Clinical Trial Summary
UC (UC) is a chronic, relapsing and destructive inflammatory disorder of the colon which can
lead to organ damage and impair quality of life.
Consensus guidelines recommend to go beyond resolution of clinical symptoms and achieve
endoscopic remission. This long-term treatment goal in UC is commonly defined by a Mayo
endoscopic subscore < 13, and is associated with prolonged clinical remission, lower rates of
hospitalization and lower rates of colectomy. However, colonoscopy is an invasive and
expensive procedure, unpleasant to patients, not without risks, especially during severe
flares. Moreover, CS is time-consuming and expensive for the Healthcare System.
Clinical symptoms correlate well with endoscopic findings, and their improvement together to
normalization of FC, are currently considered the short-term and intermediate-term targets to
achieve. However, while asymptomatic patients with FC < 50 mcg/g have < 5% probability to
have endoscopic lesions, and conversely patients with evident rectal bleeding and persistent
increased stool frequency (> 3 stools above baseline) with FC > 250 mcg/g have less than 5%
chance to have endoscopic remission, in patients in the intermediate scenarios with stool
frequency score (SFS) 2 or 3 or rectal bleeding score (RBS) > 0, with FC values between 50
and 250 mcg/g, the uncertainty increases and CS should not be avoided.
Bowel US is a well-tolerated, non-invasive, patient friendly, cheap, easy-to-use tool to
manage UC patients in clinical practice8. In addition, its ability to be performed as
point-of-care bowel US may drastically change frequency of the assessment of treatment
response, speeding the clinical decision-making process9. Recently, the investigators
developed and externally validated non-invasive ultrasonography based criteria [Milan
ultrasound criteria (MUC)] to assess and grade endoscopic activity in UC10,11. The
investigators also confirmed that a MUC score > 6.2 is a valid cut-off to discriminate
endoscopic activity, defined by a Mayo endoscopic subscore > 1 Bowel US is a well-tolerated,
non-invasive, patient friendly, cheap, easy-to-use tool to manage UC patients in clinical
practice. In addition, its ability to be performed as point-of-care bowel US may drastically
change frequency of the assessment of treatment response, speeding the clinical
decision-making process. Recently, the investigators developed and externally validated
non-invasive ultrasonography based criteria [Milan ultrasound criteria (MUC)] to assess and
grade endoscopic activity in UC14,15. The investigators also confirmed that a MUC score > 6.2
is a valid cut-off to discriminate endoscopic activity, defined by a Mayo endoscopic subscore
> 1.
Prospective interventional randomized open-label study to validate the role of US-based
treat-to-target strategies in the decision-making process in patients with UC.
Patients seen at each participating center who are eligible for this study will be randomized
1:1 (computer generated randomization list) to enter one of two study arms up to Week 52:
1. study group: the bowel US-based treat to target arm
2. control group: the routine CS treat to target arm. At Week 52 ± 4, all patients in the
two study arms will undergo CS, bowel US, PRO and FC as indicated for the screening
phase.
Patients will be followed up and will undergo the study procedures as described below:
Bowel Ultrasound-based Treat to target arm At W12 ± 4 and W24 ± 4, subjects will perform FC
and bowel US and PRO will be recorded.
If rectal bleeding > 2 + stool frequency > 2 AND FC > 250 mcg/g AND/OR MUC > 6.2, treatment
will be changed according to the standard of care and international guidelines.
Routine CS Treat to target arm At W12 ± 4 and W24 ± 4, subjects will perform FC and PRO will
be recorded for all patients.
If rectal bleeding > 2 + stool frequency > 2 AND FC > 250 mcg/g, treatment will be changed
according to the standard of care and guidelines. If rectal bleeding > 0 + stool frequency >
2 AND FC will be ≥ 50 mcg/g and ≤ 250 mcg/g, a RSS will be done in order to decide any
treatment switch.
The routine CS treat-to-target arm schedule is based on the recommendations of the STRIDE II.
When appropriate, based on the investigator's judgement, CS can be replaced by a RSS.
Since the two strategies are part of the standards of care, no additional procedures outside
the current clinical practice will be used in this study.
