Generate Real World Data On Tofacitinib Induction Therapy and Changes In Clinical and Patient Reported Outcomes. (KIC-START)

A LOW-INTERVENTIONAL, PROSPECTIVE, MULTI-CENTER STUDY TO EVALUATE REAL-WORLD CLINICAL, BIOCHEMICAL AND PATIENT-REPORTED RESPONSES TO TOFACITINIB INDUCTION THERAPY IN PATIENTS WITH MODERATELY TO SEVERELY ACTIVE ULCERATIVE COLITIS IN SWITZERLAND

This study is expected to contribute to the body of real-world data of tofacitinib's safety and efficacy profile in ulcerative colitis. Conventional clinical outcomes will give a better understanding of response and remission rates in a representative, post-marketing population.

Regular patient questionnaires and measurement of a biomarker of gut inflammation will provide detail on how patients experience induction treatment and contextualise the efficacy data.

Study Overview

• Status: Recruiting
• Conditions: Ulcerative Colitis
• Intervention / Treatment: Other: Stool sample collection

Detailed Description

This is a low-interventional study in which the intervention under study is home fecal calprotectin testing which falls outside of normal standard of care in ulcerative colitis. Tofacitinib is prescribed and administered as per the Swiss prescribing information. Accordingly, this study is registered on ClinicalTrials.gov as an interventional study. Under Swiss law, this study is considered and approved as a non-interventional study (Category A, Human Research Ordinance, Swiss Confederation).

• Study Type: Observational
• Enrollment (Estimated): 60

Contacts and Locations

• Study Contact: Name: Pfizer CT.gov Call Center; Phone Number: 1-800-718-1021; Email: ClinicalTrials.gov_Inquiries@pfizer.com

Study Locations

• Switzerland
  • Basel, Switzerland
    • Recruiting
    • Clarunis, Universitätsspital
  • Bern, Switzerland, CH - 3012
    • Recruiting
    • Verein IBD Study Group
  • Fribourg, Switzerland
    • Recruiting
    • Centre Fribourgeois de Gastroenterologie
  • Liestal, Switzerland
    • Recruiting
    • Kantonsspital Baselland
  • Zuerich, Switzerland
    • Not yet recruiting
    • Zentrum für Gastroenterologie und Hepatologie
  • Zurich, Switzerland, 8091
    • Not yet recruiting
    • University Hospital Zurich
  • Zurich, Switzerland
    • Not yet recruiting
    • University Hospital Zurich
  • Bern (de)
    • Bern, Bern (de), Switzerland, 3010
      • Not yet recruiting
      • Inselspital Bern
  • Sankt Gallen
    • St. Gallen, Sankt Gallen, Switzerland, 9007
      • Recruiting
      • Kantonsspital St, Gallen

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No

• Sampling Method: Non-Probability Sample

Study Population

60 adults both male and female who are prescribed tofacitinib for moderately to severely active UC will be enrolled in the study.

Description

Inclusion Criteria:

• Male or female participants 18 years of age or older at screening visit
• Participants with confirmed diagnosis of UC and who are prescribed tofacitinib (Xeljanz®) for moderately to severely active UC as per the Swiss label
• Participants who are willing and able to comply with all scheduled visits, treatment plan, study interventions, and other study procedures
• Capable of giving personally signed informed consent

Exclusion Criteria:

• Presence of clinical findings suggestive of Crohn's disease
• Any previous exposure to tofacitinib including participation in the tofacitinib clinical program
• Co-medication with any other advanced therapies for UC (biologics*, azathioprine, mercaptopurine and methotrexate) or any other JAK inhibitor
• Any identified contra-indications for use of tofacitinib as per the Swiss label
• Not owning a handheld digital device compatible with the Sidekick Health App, not willing to have it installed on this device or not capable of using the App
• Investigator site staff or Pfizer employees directly involved in the conduct of the study, site staff otherwise supervised by the investigator, and their respective family members.

Study Plan

How is the study designed?

Design Details

• Observational Models: Cohort
• Time Perspectives: Prospective

Number of groups / cohorts

1

Cohorts and Interventions

Group / Cohort	Intervention / Treatment
Patients with active Ulcerative Colitis	Other: Stool sample collection; collection for measuring calprotectin levels

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Proportion of participants achieving clinical response	Clinical response is defined as a reduction in the Partial Mayo Score from baseline of ≥2 points or achieving clinical remission. Clinical remission is defined as Partial Mayo Score of ≤ 2 with no subscore >1.	Week 8

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Proportion of participants achieving clinical remission	Clinical remission is defined as Partial Mayo Score of ≤ 2 with no subscore >1.	Week 8, Week 16
Proportion of participants achieving clinical response	Clinical response is defined as a reduction in the Partial Mayo Score from baseline of ≥2 points or achieving clinical remission. Clinical remission is defined as Partial Mayo Score of ≤ 2 with no subscore >1.	Week 16
Proportion of participants achieving Inflammatory Bowel Disease Questionnaire remission	Inflammatory Bowel Disease Questionnaire remission is defined as an Inflammatory Bowel Disease Questionnaire score ≥ 170. The Inflammatory Bowel Disease Questionnaire is a 32-item questionnaire grouped into four dimensions: bowel function, emotional status, systemic symptoms and social function. The total Inflammatory Bowel Disease Questionnaire score ranges from 32 to 224. For the total score and each domain, a higher score indicates better quality of life.	Week 8, Week 16
Proportion of participants achieving Inflammatory Bowel Disease Questionnaire response	Inflammatory Bowel Disease Questionnaire response is defined as an Inflammatory Bowel Disease Questionnaire score ≥16 points higher than Inflammatory Bowel Disease Questionnaire baseline score.	Week 8, Week 16
Proportion of participants achieving biochemical remission	Biochemical remission is defined as a fecal calprotectin concentration ≤250 mg/g. Fecal calprotectin is a small anti-microbial protein detected in stool that constitutes approximately 60% of neutrophil cytoplasm. As migration of neutrophils into the intestinal mucosa is a hallmark of active intestinal inflammation, fecal calprotectin serves as a noninvasive biomarker for intestinal inflammation.	Week 8, Week 16
Change from baseline in fecal calprotectin concentrations over time.	Median change from baseline in fecal calprotectin concentrations over time. Fecal calprotectin concentration is a continuous outcome.	Baseline to Week 16
Fecal calprotectin concentrations over time stratified by Week 8 clinical remission status	Median fecal calprotectin concentrations over time stratified by participants' Week 8 clinical remission status. Clinical remission status is either achieved or not achieved. Clinical remission is defined as Partial Mayo Score of ≤ 2 with no subscore >1.	Baseline to Week 16
Fecal calprotectin concentrations over time stratified by Week 16 clinical remission status	Median fecal calprotectin concentrations over time stratified by participants' Week 16 clinical remission status. Clinical remission status is either achieved or not achieved. Clinical remission is defined as Partial Mayo Score of ≤ 2 with no subscore >1.	Baseline to Week 16
Fecal calprotectin concentrations over time stratified by Week 8 clinical response status.	Median fecal calprotectin concentrations over time stratified by participants' Week 8 clinical response status. Clinical response status is either achieved or not achieved. Clinical response is defined as a reduction in the Partial Mayo Score from baseline of ≥2 points or achieving clinical remission. Clinical remission is defined as Partial Mayo Score of ≤ 2 with no subscore >1.	Baseline to Week 16
Fecal calprotectin concentrations over time stratified by Week 16 clinical response status	Median fecal calprotectin concentrations over time stratified by participants' Week 16 clinical response status. Clinical response status is either achieved or not achieved. Clinical response is defined as a reduction in the Partial Mayo Score from baseline of ≥2 points or achieving clinical remission. Clinical remission is defined as Partial Mayo Score of ≤ 2 with no subscore >1.	Baseline to Week 16
Fecal calprotectin concentrations stratified by Week 8 clinical response status	Median fecal calprotectin concentrations at Week 8 stratified by participants' Week 8 clinical response status. Clinical response status is either achieved or not achieved. Clinical response is defined as a reduction in the Partial Mayo Score from baseline of ≥2 points or achieving clinical remission. Clinical remission is defined as Partial Mayo Score of ≤ 2 with no subscore >1.	Week 8
Correlations between changes in amplitude and timing of Partial Mayo Score and Inflammatory Bowel Disease Questionnaire score	Correlations are assessed by the Spearman correlation coefficient.	Baseline to Week 16
Correlations between changes in amplitude and timing of Partial Mayo Score and fecal calprotectin concentration	Correlations are assessed by the Spearman correlation coefficient.	Baseline to Week 16
Correlations between changes in amplitude and timing of patient reported outcomes and fecal calprotectin concentration	Correlations are assessed by the Spearman correlation coefficient. There are eight individual patient reported outcomes: stool frequency, rectal bleeding, urgency of defecation, abdominal pain, quality of sleep, daily fatigue, weekly fatigue and quality of life.	Baseline to Week 16
Correlations between changes in amplitude and timing of patient reported outcomes and Inflammatory Bowel Disease Questionnaire score	Correlations are assessed by the Spearman correlation coefficient. There are eight individual patient reported outcomes: stool frequency, rectal bleeding, urgency of defecation, abdominal pain, quality of sleep, daily fatigue, weekly fatigue and quality of life.	Baseline to Week 16
Correlations between changes in amplitude and timing of Inflammatory Bowel Disease Questionnaire score and fecal calprotectin concentration.	Correlations are assessed by the Spearman correlation coefficient.	Baseline to Week 16
Change from baseline in stool frequency patient-reported outcome over time.	Median change from baseline in each stool frequency patient-reported outcome over time. The stool frequency patient-reported outcome is assessed with one question about the number of stools on a given day. The Mayo Score stool frequency subscore is used for scoring. Scores range from 0 to 3 and higher scores indicate more severe disease activity. Each participant serves as his or her own control to establish the degree of abnormality of stool frequency.	Baseline to Week 16
Change from baseline in rectal bleeding patient-reported outcome over time.	Median change from baseline in each rectal bleeding patient-reported outcome over time. The rectal bleeding patient-reported outcome is assessed with one question about most severe rectal bleeding on a given day. The Mayo Score rectal bleeding subscore is used for scoring. Scores range from 0 to 3 and higher scores indicate more severe disease activity. Each participant serves as his or her own control to establish the degree of rectal bleeding.	Baseline to Week 16
Change from baseline in urgency of defecation patient-reported outcome over time.	Median change from baseline urgency of defecation patient-reported outcome over time. The Urgency of defecation patient-reported outcome is assessed with the Urgency Numeric Rating Scale. Scoring is done on a 11-point numeric rating scale. Participant can input a score from 0 to 10. Higher scores indicate more severe urgency.	Baseline to Week 16
Change from baseline in abdominal pain patient-reported outcome over time.	Median change from baseline in abdominal pain patient-reported outcome over time. The abdominal pain patient-reported outcome is assessed with the Pain Numeric Rating Scale. Scoring is done on a 10-point horizontal numeric rating scale. Participant can input a score from 1 to 10. Higher scores indicate more severe pain.	Baseline to Week 16
Change from baseline in quality of sleep patient-reported outcome over time.	Median change from baseline in quality of sleep patient-reported outcome over time. The quality of sleep patient-reported outcome is assessed with a question from the Sleep Quality visual analogue scale survey. Scoring is done on a 11-point numeric rating scale. Participant can input a score from 0 to 10. Higher scores indicate better quality of sleep.	Baseline to Week 16
Change from baseline in daily fatigue patient-reported outcome over time.	Median change from baseline in daily fatigue patient-reported outcome over time. The daily fatigue patient-reported outcome is assessed with the Fatigue numeric rating scale. Scoring is done on a 11-point numeric rating scale. Participant can input a score from 0 to 10. Higher scores indicate more severe fatigue.	Baseline to Week 16
Change from baseline in weekly fatigue patient-reported outcome over time.	Median change from baseline in each weekly fatigue patient-reported outcome over time. The weekly fatigue patient-reported outcome is assessed with 13 questions from the ("Additional concerns" section) FACIT-F (Version 4). Scoring is done on a Responses are recorded on a 5-point Likert scale. Scores range from 0 to 52, with higher scores representing greater fatigue.	Baseline to Week 16
Change from baseline in Inflammatory Bowel Disease Questionnaire score over time.	The Inflammatory Bowel Disease Questionnaire is a 32-item questionnaire on quality of life. The total Inflammatory Bowel Disease Questionnaire score ranges from 32 to 224. For the total score and each domain, a higher score indicates better quality of life.	Baseline to Week 16
Correlations between changes in amplitude and timing of Partial Mayo Score and stool frequency patient-reported outco	Correlations are assessed by the Spearman correlation coefficient. The stool frequency patient-reported outcome is assessed with one question about the number of stools on a given day. The Mayo Score stool frequency subscore is used for scoring. Scores range from 0 to 3 and higher scores indicate more severe disease activity. Each participant serves as his or her own control to establish the degree of abnormality of stool frequency.	Baseline to Week 16
Correlations between changes in amplitude and timing of Partial Mayo Score and rectal bleeding patient-reported outcome.	Correlations are assessed by the Spearman correlation coefficient. The rectal bleeding patient-reported outcome is assessed with one question about most severe rectal bleeding on a given day. The Mayo Score rectal bleeding subscore is used for scoring. Scores range from 0 to 3 and higher scores indicate more severe disease activity. Each participant serves as his or her own control to establish the degree of rectal bleeding.	Baseline to Week 16
Correlations between changes in amplitude and timing of Partial Mayo Score and urgency of defecation patient-reported outcome.	Correlations are assessed by the Spearman correlation coefficient. The Urgency of defecation patient-reported outcome is assessed with the Urgency Numeric Rating Scale. Scoring is done on a 11-point numeric rating scale. Participant can input a score from 0 to 10. Higher scores indicate more severe urgency.	Baseline to Week 16
Correlations between changes in amplitude and timing of Partial Mayo Score and abdominal pain patient-reported outcome.	Correlations are assessed by the Spearman correlation coefficient. The abdominal pain patient-reported outcome is assessed with the Pain Numeric Rating Scale. Scoring is done on a 10-point horizontal numeric rating scale. Participant can input a score from 1 to 10. Higher scores indicate more severe pain. Scoring is done on a 10-point horizontal numeric rating scale. Participant can input a score from 1 to 10. Higher scores indicate more severe pain.	Baseline to Week 16
Correlations between changes in amplitude and timing of Partial Mayo Score and quality of sleep patient-reported outcome.	Correlations are assessed by the Spearman correlation coefficient. The quality of sleep patient-reported outcome is assessed with a question from the Sleep Quality visual analogue scale survey. Scoring is done on a 11-point numeric rating scale. Participant can input a score from 0 to 10. Higher scores indicate better quality of sleep.	Baseline to Week 16
Correlations between changes in amplitude and timing of Partial Mayo Score and daily fatigue patient-reported outcome.	Correlations are assessed by the Spearman correlation coefficient. The daily fatigue patient-reported outcome is assessed with the Fatigue numeric rating scale. Scoring is done on a 11-point numeric rating scale. Participant can input a score from 0 to 10. Higher scores indicate more severe fatigue.	Baseline to Week 16
Correlations between changes in amplitude and timing of Partial Mayo Score and weekly fatigue patient-reported outcome.	Correlations are assessed by the Spearman correlation coefficient. The weekly fatigue patient-reported outcome is assessed with 13 questions from the ("Additional concerns" section) FACIT-F (Version 4). Scoring is done on a Responses are recorded on a 5-point Likert scale. Scores range from 0 to 52, with higher scores representing greater fatigue.	Baseline to Week 16

Collaborators and Investigators

• Sponsor: Pfizer
• Investigators: Study Director: Pfizer CT.gov Call Center, Pfizer

Publications and helpful links

Helpful Links

• To obtain contact information for a study center near you, click here.

Study record dates

Study Major Dates

• Study Start (Actual): March 28, 2022
• Primary Completion (Estimated): October 21, 2024
• Study Completion (Estimated): October 21, 2024

Study Registration Dates

• First Submitted: September 1, 2021
• First Submitted That Met QC Criteria: September 27, 2021
• First Posted (Actual): October 6, 2021

Study Record Updates

• Last Update Posted (Actual): March 20, 2024
• Last Update Submitted That Met QC Criteria: March 19, 2024
• Last Verified: March 1, 2024

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Digestive System Diseases; Pathologic Processes; Gastrointestinal Diseases; Gastroenteritis; Colonic Diseases; Intestinal Diseases; Inflammatory Bowel Diseases; Ulcer; Colitis; Colitis, Ulcerative
• Other Study ID Numbers: A3921395; KIC-START (Other Identifier: Alias Study Number)

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: YES
• IPD 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.

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No