Oxytocin vs. Placebo for the Treatment Hyperphagia in Children and Adolescents With Prader-Willi Syndrome - Clinical Trial for Prader-Willi Syndrome | NCT02629991

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Primary Purpose

Status

Completed

Phase

Phase 2

Locations

United States

Study Type

Interventional

Intervention

Intranasal Oxytocin (IN-OXT)

Matched Placebo

About this trial

This is an interventional treatment trial for Prader-Willi Syndrome focused on measuring Prader-Willi Syndrome (PWS), Hyperphagia

Eligibility Criteria

5 Years - 18 Years (Child, Adult)All SexesDoes not accept healthy volunteers

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.

Sites / Locations

Montefiore Medical Center, Albert Einstein College of Medicine

Arms of the Study

Arm 1

Arm 2

Arm Type

Experimental

Placebo Comparator

Arm Label

Intranasal Oxytocin

Matched Placebo

Arm Description

Participants were instructed to take 16 IU/per day everyday (2 puffs per nostril, 4 IU each puff).

Outcomes

Primary Outcome Measures

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.

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.

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

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.

Secondary Outcome Measures

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.

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.

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.

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.

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.

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.

Full Information

NCT ID

NCT02629991

First Posted

October 18, 2015

Last Updated

September 10, 2021

Sponsor

Montefiore Medical Center

Collaborators

Foundation for Prader-Willi Research

1. Study Identification

Unique Protocol Identification Number

NCT02629991

Brief Title

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

Acronym

OXT-PWS

Official Title

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

Study Type

Interventional

2. Study Status

Record Verification Date

September 2021

Overall Recruitment Status

Completed

Study Start Date

October 2015 (undefined)

Primary Completion Date

December 2016 (Actual)

Study Completion Date

May 2018 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Principal Investigator

Name of the Sponsor

Montefiore Medical Center

Collaborators

Foundation for Prader-Willi Research

4. Oversight

Data Monitoring Committee

Yes

5. Study Description

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

Prader-Willi Syndrome, Hyperphagia

Keywords

Prader-Willi Syndrome (PWS), Hyperphagia

7. Study Design

Primary Purpose

Treatment

Study Phase

Phase 2

Interventional Study Model

Parallel Assignment

Masking

ParticipantCare ProviderInvestigatorOutcomes Assessor

Allocation

Randomized

Enrollment

23 (Actual)

8. Arms, Groups, and Interventions

Arm Title

Intranasal Oxytocin

Arm Type

Experimental

Arm Description

Participants were instructed to take 16 IU/per day everyday (2 puffs per nostril, 4 IU each puff).

Arm Title

Matched Placebo

Arm Type

Placebo Comparator

Arm Description

Participants were instructed to take 16 IU/per day everyday (2 puffs per nostril, 4 IU each puff).

Intervention Type

Drug

Intervention Name(s)

Intranasal Oxytocin (IN-OXT)

Other Intervention Name(s)

Syntocinon

Intervention Description

Participants were instructed to take 16 IU/per day everyday (2 puffs per nostril, 4 IU each puff).

Intervention Type

Other

Intervention Name(s)

Matched Placebo

Intervention Description

Participants were instructed to take 16 IU/per day everyday (2 puffs per nostril, 4 IU each puff).

Primary Outcome Measure Information:

Title

Hyperphagia Questionnaire (HQ)- Total Score

Description

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.

Time Frame

Assessed trends over multiple time points (weeks 0, 2, 4, 6, and 8), week 0 (baseline), week 4 and week 8 are reported.

Title

Hyperphagia Questionnaire (HQ)- Behavior Factor Score

Description

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.

Time Frame

Assessed trends over multiple time points (weeks 0, 2, 4, 6, and 8), week 0 (baseline), week 4 and week 8 are reported.

Title

Hyperphagia Questionnaire (HQ)- Drive Factor Score

Description

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

Time Frame

Assessed trends over multiple time points (weeks 0, 2, 4, 6, and 8), week 0 (baseline), week 4 and week 8 are reported.

Title

Hyperphagia Questionnaire (HQ) -Severity Factor Score

Description

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.

Time Frame

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 Measure Information:

Title

Repetitive Behavior Scale-Revised (RBS-R) Ritualistic Behavior Factor Score

Description

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.

Time Frame