Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
| Name | Class |
|---|---|
| Foundation for Prader-Willi Research | OTHER |
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
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.
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.
Not provided
Not provided
Not provided
Not provided
| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Intranasal Oxytocin | Experimental | Participants were instructed to take 16 IU/per day everyday (2 puffs per nostril, 4 IU each puff). |
|
| Matched Placebo | Placebo Comparator | Participants were instructed to take 16 IU/per day everyday (2 puffs per nostril, 4 IU each puff). |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Intranasal Oxytocin (IN-OXT) | Drug | Participants were instructed to take 16 IU/per day everyday (2 puffs per nostril, 4 IU each puff). |
|
| Measure | Description | Time Frame |
|---|---|---|
| Hyperphagia Questionnaire (HQ)- Total Score | 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. | Assessed trends over multiple time points (weeks 0, 2, 4, 6, and 8), week 0 (baseline), week 4 and week 8 are reported. |
| Hyperphagia Questionnaire (HQ)- Behavior Factor Score | 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. | Assessed trends over multiple time points (weeks 0, 2, 4, 6, and 8), week 0 (baseline), week 4 and week 8 are reported. |
| Hyperphagia Questionnaire (HQ)- Drive Factor Score | 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 | Assessed trends over multiple time points (weeks 0, 2, 4, 6, and 8), week 0 (baseline), week 4 and week 8 are reported. |
| Hyperphagia Questionnaire (HQ) -Severity Factor Score | 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. | Assessed trends over multiple time points (weeks 0, 2, 4, 6, and 8), week 0 (baseline), week 4 and week 8 are reported. |
| Measure | Description | Time Frame |
|---|---|---|
| Repetitive Behavior Scale-Revised (RBS-R) Ritualistic Behavior Factor Score | 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. | Trends over multiple time points (Weeks 0, 4, and 8). |
Not provided
Inclusion Criteria:
Exclusion Criteria:
Not provided
Not provided
Not provided
Not provided
Not provided
| Name | Affiliation | Role |
|---|---|---|
| Eric Hollander, MD | Montefiore Medical Center | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Montefiore Medical Center, Albert Einstein College of Medicine | The Bronx | New York | 10467 | United States |
Not provided
| Label | URL |
|---|---|
| Foundation for Prader Willi Research | View source |
Not provided
Not provided
Not provided
Not provided
| ID | Title | Description |
|---|---|---|
| FG000 | Intranasal Oxytocin | Participants were instructed to take 16 IU/per day everyday (2 puffs per nostril, 4 IU each puff). |
| FG001 | Matched Placebo | Participants were instructed to take 16 IU/per day everyday (2 puffs per nostril, 4 IU each puff). |
| Title | Milestones | Reasons Not Completed | ||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Overall Study |
|
Not provided
Not provided
| ID | Title | Description |
|---|---|---|
| BG000 | Intranasal Oxytocin | Participants were instructed to take 16 IU/per day everyday (2 puffs per nostril, 4 IU each puff). |
| BG001 | Matched Placebo | Participants were instructed to take 16 IU/per day everyday (2 puffs per nostril, 4 IU each puff). |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes |
|---|---|---|---|---|---|---|---|---|---|
| Age, Continuous | Mean |
| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | |||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | Hyperphagia Questionnaire (HQ)- Total Score | 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. | 1 subject discontinued oxytocin early and 1 subject discontinued placebo early | Posted | Mean | Standard Deviation | score on a scale | Assessed trends over multiple time points (weeks 0, 2, 4, 6, and 8), week 0 (baseline), week 4 and week 8 are reported. |
|
8 Weeks
Not provided
Not provided
| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | Intranasal Oxytocin (IN-OXT) | Participants were instructed to take 16 IU/per day everyday (2 puffs per nostril, 4 IU each puff). |
Not provided
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Fatigue | General disorders | Systematic Assessment |
Not provided
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Bonnie Taylor | Albert Einstein College of Medicine | 7188397530 | botaylor@montefiore.org |
Not provided
| ID | Term |
|---|---|
| D011218 | Prader-Willi Syndrome |
| D006963 | Hyperphagia |
| ID | Term |
|---|---|
| D008607 | Intellectual Disability |
| D019954 | Neurobehavioral Manifestations |
| D009461 | Neurologic Manifestations |
| D009422 | Nervous System Diseases |
Not provided
Not provided
| ID | Term |
|---|---|
| D010121 | Oxytocin |
| ID | Term |
|---|---|
| D010909 | Pituitary Hormones, Posterior |
| D010907 | Pituitary Hormones |
| D036361 | Peptide Hormones |
| D006728 | Hormones |
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
| Matched Placebo | Other | Participants were instructed to take 16 IU/per day everyday (2 puffs per nostril, 4 IU each puff). |
|
| Repetitive Behavior Scale-Revised (RBS-R) Sameness Factor Score | 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. | Trends over multiple time points (Weeks 0, 4, and 8). |
| Repetitive Behavior Scale-Revised (RBS-R) Stereotyped Behavior Factor Score | 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. | Trends over multiple time points (Weeks 0, 4, and 8). |
| Repetitive Behavior Scale-Revised (RBS-R) Compulsive Behavior Factor Score | 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. | Trends over multiple time points (Weeks 0, 4, and 8). |
| Repetitive Behavior Scale-Revised (RBS-R) Self-Injurious Factor Score | 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. | Trends over multiple time points (Weeks 0, 4, and 8). |
| Repetitive Behavior Scale-Revised (RBS-R) Restricted Interest Factor Score | 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. | Trends over multiple time points (Weeks 0, 4, and 8). |
| BG002 | Total | Total of all reporting groups |
| years |
|
| Sex: Female, Male | Count of Participants | Participants |
|
| Ethnicity (NIH/OMB) | Count of Participants | Participants |
|
| Race (NIH/OMB) | Count of Participants | Participants |
|
| Baseline Height (cm) | Mean | Standard Deviation | cm |
|
| Baseline Weight (kg) | Mean | Standard Deviation | kg |
|
| Baseline BMI | Mean | Standard Deviation | kg/m^2 |
|
| Hyperphagia Questionnaire (HQ)-Total | Mean | Standard Deviation | score on a scale |
|
| OG001 | Matched Placebo | Participants were instructed to take 16 IU/per day everyday (2 puffs per nostril, 4 IU each puff). |
|
|
|
| Primary | Hyperphagia Questionnaire (HQ)- Behavior Factor Score | 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. | 1 subject discontinued oxytocin early and 1 subject discontinued placebo early | Posted | Mean | Standard Deviation | score on a scale | Assessed trends over multiple time points (weeks 0, 2, 4, 6, and 8), week 0 (baseline), week 4 and week 8 are reported. |
|
|
|
|
| Primary | Hyperphagia Questionnaire (HQ)- Drive Factor Score | 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 | 1 subject discontinued oxytocin early and 1 subject discontinued placebo early | Posted | Mean | Standard Deviation | score on a scale | Assessed trends over multiple time points (weeks 0, 2, 4, 6, and 8), week 0 (baseline), week 4 and week 8 are reported. |
|
|
|
|
| Primary | Hyperphagia Questionnaire (HQ) -Severity Factor Score | 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. | 1 subject discontinued oxytocin early and 1 subject discontinued placebo early | Posted | Mean | Standard Deviation | score on a scale | Assessed trends over multiple time points (weeks 0, 2, 4, 6, and 8), week 0 (baseline), week 4 and week 8 are reported. |
|
|
|
|
| Secondary | Repetitive Behavior Scale-Revised (RBS-R) Ritualistic Behavior Factor Score | 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. | 1 subject discontinued oxytocin early and 1 subject discontinued placebo early | Posted | Mean | Standard Error | score on a scale | Trends over multiple time points (Weeks 0, 4, and 8). |
|
|
|
|
| Secondary | Repetitive Behavior Scale-Revised (RBS-R) Sameness Factor Score | 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. | 1 subject discontinued oxytocin early and 1 subject discontinued placebo early | Posted | Mean | Standard Deviation | score on a scale | Trends over multiple time points (Weeks 0, 4, and 8). |
|
|
|
|
| Secondary | Repetitive Behavior Scale-Revised (RBS-R) Stereotyped Behavior Factor Score | 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. | 1 subject discontinued oxytocin early and 1 subject discontinued placebo early | Posted | Mean | Standard Deviation | score on a scale | Trends over multiple time points (Weeks 0, 4, and 8). |
|
|
|
|
| Secondary | Repetitive Behavior Scale-Revised (RBS-R) Compulsive Behavior Factor Score | 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. | 1 subject discontinued oxytocin early and 1 subject discontinued placebo early | Posted | Mean | Standard Deviation | score on a scale | Trends over multiple time points (Weeks 0, 4, and 8). |
|
|
|
|
| Secondary | Repetitive Behavior Scale-Revised (RBS-R) Self-Injurious Factor Score | 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. | 1 subject discontinued oxytocin early and 1 subject discontinued placebo early | Posted | Mean | Standard Deviation | score on a scale | Trends over multiple time points (Weeks 0, 4, and 8). |
|
|
|
|
| Secondary | Repetitive Behavior Scale-Revised (RBS-R) Restricted Interest Factor Score | 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. | 1 subject discontinued oxytocin early and 1 subject discontinued placebo early | Posted | Mean | Standard Error | score on a scale | Trends over multiple time points (Weeks 0, 4, and 8). |
|
|
|
|
| 0 |
| 11 |
| 0 |
| 11 |
| 7 |
| 11 |
| EG001 | Matched Placebo | Participants were instructed to take 16 IU/per day everyday (2 puffs per nostril, 4 IU each puff). | 0 | 12 | 0 | 12 | 7 | 12 |
| Increased Thirst | General disorders | Systematic Assessment |
|
| Nocturia | Renal and urinary disorders | Systematic Assessment |
|
| Insomnia | Psychiatric disorders | Systematic Assessment |
|
| Increased cataplexy | Nervous system disorders | Systematic Assessment |
|
| Increased movements, involuntary | Nervous system disorders | Systematic Assessment |
|
| Headache | Nervous system disorders | Systematic Assessment |
|
| Irritability | Psychiatric disorders | Systematic Assessment |
|
| Dizziness | Nervous system disorders | Systematic Assessment |
|
| Epistaxis (nose bleed) | Respiratory, thoracic and mediastinal disorders | Systematic Assessment |
|
| Fever | General disorders | Systematic Assessment |
|
| Scraped Knee | General disorders | Systematic Assessment |
|
| Stomach Virus | General disorders | Systematic Assessment |
|
| Altitute Sickness | General disorders | Systematic Assessment |
|
| Shingles | Infections and infestations | Systematic Assessment |
|
| Upper Respiratory Infection | Infections and infestations | Systematic Assessment |
|
| Rhinitis Infective | Infections and infestations | Systematic Assessment |
|
| Pneumonia | Infections and infestations | Systematic Assessment |
|
| Sinusitis | Infections and infestations | Systematic Assessment |
|
| Cough | Respiratory, thoracic and mediastinal disorders | Systematic Assessment |
|
| Bronchitis | Respiratory, thoracic and mediastinal disorders | Systematic Assessment |
|
| Otitis | Infections and infestations | Systematic Assessment |
|
| Dry Skin | Skin and subcutaneous tissue disorders | Systematic Assessment |
|
| Worsening eczema | Skin and subcutaneous tissue disorders | Systematic Assessment |
|
| Dermatitis | Skin and subcutaneous tissue disorders | Systematic Assessment |
|
Not provided
Not provided
| D000015 | Abnormalities, Multiple |
| D000013 | Congenital Abnormalities |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D025063 | Chromosome Disorders |
| D030342 | Genetic Diseases, Inborn |
| D000096803 | Imprinting Disorders |
| D009765 | Obesity |
| D050177 | Overweight |
| D044343 | Overnutrition |
| D009748 | Nutrition Disorders |
| D009750 | Nutritional and Metabolic Diseases |
| D012817 | Signs and Symptoms, Digestive |
| D012816 | Signs and Symptoms |
| D013568 | Pathological Conditions, Signs and Symptoms |
| D006730 |
| Hormones, Hormone Substitutes, and Hormone Antagonists |
| D010455 | Peptides |
| D000602 | Amino Acids, Peptides, and Proteins |
| Week 4 |
|
|
| Week 8 |
|
|
| Week 4 |
|
|
| Week 8 |
|
|
| Week 4 |
|
|
| Week 8 |
|
|
| Week 4 |
|
|
| Week 8 |
|
|
| Week 4 |
|
|
| Week 8 |
|
|
| Week 4 |
|
|
| Week 8 |
|
|
| Week 4 |
|
|
| Week 8 |
|
|
| Week 4 |
|
|
| Week 8 |
|
|
| Week 4 |
|
|
| Week 8 |
|
|