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.
Brief Title
Oxytocin vs. Placebo for the Treatment Hyperphagia in Children and Adolescents With Prader-Willi Syndrome
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.
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.
Categories
Completion Date
Completion Date Type
Actual
Conditions
Prader-Willi Syndrome
Hyperphagia
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.
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.
Inclusion 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.
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.
Gender
All
Gender Based
false
Keywords
Prader-Willi Syndrome (PWS)
Hyperphagia
Healthy Volunteers
No
Last Update Post Date
Last Update Post Date Type
Actual
Last Update Submit Date
Maximum Age
18 Years
Minimum Age
5 Years
NCT Id
NCT02629991
Org Class
Other
Org Full Name
Montefiore Medical Center
Org Study Id
14-10-427-01
Overall Status
Completed
Phases
Phase 2
Primary Completion Date
Primary Completion Date Type
Actual
Official Title
Oxytocin vs. Placebo for the Treatment Hyperphagia in Children and Adolescents With Prader-Willi Syndrome
Primary Outcomes
Outcome Description
The Hyperphagia Questionnaire (HQ)- Total Score. The HQ is a 11-item clinician-rated instrument that was designed to measure food related behaviors in PWS. Items map onto 3 subscales; Hyperphagic Behavior, Hyperphagic Drive, and Hyperphagia Severity. The subscales are summed together to compute the Total Score. The Total Score ranges from 11-55, with higher scores indicating more hyperphagia and a worse outcome.
Outcome Measure
Hyperphagia Questionnaire (HQ)- Total Score
Outcome Time Frame
Assessed trends over multiple time points (weeks 0, 2, 4, 6, and 8), week 0 (baseline), week 4 and week 8 are reported.
Outcome Description
The Hyperphagia Questionnaire (HQ)- Behavior Factor Score assesses food seeking behaviors (e.g., stealing food). The Behavior Factor Score ranges from 5-25, with higher scores indicating more hyperphagic behaviors and a worse outcome.
Outcome Measure
Hyperphagia Questionnaire (HQ)- Behavior Factor Score
Outcome Time Frame
Assessed trends over multiple time points (weeks 0, 2, 4, 6, and 8), week 0 (baseline), week 4 and week 8 are reported.
Outcome Description
The Hyperphagia Questionnaire (HQ)- Drive Factor Score assesses the persistence in asking for food. The Drive Factor Score ranges from 4-20, with higher scores indicating higher hyperphagic drive and a worse outcome
Outcome Measure
Hyperphagia Questionnaire (HQ)- Drive Factor Score
Outcome Time Frame
Assessed trends over multiple time points (weeks 0, 2, 4, 6, and 8), week 0 (baseline), week 4 and week 8 are reported.
Outcome Description
The Hyperphagia Questionnaire (HQ)- Severity Factor Score assesses the severity of hyperphagia. The Severity Factor Score ranges from 2-10, with higher scores indicating higher severity.
Outcome Measure
Hyperphagia Questionnaire (HQ) -Severity Factor Score
Outcome Time Frame
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
Outcome Description
The Repetitive Behavior Scale-Revised (RBS-R) Ritualistic Behavior Factor Score assesses ritualistic behaviors. Scores range from 0-18, with higher scores indicating more ritualistic behaviors and a worse outcome.
Outcome Time Frame
Trends over multiple time points (Weeks 0, 4, and 8).
Outcome Measure
Repetitive Behavior Scale-Revised (RBS-R) Ritualistic Behavior Factor Score
Outcome Description
The Repetitive Behavior Scale-Revised (RBS-R) Sameness Factor Score assesses the need for sameness in the environment. Scores range from 0-33, with higher scores indicating more need for sameness and a worse outcome.
Outcome Time Frame
Trends over multiple time points (Weeks 0, 4, and 8).
Outcome Measure
Repetitive Behavior Scale-Revised (RBS-R) Sameness Factor Score
Outcome Description
The Repetitive Behavior Scale-Revised (RBS-R) Stereotyped Behavior Factor Score assesses stereotyped behaviors. Scores range from 0-18, with higher scores indicating more stereotyped behaviors and a worse outcome.
Outcome Time Frame
Trends over multiple time points (Weeks 0, 4, and 8).
Outcome Measure
Repetitive Behavior Scale-Revised (RBS-R) Stereotyped Behavior Factor Score
Outcome Description
The Repetitive Behavior Scale-Revised (RBS-R) Compulsive Behavior Factor Score assesses compulsive behaviors. Scores range from 0-24, with higher scores indicating more compulsive behaviors and a worse outcome.
Outcome Time Frame
Trends over multiple time points (Weeks 0, 4, and 8).
Outcome Measure
Repetitive Behavior Scale-Revised (RBS-R) Compulsive Behavior Factor Score
Outcome Description
The Repetitive Behavior Scale-Revised (RBS-R) Self-Injurious Factor Score assesses self-injurious behaviors. Scores range from 0-24, with higher scores indicating more self-injurious behaviors and a worse outcome.
Outcome Time Frame
Trends over multiple time points (Weeks 0, 4, and 8).
Outcome Measure
Repetitive Behavior Scale-Revised (RBS-R) Self-Injurious Factor Score
Outcome Description
The Repetitive Behavior Scale-Revised (RBS-R) Restricted Interest Factor Score assesses restricted interests. Scores range from 0-12, with higher scores indicating more restricted interests and a worse outcome.
Outcome Time Frame
Trends over multiple time points (Weeks 0, 4, and 8).
Outcome Measure
Repetitive Behavior Scale-Revised (RBS-R) Restricted Interest Factor Score
See Also Links
Start Date
Status Verified Date
First Post Date
First Post Date Type
Estimated
First Submit Date
First Submit QC Date
Std Ages
Child
Adult
Maximum Age Number (converted to Years and rounded down)
18
Minimum Age Number (converted to Years and rounded down)
5
Investigators
Investigator Type
Principal Investigator
Investigator Name
Eric Hollander
Investigator Email
eholland@montefiore.org
Investigator Phone
718-920-4287