Prader-Willi Syndrome
Conditions
Brief summary
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.
Detailed description
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.
Interventions
DRUGOxytocin
4 IU/day of oxytocin administered via nasal spray device each morning.
DRUGPlacebo
4 IU/day of placebo administered via nasal spray device each morning
Sponsors
Prader-Willi Syndrome Association
University of Florida
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
BASIC_SCIENCE
Masking
DOUBLE (Caregiver, Outcomes Assessor)
Masking description
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.
Eligibility
Sex/Gender
ALL
Age
1 Weeks to 6 Months
Healthy volunteers
No
Inclusion criteria
1. Individuals with genetically confirmed PWS who are in nutritional phase 1a, as determined by PI 2. Physical exam and laboratory results that are within the normal range. 3. Presence of a parent/caregiver/guardian that is able to consent for their participation.
Exclusion criteria
1. Exposure to any investigational agent in the 30 days prior to randomization. 2. Prior chronic treatment with oxytocin. 3. A medical condition that might interfere with the conduct of the study, confound interpretation of study results or endanger the subject's well-being.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Suck and Swallow Competency in Infants/Children With PWS Who Are in Nutritional Phase 1a | baseline to day 5 | Swallow study Overall improvement |
Countries
United States
Participant flow
Participants by arm
| Arm | Count |
|---|---|
| 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. | 10 |
| 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 | 5 |
| Total | 15 |
Baseline characteristics
| Characteristic | Intranasal Oxytocin | IN-placebo | Total |
|---|---|---|---|
| Age, Categorical <=18 years | 10 Participants | 5 Participants | 15 Participants |
| Age, Categorical >=65 years | 0 Participants | 0 Participants | 0 Participants |
| Age, Categorical Between 18 and 65 years | 0 Participants | 0 Participants | 0 Participants |
| Ethnicity (NIH/OMB) Hispanic or Latino | 2 Participants | 0 Participants | 2 Participants |
| Ethnicity (NIH/OMB) Not Hispanic or Latino | 8 Participants | 5 Participants | 13 Participants |
| Ethnicity (NIH/OMB) Unknown or Not Reported | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) American Indian or Alaska Native | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) Asian | 1 Participants | 1 Participants | 2 Participants |
| Race (NIH/OMB) Black or African American | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) More than one race | 2 Participants | 0 Participants | 2 Participants |
| Race (NIH/OMB) Native Hawaiian or Other Pacific Islander | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) Unknown or Not Reported | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) White | 7 Participants | 4 Participants | 11 Participants |
| Region of Enrollment Europe | 1 participants | 1 participants | 2 participants |
| Region of Enrollment United States | 9 participants | 4 participants | 13 participants |
| Sex: Female, Male Female | 7 Participants | 2 Participants | 9 Participants |
| Sex: Female, Male Male | 3 Participants | 3 Participants | 6 Participants |
Adverse events
| Event type | EG000 affected / at risk | EG001 affected / at risk |
|---|---|---|
| deaths Total, all-cause mortality | 0 / 10 | 0 / 5 |
| other Total, other adverse events | 0 / 10 | 0 / 5 |
| serious Total, serious adverse events | 0 / 10 | 0 / 5 |
Outcome results
Primary
Suck and Swallow Competency in Infants/Children With PWS Who Are in Nutritional Phase 1a
Swallow study Overall improvement
Time frame: baseline to day 5
| Arm | Measure | Group | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|---|
| Intranasal Oxytocin | Suck and Swallow Competency in Infants/Children With PWS Who Are in Nutritional Phase 1a | Improved Competency | 8 Participants |
| Intranasal Oxytocin | Suck and Swallow Competency in Infants/Children With PWS Who Are in Nutritional Phase 1a | No Change in Competency' | 1 Participants |
| Intranasal Oxytocin | Suck and Swallow Competency in Infants/Children With PWS Who Are in Nutritional Phase 1a | Worsened Competency | 1 Participants |
| IN-placebo | Suck and Swallow Competency in Infants/Children With PWS Who Are in Nutritional Phase 1a | Improved Competency | 4 Participants |
| IN-placebo | Suck and Swallow Competency in Infants/Children With PWS Who Are in Nutritional Phase 1a | No Change in Competency' | 1 Participants |
| IN-placebo | Suck and Swallow Competency in Infants/Children With PWS Who Are in Nutritional Phase 1a | Worsened Competency | 0 Participants |
p-value: 0.93Ordinal Categorical Analysis