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

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ClinicalTrials.gov Identifier: NCT03114371
Recruitment Status : Recruiting
First Posted : April 14, 2017
Last Update Posted : May 5, 2017
Sponsor:
Information provided by (Responsible Party):
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.

Condition or disease Intervention/treatment Phase
Prader-Willi Syndrome Drug: Oxytocin Drug: Placebo Not Applicable

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  (Clinical Trial)
Estimated Enrollment : 40 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Intervention Model Description: Children will receive after randomization either placebo or oxytocin administration (daily intranasal) for a total duration of 12 consecutive weeks.
Masking: Double (Participant, Investigator)
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.

Primary Purpose: Treatment
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.
Actual Study Start Date : November 28, 2016
Estimated Primary Completion Date : December 2019
Estimated Study Completion Date : December 2019

Resource links provided by the National Library of Medicine

Drug Information available for: Oxytocin

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 Name: 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 Name: 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: 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 Name: Intranasal administration of placebo (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 Name: Intranasal administration of oxytocin (Week 13 to 24)




Primary Outcome Measures :
  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 Outcome Measures :
  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.



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Ages Eligible for Study:   3 Years to 12 Years   (Child)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03114371


Contacts
Contact: Sophie ÇABAL-BERTHOUMIEU, Dr 5 34 55 74 32 ext 33 cabal-berthoumieu.s@chu-toulouse.fr

Locations
France
Centre de référence du syndrome de Prader-Willi Hôpital des Enfants Recruiting
Toulouse, France, 31059
Contact: Sophie ÇABAL-BERTHOUMIEU, Dr    5 34 55 74 32 ext 33    cabal-berthoumieu.s@chu-toulouse.fr   
Sponsors and Collaborators
University Hospital, Toulouse
Investigators
Principal Investigator: Sophie ÇABAL-BERTHOUMIEU, Dr Centre de référence du syndrome de Prader-Willi, Hôpital des Enfants

Publications:

Responsible Party: University Hospital, Toulouse
ClinicalTrials.gov Identifier: NCT03114371     History of Changes
Other Study ID Numbers: 15 7837 03
2016-003273-18 ( EudraCT Number )
First Posted: April 14, 2017    Key Record Dates
Last Update Posted: May 5, 2017
Last Verified: May 2017
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No

Keywords provided by University Hospital, Toulouse:
Prader-Willi syndrome
Oxytocin
Behavioural troubles

Additional relevant MeSH terms:
Prader-Willi Syndrome
Syndrome
Disease
Pathologic Processes
Intellectual Disability
Neurobehavioral Manifestations
Neurologic Manifestations
Nervous System Diseases
Abnormalities, Multiple
Congenital Abnormalities
Chromosome Disorders
Genetic Diseases, Inborn
Obesity
Overnutrition
Nutrition Disorders
Oxytocin
Oxytocics
Reproductive Control Agents
Physiological Effects of Drugs