Teduglutide treatment for 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)

This test will see if teduglutide helps Chinese children and teenagers with short bowel syndrome reduce how much intravenous nutrition they need.

Quick facts

• Study type: Observational
• Enrollment: 12 (estimated)
• Ages: 1 Year to 17 Years
• Sex: All
• Sponsor: Takeda (Industry-sponsored)
• Locations: 4 sites (Guangzhou, Guangdong and 3 other locations)
• Trial ID: NCT07319832 on ClinicalTrials.gov

What this trial studies

This observational study follows children and adolescents (1–17 years) with documented short bowel syndrome who are receiving or plan to receive teduglutide for at least 24 weeks. Investigators will record changes in parenteral support (PN/IV), growth and nutritional markers, and monitor safety events during routine care. Eligible participants typically have had stable PN/IV support for at least three months prior to enrollment, with allowance for brief interruptions that return to baseline. The study is being conducted at pediatric centers in Guangzhou and Shanghai and collects real-world treatment and safety data without assigning experimental interventions.

Who should consider this trial

Good fit: Children and adolescents aged 1–17 with documented short bowel syndrome who are receiving or will receive teduglutide for a minimum of 24 weeks and have had stable parenteral nutrition for about three months are the ideal candidates.

Not a fit: Children who are unable to advance oral or tube feeding regimens, or who are not candidates for teduglutide, are unlikely to benefit from this treatment.

Why it matters

Potential benefit: If successful, teduglutide could reduce dependence on parenteral nutrition and improve growth, nutrition, and quality of life for affected children.

How similar studies have performed: Teduglutide has previously helped many adults with SBS reduce parenteral support, and smaller pediatric reports and trials suggest similar benefits, so this approach has supportive prior data.

Eligibility criteria

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.

Where this trial is running

Guangzhou, Guangdong and 3 other locations

• Guangzhou Women And Children's Medical Center — Guangzhou, Guangdong, China (Not_yet_recruiting)
• Shanghai Children's Hospital — Shanghai, Shanghai Municipality, China (Not_yet_recruiting)
• Xinhua Hospital Affiliated to Shanghai Jiao Tong University School of Medicine — Shanghai, Shanghai Municipality, China (Recruiting)
• Children's Hospital Zhejiang University School of Medicine — Hangzhou, Zhejiang, China (Not_yet_recruiting)

Study contacts

• Study coordinator: Takeda Contact

Conditions Short Bowel Syndrome; Drug Therapy