Oxytocin Treatment in Neonates and Infants With Prader-Willi Syndrome

What Was Tested

The trial (phase 3) is built around Oxytocin.

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

Intranasal administration of OT.

Intranasal administration of placebo.

The registry classes the interventions as drug.

Who Took Part

Enrollment: 52 actual participants took part.

It was open to people of any sex aged from 1 Day up to 92 Days.

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

Examples of who could join: Male or female neonate or infant, with PWS genetically confirmed. Age \<92 days (plus a tolerance of up to 8 days maximum) (for preterm infants, born before 37 weeks amenorrhea, corrected age will be applied). Signed informed consent obtained from the parents/holders of parental authority. Parents willing and able to comply with all study procedures.

The full record lists 4 inclusion and 1 exclusion criteria; among the exclusions: 1\. neonate or infant currently admitted to the emergency care unit for ongoing life-threatening comorbidities like severe respiratory, cardiovascular or neurological abnormalities.

How the Study Was Run

It was run as a randomized, parallel-group and quadruple-blind study.

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

Groups ran in parallel: each of the participants stayed in one arm throughout.

As a double-blind Oxytocin study, the assignments were hidden from both participants and investigators until the end.

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

It started in 2020-03-10 with completion listed for 2021-10-16.

It ran at 7 sites in France (5), Belgium (1) and Germany (1).

Its stated primary purpose is treatment.

What Was Measured

The main thing measured (primary outcome): Neonatal Oral-Motor Assessment Scale (NOMAS) scale (time frame: 4 weeks).

It also tracked 3 secondary outcomes, for example Proficiency score.

What This Means in Plain Language

Put simply, this is a phase 3, randomized trial of Oxytocin in 52 people.

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.