Oxytocin vs. Placebo for the Treatment Hyperphagia in Children and Adolescents With Prader-Willi Syndrome (OXT-PWS)

Montefiore Medicine Academic Health System

Status and phase

Completed

Phase 2

Conditions

Prader-Willi Syndrome

Hyperphagia

Treatments

Drug: Intranasal Oxytocin (IN-OXT)

Other: Matched Placebo

Study type

Interventional

Funder types

Other

Identifiers

NCT02629991

14-10-427-01

Details and patient eligibility

About

The investigators propose a randomized double-blind 8 week treatment trial of intranasal oxytocin (IN-OXT) vs. placebo in 24 subjects aged 5 to 18 years with PWS in order to assess IN-OXT's affect on (1) Eating behaviors (2) Repetitive and disruptive behaviors and (3) Salivary OXT levels.

Full description

The investigators propose to conduct a treatment study of intranasal oxytocin (IN-OXT) vs. placebo in children and adolescents with Prader-Willi Syndrome (PWS). OXT has already been proven safe and effective in a treatment study of socialization and disruptive behavior in adults with PWS and is being used in infants with PWS in an ongoing clinical trial. The investigators hypothesize that OXT will be superior to placebo and have a positive effect on child and adolescent PWS eating and repetitive behaviors. Additional knowledge of OXT's ability to reduce overeating could lead to improvement of patient's quality of life and physical health and reduction in familial stress.

The investigators propose a randomized double-blind 8 week treatment trial of intranasal OXT vs. placebo in 24 subjects aged 5 to 18 years with PWS in order to assess IN-OXT's affect on (1) Eating behaviors (2) Repetitive and disruptive behaviors and (3) Salivary OXT levels. If superior to placebo, this data will add to the current knowledge that OXT is an effective treatment for hyperphagia as well as other problematic symptomatology of PWS.

Enrollment

23 patients

Sex

All

Ages

5 to 18 years old

Volunteers

No Healthy Volunteers

Inclusion criteria

Male or Female child outpatients aged 5 to 18 years
Diagnosis of PWS confirmed by genetic testing and patient medical records and history
Stable pharmacologic, educational, behavioral and/or dietary interventions for 4 weeks prior to the study start, and for the duration of the study.
Have a physical exam and laboratory results that are within the norms for PWS
Have a parent/caregiver/guardian that is able to consent for their participation and complete assessments regarding the child's development and behavior improvement throughout the study.

Exclusion criteria

Exposure to any investigational agent in the 30 days prior to randomization
Prior chronic treatment with oxytocin.
A primary psychiatric diagnosis other than ASD, including bipolar disorder, psychosis, schizophrenia, PTSD or major depressive disorder. These patients will be excluded due to potential confounding results.
Pregnant or lactating patients. IN-OXT has not been studied in pregnant or lactating women.
A medical condition that might interfere with the conduct of the study, confound interpretation of study results or endanger their own well-being.
Plan to initiate or change nonpharmacologic or pharmacologic interventions during the course of the study.
Females using an estrogen-based contraceptive. As an alternative to an estrogen based contraceptive, subjects will be counseled to use progesterone-based contraceptives; cervical caps; cervical sponges; or spermicidal foam in combination with a condom. Subjects will need to use a double barrier method to be in the study.