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

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.

Study Overview

• Status: Completed
• Conditions: Hyperphagia; Prader-Willi Syndrome
• Intervention / Treatment: Drug: Intranasal Oxytocin (IN-OXT); Other: Matched Placebo

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.

• Study Type: Interventional
• Enrollment (Actual): 23
• Phase: Phase 2

Contacts and Locations

Study Locations

• United States
  • New York
    • Bronx, New York, United States, 10467
      • Montefiore Medical Center, Albert Einstein College of Medicine

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 5 years to 18 years (Child, Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

Inclusion Criteria:

1. Male or Female child outpatients aged 5 to 18 years
2. Diagnosis of PWS confirmed by genetic testing and patient medical records and history
3. Stable pharmacologic, educational, behavioral and/or dietary interventions for 4 weeks prior to the study start, and for the duration of the study.
4. Have a physical exam and laboratory results that are within the norms for PWS
5. 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:

1. Exposure to any investigational agent in the 30 days prior to randomization
2. Prior chronic treatment with oxytocin.
3. 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.
4. Pregnant or lactating patients. IN-OXT has not been studied in pregnant or lactating women.
5. A medical condition that might interfere with the conduct of the study, confound interpretation of study results or endanger their own well-being.
6. Plan to initiate or change nonpharmacologic or pharmacologic interventions during the course of the study.
7. 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 Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: Quadruple

Number of Arms

2

Arms and Interventions

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

What is the study measuring?

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).

Collaborators and Investigators

• Sponsor: Montefiore Medical Center
• Collaborators: Foundation for Prader-Willi Research
• Investigators: Principal Investigator: Eric Hollander, MD, Montefiore Medical Center

Publications and helpful links

Helpful Links

• Foundation for Prader Willi Research

Study record dates

Study Major Dates

• Study Start: October 1, 2015
• Primary Completion (Actual): December 1, 2016
• Study Completion (Actual): May 1, 2018

Study Registration Dates

• First Submitted: October 18, 2015
• First Submitted That Met QC Criteria: December 10, 2015
• First Posted (Estimate): December 15, 2015

Study Record Updates

• Last Update Posted (Actual): October 7, 2021
• Last Update Submitted That Met QC Criteria: September 10, 2021
• Last Verified: September 1, 2021

More Information

Terms related to this study

• Keywords: Hyperphagia; Prader-Willi Syndrome (PWS)
• Additional Relevant MeSH Terms: Pathologic Processes; Nervous System Diseases; Neurologic Manifestations; Neurobehavioral Manifestations; Disease; Congenital Abnormalities; Signs and Symptoms, Digestive; Overnutrition; Nutrition Disorders; Genetic Diseases, Inborn; Intellectual Disability; Abnormalities, Multiple; Chromosome Disorders; Obesity; Syndrome; Prader-Willi Syndrome; Hyperphagia; Physiological Effects of Drugs; Reproductive Control Agents; Oxytocics; Oxytocin
• Other Study ID Numbers: 14-10-427-01

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: No