Current partner codePEPTIDESDE
NCT02682381·Phase 3·INTERVENTIONAL

Short Bowel Syndrome Research Study for Children Up To 17 Years of Age on Parenteral Nutrition

Status

Completed

Phase

Phase 3

Enrollment

59

Locations

27

Results

Posted

Publications

1

Study summary

What the protocol is testing.

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.

Interventions

Treatment arms and agents.

DRUG

Teduglutide 0.05mg/kg

0.05 mg/kg

DRUG

Teduglutide 0.025 mg/kg

0.025 mg/kg

OTHER

Standard of Care

Observational cohort for the 24-week treatment period and 4 week follow-up.

Timeline

From registration to results.

  1. First posted

    Feb 15, 2016

  2. Study start

    Jun 23, 2016

  3. Primary completion

    Aug 18, 2017

  4. Study completion

    Aug 18, 2017

  5. Results posted

    Oct 16, 2018

  6. Registry updated

    Jun 9, 2021

Outcomes

What the study measures.

Primary outcomes

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

Time frame · 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.

Secondary outcomes

Number of Participants With Treatment-emergent Adverse Events (TEAEs)

Time frame · From start of study treatment up to 28 weeks

An adverse event (AE) was defined as any untoward medical occurrence in a clinical investigation participant administered a pharmaceutical product and that did not necessarily have a causal relationship with this treatment. TEAEs were defined as AEs that started or worsened on or after the date of first dose for treatment groups and those that started or worsened on or after the baseline visit for standard of care group.

Number of Participants Who Were Completely Weaned Off Parenteral Nutrition Intravenous (PN/IV) Support at Week 24

Time frame · Week 24

A participant was considered to have achieved independence from PN/IV support (completely weaned off PN/IV) if the investigator prescribed no PN/IV at EOT and there was no use of PN/IV recorded in the participant diary during the week prior to EOT.

Change From Baseline in Parenteral Nutrition Intravenous (PN/IV) Volume at Week 24

Time frame · Baseline, Week 24

Change in PN/IV volume was reported based on the participant diary and the investigator prescribed data.

Change From Baseline in Parenteral Nutrition Intravenous (PN/IV) Caloric Intake at Week 24

Time frame · Baseline, Week 24

Change in PN/IV caloric intake was reported based on the participant diary and the investigator prescribed data.

Change From Baseline in Plasma Citrulline Levels at Week 24

Time frame · Baseline, Week 24

Plasma citrulline level was reported.

Change From Baseline in Enteral Nutrition Volume at Week 24

Time frame · Baseline, Week 24

Enteral nutrition was defined as specialized formula taken orally or by tube feeding, and excluded table foods and other fluids. Change in enteral nutrition volume was reported.

Change From Baseline in Enteral Nutrition Caloric Intake at Week 24

Time frame · Baseline, Week 24

Enteral nutrition was defined as specialized formula taken orally or by tube feeding, and excluded table foods and other fluids. Change in enteral nutrition caloric intake was reported.

Change From Week 24 in Parenteral Nutrition Intravenous (PN/IV) Volume at Week 28

Time frame · Week 24, Week 28

Change in PN/IV volume was reported.

Change From Week 24 in Parenteral Nutrition Intravenous (PN/IV) Caloric Intake at Week 28

Time frame · Week 24, Week 28

Change in PN/IV caloric intake was reported.

Change From Week 24 in Plasma Citrulline Levels at Week 28

Time frame · Week 24, Week 28

Change in plasma citrulline level was reported.

Eligibility

Who can take part.

Minimum age
0 Years
Maximum age
17 Years
Sex
ALL
Healthy volunteers
No

Inclusion Criteria: 1. Informed consent by a parent or guardian or emancipated minor prior to any study-related procedures 2. When applicable, an informed assent by the subject (as deemed appropriate by the Ethics Committee/Institutional Review Board) prior to any study-related procedures 3. Current history of SBS as a result of major intestinal resection, (eg, due to necrotizing enterocolitis, midgut volvulus, intestinal atresia, or gastroschisis) 4. Short bowel syndrome that requires PN/IV support that provides at least 30% of caloric and/or fluid/electrolyte needs prior to screening 5. 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. 6. 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 Exclusion Criteria: 1. Subjects who are not expected to be able to advance oral or tube feeding regimens 2. Serial transverse enteroplasty or any other bowel lengthening procedure performed within 3 months of screening 3. Known clinically significant untreated intestinal obstruction contributing to feeding intolerance and inability to reduce parenteral support 4. Unstable absorption due to cystic fibrosis or known DNA abnormalities 5. 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. 6. Evidence of clinically significant obstruction on upper GI series done within 6 months prior to screening. 7. 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). 8. 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. 9. 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. 10. Pregnant or lactating female subjects (in the teduglutide treatment arm only). 11. 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. 12. Previous use of teduglutide or native/synthetic glucagon-like peptide-2 (GLP-2) 13. Previous use of glucagon-like peptide-1 analog or human growth hormone within 3 months prior to screening 14. Previous use of octreotide, or dipeptidyl peptidase-4 (DPP-4) inhibitors within 3 months prior to screening 15. 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 16. 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 17. 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 18. 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 19. Body weight \< 10 kg at the screening and baseline visits 20. Signs of active severe or unstable, clinically significant hepatic impairment during the screening period, as indicated by any of the following laboratory test results : 1. Total bilirubin (TBL) ≥ 2 x upper limit of normal (ULN) 2. Aspartate aminotransferase (AST) ≥ 7x ULN 3. Alanine aminotransferase (ALT) ≥ 7x ULN For subjects with Gilbert's disease: 4. Indirect (unconjugated) bilirubin ≥ 2x ULN 21. 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 m2. 22. 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 23. Unstable, clinically significant active, untreated pancreatic or biliary disease 24. 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.

Study locations

27 registered sites.

Belgium · Canada · Finland · Germany · Italy · United Kingdom · United States. Showing up to 24 locations stored in the fast local snapshot.

Children's Hospital Los Angeles - RHU

Los Angeles, California, United States

UCLA Dept. of Medicine

Los Angeles, California, United States

UCSF Benioff Children's Hospital

San Francisco, California, United States

Georgetown Children's Research Network

Washington D.C., District of Columbia, United States

Ann & Robert H Lurie Children's Hospital of Chicago

Chicago, Illinois, United States

Riley Hospital for Children

Indianapolis, Indiana, United States

Boston Children's Hospital

Boston, Massachusetts, United States

The Nebraska Medical Center

Omaha, Nebraska, United States

Children's Hospital GI Nutrition

New York, New York, United States

Montefiore Medical Center Child Spc

The Bronx, New York, United States

Duke Medical Center

Durham, North Carolina, United States

Cincinnati Children's Hospital Medical Center

Cincinnati, Ohio, United States

Cleveland Clinic Pediatric Specialists

Cleveland, Ohio, United States

University of Pennsylvania Medical Center

Philadelphia, Pennsylvania, United States

Children's Medical Center Dallas

Dallas, Texas, United States

Texas Children's Hospital

Houston, Texas, United States

Seattle Children's Hospital

Seattle, Washington, United States

University of Wisconsin School of Medicine and Public Health

Madison, Wisconsin, United States

Cliniques Universitaires Saint-Luc

Brussels, Belgium

Walter C. Mackenzie Health Science Center

Edmonton, Alberta, Canada

British Columbia Children's & Women's Hospital Center

Vancouver, British Columbia, Canada

The Hospital for Sick Children

Toronto, Ontario, Canada

Helsingin yliopistollinen keskussairaala

Helsinki, Finland

Universitaetsklinikum Tuebingen

Tübingen, Baden Wuertternberg, Germany

Related trials

More studies on Teduglutide.

Related PeptideStat pages

Put the record in context.

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