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

Официальное название

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)

Обзор

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.

Вмешательства

• Другое No Intervention
  No Intervention

Первичные конечные точки

• Percentage of Participants Who Achieve at Least a 20% Reduction from Baseline in Weekly Parenteral Support (PS) Volume at Week 24 [Срок оценки: At Week 24]

Вторичные конечные точки (12)

• Number of Participants Able to Completely Wean off PS Support at Week 24 [Срок оценки: At Week 24]
• Absolute Change from Baseline in PN/Intravenous (IV) Support Volume [Срок оценки: Baseline up to Week 24]
• Percent Change From Baseline in PN/IV Support Volume [Срок оценки: Baseline up to Week 24]
• Absolute Change From Baseline in PN/IV Support Calories [Срок оценки: Baseline up to Week 24]
• Percent Change From Baseline in PN/IV Support Calories [Срок оценки: Baseline up to Week 24]
• Absolute Change From Baseline in Plasma Citrulline [Срок оценки: Baseline, Week 24]
• Percent Change From Baseline in Plasma Citrulline [Срок оценки: Baseline, Week 24]
• Absolute Change From Baseline in Enteral Nutrition (EN) Support Volume [Срок оценки: Baseline up to Week 24]
• Percent Change From Baseline in EN Support Volume [Срок оценки: Baseline up to Week 24]
• Absolute Change From Baseline in EN Support Calories [Срок оценки: Baseline up to Week 24]
• Percent Change From Baseline in EN Support Calories [Срок оценки: Baseline up to Week 24]
• Change From Baseline in Hours per day of PN/IV Support [Срок оценки: Baseline up to Week 24]

Критерии участия

Критерии включения

• 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.

Критерии исключения

• 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:

• Total Bilirubin Level (TBL) >= 2 × upper limit of normal (ULN)
• Aspartate Aminotransferase (AST) >=7 × ULN
• Alanine Aminotransferase (ALT) >=7 × ULN

For Participants with Gilbert's disease:

• 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.

Критерии приведены из реестра в оригинале (на английском). Окончательную оценку соответствия проводит исследовательский центр.

Здоровые добровольцы: Нет

Дизайн исследования

Модель наблюдения

Когортное

Центры проведения

Китай · 4 центра

• Guangzhou Women And Children's Medical Center — Гуанчжоу
• Shanghai Children's Hospital — Шанхай
• Xinhua Hospital Affiliated to Shanghai Jiao Tong University School of Medicine — Шанхай
• Children's Hospital Zhejiang University School of Medicine — Ханчжоу

Идентификаторы

NCT: NCT07319832 · TAK-633-5002

Первоисточники (государственные реестры)

• ClinicalTrials.gov (NIH, США)

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