Intranasal Oxytocin vs. Placebo for the Treatment of Hyperphagia in Prader-Willi Syndrome

Inclusion Criteria

• Male or female pediatric outpatients aged 5 to 17 years
• Must be in PWS nutritional phase 2b or 3 as determined by PI
• Must be on growth hormone treatment and have been receiving stable doses of growth hormone treatment for at least 3 months prior to screening date. Treatment cannot have been interrupted for more than one week within 3 months of screening.
• Diagnosis of PWS confirmed by patient medical records.
• A score of at least moderate severity on the Hyperphagia Questionnaire for Clinical Trials at both screening and baseline visits.
• Stable dosages of hormone treatments (including testosterone and estrogen supplements) for 4 weeks prior to randomization and for the duration of the study.
• Stable dosages of metabolic treatments that could affect appetite (including metformin) for 4 weeks prior to randomization and for the duration of the study.
• Physical exam and laboratory results that are within the normal range for individuals with PWS.
• Presence of a parent/caregiver/guardian that is able to consent for their participation and complete assessments regarding the child's development and behavior change throughout the study.

Exclusion Criteria

• Exposure to any investigational agent in the 30 days prior to randomization.
• Child not receiving growth hormone treatment
• Children weighing less than 40 lbs
• Children with unstable Type 2 Diabetes confirmed by Hemoglobin A1C levels at screening
• Children with unstable medical co-morbidities at baseline.
• Children with active upper respiratory infections at screening.
• A primary psychiatric diagnosis other than Autism Spectrum Disorder (ASD), including bipolar disorder, psychosis, schizophrenia, Post-traumatic stress disorder (PTSD) or major depressive disorder (MDD). These patients will be excluded due to potential confounding results.
• Pregnant or lactating patients or patients who will not agree to use a double barrier method of contraception. IN-OXT has not been studied in pregnant or lactating women.
• Females using an estrogen-based contraceptive. As an alternative to an estrogen based contraceptive, subjects will be counseled to use progesterone-based contraceptives; cervical cap; cervical sponges; or spermicidal foam in combination with a condom. Subjects will need to use a double barrier method to be in the study.
• A medical condition that might interfere with the conduct of the study, confound interpretation of study results or endanger the subject's well-being.
• A known diagnosis of Rett's Syndrome of Childhood Disintegrative Disorder or marked sensory impairment such as deafness or blindness.
• Subjects who have changes in allied health therapies, behavioral or educational interventions within four weeks prior to randomization other than those associated with school holidays.
• Subjects who have had changes in medications or medication doses of risperidone, aripiprazole, other antipsychotic medications, clonidine, guanfacine, stimulants or anti-convulsants within four weeks of randomization.