Clinical trial NCT04338204: Observational Study To Assess The Effectiveness and Treatment Adherence Of Tofacitinib of Ulcerative Colitis In Clinical Practice In Sweden
Official title: Observational Study of Tofacitinib in Ulcerative Colitis in Sweden (ODEN)
Study type: OBSERVATIONAL
Overall status: RECRUITING
Conditions: Ulcerative Colitis
Lead sponsor: Pfizer
Sponsor class: INDUSTRY
First posted: 2020-04-08
Enrollment: 120
Sex: ALL
Minimum age (years): 18
Maximum age (years): 99
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Patients with a confirmed diagnosis of ulcerative colitis, ≥18years of age, with confirmed active disease (biomarker or endoscopy) initiating tofacitinib as per the Swedish summary of product characteristics (SmPC)

Brief summary: This is a prospective observational study using data from an existing, ongoing National Swedish registry (SWIBREG). This study is designed to assess the effectiveness and treatment adherence of tofacitinib on clinical disease activity parameters in patients with ulcerative colitis in Swedish clinical practice. The study will also assess treatment adherence of tofacitinib using the Swedish Prescribed Drug Register.


Eligibility criteria: Inclusion Criteria:

* The assignment of the patient to tofacitinib is not decided in advance by the protocol but falls within clinical practice and the prescription of the medicine is done according to the SmPC and is clearly separated from the decision to include the patient in the study.
* The patient must sign the informed consent before enrollment in the study. The informed consent permits extraction of data from SWIBREG at baseline and during the duration of the study. For patients not registered in SWIBREG, they must complete all SWIBREG consents and registration at the time of treatment initiation. Evidence of a personally signed and dated informed consent document indicating that the patient (or a legally acceptable representative) has been informed of all pertinent aspects of the study.
* Patients, male or female, must be 18 years old or above.
* The patient must have active disease as confirmed by fecal calprotectin \>250 mg/kg or endoscopic assessment corresponding to a Mayo endoscopic subscore ≥2 not more than 4 weeks prior onset to the initiation of tofacitinib treatment. This inclusion criteria applies also to patients that have already been enrolled in the study.

Exclusion Criteria:

* The patient is enrolled in a clinical trial in which the treatment of ulcerative colitis is dictated by a study protocol. If the patient is participating in another ongoing observational study (non-interventional), the patient may be included in this observational study.
* Patients that fulfill any of the contraindications according to the latest version of the SmPC. Any SmPC label updates will be communicated to all study sites.
* For whatever reason the physician feels the patient unsuitable to participate in the study.

Primary outcomes:
measure: Proportion of Participants in Remission as Measured by Partial Mayo Score
timeFrame: Week 52
Secondary outcomes:
measure: Proportion of Participants who are taking tofacitinib
timeFrame: Baseline, Weeks 8, 16, 52, 104
measure: Proportion of Participants in Clinical Remission Based on Total Mayo Score
timeFrame: Weeks 8, 16, 52, and 104
measure: Proportion of Participants in Clinical Response Based on Total Mayo Score
timeFrame: Weeks 8, 16, 52, and 104
measure: Proportion of Participants in Clinical Remission Based on Partial Mayo Score
timeFrame: Weeks 8, 16, 52 and 104
measure: Proportion of Participants in Clinical Response Based on Partial Mayo Score
timeFrame: Weeks 8, 16, 52 and 104
measure: Proportion of Participants in Steroid-Free Clinical Remission
timeFrame: Baseline, weeks 8, 16, 52, and 104
measure: Change from Baseline in Total Mayo Score
timeFrame: Baseline, Weeks 8, 16, 52, and 104
measure: Change From Baseline In Partial Mayo Score
timeFrame: Baseline, Weeks 8, 16, 52 and 104
measure: Change From Baseline In Level of Fecal Calprotectin (f-calprotectin)
timeFrame: Baseline, Weeks 8, 16, 52 and 104
measure: Proportion of Responders defined by a Fecal Calprotectin (f-calprotectin) Reduction of ≥50%, ≥75% or ≥90%
timeFrame: Weeks 8, 16, 52, and 104
measure: Change from Baseline of C-Reactive Protein (CRP)
timeFrame: Baseline, Weeks 8, 16, 52, and 104
measure: Proportion of Participants In Sustained Remission (Partial Mayo score)
timeFrame: Week 8 To Weeks 16, 52 and 104
measure: Proportion of Participants In Sustained Remission (Partial Mayo score)
timeFrame: Week 16 To Weeks 52 and 104
measure: Proportion of Participants in Sustained Steroid Free Remission (Partial Mayo Score) (for all patients and for those treated with corticosteroids at baseline)
timeFrame: Weeks 16 through 52 and at Week 104
measure: Proportion of Participants in Endoscopic Remission, Mucosal Healing or Endoscopic Response
timeFrame: Week 8, 16, 52 and 104
measure: Proportion of Participants in Sustained Endoscopic Remission, Mucosal Healing or Endoscopic Response
timeFrame: Week 8 to Week 16, 52 and 104
measure: Proportion of Participants in Sustained Endoscopic Remission, Mucosal Healing or Endoscopic Response
timeFrame: Week 16 to 52 and at Week 104
measure: Proportion of Participants in Sustained Steroid Free Remission (Partial Mayo Score) (For All Participants and for Those Treated With Corticosteroids at Baseline) and Endoscopic Remission, Mucosal Healing or Endoscopic Response
timeFrame: Week 16 to 52 and 104
measure: Change From Baseline In Inflammatory Bowel Disease Fatigue (IBD-F) Score
timeFrame: Baseline, Weeks 8, 16, 52 and 104
measure: Change From Baseline In EuroQol 5 Dimensions 5 Levels (EQ5D-5L)
timeFrame: Baseline, Weeks 8, 16, 52, and 104
measure: Change From Baseline In Short Health Scale (SHS)
timeFrame: Baseline, Weeks 8, 16, 52 and 104
measure: Proportion of Participants Who Had a Colectomy
timeFrame: Weeks 8, 16, 52, and 104
measure: Comparison of Response and Remission (Partial Mayo Score) Based on the Extent of Ulcerative Colitis According To the Montreal Classification
timeFrame: Weeks 8, 16, 52 and 104
measure: Proportion of Participants In Sustained Remission (Total Mayo score)
timeFrame: Week 16 To Weeks 52 and 104
measure: Proportion of Participants In Sustained Remission (Total Mayo score)
timeFrame: Week 8 To Weeks 16, 52 and 104
measure: Proportion of Participants in Sustained Steroid Free Remission (Total Mayo Score) (For All Participants and for Those Treated With Corticosteroids at Baseline) and Endoscopic Remission, Mucosal Healing or Endoscopic Response
timeFrame: Week 16 to 52 and 104
measure: Proportion of Participants in Sustained Steroid Free Remission (Total Mayo Score) (for all patients and for those treated with corticosteroids at baseline)
timeFrame: Weeks 16 through 52 and at Week 104
measure: Comparison of Response and Remission (Total Mayo Score) Based on the Extent of Ulcerative Colitis According To the Montreal Classification
timeFrame: Weeks 8, 16, 52 and 104
measure: Proportion of Participants in Steroid-Free Clinical Remission
timeFrame: Baseline, weeks 8, 16, 52, and 104
measure: Proportion of participants reaching f-calprotectin below 250 mg/kg of those that had f-calprotectin above 250 mg/kg at baseline
timeFrame: Baseline, week 8, 16, 52 and 104
measure: Portion of participants with dose change of tofacitinib
timeFrame: Week 8, 16, 52 and 104
measure: Portion of participants with a termination of tofacitinib
timeFrame: Week 8, 16, 52 and 104
measure: Portion of participants with dose and dose changes of tofacitinib and corticosteroids
timeFrame: Week 8, 16, 52 and 104
measure: Proportion of particpants with changes in rectal bleeding and stool frequency
timeFrame: Baseline, Week 2
measure: Proportion of participants with changes in EQ5D and SHS
timeFrame: Baseline, Week 2
measure: Proportion of particpants with rectal bleeding and stool frequency sub score indicative of mild disease
timeFrame: Baseline, Week 2
measure: Proportion of participants with stool frequency and rectal bleeding subscore of 0
timeFrame: Baseline, Week 2
measure: Proportion of participants with mild abdominal pain
timeFrame: Baseline, week 2, 8, 16, 52 and 104
measure: Proportion of participants with no abdominal pain
timeFrame: Baseline, week 2, 8, 16, 52 and 104
measure: Proportion of participants with no bowel urgency
timeFrame: Baseline, Weeks 2, 8, 16, 52 and 104.
measure: Proportion of participants with reduction of ≥ 1 point from baseline rectal bleeding and stool frequency sub score
timeFrame: Baseline, Week 2
measure: Proportion of patients achieving symptomatic remission
timeFrame: Baseline, weeks 2, 8, 16, 52 and 104.
measure: Proportion of patients achieving symptomatic remission stratified on steroid use at baseline or not.
timeFrame: Baseline, weeks 2, 8, 16, 52 and 104
measure: Proportion of patients achieving symptomatic response
timeFrame: Baseline, weeks 2, 8, 16, 52 and 104
measure: Proportion of patients achieving symptomatic response stratified on steroid use at baseline
timeFrame: Baseline, weeks 2, 8, 16, 52 and 104
measure: Proportion of patients in steroid-free clinical remission (pMayo and full Mayo score)stratified based on steroid use at baseline or not.
timeFrame: Baseline, weeks 8, 16, 52 and 104
measure: Change from baseline in bowel urgency (as measured by SCCAI)
timeFrame: Weeks 2, 8, 16, 52 and 104
Other outcomes:
Locations:
city: Alingsås
country: Sweden
facility: Ulf Eriksson
city: Borås
country: Sweden
facility: Medicinkliniken, Södra Älvsborgs Sjukhus Borås, Brämhultsvägen 53
city: Gävle
country: Sweden
facility: Gävle Hospital
city: Göteborg
country: Sweden
facility: SU/Sahlgrenska, Gastroenterologi & Hepatologi
city: Jönköping
country: Sweden
facility: Medicinkliniken, Länssjukhuset Ryhov, Sjukhusgatan
city: Kristianstad
country: Sweden
facility: Daniel Molin
city: Lidingö
country: Sweden
facility: Shiprock Consulting AB,
city: Linköping
country: Sweden
facility: Mag-Tarmmedicinska kliniken, Universitetssjukhuset i Linköping
city: Malmö
country: Sweden
facility: Region skåne, Skånes Universitetssjukhus
city: Stockholm
country: Sweden
facility: Stockholm Gastro Center
city: Stockholm
country: Sweden
facility: Ersta Sjukhus, Medicinkliniken, Fjällgatan 44
city: Stockholm
country: Sweden
facility: Karolinska Universitetssjukhuset i Solna, Eugeniavägen 3, B4-09,
city: Stockholm
country: Sweden
facility: Danderyds Hospital
city: Umeå
country: Sweden
facility: Medicinkliniken, Umeås Universitetssjukhus
city: Uppsala
country: Sweden
facility: Specialmedicin, Akademiska Sjukhuset, Sjukhusvägen ing 40
city: Västerås
country: Sweden
facility: Medicinmottagningen gastroenterologi, Västmanlands sjukhus
city: Örebro
country: Sweden
facility: Medicinmottagning 4, Medicinska Kliniken, Universitetssjukhuset Örebro
Study officials:
affiliation: Pfizer
name: Pfizer CT.gov Call Center
role: STUDY_DIRECTOR
References: