A Study of Teduglutide in Chinese Children and Teenagers With Short Bowel Syndrome

A Multicenter, Retrospective and Prospective, Observational Study to Evaluate the Efficacy and Safety of Subcutaneous Teduglutide in the Treatment of Short Bowel Syndrome (SBS) in Parenteral Nutrition (PN) Dependent Chinese Pediatric Subjects (≥1 Through 17 Years Old)

Short Bowel Syndrome (SBS) is a rare condition that happens when a large part of the bowel (also called intestine) is missing or has been removed because of illness or surgery. In children, SBS means that the intestine cannot absorb enough food, water and important part of food the body needs (called nutrients) because a big part of it has been removed, bypassed or did not develop normally at birth and the children need support through a vein (parenteral support or PS) for more than 42 days to stay healthy and keep their energy. SBS in children is defined mainly by how well the intestine works and how long the children need this support, not just by how long the intestine is.

The main aim of the study is to learn how well the teduglutide works in children and teenagers with SBS and who need PS. Another aim is to find out how well teduglutide works for participants to lower the amount of PS needed. Also, the study wants to learn more about how safe teduglutide is in children and teenagers with SBS who need PS.

The study will review data already existing in the medical records of participants as well as collect new data during the study.

Study Overview

• Status: Recruiting
• Conditions: Short Bowel Syndrome (SBS)
• Intervention / Treatment: Other: No Intervention

• Study Type: Observational
• Enrollment (Estimated): 12

Contacts and Locations

• Study Contact: Name: Takeda Contact; Phone Number: +1-877-825-3327; Email: medinfoUS@takeda.com

Study Locations

• China
  • Guangdong
    • Guangzhou, Guangdong, China, 510040
      • Not yet recruiting
      • Guangzhou Women and Children's Medical Center
      • Principal Investigator: Jiakang Yu
  • Shanghai Municipality
    • Shanghai, Shanghai Municipality, China, 200062
      • Not yet recruiting
      • Shanghai Children's Hospital
      • Principal Investigator: Zhibao Lv
    • Shanghai, Shanghai Municipality, China, 200092
      • Recruiting
      • Xinhua Hospital Affiliated to Shanghai Jiao Tong University School of Medicine
      • Principal Investigator: Fan Lv
  • Zhejiang
    • Hangzhou, Zhejiang, China, 310006
      • Not yet recruiting
      • Children's Hospital ZheJiang University School of Medicine
      • Principal Investigator: Jinfa Tou

Participation Criteria

Eligibility Criteria

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

• Sampling Method: Non-Probability Sample

Study Population

The study population consists of Chinese pediatric participants with SBS who are dependent on PS.

Description

Inclusion criteria

• Children and adolescents greater than or equal to (>=)1 through 17 years of age at Day 1 (D1).
• Documented diagnosis of SBS.
• Received or plan to receive Teduglutide treatment for a minimum of 24 weeks.
• Stable PN/IV support, defined as inability to significantly reduce PN/IV support, usually associated with minimal or no advance in enteral feeds (i.e., 10% or less change in PN or advance in feeds) for at least 3 months prior to D1, as assessed by the investigator. Transient instability for events such as interruption of central access or treatment for sepsis is allowed if the PN/IV support returns to within 10% of baseline prior to the event.
• Informed consent obtained from the patient aged 8 to 17 years and their guardians, while informed consent from the guardians for participants under 8 years old, unless waived by the Institution's Ethics Committee.

Exclusion criteria

• Participants who are not expected to be able to advance oral or tube feeding regimens.
• Serial Transverse Enteroplasty (STEP) or any other bowel lengthening procedure performed within 3 months prior to baseline.
• Known clinically significant untreated intestinal obstruction contributing to feeding intolerance and inability to reduce PS.
• Evidence of clinically significant obstruction on upper GI series done within 6 months prior to baseline.
• Previous use of octreotide or Dipeptidyl peptidase-4 (DPP-4) inhibitors within 3 months prior to baseline.

• Signs of active, severe, or unstable clinically significant hepatic impairment during the screening or baseline period, indicative by any of the following laboratory test results:

  1. Total Bilirubin Level (TBL) >= 2 × upper limit of normal (ULN)
  2. Aspartate Aminotransferase (AST) >=7 × ULN

  3. Alanine Aminotransferase (ALT) >=7 × ULN

     For Participants with Gilbert's disease:

  4. Indirect (unconjugated) bilirubin >=2 × ULN
• Signs of known continuous active or unstable, clinically significant renal dysfunction shown by results of an estimated glomerular filtration rate (eGFR) below 50 millilitres per minutes per 1.73 meter square (mL/min/1.73 m^2).
• Known hypersensitivity of the active substance or excipient of teduglutide.
• Body weight less than (<) 10 kg at baseline.
• Previous use of teduglutide or native/synthetic Glucagon-like Peptide-2 (GLP-2).
• Previous use of GLP-1 analog or human growth hormone within 3 months prior to baseline.
• Any condition, disease, illness, or circumstance that in the investigator's opinion puts the patient at any undue risk, prevents completion of the study, or interferes with analysis of the study results.

Study Plan

How is the study designed?

Number of groups / cohorts

3

Cohorts and Interventions

Group / Cohort	Intervention / Treatment
Retrospective Group; Participants who completed the 24-week teduglutide treatment period before enrollment will have their clinical data collected retrospectively from their medical records.	Other: No Intervention; No Intervention
Mixed Group; Participants who initiated treatment before the study start date but have not completed the 24-week teduglutide treatment period at the time of enrollment will have their clinical data collected retrospectively before enrollment and prospectively after enrollment.	Other: No Intervention; No Intervention
Prospective Group; Participants who will initiate the teduglutide treatment period after enrollment will have their clinical data collected prospectively for 48 weeks.	Other: No Intervention; No Intervention

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Percentage of Participants Who Achieve at Least a 20% Reduction from Baseline in Weekly Parenteral Support (PS) Volume at Week 24	Percentage of participants who achieve at least a 20 percent (%) reduction from baseline in weekly PS volume at Week 24 will be reported.	At Week 24

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Number of Participants Able to Completely Wean off PS Support at Week 24	Number of participants who are able to completely wean off PS support at Week 24 will be reported.	At Week 24
Absolute Change from Baseline in PN/Intravenous (IV) Support Volume	Absolute change from baseline in PN/IV support volume will be reported.	Baseline up to Week 24
Percent Change From Baseline in PN/IV Support Volume	Percent change from baseline in PN/IV support volume will be reported.	Baseline up to Week 24
Absolute Change From Baseline in PN/IV Support Calories	Absolute change from baseline in PN/IV support calories will be reported.	Baseline up to Week 24
Percent Change From Baseline in PN/IV Support Calories	Percent change from baseline in PN/IV support calories will be reported.	Baseline up to Week 24
Absolute Change From Baseline in Plasma Citrulline	Absolute change from baseline in plasma citrulline will be reported.	Baseline, Week 24
Percent Change From Baseline in Plasma Citrulline	Percent change from baseline in plasma citrulline will be reported.	Baseline, Week 24
Absolute Change From Baseline in Enteral Nutrition (EN) Support Volume	Absolute change from baseline in EN support volume will be reported.	Baseline up to Week 24
Percent Change From Baseline in EN Support Volume	Percent change from baseline in EN support volume will be reported.	Baseline up to Week 24
Absolute Change From Baseline in EN Support Calories	Absolute change from baseline in EN support calories will be reported.	Baseline up to Week 24
Percent Change From Baseline in EN Support Calories	Percent change from baseline in EN support calories will be reported.	Baseline up to Week 24
Change From Baseline in Hours per day of PN/IV Support	Change from baseline in hours per day of PN/IV support will be reported.	Baseline up to Week 24
Change From Baseline in Days per Week of PN/IV Support	Change from baseline in days per week of PN/IV support will be reported.	Baseline up to Week 24
Percentage of Participants with at Least a 20% Reduction From Baseline in PN/IV Volume	Percentage of participants with at least a 20% reduction from baseline in PN/IV volume will be reported.	Baseline up to Week 24
Change in Body Weight	Change in body weight will be reported.	Baseline up to Week 24
Change in Height	Change in height will be reported.	Baseline up to Week 24
Change in Head Circumference	Head circumference will be measured in participants 36 months of age and younger.	Baseline up to Week 24
Change in Height Z-score	Height is measured using Z-score. Z score is calculated as ([observed value/M]^L-1)/S*L, where observed value is the child's height. The L, M, and S values vary according to the child's sex and age.	Baseline up to Week 24
Change in Weight Z-score	Weight is measured using Z-score. Z score is calculated as ([observed value/M]^L-1)/S*L, where observed value is the child's weight. The L, M, and S values vary according to the child's sex and age.	Baseline up to Week 24
Change in Body Mass Index (BMI)	BMI is a simple measure that uses a participant's weight and height to estimate whether they are underweight, normal weight, overweight, or obese. It is calculated as weight in kilograms (kg) divided by height in meters squared (kg/m^2). Change in BMI will be reported.	Baseline up to Week 24
Change in BMI Z-score	BMI is measured using Z-score. Z score is calculated as ([observed value/M]^L-1)/S*L, where observed value is the child's BMI. The L, M, and S values vary according to the child's sex and age.	Baseline up to Week 24
Number of Participants with Adverse Events (AEs). Serious Adverse Events (SAEs) and Adverse Events of Special Interest (AESIs)	An AE is defined as any untoward medical occurrence in a clinical investigation participant administered a pharmaceutical product. An AE does not necessarily have a causal relationship withthis treatment. An AE can therefore be any unfavorable and unintended sign (including a clinically significant abnormal laboratory finding, for example), symptom, or disease temporally associated with the use of a medicinal product (investigational or marketed), whether or not considered related to treatment with the medicinal product. SAE is any untoward medical occurrence that results in death, is life-threatening, requires inpatient hospitalization or prolongation of hospitalization, results in persistent or significant disability/incapacity, results in a congenital abnormality/birth defect, or is an important medical event. An AESI is an AE (serious or nonserious) of scientific and medical concern specific to the product or program.	Up to 50 weeks
Number of Participants with Clinically Significant Change in Vital Sign Values	Vital signs will include measurement of temperature, heart rate, blood pressure and respiratory rate. Any clinically significant change in vital signs will be determined at the investigator's discretion.	Up to 24 weeks
Body Weight in Participants Dependent on PS	Body weight in kg will be collected and reported.	Baseline up to Week 24
Head Circumference in Participants Dependent on PS	Head circumference in participants 36 months of age and younger will be collected and reported.	Baseline up to Week 24
Height in Participants Dependent on PS	Height in centimeters (cm) will be collected and reported.	Baseline up to Week 24
Trends on Growth Chart	Trends on growth chart will be collected and reported.	Baseline up to Week 24
Urine Output Determined up to Week 24	Collected and calculated values of urine output will be recorded over a 48-hour period of PN/IV and EN stability before every clinic visit and within 1 week of implementing a change in the PN/IV prescription.	Baseline up to Week 24
Fecal Output Measured by Volume	Collected and calculated values of fecal output should be recorded over a 48-hour period of PN/IV and EN stability before every clinic visit and within 1 week of implementing a change in the PN/IV prescription.	Baseline up to Week 24
Fecal Output Measured by Number of Bowel Movements per Day	Fecal output should be recorded over a 48-hour period of PN/IV and EN stability before every clinic visit and within 1 week of implementing a change in the PN/IV prescription.	Baseline up to Week 24
Number of Participants with Gastrointestinal-specific Testing	Gastrointestinal-specific testing will include colonoscopy or sigmoidoscopy or upper gastrointestinal (GI) endoscopy, abdominal ultrasound, fecal occult blood testing (FOBT), upper gastrointestinal series small bowel follow-through (UGI SBFT).	Baseline up to Week 24
Number of Participants With Clinically Significant Changes in Physical Examinations	Physical examination will include assessing the participants physical status. (general surface, skin, head and neck, chest [heart, lungs, breasts], abdomen [gastrointestinal, liver and gallbladder], back, urinary system, limbs, nervous system, lymph nodes, etc.). Any clinically significant change in physical examination will be determined at the investigator's discretion.	Up to 24 weeks
Number of Participants With Clinically Significant Changes in Laboratory Values	Laboratory parameters will include hematology, chemistry and urinalysis. Any clinically significant change in laboratory values will be determined at the investigator's discretion.	Up to 24 weeks
Number of Participants With Clinically Significant Changes in 12-Lead Electrocardiogram (ECG) Parameters	The 12-lead ECG will be evaluated. Any clinically significant change in ECG assessment will be determined at the investigator's discretion.	Up to 24 weeks

Collaborators and Investigators

• Sponsor: Takeda
• Investigators: Study Director: Study Director, Takeda

Publications and helpful links

Helpful Links

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• Click here for more information about this trial in easy-to-understand language.

Study record dates

Study Major Dates

• Study Start (Actual): March 5, 2026
• Primary Completion (Estimated): April 30, 2027
• Study Completion (Estimated): September 30, 2027

Study Registration Dates

• First Submitted: December 22, 2025
• First Submitted That Met QC Criteria: December 22, 2025
• First Posted (Actual): January 6, 2026

Study Record Updates

• Last Update Posted (Actual): March 13, 2026
• Last Update Submitted That Met QC Criteria: March 11, 2026
• Last Verified: March 1, 2026

More Information

Terms related to this study

• Keywords: Drug Therapy
• 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
• Other Study ID Numbers: TAK-633-5002

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: YES
• IPD Plan Description: Takeda provides access to the de-identified individual participant data (IPD) for eligible studies to aid qualified researchers in addressing legitimate scientific objectives (Takeda's data sharing commitment is available on https://clinicaltrials.takeda.com/takedas-commitment?commitment=5). These IPDs will be provided in a secure research environment following approval of a data sharing request, and under the terms of a data sharing agreement.
• IPD Sharing Access Criteria: IPD from eligible studies will be shared with qualified researchers according to the criteria and process described on https://vivli.org/ourmember/takeda/. For approved requests, the researchers will be provided access to anonymized data (to respect patient privacy in line with applicable laws and regulations) and with information necessary to address the research objectives under the terms of a data sharing agreement.
• IPD Sharing Supporting Information Type: STUDY_PROTOCOL; SAP; ICF; CSR

Drug and device information, study documents

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