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

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

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.

Study Overview

• Status: Completed
• Conditions: Mild-to-moderate Ulcerative Colitis
• Intervention / Treatment: Other: Faecal Calprotectin Home Test; Other: PRO-2 Scoring

• Study Type: Interventional
• Enrollment (Actual): 250
• Phase: Not Applicable

Contacts and Locations

Study Locations

• Czechia
  • Brno, Czechia, 63600
    • Vojenska nemocnice Brno p.o.
  • Hradec Králové, Czechia, 50012
    • Hepato-Gastroenterologie HK, s.r.o.
  • Praha, Czechia, 13000
    • EGK s.r.o. - Sanatorium sv. Anny
  • Praha, Czechia, 17000
    • ISCARE IVF a.s.
  • Praha, Czechia, 18600
    • Mediendo s.r.o.
  • Usti nad Labem, Czechia, 40113
    • KZ a.s. - Masaryk Hospital
• Hungary
  • Budapest, Hungary, 1088
    • Semmelweis Egyetem, Isz. Sebeszeti es Intervencios Gasztroenterologiai Klinika
  • Budapest, Hungary, 1136
    • Pannonia Mgánorvosi Centrum Kft
  • Gyongyos, Hungary, 3200
    • Bugat Pal Hospital
  • Kecskemet, Hungary, 6000
    • Bacs-Kiskun Megyei Oktatokorhaz
  • Pécs, Hungary, 7624
    • University of Pecs
  • Szeged, Hungary, 6725
    • Szegedi Tudomnyegyetem
  • Vac, Hungary, 2600
    • Javorszky Odon Hospital
• Italy
  • Ancona, Italy, 60126
    • AOU Ospedali Riuniti di Ancona
  • Milan, Italy, 20157
    • ASST-FBF Luigi Sacco Hospital
  • Milano, Italy, 20132
    • Ospedale San Raffaele S.R.L
  • Palermo, Italy, 90146
    • Ospedali Riuniti Villa Sofia-Cervello
  • Pisa, Italy, 56124
    • Azienda Ospedaliero - Universitaria Pisana
  • Torino, Italy, 10126
    • A.O.Città della salute e della scienza diTorino
• Netherlands
  • Den Bosch, Netherlands, 5223GZ
    • Jeroen Bosch Ziekenhuis
  • Eindhoven, Netherlands, 5623EJ
    • Catharina Ziekenhuis
  • Rotterdam, Netherlands, 3045 BM
    • Franciscus Gasthuis & Vlietland
• Poland
  • Bydgoszcz, Poland, 85-079
    • NZOZ Vitamed
  • Bydgoszcz, Poland, 85168
    • Szpital Uniwersytecki
  • Bydgoszcz, Poland
    • Osrodek Sadan Klinicznych CLINSANTE
  • Chojnice, Poland, 89-600
    • Centrum Medyczne Lukamed
  • Jelenia Góra, Poland, 58-500
    • Karkonoskie Centrum Bada Klinicznych - Lexmedica SP Z O O
  • Krakow, Poland, 31-513
    • Centrum Medyczne Promed
  • Nowy Targ, Poland, 34-400
    • Orodek Bada Klinicznych Allmedica
  • Rzeszow, Poland, 35-302
    • Korczowski Bartosz, Gabinet Lekarski
  • Rzeszow, Poland, 35-236
    • Centrum Medyczne Medyk
  • Tychy, Poland, 43-110
    • H-T. Centrum Medyczne sp. z o.o.
  • Warszawa, Poland, 02-507
    • Centralny Szpital Kliniczny Ministerstwa Spraw
  • Wloclawek, Poland, 87-806
    • Centrum Diagnostyczno Lecznicze Barska
  • Wodzislaw Slaski, Poland, 44-300
    • NZOZ Centrum Gastroenterologii w Wodzislawiu Slaskim
  • Wrocław, Poland, 50-529
    • Melita Medical
  • Zabrze, Poland, 41-807
    • Clinhouse Centrum Medyczne
  • Łódź, Poland, 90302
    • SANTA FAMILIA Centrum Badan Profilaktyki i Leczenia
• Slovakia
  • Kosice, Slovakia, 4013
    • ENDOMED s.r.o.
  • Martin, Slovakia, 03601
    • Pigeas s.r.o.
  • Nitra, Slovakia, 94901
    • Fakuktna Nemocnica Nitra
  • Presov, Slovakia, 8001
    • Gastro LM s.r.o.

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 16 years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No

Description

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

Study Plan

How is the study designed?

Design Details

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

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Intervention arm; 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; 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.; Other: PRO-2 Scoring; 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.
Active Comparator: Reference arm; 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; 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.

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

Collaborators and Investigators

• Sponsor: Ferring Pharmaceuticals
• Investigators: Study Director: Global Clinical Compliance, Ferring Pharmaceuticals

Publications and helpful links

General Publications

• Dal Buono A, Roda G, Argollo M, Paridaens K, Peyrin-Biroulet L, Danese S. 'Treat to Target' in Mild to Moderate Ulcerative Colitis: Evidence to Support this Strategy. Curr Drug Targets. 2021;22(1):117-125. doi: 10.2174/1389450121666200727120305.

Study record dates

Study Major Dates

• Study Start (Actual): October 14, 2019
• Primary Completion (Actual): October 20, 2023
• Study Completion (Actual): October 20, 2023

Study Registration Dates

• First Submitted: April 7, 2020
• First Submitted That Met QC Criteria: April 7, 2020
• First Posted (Actual): April 10, 2020

Study Record Updates

• Last Update Posted (Estimated): November 9, 2023
• Last Update Submitted That Met QC Criteria: November 8, 2023
• Last Verified: November 1, 2023

More Information

Terms related to this study

• Keywords: Ulcerative Colitis; Pragmatic trial; Management strategies
• 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: 7002661

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

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