Trial · NCT02710084
Piloting Treatment With Intranasal Oxytocin in Phelan-McDermid Syndrome
NCT02710084, Phase 2, Completed, Alexander Kolevzon; results first posted 2021-12-06
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. Piloting Treatment With Intranasal Oxytocin in Phelan-McDermid Syndrome. Trials, record NCT02710084. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT02710084
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Piloting Treatment With Intranasal Oxytocin in Phelan-McDermid Syndrome
- NCT number
- NCT02710084
- Status
- Completed
- Phases
- Phase 2
- Enrollment
- 18 participants, actual
- Lead sponsor
- Alexander Kolevzon
- Conditions
- Phelan-McDermid Syndrome
- Interventions
- Oxytocin, Saline
- First posted
- 2016-03-16
- Last updated
- 2021-12-06
- Results first posted
- 2021-12-06
- Limitations and caveats
- The small sample size, potential challenges with drug administration, and expectancy bias due to relying on parent reported outcome measures may all contribute to limitations in interpreting results.
Source ClinicalTrials.gov
Registered outcomes13
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Change in Aberrant Behavior Checklist | Baseline and Week 12 | Change in scale at week 12 as compared to baseline. Aberrant Behavior Checklist - behavior rating subscales for the assessment of treatment effects.16 items, Each item is scored as 0 (never a problem), 1 (slight problem), 2 (moderately serious problem), or 3 (severe problem). Total score range from 0 to 48, with higher score indicating poorer health outcomes. |
| Secondary | Change in Visual Paired Comparison (VPC) Task | Baseline and Week 12 | Change in VPC Task has 2 subscores at week 12 as compared to baseline. The subject is initially presented with a target for familiarization and the familiar target is then paired with a novel target. Recognition is indexed by a novelty score derived from the percentage of time looking at the novel stimulus vs. the familiar stimulus. The Social-Novel Preference score refers to the proportion of time spent looking at novel stimuli out of all the time spent looking at both novel and familiar stimuli in the social test. The Nonsocial-Novel Preference score refers to the proportion of time spent looking at novel stimuli out of all the time spent looking at both novel and familiar stimuli in the nonsocial test.The scores are given in proportions with a range from -1 to 1. A score of -1 would mean the participant shows a preference for familiar stimuli and a score of 1 demonstrates a preference for novel stimuli. |
| Secondary | Change in Gap-Overlap Task | Baseline and Week 12 | One of three computerized eye tracking tasks to measure attention. three computer screens are set up side by side. A stimulus initially appears on the central screen, and once the participant orients to that stimulus, another stimulus will appear on one of the lateral screens. In the "gap" condition, the central stimulus will disappear before the peripheral stimulus appears. In the "overlap" condition, the central stimulus will remain on the screen while the peripheral stimulus appears. In both conditions, reaction times of the participant's eye movements (saccadic reaction time) from the central stimulus to the peripheral stimulus will be measured. Gap Effect measures the difference between average Gap and Overlap saccade latencies. |
| Secondary | Change in Flicker Tasks | Baseline and Week 12 | One of three computerized eye tracking tasks to measure attention. The "flicker task," or the four-alternative forced-choice preferential looking paradigm - the time interval over which the visual system is able to parse information. Scores range from -1 to 1 with higher scores indicating better (more typical) performance. |
| Secondary | Change in Vineland Adaptive Behavior Scales | Baseline and Week 12 | Mean change in scale at week 12 as compared to baseline. Vineland Adaptive Behavior Communication Domain measures adaptive functioning. Vineland-II subdomains (Communication, Daily Living Skills, Socialization, Motor, Adaptive Behavior Composite, Internalizing, Externalizing and maladaptive) are V-scale scores (M=15, SD=3). Higher scores indicate better developed adaptive social behavior. Vineland-II domain and composite values are standard scores (M=100, SD=15). Higher scores indicate better developed adaptive social behavior. |
| Secondary | Change in Repetitive Behavior Scale-Revised (RBS-R) | Baseline and Week 12 | Change in Repetitive Behavior Scale at week 12 as compared to baseline. the RBS-R is a 44-item self-report questionnaire consisting of subscales and one additional global rating score from 1-100. Score range for Stereotypic Behavior subscale is 0-27, Self-Injurious Behavior subscale is 0-24, Compulsive Behavior subscale is 0-18, Ritualistic/Sameness Behavior subscale is 0-36, and Restricted Interests subscale 0-9. Total scale for RBS-R is 0-214, with higher score indicating more severe problem. |
| Secondary | Change in Clinical Global Impression - Severity Scales | Baseline and Week 12 | Mean Change in Clinical Global Impression-Severity Scales at week 12 as compared to baseline. Clinical Global Impressions (CGI) Rating Scales are commonly used to measure symptom severity and global improvement in treatment studies of patients with developmental disorders. There Severity Scale (CGI-S) is a 7-point scale that requires the clinician to rate the severity of illness at the time of assessment. Full scale from 1 - 7. Higher scores indicate greater symptom severity. |
| Secondary | Change in Short Sensory Profile (SSP) | Baseline and Week 12 | Mean Change in Short Sensory Profile Scale at week 12 as compared to baseline, measuring sensory sensitivity and sensory seeking behavior. The SSP is a 38-item caregiver questionnaire. Items are based on a 5-point Liker scale ranging from 1=always to 5=never. Tactile Sensitivity ranges from 7-35. Taste/Smell Sensitivity ranges from 4-20. Movement Sensitivity ranges from 3-15. Underresponsive/Seeks Sensation ranges from 7-35. Auditory Filtering ranges from 6-30. Low Energy/Weak ranges from 6-30. Visual/Auditory Sensitivity ranges from 5-25. Total scale range from 38-190. Lower scores indicate a higher probability of sensory processing abnormalities. |
| Secondary | Change in Macarthur-Bates Communicative Development Inventory (MCDI) | Baseline and Week 12 | Macarthur-Bates Communicative Development Inventory at week 12 as compared to baseline, measuring language. Words and Gestures Forms are for children ages 8-18 months. In the form, the first part prompts parents to document the child's understanding of hundreds of early vocabulary items separated into semantic categories such as animal names, household items, and action words. Parents mark the words understood or used, and the forms yield separate indexes of words understood and words produced. The second part of each form asks parents to record the communicative and symbolic gestures the child has tried or completed. Ranges for each subscale score are as follows: Phrases understood (0-28), Words understood (0-396), Words produced (0-396), Early gestures (0-18), Later gestures (0-45), Total gestures (0-63). Higher scores indicate a great number of words, phrases, or gestures understood or produced. |
| Secondary | Change in EEG Tasks: Auditory Habituation | Baseline and Week 12 | In the Auditory Habituation EEG task, participants listen to a series of 4 tones and the neural responses to each of these tones (event related potentials, ERPs ) are averaged. The N1 and P2 components are typical responses to auditory stimuli. The N1 response is the initial, quick response to hearing a stimulus, and P2 reflects the later processing of the stimulus. Both of these should be greater in response to the first tone, and then reduce in response to each subsequent tone. This is known as habituation, and is calculated as the change in Tone 1 minus Tone 2 response for both N1 and P2 components. |
1 to 10 of 13Source ClinicalTrials.gov
See all 13 registered outcomesOutcomes13
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Change in Aberrant Behavior Checklist | Baseline and Week 12 | In the case of two participants who dropped out after week 4, difference scores were calculated for each outcome measure using the last observation carried forward (This is why some measures have N's of 9 and 7). Two participants dropped out after BL and were not included in the analysis. | score on a scale | MEAN |
| SECONDARY | Change in Visual Paired Comparison (VPC) Task | Baseline and Week 12 | Only 3 participants with data from baseline and week 12 | score on a scale | MEAN |
| SECONDARY | Change in Gap-Overlap Task | Baseline and Week 12 | Only 2 participants with data from both baseline and Week 12 | milliseconds (ms) | MEAN |
| SECONDARY | Change in Flicker Tasks | Baseline and Week 12 | Only 1 participant with data from baseline and Week 12 | score on a scale | MEAN |
| SECONDARY | Change in Vineland Adaptive Behavior Scales | Baseline and Week 12 | Participants were only included in this analysis if they had Vinelands at BL and Week 12. One incomplete Maladaptive subdomain form for one participant. | score on a scale | MEAN |
| SECONDARY | Change in Repetitive Behavior Scale-Revised (RBS-R) | Baseline and Week 12 | Patients were only included in this analysis if they had RBS-R at BL and Week 12 | score on a scale | MEAN |
| SECONDARY | Change in Clinical Global Impression - Severity Scales | Baseline and Week 12 | Data from all participants who had data collected at 12 weeks. | score on a scale | MEAN |
| SECONDARY | Change in Short Sensory Profile (SSP) | Baseline and Week 12 | In the case of two participants who dropped out after week 4, difference scores were calculated for each outcome measure using the last observation carried forward (This is why some measures have N's of 9 and 7). Two participants dropped out after BL and were not included in the analysis. | score on a scale | MEAN |
| SECONDARY | Change in Macarthur-Bates Communicative Development Inventory (MCDI) | Baseline and Week 12 | score on a scale | MEAN | |
| SECONDARY | Change in EEG Tasks: Auditory Habituation | Baseline and Week 12 | Data results available only for those who underwent Auditory Habituation EEG task | microvolts (µV) | MEAN |
1 to 10 of 13Source ClinicalTrials.gov
See all 13 outcomesResults by group97
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Change in Aberrant Behavior Checklist | Saline Then Oxytocin | Social Withdrawal | -7.44 | Standard Deviation 4.58 | score on a scale |
| Change in Aberrant Behavior Checklist | Intranasal Oxytocin | Social Withdrawal | -2.42 | Standard Deviation 3.15 | score on a scale |
| Change in Aberrant Behavior Checklist | Saline Then Oxytocin | Irritability | -5 | Standard Deviation 6.63 | score on a scale |
| Change in Aberrant Behavior Checklist | Intranasal Oxytocin | Irritability | -1.71 | Standard Deviation 7.13 | score on a scale |
| Change in Aberrant Behavior Checklist | Saline Then Oxytocin | Stereotypy | -2.22 | Standard Deviation 4.66 | score on a scale |
| Change in Aberrant Behavior Checklist | Intranasal Oxytocin | Stereotypy | 0.57 | Standard Deviation 2.99 | score on a scale |
| Change in Aberrant Behavior Checklist | Saline Then Oxytocin | Hyperactivity | -8.11 | Standard Deviation 11.85 | score on a scale |
| Change in Aberrant Behavior Checklist | Intranasal Oxytocin | Hyperactivity | -2.71 | Standard Deviation 7.34 | score on a scale |
| Change in Aberrant Behavior Checklist | Saline Then Oxytocin | Inappropriate speech | -1.67 | Standard Deviation 3.04 | score on a scale |
| Change in Aberrant Behavior Checklist | Intranasal Oxytocin | Inappropriate speech | -7.14 | Standard Deviation 2.21 | score on a scale |
1 to 10 of 97Source ClinicalTrials.gov
See all 97 results by groupParticipant flow12
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| First Phase - 12 Weeks | STARTED | Saline Then Oxytocin | 10 | |
| First Phase - 12 Weeks | STARTED | Intranasal Oxytocin | 8 | |
| First Phase - 12 Weeks | COMPLETED | Saline Then Oxytocin | 7 | |
| First Phase - 12 Weeks | COMPLETED | Intranasal Oxytocin | 7 | |
| First Phase - 12 Weeks | NOT COMPLETED | Saline Then Oxytocin | 3 | |
| First Phase - 12 Weeks | NOT COMPLETED | Intranasal Oxytocin | 1 | |
| Open Label Phase: Week 12 to 24 | STARTED | Saline Then Oxytocin | 7 | |
| Open Label Phase: Week 12 to 24 | STARTED | Intranasal Oxytocin | 7 | |
| Open Label Phase: Week 12 to 24 | COMPLETED | Saline Then Oxytocin | 7 | |
| Open Label Phase: Week 12 to 24 | COMPLETED | Intranasal Oxytocin | 7 |
1 to 10 of 12Source ClinicalTrials.gov
See all 12 participant flowAdverse events64
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 | Sedation | General disorders | Saline | 2 | 10 | |
| other | Sedation | General disorders | Intranasal Oxytocin | 1 | 15 | |
| other | Decreased appetite | Metabolism and nutrition disorders | Saline | 2 | 10 | |
| other | Decreased appetite | Metabolism and nutrition disorders | Intranasal Oxytocin | 0 | 15 | |
| other | Periorbital / facial swelling | General disorders | Saline | 1 | 10 | |
| other | Periorbital / facial swelling | General disorders | Intranasal Oxytocin | 1 | 15 | |
| other | Diarrhea | Gastrointestinal disorders | Saline | 1 | 10 | |
| other | Diarrhea | Gastrointestinal disorders | Intranasal Oxytocin | 2 | 15 | |
| other | Upper respiratory tract infection | Infections and infestations | Saline | 1 | 10 | |
| other | Upper respiratory tract infection | Infections and infestations | Intranasal Oxytocin | 2 | 15 |
1 to 10 of 64Source ClinicalTrials.gov
See all 64 adverse eventsAdverse event totals6
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Saline | 0 | 10 |
| Total, all-cause mortality | Intranasal Oxytocin | 15 | 15 |
| Total, other adverse events | Saline | 9 | 10 |
| Total, other adverse events | Intranasal Oxytocin | 15 | 15 |
| Total, serious adverse events | Saline | 0 | 10 |
| Total, serious adverse events | Intranasal Oxytocin | 0 | 15 |
Source ClinicalTrials.gov
Papers1
The papers the trial lists on ClinicalTrials.gov.
| PMID | Listed as |
|---|---|
| 34593045 | RESULT |
Source PubMed
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 |
|---|---|
| Saline vs Intranasal Oxytocin: deaths from any cause | Trial comparisons |
| Saline vs Intranasal Oxytocin: 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.
For software
Read this trial from software.
The record and each of its lists are JSON, twenty rows a page. No key.
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