Oxytocin Treatment in Neonates and Infants With Prader-Willi Syndrome

Study Overview
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Brief Summary
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Prospective, randomized, placebo-controlled, double-blind part of the phase III trial to assess the safety and efficacy of 4 weeks oxytocin (OT) administration on oral and social skills in neonates/infants with Prader-Willi Syndrome (PWS) aged less than 3 months at inclusion. Phase III clinical trial.

Official Title
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Oxytocin Treatment in Neonates and Infants Aged From 0 to 3 Months With Prader-Willi Syndrome : a Study of Safety and Efficacy on Oral and Social Skills and Feeding Behavior of Intranasal Administration of Oxytocin vs Placebo

Conditions
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Prader-Willi Syndrome

Intervention / Treatment
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* Drug: OT
* Drug: Placebo comparator


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: 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. Exclusion Criteria: 1. Neonate or infant currently admitted to the emergency care unit for ongoing life-threatening comorbidities like severe respiratory, cardiovascular or neurological abnormalities. 2. Neonate or infant with prolongation of the QT interval. 3. Neonate or infant without medical insurance. 4. Neonate or infant with hypersensitivity to oxytocin or excipients of the product. 5. Neonate or infant with concomitant treatment prolonging QT interval 6. Neonate or infant with family history of genetic pathology causing QT interval prolongation. 7. Neonate or infant with hypokalemia (clinically relevant at the discretion of the doctor). 8. Neonate or infant participating simultaneously in another interventional study. 9. Neonates or infants whose parents' situations may jeopardize the interpretation of the results. 10. Neonates or infants whose parents' refuse video recording, required to respond to the primary objective of the study.

Ages Eligible for Study
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Minimum Age: 1 Day
Maximum Age: 92 Days

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Oxytocin<br>intranasal administration of OT | Drug: OT<br>* One group will receive OT for 4 weeks, then placebo or OT for 8 weeks<br>* Other names: oxytocin;Drug: Placebo comparator<br>* One group will receive Placebo for 4 weeks, then Placebo or OT for 8 weeks.<br>|
| Placebo Comparator: Placebo<br>intranasal administration of placebo | Drug: OT<br>* One group will receive OT for 4 weeks, then placebo or OT for 8 weeks<br>* Other names: oxytocin;Drug: Placebo comparator<br>* One group will receive Placebo for 4 weeks, then Placebo or OT for 8 weeks.<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Neonatal Oral-Motor Assessment Scale (NOMAS) scale | the score goes from 8 to 28, the higher the score meaning a worse outcome | 4 weeks | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Ghrelin dosage | Concentration of ghrelin (unacylated/UAG and acylated/AG) | Day 0, Week 1 and week 4 | 
| Oxytocin dosage | Plasma Oxytocin concentration | Day 0, week 4 | 
| Proficiency score | The volume of milk taken in the first five minutes of feeding | Day 0, Week and week 4 |