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Study · Randomized controlled trial · n = 64 · 2026

5 results

Oxytocin in infants with Prader-Willi syndrome to improve dysphagia and disease trajectory.

Tauber M, Diene G, Fichaux-Bourin P, Pinto G, Gueorguieva I, Nicolino M, Reynaud R, Bernoux D, Beauloye V, Abrahimians EM, Kiewert C, Payoux P and 8 more

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Cabal S, Molinas C, Glattard M, Viaux-Savelon S, Guedeney A, Cohen D, Arnaud C, Valette M

Orphanet journal of rare diseases4 Feb 2026

PMID41639888 (opens in a new tab)PMCIDPMC12964787 (opens in a new tab)DOI10.1186/s13023-026-04214-8 (opens in a new tab)PepBankPUB-41639888
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Summary

OBJECTIVE: Prader-Willi syndrome (PWS) is a genetic neurodevelopmental disorder with a characteristic trajectory. Infants display hypotonia, poor social and feeding skills, and high risk of choking, which have been shown to improve after oxytocin (OT) treatment. Our aim is to demonstrate the efficacy of intranasal OT treatment administered in the postnatal critical period on infant feeding skills and document its long-term effects. METHODS: We enrolled 52 infants with PWS (median age 2.2 months) in a European double-blind randomized placebo-controlled study. Infants were randomly assigned in a 1:1 ratio to either 4 IU/day of OT or placebo for a 4-week evaluation of efficacy. A second randomization in each group was performed. Infants in the placebo group were randomized into 4 weeks of OT followed by 4 weeks of placebo or 8 consecutive weeks of OT. Those in the OT group were randomized into additional 4 weeks of OT followed by 4 weeks of placebo or 8 consecutive weeks of placebo. Infants were followed-up to 26 weeks from baseline. Feeding skills were evaluated using Neonatal Oral-Motor Scale (NOMAS) as primary endpoint and videofluoroscopy of swallowing study (VFSS) as key secondary endpoint. Subsequently, 40 infants included in France (OT-exposed cohort) participated in a new study to document long term safety of OT treatment and to compare them with an unexposed cohort (n = 24) at about 3 years of age. Prevalence and severity of comorbidities of the disorder were compared between the two cohorts. RESULTS: OT was well tolerated. At 4 weeks, NOMAS normalization rates were similar between OT and placebo, but OT yielded a higher VFSS responder rate (53.3% vs 16.7%, p = 0.05) and a greater reduction in VFSS total score (LS mean difference −1.55; 95% CI −2.9 to −0.2; p = 0.03). At 3 years, the OT-exposed cohort demonstrated consistently better motor, adaptive and behavioral outcomes than unexposed controls. CONCLUSIONS: The primary endpoint did not show significant difference between OT and placebo groups. However, using VFSS we showed for the first time a positive effect of 4 weeks intranasal OT treatment on swallowing. We also document long-term effect on disease trajectory, with less severe comorbidities.
CC BYText from Europe PMC. Highlighted sentences are the source of results.

Results from this study5

5 results from studies that gave the peptide. 5 results from people.

  1. 01HumanRandomized controlled trialEfficacy outcome

    In infants with Prader-Willi syndrome, NOMAS normalization rates at 4 weeks were similar between oxytocin and placebo.

    In plain wordsIn infants with Prader-Willi syndrome, feeding skill normalization on the NOMAS scale was similar with oxytocin and placebo.

    Peptide
    Oxytocin
    Outcome
    No significant changeNOMAS normalization rate at 4 weeks
    Study

    n = 52

    Comparator
    Placebo
    Analysis
    Primary
    Outcome group
    Exercise and physical function · Nervous system and cognition
    Studied dose
    4 IU/day · intranasal · 4 weeksAs given in this study (human).

    Source sentences

    Summary
    At 4 weeks, NOMAS normalization rates were similar between OT and placebo
    3 context sentences
    Summary · context
    Feeding skills were evaluated using Neonatal Oral-Motor Scale (NOMAS) as primary endpoint and videofluoroscopy of swallowing study (VFSS) as key secondary endpoint.
    Summary · context
    We enrolled 52 infants with PWS (median age 2.2 months) in a European double-blind randomized placebo-controlled study.
    Summary · context
    Infants were randomly assigned in a 1:1 ratio to either 4 IU/day of OT or placebo for a 4-week evaluation of efficacy.
  2. 02HumanRandomized controlled trialEfficacy outcome

    In infants with Prader-Willi syndrome, oxytocin gave a higher swallowing study (VFSS) responder rate than placebo at 4 weeks.

    In plain wordsIn infants with Prader-Willi syndrome, more babies responded on the swallowing study with oxytocin than with placebo.

    Peptide
    Oxytocin
    Outcome
    IncreaseVFSS responder rate at 4 weeks
    Study

    n: not reported in source

    Comparator
    Placebo
    Analysis
    Secondary
    Outcome group
    Exercise and physical function · Nervous system and cognition
    Studied dose
    4 IU/day · intranasal · 4 weeksAs given in this study (human).

    Measurements

    VFSS responder rate
    53.3%
    oxytocin
    VFSS responder rate
    16.7%
    placebo

    Source sentences

    Summary
    OT yielded a higher VFSS responder rate (53.3% vs 16.7%, p = 0.05)
    2 context sentences
    Summary · context
    We enrolled 52 infants with PWS (median age 2.2 months) in a European double-blind randomized placebo-controlled study.
    Summary · context
    Infants were randomly assigned in a 1:1 ratio to either 4 IU/day of OT or placebo for a 4-week evaluation of efficacy.
  3. 03HumanRandomized controlled trialEfficacy outcome

    In infants with Prader-Willi syndrome, oxytocin gave a greater reduction in the VFSS total score than placebo at 4 weeks.

    In plain wordsIn infants with Prader-Willi syndrome, oxytocin lowered the swallowing study score more than placebo, which indicates better swallowing.

    Peptide
    Oxytocin
    Outcome
    DecreaseVFSS total score change at 4 weeks
    Study

    n: not reported in source

    Comparator
    Placebo
    Analysis
    Secondary
    Outcome group
    Exercise and physical function · Nervous system and cognition
    Studied dose
    4 IU/day · intranasal · 4 weeksAs given in this study (human).

    Measurements

    LS mean difference
    −1.55
    oxytocin vs placebo

    Source sentences

    Summary
    a greater reduction in VFSS total score (LS mean difference −1.55; 95% CI −2.9 to −0.2; p = 0.03).
    2 context sentences
    Summary · context
    We enrolled 52 infants with PWS (median age 2.2 months) in a European double-blind randomized placebo-controlled study.
    Summary · context
    Infants were randomly assigned in a 1:1 ratio to either 4 IU/day of OT or placebo for a 4-week evaluation of efficacy.
  4. 04HumanRandomized controlled trialSafety outcome

    In infants with Prader-Willi syndrome, intranasal oxytocin was reported as well tolerated.

    In plain wordsIn infants with Prader-Willi syndrome, oxytocin was reported as well tolerated.

    Peptide
    Oxytocin
    Outcome
    No significant changeTolerability of oxytocin
    Study

    n = 52

    Outcome group
    Adverse events · Whole body
    Studied dose
    4 IU/day · intranasal · 4 weeksAs given in this study (human).

    Source sentences

    Summary
    OT was well tolerated.
    2 context sentences
    Summary · context
    We enrolled 52 infants with PWS (median age 2.2 months) in a European double-blind randomized placebo-controlled study.
    Summary · context
    Infants were randomly assigned in a 1:1 ratio to either 4 IU/day of OT or placebo for a 4-week evaluation of efficacy.
  5. 05HumanObservational studyEfficacy outcome

    At about 3 years, children with Prader-Willi syndrome who had received oxytocin as infants had consistently better motor, adaptive and behavioral outcomes than unexposed controls.

    In plain wordsAt about 3 years, children who got oxytocin as infants were linked to better motor, adaptive and behavioral outcomes than children who did not.

    Peptide
    Oxytocin
    Outcome
    IncreaseMotor, adaptive and behavioral outcomes at about 3 years
    Study

    n = 64

    Comparator
    Unexposed cohort
    Outcome group
    Exercise and physical function · Nervous system and cognition
    Studied dose
    Dose not stated

    Source sentences

    Summary
    At 3 years, the OT-exposed cohort demonstrated consistently better motor, adaptive and behavioral outcomes than unexposed controls.
    1 context sentence
    Summary · context
    Subsequently, 40 infants included in France (OT-exposed cohort) participated in a new study to document long term safety of OT treatment and to compare them with an unexposed cohort (n = 24) at about 3 years of age.

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

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.

  1. Version 2Current

    11 Oct 2026, 00:50 UTC

    Study results updated

  2. Version 1

    9 Oct 2026, 02:47 UTC

    First added · source update from Europe PMC

Subjects

Terms as the source lists them.

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