Prader-Willi Syndrome
Conditions
Interventions
Product Name: OXYTOCIN
Product Code: H01BB02
Pharmaceutical Form: Nasal spray, solution
Pharmaceutical form of the placebo: Nasal spray
Route of administration of the placebo: Nasal use
Sponsors
Toulouse University Hospital
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: 1. Male or female patients aged between 2 and 17,5 years. 2. Genetically confirmed diagnosis of PWS. 3. Parents (or legal representative) have signed the informed consent form and are willing to comply with all study procedures. Are the trial subjects under 18? yes Number of subjects for this age range: 36 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
Exclusion criteria: 1. A history of hypersensitivity to the study drug or drugs with similar chemical structures, to excipients of the product, or to latex; 2. Intolerance of intranasal administrations (including when due to a major behavioural problem); 3. Hyponatremia (clinically relevant at the discretion of the investigator); 4. Hypokalaemia (clinically relevant at the discretion of the investigator); 5. Prolongation of the QT interval and/or family history of prolongation of the QT interval; 6. Concomitant treatment prolonging the QT interval; 7. History of abnormal electrocardiogram (ECG) (validated by a cardiologist); 8. Pregnant girls; (for girls aged 14 years of above who do not have contraception and are sexually active, a negative pregnancy test will be required) 9. Patient with clinical signs in the context of contact with SARs-COV2 infected person 10. Patient included in another study protocol on a medicinal product within the last 6 months 11. Administrative problems:
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: To compare the effect of a 12-week intranasal administration of OT versus (vs) placebo on dysphagia related to OPOD in children and adolescents with PWS.;Secondary Objective: To compare the effect of a 12-week intranasal administration of OT vs placebo on: •Swallowing symptoms; •Gastrointestinal disorders; •Feeding events •Behavioural problems such as: oTemper outbursts; oSkin picking; •Patient global improvement. To document the effect of 12-week OT treatment withdrawal on dysphagia related to OPOD and other efficacy parameters. To document the maintenance of efficacy over a 24 week-intranasal administration of OT. To document the long-term efficacy up to 48-weeks of intranasal administration of OT. ;Primary end point(s): The percentage of patients showing normalisation of at least 1 of the following abnormal OPOD subscores after 12 weeks OT / placebo at V2 (groups 1 and 3 vs group 2): •Oral and pharyngeal propulsion; •Initiation and synchronisation of swallowing; •Oesophageal motility. ;Timepoint(s) of evaluation of this end point: visit 2 and (visit 3 for complementary analysis) | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): Secondary Endpoints Secondary Efficacy Endpoints: The following parameters will be assessed for differences between OT and placebo at V2 or change from baseline (groups 1 and 3 vs group 2): • Swallowing symptoms • Digestive questionnaire • Occurrence of feeding events – • Bristol stool scale • Temper outbursts questionnaire • Percentage of patients with improvement in CGI score •Percentage of patients with improvement in parent/caregiver’s global impression score (overall and by level of improvement); • Percentage of patients with improvement in CGI specific for skin picking (overall and by level of improvement). The percentage of normalised OPOD subscores at V2 which became abnormal at V3, in Group 1. All above parameters presented, as percentage of patients at V3 and as change from baseline to V3 vs change from V2 to V3 in Group 1 (withdrawal effect). The percentage of normalised OPOD subscores at V2 which remains normal at V3, in Group 3; all above parameters presented, as percentage of patients and as change from baseline to V3 vs change from V2 to V3 in Group 3. (Maintenance of efficacy) All parameters presented, as percentage of patients and as change from baseline to V4 in all groups (48 weeks for group 3, 36 weeks for group 2 and 24 weeks for group 1) (long-term efficacy). Other Endpoints: Plasma OT at V1, V2 and V3. Acylated ghrelin (AG), unacylated ghrelin (UAG), AG/UAG at V1, V2 and V3. Safety Endpoints: • Adverse events; • Laboratory safety parameters at V1, V2, V3 and V4; • Vital signs (SBP, DBP and HR) at V1, V2, V3 and V4; • ECG at V1, V2, and V3. ;Timepoint(s) of evaluation of this end point: Visit 2, Visit 3 and Visit 4 | — |
Countries
France
Contacts
Public ContactTAUBER
Centre de référence Prader-Willi
Outcome results
None listed