Clinical trial NCT04283578: Oxytocin Treatment in Neonates and Infants With Prader-Willi Syndrome
Official title: 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
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE3
Conditions: Prader-Willi Syndrome
Lead sponsor: University Hospital, Toulouse
Sponsor class: OTHER
Collaborators: name: International Clinical Trials Association
name: Epidemiological and Clinical Research Information Network
First posted: 2020-02-25
Enrollment: 52
Sex: ALL
Minimum age (years): 1
Maximum age (years): 92
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief summary: 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.


Eligibility criteria: Inclusion Criteria:

1. Male or female neonate or infant, with PWS genetically confirmed.
2. 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).
3. Signed informed consent obtained from the parents/holders of parental authority.
4. 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.

Primary outcomes:
measure: Neonatal Oral-Motor Assessment Scale (NOMAS) scale
timeFrame: 4 weeks
Secondary outcomes:
measure: Ghrelin dosage
timeFrame: Day 0, Week 1 and week 4
measure: Oxytocin dosage
timeFrame: Day 0, week 4
measure: Proficiency score
timeFrame: Day 0, Week and week 4
Other outcomes:
Locations:
city: Bruxelles
country: Belgium
facility: Cliniques Saint Luc
city: Bron
country: France
facility: Hôpital Femme Mère Enfant
city: Lille
country: France
facility: Hôpital Jeanne de Flandre
city: Marseille
country: France
facility: Hôpital de la Timone Enfant
city: Paris
country: France
facility: Groupe Hospitalier Necker - Enfants Malades
city: Toulouse
country: France
facility: Centre de réfrence Prader-Willi, Hospital of infants
city: Essen
country: Germany
facility: Klinik für Kinderheilkunde II
Study officials:
affiliation: University Hospital, Toulouse
name: Maithé TAUBER, MD
role: PRINCIPAL_INVESTIGATOR
References:
citation: Borie AM, Dromard Y, Guillon G, Olma A, Manning M, Muscatelli F, Desarmenien MG, Jeanneteau F. Correction of vasopressin deficit in the lateral septum ameliorates social deficits of mouse autism model. J Clin Invest. 2021 Jan 19;131(2):e144450. doi: 10.1172/JCI144450.