Mild-to-moderate Ulcerative ColitisOPTIMISENCT04340895
Study to Assess Effectiveness of Therapy Timely Adjustment Based on Self-monitoring in Patients Suffering From Mild-to-moderate Ulcerative Colitis (OPTIMISE Study)
A study testing Faecal Calprotectin Home Test and PRO-2 Scoring in people with Mild-to-moderate Ulcerative Colitis, run by Ferring Pharmaceuticals at 42 sites in 6 countries.
Trial 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… Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Mild-to-moderate Ulcerative Colitis
- What is being tested?
- Faecal Calprotectin Home Test and PRO-2 Scoring
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 250 participants
- Where
- 42 sitesin 6 countries
- Study dates
- 14 Oct 2019to 20 Oct 2023
- Phase
- Interventional
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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
Who cannot join
- 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
Trial locations and contacts
42 sites in 6 countries.
42 sites in 6 countries
Poland16 sites
NZOZ Vitamed
Bydgoszcz, 85-079
Szpital Uniwersytecki
Bydgoszcz, 85168
Osrodek Sadan Klinicznych CLINSANTE
Bydgoszcz
Centrum Medyczne LukaMed
Chojnice, 89-600
Karkonoskie Centrum Bada Klinicznych - Lexmedica SP Z O O
Jelenia Góra, 58-500
Centrum Medyczne Promed
Krakow, 31-513
SANTA FAMILIA Centrum Badan Profilaktyki i Leczenia
Lodz, 90302
Orodek Bada Klinicznych Allmedica
Nowy Targ, 34-400
Centrum Medyczne Medyk
Rzeszów, 35-236
Korczowski Bartosz, Gabinet Lekarski
Rzeszów, 35-302
H-T. Centrum Medyczne sp. z o.o.
Tychy, 43-110
Centralny Szpital Kliniczny Ministerstwa Spraw
Warsaw, 02-507
Centrum Diagnostyczno Lecznicze Barska
Włocławek, 87-806
NZOZ Centrum Gastroenterologii w Wodzislawiu Slaskim
Wodzisław Śląski, 44-300
Melita Medical
Wroclaw, 50-529
Clinhouse Centrum Medyczne
Zabrze, 41-807
Hungary7 sites
Semmelweis Egyetem, Isz. Sebeszeti es Intervencios Gasztroenterologiai Klinika
Budapest, 1088
Pannonia Mgánorvosi Centrum Kft
Budapest, 1136
Bugat Pal Hospital
Gyöngyös, 3200
Bacs-Kiskun Megyei Oktatokorhaz
Kecskemét, 6000
University of Pecs
Pécs, 7624
Szegedi Tudomnyegyetem
Szeged, 6725
Javorszky Odon Hospital
Vác, 2600
Czechia6 sites
Vojenska nemocnice Brno p.o.
Brno, 63600
Hepato-Gastroenterologie HK, s.r.o.
Hradec Králové, 50012
EGK s.r.o. - Sanatorium sv. Anny
Prague, 13000
ISCARE IVF a.s.
Prague, 17000
Mediendo s.r.o.
Prague, 18600
KZ a.s. - Masaryk Hospital
Ústí nad Labem, 40113
Italy6 sites
AOU Ospedali Riuniti di Ancona
Ancona, 60126
ASST-FBF Luigi Sacco Hospital
Milan, 20157
Ospedale San Raffaele S.R.L
Milan, 20132
Ospedali Riuniti Villa Sofia-Cervello
Palermo, 90146
Azienda Ospedaliero - Universitaria Pisana
Pisa, 56124
A.O.Città della salute e della scienza diTorino
Torino, 10126
Slovakia4 sites
ENDOMED s.r.o.
Košice, 4013
Pigeas s.r.o.
Martin, 03601
Fakuktna Nemocnica Nitra
Nitra, 94901
Gastro LM s.r.o.
Prešov, 8001
Netherlands3 sites
Jeroen Bosch Ziekenhuis
's-Hertogenbosch, 5223GZ
Catharina Ziekenhuis
Eindhoven, 5623EJ
Franciscus Gasthuis & Vlietland
Rotterdam, 3045 BM
About this study
Official title: 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.
- Conditions
- Mild-to-moderate Ulcerative Colitis
- Being tested
- Faecal Calprotectin Home Test, PRO-2 Scoring
- Keywords
- Ulcerative Colitis, Management strategies, Pragmatic trial
- Medical subject headings
- Colitis, Ulcerative
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Faecal Calprotectin Home TestOther
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.
PRO-2 ScoringOther
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.
| Study group | Role | What they receive |
|---|---|---|
| Intervention arm | Experimental | 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. Receives: Other: Faecal Calprotectin Home Test; Other: PRO-2 Scoring |
| Reference arm | Active comparator | 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. Receives: Other: PRO-2 Scoring |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: At 12 months
Secondary outcomes (11)
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
Measured: 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
Measured: 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
Measured: 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).
Measured: 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.
Measured: From Baseline over 12 Months
Percentage of Participants Escalated to Rescue Therapy
Rescue therapy included systemic corticosteroids/ immunosuppressants /biologics.
Measured: 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.
Measured: From Baseline over 12 Months
Number of Hospitalization Days
Number of hospitalization days will be determined by UC related medical resources.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: Over 12 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted10 Apr 2020
- Study start14 Oct 2019
- Primary completion20 Oct 2023
- Study completion20 Oct 2023
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (2)Papers the sponsor has linked to this study.
- Other trials for Mild-to-moderate Ulcerative ColitisStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase N/A
- Primary purpose
- Other
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 250 participants (actual)
Sponsor and investigators
- Lead sponsor
- Ferring Pharmaceuticals
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Global Clinical Compliance, Study director, Ferring Pharmaceuticals
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04340895
- Sponsor's protocol ID
- 7002661
- Acronym
- OPTIMISE
- Status last verified
- May 2024
- First submitted
- 7 Apr 2020
- Last updated
- 19 Sep 2024
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
Danese S, Fiorino G, Vicaut E, Paridaens K, Ugur A, Clark B, Vanasek T, Stepek D, D'Amico F, West R, Gilissen LPL, Wisniewska Jarosinka M, Drobinski P, Fronik G, Fic M, Walczak M, Kowalski M, Korczowski B, Wiatr M, Peyrin-Biroulet L. Clinical Trial: A Pragmatic Randomised Controlled Study to Assess the Effectiveness of Two Patient Management Strategies in Mild to Moderate Ulcerative Colitis-The OPTIMISE Study. J Clin Med. 2024 Aug 30;13(17):5147. doi: 10.3390/jcm13175147.
PubMed 39274360Dal 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.
PubMed 32718289
Study at a glance
From the registry record, last updated by the sponsor on 19 Sep 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Pragmatic Randomized Controlled Study to Assess the Effectiveness of Two Patient Management Strategies in Mild to Moderate Ulcerative Colitis
- ClinicalTrials.gov ID
- NCT04340895
- Acronym / sponsor ID
- OPTIMISE / 7002661
- Conditions
- Mild-to-moderate Ulcerative Colitis
- Keywords
- Ulcerative Colitis, Management strategies, Pragmatic trial
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 250 participants (actual)
- Lead sponsor
- Ferring PharmaceuticalsIndustry (pharma, biotech, devices)
- Study start
- 14 Oct 2019 (actual)
- Primary completion
- 20 Oct 2023 (actual)
- Study completion
- 20 Oct 2023 (actual)
- First posted
- 10 Apr 2020 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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 design
- Primary purpose
- Other
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Intervention arm | Experimental | Other: Faecal Calprotectin Home Test, Other: PRO-2 ScoringTreatment 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. |
| Reference arm | Active comparator | Other: PRO-2 ScoringTreatment 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. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (5 listed)
- 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
Key exclusion criteria (4 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Faecal Calprotectin Home TestA 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 | Intervention arm |
| PRO-2 ScoringMonitoring 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. | Other | Intervention arm, Reference arm |
Primary outcome
Percentage of Participants with Mayo Endoscopic Sub-score = 0
Time frame: At 12 months
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.
11 secondary outcomes
Percentage of Participants with Rectal Bleeding = 0
Time frame: At 12 months
Percentage of Participants with Stool Frequency <=1
Time frame: At 12 months
Percentage of Participants with Mayo Endoscopic Sub-score <=1
Time frame: At 12 months
Change in Short Inflammatory Bowel Disease Questionnaire (SIBDQ)
Time frame: From Baseline over 12 Months
Change in Short Form Questionnaire-36 (SF-36)
Time frame: From Baseline over 12 Months
Percentage of Participants Escalated to Rescue Therapy
Time frame: Over 12 months
Change in Work Productivity and Activity Impairment: Ulcerative Colitis (WPAI: UC) Questionnaire
Time frame: From Baseline over 12 Months
Number of Hospitalization Days
Time frame: Over 12 months
Number of Outpatients Visits and Calls (Outside of Study Schedule)
Time frame: Over 12 months
Number of Ulcerative Colitis Related Procedures and/or Surgeries
Time frame: Over 12 months
Faecal Calprotectin Value Changes at the Defined Study Time Points (Interventional Arm Only)
Time frame: Over 12 months
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 19 Sep 2024.
- Results publication: PubMed 39274360Danese S, Fiorino G, Vicaut E, Paridaens K, Ugur A, Clark B, Vanasek T, Stepek D, D'Amico F, West R, Gilissen LPL, Wisniewska Jarosinka M, Drobinski P, Fronik G, Fic M, Walczak M, Kowalski M, Korczowski B, Wiatr M, Peyrin-Biroulet L. Clinical Trial: A Pragmatic Randomised Controlled Study to Assess the Effectiveness of Two Patient Management Strategies in Mild to Moderate Ulcerative Colitis-The OPTIMISE Study. J Clin Med. 2024 Aug 30;13(17):5147. doi: 10.3390/jcm13175147.
- Derived from this study: PubMed 32718289Dal 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.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Global Clinical Compliance study director
Ferring Pharmaceuticals
42 sites in 6 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing Faecal Calprotectin Home Test and PRO-2 Scoring in people with Mild-to-moderate Ulcerative Colitis, run by Ferring Pharmaceuticals at 42 sites in 6 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 5 inclusion and 4 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 19 Sep 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 42 sites in Poland, Hungary, Czechia, Italy, Slovakia and 1 more country. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Percentage of Participants with Mayo Endoscopic Sub-score = 0 (time frame: At 12 months). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT04340895, which also lists 2 linked publications.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 19 Sep 2024.