Development of a Novel Endoscopic Index and Patient-reported Outcome Measure for Clinical Trials in Participants With Permanent Ileostomy

To date, patients with Crohn's Disease (CD) and permanent ileostomies have been excluded from clinical trials for new treatments. To allow this patient population to be included in clinical trials, outcome and measurement tools are needed. This study aims to develop a Patient Reported Outcome (PRO) and Endoscopic Index (EI) for patients with Crohn's Disease (CD) and permanent ileostomy. The study will enroll about 50 participants and collect videos of endoscopies that are done as part of standard of care. The videos will be centrally read to identify features that represent a broad range of inflammation and will be used to develop the EI. Participants will also be asked to participate in interviews, to understand the symptoms and impacts that are most important to participants for the development of a PRO.

Study Overview

• Status: Recruiting
• Conditions: Crohns Disease

• Study Type: Observational
• Enrollment (Estimated): 50

Contacts and Locations

• Study Contact: Name: Robyn Garrels-Lierman; Phone Number: 642-204-2047; Email: robyn.garrels@alimentiv.com
• Study Contact Backup: Name: Ana Olteanu; Phone Number: 416-704-7734; Email: ana.olteanu@alimentiv.com

Study Locations

• Canada
  • Ontario
    • Hamilton, Ontario, Canada, L6S 0E2
      • Recruiting
      • McMaster University Medical Centre
      • Principal Investigator: Neeraj Narula
      • Contact: Jaimin Patel; Email: patej102@mcmaster.ca
    • London, Ontario, Canada, L6S 0E2
      • Recruiting
      • LHSC - University Campus
      • Principal Investigator: Vipul Jairath
      • Contact: Taylor Toth; Email: taylor.toth@lhsc.on.ca
• United States
  • Minnesota
    • Rochester, Minnesota, United States, 55905
      • Recruiting
      • Mayo Clinic- Rochester
      • Contact: Brian Will; Email: will.brian@mayo.edu
      • Principal Investigator: David Bruning
  • Missouri
    • St Louis, Missouri, United States, 63110
      • Recruiting
      • Washington University in St Louis School of Medicine
      • Contact: Lulu Huang; Email: luluhuang@wustl.edu
      • Principal Investigator: Parakkal Deepak
  • Ohio
    • Cleveland, Ohio, United States, 44195
      • Recruiting
      • Cleveland Clinic Foundation
      • Contact: Catherine Larson; Email: larsenc@ccf.org
      • Principal Investigator: Florian Rieder

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Adult; Older Adult
• Accepts Healthy Volunteers: No

• Sampling Method: Non-Probability Sample

Study Population

Approximately 50 participants who meet the inclusion and exclusion criteria will be enrolled in the study. The study population will ideally include approximately equal distributions of participants with disease activity categorized by a central reader as normal, mild, moderate, and severe, according to the VAS assessment. It is not possible to predetermine the number of participants with specific disease activity levels. The distribution of inflammatory disease activity across the study population will be monitored on an ongoing basis and recruitment strategies may be reviewed for opportunities to help maintain a balanced distribution. No more than 10 asymptomatic participants will be enrolled.

Description

Inclusion Criteria:

1. Adults (age ≥ 18 years) with a diagnosis of CD treated surgically with subtotal colectomy/proctocolectomy end ileostomy at least 12 months prior to the scheduled endoscopy procedure.
2. Planning to have an ileoscopy procedure scheduled as part of routine medical care.
3. Be proficient in the English language (ie, ability to read, write, speak, and understand English well enough to take part in the interview process without the aid of an interpreter).
4. Able and willing to participate fully in all aspects of this study.
5. Written informed consent must be obtained and documented.

Exclusion Criteria:

1. End or loop colostomy, end ileostomy for UC, continent ileostomy, ileal pouch anal anastomosis, end ileostomy with mucous fistula, double barrel ileostomy, urostomy, loop ostomy, or any type of end ileostomy not considered permanent at the time of enrolment (with no intent to restore continuity).
2. Peristomal skin complications such as pyoderma gangrenosum, abscess, or any other severe peristomal skin inflammation.
3. Stomal stenosis obviating ileoscopy, known small bowel stricture, major stoma prolapse, symptomatic parastomal hernia, parastomal hernia with subcutaneous loops of small bowel within the hernia, stoma in a crease/poorly fitting applicable resulting in severe peristomal skin irritation, or retracted stoma that prevents endoscopic evaluation.
4. Any actively draining fistula (eg, peristomal or peri-anal).
5. Evidence of a known small bowel stricture on cross-sectional imaging or inability to pass an endoscope within the past 6 months.
6. Known active Clostridoides difficile or other enteric infection.
7. Short bowel syndrome.
8. Predominant symptom(s) arising from a retained rectal stump.
9. Serious underlying disease other than CD that in the opinion of the investigator may interfere with the participant's ability to participate fully in the study procedures or provide nonconfounded descriptions of their CD and ostomy symptom experiences during the interview.
10. Prior enrolment in the current study.

Study Plan

How is the study designed?

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Develop a PRO instrument for patients with Crohn's Disease and Permanent Ileostomy	Concept elicitation interviews focusing on the symptoms and impacts relevant to patients with CD and permanent ileostomy will be conducted and used to develop a PRO. Cognitive interviews will subsequently be conducted to determine respondent comprehension and relevance of the items.	through study completion, and average of 1 year
Evaluate the intrarater reliability of 100-mm VAS.	Central readers will score endoscopy videos using the VAS, on 2 separate occasions at least 2 weeks apart. Intrarater reliability will be quantified using ICC, which are equivalent to weighted Kappa statistics in the case of ordinal data.	At baseline and 2 weeks
Evaluate the intrarater reliability of RAM identified items.	Central readers will score endoscopy videos for items identified during a RAND/UCLA appropriateness method, on 2 separate occasions at least 2 weeks apart. Intrarater reliability will be quantified using ICC, which are equivalent to weighted Kappa statistics in the case of ordinal data.	At baseline and 2 weeks
Evaluate the intrarater reliability of Rutgeerts score and its component items.	Central readers will score endoscopy videos using the Rutgeerts Score, on 2 separate occasions at least 2 weeks apart. Intrarater reliability will be quantified using ICC, which are equivalent to weighted Kappa statistics in the case of ordinal data.	At baseline and 2 weeks
Evaluate the intrarater reliability of CDEIS and its component items.	Central readers will score endoscopy videos using the CDEIS, on 2 separate occasions at least 2 weeks apart. Intrarater reliability will be quantified using ICC, which are equivalent to weighted Kappa statistics in the case of ordinal data.	At baseline and 2 weeks
Evaluate the intrarater reliability of SES-CD and its component items.	Central readers will score endoscopy videos using the SES-CD, on 2 separate occasions at least 2 weeks apart. Intrarater reliability will be quantified using ICC, which are equivalent to weighted Kappa statistics in the case of ordinal data.	At baseline and 2 weeks
Evaluate the interrater reliability of 100-mm VAS.	Central readers will score endoscopy videos using the VAS, on 2 separate occasions at least 2 weeks apart. Interrater reliability will be quantified using ICC, which are equivalent to weighted Kappa statistics in the case of ordinal data.	At baseline
Evaluate the interrater reliability of RAM identified items.	Central readers will score endoscopy videos for items identified during a RAND/UCLA appropriateness method, on 2 separate occasions at least 2 weeks apart. Interrater reliability will be quantified using ICC, which are equivalent to weighted Kappa statistics in the case of ordinal data.	At baseline
Evaluate the interrater reliability of Rutgeerts score and its component items.	Central readers will score endoscopy videos using the Rutgeerts score, on 2 separate occasions at least 2 weeks apart. Interrater reliability will be quantified using ICC, which are equivalent to weighted Kappa statistics in the case of ordinal data.	At baseline
Evaluate the interrater reliability of CDEIS and its component items.	Central readers will score endoscopy videos using the CDEIS, on 2 separate occasions at least 2 weeks apart. Interrater reliability will be quantified using ICC, which are equivalent to weighted Kappa statistics in the case of ordinal data.	At baseline
Evaluate the interrater reliability of SES-CD and its component items.	Central readers will score endoscopy videos using the SES-CD, on 2 separate occasions at least 2 weeks apart. Interrater reliability will be quantified using ICC, which are equivalent to weighted Kappa statistics in the case of ordinal data.	At baseline
Develop a novel index using a multiple regression approach with the dependent variables being the VAS and candidate independent variables including items having at least moderate interrater reliability.	A prototype index will be created using the VAS for global assessment of severity as the dependent criterion. Prospective validation of the endoscopic index will be performed in a future initiative outside of the scope of this study.	through study completion, and average of 1 year

Collaborators and Investigators

• Sponsor: Alimentiv Inc.
• Collaborators: The Cleveland Clinic; University of Western Ontario, Canada; The Leona M. and Harry B. Helmsley Charitable Trust; OPEN Health (Pharmerit)
• Investigators: Principal Investigator: Florian Rieder, The Cleveland Clinic; Principal Investigator: Vipul Jairath, Western University, Canada

Study record dates

Study Major Dates

• Study Start (Actual): June 19, 2024
• Primary Completion (Estimated): June 30, 2026
• Study Completion (Estimated): February 28, 2027

Study Registration Dates

• First Submitted: October 30, 2024
• First Submitted That Met QC Criteria: October 31, 2024
• First Posted (Actual): November 1, 2024

Study Record Updates

• Last Update Posted (Actual): November 12, 2025
• Last Update Submitted That Met QC Criteria: November 7, 2025
• Last Verified: November 1, 2025

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Intestinal Diseases; Digestive System Diseases; Gastrointestinal Diseases; Gastroenteritis; Inflammatory Bowel Diseases; Crohn Disease
• Other Study ID Numbers: HEL01847

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: No