Oxytocin Trial in Prader-Willi Syndrome
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|ClinicalTrials.gov Identifier: NCT02013258|
Recruitment Status : Completed
First Posted : December 17, 2013
Last Update Posted : April 5, 2018
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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?
|Condition or disease||Intervention/treatment||Phase|
|Prader Willi Syndrome||Drug: Intranasal oxytocin Other: Placebo||Phase 1|
|Study Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||24 participants|
|Intervention Model:||Crossover Assignment|
|Masking:||Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)|
|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|
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.
- 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.
- 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.
- 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:
- 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 ]Evaluation of plasma OT, ghrelin and other neuroendocrine hormones involved in appetite regulation (cortisol, orexin A, ghrelin, leptin, oxytocin, insulin).
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|Ages Eligible for Study:||5 Years to 11 Years (Child)|
|Sexes Eligible for Study:||All|
|Accepts Healthy Volunteers:||No|
- 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
- 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
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
|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|
|Principal Investigator:||Jennifer L Miller, MD||University of Florida|
|Responsible Party:||University of Florida|
|Other Study ID Numbers:||
|First Posted:||December 17, 2013 Key Record Dates|
|Last Update Posted:||April 5, 2018|
|Last Verified:||April 2018|
Prader Willi syndrome
Nervous System Diseases
Genetic Diseases, Inborn
Reproductive Control Agents
Physiological Effects of Drugs