Oxytocin Trial in Prader-Willi Syndrome

April 4, 2018 updated by: University of Florida

Individuals with Prader-Willi syndrome (PWS) have been found to have a deficit of oxytocin-producing neurons and decreased oxytocin receptor gene function, so the purpose of this study is to determine if oxytocin (OT) administration will improve some of the aspects of Prader-Willi syndrome that are particularly troublesome for children and their families (the insatiable appetite and social behaviors).

The research questions are:

  1. Does intranasal oxytocin cause any side effects in children with PWS?
  2. Does intranasal oxytocin administration alter appetite or behaviors in PWS?

Study Overview

Status

Status

Indicates the current recruitment status or the expanded access status.
Completed

Conditions

Conditions

The disease, disorder, syndrome, illness, or injury that is being studied. On ClinicalTrials.gov, conditions may also include other health-related issues, such as lifespan, quality of life, and health risks.

Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.

Detailed Description

This study is to investigate if intranasal oxytocin will improve hyperphagia, social skills, and behaviors in subjects with Prader-Willi syndrome. This will be a randomized placebo controlled pilot study. The primary outcome measure is to determine if intranasal administration of oxytocin will cause any adverse events in subjects with Prader-Willi syndrome. Secondarily, the investigators will also perform evaluations to determine if intranasal oxytocin has any effect on social skills, behaviors, or appetite in children with Prader-Willi syndrome.

Study Type

Study Type

Describes the nature of a clinical study. Study types include interventional studies (also called clinical trials), observational studies (including patient registries), and expanded access.
Interventional

Enrollment (Actual)

Enrollment

The number of participants in a clinical study. The "anticipated" enrollment is the target number of participants that the researchers need for the study.
24

Phase

Phase

The stage of a clinical trial studying a drug or biological product, based on definitions developed by the U.S. Food and Drug Administration (FDA). The phase is based on the study's objective, the number of participants, and other characteristics. There are five phases: Early Phase 1 (formerly listed as Phase 0), Phase 1, Phase 2, Phase 3, and Phase 4. Not Applicable is used to describe trials without FDA-defined phases, including trials of devices or behavioral interventions.
  • Phase 1

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • United States
    • California
      • Orange, California, United States, 92686
        • University of California, Irvine
    • Florida
      • Gainesville, Florida, United States, 32610
        • University of Florida
    • Kansas
      • Kansas City, Kansas, United States, 66160
        • University of Kansas Medical Center

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Eligibility Criteria

The key requirements that people who want to participate in a clinical study must meet or the characteristics they must have. Eligibility criteria consist of both inclusion criteria (which are required for a person to participate in the study) and exclusion criteria (which prevent a person from participating). Types of eligibility criteria include whether a study accepts healthy volunteers, has age or age group requirements, or is limited by sex.

Ages Eligible for Study

5 years to 11 years (Child)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

Inclusion Criteria:

  • Children with genetically confirmed PWS
  • Ages at ≥ 5 years and ≤ 11 years (must start treatment prior to 11th birthday)
  • Child must be in nutritional phase 2b or 3, as determined by the PI at each site.
  • Must currently be on growth hormone treatment, and have been receiving growth hormone treatment for at least one year prior to screening date.
  • Treatment cannot have been interrupted for more than 1 week within 3 months prior to screening date.
  • Priority will be given to children currently enrolled in the RDCRN Natural History study

Exclusion Criteria:

  • Inability to tolerate intranasal administration of medication
  • Hepatic insufficiency (AST/ALT greater than 3 times the normal levels for age)
  • Renal insufficiency (BUN/Creatinine greater than 3 times the normal levels for age)
  • History of an abnormal ECG (as determined by a cardiologist). If there is any question about cardiac function, ECG reports will be reviewed with a cardiologist prior to enrollment in the study.
  • Child not receiving growth hormone treatment
  • Child with hypertension or hypotension for age and sex (blood pressure >97% for age and sex or blood pressure <3% for age and sex)
  • Diabetes mellitus
  • Pregnant or lactating.
  • Schizophrenia or psychosis
  • Taking any psychotropic medications

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

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

Arms and Interventions

Participant Group / Arm

Participant Group / Arm

A group or subgroup of participants in a clinical trial that receives a specific intervention/treatment, or no intervention, according to the trial's protocol.
Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.
Active Comparator: Intranasal oxytocin
Intranasal oxytocin. 16 IU intranasal oxytocin x 5 days. One month interval between arms of treatment.
Drug: Intranasal oxytocin
This is a double-blind placebo controlled 2x2 study. Subjects will receive OT for 5 consecutive days during their 7 day stay. This will be followed by a wash out period of 4-6 weeks.
Other Names:
  • Pitocin
  • Syntocinon
Placebo Comparator: Placebo
Placebo will be administered via nasal spray - 1 spray in each nostril x5 days.
Other: Placebo
This is a double-blind placebo controlled 2x2 study. Placebo will be given via intranasal spray, one spray in each nostril daily x 5 days. One month interval between arms of treatment.

What is the study measuring?

Primary Outcome Measures

Primary Outcome Measures

In a clinical study's protocol, the planned outcome measure that is the most important for evaluating the effect of an intervention/treatment. Most clinical studies have one primary outcome measure, but some have more than one.
Outcome Measure
Measure Description
Time Frame
Safety of intranasal oxytocin in children with Prader-Willi syndrome
Time Frame: 3 months
Occurrence of adverse event, description and quantification of clinical and behavior severity, pre- and post- intranasal oxytocin and placebo administration.
3 months

Secondary Outcome Measures

Secondary Outcome Measures

In a clinical study's protocol, a planned outcome measure that is not as important as the primary outcome measure for evaluating the effect of an intervention but is still of interest. Most clinical studies have more than one secondary outcome measure.
Outcome Measure
Measure Description
Time Frame
Evaluation of food intake in Prader-Willi syndrome
Time Frame: 3 months
Quantitative evaluation of hyperphagia via the Hyperphagia Questionnaire obtained on days 1, 4 and 6 during the 7 day study protocol and conducted in the evening on these days. Score will range from 0 (no hyperphagia behaviors) to 96 (most severe hyperphagia behaviors). Additionally quantity of food consumed will be recorded on Days 1,4, and 6.
3 months

Other Outcome Measures

Other Outcome Measures

For clinical trials, a planned measurement described in the protocol that is used to determine the effect of an intervention/treatment on participants. For observational studies, a measurement or observation that is used to describe patterns of diseases or traits, or associations with exposures, risk factors, or treatment. Types of outcome measures include primary outcome measure and secondary outcome measure.
Outcome Measure
Measure Description
Time Frame
Evaluation of social effects of intranasal oxytocin in children with Prader-Willi syndrome
Time Frame: 3 months

Evaluation of anxiety, food issues, irritability, social communication and behavioral issues will be measured on days 1, 4 and 6 using:

  1. Aberrant Behavior Checklist: Behavior rating scale used to measure behavior problems of children and adults with intellectual disabilities ages 6 - 54 years. Scores will range from 0 (no problem behaviors) to 174 (severe aberrant behaviors).
  2. Social Responsiveness Scale:designed to measure the breadth of repetitive behaviors. Scores will range from 64 (no repetitive behaviors) to 260 (severe repetitive behaviors).
  3. Repetitive Behavior Scales-Revised: Designed to measure the breadth of repetitive behaviors in autism spectrum disorder including: Ritualistic/Sameness Behavior; Stereotypic Behavior; Self-injurious Behavior; Compulsive Behavior; and Restricted Interests. Scores will range from 0 (no autistic behaviors) to 100 (severe autistic behaviors).
3 months
Effects of intranasal oxytocin on appetite-regulating hormones
Time Frame: 3 months
Evaluation of plasma OT, ghrelin and other neuroendocrine hormones involved in appetite regulation (cortisol, orexin A, ghrelin, leptin, oxytocin, insulin).
3 months

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Sponsor

The organization or person who initiates the study and who has authority and control over the study.
University of Florida

Collaborators

Collaborators

An organization other than the sponsor that provides support for a clinical study. This support may include activities related to funding, design, implementation, data analysis, or reporting.
National Institutes of Health (NIH)

Investigators

Investigators

A researcher involved in a clinical study. Related terms include site principal investigator, site sub-investigator, study chair, study director, and study principal investigator.
  • Principal Investigator: Jennifer L Miller, MD, University of Florida

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Helpful Links

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start

Study Start

The actual date on which the first participant was enrolled in a clinical study. The "anticipated" study start date is the date that the researchers think will be the study start date.
March 1, 2015

Primary Completion (Actual)

Primary Completion

The date on which the last participant in a clinical study was examined or received an intervention to collect final data for the primary outcome measure. Whether the clinical study ended according to the protocol or was terminated does not affect this date. For clinical studies with more than one primary outcome measure with different completion dates, this term refers to the date on which data collection is completed for all the primary outcome measures. The "anticipated" primary completion date is the date that the researchers think will be the primary completion date for the study.
August 1, 2015

Study Completion (Actual)

Study Completion

The date on which the last participant in a clinical study was examined or received an intervention/treatment to collect final data for the primary outcome measures, secondary outcome measures, and adverse events (that is, the last participant's last visit). The "anticipated" study completion date is the date that the researchers think will be the study completion date.
August 1, 2015

Study Registration Dates

First Submitted

First Submitted

The date on which the study sponsor or investigator first submitted a study record to ClinicalTrials.gov. There is typically a delay of a few days between the first submitted date and the record's availability on ClinicalTrials.gov (the first posted date).
December 11, 2013

First Submitted That Met QC Criteria

First Submitted That Met QC Criteria

The date on which the study sponsor or investigator first submits a study record that is consistent with National Library of Medicine (NLM) quality control (QC) review criteria. The sponsor or investigator may need to revise and submit a study record one or more times before NLM's QC review criteria are met. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
December 16, 2013

First Posted (Estimate)

First Posted

The date on which the study record was first available on ClinicalTrials.gov after National Library of Medicine (NLM) quality control (QC) review has concluded. There is typically a delay of a few days between the date the study sponsor or investigator submitted the study record and the first posted date.
December 17, 2013

Study Record Updates

Last Update Posted (Actual)

Last Update Posted

The most recent date on which changes to a study record were made available on ClinicalTrials.gov. There may be a delay between when the changes were submitted to ClinicalTrials.gov by the study's sponsor or investigator (the last update submitted date) and the last update posted date.
April 5, 2018

Last Update Submitted That Met QC Criteria

Last Update Submitted That Met QC Criteria

The most recent date on which the study sponsor or investigator submitted changes to a study record that are consistent with National Library of Medicine (NLM) quality control (QC) review criteria. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
April 4, 2018

Last Verified

Last Verified

The most recent date on which the study sponsor or investigator confirmed the information about a clinical study on ClinicalTrials.gov as accurate and current. If a study with a recruitment status of recruiting; not yet recruiting; or active, not recruiting has not been confirmed within the past 2 years, the study's recruitment status is shown as unknown.
April 1, 2018

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

Other Study ID Numbers

Identifiers or ID numbers other than the NCT number that are assigned to a clinical study by the study's sponsor, funders, or others. These numbers may include unique identifiers from other trial registries and National Institutes of Health grant numbers.
  • 5208-N

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