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)
试验速览
- 阶段
- 不适用
- 状态
- 招募中
- 发起方
- 入组人数
- 12
- 试验地点
- 4
- 主要终点
- Percentage of Participants Who Achieve at Least a 20% Reduction from Baseline in Weekly Parenteral Support (PS) Volume at Week 24
研究概览
简要总结
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.
研究设计
- 研究类型
- Observational
- 观察模型
- Cohort
- 时间视角
- Other
入排标准
- 年龄范围
- 1 Year 至 17 Years（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •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.
排除标准
- •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.
研究组 & 干预措施
Retrospective Group
Participants who completed the 24-week teduglutide treatment period before enrollment will have their clinical data collected retrospectively from their medical records.
干预措施: No Intervention (Other)
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.
干预措施: No Intervention (Other)
Prospective Group
Participants who will initiate the teduglutide treatment period after enrollment will have their clinical data collected prospectively for 48 weeks.
干预措施: No Intervention (Other)
结局指标
主要结局
Percentage of Participants Who Achieve at Least a 20% Reduction from Baseline in Weekly Parenteral Support (PS) Volume at Week 24
时间窗: 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.
次要结局
- Number of Participants Able to Completely Wean off PS Support at Week 24(At Week 24)
- Absolute Change from Baseline in PN/Intravenous (IV) Support Volume(Baseline up to Week 24)
- Percent Change From Baseline in PN/IV Support Volume(Baseline up to Week 24)
- Absolute Change From Baseline in PN/IV Support Calories(Baseline up to Week 24)
- Percent Change From Baseline in PN/IV Support Calories(Baseline up to Week 24)
- Absolute Change From Baseline in Plasma Citrulline(Baseline, Week 24)
- Percent Change From Baseline in Plasma Citrulline(Baseline, Week 24)
- Absolute Change From Baseline in Enteral Nutrition (EN) Support Volume(Baseline up to Week 24)
- Percent Change From Baseline in EN Support Volume(Baseline up to Week 24)
- Absolute Change From Baseline in EN Support Calories(Baseline up to Week 24)
- Percent Change From Baseline in EN Support Calories(Baseline up to Week 24)
- Change From Baseline in Hours per day of PN/IV Support(Baseline up to Week 24)
- Change From Baseline in Days per Week of PN/IV Support(Baseline up to Week 24)
- Percentage of Participants with at Least a 20% Reduction From Baseline in PN/IV Volume(Baseline up to Week 24)
- Change in Body Weight(Baseline up to Week 24)
- Change in Height(Baseline up to Week 24)
- Change in Head Circumference(Baseline up to Week 24)
- Change in Height Z-score(Baseline up to Week 24)
- Change in Weight Z-score(Baseline up to Week 24)
- Change in Body Mass Index (BMI)(Baseline up to Week 24)
- Change in BMI Z-score(Baseline up to Week 24)
- Number of Participants with Adverse Events (AEs). Serious Adverse Events (SAEs) and Adverse Events of Special Interest (AESIs)(Up to 50 weeks)
- Number of Participants with Clinically Significant Change in Vital Sign Values(Up to 24 weeks)
- Body Weight in Participants Dependent on PS(Baseline up to Week 24)
- Head Circumference in Participants Dependent on PS(Baseline up to Week 24)
- Height in Participants Dependent on PS(Baseline up to Week 24)
- Trends on Growth Chart(Baseline up to Week 24)
- Urine Output Determined up to Week 24(Baseline up to Week 24)
- Fecal Output Measured by Volume(Baseline up to Week 24)
- Fecal Output Measured by Number of Bowel Movements per Day(Baseline up to Week 24)
- Number of Participants with Gastrointestinal-specific Testing(Baseline up to Week 24)
- Number of Participants With Clinically Significant Changes in Physical Examinations(Up to 24 weeks)
- Number of Participants With Clinically Significant Changes in Laboratory Values(Up to 24 weeks)
- Number of Participants With Clinically Significant Changes in 12-Lead Electrocardiogram (ECG) Parameters(Up to 24 weeks)