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

What Was Tested

In this phase 3 trial, researchers set out to test Teduglutide (GLP-2 analog).

Teduglutide is approved for treatment of adults with short bowel syndrome (SBS).

Subjects may choose whether to receive the study drug or to participate in a standard-of-care arm.

It had three arms (groups): one (experimental), one (experimental) and 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.

The registry classes the interventions as drug and other.

Who Took Part

59 actual participants took part.

It was open to people of any sex aged from 0 years up to 17 years.

Healthy volunteers were not accepted; participants needed the condition being studied.

Examples of who could join: 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). 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.

The full record lists 6 inclusion and 28 exclusion criteria; among the exclusions: 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; unstable absorption due to cystic fibrosis or known dna abnormalities.

How the Study Was Run

It was run as a randomized, single-group and triple-blind study.

Random assignment meant chance, not the researchers, decided who got Teduglutide (GLP-2 analog) and who got the comparison, which limits bias.

Blinding on both sides kept the participants and researchers unaware of who received Teduglutide (GLP-2 analog).

Everyone received the same intervention, so there was no separate comparison group inside the trial.

Those kept unaware of assignments: participant, care provider and investigator.

It started in 2016-06-23 with completion listed for 2017-08-18.

It ran at 27 sites in United States (18), Canada (3), United Kingdom (2), Belgium (1), Finland (1) and Germany (1).

Its stated primary purpose is treatment.

What Was Measured

It also tracked 19 secondary outcomes, for example Number of Participants With Treatment-emergent Adverse Events (TEAEs), Number of Participants Who Were Completely Weaned Off Parenteral Nutrition Intravenous (PN/IV) Support at Week 24, Change From Baseline in Parenteral Nutrition Intravenous (PN/IV) Volume at Week 24, Change From Baseline in Parenteral Nutrition Intravenous (PN/IV) Caloric Intake at Week 24, Change From Baseline in Plasma Citrulline Levels at Week 24 and Change From Baseline in Enteral Nutrition Volume at Week 24.

What This Means in Plain Language

Put simply, this is a phase 3, randomized trial of Teduglutide (GLP-2 analog) in 59 people.

The trial is marked completed; results are posted on the registry, and this page does not restate them.

More context: the Teduglutide (GLP-2 analog) compound page, all human trials.