Oxytocin Treatment in Neonates and Infants With Prader-Willi Syndrome (OTBB3)

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

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.

Study Overview

• Status: Completed
• Conditions: Prader-Willi Syndrome
• Intervention / Treatment: Drug: OT; Drug: Placebo comparator

• Study Type: Interventional
• Enrollment (Actual): 52
• Phase: Phase 3

Contacts and Locations

Study Locations

• Belgium
  • Bruxelles, Belgium
    • Cliniques Saint Luc
• France
  • Bron, France
    • Hopital Femme Mère Enfant
  • Lille, France
    • Hopital Jeanne de Flandre
  • Marseille, France
    • Hôpital de la Timone Enfant
  • Paris, France
    • Groupe Hospitalier Necker - Enfants Malades
  • Toulouse, France, 31059
    • Centre de réfrence Prader-Willi, Hospital of infants
• Germany
  • Essen, Germany
    • Klinik für Kinderheilkunde II

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 1 day to 3 months (Child)
• Accepts Healthy Volunteers: No

Description

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.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: Quadruple

Number of Arms

2

Arms and Interventions

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

What is the study measuring?

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

Collaborators and Investigators

• Sponsor: University Hospital, Toulouse
• Collaborators: Epidemiological and Clinical Research Information Network; International Clinical Trials Association
• Investigators: Principal Investigator: Maithé TAUBER, MD, University Hospital, Toulouse

Publications and helpful links

General Publications

• 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.

Study record dates

Study Major Dates

• Study Start (Actual): March 10, 2020
• Primary Completion (Actual): October 16, 2021
• Study Completion (Actual): March 14, 2022

Study Registration Dates

• First Submitted: February 19, 2020
• First Submitted That Met QC Criteria: February 21, 2020
• First Posted (Actual): February 25, 2020

Study Record Updates

• Last Update Posted (Actual): June 1, 2023
• Last Update Submitted That Met QC Criteria: May 31, 2023
• Last Verified: May 1, 2023

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Pathologic Processes; Nervous System Diseases; Neurologic Manifestations; Neurobehavioral Manifestations; Disease; Congenital Abnormalities; Overnutrition; Nutrition Disorders; Overweight; Genetic Diseases, Inborn; Intellectual Disability; Abnormalities, Multiple; Chromosome Disorders; Obesity; Syndrome; Prader-Willi Syndrome; Physiological Effects of Drugs; Reproductive Control Agents; Oxytocics; Oxytocin
• Other Study ID Numbers: RC31/15/7825

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No