Early oxytocin treatment in infants with Prader-Willi syndrome is safe and is associated with better endocrine, metabolic and behavioral outcomes.

Valette M, Diene G, Glattard M, Cortadellas J, Molinas C, Faye S, Benvegnu G, Boulanouar K, Payoux P, Salles JP, Arnaud C, Çabal S, Tauber M

Orphanet journal of rare diseases1 Mar 2025Cited by 11 (Europe PMC)

PMID40025514PMCIDPMC11872305DOI10.1186/s13023-025-03560-3PepBankPUB-40025514

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Summary

BackgroundOxytocin (OT) plays an important role in modulating behavior, social interactions and feeding. Prader-Willi syndrome (PWS), a rare genetic neurodevelopmental disorder, is a model of hypothalamic disorder including OT dysfunction. We previously showed that infants with PWS who had received an early short course (7 days) of intranasal OT treatment improved their oral and social skills. We aim to document the long-term tolerance and effects of early intranasal OT treatment on the disease trajectory.MethodsWe performed a comparative clinical trial including the 17 children who had received OT as infants in our previous study and compared them to 17 PWS non-exposed children at 3-4 years old. Primary endpoint was the total communication score on the Vineland Adaptive Behavior Scales-2nd edition (VABS-II). Secondary endpoints were the other domains of VABS-II, behavior scored by the Child Behavior Checklist, feeding skills, endocrine and metabolic profiles, and brain connectivity on functional magnetic resonance imaging.ResultsWe documented the long-term safety of early OT treatment. The VABS-II communication score was not different between the two groups, defined as OT-exposed and non-exposed, whereas a trend toward a higher socialization score was found in the OT-exposed children (p = 0.06). Circulating IGF-1 and HDL cholesterol were significantly higher in the OT-exposed group (p < 0.05). OT-exposed children had normal acylated ghrelin levels, which were lower than those observed in non-exposed children (p = 0.06), and they displayed higher connectivity of the orbitofrontal cortex brain region.ConclusionEarly OT treatment in infants with PWS is safe up to 3-4 years of age. OT-exposed children display better social, endocrine and metabolic outcomes. This study documents for the first time in human the biological window of opportunity of early OT treatment, which may change the trajectory of the PWS condition.Trial registrationClinical trial NCT03081832 Retrospectively registered https://clinicaltrials.gov/search?cond=NCT03081832 .

CC BYText from Europe PMC. Highlighted sentences are the source of results.

Results from this study7

7 results from studies that gave the peptide. 7 results from people.

01HumanNon-randomized trialEfficacy outcome
In children with Prader-Willi syndrome aged 3-4 years, the VABS-II communication score did not differ between those exposed to oxytocin as infants and non-exposed children.
In plain wordsIn children with Prader-Willi syndrome, communication scores were not different between children who had oxytocin as infants and those who had not.
Peptide
Oxytocin
Outcome
No significant changeVABS-II total communication score
Study
n = 34
Comparator
Non-exposed children with Prader-Willi syndrome
Analysis
Primary
Outcome group
Social behavior and cognition · Nervous system and cognition
Studied dose
Dose not stated · intranasal · 7 daysAs given in this study (human).
Source sentences
Summary
The VABS-II communication score was not different between the two groups, defined as OT-exposed and non-exposed
3 context sentences(design, population, supporting numbers)
Summary · context
Primary endpoint was the total communication score on the Vineland Adaptive Behavior Scales-2nd edition (VABS-II).
Summary · context
We previously showed that infants with PWS who had received an early short course (7 days) of intranasal OT treatment improved their oral and social skills.
Summary · context
We performed a comparative clinical trial including the 17 children who had received OT as infants in our previous study and compared them to 17 PWS non-exposed children at 3-4 years old.
Show in the study text Report a problemFND-4002551402
02HumanNon-randomized trialEfficacy outcome
In children with Prader-Willi syndrome aged 3-4 years, a trend toward a higher socialization score in oxytocin-exposed children did not reach significance (p = 0.06).
In plain wordsIn children with Prader-Willi syndrome, children given oxytocin as infants showed a possible, not clearly significant, trend to better social scores.
Peptide
Oxytocin
Outcome
No significant changeVABS-II socialization score
Study
n = 34
Comparator
Non-exposed children with Prader-Willi syndrome
Analysis
Secondary
Outcome group
Social behavior and cognition · Nervous system and cognition
Studied dose
Dose not stated · intranasal · 7 daysAs given in this study (human).
Measurements
p value
0.06
socialization score
Source sentences
Summary
whereas a trend toward a higher socialization score was found in the OT-exposed children (p = 0.06).
2 context sentences(design, population, supporting numbers)
Summary · context
We previously showed that infants with PWS who had received an early short course (7 days) of intranasal OT treatment improved their oral and social skills.
Summary · context
We performed a comparative clinical trial including the 17 children who had received OT as infants in our previous study and compared them to 17 PWS non-exposed children at 3-4 years old.
Show in the study text Report a problemFND-4002551403
03HumanNon-randomized trialEfficacy outcome
In children with Prader-Willi syndrome aged 3-4 years, circulating IGF-1 was significantly higher in oxytocin-exposed than in non-exposed children.
In plain wordsIn children with Prader-Willi syndrome, children given oxytocin as infants had higher IGF-1 levels than those who were not.
Peptide
Oxytocin
Outcome
IncreaseCirculating IGF-1
Study
n = 34
Comparator
Non-exposed children with Prader-Willi syndrome
Analysis
Secondary
Outcome group
Growth hormone and IGF-1 · Endocrine and hormones
Studied dose
Dose not stated · intranasal · 7 daysAs given in this study (human).
Source sentences
Summary
Circulating IGF-1 and HDL cholesterol were significantly higher in the OT-exposed group (p < 0.05).
2 context sentences(design, population, supporting numbers)
Summary · context
We previously showed that infants with PWS who had received an early short course (7 days) of intranasal OT treatment improved their oral and social skills.
Summary · context
We performed a comparative clinical trial including the 17 children who had received OT as infants in our previous study and compared them to 17 PWS non-exposed children at 3-4 years old.
Show in the study text Report a problemFND-4002551404
04HumanNon-randomized trialEfficacy outcome
In children with Prader-Willi syndrome aged 3-4 years, HDL cholesterol was significantly higher in oxytocin-exposed than in non-exposed children.
In plain wordsIn children with Prader-Willi syndrome, children given oxytocin as infants had higher HDL cholesterol than those who were not.
Peptide
Oxytocin
Outcome
IncreaseHDL cholesterol
Study
n = 34
Comparator
Non-exposed children with Prader-Willi syndrome
Analysis
Secondary
Outcome group
Lipids · Metabolic and weight
Studied dose
Dose not stated · intranasal · 7 daysAs given in this study (human).
Source sentences
Summary
Circulating IGF-1 and HDL cholesterol were significantly higher in the OT-exposed group (p < 0.05).
2 context sentences(design, population, supporting numbers)
Summary · context
We previously showed that infants with PWS who had received an early short course (7 days) of intranasal OT treatment improved their oral and social skills.
Summary · context
We performed a comparative clinical trial including the 17 children who had received OT as infants in our previous study and compared them to 17 PWS non-exposed children at 3-4 years old.
Show in the study text Report a problemFND-4002551405
05HumanNon-randomized trialEfficacy outcome
In children with Prader-Willi syndrome aged 3-4 years, acylated ghrelin was normal in oxytocin-exposed children and tended to be lower than in non-exposed children (p = 0.06).
In plain wordsIn children with Prader-Willi syndrome, children given oxytocin as infants had normal ghrelin levels, with a possible, not clearly significant, difference from others.
Peptide
Oxytocin
Outcome
No significant changeAcylated ghrelin
Study
n = 34
Comparator
Non-exposed children with Prader-Willi syndrome
Analysis
Secondary
Outcome group
Appetite and food intake · Endocrine and hormones
Studied dose
Dose not stated · intranasal · 7 daysAs given in this study (human).
Measurements
p value
0.06
acylated ghrelin
Source sentences
Summary
OT-exposed children had normal acylated ghrelin levels, which were lower than those observed in non-exposed children (p = 0.06)
2 context sentences(design, population, supporting numbers)
Summary · context
We previously showed that infants with PWS who had received an early short course (7 days) of intranasal OT treatment improved their oral and social skills.
Summary · context
We performed a comparative clinical trial including the 17 children who had received OT as infants in our previous study and compared them to 17 PWS non-exposed children at 3-4 years old.
Show in the study text Report a problemFND-4002551406
06HumanNon-randomized trialEfficacy outcome
In children with Prader-Willi syndrome aged 3-4 years, oxytocin-exposed children showed higher connectivity of the orbitofrontal cortex than non-exposed children.
In plain wordsIn children with Prader-Willi syndrome, children given oxytocin as infants showed higher brain connectivity in one frontal region than those who were not.
Peptide
Oxytocin
Outcome
IncreaseOrbitofrontal cortex connectivity on functional MRI
Study
n = 34
Comparator
Non-exposed children with Prader-Willi syndrome
Analysis
Secondary
Outcome group
Cognition and memory · Nervous system and cognition
Studied dose
Dose not stated · intranasal · 7 daysAs given in this study (human).
Source sentences
Summary
and they displayed higher connectivity of the orbitofrontal cortex brain region.
2 context sentences(design, population, supporting numbers)
Summary · context
We previously showed that infants with PWS who had received an early short course (7 days) of intranasal OT treatment improved their oral and social skills.
Summary · context
We performed a comparative clinical trial including the 17 children who had received OT as infants in our previous study and compared them to 17 PWS non-exposed children at 3-4 years old.
Show in the study text Report a problemFND-4002551407
07HumanNon-randomized trialSafety outcome
In children with Prader-Willi syndrome, early intranasal oxytocin in infancy was documented as safe up to 3-4 years of age.
In plain wordsIn children with Prader-Willi syndrome, a short oxytocin course in infancy was reported as safe up to age 3-4 years.
Peptide
Oxytocin
Outcome
Not applicableLong-term safety of early oxytocin treatment
Study
n = 34
Comparator
Non-exposed children with Prader-Willi syndrome
Outcome group
Adverse events · Whole body
Studied dose
Dose not stated · intranasal · 7 daysAs given in this study (human).
Source sentences
Summary
We documented the long-term safety of early OT treatment.
3 context sentences(design, population, supporting numbers)
Summary · context
Early OT treatment in infants with PWS is safe up to 3-4 years of age.
Summary · context
We previously showed that infants with PWS who had received an early short course (7 days) of intranasal OT treatment improved their oral and social skills.
Summary · context
We performed a comparative clinical trial including the 17 children who had received OT as infants in our previous study and compared them to 17 PWS non-exposed children at 3-4 years old.
Show in the study text Report a problemFND-4002551401

AI wrote and checked these results. AI can make mistakes, so check important results against the source.

Registered trials1

Trials this study reports on, or that list this study as a reference. Registry data from ClinicalTrials.gov.

1 trial · 1 completed · source ClinicalTrials.gov (opens in a new tab)

CompletedNot applicableNCT03081832

Follow-up of Prader Willi Syndrome Infants Treated by Oxytocin and Comparison With Not-treated Infants.

University Hospital, Toulousen = 34Jan 2017 to Dec 2018

Prader-Willi Syndrome

Trial page ClinicalTrials.govUpdated 30 Jan 2019

Version history

PepBank keeps every version of the data on this study. Status changes from Retraction Watch, Europe PMC and Crossref are listed with their source.

Version 2Current
11 Oct 2026, 00:50 UTC
Study results updated
Version 1
9 Oct 2026, 02:48 UTC
First added · source update from Europe PMC

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