Oxytocin vs. Placebo for the Treatment Hyperphagia in Children and Adolescents With Prader-Willi Syndrome

Study Overview
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Brief Summary
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The investigators propose a randomized double-blind 8 week treatment trial of intranasal oxytocin (IN-OXT) vs. placebo in 24 subjects aged 5 to 18 years with PWS in order to assess IN-OXT's affect on (1) Eating behaviors (2) Repetitive and disruptive behaviors and (3) Salivary OXT levels.

Detailed Description
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The investigators propose to conduct a treatment study of intranasal oxytocin (IN-OXT) vs. placebo in children and adolescents with Prader-Willi Syndrome (PWS). OXT has already been proven safe and effective in a treatment study of socialization and disruptive behavior in adults with PWS and is being used in infants with PWS in an ongoing clinical trial. The investigators hypothesize that OXT will be superior to placebo and have a positive effect on child and adolescent PWS eating and repetitive behaviors. Additional knowledge of OXT's ability to reduce overeating could lead to improvement of patient's quality of life and physical health and reduction in familial stress. The investigators propose a randomized double-blind 8 week treatment trial of intranasal OXT vs. placebo in 24 subjects aged 5 to 18 years with PWS in order to assess IN-OXT's affect on (1) Eating behaviors (2) Repetitive and disruptive behaviors and (3) Salivary OXT levels. If superior to placebo, this data will add to the current knowledge that OXT is an effective treatment for hyperphagia as well as other problematic symptomatology of PWS.

Official Title
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Oxytocin vs. Placebo for the Treatment Hyperphagia in Children and Adolescents With Prader-Willi Syndrome

Conditions
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Prader-Willi Syndrome, Hyperphagia

Intervention / Treatment
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* Drug: Intranasal Oxytocin (IN-OXT)
* Other: Matched Placebo


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Male or Female child outpatients aged 5 to 18 years Diagnosis of PWS confirmed by genetic testing and patient medical records and history Stable pharmacologic, educational, behavioral and/or dietary interventions for 4 weeks prior to the study start, and for the duration of the study. Have a physical exam and laboratory results that are within the norms for PWS Have a parent/caregiver/guardian that is able to consent for their participation and complete assessments regarding the child's development and behavior improvement throughout the study. Exclusion Criteria: Exposure to any investigational agent in the 30 days prior to randomization Prior chronic treatment with oxytocin. A primary psychiatric diagnosis other than ASD, including bipolar disorder, psychosis, schizophrenia, PTSD or major depressive disorder. These patients will be excluded due to potential confounding results. Pregnant or lactating patients. IN-OXT has not been studied in pregnant or lactating women. A medical condition that might interfere with the conduct of the study, confound interpretation of study results or endanger their own well-being. Plan to initiate or change nonpharmacologic or pharmacologic interventions during the course of the study. Females using an estrogen-based contraceptive. As an alternative to an estrogen based contraceptive, subjects will be counseled to use progesterone-based contraceptives; cervical caps; cervical sponges; or spermicidal foam in combination with a condom. Subjects will need to use a double barrier method to be in the study.

Ages Eligible for Study
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Minimum Age: 5 Years
Maximum Age: 18 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Intranasal Oxytocin<br>Participants were instructed to take 16 IU/per day everyday (2 puffs per nostril, 4 IU each puff). | Drug: Intranasal Oxytocin (IN-OXT)<br>* Participants were instructed to take 16 IU/per day everyday (2 puffs per nostril, 4 IU each puff).<br>* Other names: Syntocinon;|
| Placebo Comparator: Matched Placebo<br>Participants were instructed to take 16 IU/per day everyday (2 puffs per nostril, 4 IU each puff). | Other: Matched Placebo<br>* Participants were instructed to take 16 IU/per day everyday (2 puffs per nostril, 4 IU each puff).<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Hyperphagia Questionnaire (HQ)- Total Score | The Hyperphagia Questionnaire (HQ)- Total Score. The HQ is a 11-item clinician-rated instrument that was designed to measure food related behaviors in PWS. Items map onto 3 subscales; Hyperphagic Behavior, Hyperphagic Drive, and Hyperphagia Severity. The subscales are summed together to compute the Total Score. The Total Score ranges from 11-55, with higher scores indicating more hyperphagia and a worse outcome. | Assessed trends over multiple time points (weeks 0, 2, 4, 6, and 8), week 0 (baseline), week 4 and week 8 are reported. | 
| Hyperphagia Questionnaire (HQ)- Behavior Factor Score | The Hyperphagia Questionnaire (HQ)- Behavior Factor Score assesses food seeking behaviors (e.g., stealing food). The Behavior Factor Score ranges from 5-25, with higher scores indicating more hyperphagic behaviors and a worse outcome. | Assessed trends over multiple time points (weeks 0, 2, 4, 6, and 8), week 0 (baseline), week 4 and week 8 are reported. | 
| Hyperphagia Questionnaire (HQ)- Drive Factor Score | The Hyperphagia Questionnaire (HQ)- Drive Factor Score assesses the persistence in asking for food. The Drive Factor Score ranges from 4-20, with higher scores indicating higher hyperphagic drive and a worse outcome | Assessed trends over multiple time points (weeks 0, 2, 4, 6, and 8), week 0 (baseline), week 4 and week 8 are reported. | 
| Hyperphagia Questionnaire (HQ) -Severity Factor Score | The Hyperphagia Questionnaire (HQ)- Severity Factor Score assesses the severity of hyperphagia. The Severity Factor Score ranges from 2-10, with higher scores indicating higher severity. | Assessed trends over multiple time points (weeks 0, 2, 4, 6, and 8), week 0 (baseline), week 4 and week 8 are reported. | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Repetitive Behavior Scale-Revised (RBS-R) Ritualistic Behavior Factor Score | The Repetitive Behavior Scale-Revised (RBS-R) Ritualistic Behavior Factor Score assesses ritualistic behaviors. Scores range from 0-18, with higher scores indicating more ritualistic behaviors and a worse outcome. | Trends over multiple time points (Weeks 0, 4, and 8). | 
| Repetitive Behavior Scale-Revised (RBS-R) Sameness Factor Score | The Repetitive Behavior Scale-Revised (RBS-R) Sameness Factor Score assesses the need for sameness in the environment. Scores range from 0-33, with higher scores indicating more need for sameness and a worse outcome. | Trends over multiple time points (Weeks 0, 4, and 8). | 
| Repetitive Behavior Scale-Revised (RBS-R) Stereotyped Behavior Factor Score | The Repetitive Behavior Scale-Revised (RBS-R) Stereotyped Behavior Factor Score assesses stereotyped behaviors. Scores range from 0-18, with higher scores indicating more stereotyped behaviors and a worse outcome. | Trends over multiple time points (Weeks 0, 4, and 8). | 
| Repetitive Behavior Scale-Revised (RBS-R) Compulsive Behavior Factor Score | The Repetitive Behavior Scale-Revised (RBS-R) Compulsive Behavior Factor Score assesses compulsive behaviors. Scores range from 0-24, with higher scores indicating more compulsive behaviors and a worse outcome. | Trends over multiple time points (Weeks 0, 4, and 8). | 
| Repetitive Behavior Scale-Revised (RBS-R) Self-Injurious Factor Score | The Repetitive Behavior Scale-Revised (RBS-R) Self-Injurious Factor Score assesses self-injurious behaviors. Scores range from 0-24, with higher scores indicating more self-injurious behaviors and a worse outcome. | Trends over multiple time points (Weeks 0, 4, and 8). | 
| Repetitive Behavior Scale-Revised (RBS-R) Restricted Interest Factor Score | The Repetitive Behavior Scale-Revised (RBS-R) Restricted Interest Factor Score assesses restricted interests. Scores range from 0-12, with higher scores indicating more restricted interests and a worse outcome. | Trends over multiple time points (Weeks 0, 4, and 8). | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Prader-Willi Syndrome (PWS), Hyperphagia