TED_ORG: Study on Short Bowel Syndrome

Short Bowel Syndrome: Human Intestinal Organoids to Investigate the Different Efficacy of the GLP-2 Analogue Teduglutide in Pediatric Patients With Short Bowel Syndrome

The goal of this clinical trial is to understand why pediatric patients with short bowel syndrome respond differently to treatment with the glucagon-like peptide-2 (GLP-2) analogue teduglutide. Short bowel syndrome is a rare and severe condition in children that results from extensive intestinal resection and leads to impaired nutrient absorption, chronic diarrhea, and dependence on parenteral nutrition. Although teduglutide is known to promote intestinal adaptation and improve absorption, the clinical response varies widely among patients, and the biological mechanisms underlying this variability are not fully understood.

This study aims to investigate the effects of teduglutide using human intestinal organoids derived from intestinal tissue samples of pediatric patients with short bowel syndrome. Intestinal organoids are three-dimensional structures grown from patient-derived stem cells that reproduce key structural and functional characteristics of the human intestine. These organoids provide a human-based experimental model that allows the study of intestinal morphology, cellular behavior, and nutrient absorption in a controlled in vitro environment.

The main questions this study aims to answer are:

Does treatment with teduglutide improve the absorptive capacity of human intestinal organoids derived from pediatric patients with short bowel syndrome?

Are there differences in intestinal structure, cellular proliferation, and gene expression between teduglutide-treated organoids and untreated organoids?

Are specific molecular or cellular features associated with different responses to teduglutide?

Researchers will compare intestinal organoids treated with teduglutide to untreated organoids obtained from the same patients. This comparison will be used to evaluate changes in organoid morphology, expression of receptors involved in intestinal growth and absorption, activity of nutrient transporters, and overall absorptive function. The study will also explore differences between organoids derived from patients who show different clinical responses to teduglutide.

Participants in this study are pediatric patients with short bowel syndrome or patients undergoing intestinal resection surgery as part of their standard clinical care. No experimental treatment is administered directly to participants as part of this study. Intestinal tissue samples are collected only during clinically indicated surgical procedures and are not obtained specifically for research purposes.

Participants will:

Provide intestinal tissue samples collected during routine or clinically indicated intestinal surgery

Have intestinal organoids generated from their tissue samples using established laboratory techniques

Have their organoids studied in vitro with and without exposure to teduglutide to evaluate intestinal structure, gene and protein expression, and nutrient absorption mechanisms

The results of this study are expected to improve understanding of the biological mechanisms underlying variable responses to teduglutide and may contribute to the development of more personalized treatment strategies for pediatric patients with short bowel syndrome in the future.

Study Overview

• Status: Recruiting
• Conditions: Short Bowel Syndrome (SBS)
• Intervention / Treatment: Drug: Teduglutide (ALX-0600); Other: No Treatment Added

• Study Type: Interventional
• Enrollment (Estimated): 50
• Phase: Not Applicable

Contacts and Locations

Study Locations

• Italy
  • Florence
    • Florence, Florence, Italy
      • Recruiting
      • Aou Meyer IRCSS
      • Contact: Antonino Morabito; Phone Number: +390555662545; Email: antonino.morabito@meyer.it

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Child
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

• Patients or His/her Parents/ legal guardian must provide informed consent before they can participate in the study.
• Paediatric patients: Male and female patients aged ≥ 4 months old and ≤ 18 years;
• SBS patient or patient undergoing intestinal resective surgery

Exclusion Criteria:

• Adult patients (≥ 18 years old);
• Patients who have never undergone intestinal resective surgery
• Current or past use of teduglutide

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Basic Science
• Allocation: Non-Randomized
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Other: teduglutide-treated organoids; Human intestinal organoids derived from pediatric patients with short bowel syndrome are treated in vitro with teduglutide to assess its effects on intestinal morphology, gene and protein expression, and nutrient absorption.	Drug: Teduglutide (ALX-0600); Teduglutide is a glucagon-like peptide-2 (GLP-2) analogue. In this study, teduglutide is used in vitro on human intestinal organoids derived from pediatric patients with short bowel syndrome. The intervention is applied only to organoid cultures and is not administered directly to study participants. Teduglutide-treated organoids are compared with untreated control organoids to evaluate differences in intestinal structure, gene and protein expression, and nutrient absorption.
Other: Untreated organoids; Human intestinal organoids derived from pediatric patients with short bowel syndrome are maintained in vitro without exposure to teduglutide and serve as a control group for comparison.	Other: No Treatment Added; No Treatment Added

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Intestinal organoid diameter after in vitro exposure to teduglutide	Quantitative measurement of intestinal organoid diameter assessed by image-based analysis in patient-derived human intestinal organoids treated in vitro with teduglutide and compared with untreated organoids from the same participant.	During in vitro treatment period (up to 7 days)
Crypt-villus-like architecture in intestinal organoids	Quantitative assessment of crypt-villus-like architectural features in patient-derived human intestinal organoids following in vitro exposure to teduglutide, evaluated by image-based morphological analysis and compared with untreated organoids from the same participant.	During in vitro treatment period (up to 7 days)
Organoid shape (sphericity) after in vitro teduglutide treatment	Quantitative measurement of organoid shape, expressed as sphericity index, in patient-derived human intestinal organoids treated in vitro with teduglutide and compared with untreated organoids from the same participant.	During in vitro treatment period (up to 7 days)
Lumen size in intestinal organoids after in vitro exposure to teduglutide	Quantitative measurement of lumen size assessed by image-based analysis in patient-derived human intestinal organoids treated in vitro with teduglutide and compared with untreated organoids from the same participant.	During in vitro treatment period (up to 7 days)
Intestinal organoid volume after in vitro exposure to teduglutide	Quantitative measurement of intestinal organoid volume assessed by image-based analysis in patient-derived human intestinal organoids treated in vitro with teduglutide and compared with untreated organoids from the same participant.	During in vitro treatment period (up to 7 days)
Lumen integrity in intestinal organoids after in vitro exposure to teduglutide	Quantitative assessment of lumen integrity assessed by image-based analysis in patient-derived human intestinal organoids treated in vitro with teduglutide and compared with untreated organoids from the same participant.	During in vitro treatment period (up to 7 days)

Collaborators and Investigators

• Sponsor: Meyer Children's Hospital IRCCS

Study record dates

Study Major Dates

• Study Start (Actual): October 30, 2024
• Primary Completion (Estimated): March 1, 2026
• Study Completion (Estimated): October 1, 2026

Study Registration Dates

• First Submitted: January 20, 2026
• First Submitted That Met QC Criteria: February 6, 2026
• First Posted (Actual): February 10, 2026

Study Record Updates

• Last Update Posted (Actual): February 10, 2026
• Last Update Submitted That Met QC Criteria: February 6, 2026
• Last Verified: January 1, 2026

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Postoperative Complications; Pathologic Processes; Intestinal Diseases; Digestive System Diseases; Gastrointestinal Diseases; Malabsorption Syndromes; Pathological Conditions, Signs and Symptoms; Short Bowel Syndrome; teduglutide; ALX-0600
• Other Study ID Numbers: TED_ORG

Drug and device information, study documents

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