Octreotide Therapy in Children and Young Adults With Prader-Willi Syndrome (PWS)

What Was Tested

This registered not phased trial tested Octreotide.

The purpose of this study is to investigate over a 6 month period the effect of octreotide therapy on food intake, sense of hunger, body weight, body composition, efficiency of burning calories, biomarkers of weight regulation and growth hormone markers in children and young Adults with Prader-Willi Syndrome(PWS).

It had two arms (groups): Octreotide (experimental) and Placebo (placebo comparator).

Octreotide to be administered by subcutaneous injection three times daily while on study.

Placebo to be administered by subcutaneous injection three times daily while on study.

The registry classes the interventions as drug.

More on the Plan

Studies show that obese children are likely to become obese adults (2-5).

Recent studies have identified a new gastroenteric hormone, ghrelin, as a long-term regulator of energy balance in humans (12).

Ghrelin is a 28 amino acid acylated peptide which is an endogenous ligand of the growth hormone secretagogue receptor (GHS-R), a hypothalamic G-protein-coupled receptor (13).

Enteroendocrine cells (X/A-like cells) of the stomach are the major site of ghrelin synthesis, although a minor proportion of ghrelin synthesis occurs in other sites such as the hypothalamus, pituitary, duodenum, jejunum and lung (14) (15, 16).

Who Took Part

5 actual participants took part.

It was open to people of any sex aged from 5 years up to 21 years.

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

Examples of who could join: Diagnosis of PWS confirmed by chromosome analysis. Ages 5 years to 21 years. BMI for age ≥ (greater-than or equal to)85th percentile. Written informed consent and assent obtained and willingness to comply with the study schedule and procedures. Free T4, Thyroid stimulating hormone (TSH) values in the normal range (either endogenous or with thyroxine replacement).

The full record lists 5 inclusion and 3 exclusion criteria; among the exclusions: concomitant use of an investigational drug or octreotide in the past year; use of steroids for longer than 7 days within the past 30 days.

How the Study Was Run

The set-up was randomized, parallel-group and triple-blind.

Because people in this Octreotide study were split into groups at random, differences between the groups are less likely to reflect who happened to join each one.

The Octreotide group and the comparison group ran side by side rather than switching.

Neither the participants nor the staff measuring them knew who was on Octreotide, so expectations could not easily colour the readings.

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

It started in 2006-12 with completion listed for 2009-04.

It ran at 1 site in United States.

Its stated primary purpose is treatment.

What This Means in Plain Language

Put simply, this is a not phased, randomized trial of Octreotide in 5 people.

Its size is small, so on its own it can only give an early look.

It ended early (terminated); results are posted on the registry, and this page does not restate them.

More context: the Octreotide compound page, all human trials.