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

November 10, 2020 updated by: University Hospital, Toulouse

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.

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.

Study Overview

Status

Completed

Conditions

Intervention / Treatment

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 Type

Interventional

Enrollment (Actual)

40

Phase

  • Not Applicable

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • France
      • Toulouse, France, 31059
        • Centre de référence du syndrome de Prader-Willi Hôpital des Enfants

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

3 years to 12 years (Child)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

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 Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Randomized
  • Interventional Model: Parallel Assignment
  • Masking: Double

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: 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.
Drug: Oxytocin
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.
Other Names:
  • Intranasal administration of oxytocin (Week 1 to 12)
Drug: Oxytocin
Each patient will receive oxytocin in open label (Syntocinon® not reconditioned) from week 13 to week 24 according to the same dosages.
Other Names:
  • Intranasal administration of oxytocin (Week 13 to 24)
Placebo Comparator: 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.
Drug: Oxytocin
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.
Other Names:
  • Intranasal administration of oxytocin (Week 1 to 12)
Drug: Oxytocin
Each patient will receive oxytocin in open label (Syntocinon® not reconditioned) from week 13 to week 24 according to the same dosages.
Other Names:
  • Intranasal administration of oxytocin (Week 13 to 24)
Drug: Placebo
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.
Other Names:
  • Intranasal administration of placebo (Week 1 to 12)

What is the study measuring?

Primary Outcome Measures

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

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Evaluation of hyperphagia after 12 weeks of oxytocin/placebo treatment.
Time Frame: 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.
Week 12
Evaluation of social skills after 12 weeks of oxytocin/placebo treatment.
Time Frame: 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.
Week 12
Evaluation of auto- and hetero-aggressive after 12 weeks of oxytocin/placebo treatment.
Time Frame: Week 12
It is variation between day 0 and week 12 of the total score obtained from the self-aggression assessment questionnaire.
Week 12
Evaluation of psychopathology after 12 weeks of oxytocin/placebo treatment.
Time Frame: 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.
Week 12
Evaluation of global clinical status after 12 weeks of oxytocin/placebo treatment.
Time Frame: 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.
Weeks 12
Evaluation of acyl and desacyl ghrelin plasma levels after 12 weeks of oxytocin/placebo treatment.
Time Frame: 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.
Week 12
Evaluation of attentional abilities after 12 weeks of oxytocin/placebo treatment, for patients aged from 7 to 12 years at inclusion.
Time Frame: Week 12
It is evolution of attentional abilities is evaluated by a computerized test, the Attention Network Test.
Week 12
Evaluation of metabolic brain resting state after 12 weeks of oxytocin/placebo treatment, for patients aged from 7 to 12 years at inclusion.
Time Frame: 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.
Week 12

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

University Hospital, Toulouse

Investigators

  • Principal Investigator: Sophie ÇABAL-BERTHOUMIEU, Dr, Centre de référence du syndrome de Prader-Willi, Hôpital des Enfants

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

November 28, 2016

Primary Completion (Actual)

January 1, 2019

Study Completion (Actual)

January 11, 2019

Study Registration Dates

First Submitted

March 10, 2017

First Submitted That Met QC Criteria

April 13, 2017

First Posted (Actual)

April 14, 2017

Study Record Updates

Last Update Posted (Actual)

November 12, 2020

Last Update Submitted That Met QC Criteria

November 10, 2020

Last Verified

November 1, 2020

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • 15 7837 03
  • 2016-003273-18 (EudraCT Number)

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

No

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

product manufactured in and exported from the U.S.

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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