OXYTOCIN TREATMENT IN NEONATES AND INFANTS AGED FROM 0 TO 3 MONTHS WITH PRADER-WILLI SYNDROME

Phase 1

Conditions: Prader-Willi Syndrom
Therapeutic area: Diseases [C] - Hormonal diseases [C19]

Registration Number: EUCTR2019-002385-12-BE

Lead Sponsor: niversity Hospital of Toulouse

Brief Summary: Not available

Detailed Description: Not available

Recruitment & Eligibility

Status: ot Recruiting

Sex: All

Target Recruitment: 48

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.

Are the trial subjects under 18? yes
Number of subjects for this age range: 48
F.1.2 Adults (18-64 years) no
F.1.2.1 Number of subjects for this age range
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

Exclusion Criteria

- Neonate or infant admitted to the emergency care unit for ongoing life-threatening comorbidities like severe respiratory, cardiovascular or neurological abnormalities
- Neonate or infant with prolongation of the QT interval
- Neonate or infant with hypokaliema
- Neonate or infant without medical insurance
- Neonates or infants whose parents’ situations may jeopardize the interpretation of the results
- Neonate or infant with known hypersensitivity to the excipients of the product
- Neonate or infant participating simultaneously in another interventional study.

Study & Design

Study Type: Interventional clinical trial of medicinal product

Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Main Objective: The primary objective is to demonstrate the superiority versus placebo of a 4 weeks intranasal OT administration on oral skills assessed by the Neonatal Oral-Motor Assessment Scale (NOMAS) in infants with Prader Willi Syndrom (PWS) aged less than or equal to 3 months at inclusion;Secondary Objective: The secondary objectives are to document the effects of 1 week and 4 weeks intranasal OT administration versus Placebo on:<br>- Sucking/swallowing troubles <br>- Social Withdrawal score ADBB<br>- CIB subscores <br>- Food intake (medical assesment and parental assesment)<br>- Circulating forms of ghrelin<br>- OT levels<br><br>To document the safety of repeated OT administration for 4 weeks or 8 weeks with total follow-up over 26 weeks.<br><br><br><br>;Primary end point(s): The primary endpoint is the proportion of neonates/infants who achieve a quasi-normal score on sucking/swallowing after 4 weeks (V4) OT/ Placebo intranasal treatment.<br>;Timepoint(s) of evaluation of this end point: 4 weeks

Secondary Outcome Measures

Name	Time	Method
Secondary end point(s): - Change from baseline global score of swallowing at 4 weeks.<br>- Proportion of infants with abnormal score at baseline who reached a normal score after 4 weeks treatment for all of the 3 items, namely i) velopharyngeal continence ii) pharyngeal propulsion iii) swallowing troubles at 4 weeks.<br>- The change from baseline of the Alarm Distress Baby Scale (ADBB) score at 4 weeks.<br>- The change from baseline of the Coding Interacting Behaviour (CIB) subscores at 4 weeks.<br>- The change from baseline of volume of milk taken in the first five minutes of feeding at 1 week (V3) and at 4 weeks.<br>- The change from baseline of ghrelin (unacylated/UAG and acylated/AG) concentration at 1 week (V3) and at 4 weeks.<br>- The change from baseline of blood OT concentration at 4 weeks.<br>- Biological safety parameters vital signs, ECG and emergent adverse events at all study time points in all groups of patients.<br><br>;Timepoint(s) of evaluation of this end point: 1 week and 4 week

Related Trials