Tolerance of Intranasal Administration of OT in Prader-Willi Newborn Babies

What Was Tested

The trial (phase 1, phase 2) is built around Oxytocin.

Background: Prader-Willi syndrome (PWS) is a rare, complex multisystem genetic disorder arising from the lack of expression of paternally inherited imprinted genes on chromosome 15q11-q13.

The syndrome includes severe neonatal hypotonia with impaired suckling leading to failure to thrive in the most severe cases, subsequently followed by an early onset of morbid obesity with insatiable hunger, combined with other endocrine dysfunction probably due to hypothalamic dysfunction.

The pathophysiological mechanism of the occurrence of the 2 main nutritional phases of PWS is unknown.

It had 1 arm (groups): Oxytocin (experimental).

The registry classes the interventions as drug.

More on the Plan

The researchers want to evaluate the tolerance of the intranasal administration of OT in 6 infants with PWS genetically confirmed and its effect on suckling, milk intake and weight gain.

The researchers will monitor cardiac pulse, blood pressure, urine emission and measure biological safety parameters (glycemia, natremia and kaliemia).

Video will be performed during 1 day before the drug administration and the 3 days after in order to qualify the suckling.

Quantitative evaluation of the milk intake during each feeding and per day will be also evaluated.

Who Took Part

5 actual participants took part.

It was open to people of any sex aged from 15 Days up to 5 months.

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

Examples of who could join: Neonates with genetic diagnosis of Prader-Willi syndrome. Aged from 15 days to 5 months.

The full record lists 2 inclusion and 2 exclusion criteria; among the exclusions: exclusive tube feeding.

How the Study Was Run

Design: single-group and open-label.

Everyone knew when Oxytocin was being given (open label), which is simpler to run but leaves more room for expectations to affect what is reported.

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

It started in 2011-07 with completion listed for 2012-02.

It ran at 1 site in France.

Its stated primary purpose is treatment.

What Was Measured

Primary outcome: Occurrence of adverse event, description and quantification of their severity, imputability to oxytocin administration (time frame: up to day 8).

It also tracked 2 secondary outcomes, for example Quantitative evaluation of food intake.

What This Means in Plain Language

Put simply, this is a phase 1, phase 2 trial of Oxytocin in 5 people.

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

The trial is marked completed; no results are posted to the registry yet, so nothing here describes outcomes.

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