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

Toulouse University Hospital

Status: Completed

Conditions: Prader-Willi Syndrome

Treatments: Drug: Oxytocin; Drug: Placebo

Study type: Interventional

Funder types: Other

Identifiers

NCT03114371

15 7837 03

2016-003273-18 (EudraCT Number)

Details and patient eligibility

About

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.

Full 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.

Enrollment: 40 patients

Sex: All

Ages: 3 to 12 years old

Volunteers: No Healthy Volunteers

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

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Double Blind

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40 participants in 2 patient groups, including a placebo group

Oxytocin

Experimental group

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.

Treatment:

Drug: Oxytocin

Drug: Oxytocin

Placebo

Placebo Comparator group

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.

Treatment:

Drug: Placebo

Drug: Oxytocin

Drug: Oxytocin