Prader-Willi SyndromePhase 1/2NCT03245762
Intranasal Oxytocin for Infants With Prader-Willi Syndrome
A Phase 1/2 study testing Oxytocin in people with Prader-Willi Syndrome, run by University of Florida at 1 site in United States.
Trial 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. Read more about this study
- Who is it for?
- Ages 1 week to 6 months
- Conditions the study is for
- Prader-Willi Syndrome
- What is being tested?
- Oxytocin
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 15 participants
- Where
- 1 siteUnited States
- Study dates
- 1 Aug 2017to 4 Jan 2018
- Phase
- Phase 1/2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 1 week to 6 months
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- Individuals with genetically confirmed PWS who are in nutritional phase 1a, as determined by PI
- Physical exam and laboratory results that are within the normal range.
- Presence of a parent/caregiver/guardian that is able to consent for their participation.
Who cannot join
- Exposure to any investigational agent in the 30 days prior to randomization.
- Prior chronic treatment with oxytocin.
- A medical condition that might interfere with the conduct of the study, confound interpretation of study results or endanger the subject's well-being.
Trial locations and contacts
1 site in 1 country.
About this study
Official title: Intranasal Oxytocin for Treatment of Infants and Children With Prader-Willi Syndrome in Nutritional Phase 1a - Phase 2 Study
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.
Read the 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.
- Conditions
- Prader-Willi Syndrome
- Being tested
- Oxytocin, Placebo
- Medical subject headings
- Prader-Willi Syndrome
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
OxytocinDrug
Also called Syntocinon
4 IU/day of oxytocin administered via nasal spray device each morning.
PlaceboDrug
Also called Saline
4 IU/day of placebo administered via nasal spray device each morning
| Study group | Role | What they receive |
|---|---|---|
| Intranasal oxytocin | Active comparator | Intervention: 4 IU/day of intranasal oxytocin via a nasal spray device each morning. Receives: Drug: Oxytocin |
| IN-placebo | Placebo comparator | Intervention: 4 IU/day of placebo via nasal spray device each morning. Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Suck and Swallow Competency in Infants/Children With PWS Who Are in Nutritional Phase 1a
Swallow study Overall improvement
Measured: baseline to day 5
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted10 Aug 2017
- Study start1 Aug 2017
- Primary completion4 Jan 2018
- Study completion4 Jan 2018
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Prader-Willi SyndromeStudies of the same condition, open and under way.
- More trials run by University of FloridaThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1/2
- Primary purpose
- Basic science
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind
- Who is masked
- Care provider, Outcomes assessor
- Enrolment
- 15 participants (actual)
Sponsor and investigators
- Lead sponsor
- University of Florida
- Sponsor type
- University, hospital or other
- Collaborators
- Prader-Willi Syndrome Association
- Responsible party
- Sponsor
- Investigators
- Jennifer Miller, MD, Principal investigator, University of Florida
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03245762
- Sponsor's protocol ID
- IRB201700423 -A
- Other IDs
- OCR16237
- Status last verified
- Mar 2020
- First submitted
- 7 Aug 2017
- Last updated
- 26 Mar 2020
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Undecided
Study at a glance
From the registry record, last updated by the sponsor on 26 Mar 2020. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Intranasal Oxytocin for Treatment of Infants and Children With Prader-Willi Syndrome in Nutritional Phase 1a - Phase 2 Study
- ClinicalTrials.gov ID
- NCT03245762
- Acronym / sponsor ID
- IRB201700423 -A
- Conditions
- Prader-Willi Syndrome
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 15 participants (actual)
- Lead sponsor
- University of FloridaUniversity, hospital or other
- Collaborators
- Prader-Willi Syndrome Association
- Study start
- 1 Aug 2017 (actual)
- Primary completion
- 4 Jan 2018 (actual)
- Study completion
- 4 Jan 2018 (actual)
- First posted
- 10 Aug 2017 (actual)
- Results
- Posted 25 Jun 2019
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Basic science
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind (care provider, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Intranasal oxytocin | Active comparator | Drug: OxytocinIntervention: 4 IU/day of intranasal oxytocin via a nasal spray device each morning. |
| IN-placebo | Placebo comparator | Drug: PlaceboIntervention: 4 IU/day of placebo via nasal spray device each morning. |
Who may qualify
- Ages
- 1 week to 6 months
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (3 listed)
- Individuals with genetically confirmed PWS who are in nutritional phase 1a, as determined by PI
- Physical exam and laboratory results that are within the normal range.
- Presence of a parent/caregiver/guardian that is able to consent for their participation.
Key exclusion criteria (3 listed)
- Exposure to any investigational agent in the 30 days prior to randomization.
- Prior chronic treatment with oxytocin.
- A medical condition that might interfere with the conduct of the study, confound interpretation of study results or endanger the subject's well-being.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| OxytocinAlso: Syntocinon4 IU/day of oxytocin administered via nasal spray device each morning. | Drug | Intranasal oxytocin |
| PlaceboAlso: Saline4 IU/day of placebo administered via nasal spray device each morning | Drug | IN-placebo |
Primary outcome
Suck and Swallow Competency in Infants/Children With PWS Who Are in Nutritional Phase 1a
Time frame: baseline to day 5
Swallow study Overall improvement
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 26 Mar 2020.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Individual participant dataSharing planned: Undecided
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Jennifer Miller, MD principal investigator
University of Florida
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing Oxytocin in people with Prader-Willi Syndrome, run by University of Florida at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 3 inclusion and 3 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 26 Mar 2020. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 25 Jun 2019). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Suck and Swallow Competency in Infants/Children With PWS Who Are in Nutritional Phase 1a (time frame: baseline to day 5). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 26 Mar 2020; AzTrials last refreshed its copy on 7 Nov 2026.