Oxytocin Treatment in Neonates and Infants With Prader-Willi Syndrome

Prader-Willi Syndrome Clinical Trial

— OTBB3  

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT04283578
• Other study ID #: RC31/15/7825
• Status: Completed
• Phase: Phase 3
• Start date: March 10, 2020
• Est. completion date: March 14, 2022

Study information

• Verified date: May 2023
• Source: University Hospital, Toulouse
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 52
• Est. completion date: March 14, 2022
• Est. primary completion date: October 16, 2021
• Accepts healthy volunteers: No
• Gender: All
• Age group: 1 Day to 92 Days

Eligibility

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.

Related Conditions & MeSH terms

• Prader-Willi Syndrome
• Syndrome

Intervention

Drug:
• OT
One group will receive OT for 4 weeks, then placebo or OT for 8 weeks
• Placebo comparator
One group will receive Placebo for 4 weeks, then Placebo or OT for 8 weeks.

Locations

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

Sponsors (3)

Lead Sponsor	Collaborator
University Hospital, Toulouse	Epidemiological and Clinical Research Information Network, International Clinical Trials Association

Countries where clinical trial is conducted

Belgium,  France,  Germany, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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	Ghrelin dosage	Concentration of ghrelin (unacylated/UAG and acylated/AG)	Day 0, Week 1 and week 4
Secondary	Oxytocin dosage	Plasma Oxytocin concentration	Day 0, week 4
Secondary	Proficiency score	The volume of milk taken in the first five minutes of feeding	Day 0, Week and week 4