Tofacitinib Registry of Patients With Ulcerative Colitis in Germany (TOFA-UC)

Tofacitinib Registry of Patients With Ulcerative Colitis in Germany - Documentation of Tofacitinib Induction and Maintenance Therapy in Conjunction With Long-term Outcome and Predictors of Response

This registry on Tofacitinib and biologics (anti-integrin/anti-TNF) in the treatment of ulcerative colitis (UC) patients in Germany will extend the prospective documentation of safety issues and efficacy in induction and maintenance therapy of Tofacitinib (Xeljanz®) in addition to other biologics used in Germany with a particular interest in predictors of long-term responses and favorable disease outcome or to predict severe side effects caused by therapy with Januskinase(JAK)- inhibitors/biologics.

Study Overview

• Status: Terminated
• Conditions: Colitis, Ulcerative; Bowel Disease; Bowel Diseases, Inflammatory; Biologics; Chronic Inflammatory Small Bowel Disease; Tofacitiniib; CED

Detailed Description

This is an Investigator-initiated non-randomized, non-interventional observational prospective long- term research (IIR) as part of the normal treatment of UC patients for the safety and efficacy of Tofacitinib / biologic therapies. As of 01st July 2019, UC patients who have been provided Tofacitinib / biologics for UC according the respective label by the treating physician may be prospectively included in an online database by the participating study centers by 31st March 2021.

An interim analysis is planned at the end of patients ́ recruitment with respect of the induction phase (week 16) for October 2021 with a first publication of the data in a peer-reviewed journal (e.g., JCC, GUT, UEG Journal).

Up to 480 patients (including about 360 UC patients with Tofacitinib and about 120 UC patients with biologics) will be included in the online documentation. A specification to the study centers regarding the group inclusion (patients treated with Tofacitinib (group 1) or a biologics (group 2)) will not be made; this means, each study center can include patients in both groups.

The CED Service GmbH assume that approximately 20-30% of Tofacitinib patients will be biologic-naïve UC patients, many of whom will represent early disease patients (< 2 years after first diagnosis).

• Study Type: Observational
• Enrollment (Actual): 50

Contacts and Locations

Study Locations

• Germany
  • Niedersachsen
    • Minden, Niedersachsen, Germany, 32423
      • Gastroenterologische Gemeinschaftspraxis Minden

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years to 80 years (ADULT, OLDER_ADULT)
• Accepts Healthy Volunteers: N/A
• Genders Eligible for Study: All

• Sampling Method: Probability Sample

Study Population

Up to 480 patients (aged 18-80 years at enrollment) (including about 360 UC patients with Tofacitinib and about 120 UC patients with biologics) will be included in the online documentation. A specification to the study centers regarding the group inclusion (patients treated with Tofacitinib (group 1) or a biologics (group 2)) will not be made; this means, each study center can include patients in both groups.

The CED Service GmbH assume that approximately 20-30% of Tofacitinib patients will be biologic-naïve UC patients, many of whom will represent early disease patients (< 2 years after first diagnosis).

Description

Inclusion Criteria:

• Patients with moderate to severe Ulcerative Colitis aged 18-80 years at enrollment
• Written informed consent is given

Exclusion Criteria:

• Malignant disease in history (except for non-melanoma skin cancer)
• Any contraindication according to the SmPC of the respective medication

Study Plan

How is the study designed?

Number of groups / cohorts

2

Cohorts and Interventions

Group / Cohort
Group 1; UC-patients (age at enrollment: 18-80 years) receiving a newly introduced Tofacitinib therapy (n=360). Previous treatment(s) with biologics or immunosuppressants is (are) permitted. About 20-30% of the Tofacitinib patients will biologic-naiv.
Group 2; UC-patients (age at enrollment: 18-80 years) receiving a newly introduced biologics therapy (n=120). Previous treatment(s) with biologics or immunosuppressants is (are) allowed.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
partial Mayo score ≤ 1 plus a bleeding subscore of 0) in the inductionphase (week 16)	The primary endpoint is steroid-free remission (remission: partial Mayo score ≤ 1 plus a bleeding subscore of 0) in the inductionphase (week 16). For this endpoint it will be only captured if there is a current use of steroids at the time of the visit. So the patient will be asked for current steroid use at time of visit at week 16 and if he has received steroids within the last 4 weeks prior to time point of the visit.	07/01/2019 - 03/31/2023

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
occurrence of serious side effects (e.g., death, tumor, tuberculosis, severe infections, or side effects leading to hospitalization)	Online documentation of safety and efficacy in induction and maintenance therapy including the occurrence of serious side effects (e.g., death, tumor, tuberculosis, severe infections, or side effects leading to hospitalization)	07/01/2019 - 03/31/2023
response: partial Mayo Score reduction of ≥ 3 accompanied by a decrease of at least 30% from baseline and remission: partial Mayo score ≤ 1 plus a bleeding subscore of 0 - Efficacy	Efficacy (response: partial Mayo Score reduction of ≥ 3 accompanied by a decrease of at least 30% from baseline and remission: partial Mayo score ≤ 1 plus a bleeding subscore of 0) of induction therapy (week 8 and 16) and maintenance therapy (months 6 to 24) and efficacy (response and remission) in different subpopulations, e.g. based on a previous biologic therapy or not.	07/01/2019 - 03/31/2023
hospitalization days via question in questionnaire	Obtaining health economic data in UC patients on Tofacitinib / biologics therapy (hospitalization)	07/01/2019 - 03/31/2023
psychosocial impairments - EQ-5D	Obtaining data of the course from UC patients with a new therapy (Tofacitinib / biologics) related to treatment strategy and psychosocial impairments	07/01/2019 - 03/31/2023
EQ-5D quality of life	Obtaining health economic data in UC patients on Tofacitinib / biologics therapy (quality of life)	07/01/2019 - 03/31/2023
early retirement via question in questionnaire	Obtaining health economic data in UC patients on Tofacitinib / biologics therapy (early retirement)	07/01/2019 - 03/31/2023

Collaborators and Investigators

• Sponsor: Ced Service GmbH
• Collaborators: Pfizer

Study record dates

Study Major Dates

• Study Start (ACTUAL): August 1, 2019
• Primary Completion (ACTUAL): November 15, 2020
• Study Completion (ACTUAL): November 15, 2020

Study Registration Dates

• First Submitted: July 19, 2019
• First Submitted That Met QC Criteria: July 23, 2019
• First Posted (ACTUAL): July 25, 2019

Study Record Updates

• Last Update Posted (ACTUAL): October 8, 2021
• Last Update Submitted That Met QC Criteria: September 30, 2021
• Last Verified: September 1, 2021

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Digestive System Diseases; Pathologic Processes; Gastrointestinal Diseases; Gastroenteritis; Colonic Diseases; Ulcer; Inflammatory Bowel Diseases; Intestinal Diseases; Colitis; Colitis, Ulcerative
• Other Study ID Numbers: Protocol V1.4 31.05.2019

Drug and device information, study documents

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