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

Prader-Willi Syndrome Clinical Trial

— OXT-PWS  

Official title:

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT02629991
• Other study ID #: 14-10-427-01
• Status: Completed
• Phase: Phase 2
• Start date: October 2015
• Est. completion date: May 2018

Study information

• Verified date: September 2021
• Source: Montefiore Medical Center
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

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 information / eligibility

• Status: Completed
• Enrollment: 23
• Est. completion date: May 2018
• Est. primary completion date: December 2016
• Accepts healthy volunteers: No
• Gender: All
• Age group: 5 Years to 18 Years

Eligibility

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.

Related Conditions & MeSH terms

• Hyperphagia
• Prader-Willi Syndrome
• Syndrome

Intervention

Drug:
• Intranasal Oxytocin (IN-OXT)
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).

Locations

Country	Name	City	State
United States	Montefiore Medical Center, Albert Einstein College of Medicine	Bronx	New York

Sponsors (2)

Lead Sponsor	Collaborator
Montefiore Medical Center	Foundation for Prader-Willi Research

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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.
Primary	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.
Primary	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.
Primary	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	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).
Secondary	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).
Secondary	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).
Secondary	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).
Secondary	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).
Secondary	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).

External Links

•   Foundation for Prader Willi Research