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Oxytocin Trial in Prader-Willi Syndrome

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT02013258
Recruitment Status : Completed
First Posted : December 17, 2013
Last Update Posted : April 5, 2018
Sponsor:
Collaborator:
National Institutes of Health (NIH)
Information provided by (Responsible Party):
University of Florida

Study Description
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Brief Summary:

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?

Condition or disease Intervention/treatment Phase
Prader Willi Syndrome Drug: Intranasal oxytocin Other: Placebo Phase 1

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 Design
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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 24 participants
Allocation: Randomized
Intervention Model: Crossover Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Official Title: Oxytocin Trial in Prader-Willi Syndrome
Study Start Date : March 2015
Actual Primary Completion Date : August 2015
Actual Study Completion Date : August 2015

Resource links provided by the National Library of Medicine

Drug Information available for: Oxytocin

Arms and Interventions
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Arm Intervention/treatment
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.


Outcome Measures
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Primary Outcome Measures :
  1. 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.


Secondary Outcome Measures :
  1. 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.


Other Outcome Measures:
  1. 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).

  2. 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).


Eligibility Criteria
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Ages Eligible for Study:   5 Years to 11 Years   (Child)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

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
Contacts and Locations
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02013258


Locations
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United States, California
University of California, Irvine
Orange, California, United States, 92686
United States, Florida
University of Florida
Gainesville, Florida, United States, 32610
United States, Kansas
University of Kansas Medical Center
Kansas City, Kansas, United States, 66160
Sponsors and Collaborators
University of Florida
National Institutes of Health (NIH)
Investigators
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Principal Investigator: Jennifer L Miller, MD University of Florida
More Information
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Additional Information:

Publications:

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Responsible Party: University of Florida
ClinicalTrials.gov Identifier: NCT02013258    
Other Study ID Numbers: 5208-N
First Posted: December 17, 2013    Key Record Dates
Last Update Posted: April 5, 2018
Last Verified: April 2018
Keywords provided by University of Florida:
Prader Willi syndrome
Oxytocin
Additional relevant MeSH terms:
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Prader-Willi Syndrome
Syndrome
Disease
Pathologic Processes
Intellectual Disability
Neurobehavioral Manifestations
Neurologic Manifestations
Nervous System Diseases
Abnormalities, Multiple
Congenital Abnormalities
 Chromosome Disorders
Genetic Diseases, Inborn
Obesity
Overweight
Overnutrition
Nutrition Disorders
Oxytocin
Oxytocics
Reproductive Control Agents
Physiological Effects of Drugs