Prader-Willi Syndrom
Conditions
Interventions
Product Name: Oxytocin
Pharmaceutical Form: Nasal spray, solution
INN or Proposed INN: OXYTOCIN
CAS Number: 50-56-6
Other descriptive name: OXYTOCIN
Concentration unit: IU/ml international unit(s)/mil
Sponsors
University Hospital of Toulouse
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: - Male or female neonate or infant, with PWS genetically confirmed - Age =65 years) no F.1.3.1 Number of subjects for this age range
Exclusion criteria
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 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.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| 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: - Sucking/swallowing troubles assessed by videofluoroscopy - Social Withdrawal assessed by Alarm Distress Baby Scale ADBB - CIB subscores (child state, child social engagement and dyadic reciprocity) - Food intake: proficiency score (PRO) - Circulating forms of ghrelin - OT levels To document the safety of repeated OT administration for 4 weeks or 8 weeks with total follow-up over 26 weeks. The exploratory objectives are to: -Evaluate in more detail the effects of intranasal OT versus placebo on the parameters: CIB scale, duration of feeding by Naso-gastric probe, brain connectivity at rest (MRI)... Compare the effects on all the criteria of an administration of 4 versus 8 weeks, delayed versus non-delayed and maintaining of the effect ;Primary end point(s): The primary endpoint is the proportion of neonates/infants who achieve a quasi-normal score (=10) (defined as responders) on sucking/swallowing as assessed by the Neonatal Oral-Motor Assessment Scale (NOMAS) centrally scored on videos, after 4 weeks (V4) OT/ Placebo intranasal treatment. ;Timepoint(s) of evaluation of this end point: 4 weeks | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): - Change from baseline global score of videofluoroscopy of swallowing at 4 weeks. - Proportion of infants with abnormal score at baseline (on at least one of the three items) 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. - The change from baseline of the Alarm Distress Baby Scale (ADBB) score at 4 weeks. - The change from baseline of the Coding Interacting Behaviour (CIB) subscores (child state, social engagement and mother-infant interactions) at 4 weeks. - The change from baseline of proficiency score (PRO) which is the volume of milk taken in the first five minutes of feeding at 1 week (V3) and at 4 weeks. - The change from baseline of ghrelin (unacylated/UAG and acylated/AG) concentration at 1 week (V3) and at 4 weeks. - The change from baseline of blood OT concentration at 4 weeks. - Biological safety parameters (natremia, plasmatic osmolality, capillary blood glucose, total bilirubin level, urinary density), vital signs, ECG and emergent adverse events at all study time points in all groups of patients. The exploratory endpoints are: -Comparisons of treatment with OT vs. placebo at 4 weeks (V4) for the below parameters: -CIB subscores: Parental sensitivity, Parental intrusiveness, Child withdrawal, Dyadic joint negative state -Proportion of infants with NGT feeding at inclusion, who have stopped it at 4 weeks (V4) in both groups and duration of the NGT. -Centrally analyzed Bold functional MRI (using the Independent component analysis (ICA)-based method (Group ICA fMRI Toolbox)) at 4 weeks (V4). -The change from baseline of infants’ development of body weight (Kg and z score), growth and head circumference (cm and z scores) at 4 weeks (V4). -Nutritional phase: % of patients in phase 1b at 4 weeks (V4). -The change from baseline of the Parent-Infant Global Assessment Scale (PIRGAS) at 4 weeks (V4) | — |
Countries
Belgium, France, Germany, Italy, Netherlands
Contacts
Public ContactNadège ALGANS
University Hospital of Toulouse
Outcome results
None listed