Trial · NCT03197662
Intranasal Oxytocin vs. Placebo for the Treatment of Hyperphagia in Prader-Willi Syndrome
NCT03197662, Phase 2, Completed, Eric Hollander; results first posted 2025-02-07
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. Intranasal Oxytocin vs. Placebo for the Treatment of Hyperphagia in Prader-Willi Syndrome. Trials, record NCT03197662. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT03197662
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Intranasal Oxytocin vs. Placebo for the Treatment of Hyperphagia in Prader-Willi Syndrome
- NCT number
- NCT03197662
- Status
- Completed
- Phases
- Phase 2
- Enrollment
- 32 participants, actual
- Lead sponsor
- Eric Hollander
- Conditions
- Prader-Willi Syndrome, Hyperphagia
- Interventions
- Intranasal Oxytocin (IN-OXT), Matched Placebo
- First posted
- 2017-06-23
- Last updated
- 2025-02-07
- Results first posted
- 2025-02-07
Source ClinicalTrials.gov
Registered outcomes14
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Change in Efficacy of Peptide Intranasal Oxytocin (IN-OXT) | From Randomization at Baseline until Endpoint at Week 8 (Change over 8 weeks) | Change in efficacy of peptide IN-OXT as measured by changes in hyperphagia from baseline will be assessed using the Hyperphagia Questionnaire for Clinical Trials (HQ-CT). HQ-CT is a caregiver-rated assessment comprised of 9-items that measures the frequency and intensity of hyperphagic behaviors, over the preceding two weeks, in participants with Prader-Willi Syndrome (PWS). The HQ-CT total score is created by summing the 9 item-level responses (ranging from 0 to 4) for a possible range of 0-36 with higher scores indicate more extreme hyperphagia. For purposes of this outcome measure, negative scores represent a decrease in hyperphagia from baseline and positive scores represent an increase in hyperphagia from baseline. |
| Secondary | Change in Repetitive Behavior | From Randomization at Baseline until Endpoint at Week 8 (Change over 8 weeks) | Change in Repetitive Behavior from baseline was determined using the Repetitive Behavior Scale Revised (RBS-R) score. The RBS-R is a 44-item form used to measure the full spectrum of repetitive behavior of participants. The child's parent will choose a score that describes how much of a problem the behavior has been over the last month on a 4-point scale ranging from 0 ("Behavior does not occur") to 3 ("Behavior occurs and is severe problem"). The final question asks participants to "lump together" all behaviors described in the RBS-R, and provide a rating (0-100) for how much of a problem these repetitive behaviors are overall: 1 ("Not a problem at all") to 100 ("As bad as you can imagine"). For purposes of this outcome, a change in group mean score from baseline is summarized. A negative score is indicative of a decrease in the frequency and severity of behavioral problems; whereas, a positive score is indicative of an increase in the frequency and severity of behavioral problems. |
| Secondary | Change in Rigid Behavior | From Randomization at Baseline until Endpoint at Week 8 (Change over 8 weeks) | Change in Rigid Behavior from baseline was assessed using the Montefiore-Einstein Rigidity Scale-Revised-Prader-Willi Syndrome (MERS-R-PWS). The MERS-R-PWS is designed to assess 3 domains of rigid behavior in children with PWS: 1. Behavioral Rigidity (e.g., insistence on sameness, things must be done his/her way, etc.) 2. Cognitive Rigidity (e.g., special interests, inflexible adherence to rules, etc.) 3. Protest (in response to deviation from rigidity (e.g., verbal objection, tantrum, physical aggression). Four items are rated on a 5-point scale ranging from 0 ("No/None") to 4 ("Extreme/Extremely difficult") for each of the 3 domains. The domains are added yielding a possible Total Domain Score which could range from 0-48. For purposes of this outcome measure, a change in group mean score from baseline is summarized. A negative score is indicative of a decrease in rigid behaviors from baseline; whereas, a positive score is indicative of an increase in rigid behavior from baseline. |
| Secondary | Change in Body Weight | From Randomization at Baseline until Endpoint at Week 8 (Change over 8 weeks) | Change in body weight from baseline was determined by obtaining the participant's body weight, in kilograms, using a regularly calibrated scale. Change in body weights from baseline are summarized by study arm using basic descriptive statistics. A negative result indicates a decrease in body weight from baseline and may be associated with improved health-related outcomes including a decrease in hyperphagia. |
| Secondary | Body Composition | 8 weeks | Body composition, in particular fat mass, was assessed using bioelectrical impedance analysis (BIA). BIA was conducted with equipment and oversight provided by the Einstein-Mount Sinai Diabetes Research Center. The equipment measured resistance in ohms which represents the resistance to a small alternating current passing through the body. These values are converted to body fat percentages using built-in software, after calibrating for variables, for purposes of reporting. Percentages are summarized by study arm using basic descriptive statistics. |
| Secondary | Change in Body Mass Index (BMI) | From Randomization at Baseline until Endpoint at Week 8 (Change over 8 weeks) | Change in BMI from baseline will be calculated by dividing the participant's weight in kilograms by their height in meters squared (kg/m\^2). Change in mean BMI from baseline will summarized and reported using basic descriptive statistics. A negative BMI score may be indicative of a decrease in hyperphagia from baseline; whereas, a positive BMI score may be indicative of an increase in hyperphagia when compared to baseline. |
| Secondary | Change in Quality of Life (QOL) and Caregiver Burden as Determined by the World Health Organization Quality of Life (WHOQOL-BREF) Questionnaire: Domain 1 (Physical Health) | From Randomization at Baseline until Endpoint at Week 8 (Change over 8 weeks) | Change in QOL/Caregiver burden was assessed using the 7-item Physical Health domain subscale of the WHOQOL-BREF questionnaire. The questionnaire was completed by the parent/caregiver and quantified the burden placed on the caregiver/family by the PWS disorder. Each item of the Physical Health domain was scored on a 5-point ordinal scale ranging from 1-5, for an overall possible raw scoring range of 7-35. A mean overall Physical Health score was calculated by summing the scores from the seven items (response values for 2 of the items are inversely-coded) and dividing by the total # of questions (7) for an algebraic mean. A transformed scale score was obtained by subtracting 7 (lowest possible score) from the raw score and dividing by 28 (possible raw score range). For purposes of this outcome measure change from baseline was reported. Increases in Physical Health scores from baseline were associated with higher physical health QOL and reduced caregiver physical burden. |
| Secondary | Change in Quality of Life (QOL) and Caregiver Burden as Determined by the World Health Organization Quality of Life (WHOQOL-BREF) Questionnaire - Domain 2 (Psychological Health) | From Randomization at Baseline until Endpoint at Week 8 (Change over 8 weeks) | Change in QOL/caregiver burden was assessed using the abbreviated WHOQOL-BREF. The WHOQOL-BREF measures 4 broad domains: Physical Health, Health, Social Relationships, and Environment. The questionnaire was completed by the parent/caregiver quantified the burden placed on the caregiver/family by the PWS disorder. For the Psychological Health domain (6 questions), responses were assigned to corresponding predefined scores ranging from 1-5 points. A mean overall Psychological Health score was calculated by summing the scores from 5 of the questions (#s 5, 6, 7, 11, and 19) and inverting the response value from Question #26 and adding it to the sum of the other 5 scores and dividing by the total number of questions (6). This result was multiplied by 4 to get a final Psychological Health domain score. Raw scoring ranges from 6-30; however, for purposes of this outcome measure change from baseline was reported. Positive values were associated with higher QOL and reduced caregiver burden. |
| Secondary | Change in Quality of Life (QOL) and Caregiver Burden as Determined by the World Health Organization Quality of Life (WHOQOL-BREF) Questionnaire - Domain 3 (Social Relationships) | From Randomization at Baseline until Endpoint at Week 8 (Change over 8 weeks) | Change in QOL and caregiver burden was assessed using the abbreviated WHOQOL-BREF. The WHOQOL-BREF measures 4 broad domains: Physical Health, Psychological Health, Social Relationships, and Environment. The questionnaire was completed by the parent/caregiver and quantified the burden placed on the caregiver/family by the PWS disorder. For the Social Relationships domain (3 questions), responses were assigned to corresponding predefined scores ranging from 1-5 points. A mean overall Social Relationships score was calculated by summing the 3 scores (#s 20, 21, and 22) and dividing by the total number of questions (3). This result was multiplied by 4 to get a final Social Relationships domain score. Social Relationships raw scoring ranges from 3-15; however, for purposes of this outcome measure change from baseline was reported. Positive values were associated with higher QOL and reduced caregiver burden. |
| Secondary | Change in Quality of Life (QOL) and Caregiver Burden as Determined by the World Health Organization Quality of Life (WHOQOL-BREF) Questionnaire - Domain 4 (Environment) | From Randomization at Baseline until Endpoint at Week 8 (Change over 8 weeks) | Change in QOL and caregiver burden was assessed using the abbreviated WHOQOL-BREF. The WHOQOL-BREF is comprised of 26 items which measure 4 broad domains: Physical Health, Psychological Health, Social Relationships, and Environment. The questionnaire was completed by the parent/caregiver and quantified the burden placed on the caregiver/family by the PWS disorder. For the Environment domain (8 questions), responses were assigned to corresponding predefined scores ranging from 1-5 points. A mean overall Environment score was calculated by summing the scores from the 8 questions (#s 8, 9, 12, 13, 14, 23, 24, and 25) and dividing by the total number of questions (8). This result was multiplied by 4 to get a final Environmental domain score. Environment raw scoring ranges from 8-40; however, for purposes of this outcome measure change from baseline was reported. Positive values were associated with higher QOL and reduced caregiver burden. |
1 to 10 of 14Source ClinicalTrials.gov
See all 14 registered outcomesOutcomes14
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Change in Efficacy of Peptide Intranasal Oxytocin (IN-OXT) | From Randomization at Baseline until Endpoint at Week 8 (Change over 8 weeks) | Data was unable to be collected from 2 participants in the matched Placebo study arm who withdrew from the study. | score on a scale | MEAN |
| SECONDARY | Change in Repetitive Behavior | From Randomization at Baseline until Endpoint at Week 8 (Change over 8 weeks) | Data was unable to be collected from 2 participants in the matched Placebo study arm who withdrew from the study. | score on a scale | MEAN |
| SECONDARY | Change in Rigid Behavior | From Randomization at Baseline until Endpoint at Week 8 (Change over 8 weeks) | Data was unable to be collected from 2 participants in the matched Placebo study arm who withdrew from the study. | score on a scale | MEAN |
| SECONDARY | Change in Body Weight | From Randomization at Baseline until Endpoint at Week 8 (Change over 8 weeks) | Data was missing or unable to be collected from 3 participants in the matched Placebo study arm (including the 2 participants who withdrew from the study). | kilograms | MEAN |
| SECONDARY | Body Composition | 8 weeks | Data was missing or unable to be collected from 4 participants in the IN-OXT study arm and 8 participants in the matched Placebo study arm (including the 2 participants who withdrew from the study). | percentage of fat | MEDIAN |
| SECONDARY | Change in Body Mass Index (BMI) | From Randomization at Baseline until Endpoint at Week 8 (Change over 8 weeks) | Data was missing or unable to be collected from 1 participant in the IN-OXT arm and 3 participants in the matched Placebo study arm (including the 2 participants who withdrew from the study). | kg/m2 | MEAN |
| SECONDARY | Change in Quality of Life (QOL) and Caregiver Burden as Determined by the World Health Organization Quality of Life (WHOQOL-BREF) Questionnaire: Domain 1 (Physical Health) | From Randomization at Baseline until Endpoint at Week 8 (Change over 8 weeks) | Data was missing or unable to be collected from 1 participant in the IN-OXT arm and 3 participants in the matched Placebo study arm (including the 2 participants who withdrew from the study). | score on a scale | MEAN |
| SECONDARY | Change in Quality of Life (QOL) and Caregiver Burden as Determined by the World Health Organization Quality of Life (WHOQOL-BREF) Questionnaire - Domain 2 (Psychological Health) | From Randomization at Baseline until Endpoint at Week 8 (Change over 8 weeks) | Data was missing or unable to be collected from 1 participant in the IN-OXT arm and 3 participants in the matched Placebo study arm (including the 2 participants who withdrew from the study). | score on a scale | MEAN |
| SECONDARY | Change in Quality of Life (QOL) and Caregiver Burden as Determined by the World Health Organization Quality of Life (WHOQOL-BREF) Questionnaire - Domain 3 (Social Relationships) | From Randomization at Baseline until Endpoint at Week 8 (Change over 8 weeks) | Data was missing or unable to be collected from 1 participant in the IN-OXT arm and 3 participants in the matched Placebo study arm (including the 2 participants who withdrew from the study). | score on a scale | MEAN |
| SECONDARY | Change in Quality of Life (QOL) and Caregiver Burden as Determined by the World Health Organization Quality of Life (WHOQOL-BREF) Questionnaire - Domain 4 (Environment) | From Randomization at Baseline until Endpoint at Week 8 (Change over 8 weeks) | Data was missing or unable to be collected from 1 participant in the IN-OXT arm and 3 participants in the matched Placebo study arm (including the 2 participants who withdrew from the study). | score on a scale | MEAN |
1 to 10 of 14Source ClinicalTrials.gov
See all 14 outcomesResults by group26
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Change in Efficacy of Peptide Intranasal Oxytocin (IN-OXT) | Experimental: Intranasal Oxytocin (IN-OXT) | -6.08 | Standard Deviation 5.65 | score on a scale | |
| Change in Efficacy of Peptide Intranasal Oxytocin (IN-OXT) | Placebo Comparator: Matched Placebo | -6.64 | Standard Deviation 5.05 | score on a scale | |
| Change in Repetitive Behavior | Experimental: Intranasal Oxytocin (IN-OXT) | -13.2 | Standard Deviation 14.4 | score on a scale | |
| Change in Repetitive Behavior | Placebo Comparator: Matched Placebo | -8.53 | Standard Deviation 8.89 | score on a scale | |
| Change in Rigid Behavior | Experimental: Intranasal Oxytocin (IN-OXT) | -8.43 | Standard Deviation 5.15 | score on a scale | |
| Change in Rigid Behavior | Placebo Comparator: Matched Placebo | -11.2 | Standard Deviation 7.86 | score on a scale | |
| Change in Body Weight | Experimental: Intranasal Oxytocin (IN-OXT) | 0.585 | Standard Deviation 0.709 | kilograms | |
| Change in Body Weight | Placebo Comparator: Matched Placebo | -0.0669 | Standard Deviation 2.14 | kilograms | |
| Body Composition | Experimental: Intranasal Oxytocin (IN-OXT) | 23.5 | Full Range 15.0 to 53.7 | percentage of fat | |
| Body Composition | Placebo Comparator: Matched Placebo | 24.3 | Full Range 18.2 to 43.4 | percentage of fat |
1 to 10 of 26Source ClinicalTrials.gov
See all 26 results by groupParticipant flow6
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | Experimental: Intranasal Oxytocin (IN-OXT) | 15 | |
| Overall Study | STARTED | Placebo Comparator: Matched Placebo | 17 | |
| Overall Study | COMPLETED | Experimental: Intranasal Oxytocin (IN-OXT) | 15 | |
| Overall Study | COMPLETED | Placebo Comparator: Matched Placebo | 15 | |
| Overall Study | NOT COMPLETED | Experimental: Intranasal Oxytocin (IN-OXT) | 0 | |
| Overall Study | NOT COMPLETED | Placebo Comparator: Matched Placebo | 2 |
Source ClinicalTrials.gov
Adverse events52
Each adverse event the trial posted, group by group: the participants affected out of those at risk.
| Type | Event | Organ system | Group | Affected | At risk | Events |
|---|---|---|---|---|---|---|
| other | Fatigue | General disorders | Experimental: Intranasal Oxytocin (IN-OXT) | 0 | 15 | 0 |
| other | Fatigue | General disorders | Placebo Comparator: Matched Placebo | 3 | 17 | 3 |
| other | Increased Irritability | Psychiatric disorders | Experimental: Intranasal Oxytocin (IN-OXT) | 0 | 15 | 0 |
| other | Increased Irritability | Psychiatric disorders | Placebo Comparator: Matched Placebo | 2 | 17 | 2 |
| other | Headache | Nervous system disorders | Experimental: Intranasal Oxytocin (IN-OXT) | 2 | 15 | 2 |
| other | Headache | Nervous system disorders | Placebo Comparator: Matched Placebo | 3 | 17 | 3 |
| other | Nausea | Gastrointestinal disorders | Experimental: Intranasal Oxytocin (IN-OXT) | 0 | 15 | 0 |
| other | Nausea | Gastrointestinal disorders | Placebo Comparator: Matched Placebo | 1 | 17 | 1 |
| other | Aggressive Episode | Psychiatric disorders | Experimental: Intranasal Oxytocin (IN-OXT) | 1 | 15 | 1 |
| other | Aggressive Episode | Psychiatric disorders | Placebo Comparator: Matched Placebo | 0 | 17 | 0 |
1 to 10 of 52Source ClinicalTrials.gov
See all 52 adverse eventsAdverse event totals6
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Experimental: Intranasal Oxytocin (IN-OXT) | 0 | 15 |
| Total, all-cause mortality | Placebo Comparator: Matched Placebo | 0 | 17 |
| Total, other adverse events | Experimental: Intranasal Oxytocin (IN-OXT) | 7 | 15 |
| Total, other adverse events | Placebo Comparator: Matched Placebo | 13 | 17 |
| Total, serious adverse events | Experimental: Intranasal Oxytocin (IN-OXT) | 0 | 15 |
| Total, serious adverse events | Placebo Comparator: Matched Placebo | 0 | 17 |
Source ClinicalTrials.gov
Papers0
The papers the trial lists on ClinicalTrials.gov.
None in this release.
Grants0
The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.
None in this release.
Trial comparisons2
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
| Comparison | Source |
|---|---|
| Experimental: Intranasal Oxytocin (IN-OXT) vs Placebo Comparator: Matched Placebo: deaths from any cause | Trial comparisons |
| Experimental: Intranasal Oxytocin (IN-OXT) vs Placebo Comparator: Matched Placebo: serious adverse events | Trial comparisons |
Tests0
The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.
None in this release.
Data dates
The day each source was copied for this release.
Sources 3
- ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
- ClinicalTrials.govNo license stated
- NIH RePORTER's clinical studies and project filesPublic domain
Each source's terms
- ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
- ClinicalTrials.govnone stated
- NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)
ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.
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