)

Eligibility Criteria

Inclusion Criteria:

• Patients ≥ 18 years of age at the time of recruitment
• Patient diagnosed with moderate to severe RA ≥ 6 months before enrollment
• Patients who have had an inadequate response to the continuous use of methotrexate or combination of conventional DMARDs for at least 12 weeks before the study without dose change within the last 8 weeks before enrollment in the study
• Patients with no biological DMARDs use in patient history.
• Patients prescribed with tofacitinib or biological DMARDs in the last two weeks at doses established in ACR guidelines published in 2015 and following medical criteria.
• Acceptance for patients to participate in the study and signing of the informed consent.

Exclusion Criteria:

• Patients who do not have the ability to answer the questionnaires by themselves or who have any kind of mental disorder that may affect their answers.
• Patients diagnosed with autoimmune rheumatic diseases other than RA and Sjogren's syndrome.
• Patients treated with biological DMARDs in monotherapy.
• Participation in other studies involving investigational drug(s) (Phases 1-4) within 4 weeks or 5 half-lives (whichever is longer) after discontinuation of the investigational compound before the current study begins and/or during study participation.
• Patients with any current malignancy or a history of malignancy, with the exception of adequately treated or excised non-metastatic basal cell or squamous cell cancer of the skin or cervical carcinoma in situ.
• Patients with lymphoproliferative disorders (e.g., Epstein Barr Virus (EBV) related lymphoproliferative disorder), a history of lymphoma, leukemia, or signs and symptoms suggestive of current lymphatic disease.

Sex/Gender

• Sexes Eligible for Study: All

• Ages: 18 Years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Contacts: Contact information is only displayed when the study is recruiting subjects
• Listed Location Countries: Colombia, Peru
• Removed Location Countries: Ecuador

Administrative Information

• NCT Number: NCT03073109
• Other Study ID Numbers: A3921284
• Has Data Monitoring Committee: No
• U.S. FDA-regulated Product: Not Provided

IPD Sharing Statement

• Plan to Share IPD: No
• Plan Description: Pfizer will provide access to individual de-identified participant data and related study documents (e.g. protocol, Statistical Analysis Plan (SAP), Clinical Study Report (CSR)) upon request from qualified researchers, and subject to certain criteria, conditions, and exceptions. Further details on Pfizer's data sharing criteria and process for requesting access can be found at: https://www.pfizer.com/science/clinical_trials/trial_data_and_results/data_requests.

• Responsible Party: Pfizer
• Study Sponsor: Pfizer
• Collaborators: Not Provided

Investigators

• Study Director: Pfizer CT.gov Call Center; Pfizer

• PRS Account: Pfizer
• Verification Date: October 2020