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

NCT ID: NCT03114371

Last Updated: 2020-11-12

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

NA

Total Enrollment

40 participants

Study Classification

INTERVENTIONAL

Study Start Date

2016-11-28

Study Completion Date

2019-01-11

Brief Summary

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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

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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.

Conditions

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Prader-Willi Syndrome

Keywords

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Prader-Willi syndrome Oxytocin Behavioural troubles

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Children will receive after randomization either placebo or oxytocin administration (daily intranasal) for a total duration of 12 consecutive weeks.
Primary Study Purpose

TREATMENT

Blinding Strategy

DOUBLE

Participants Investigators
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.

Study Groups

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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.

Group Type EXPERIMENTAL

Oxytocin

Intervention Type 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.

Oxytocin

Intervention Type DRUG

Each patient will receive oxytocin in open label (Syntocinon® not reconditioned) from week 13 to week 24 according to the same dosages.

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.

Group Type PLACEBO_COMPARATOR

Placebo

Intervention Type 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.

Oxytocin

Intervention Type DRUG

Each patient will receive oxytocin in open label (Syntocinon® not reconditioned) from week 13 to week 24 according to the same dosages.

Interventions

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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.

Intervention Type 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.

Intervention Type DRUG

Oxytocin

Each patient will receive oxytocin in open label (Syntocinon® not reconditioned) from week 13 to week 24 according to the same dosages.

Intervention Type DRUG

Other Intervention Names

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Intranasal administration of oxytocin (Week 1 to 12) Intranasal administration of placebo (Week 1 to 12) Intranasal administration of oxytocin (Week 13 to 24)

Eligibility Criteria

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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
Minimum Eligible Age

3 Years

Maximum Eligible Age

12 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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University Hospital, Toulouse

OTHER

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Sophie ÇABAL-BERTHOUMIEU, Dr

Role: PRINCIPAL_INVESTIGATOR

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

Locations

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

Toulouse, , France

Site Status

Countries

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France

References

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Other Identifiers

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2016-003273-18

Identifier Type: EUDRACT_NUMBER

Identifier Source: secondary_id

15 7837 03

Identifier Type: -

Identifier Source: org_study_id