Oxytocin Intranasal Administrations in Children With Prader-Willi Syndrome Aged From 3 to 12 Years

Not Applicable

Completed

• Conditions: Prader-Willi Syndrome
• Interventions: Drug: Placebo; Drug: Oxytocin

• Registration Number: NCT03114371
• Lead Sponsor: University Hospital, Toulouse

Brief Summary

Positive results in preclinical and clinical studies in adults and infants with Prader-Willi syndrome lead investigators to set up a new study in children with Prader-Willi syndrome. The objective of this study is to document effects of oxytocin intranasal administrations on behavioural troubles in children with Prader-Willi syndrome aged from 3 to 12 years.

Detailed Description

Two groups of patients will be constituted according to their age; "Group 1" children aged from 3 to 6 years (n = 20) and "Group 2" children aged from 7 to 12 years (n = 20). Within each group, subjects will be randomized to receive either oxytocin or placebo for 12 consecutive weeks. A second period of 12 consecutive oxytocin treatment weeks will then be performed for all patients, followed by a 4-week observation period to document effects after discontinuation of treatment.

Study Timeline

• Study Start: 2016-11-28
• Study First Submitted: 2017-03-10
• Study First Submitted QC: 2017-04-13
• Study First Posted: 2017-04-14
• Primary Completion: 2019-01
• Study Completion: 2019-01-11
• Status Verified: 2020-11
• Last Update Submitted: 2020-11-10
• Last Update Posted: 2020-11-12

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 40

Inclusion Criteria

• patient with a complete genetic diagnosis of Prader-Willi syndrome
• patient treated by growth hormone for at least 1 year
• patient naïve for oxytocin for at least 5 years

Exclusion Criteria

• patient who do not accept intranasal administrations (major behavioural trouble)
• patient with hepatic insufficiency : serum transaminases (SGOT, SGPT) higher than 3 times normal values for age
• patient with renal insufficiency : serum creatinine higher than 3 times normal values for age
• patient with an antecedent of abnormal electrocardiogram
• patient with arterial hypertension or hypotension
• patient with type 1 or 2 diabetes

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
Placebo	Oxytocin	Daily intranasal administrations of placebo for 12 weeks, followed by an open-label period of 12 weeks of oxytocin. Oxytocin dose will be International Unit for patients aged from 3 to 6 years and 16 International Unit for patients aged from 7 to 12 years.
Placebo	Placebo	Daily intranasal administrations of placebo for 12 weeks, followed by an open-label period of 12 weeks of oxytocin. Oxytocin dose will be International Unit for patients aged from 3 to 6 years and 16 International Unit for patients aged from 7 to 12 years.
Oxytocin	Oxytocin	Daily intranasal administrations of oxytocin for 12 weeks, followed by an open-label period of 12 weeks of oxytocin. Oxytocin dose will be 8 International Unit for patients aged from 3 to 6 years and 16 International Unit for patients aged from 7 to 12 years.

Primary Outcome Measures

Name	Time	Method
Evolution of behavioural troubles evaluated by the global score of Child Behavior Check List Questionnaire after 12 weeks of oxytocin/placebo treatment.	Week 12	It's variation between inclusion and 12 weeks of total score total problems from the Child Behavior Check List Questionnaire.

Secondary Outcome Measures

Name	Time	Method
Evaluation of hyperphagia after 12 weeks of oxytocin/placebo treatment.	Week 12	It is the variation of each of the three sub-scores obtained from the Dykens hyperphagia questionnaire between day 0 and week 12.
Evaluation of social skills after 12 weeks of oxytocin/placebo treatment.	Week 12	It is variation between day 0 and week 12 of the total score obtained from the social skills assessment questionnaire for children aged from 3 to 6 years and the Social Responsiveness Scale questionnaire for children aged from 7 to 12 years.
Evaluation of auto- and hetero-aggressive after 12 weeks of oxytocin/placebo treatment.	Week 12	It is variation between day 0 and week 12 of the total score obtained from the self-aggression assessment questionnaire.
Evaluation of psychopathology after 12 weeks of oxytocin/placebo treatment.	Week 12	It is the variation of the following sub-scores obtained from the Child Behaviour Check List questionnaire (only the following 3 subscales) between day 0 and week 12.
Evaluation of global clinical status after 12 weeks of oxytocin/placebo treatment.	Weeks 12	It is improvement of the patient's overall clinical condition after 12 weeks of treatment with oxytocin/placebo. It's assessed by the Clinical Global Impression Scale's score.; This scale of improvement of Clinical Global Impression is a 7-point ordinal qualitative scale of "very greatly improved" rated + 3 to "very strongly aggravated" rated -3. The result is expressed in total score which varies from -3 to +3.
Evaluation of acyl and desacyl ghrelin plasma levels after 12 weeks of oxytocin/placebo treatment.	Week 12	It is evolution of circulating levels of acylated and deacylated ghrelin will be the variations of these rates and the variation of the relationships between day 0 and week 12.
Evaluation of attentional abilities after 12 weeks of oxytocin/placebo treatment, for patients aged from 7 to 12 years at inclusion.	Week 12	It is evolution of attentional abilities is evaluated by a computerized test, the Attention Network Test.
Evaluation of metabolic brain resting state after 12 weeks of oxytocin/placebo treatment, for patients aged from 7 to 12 years at inclusion.	Week 12	It is evolution of metabolic brain resting state is evaluated by a magnetic resonance imaging to study the cerebral metabolism between day 0 and week 12.

Trial Locations

Locations (1)

Centre de référence du syndrome de Prader-Willi Hôpital des Enfants; 🇫🇷 Toulouse, France