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| ID | Type | Description | Link |
|---|---|---|---|
| OCR16237 | Other Identifier | University of Florida |
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| Name | Class |
|---|---|
| Prader-Willi Syndrome Association | OTHER |
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The purpose of this study is to compare the change in suck and swallow competency from baseline to morning of day 6 with intranasal oxytocin spray vs placebo in infants/children with Prader-Willi Syndrome who are in nutritional phase 1a. Videofluoroscopic swallow studies will be performed on treatment day 1 and on the day following treatment morning of day 6.
The overall objective of this Phase 2 trial is to compare the change from baseline to morning of day 6 of Internasal Oxytocin (IN-OT) on suck and swallow competency in infants/children with Prader-Willi Syndrome (PWS) who are in nutritional phase 1a.
Study Hypothesis 1: The Study team hypothesize that replacing Oxytocin (OT) in infants and children who are in nutritional phase 1a will improve their suck and swallow, potentially even eliminating the need for gastrostomy tubes and nasogastric tubes for feeding, and decreasing the risk of aspiration with oral feeding.
Study Hypothesis 2: The Study team hypothesize that replacing OT in infants and children with PWS will result in improved eye contact, daytime alertness, and feelings of bonding between the parents and the infant.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Intranasal oxytocin | Active Comparator | Intervention: 4 IU/day of intranasal oxytocin via a nasal spray device each morning. |
|
| IN-placebo | Placebo Comparator | Intervention: 4 IU/day of placebo via nasal spray device each morning. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Oxytocin | Drug | 4 IU/day of oxytocin administered via nasal spray device each morning. |
|
| Measure | Description | Time Frame |
|---|---|---|
| Suck and Swallow Competency in Infants/Children With PWS Who Are in Nutritional Phase 1a | Swallow study Overall improvement | baseline to day 5 |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Jennifer Miller, MD | University of Florida | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| University of Florida | Gainesville | Florida | 32610 | United States |
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| ID | Title | Description |
|---|---|---|
| FG000 | Intranasal Oxytocin | Intervention: 4 IU/day of intranasal oxytocin via a nasal spray device each morning. Oxytocin: 4 IU/day of oxytocin administered via nasal spray device each morning. |
| FG001 | IN-placebo | Intervention: 4 IU/day of placebo via nasal spray device each morning. Placebo: 4 IU/day of placebo administered via nasal spray device each morning |
| Title | Milestones | Reasons Not Completed | ||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Overall Study |
|
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| ID | Title | Description |
|---|---|---|
| BG000 | Intranasal Oxytocin | Intervention: 4 IU/day of intranasal oxytocin via a nasal spray device each morning. Oxytocin: 4 IU/day of oxytocin administered via nasal spray device each morning. |
| BG001 | IN-placebo |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes |
|---|---|---|---|---|---|---|---|---|---|
| Age, Categorical | Count of Participants |
| 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 | Suck and Swallow Competency in Infants/Children With PWS Who Are in Nutritional Phase 1a | Swallow study Overall improvement | Posted | Count of Participants | Participants | baseline to day 5 |
|
7 days
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| 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 | Intervention: 4 IU/day of intranasal oxytocin via a nasal spray device each morning. Oxytocin: 4 IU/day of oxytocin administered via nasal spray device each morning. |
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Short study with small number of participants
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Jennifer Miller, MD | University of Florida | 352-265-7337 | millejl@peds.ufl.edu |
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| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| Prot_SAP | Yes | Yes | No | Study Protocol and Statistical Analysis Plan | Aug 17, 2017 | Jan 2, 2019 | Prot_SAP_000.pdf |
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| ID | Term |
|---|---|
| D011218 | Prader-Willi Syndrome |
| ID | Term |
|---|---|
| D008607 | Intellectual Disability |
| D019954 | Neurobehavioral Manifestations |
| D009461 | Neurologic Manifestations |
| D009422 | Nervous System Diseases |
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| ID | Term |
|---|---|
| D010121 | Oxytocin |
| D012965 | Sodium Chloride |
| ID | Term |
|---|---|
| D010909 | Pituitary Hormones, Posterior |
| D010907 | Pituitary Hormones |
| D036361 | Peptide Hormones |
| D006728 | Hormones |
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This will be a double-blinded study, as no studies to date have compared this treatment in infants with placebo. Board-certified speech and language pathologist who will be blinded to the treatment arm of each patient.
| Placebo | Drug | 4 IU/day of placebo administered via nasal spray device each morning |
|
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Intervention: 4 IU/day of placebo via nasal spray device each morning.
Placebo: 4 IU/day of placebo administered via nasal spray device each morning
| BG002 | Total | Total of all reporting groups |
| Participants |
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| Sex: Female, Male | Count of Participants | Participants |
|
| Ethnicity (NIH/OMB) | Count of Participants | Participants |
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| Race (NIH/OMB) | Count of Participants | Participants |
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| Region of Enrollment | Number | participants |
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| Units | Counts |
|---|---|
| Participants |
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| 0 |
| 10 |
| 0 |
| 10 |
| 0 |
| 10 |
| EG001 | IN-placebo | Intervention: 4 IU/day of placebo via nasal spray device each morning. Placebo: 4 IU/day of placebo administered via nasal spray device each morning | 0 | 5 | 0 | 5 | 0 | 5 |
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| 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 |
| D006730 |
| Hormones, Hormone Substitutes, and Hormone Antagonists |
| D010455 | Peptides |
| D000602 | Amino Acids, Peptides, and Proteins |
| D002712 | Chlorides |
| D006851 | Hydrochloric Acid |
| D017606 | Chlorine Compounds |
| D007287 | Inorganic Chemicals |
| D017670 | Sodium Compounds |