Oxytocin Treatment in Neonates and Infants With Prader-Willi Syndrome

Phase 3

Completed

• Conditions: Prader-Willi Syndrome
• Interventions: Drug: OT; Drug: Placebo comparator

• Registration Number: NCT04283578
• Lead Sponsor: University Hospital, Toulouse

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.

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2020-02-19
• Study First Submitted QC: 2020-02-21
• Study First Posted: 2020-02-25
• Study Start: 2020-03-10
• Primary Completion: 2021-10-16
• Study Completion: 2022-03-14
• Status Verified: 2023-05
• Last Update Submitted: 2023-05-31
• Last Update Posted: 2023-06-01

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 52

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.

  3. Neonate or infant with concomitant treatment prolonging QT interval 6. Neonate or infant with family history of genetic pathology causing QT interval prolongation.

  4. Neonate or infant with hypokalemia (clinically relevant at the discretion of the doctor).

  5. Neonate or infant participating simultaneously in another interventional study.

  6. Neonates or infants whose parents' situations may jeopardize the interpretation of the results.

  7. Neonates or infants whose parents' refuse video recording, required to respond to the primary objective of the study.

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
Oxytocin	OT	intranasal administration of OT
Oxytocin	Placebo comparator	intranasal administration of OT
Placebo	OT	intranasal administration of placebo
Placebo	Placebo comparator	intranasal administration of placebo

Primary Outcome Measures

Name	Time	Method
Neonatal Oral-Motor Assessment Scale (NOMAS) scale	4 weeks	the score goes from 8 to 28, the higher the score meaning a worse outcome

Secondary Outcome Measures

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

Trial Locations

Locations (7)

Cliniques Saint Luc; 🇧🇪 Bruxelles, Belgium

Klinik für Kinderheilkunde II; 🇩🇪 Essen, Germany

Groupe Hospitalier Necker - Enfants Malades; 🇫🇷 Paris, France

Centre de réfrence Prader-Willi, Hospital of infants; 🇫🇷 Toulouse, France

Hôpital de la Timone Enfant; 🇫🇷 Marseille, France

Hôpital Jeanne de Flandre; 🇫🇷 Lille, France

Hôpital Femme Mère Enfant; 🇫🇷 Bron, France