Oxytocin Trial in Prader-Willi Syndrome
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:
- Does intranasal oxytocin cause any side effects in children with PWS?
- Does intranasal oxytocin administration alter appetite or behaviors in PWS?
|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|
- 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.
- 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.
- 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:
- 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).
- Social Responsiveness Scale:designed to measure the breadth of repetitive behaviors. Scores will range from 64 (no repetitive behaviors) to 260 (severe repetitive behaviors).
- 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).
|Study Start Date:||March 2015|
|Estimated Study Completion Date:||August 2015|
|Estimated Primary Completion Date:||August 2015 (Final data collection date for primary outcome measure)|
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.
Placebo Comparator: Placebo
Placebo will be administered via nasal spray - 1 spray in each nostril x5 days.
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.
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.
Please refer to this study by its ClinicalTrials.gov identifier: NCT02013258
|Contact: Christine Keeling, RNfirstname.lastname@example.org|
|United States, California|
|University of California, Irvine||Recruiting|
|Orange, California, United States, 92686|
|Contact: Virginia Kimonis, MD 714-456-2942 email@example.com|
|Principal Investigator: Virginia Kimonis, MD|
|United States, Florida|
|University of Florida||Recruiting|
|Gainesville, Florida, United States, 32610|
|Contact: Jennifer L Miller, MD 352-334-1390 firstname.lastname@example.org|
|Contact: Christine Keeling, RN 352-294-5280 email@example.com|
|Principal Investigator: Jennifer L Miller, MD|
|United States, Kansas|
|University of Kansas Medical Center||Recruiting|
|Kansas City, Kansas, United States, 66160|
|Contact: Merlin Butler, MD, PhD 913-588-1873 firstname.lastname@example.org|
|Principal Investigator: Merlin Butler, MD, PhD|
|Principal Investigator:||Jennifer L Miller, MD||University of Florida|