Prader Willi SyndromePhase 1NCT02013258
Oxytocin Trial in Prader-Willi Syndrome
A Phase 1 study testing Intranasal oxytocin in people with Prader Willi Syndrome, run by University of Florida at 3 sites in United States.
Trial summary
Individuals with Prader-Willi syndrome (PWS) have been found to have a deficit of oxytocin-producing neurons and decreased oxytocin receptor gene function, so the purpose of this study is to determine if oxytocin (OT) administration will improve some of the aspects of Prader-Willi syndrome that are particularly… Read more about this study
- Who is it for?
- Ages 5 to 11 years
- Conditions the study is for
- Prader Willi Syndrome
- What is being tested?
- Intranasal oxytocin
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 24 participants
- Where
- 3 sitesUnited States
- Study dates
- Mar 2015to Aug 2015
- Phase
- Phase 1
- Are there results?
- None posted in the registry
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 5 to 11 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- Children with genetically confirmed PWS
- Ages at ≥ 5 years and ≤ 11 years (must start treatment prior to 11th birthday)
- Child must be in nutritional phase 2b or 3, as determined by the PI at each site.
- Must currently be on growth hormone treatment, and have been receiving growth hormone treatment for at least one year prior to screening date.
- Treatment cannot have been interrupted for more than 1 week within 3 months prior to screening date.
- Priority will be given to children currently enrolled in the RDCRN Natural History study
Who cannot join
- Inability to tolerate intranasal administration of medication
- Hepatic insufficiency (AST/ALT greater than 3 times the normal levels for age)
- Renal insufficiency (BUN/Creatinine greater than 3 times the normal levels for age)
- History of an abnormal ECG (as determined by a cardiologist). If there is any question about cardiac function, ECG reports will be reviewed with a cardiologist prior to enrollment in the study.
- Child not receiving growth hormone treatment
- Child with hypertension or hypotension for age and sex (blood pressure >97% for age and sex or blood pressure <3% for age and sex)
Show all 10Show fewer
- Diabetes mellitus
- Pregnant or lactating.
- Schizophrenia or psychosis
- Taking any psychotropic medications
Trial locations and contacts
3 sites in 1 country.
United States3 sites
University of Florida
Gainesville, Florida, 32610
University of Kansas Medical Center
Kansas City, Kansas, 66160
University of California, Irvine
Orange, California, 92686
About this study
Individuals with Prader-Willi syndrome (PWS) have been found to have a deficit of oxytocin-producing neurons and decreased oxytocin receptor gene function, so the purpose of this study is to determine if oxytocin (OT) administration will improve some of the aspects of Prader-Willi syndrome that are particularly troublesome for children and their families (the insatiable appetite and social behaviors).
The research questions are:
- Does intranasal oxytocin cause any side effects in children with PWS?
- Does intranasal oxytocin administration alter appetite or behaviors in PWS?
Read the detailed description
This study is to investigate if intranasal oxytocin will improve hyperphagia, social skills, and behaviors in subjects with Prader-Willi syndrome. This will be a randomized placebo controlled pilot study. The primary outcome measure is to determine if intranasal administration of oxytocin will cause any adverse events in subjects with Prader-Willi syndrome. Secondarily, the investigators will also perform evaluations to determine if intranasal oxytocin has any effect on social skills, behaviors, or appetite in children with Prader-Willi syndrome.
- Conditions
- Prader Willi Syndrome
- Being tested
- Intranasal oxytocin, Placebo
- Keywords
- Prader Willi syndrome, Oxytocin
- 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.
Intranasal oxytocinDrug
Also called Pitocin, Syntocinon
This is a double-blind placebo controlled 2x2 study. Subjects will receive OT for 5 consecutive days during their 7 day stay. This will be followed by a wash out period of 4-6 weeks.
PlaceboOther
This is a double-blind placebo controlled 2x2 study. Placebo will be given via intranasal spray, one spray in each nostril daily x 5 days. One month interval between arms of treatment.
| Study group | Role | What they receive |
|---|---|---|
| Intranasal oxytocin | Active comparator | Intranasal oxytocin. 16 IU intranasal oxytocin x 5 days. One month interval between arms of treatment. Receives: Drug: Intranasal oxytocin |
| Placebo | Placebo comparator | Placebo will be administered via nasal spray - 1 spray in each nostril x5 days. Receives: Other: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Safety of intranasal oxytocin in children with Prader-Willi syndrome
Occurrence of adverse event, description and quantification of clinical and behavior severity, pre- and post- intranasal oxytocin and placebo administration.
Measured: 3 months
Secondary outcomes (1)
Evaluation of food intake in Prader-Willi syndrome
Quantitative evaluation of hyperphagia via the Hyperphagia Questionnaire obtained on days 1, 4 and 6 during the 7 day study protocol and conducted in the evening on these days. Score will range from 0 (no hyperphagia behaviors) to 96 (most severe hyperphagia behaviors). Additionally quantity of food consumed will be recorded on Days 1,4, and 6.
Measured: 3 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted17 Dec 2013 (estimated)
- Study startMar 2015
- Primary completionAug 2015
- Study completionAug 2015
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- 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
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 24 participants (actual)
Sponsor and investigators
- Lead sponsor
- University of Florida
- Sponsor type
- University, hospital or other
- Collaborators
- National Institutes of Health (NIH)
- Responsible party
- Sponsor
- Investigators
- Jennifer L Miller, MD, Principal investigator, University of Florida
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02013258
- Sponsor's protocol ID
- 5208-N
- Status last verified
- Apr 2018
- First submitted
- 11 Dec 2013
- Last updated
- 5 Apr 2018
- Results posted
- No
- Data monitoring committee
- Yes
Publications
Tauber M, Mantoulan C, Copet P, Jauregui J, Demeer G, Diene G, Roge B, Laurier V, Ehlinger V, Arnaud C, Molinas C, Thuilleaux D. Oxytocin may be useful to increase trust in others and decrease disruptive behaviours in patients with Prader-Willi syndrome: a randomised placebo-controlled trial in 24 patients. Orphanet J Rare Dis. 2011 Jun 24;6:47. doi: 10.1186/1750-1172-6-47.
PubMed 21702900
Study at a glance
From the registry record, last updated by the sponsor on 5 Apr 2018. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Oxytocin Trial in Prader-Willi Syndrome
- ClinicalTrials.gov ID
- NCT02013258
- Acronym / sponsor ID
- 5208-N
- Conditions
- Prader Willi Syndrome
- Keywords
- Prader Willi syndrome, Oxytocin
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 24 participants (actual)
- Lead sponsor
- University of FloridaUniversity, hospital or other
- Collaborators
- National Institutes of Health (NIH)
- Study start
- Mar 2015
- Primary completion
- Aug 2015 (actual)
- Study completion
- Aug 2015 (actual)
- First posted
- 17 Dec 2013 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
Individuals with Prader-Willi syndrome (PWS) have been found to have a deficit of oxytocin-producing neurons and decreased oxytocin receptor gene function, so the purpose of this study is to determine if oxytocin (OT) administration will improve some of the aspects of Prader-Willi syndrome that are particularly troublesome for children and their families (the insatiable appetite and social behaviors).
The research questions are:
- Does intranasal oxytocin cause any side effects in children with PWS?
- Does intranasal oxytocin administration alter appetite or behaviors in PWS?
Detailed description
This study is to investigate if intranasal oxytocin will improve hyperphagia, social skills, and behaviors in subjects with Prader-Willi syndrome. This will be a randomized placebo controlled pilot study. The primary outcome measure is to determine if intranasal administration of oxytocin will cause any adverse events in subjects with Prader-Willi syndrome. Secondarily, the investigators will also perform evaluations to determine if intranasal oxytocin has any effect on social skills, behaviors, or appetite in children with Prader-Willi syndrome.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Intranasal oxytocin | Active comparator | Drug: Intranasal oxytocinIntranasal oxytocin. 16 IU intranasal oxytocin x 5 days. One month interval between arms of treatment. |
| Placebo | Placebo comparator | Other: PlaceboPlacebo will be administered via nasal spray - 1 spray in each nostril x5 days. |
Who may qualify
- Ages
- 5 to 11 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (6 listed)
- Children with genetically confirmed PWS
- Ages at ≥ 5 years and ≤ 11 years (must start treatment prior to 11th birthday)
- Child must be in nutritional phase 2b or 3, as determined by the PI at each site.
- Must currently be on growth hormone treatment, and have been receiving growth hormone treatment for at least one year prior to screening date.
- Treatment cannot have been interrupted for more than 1 week within 3 months prior to screening date.
Key exclusion criteria (10 listed)
- Inability to tolerate intranasal administration of medication
- Hepatic insufficiency (AST/ALT greater than 3 times the normal levels for age)
- Renal insufficiency (BUN/Creatinine greater than 3 times the normal levels for age)
- History of an abnormal ECG (as determined by a cardiologist). If there is any question about cardiac function, ECG reports will be reviewed with a cardiologist prior to enrollment in the study.
- Child not receiving growth hormone treatment
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 |
|---|---|---|
| Intranasal oxytocinAlso: Pitocin, SyntocinonThis is a double-blind placebo controlled 2x2 study. Subjects will receive OT for 5 consecutive days during their 7 day stay. This will be followed by a wash out period of 4-6 weeks. | Drug | Intranasal oxytocin |
| PlaceboThis is a double-blind placebo controlled 2x2 study. Placebo will be given via intranasal spray, one spray in each nostril daily x 5 days. One month interval between arms of treatment. | Other | Placebo |
Primary outcome
Safety of intranasal oxytocin in children with Prader-Willi syndrome
Time frame: 3 months
Occurrence of adverse event, description and quantification of clinical and behavior severity, pre- and post- intranasal oxytocin and placebo administration.
1 secondary outcome
Evaluation of food intake in Prader-Willi syndrome
Time frame: 3 months
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 5 Apr 2018.
- Background: PubMed 21702900Tauber M, Mantoulan C, Copet P, Jauregui J, Demeer G, Diene G, Roge B, Laurier V, Ehlinger V, Arnaud C, Molinas C, Thuilleaux D. Oxytocin may be useful to increase trust in others and decrease disruptive behaviours in patients with Prader-Willi syndrome: a randomised placebo-controlled trial in 24 patients. Orphanet J Rare Dis. 2011 Jun 24;6:47. doi: 10.1186/1750-1172-6-47.
- Oxytocin treatment in children with Prader-Willi syndrome: A double-blind, placebo-controlled, crossover study. Miller JL, Tamura R, Butler MG, Kimonis V, Sulsona C, Gold JA, Driscoll DJ. Am J Med Genet A. 2017 May;173(5):1243-1250.Link provided by the sponsor.
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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 L Miller, MD principal investigator
University of Florida
3 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing Intranasal oxytocin in people with Prader Willi Syndrome, run by University of Florida at 3 sites 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 6 inclusion and 10 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 5 Apr 2018. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 3 sites 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 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.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
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?
Safety of intranasal oxytocin in children with Prader-Willi syndrome (time frame: 3 months). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT02013258, which also lists 1 linked publication.
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 5 Apr 2018.