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NCT Number: NCT03114371

Oxytocin Intranasal Administrations 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.

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Key information

Conditions

Prader-Willi Syndrome Abnormalities, Multiple Chromosome Disorders Congenital Abnormalities Congenital, Hereditary, and Neonatal Diseases and Abnormalities Genetic Diseases, Inborn Imprinting Disorders Intellectual Disability Nervous System Diseases Neurobehavioral Manifestations Neurologic Manifestations Nutrition Disorders Nutritional and Metabolic Diseases Obesity Overnutrition Overweight

Age range

3 year–12 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Not applicable

Primary location

Centre de référence du syndrome de Prader-Willi Hôpital des Enfants

Toulouse, 31059, France

About this study

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.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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

Treatment and study plan

Oxytocin

Drug

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)

Placebo

Drug

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)

Primary outcomes

  1. 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.

Secondary outcomes

  1. 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.

  2. 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.

  3. 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.

  4. 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.

  5. 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.

  6. 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.

  7. 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.

  8. 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.

Sponsors and collaborators

Lead sponsor

University Hospital, Toulouse

Other

Registry information

Official study 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.

Acronym: Oxyjeune

Important dates

Study start
2016
Primary completion
2019
Study completion
2019
First posted
Apr 14, 2017
Registry last updated
Nov 12, 2020

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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