Study to Assess Effectiveness of Therapy Timely Adjustment Based on Self-monitoring in Patients Suffering From Mild-to-moderate Ulcerative Colitis (OPTIMISE Study)

Study Overview
=================
Brief Summary
-----------------
The purpose of this study is to provide evidence that a therapy of Ulcerative Colitis (UC) disease adjusted on tight monitoring of non-invasive parameters, such as clinical symptoms and faecal calprotectin (FC) (substance that is released when intestines are inflamed and that can be measured in faeces), can provide significantly higher benefit for the participants in terms of disease control and quality of life (QoL) improvement, compared to a symptom-based approach only.

Official Title
-----------------
Pragmatic Randomized Controlled Study to Assess the Effectiveness of Two Patient Management Strategies in Mild to Moderate Ulcerative Colitis

Conditions
-----------------
Mild-to-moderate Ulcerative Colitis

Intervention / Treatment
-----------------
* Other: Faecal Calprotectin Home Test
* Other: PRO-2 Scoring


Participation Criteria
=================
Eligibility Criteria
-----------------
Inclusion Criteria: Adult Participants (≥ 18 years old) Participants with active mild-to-moderate UC (with RB and bowel inflammation confirmed by endoscopy) Participants with treatment with 5-aminosalicylic acid (5-ASA) ≤ 2.4 g/day or with no treatment regimen at Baseline Participants who agree to use FC home test (to dose FC in faeces as a marker of inflammation) Participants with internet access and smartphone with camera Exclusion Criteria: Participants currently enrolled in another interventional study Participants not willing to undergo an endoscopy at the end of study Participants with contraindications to treatment with 5-ASA and/or 2nd generation corticosteroids Participants not willing to perform FC self-testing in faeces at home

Ages Eligible for Study
-----------------
Minimum Age: 18 Years

Sexes Eligible for Study
-----------------
All

Accepts Healthy Volunteers
-----------------
No

Study Plan
=================
How is the study designed?
-----------------

Design Details

Primary Purpose: Other
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Intervention arm<br>Treatment optimization (escalation/de-escalation) performed by the investigator based on participant self-monitoring of FC values and/or clinical symptoms (patient-reported outcome-2 [PRO-2] scoring). The FC Test and/or PRO-2 scoring will be done by the participant at home every month during active disease, every 3 months in remission, or when a participant feels the need/presents clinical symptoms. | Other: Faecal Calprotectin Home Test<br>* A non-invasive, in vitro diagnostic test for the determination of FC levels in human stool samples in combination with the dedicated 'CalproSmart' smartphone application. This test helps participants to self-monitor their FC levels at their own homes, ensuring an adequate and effective medical treatment regimen performed by the investigator.<br>Other: PRO-2 Scoring<br>* Monitoring of clinical symptoms will be performed by PRO-2 scoring, which includes rectal bleeding (RB) and stool frequency (SF) assessment obtained within the last 3-days.<br>|
| Active Comparator: Reference arm<br>Treatment optimization (escalation/de-escalation) performed by the investigator based on clinical symptoms (PRO-2 scoring) only. The PRO-2 scoring will be assessed during clinic visits every 3-months during active disease, every 6 months during remission, or when a participant feels the need; as per recommended standard practice. | Other: PRO-2 Scoring<br>* Monitoring of clinical symptoms will be performed by PRO-2 scoring, which includes rectal bleeding (RB) and stool frequency (SF) assessment obtained within the last 3-days.<br>|

What is the study measuring?
-----------------
Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Percentage of Participants with Mayo Endoscopic Sub-score = 0 | Defined as the percentage of participants achieved mucosal healing (Mayo endoscopic sub-score of 0 [normal friability]). The Mayo endoscopic scoring ranges from 0-3 with higher scores indicating greater disease severity. The score is defined as 0-3: 0= Normal; 1=Mild friability; 2 = Moderate friability; 3 = Exudation, spontaneous hemorrhage. | At 12 months | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Percentage of Participants with Rectal Bleeding = 0 | Defined as the percentage of participants with no RB (RB=0). The RB score ranges from 0-3 with higher scores indicating greater disease severity. The score was defined as 0-3: 0 = None; 1 = Streak of blood; 2 = Obvious blood; 3 = Mostly blood | At 12 months | 
| Percentage of Participants with Stool Frequency <=1 | Defined as the percentage of participants with SF<=1. The SF score ranges from 0-3 with higher scores indicating greater disease severity. The score was defined as 0-3: 0 = None; 1 = 1-2 stools/day > normal; 2 = 3-4 stools/day > normal; 3 = >4 stools/day > normal | At 12 months | 
| Percentage of Participants with Mayo Endoscopic Sub-score <=1 | Defined as the percentage of participants achieved endoscopic remission (Mayo endoscopic sub-score <=1 [normal or mild friability]). The Mayo endoscopic scoring ranges from 0-3 with higher scores indicating greater disease severity. The sub-score was defined as 0-3: 0 = Normal friability; 1 = Mild friability; 2 = Moderate friability; 3 = Exudation, spontaneous hemorrhage | At 12 months | 
| Change in Short Inflammatory Bowel Disease Questionnaire (SIBDQ) | Defined as a change in the QoL using SIBDQ over 12 months. The SIBDQ consists of 10 questions, measuring systemic, social, Bowel, and emotional status; each question is scored on a scale from 1 (poor QoL) to 7 (optimum QoL). A higher score indicates a better QoL. Total scores range from 10 (poor QoL) to 70 (good QoL). | From Baseline over 12 Months | 
| Change in Short Form Questionnaire-36 (SF-36) | The SF-36 is a health-related survey that assesses participant's QoL and consists of 36 questions covering 8 health domains: physical functioning, bodily pain, role limitations due to physical problems and emotional problems, general health, mental health, social functioning, vitality, and 2 component scores (mental component score and physical component score). Each domain is scored by summing the individual items and transforming the scores into a 0 (poorest health) to 100 (best health) scale with higher scores indicating better health status or functioning. | From Baseline over 12 Months | 
| Percentage of Participants Escalated to Rescue Therapy | Rescue therapy included systemic corticosteroids/ immunosuppressants /biologics. | Over 12 months | 
| Change in Work Productivity and Activity Impairment: Ulcerative Colitis (WPAI: UC) Questionnaire | The WPAI-UC is a questionnaire with 6 questions used to evaluate lost productivity (work time missed and work and activity impairment) during the past 7 days due to UC. The scores are presented as percentages. | From Baseline over 12 Months | 
| Number of Hospitalization Days | Number of hospitalization days will be determined by UC related medical resources. | Over 12 months | 
| Number of Outpatients Visits and Calls (Outside of Study Schedule) | Number of outpatients visits and calls (outside of study schedule) will be determined by UC related medical resources. | Over 12 months | 
| Number of Ulcerative Colitis Related Procedures and/or Surgeries | Number of UC related procedures and/or surgeries will be determined by UC related medical resources. | Over 12 months | 
| Faecal Calprotectin Value Changes at the Defined Study Time Points (Interventional Arm Only) | The FC Test will be done by the participant at home every month during active disease, every 3 months in remission, or when participant feels the need/presents clinical symptoms. | Over 12 months | 

 Terms related to the study
=================
Keywords Provided by Centre Hospitalier Valida
-----------------
Pragmatic trial, Ulcerative Colitis, Management strategies