Tofacitinib Response in Ulcerative Colitis - a Real World Prospective Multi-center Study (TOUR)

• ClinicalTrials.gov Identifier: NCT03772145
• Recruitment Status: Enrolling by invitation
• First Posted: December 11, 2018
• Last Update Posted: January 20, 2021
• Sponsor: University of North Carolina, Chapel Hill
• Collaborator: Pfizer
• Information provided by (Responsible Party): University of North Carolina, Chapel Hill

Tracking Information

• First Submitted Date: December 4, 2018
• First Posted Date: December 11, 2018
• Last Update Posted Date: January 20, 2021
• Actual Study Start Date: November 27, 2018
• Estimated Primary Completion Date: December 31, 2023 (Final data collection date for primary outcome measure)

Current Primary Outcome Measures (submitted: December 10, 2018)

Change in clinical response from baseline [ Time Frame: At baseline and at approximately 2, 6, 10, 22 and 54 weeks after start of tofacitinib ]

Clinical response as assessed by the Simple Clinical Colitis Activity Index (SCCAI)

• Original Primary Outcome Measures: Same as current

Current Secondary Outcome Measures (submitted: December 10, 2018)

• Change in clinical remission from baseline [ Time Frame: At baseline and at approximately 2, 6, 10, 22 and 54 weeks after start of tofacitinib ]
  Clinical remission as assessed by the Simple Clinical Colitis Activity Index (SCCAI)
• Persistence of the initial tofacitinib dose [ Time Frame: 1 year ]
  Persistence of initial tofacitinib dose

• Original Secondary Outcome Measures: Same as current
• Current Other Pre-specified Outcome Measures: Not Provided
• Original Other Pre-specified Outcome Measures: Not Provided

Descriptive Information

• Brief Title: Tofacitinib Response in Ulcerative Colitis - a Real World Prospective Multi-center Study
• Official Title: TOUR: Tofacitinib Response in Ulcerative Colitis - a Real World Prospective Multi-center Study

Brief Summary

The overarching goal of this study, is to create a longitudinally followed, well phenotyped cohort of patients with UC starting treatment with tofacitinib in the setting of standard of care who have linked clinical data and self-reported outcome data that will lead to evaluation of efficacy and safety of tofacitinib in the real-life setting. The specific aims for the study are:

1. Create a prospective cohort of well phenotyped (proctitis vs. rectosigmoiditis vs. extensive) adult UC patients with serial clinical and patient reported data collected throughout the course of 12 months of tofacitinib therapy.
2. To determine clinical response rates and persistence of therapy with tofacitinib for induction and maintenance therapy
3. Describe the incidence of specific drug associated adverse event (shingles, serious infections), hospitalizations and surgeries in the standard of care setting.
4. Assess the correlation of various outcome measures in ulcerative colitis (Simple Clinical Colitis Activity Index (SCCAI), partial Mayo index, 6-point index) and endoscopic outcomes via the endoscopic Mayo Score

Detailed Description

The proposed prospective tofacitinib registry is a prospective patient cohort with the goal of evaluating the efficacy and safety of this drug in "real life". All patients starting tofacitinib for ulcerative colitis in the setting of standard of care therapy will be eligible within of 4 weeks of start of therapy. Preferentially, patients will be enrolled at the visit where therapy is initiated. At baseline patient characteristics including extent of disease, previous therapies (including number of previously failed biologics) and pro-inflammatory laboratory markers (CRP, if available calprotectin) will be captured in a prospective web-based database. Additionally, each consented study participant will be enrolled in a web-based prospective patient powered registry (PPR) to evaluate patient report outcomes (PRO's) (such as validated measures for depression, social satisfaction, pain, fatigue) and safety events (e.g. hospitalizations; herpes zoster, complications requiring discontinuation of tofacitinib).

The following outcomes will be assessed: Response and remission as assessed by the Simple Clinical Colitis Activity Index (SCCAI) (primary endpoint), in addition secondary outcomes will include: the partial Mayo score, the 6-point subscore of the Mayo score, and the Manitoba index, need for steroids, need for surgery or hospitalization, need for dosing intensification or reduction of dosing as well as persistence of tofacitinib therapy. Endoscopy information will be collected if done as part of routine care, but the study will not have an endoscopic requirement. Sites will be encouraged to report a Mayo endoscopic score if done as part of routine care as a secondary endpoint. The outcomes will be determined in the setting of standard of care visits, which normally are scheduled at the end of induction (approx. 6-12 weeks after start of tofacitinib) and during maintenance therapy after 4-6 and 9-12 months. PRO's and safety will be assessed with the PPR instrument at baseline, daily in the first 2 weeks to better understand rapidity of onset, and after 6,10,14,22,30,38,46 and 54 weeks. The analyses of these data will facilitate UC patient care and improve the overall quality of IBD therapy in the US and world-wide. This study is to be conducted according to US and international standards of Good Clinical Practice (FDA Title 21 part 312 and International Conference on Harmonization guidelines), applicable government regulations and Institutional research policies and procedures.

• Study Type: Observational [Patient Registry]
• Study Design: Observational Model: Cohort; Time Perspective: Prospective
• Target Follow-Up Duration: 1 Year
• Biospecimen: Not Provided
• Sampling Method: Non-Probability Sample
• Study Population: Adult patients, age 18 years or older, with UC, who within 2 weeks have been started on tofacitinib therapy for moderate to severe UC or who plan to start this therapy within the next 2 weeks. The start of the tofacitinib therapy must have been or be initiated in the setting of standard of care therapy
• Condition: Ulcerative Colitis
• Intervention: Not Provided

Study Groups/Cohorts

Tofacitinib

Adult patients, age 18 years or older, with UC, who within 2 weeks have been started on tofacitinib therapy for moderate to severe UC or who plan to start this therapy within the next 2 weeks. The start of the tofacitinib therapy must have been or be initiated in the setting of standard of care therapy

• Publications *: Not Provided

Recruitment Information

• Recruitment Status: Enrolling by invitation
• Estimated Enrollment (submitted: December 10, 2018): 100
• Original Estimated Enrollment: Same as current
• Estimated Study Completion Date: December 31, 2024
• Estimated Primary Completion Date: December 31, 2023 (Final data collection date for primary outcome measure)

Eligibility Criteria

Inclusion Criteria:

1. Adult patients, age 18 years or older, with UC, who within 2 weeks have been started on tofacitinib therapy for moderate to severe UC or who plan to start this therapy within the next 2 weeks. The start of the tofacitinib therapy must have been or be initiated in the setting of standard of care therapy
2. Anticipation that the patient will be followed by the participating center for the next 12 months
3. Diagnosis of UC must be established on the basis of standard clinical, radiographic, endoscopic, and histologic criteria as described below.

Exclusion Criteria:

• 1. Patients will be excluded if they meet any of the following criteria:

  1. Inability to provide informed consent
  2. Patients presenting for a one-time consultation

Sex/Gender

• Sexes Eligible for Study: All

• Ages: 18 Years to 80 Years (Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Contacts: Contact information is only displayed when the study is recruiting subjects
• Listed Location Countries: United States

Administrative Information

• NCT Number: NCT03772145
• Other Study ID Numbers: 18-2435
• Has Data Monitoring Committee: No

U.S. FDA-regulated Product

• Studies a U.S. FDA-regulated Drug Product: No
• Studies a U.S. FDA-regulated Device Product: No
• Product Manufactured in and Exported from the U.S.: No

IPD Sharing Statement

• Plan to Share IPD: Undecided
• Plan Description: There is currently no plan to share the anonymized data with other investigators. But this may change in the future

• Responsible Party: University of North Carolina, Chapel Hill
• Study Sponsor: University of North Carolina, Chapel Hill
• Collaborators: Pfizer

Investigators

• Principal Investigator: Hans Herfarth, MD; University of North Carolina
• Principal Investigator: Millie Long; University of North Carolina

• PRS Account: University of North Carolina, Chapel Hill
• Verification Date: January 2021