Prader-Willi SyndromePhase 2OXT-PWSNCT02629991

Oxytocin vs. Placebo for the Treatment Hyperphagia in Children and Adolescents With Prader-Willi Syndrome

A Phase 2 study testing Intranasal Oxytocin (IN-OXT) in people with Prader-Willi Syndrome and Hyperphagia, run by Montefiore Medical Center at 1 site in United States.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record

Search all trials

Trial summary

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. Read more about this study

Who is it for?: Ages 5 to 18 years
Conditions the study is for: Prader-Willi Syndrome, Hyperphagia
What is being tested?: Intranasal Oxytocin (IN-OXT)
Could I receive a placebo?: Yes, some participants may receive a placebo
How many people: 23 participants
Where: 1 siteUnited States
Study dates: Oct 2015to May 2018
Phase: Phase 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 5 to 18 years
All sexes
No healthy volunteers
Child, Adult

Who may be able to join

Male or Female child outpatients aged 5 to 18 years
Diagnosis of PWS confirmed by genetic testing and patient medical records and history
Stable pharmacologic, educational, behavioral and/or dietary interventions for 4 weeks prior to the study start, and for the duration of the study.
Have a physical exam and laboratory results that are within the norms for PWS
Have a parent/caregiver/guardian that is able to consent for their participation and complete assessments regarding the child's development and behavior improvement throughout the study.

Who cannot join

Exposure to any investigational agent in the 30 days prior to randomization
Prior chronic treatment with oxytocin.
A primary psychiatric diagnosis other than ASD, including bipolar disorder, psychosis, schizophrenia, PTSD or major depressive disorder. These patients will be excluded due to potential confounding results.
Pregnant or lactating patients. IN-OXT has not been studied in pregnant or lactating women.
A medical condition that might interfere with the conduct of the study, confound interpretation of study results or endanger their own well-being.
Plan to initiate or change nonpharmacologic or pharmacologic interventions during the course of the study.
Females using an estrogen-based contraceptive. As an alternative to an estrogen based contraceptive, subjects will be counseled to use progesterone-based contraceptives; cervical caps; cervical sponges; or spermicidal foam in combination with a condom. Subjects will need to use a double barrier method to be in the study.

Trial locations and contacts

1 site in 1 country.

United States1 site

Montefiore Medical Center, Albert Einstein College of Medicine
The Bronx, New York, 10467

Other trials in United States

About this study

Read the detailed description

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.

Conditions: Prader-Willi Syndrome, Hyperphagia
Being tested: Intranasal Oxytocin (IN-OXT), Matched Placebo
Keywords: Prader-Willi Syndrome (PWS), Hyperphagia
Medical subject headings: Prader-Willi Syndrome, Hyperphagia

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

Intranasal Oxytocin (IN-OXT)Drug
Also called Syntocinon
Participants were instructed to take 16 IU/per day everyday (2 puffs per nostril, 4 IU each puff).
Matched PlaceboOther
Participants were instructed to take 16 IU/per day everyday (2 puffs per nostril, 4 IU each puff).

Study groups in NCT02629991
Study group	Role	What they receive
Intranasal Oxytocin	Experimental	Participants were instructed to take 16 IU/per day everyday (2 puffs per nostril, 4 IU each puff). Receives: Drug: Intranasal Oxytocin (IN-OXT)
Matched Placebo	Placebo comparator	Participants were instructed to take 16 IU/per day everyday (2 puffs per nostril, 4 IU each puff). Receives: Other: Matched Placebo

What is being measured

The primary outcome is the main question the study is designed to answer.

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.
Measured: 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.
Measured: 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
Measured: 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.
Measured: Assessed trends over multiple time points (weeks 0, 2, 4, 6, and 8), week 0 (baseline), week 4 and week 8 are reported.

Secondary outcomes (6)

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.
Measured: Trends over multiple time points (Weeks 0, 4, and 8).
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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: Trends over multiple time points (Weeks 0, 4, and 8).

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

Oct 2015May 2018

First posted15 Dec 2015 (estimated)
Study startOct 2015
Primary completionDec 2016
Study completionMay 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 Montefiore Medical CenterThe 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 2
Primary purpose: Treatment
Allocation: Randomised
Intervention model: Parallel groups
Masking: Quadruple blind
Who is masked: Participant, Care provider, Investigator, Outcomes assessor
Enrolment: 23 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT02629991
Sponsor's protocol ID: 14-10-427-01
Acronym: OXT-PWS
Status last verified: Sep 2021
First submitted: 18 Oct 2015
Last updated: 7 Oct 2021
Results posted: Yes, on ClinicalTrials.gov
Data monitoring committee: Yes

Want to know more?

The sponsor has not listed a contact. The official record may have more details.

Talk to your doctor first and take this page with you. Only the study team can confirm whether you can join.

Questions to ask the study team

What is the study trying to find out?
What would I need to do, and how often would I visit?
Could I receive a placebo instead of the treatment?
What are the possible risks and benefits for me?
Will it cost me anything? Are travel or time paid for?
Can I keep seeing my own doctor during the study?
What happens when the study ends?

See locations

RecruitingPhase 2
A Study of CSTI-500 in Patients With Prader-Willi Syndrome
Prader-Willi Syndrome
Recruiting
Cerebellar TMS and Satiety in Prader-Willi Syndrome
Prader-Willi Syndrome
Enrolling by invitationObservational study
Observational Study of VYKAT™ XR in Patients With Prader-Willi Syndrome
Prader-Willi Syndrome
Active, not recruitingPhase 1/2
A Study of RM-718 in Healthy Subjects and Patients With MC4R Pathway Impairment
Hypothalamic Obesity, Prader-Willi Syndrome
Active, not recruiting
Impact of Bright Light Therapy on Prader-Willi Syndrome
Prader-Willi Syndrome, Excessive Daytime Sleepiness

All Prader-Willi Syndrome trials

Study at a glance

From the registry record, last updated by the sponsor on 7 Oct 2021. Recruitment status is as the sponsor reported it: confirm with the study team.

Official title: Oxytocin vs. Placebo for the Treatment Hyperphagia in Children and Adolescents With Prader-Willi Syndrome
ClinicalTrials.gov ID: NCT02629991
Acronym / sponsor ID: OXT-PWS / 14-10-427-01
Conditions: Prader-Willi Syndrome, Hyperphagia
Keywords: Prader-Willi Syndrome (PWS), Hyperphagia
Phase: Phase 2
Study type: Interventional
Recruitment status: Completed
Enrolment: 23 participants (actual)
Lead sponsor: Montefiore Medical CenterUniversity, hospital or other
Collaborators: Foundation for Prader-Willi Research
Study start: Oct 2015
Primary completion: Dec 2016 (actual)
Study completion: May 2018 (actual)
First posted: 15 Dec 2015 (estimated)
Results: Posted 7 Oct 2021

Clinical summary

The research question as the sponsor registered it. This describes what the study plans to test, not what it found.

Detailed description

Study design

Primary purpose: Treatment
Allocation: Randomised
Intervention model: Parallel groups
Masking: Quadruple blind (participant, care provider, investigator, outcomes assessor)
Number of arms: 2

Arm	Type	Receives
Intranasal Oxytocin	Experimental	Drug: Intranasal Oxytocin (IN-OXT)Participants were instructed to take 16 IU/per day everyday (2 puffs per nostril, 4 IU each puff).
Matched Placebo	Placebo comparator	Other: Matched PlaceboParticipants were instructed to take 16 IU/per day everyday (2 puffs per nostril, 4 IU each puff).

Who may qualify

Ages: 5 to 18 years
Sex: All
Healthy volunteers: Not accepted
Age groups: Child, Adult

Key inclusion criteria (5 listed)

Male or Female child outpatients aged 5 to 18 years
Diagnosis of PWS confirmed by genetic testing and patient medical records and history
Stable pharmacologic, educational, behavioral and/or dietary interventions for 4 weeks prior to the study start, and for the duration of the study.
Have a physical exam and laboratory results that are within the norms for PWS
Have a parent/caregiver/guardian that is able to consent for their participation and complete assessments regarding the child's development and behavior improvement throughout the study.

Key exclusion criteria (7 listed)

Exposure to any investigational agent in the 30 days prior to randomization
Prior chronic treatment with oxytocin.
A primary psychiatric diagnosis other than ASD, including bipolar disorder, psychosis, schizophrenia, PTSD or major depressive disorder. These patients will be excluded due to potential confounding results.
Pregnant or lactating patients. IN-OXT has not been studied in pregnant or lactating women.
A medical condition that might interfere with the conduct of the study, confound interpretation of study results or endanger their own 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
Intranasal Oxytocin (IN-OXT)Also: SyntocinonParticipants were instructed to take 16 IU/per day everyday (2 puffs per nostril, 4 IU each puff).	Drug	Intranasal Oxytocin
Matched PlaceboParticipants were instructed to take 16 IU/per day everyday (2 puffs per nostril, 4 IU each puff).	Other	Matched Placebo

Primary outcome

Hyperphagia Questionnaire (HQ)- Total Score
Time frame: Assessed trends over multiple time points (weeks 0, 2, 4, 6, and 8), week 0 (baseline), week 4 and week 8 are reported.
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.
Hyperphagia Questionnaire (HQ)- Behavior Factor Score
Time frame: Assessed trends over multiple time points (weeks 0, 2, 4, 6, and 8), week 0 (baseline), week 4 and week 8 are reported.
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.
Hyperphagia Questionnaire (HQ)- Drive Factor Score
Time frame: Assessed trends over multiple time points (weeks 0, 2, 4, 6, and 8), week 0 (baseline), week 4 and week 8 are reported.
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
Hyperphagia Questionnaire (HQ) -Severity Factor Score
Time frame: Assessed trends over multiple time points (weeks 0, 2, 4, 6, and 8), week 0 (baseline), week 4 and week 8 are reported.
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.

6 secondary outcomes

Repetitive Behavior Scale-Revised (RBS-R) Ritualistic Behavior Factor Score
Time frame: Trends over multiple time points (Weeks 0, 4, and 8).
Repetitive Behavior Scale-Revised (RBS-R) Sameness Factor Score
Time frame: Trends over multiple time points (Weeks 0, 4, and 8).
Repetitive Behavior Scale-Revised (RBS-R) Stereotyped Behavior Factor Score
Time frame: Trends over multiple time points (Weeks 0, 4, and 8).
Repetitive Behavior Scale-Revised (RBS-R) Compulsive Behavior Factor Score
Time frame: Trends over multiple time points (Weeks 0, 4, and 8).
Repetitive Behavior Scale-Revised (RBS-R) Self-Injurious Factor Score
Time frame: Trends over multiple time points (Weeks 0, 4, and 8).
Repetitive Behavior Scale-Revised (RBS-R) Restricted Interest Factor Score
Time frame: Trends over multiple time points (Weeks 0, 4, and 8).

Results and safety

Results posted to the registry on 7 Oct 2021, as reported by the sponsor. Figures are copied from the record, not re-analysed. Full results on ClinicalTrials.gov.

Participant flow

Group	Started	Completed
Intranasal Oxytocin	11	10
Matched Placebo	12	11

Primary outcome: Hyperphagia Questionnaire (HQ)- Total Score

Time frame: Assessed trends over multiple time points (weeks 0, 2, 4, 6, and 8), week 0 (baseline), week 4 and week 8 are reported.. Mean (Standard Deviation). Unit: score on a scale

Measure	Intranasal Oxytocin n = 11	Matched Placebo n = 12
Baseline	27.91 (10.77)	30.58 (8.08)
Week 4	27.4 (9.85)	23.33 (8.41)
Week 8	26.91 (11.67)	25.09 (9.86)

Intranasal Oxytocin vs Matched Placebo: p = 0.059, Mixed effects model

Population analysed: 1 subject discontinued oxytocin early and 1 subject discontinued placebo early

Primary outcome: Hyperphagia Questionnaire (HQ)- Behavior Factor Score

Time frame: Assessed trends over multiple time points (weeks 0, 2, 4, 6, and 8), week 0 (baseline), week 4 and week 8 are reported.. Mean (Standard Deviation). Unit: score on a scale

Measure	Intranasal Oxytocin n = 11	Matched Placebo n = 12
Baseline	9.55 (5.01)	9.75 (3.47)
Week 4	9.4 (4.27)	7.22 (2.64)
Week 8	9.64 (4.9)	8.09 (4.25)

Intranasal Oxytocin vs Matched Placebo: p = 0.088, Mixed effects model

Population analysed: 1 subject discontinued oxytocin early and 1 subject discontinued placebo early

Primary outcome: Hyperphagia Questionnaire (HQ)- Drive Factor Score

Time frame: Assessed trends over multiple time points (weeks 0, 2, 4, 6, and 8), week 0 (baseline), week 4 and week 8 are reported.. Mean (Standard Deviation). Unit: score on a scale

Measure	Intranasal Oxytocin n = 11	Matched Placebo n = 12
Baseline	10.82 (4.81)	13.58 (4.44)
Week 4	11 (4.03)	10.44 (5.46)
Week 8	10.36 (4.84)	10.55 (4.48)

Intranasal Oxytocin vs Matched Placebo: p = 0.027, Mixed effects model

Population analysed: 1 subject discontinued oxytocin early and 1 subject discontinued placebo early

Primary outcome: Hyperphagia Questionnaire (HQ) -Severity Factor Score

Time frame: Assessed trends over multiple time points (weeks 0, 2, 4, 6, and 8), week 0 (baseline), week 4 and week 8 are reported.. Mean (Standard Deviation). Unit: score on a scale

Measure	Intranasal Oxytocin (IN-OXT) n = 11	Matched Placebo n = 12
Baseline	7.55 (2.62)	7.25 (2.09)
Week 4	7.00 (2.54)	5.67 (2.45)
Week 8	6.91 (2.74)	6.45 (2.54)

Intranasal Oxytocin (IN-OXT) vs Matched Placebo: p = 0.16, Mixed effects model

Population analysed: 1 subject discontinued oxytocin early and 1 subject discontinued placebo early

6 secondary outcomes are reported in the full results.

Adverse events

Time frame: 8 Weeks. Other adverse events reported at 0% frequency or more

Group	Serious adverse events	Other adverse events	Deaths
Intranasal Oxytocin (IN-OXT)	0 of 11 (0%)	7 of 11 (63.6%)	0 of 11
Matched Placebo	0 of 12 (0%)	7 of 12 (58.3%)	0 of 12

Loading the posted results from ClinicalTrials.gov…

Research links

Official record on ClinicalTrials.govLast updated by the sponsor 7 Oct 2021.
Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
Foundation for Prader Willi ResearchLink provided by the sponsor.

Related trials for Prader-Willi Syndrome

RecruitingPhase 2
A Study of CSTI-500 in Patients With Prader-Willi Syndrome
Prader-Willi Syndrome
Recruiting
Cerebellar TMS and Satiety in Prader-Willi Syndrome
Prader-Willi Syndrome
Enrolling by invitationObservational study
Observational Study of VYKAT™ XR in Patients With Prader-Willi Syndrome
Prader-Willi Syndrome
Active, not recruitingPhase 1/2
A Study of RM-718 in Healthy Subjects and Patients With MC4R Pathway Impairment
Hypothalamic Obesity, Prader-Willi Syndrome
Active, not recruiting
Impact of Bright Light Therapy on Prader-Willi Syndrome
Prader-Willi Syndrome, Excessive Daytime Sleepiness

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.

Eric Hollander, MD principal investigator

Montefiore Medical Center

1 site in 1 country: see every site and its status.

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing Intranasal Oxytocin (IN-OXT) in people with Prader-Willi Syndrome and Hyperphagia, run by Montefiore Medical Center 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 5 inclusion and 7 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 7 Oct 2021. 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 phase mean?

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 7 Oct 2021). 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?

Hyperphagia Questionnaire (HQ)- Total Score (time frame: Assessed trends over multiple time points (weeks 0, 2, 4, 6, and 8), week 0 (baseline), week 4 and week 8 are reported.). The study lists 4 primary outcomes in all. 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 NCT02629991.

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 7 Oct 2021.