Oxytocin Trial in Prader-Willi Syndrome

This study is not yet open for participant recruitment. (see Contacts and Locations)
Verified October 2014 by University of Florida
Sponsor:
Collaborator:
Information provided by (Responsible Party):
University of Florida
ClinicalTrials.gov Identifier:
NCT02013258
First received: December 11, 2013
Last updated: October 21, 2014
Last verified: October 2014
  Purpose

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 Intervention Phase
Prader Willi Syndrome
Drug: Intranasal oxytocin
Other: Placebo
Phase 1

Study Type: Interventional
Study Design: Allocation: Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Crossover Assignment
Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Official Title: Oxytocin Trial in Prader-Willi Syndrome

Resource links provided by NLM:


Further study details as provided by University of Florida:

Primary Outcome Measures:
  • Safety of intranasal oxytocin in children with Prader-Willi syndrome [ Time Frame: 3 months ] [ Designated as safety issue: Yes ]
    Occurrence of adverse event, description and quantification of clinical and behavior severity, pre- and post- intranasal oxytocin and placebo administration.


Secondary Outcome Measures:
  • Evaluation of food intake in Prader-Willi syndrome [ Time Frame: 3 months ] [ Designated as safety issue: No ]
    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:
  • Evaluation of social effects of intranasal oxytocin in children with Prader-Willi syndrome [ Time Frame: 3 months ] [ Designated as safety issue: No ]

    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).

  • Effects of intranasal oxytocin on appetite-regulating hormones [ Time Frame: 3 months ] [ Designated as safety issue: No ]
    Evaluation of plasma OT, ghrelin and other neuroendocrine hormones involved in appetite regulation (cortisol, orexin A, ghrelin, leptin, oxytocin, insulin).


Estimated Enrollment: 24
Study Start Date: November 2014
Estimated Study Completion Date: August 2015
Estimated Primary Completion Date: August 2015 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
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.

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.

  Eligibility

Ages Eligible for Study:   5 Years to 11 Years
Genders Eligible for Study:   Both
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
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT02013258

Contacts
Contact: Christine Keeling, RN 352-294-5280 ckeeling@peds.ufl.edu

Locations
United States, California
University of California, Irvine Not yet recruiting
Orange, California, United States, 92686
Contact: Virginia Kimonis, MD    714-456-2942    vkimonis@uci.edu   
Principal Investigator: Virginia Kimonis, MD         
United States, Florida
University of Florida Not yet recruiting
Gainesville, Florida, United States, 32610
Contact: Jennifer L Miller, MD    352-334-1390    millejl@peds.uf.edu   
Contact: Christine Keeling, RN    352-294-5280    ckeeling@peds.ufl.edu   
Principal Investigator: Jennifer L Miller, MD         
United States, Kansas
University of Kansas Medical Center Not yet recruiting
Kansas City, Kansas, United States, 66160
Contact: Merlin Butler, MD, PhD    913-588-1873    mbutler4@kumc.edu   
Principal Investigator: Merlin Butler, MD, PhD         
Sponsors and Collaborators
University of Florida
Investigators
Principal Investigator: Jennifer L Miller, MD University of Florida
  More Information

Publications:
Responsible Party: University of Florida
ClinicalTrials.gov Identifier: NCT02013258     History of Changes
Other Study ID Numbers: 5208
Study First Received: December 11, 2013
Last Updated: October 21, 2014
Health Authority: United States: Food and Drug Administration
United States: Federal Government
United States: Institutional Review Board

Keywords provided by University of Florida:
Prader Willi syndrome
Oxytocin

Additional relevant MeSH terms:
Prader-Willi Syndrome
Syndrome
Abnormalities, Multiple
Chromosome Disorders
Congenital Abnormalities
Disease
Genetic Diseases, Inborn
Intellectual Disability
Nervous System Diseases
Neurobehavioral Manifestations
Neurologic Manifestations
Nutrition Disorders
Obesity
Overnutrition
Pathologic Processes
Oxytocin
Oxytocics
Pharmacologic Actions
Physiological Effects of Drugs
Reproductive Control Agents
Therapeutic Uses

ClinicalTrials.gov processed this record on October 22, 2014