Clinical trial NCT02629991: Oxytocin vs. Placebo for the Treatment Hyperphagia in Children and Adolescents With Prader-Willi Syndrome
Official title: Oxytocin vs. Placebo for the Treatment Hyperphagia in Children and Adolescents With Prader-Willi Syndrome
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Prader-Willi Syndrome
Hyperphagia
Keywords: Prader-Willi Syndrome (PWS)
Hyperphagia
Lead sponsor: Montefiore Medical Center
Sponsor class: OTHER
Collaborators: name: Foundation for Prader-Willi Research
First posted: 2015-12-15
Enrollment: 23
Sex: ALL
Minimum age (years): 5
Maximum age (years): 18
Standard ages: CHILD
ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief summary: 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.

Detailed 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.

Eligibility criteria: Inclusion Criteria:

1. Male or Female child outpatients aged 5 to 18 years
2. Diagnosis of PWS confirmed by genetic testing and patient medical records and history
3. Stable pharmacologic, educational, behavioral and/or dietary interventions for 4 weeks prior to the study start, and for the duration of the study.
4. Have a physical exam and laboratory results that are within the norms for PWS
5. 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:

1. Exposure to any investigational agent in the 30 days prior to randomization
2. Prior chronic treatment with oxytocin.
3. 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.
4. Pregnant or lactating patients. IN-OXT has not been studied in pregnant or lactating women.
5. A medical condition that might interfere with the conduct of the study, confound interpretation of study results or endanger their own well-being.
6. Plan to initiate or change nonpharmacologic or pharmacologic interventions during the course of the study.
7. 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.

Primary outcomes:
measure: Hyperphagia Questionnaire (HQ)- Total Score
timeFrame: Assessed trends over multiple time points (weeks 0, 2, 4, 6, and 8), week 0 (baseline), week 4 and week 8 are reported.
measure: Hyperphagia Questionnaire (HQ)- Behavior Factor Score
timeFrame: Assessed trends over multiple time points (weeks 0, 2, 4, 6, and 8), week 0 (baseline), week 4 and week 8 are reported.
measure: Hyperphagia Questionnaire (HQ)- Drive Factor Score
timeFrame: Assessed trends over multiple time points (weeks 0, 2, 4, 6, and 8), week 0 (baseline), week 4 and week 8 are reported.
measure: Hyperphagia Questionnaire (HQ) -Severity Factor Score
timeFrame: Assessed trends over multiple time points (weeks 0, 2, 4, 6, and 8), week 0 (baseline), week 4 and week 8 are reported.
Secondary outcomes:
measure: Repetitive Behavior Scale-Revised (RBS-R) Ritualistic Behavior Factor Score
timeFrame: Trends over multiple time points (Weeks 0, 4, and 8).
measure: Repetitive Behavior Scale-Revised (RBS-R) Sameness Factor Score
timeFrame: Trends over multiple time points (Weeks 0, 4, and 8).
measure: Repetitive Behavior Scale-Revised (RBS-R) Stereotyped Behavior Factor Score
timeFrame: Trends over multiple time points (Weeks 0, 4, and 8).
measure: Repetitive Behavior Scale-Revised (RBS-R) Compulsive Behavior Factor Score
timeFrame: Trends over multiple time points (Weeks 0, 4, and 8).
measure: Repetitive Behavior Scale-Revised (RBS-R) Self-Injurious Factor Score
timeFrame: Trends over multiple time points (Weeks 0, 4, and 8).
measure: Repetitive Behavior Scale-Revised (RBS-R) Restricted Interest Factor Score
timeFrame: Trends over multiple time points (Weeks 0, 4, and 8).
Other outcomes:
Locations:
city: Bronx
country: United States
facility: Montefiore Medical Center, Albert Einstein College of Medicine
state: New York
Study officials:
affiliation: Montefiore Medical Center
name: Eric Hollander, MD
role: PRINCIPAL_INVESTIGATOR
References: