Back to resultsOxytocin Intranasal Administrations in Children With Prader-Willi Syndrome Aged From 3 to 12 Years (Oxyjeune)
Primary Purpose
Prader-Willi Syndrome
Status
Completed
Phase
Not Applicable
Locations
France
Study Type
Interventional
Intervention
Oxytocin
Placebo
Oxytocin
Sponsored by
About this trial
This is an interventional treatment trial for Prader-Willi Syndrome focused on measuring Prader-Willi syndrome, Oxytocin, Behavioural troubles
Eligibility Criteria
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
Sites / Locations
- Centre de référence du syndrome de Prader-Willi Hôpital des Enfants
Arms of the Study
Arm 1
Arm 2
Arm Type
Experimental
Placebo Comparator
Arm Label
Oxytocin
Placebo
Arm Description
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.
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.
Outcomes
Primary Outcome Measures
Evolution of behavioural troubles evaluated by the global score of Child Behavior Check List Questionnaire after 12 weeks of oxytocin/placebo treatment.
It's variation between inclusion and 12 weeks of total score total problems from the Child Behavior Check List Questionnaire.
Secondary Outcome Measures
Evaluation of hyperphagia after 12 weeks of oxytocin/placebo treatment.
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.
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.
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.
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.
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.
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.
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.
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.
Full Information
NCT ID
NCT03114371
First Posted
March 10, 2017
Last Updated
November 10, 2020
Sponsor
University Hospital, Toulouse
1. Study Identification
Unique Protocol Identification Number
NCT03114371
Brief Title
Oxytocin Intranasal Administrations in Children With Prader-Willi Syndrome Aged From 3 to 12 Years
Acronym
Oxyjeune
Official Title
Effects of Intranasal Administrations of Oxytocin on Behavioural Troubles, Hyperphagia and Social Skills in Children With Prader-Willi Syndrome Aged From 3 to 12 Years.
Study Type
Interventional
2. Study Status
Record Verification Date
November 2020
Overall Recruitment Status
Completed
Study Start Date
November 28, 2016 (Actual)
Primary Completion Date
January 2019 (Actual)
Study Completion Date
January 11, 2019 (Actual)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
University Hospital, Toulouse
4. Oversight
Studies a U.S. FDA-regulated Drug Product
No
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
No
5. Study Description
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.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Prader-Willi Syndrome
Keywords
Prader-Willi syndrome, Oxytocin, Behavioural troubles
7. Study Design
Primary Purpose
Treatment
Study Phase
Not Applicable
Interventional Study Model
Parallel Assignment
Model Description
Children will receive after randomization either placebo or oxytocin administration (daily intranasal) for a total duration of 12 consecutive weeks.
Masking
ParticipantInvestigator
Masking Description
The packaging, presentation and labelling of the bottles used during the blind phase will guarantee the blindness to the healthcare team and the patient.
The allocation of the treatment arms to each treatment number will be carried out according to the randomization table established beforehand.
Until the end of the study, neither the investigating physician nor the patient will know the group to which the patient has been randomly assigned.
Allocation
Randomized
Enrollment
40 (Actual)
8. Arms, Groups, and Interventions
Arm Title
Oxytocin
Arm Type
Experimental
Arm Description
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.
Arm Title
Placebo
Arm Type
Placebo Comparator
Arm Description
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.
Intervention Type
Drug
Intervention Name(s)
Oxytocin
Other Intervention Name(s)
Intranasal administration of oxytocin (Week 1 to 12)
Intervention Description
The study drug is oxytocin in intra-nasal administration, Syntocinon®, reconditioned as a placebo-like spray. The dosage administered will be 8 International Unit, ie 1 spray (4 International Unit per spray) in each nostril per day for the first 12 weeks, in 3 and 6 years old patients. The dosage administered will be 16 International Unit or 2 sprays in each nostril per day for the first 12 weeks, in 3 to 6 years old patients.
Intervention Type
Drug
Intervention Name(s)
Placebo
Other Intervention Name(s)
Intranasal administration of placebo (Week 1 to 12)
Intervention Description
Placebo should be used as a spray, similar to that of the oxytocin. The dosage administered will be 1 spray in each nostril per day for the first 12 weeks in 3 to 6 years old patients. The dosage administered will be 2 sprays in each nostril per day for the first 12 weeks, in 7 to 12 years old patients.
Intervention Type
Drug
Intervention Name(s)
Oxytocin
Other Intervention Name(s)
Intranasal administration of oxytocin (Week 13 to 24)
Intervention Description
Each patient will receive oxytocin in open label (Syntocinon® not reconditioned) from week 13 to week 24 according to the same dosages.
Primary Outcome Measure Information:
Title
Evolution of behavioural troubles evaluated by the global score of Child Behavior Check List Questionnaire after 12 weeks of oxytocin/placebo treatment.
Description
It's variation between inclusion and 12 weeks of total score total problems from the Child Behavior Check List Questionnaire.
Time Frame
Week 12
Secondary Outcome Measure Information:
Title
Evaluation of hyperphagia after 12 weeks of oxytocin/placebo treatment.
Description
It is the variation of each of the three sub-scores obtained from the Dykens hyperphagia questionnaire between day 0 and week 12.
Time Frame
Week 12
Title
Evaluation of social skills after 12 weeks of oxytocin/placebo treatment.
Description
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.
Time Frame
Week 12
Title
Evaluation of auto- and hetero-aggressive after 12 weeks of oxytocin/placebo treatment.
Description
It is variation between day 0 and week 12 of the total score obtained from the self-aggression assessment questionnaire.
Time Frame
Week 12
Title
Evaluation of psychopathology after 12 weeks of oxytocin/placebo treatment.
Description
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.
Time Frame
Week 12
Title
Evaluation of global clinical status after 12 weeks of oxytocin/placebo treatment.
Description
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.
Time Frame
Weeks 12
Title
Evaluation of acyl and desacyl ghrelin plasma levels after 12 weeks of oxytocin/placebo treatment.
Description
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.
Time Frame
Week 12
Title
Evaluation of attentional abilities after 12 weeks of oxytocin/placebo treatment, for patients aged from 7 to 12 years at inclusion.
Description
It is evolution of attentional abilities is evaluated by a computerized test, the Attention Network Test.
Time Frame
Week 12
Title
Evaluation of metabolic brain resting state after 12 weeks of oxytocin/placebo treatment, for patients aged from 7 to 12 years at inclusion.
Description
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.
Time Frame
Week 12
10. Eligibility
Sex
All
Minimum Age & Unit of Time
3 Years
Maximum Age & Unit of Time
12 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
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
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Sophie ÇABAL-BERTHOUMIEU, Dr
Organizational Affiliation
Centre de référence du syndrome de Prader-Willi, Hôpital des Enfants
Official's Role
Principal Investigator
Facility Information:
Facility Name
Centre de référence du syndrome de Prader-Willi Hôpital des Enfants
City
Toulouse
ZIP/Postal Code
31059
Country
France
12. IPD Sharing Statement
Plan to Share IPD
No
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Oxytocin Intranasal Administrations in Children With Prader-Willi Syndrome Aged From 3 to 12 Years
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