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

Phase 2

Completed

Conditions: Prader-Willi Syndrome
Hyperphagia

Interventions: Drug: Intranasal Oxytocin (IN-OXT)
Other: Matched Placebo

Registration Number: NCT02629991

Lead Sponsor: Montefiore Medical Center

Brief Summary: 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: 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.

Recruitment & Eligibility

Status: COMPLETED

Sex: All

Target Recruitment: 23

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.

Study & Design

Study Type: INTERVENTIONAL

Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
Intranasal Oxytocin	Intranasal Oxytocin (IN-OXT)	Participants were instructed to take 16 IU/per day everyday (2 puffs per nostril, 4 IU each puff).
Matched Placebo	Matched Placebo	Participants were instructed to take 16 IU/per day everyday (2 puffs per nostril, 4 IU each puff).

Primary Outcome Measures

Name	Time	Method
Hyperphagia Questionnaire (HQ)- Total Score	Assessed trends over multiple time points (weeks 0, 2, 4, 6, and 8), week 0 (baseline), week 4 and week 8 are reported.	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.
Hyperphagia Questionnaire (HQ)- Behavior Factor Score	Assessed trends over multiple time points (weeks 0, 2, 4, 6, and 8), week 0 (baseline), week 4 and week 8 are reported.	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.
Hyperphagia Questionnaire (HQ)- Drive Factor Score	Assessed trends over multiple time points (weeks 0, 2, 4, 6, and 8), week 0 (baseline), week 4 and week 8 are reported.	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
Hyperphagia Questionnaire (HQ) -Severity Factor Score	Assessed trends over multiple time points (weeks 0, 2, 4, 6, and 8), week 0 (baseline), week 4 and week 8 are reported.	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.

Secondary Outcome Measures

Name	Time	Method
Repetitive Behavior Scale-Revised (RBS-R) Ritualistic Behavior Factor Score	Trends over multiple time points (Weeks 0, 4, and 8).	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.
Repetitive Behavior Scale-Revised (RBS-R) Sameness Factor Score	Trends over multiple time points (Weeks 0, 4, and 8).	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.
Repetitive Behavior Scale-Revised (RBS-R) Stereotyped Behavior Factor Score	Trends over multiple time points (Weeks 0, 4, and 8).	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.
Repetitive Behavior Scale-Revised (RBS-R) Compulsive Behavior Factor Score	Trends over multiple time points (Weeks 0, 4, and 8).	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.
Repetitive Behavior Scale-Revised (RBS-R) Self-Injurious Factor Score	Trends over multiple time points (Weeks 0, 4, and 8).	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.
Repetitive Behavior Scale-Revised (RBS-R) Restricted Interest Factor Score	Trends over multiple time points (Weeks 0, 4, and 8).	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.