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临床试验/NCT02682381
NCT02682381已完成3 期

A 24-Week Double-blind, Safety, Efficacy, and Pharmacodynamic Study Investigating Two Doses of Teduglutide in Pediatric Subjects Through 17 Years of Age With Short Bowel Syndrome Who Are Dependent on Parenteral Support

Shire27 个研究点 分布在 7 个国家目标入组 59 人开始时间: 2016年6月23日最近更新:
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适应症
干预措施
相关药物

试验速览

阶段
3 期
状态
已完成
发起方
Shire
入组人数
59
试验地点
27
主要终点
Number of Participants Who Achieved at Least a 20 Percent (%) Reduction in Weight-Normalized Average Daily Parenteral Nutrition Intravenous (PN/IV) Volume at Week 24
研究概览研究设计入排标准研究组 & 干预措施结局指标研究者研究点记录与标识符相似试验

研究概览

简要总结

Teduglutide is approved for treatment of adults with short bowel syndrome (SBS). The purpose of this study is to evaluate the safety and efficacy of teduglutide in children up to the age of 17 with SBS who are dependent on parenteral support. Subjects may choose whether to receive the study drug or to participate in a standard-of-care arm. All participants who complete the study may be eligible to receive the study drug in a long-term extension study.

研究设计

研究类型
Interventional
分配方式
Randomized
干预模型
Single Group
主要目的
Treatment
盲法
Triple (Participant, Care Provider, Investigator)

入排标准

年龄范围
0 Years 至 17 Years（Child）
性别
All
接受健康志愿者
否

入选标准

  • •Informed consent by a parent or guardian or emancipated minor prior to any study-related procedures
  • •When applicable, an informed assent by the subject (as deemed appropriate by the Ethics Committee/Institutional Review Board) prior to any study-related procedures
  • •Current history of SBS as a result of major intestinal resection, (eg, due to necrotizing enterocolitis, midgut volvulus, intestinal atresia, or gastroschisis)
  • •Short bowel syndrome that requires PN/IV support that provides at least 30% of caloric and/or fluid/electrolyte needs prior to screening
  • •Stable PN/IV support, defined as inability to significantly reduce PN/IV support, usually associated with minimal or no advance in enteral feeds (ie, 10% or less change in PN or advance in feeds) for at least 3 months prior to and during screening, as assessed by the investigator.
  • •Sexually active female subjects of child-bearing potential (in the teduglutide treatment arm only) must use medically acceptable methods of birth control during and 4 weeks after the treatment period

排除标准

  • •Subjects who are not expected to be able to advance oral or tube feeding regimens
  • •Serial transverse enteroplasty or any other bowel lengthening procedure performed within 3 months of screening
  • •Known clinically significant untreated intestinal obstruction contributing to feeding intolerance and inability to reduce parenteral support
  • •Unstable absorption due to cystic fibrosis or known DNA abnormalities
  • •Severe, known dysmotility syndrome, such as pseudo-obstruction or persistent, severe, active gastroschisis-related dysmotility, that is the primary contributing factor to feeding intolerance and inability to reduce parenteral support, prior to screening. Dysmotility is defined as severe if it is expected to limit the advancement of enteral feeding.
  • •Evidence of clinically significant obstruction on upper GI series done within 6 months prior to screening.
  • •Major GI surgical intervention including significant intestinal resection within 3 months prior to the screening visit (insertion of feeding tube, anastomotic ulcer repair, minor intestinal resections ≤ 10 cm, or endoscopic procedure is allowed).
  • •Unstable cardiac disease, congenital heart disease or cyanotic disease, with the exception of subjects who had undergone ventricular or atrial septal defect repair, and patent ductus arteriosus (PDA) ligation.
  • •History of cancer or clinically significant lymphoproliferative disease, not including resected cutaneous basal or squamous cell carcinoma, or in situ non aggressive and surgically resected cancer.
  • •Pregnant or lactating female subjects (in the teduglutide treatment arm only).
  • •Participation in a clinical study using an experimental drug (other than glutamine or Omegaven) within 3 months or 5.5 half-lives of the experimental drug, whichever is longer, prior to screening, and for the duration of the study.
  • •Previous use of teduglutide or native/synthetic glucagon-like peptide-2 (GLP-2)
  • •Previous use of glucagon-like peptide-1 analog or human growth hormone within 3 months prior to screening
  • •Previous use of octreotide, or dipeptidyl peptidase-4 (DPP-4) inhibitors within 3 months prior to screening
  • •Subjects with active Crohn's disease who had been treated with biological therapy (eg, antitumor necrosis factor [anti-TNF]) within the 6 months prior to the screening visit
  • •Subjects with inflammatory bowel disease (IBD) who require chronic systemic immunosuppressant therapy that had been introduced or changed during the 3 months prior to screening
  • •More than 3 SBS-related or PN-related hospital admissions (eg, documented infection-related catheter sepsis, clots, bowel obstruction, severe water-electrolyte disturbances) within 3 months prior to the screening visit
  • •Any major unscheduled hospital admission which affects parenteral support requirements within 1 month prior to or during screening, excluding uncomplicated treatment of bacteremia, central line replacement/repair, or issues of similar magnitude in an otherwise stable subject
  • •Body weight < 10 kg at the screening and baseline visits
  • •Signs of active severe or unstable, clinically significant hepatic impairment during the screening period, as indicated by any of the following laboratory test results :
  • •Total bilirubin (TBL) ≥ 2 x upper limit of normal (ULN)
  • •Aspartate aminotransferase (AST) ≥ 7x ULN
  • •Alanine aminotransferase (ALT) ≥ 7x ULN
  • •For subjects with Gilbert's disease:
  • •Indirect (unconjugated) bilirubin ≥ 2x ULN
  • •Signs of known continuous active or unstable, clinically significant renal dysfunction shown by results of an estimated glomerular filtration rate (eGFR) below 50 mL/min/1.73 m
  • •Parent(s) and/or subjects who are not capable of understanding or not willing to adhere to the study visit schedule and other protocol requirements
  • •Unstable, clinically significant active, untreated pancreatic or biliary disease
  • •Any condition, disease, illness, or circumstance that in the investigator's opinion puts the subject at any undue risk, prevents completion of the study, or interferes with analysis of the study results.

研究组 & 干预措施

0.025 mg/kg/day Teduglutide

Experimental

0.025 milligrams per kilogram per day (mg/kg/day) of teduglutide for 24 weeks.

干预措施: Teduglutide 0.025 mg/kg (Drug)

0.05 mg/kg/day Teduglutide

Experimental

0.05 mg/kg/day of teduglutide for 24 weeks.

干预措施: Teduglutide 0.05mg/kg (Drug)

Standard of care

Active Comparator

Observational cohort for the 24-week treatment period and 4 week follow-up. The subjects in the standard of care group will follow the same visit schedule as the randomized subjects.

干预措施: Standard of Care (Other)

结局指标

主要结局

Number of Participants Who Achieved at Least a 20 Percent (%) Reduction in Weight-Normalized Average Daily Parenteral Nutrition Intravenous (PN/IV) Volume at Week 24

时间窗: Baseline through Week 24

Reduction in weight-normalized PN/IV volume was performed using both participant diary and investigator prescribed data. Number of participants who achieved at least a 20% reduction in weight-normalized PN/IV volume between the baseline and week 24/EOT visit were reported.

次要结局

  • Number of Participants With Treatment-emergent Adverse Events (TEAEs)(From start of study treatment up to 28 weeks)
  • Change From Baseline in Enteral Nutrition Volume at Week 24(Baseline, Week 24)
  • Change From Baseline in Plasma Citrulline Levels at Week 24(Baseline, Week 24)
  • Number of Participants Who Were Completely Weaned Off Parenteral Nutrition Intravenous (PN/IV) Support at Week 24(Week 24)
  • Change From Baseline in Parenteral Nutrition Intravenous (PN/IV) Volume at Week 24(Baseline, Week 24)
  • Change From Week 24 in Enteral Nutrition Volume at Week 28(Week 24, Week 28)
  • Change From Baseline in Parenteral Nutrition Intravenous (PN/IV) Caloric Intake at Week 24(Baseline, Week 24)
  • Change From Week 24 in Parenteral Nutrition Intravenous (PN/IV) Volume at Week 28(Week 24, Week 28)
  • Change From Week 24 in Enteral Nutrition Caloric Intake at Week 28(Week 24, Week 28)
  • Change From Baseline in Body Weight Z-score at Week 28(Baseline, Week 28)
  • Change From Baseline in Body Height Z-score at Week 28(Baseline, Week 28)
  • Change From Baseline in Enteral Nutrition Caloric Intake at Week 24(Baseline, Week 24)
  • Change From Week 24 in Parenteral Nutrition Intravenous (PN/IV) Caloric Intake at Week 28(Week 24, Week 28)
  • Change From Week 24 in Plasma Citrulline Levels at Week 28(Week 24, Week 28)
  • Change From Baseline in Head Circumference Z-score at Week 28(Baseline, Week 28)
  • Change From Baseline in Body Mass Index (BMI) Z-score at Week 28(Baseline, Week 28)
  • Change From Baseline in Participants' Stool Consistency at Week 28(Baseline, Week 28)
  • Change From Baseline in Hours Per Day of Parenteral Nutrition Intravenous (PN/IV) Support at Week 24(Baseline, Week 24)
  • Change From Baseline in Days Per Week of Parenteral Nutrition Intravenous (PN/IV) Support at Week 24(Baseline, Week 24)

研究者

发起方
Shire
申办方类型
Industry
责任方
Sponsor

研究点 (27)

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