Autism Spectrum Disorders
Conditions
Brief summary
Researchers at the Stanford University School of Medicine are seeking participants for a study examining the effectiveness of vasopressin, a neuropeptide, in treating children with autism spectrum disorder. Difficulty with social interactions is characteristic of people with autism, who often have problems interpreting facial expressions or maintaining eye contact while talking with someone. There are currently no effective medicines available to treat social problems in individuals with autism. Neuropeptides, such as vasopressin and oxytocin, are molecules used by neurons in the brain to communicate with one another. Vasopressin is closely related to oxytocin, which is currently being tested as a treatment for autism, and has been shown to enhance social functioning in animals. Animal studies have shown that when the proper functioning of vasopressin is experimentally altered, animals develop a variety of social deficits, including impaired memory for peers and a reduced interest in social interaction. Researchers found that when vasopressin was administered to mice with a genetically induced form of autism, their social functioning improved. Vasopressin is already approved by the Food and Drug Administration for use in humans, and has proved to be a successful treatment for some common pediatric conditions, including bedwetting. Similar to oxytocin, it also has been shown to improve social cognition and memory in people who do not have autism. The researchers will test the effects of vasopressin on social impairments in 50 boys and girls with autism, ages 6 to 12 years old. The study will last four weeks for each participant. Participants will receive either vasopressin or a placebo nasal spray. At the end of this phase of the study, those who received the placebo will have the option of participating in a four-week trial during which they will be given vasopressin. Stanford is the only site for the study. Participants do not need to live locally but will need to come to the Stanford University Department of Psychiatry and Behavioral Sciences for study visits.
Interventions
DRUGVasopressin
Participants aged 6 to 9.5 years of age will receive the maximum dose of 24 IU (12 IU twice daily). Participants aged 9.6 to 12 years of age will receive the maximum dose of 32 IU (16 IU twice daily).
DRUGPlacebo
Sponsors
National Institute of Mental Health (NIMH)
Stanford University
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
TRIPLE (Subject, Caregiver, Investigator)
Eligibility
Sex/Gender
ALL
Age
6 Years to 12 Years
Healthy volunteers
No
Inclusion criteria
* medically healthy outpatients between 6 and 12 years of age (cut off: 12 years and 11 months) * Intelligence Quotient (IQ) equal to or greater than 50 (Stanford-Binet) * Social Responsiveness Scale (SRS) Total Score equal to or greater than 70 * ability to complete laboratory and cognitive testing * diagnosis of Autism Spectrum Disorder (ASD) based on expert clinical opinion and confirmed on the Autism Diagnostic Interview-Revised (ADI-R), Autism Diagnostic Observation Schedule (ADOS) * Clinical Global Impression (CGI) severity rating of 4 or higher * care provider who can reliably bring participant to clinic visits, provide trustworthy ratings, and interact with the participant on a regular basis * stable medications for at least 4 weeks * no planned changes in psychosocial interventions during the trial * no concurrent participation in any other clinical research trials * willingness to provide blood samples and electrocardiogram
Exclusion criteria
* diagnosis of schizophrenia, schizoaffective disorder, bipolar disorder, or psychotic disorder * regular nasal obstruction or nosebleeds * active and unstable medical problems (e.g., migraine; asthma; seizure disorder; anaphylaxis; epilepsy; diabetes; serious liver, renal, or cardiac pathology) * clinically significant abnormal vital signs or ECG reading * evidence of a genetic mutation know to cause ASD (e.g., Fragile X Syndrome) or metabolic disorder * significant hearing or vision impairments * drinks large volumes of water (e.g., habitual or psychogenic polydipsia) * pregnant or sexually active females not using a reliable method of contraception (urine pregnancy test will be conducted) * history of hypersensitivity to vasopressin, its analogs (e.g., Desmopressin), or compounding preservatives (e.g., chlorobutanol) * current use of any medications known to interact with vasopressin including: 1) carbamazepine (i.e., Tegretol); chlorpropamide; clofibrate; urea; fludrocortisone; tricyclic antidepressants (all of which may potentiate the antidiuretic effect of vasopressin when used concurrently); 2) demeclocycline; norepinephrine; lithium; heparin; alcohol (all of which may decrease the antidiuretic effect of vasopressin when used concurrently); 3) ganglionic blocking agents including benzohexonium, chlorisondamine, pentamine (all of which may produce a marked increase in sensitivity to the pressor effects of vasopressin) * prior or current use of vasopressin * abnormal chemistry result
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Change From Baseline in Parent Rated Social Responsiveness Scale, 2nd Edition (SRS-2) T-Score After Treatment. | Baseline; Week 4 | Social Responsiveness Scale, 2nd Edition (SRS) scores measure social abilities with lower scores meaning better social abilities. (T-Score Range: 37- above 90 ) |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Change From Baseline in Reading the Mind in the Eyes Test, Child Version (RMET-child) Scores During Treatment. | Baseline; Week 4 | Higher scores mean better ability to read emotions and lower scores mean worse ability to read emotions. Range 0-28. |
| Change From Baseline in Laboratory Based Facial Emotion Recognition Abilities During Treatment. | Baseline; Week 4 | Higher scores mean better facial emotion recognition abilities. Lower scores mean worse facial emotion recognition abilities (Range: 0-42). |
| Change From Baseline in Parent Rated Repetitive Behavior Scale Revised (RBS-R) Scores During Treatment. | Baseline; Week 4 | Higher scores on the Repetitive Behavior Scale- Revised mean higher levels of repetitive and restricted behaviors. (Raw Score Total Range: 0 - 129) |
| Change From Baseline in Parent Rated Spence Children's Anxiety Scale (SCAS) During Treatment. | Baseline; Week 4 | Scale measuring severity of anxiety symptoms. Higher scores mean higher levels of anxiety, lower scores mean lower levels of anxiety. (Raw Score Range: 0 - 114) |
| Change From Baseline in Laboratory Based Eye-gaze to Social Cues During Treatment. | Baseline; Week 4 | — |
| Number of Participants With Side Effects Assessed Using Parent Rated Dosage Record Treatment Emergent Symptom Scale (DOTES) Scores During Treatment | Baseline through Week 4 | Dosage Record Treatment Emergent Symptom Scale (DOTES) side effects reported by parents during 4-weeks of treatment. Participant Counts are used. |
| Change From Baseline on the Overt Aggression Scale (OAS) During Treatment. | Baseline through Week 4 | Count of participants reporting an increase of aggression during treatment compared to baseline (pretreatment). |
| Change From Baseline in Heart Rate After Treatment. | Baseline; Week 4 | Sitting heart rate (beats per minute). |
| Change From Baseline in Parent Rated Aberrant Behavior Checklist (ABC) Scores During Treatment. | Baseline; Week 4 | Higher scores indicate more symptoms, lower scores indicate fewer symptoms. Irritability scores can range from 0-45. Lethargy scores can range from 0-48. Stereotypy scores can range from 0-21. Hyperactivity scores can range from 0-48. Inappropriate speech scores can from 0-12. |
| Change From Baseline in Clinical Global Impression (CGI) Severity, Social and Communication Scores During Treatment. | Baseline; Week 4 | Higher Scores on the CGI severity scale mean more greater social and communication deficits (Range 1-7) |
| Change From Baseline in Parent Rated Vineland Adaptive Behavior Scales Second Edition (VABS-II) - Social and Communication Subscales During Treatment. | Baseline; Week 4 | Higher Social Standard Score means better social skills, lower Social Standard Score means worse social skills. Higher Communication Standard Score means better communication skills, lower Communication Standard Score means worse communication skills. Standard Scores can range from 20 to 160. |
| Change From Baseline in Clinical Chemistry Labs (NA+, K+, Cl-) During Treatment. | Baseline; Week 4 | Clinical chemistry labs(sodium, potassium, chloride) |
| Change From Baseline in Laboratory Based Social Mimicry Abilities During Treatment. | Baseline; Week 4 | — |
| Change From Baseline in Blood Pressure After Treatment | Baseline; Week 4 | Sitting Systolic and Diastolic blood pressure. |
| Change From Baseline in Body Weight After Treatment. | Baseline; Week 4 | — |
| Change From Baseline in Body Temperature After Treatment | Baseline; Week 4 | — |
| Change From Baseline in the Awareness of Social Inference Test Revised (TASIT-R) Scores During Treatment. | Baseline, Week 4 | — |
| Change From Baseline in a Developmental Neuropsychological Assessment, Second Edition. (NEPSY-II) Affect Recognition Scores During Treatment. | Baseline; Week 4 | Higher Affect Recognition scores mean better affect recognition abilities, lower Affect Recognition scores mean worse affect recognition abilities. Scores can range from 1 to 19. |
| Change From Baseline in Plasma Vasopressin Levels During Treatment. | Baseline | There are no clinical laboratory tests that establish a normative range for vasopressin. Measurements prior to treatment were intended to evaluate vasopressin level as a predictor of response. Plasma vasopressin levels post treatment were not quantified. Baseline vasopressin levels are included in the outcome data below. |
| Change From Baseline in Parent Rated Pediatric Quality of Life (PedQL) Inventory Scores During Treatment. | Baseline; Week 4 | Higher scores mean better quality of life and lower scores mean worse quality of life (Range: Minimum=0; Maximum=100). |
Countries
United States
Participant flow
Recruitment details
This study was conducted in the Autism and Developmental Disorders Clinic in the Division of Child and Adolescent Psychiatry at Stanford University. Recruitment began in December 2013 and ended in May 2017.
Pre-assignment details
68 participants signed the consent form and enrolled in the study. Of those 68 participants, 27 participants did not meet the eligibility criteria for the study and 11 participants declined to participate prior to randomization. 30 participants were randomized.
Participants by arm
| Arm | Count |
|---|---|
| Placebo Placebo Nasal Spray
Placebo | 13 |
| Vasopressin Vasopressin Nasal Spray
Vasopressin: Participants aged 6 to 9.5 years of age will receive the maximum dose of 24 IU (12 IU twice daily). Participants aged 9.6 to 12 years of age will receive the maximum dose of 32 IU (16 IU twice daily). | 17 |
| Total | 30 |
Baseline characteristics
| Characteristic | Placebo | Vasopressin | Total |
|---|---|---|---|
| Age, Categorical <=18 years | 13 Participants | 17 Participants | 30 Participants |
| Age, Categorical >=65 years | 0 Participants | 0 Participants | 0 Participants |
| Age, Categorical Between 18 and 65 years | 0 Participants | 0 Participants | 0 Participants |
| Race/Ethnicity, Customized Caucasian | 11 Participants | 8 Participants | 19 Participants |
| Race/Ethnicity, Customized Other | 2 Participants | 9 Participants | 11 Participants |
| Region of Enrollment United States | 13 participants | 17 participants | 30 participants |
| Sex: Female, Male Female | 2 Participants | 3 Participants | 5 Participants |
| Sex: Female, Male Male | 11 Participants | 14 Participants | 25 Participants |
Adverse events
| Event type | EG000 affected / at risk | EG001 affected / at risk |
|---|---|---|
| deaths Total, all-cause mortality | 0 / 13 | 0 / 17 |
| other Total, other adverse events | 12 / 13 | 13 / 17 |
| serious Total, serious adverse events | 0 / 13 | 0 / 17 |
Outcome results
Primary
Change From Baseline in Parent Rated Social Responsiveness Scale, 2nd Edition (SRS-2) T-Score After Treatment.
Social Responsiveness Scale, 2nd Edition (SRS) scores measure social abilities with lower scores meaning better social abilities. (T-Score Range: 37- above 90 )
Time frame: Baseline; Week 4
Population: Participants who completed the protocol are included in the analysis.
| Arm | Measure | Value (LEAST_SQUARES_MEAN) | Dispersion |
|---|---|---|---|
| Placebo | Change From Baseline in Parent Rated Social Responsiveness Scale, 2nd Edition (SRS-2) T-Score After Treatment. | 10.8 units on a scale | Standard Error 2.11 |
| Vasopressin | Change From Baseline in Parent Rated Social Responsiveness Scale, 2nd Edition (SRS-2) T-Score After Treatment. | 17.6 units on a scale | Standard Error 1.37 |
Secondary
Change From Baseline in a Developmental Neuropsychological Assessment, Second Edition. (NEPSY-II) Affect Recognition Scores During Treatment.
Higher Affect Recognition scores mean better affect recognition abilities, lower Affect Recognition scores mean worse affect recognition abilities. Scores can range from 1 to 19.
Time frame: Baseline; Week 4
Population: Participants with available data were included.
| Arm | Measure | Value (LEAST_SQUARES_MEAN) | Dispersion |
|---|---|---|---|
| Placebo | Change From Baseline in a Developmental Neuropsychological Assessment, Second Edition. (NEPSY-II) Affect Recognition Scores During Treatment. | -2.58 units on a scale | Standard Error 1.9 |
| Vasopressin | Change From Baseline in a Developmental Neuropsychological Assessment, Second Edition. (NEPSY-II) Affect Recognition Scores During Treatment. | 0.094 units on a scale | Standard Error 2.2 |
Secondary
Change From Baseline in Blood Pressure After Treatment
Sitting Systolic and Diastolic blood pressure.
Time frame: Baseline; Week 4
Population: Participants with available data are included in the analysis.
| Arm | Measure | Group | Value (LEAST_SQUARES_MEAN) | Dispersion |
|---|---|---|---|---|
| Placebo | Change From Baseline in Blood Pressure After Treatment | Change from Baseline in Systolic Blood Pressure | -1.1 mmHG | Standard Error 2.89 |
| Placebo | Change From Baseline in Blood Pressure After Treatment | Change from Baseline in Diastolic Blood Pressure | -1.8 mmHG | Standard Error 2.39 |
| Vasopressin | Change From Baseline in Blood Pressure After Treatment | Change from Baseline in Systolic Blood Pressure | 5.5 mmHG | Standard Error 2.53 |
| Vasopressin | Change From Baseline in Blood Pressure After Treatment | Change from Baseline in Diastolic Blood Pressure | 3.2 mmHG | Standard Error 2.09 |
Secondary
Change From Baseline in Body Temperature After Treatment
Time frame: Baseline; Week 4
Population: Participants with available data are included in the analysis.
| Arm | Measure | Value (LEAST_SQUARES_MEAN) | Dispersion |
|---|---|---|---|
| Placebo | Change From Baseline in Body Temperature After Treatment | -0.2 Degrees (Farenheit) | Standard Error 0.19 |
| Vasopressin | Change From Baseline in Body Temperature After Treatment | 0.2 Degrees (Farenheit) | Standard Error 0.17 |
Secondary
Change From Baseline in Body Weight After Treatment.
Time frame: Baseline; Week 4
Population: Participants with available data are included in the analysis.
| Arm | Measure | Value (LEAST_SQUARES_MEAN) | Dispersion |
|---|---|---|---|
| Placebo | Change From Baseline in Body Weight After Treatment. | 0.2 kilograms | Standard Error 0.34 |
| Vasopressin | Change From Baseline in Body Weight After Treatment. | 0.3 kilograms | Standard Error 0.3 |
Secondary
Change From Baseline in Clinical Chemistry Labs (NA+, K+, Cl-) During Treatment.
Clinical chemistry labs(sodium, potassium, chloride)
Time frame: Baseline; Week 4
Population: Participants with available data are included in the analysis.
| Arm | Measure | Group | Value (LEAST_SQUARES_MEAN) | Dispersion |
|---|---|---|---|---|
| Placebo | Change From Baseline in Clinical Chemistry Labs (NA+, K+, Cl-) During Treatment. | Change From Baseline in Sodium | 0.15 mmol/L | Standard Error 0.72 |
| Placebo | Change From Baseline in Clinical Chemistry Labs (NA+, K+, Cl-) During Treatment. | Change from Baseline in Potassium | 0.06 mmol/L | Standard Error 0.13 |
| Placebo | Change From Baseline in Clinical Chemistry Labs (NA+, K+, Cl-) During Treatment. | Change from Baseline on Chloride | -0.31 mmol/L | Standard Error 0.64 |
| Vasopressin | Change From Baseline in Clinical Chemistry Labs (NA+, K+, Cl-) During Treatment. | Change From Baseline in Sodium | 0.24 mmol/L | Standard Error 0.63 |
| Vasopressin | Change From Baseline in Clinical Chemistry Labs (NA+, K+, Cl-) During Treatment. | Change from Baseline in Potassium | 0.15 mmol/L | Standard Error 0.11 |
| Vasopressin | Change From Baseline in Clinical Chemistry Labs (NA+, K+, Cl-) During Treatment. | Change from Baseline on Chloride | 1.0 mmol/L | Standard Error 0.56 |
Secondary
Change From Baseline in Clinical Global Impression (CGI) Severity, Social and Communication Scores During Treatment.
Higher Scores on the CGI severity scale mean more greater social and communication deficits (Range 1-7)
Time frame: Baseline; Week 4
Population: Participants with available data are included.
| Arm | Measure | Value (LEAST_SQUARES_MEAN) | Dispersion |
|---|---|---|---|
| Placebo | Change From Baseline in Clinical Global Impression (CGI) Severity, Social and Communication Scores During Treatment. | 0.712 units on a scale | Standard Error 0.202 |
| Vasopressin | Change From Baseline in Clinical Global Impression (CGI) Severity, Social and Communication Scores During Treatment. | 0.873 units on a scale | Standard Error 0.126 |
Secondary
Change From Baseline in Heart Rate After Treatment.
Sitting heart rate (beats per minute).
Time frame: Baseline; Week 4
Population: Participants who completed the protocol are included in the analysis.
| Arm | Measure | Value (LEAST_SQUARES_MEAN) | Dispersion |
|---|---|---|---|
| Placebo | Change From Baseline in Heart Rate After Treatment. | 7.5 Beats per minute | Standard Error 4.43 |
| Vasopressin | Change From Baseline in Heart Rate After Treatment. | 1.9 Beats per minute | Standard Error 3.87 |
Secondary
Change From Baseline in Laboratory Based Eye-gaze to Social Cues During Treatment.
Time frame: Baseline; Week 4
Population: Data were not not collected for this outcome.
Secondary
Change From Baseline in Laboratory Based Facial Emotion Recognition Abilities During Treatment.
Higher scores mean better facial emotion recognition abilities. Lower scores mean worse facial emotion recognition abilities (Range: 0-42).
Time frame: Baseline; Week 4
Population: Participants with available data are included in the analysis.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Placebo | Change From Baseline in Laboratory Based Facial Emotion Recognition Abilities During Treatment. | -7.19 units on a scale | Standard Error 1.81 |
| Vasopressin | Change From Baseline in Laboratory Based Facial Emotion Recognition Abilities During Treatment. | 3.10 units on a scale | Standard Error 2.42 |
Secondary
Change From Baseline in Laboratory Based Social Mimicry Abilities During Treatment.
Time frame: Baseline; Week 4
Population: Data were not not collected for this outcome.
Secondary
Change From Baseline in Parent Rated Aberrant Behavior Checklist (ABC) Scores During Treatment.
Higher scores indicate more symptoms, lower scores indicate fewer symptoms. Irritability scores can range from 0-45. Lethargy scores can range from 0-48. Stereotypy scores can range from 0-21. Hyperactivity scores can range from 0-48. Inappropriate speech scores can from 0-12.
Time frame: Baseline; Week 4
Population: Participants with available data were included in analysis.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Placebo | Change From Baseline in Parent Rated Aberrant Behavior Checklist (ABC) Scores During Treatment. | Irritability Baseline | 14.00 units on a scale | Standard Deviation 10.23 |
| Placebo | Change From Baseline in Parent Rated Aberrant Behavior Checklist (ABC) Scores During Treatment. | Irritability Week 4 | 8.38 units on a scale | Standard Deviation 7.23 |
| Placebo | Change From Baseline in Parent Rated Aberrant Behavior Checklist (ABC) Scores During Treatment. | Lethargy Baseline | 14.77 units on a scale | Standard Deviation 10.69 |
| Placebo | Change From Baseline in Parent Rated Aberrant Behavior Checklist (ABC) Scores During Treatment. | Lethargy Week 4 | 12.77 units on a scale | Standard Deviation 9.81 |
| Placebo | Change From Baseline in Parent Rated Aberrant Behavior Checklist (ABC) Scores During Treatment. | Stereotypy Baseline | 5.08 units on a scale | Standard Deviation 5.36 |
| Placebo | Change From Baseline in Parent Rated Aberrant Behavior Checklist (ABC) Scores During Treatment. | Stereotypy Week 4 | 4.54 units on a scale | Standard Deviation 4.91 |
| Placebo | Change From Baseline in Parent Rated Aberrant Behavior Checklist (ABC) Scores During Treatment. | Hyperactivity Baseline | 21.38 units on a scale | Standard Deviation 9.17 |
| Placebo | Change From Baseline in Parent Rated Aberrant Behavior Checklist (ABC) Scores During Treatment. | Hyperactivity Week 4 | 14.54 units on a scale | Standard Deviation 7.34 |
| Placebo | Change From Baseline in Parent Rated Aberrant Behavior Checklist (ABC) Scores During Treatment. | Inappropriate Speech Baseline | 4.62 units on a scale | Standard Deviation 3.74 |
| Placebo | Change From Baseline in Parent Rated Aberrant Behavior Checklist (ABC) Scores During Treatment. | Inappropriate Speech Week 4 | 3.69 units on a scale | Standard Deviation 3.52 |
| Vasopressin | Change From Baseline in Parent Rated Aberrant Behavior Checklist (ABC) Scores During Treatment. | Hyperactivity Week 4 | 12.29 units on a scale | Standard Deviation 9.06 |
| Vasopressin | Change From Baseline in Parent Rated Aberrant Behavior Checklist (ABC) Scores During Treatment. | Irritability Baseline | 8.29 units on a scale | Standard Deviation 7.4 |
| Vasopressin | Change From Baseline in Parent Rated Aberrant Behavior Checklist (ABC) Scores During Treatment. | Stereotypy Week 4 | 3.53 units on a scale | Standard Deviation 3.59 |
| Vasopressin | Change From Baseline in Parent Rated Aberrant Behavior Checklist (ABC) Scores During Treatment. | Irritability Week 4 | 6.24 units on a scale | Standard Deviation 7.05 |
| Vasopressin | Change From Baseline in Parent Rated Aberrant Behavior Checklist (ABC) Scores During Treatment. | Inappropriate Speech Week 4 | 3.41 units on a scale | Standard Deviation 3.08 |
| Vasopressin | Change From Baseline in Parent Rated Aberrant Behavior Checklist (ABC) Scores During Treatment. | Lethargy Baseline | 10.76 units on a scale | Standard Deviation 6.32 |
| Vasopressin | Change From Baseline in Parent Rated Aberrant Behavior Checklist (ABC) Scores During Treatment. | Hyperactivity Baseline | 16.00 units on a scale | Standard Deviation 11.69 |
| Vasopressin | Change From Baseline in Parent Rated Aberrant Behavior Checklist (ABC) Scores During Treatment. | Lethargy Week 4 | 7.41 units on a scale | Standard Deviation 6.88 |
| Vasopressin | Change From Baseline in Parent Rated Aberrant Behavior Checklist (ABC) Scores During Treatment. | Inappropriate Speech Baseline | 3.94 units on a scale | Standard Deviation 2.86 |
| Vasopressin | Change From Baseline in Parent Rated Aberrant Behavior Checklist (ABC) Scores During Treatment. | Stereotypy Baseline | 5.18 units on a scale | Standard Deviation 3.88 |
Secondary
Change From Baseline in Parent Rated Pediatric Quality of Life (PedQL) Inventory Scores During Treatment.
Higher scores mean better quality of life and lower scores mean worse quality of life (Range: Minimum=0; Maximum=100).
Time frame: Baseline; Week 4
Population: Participants with available data were analyzed.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Placebo | Change From Baseline in Parent Rated Pediatric Quality of Life (PedQL) Inventory Scores During Treatment. | Baseline | 57.14 units on a scale | Standard Deviation 16.67 |
| Placebo | Change From Baseline in Parent Rated Pediatric Quality of Life (PedQL) Inventory Scores During Treatment. | Week 4 | 65.96 units on a scale | Standard Deviation 14.93 |
| Vasopressin | Change From Baseline in Parent Rated Pediatric Quality of Life (PedQL) Inventory Scores During Treatment. | Baseline | 64.53 units on a scale | Standard Deviation 13.86 |
| Vasopressin | Change From Baseline in Parent Rated Pediatric Quality of Life (PedQL) Inventory Scores During Treatment. | Week 4 | 74.52 units on a scale | Standard Deviation 15.38 |
Secondary
Change From Baseline in Parent Rated Repetitive Behavior Scale Revised (RBS-R) Scores During Treatment.
Higher scores on the Repetitive Behavior Scale- Revised mean higher levels of repetitive and restricted behaviors. (Raw Score Total Range: 0 - 129)
Time frame: Baseline; Week 4
Population: Participants with available data were analyzed.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Placebo | Change From Baseline in Parent Rated Repetitive Behavior Scale Revised (RBS-R) Scores During Treatment. | 21.3 units on a scale | Standard Error 3.18 |
| Vasopressin | Change From Baseline in Parent Rated Repetitive Behavior Scale Revised (RBS-R) Scores During Treatment. | 17.2 units on a scale | Standard Error 1.83 |
Secondary
Change From Baseline in Parent Rated Spence Children's Anxiety Scale (SCAS) During Treatment.
Scale measuring severity of anxiety symptoms. Higher scores mean higher levels of anxiety, lower scores mean lower levels of anxiety. (Raw Score Range: 0 - 114)
Time frame: Baseline; Week 4
Population: Participants with available data were analyzed.
| Arm | Measure | Value (LEAST_SQUARES_MEAN) | Dispersion |
|---|---|---|---|
| Placebo | Change From Baseline in Parent Rated Spence Children's Anxiety Scale (SCAS) During Treatment. | 9.14 units on a scale | Standard Error 2.68 |
| Vasopressin | Change From Baseline in Parent Rated Spence Children's Anxiety Scale (SCAS) During Treatment. | 17.9 units on a scale | Standard Error 1.66 |
Secondary
Change From Baseline in Parent Rated Vineland Adaptive Behavior Scales Second Edition (VABS-II) - Social and Communication Subscales During Treatment.
Higher Social Standard Score means better social skills, lower Social Standard Score means worse social skills. Higher Communication Standard Score means better communication skills, lower Communication Standard Score means worse communication skills. Standard Scores can range from 20 to 160.
Time frame: Baseline; Week 4
Population: Not all participants were able to fully complete the measure so not all of the numbers analyzed are the same.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Placebo | Change From Baseline in Parent Rated Vineland Adaptive Behavior Scales Second Edition (VABS-II) - Social and Communication Subscales During Treatment. | Baseline Social Standard Score | 61.33 units on a scale | Standard Deviation 13.93 |
| Placebo | Change From Baseline in Parent Rated Vineland Adaptive Behavior Scales Second Edition (VABS-II) - Social and Communication Subscales During Treatment. | Week 4 Social Standard Score | 62.25 units on a scale | Standard Deviation 7.78 |
| Placebo | Change From Baseline in Parent Rated Vineland Adaptive Behavior Scales Second Edition (VABS-II) - Social and Communication Subscales During Treatment. | Baseline Communication Standard Score | 75.67 units on a scale | Standard Deviation 13.57 |
| Placebo | Change From Baseline in Parent Rated Vineland Adaptive Behavior Scales Second Edition (VABS-II) - Social and Communication Subscales During Treatment. | Week 4 Communication Standard Score | 77.40 units on a scale | Standard Deviation 10.55 |
| Vasopressin | Change From Baseline in Parent Rated Vineland Adaptive Behavior Scales Second Edition (VABS-II) - Social and Communication Subscales During Treatment. | Week 4 Communication Standard Score | 81.12 units on a scale | Standard Deviation 14.26 |
| Vasopressin | Change From Baseline in Parent Rated Vineland Adaptive Behavior Scales Second Edition (VABS-II) - Social and Communication Subscales During Treatment. | Baseline Social Standard Score | 71.08 units on a scale | Standard Deviation 14.38 |
| Vasopressin | Change From Baseline in Parent Rated Vineland Adaptive Behavior Scales Second Edition (VABS-II) - Social and Communication Subscales During Treatment. | Baseline Communication Standard Score | 74.77 units on a scale | Standard Deviation 8.6 |
| Vasopressin | Change From Baseline in Parent Rated Vineland Adaptive Behavior Scales Second Edition (VABS-II) - Social and Communication Subscales During Treatment. | Week 4 Social Standard Score | 79.67 units on a scale | Standard Deviation 16.83 |
Secondary
Change From Baseline in Plasma Vasopressin Levels During Treatment.
There are no clinical laboratory tests that establish a normative range for vasopressin. Measurements prior to treatment were intended to evaluate vasopressin level as a predictor of response. Plasma vasopressin levels post treatment were not quantified. Baseline vasopressin levels are included in the outcome data below.
Time frame: Baseline
Population: Participants with available data were included in the analysis.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Placebo | Change From Baseline in Plasma Vasopressin Levels During Treatment. | 1.28 pg/ml | Standard Error 0.29 |
| Vasopressin | Change From Baseline in Plasma Vasopressin Levels During Treatment. | 1.32 pg/ml | Standard Error 0.25 |
Secondary
Change From Baseline in Reading the Mind in the Eyes Test, Child Version (RMET-child) Scores During Treatment.
Higher scores mean better ability to read emotions and lower scores mean worse ability to read emotions. Range 0-28.
Time frame: Baseline; Week 4
Population: Participants with available data are included in the analysis.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Placebo | Change From Baseline in Reading the Mind in the Eyes Test, Child Version (RMET-child) Scores During Treatment. | -1.28 units on a scale | Standard Error 1.24 |
| Vasopressin | Change From Baseline in Reading the Mind in the Eyes Test, Child Version (RMET-child) Scores During Treatment. | 4.04 units on a scale | Standard Error 1.63 |
Secondary
Change From Baseline in the Awareness of Social Inference Test Revised (TASIT-R) Scores During Treatment.
Time frame: Baseline, Week 4
Population: Data were not not collected for this outcome.
Secondary
Change From Baseline on the Overt Aggression Scale (OAS) During Treatment.
Count of participants reporting an increase of aggression during treatment compared to baseline (pretreatment).
Time frame: Baseline through Week 4
Population: Participants with available data are included in the analysis.
| Arm | Measure | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|
| Placebo | Change From Baseline on the Overt Aggression Scale (OAS) During Treatment. | 1 Participants |
| Vasopressin | Change From Baseline on the Overt Aggression Scale (OAS) During Treatment. | 1 Participants |
Secondary
Number of Participants With Side Effects Assessed Using Parent Rated Dosage Record Treatment Emergent Symptom Scale (DOTES) Scores During Treatment
Dosage Record Treatment Emergent Symptom Scale (DOTES) side effects reported by parents during 4-weeks of treatment. Participant Counts are used.
Time frame: Baseline through Week 4
Population: Participants who completed the protocol are included in the analysis.
| Arm | Measure | Group | Value (NUMBER) |
|---|---|---|---|
| Placebo | Number of Participants With Side Effects Assessed Using Parent Rated Dosage Record Treatment Emergent Symptom Scale (DOTES) Scores During Treatment | Nasal Congestion | 4 participants |
| Placebo | Number of Participants With Side Effects Assessed Using Parent Rated Dosage Record Treatment Emergent Symptom Scale (DOTES) Scores During Treatment | Fever | 1 participants |
| Placebo | Number of Participants With Side Effects Assessed Using Parent Rated Dosage Record Treatment Emergent Symptom Scale (DOTES) Scores During Treatment | Dry Mouth | 3 participants |
| Placebo | Number of Participants With Side Effects Assessed Using Parent Rated Dosage Record Treatment Emergent Symptom Scale (DOTES) Scores During Treatment | Drowsiness | 3 participants |
| Placebo | Number of Participants With Side Effects Assessed Using Parent Rated Dosage Record Treatment Emergent Symptom Scale (DOTES) Scores During Treatment | Blurred Vision | 1 participants |
| Placebo | Number of Participants With Side Effects Assessed Using Parent Rated Dosage Record Treatment Emergent Symptom Scale (DOTES) Scores During Treatment | Insomnia | 1 participants |
| Placebo | Number of Participants With Side Effects Assessed Using Parent Rated Dosage Record Treatment Emergent Symptom Scale (DOTES) Scores During Treatment | Ear Infection | 1 participants |
| Placebo | Number of Participants With Side Effects Assessed Using Parent Rated Dosage Record Treatment Emergent Symptom Scale (DOTES) Scores During Treatment | Decreased Motor Activity | 2 participants |
| Placebo | Number of Participants With Side Effects Assessed Using Parent Rated Dosage Record Treatment Emergent Symptom Scale (DOTES) Scores During Treatment | Runny Nose | 1 participants |
| Placebo | Number of Participants With Side Effects Assessed Using Parent Rated Dosage Record Treatment Emergent Symptom Scale (DOTES) Scores During Treatment | Body Ache | 0 participants |
| Placebo | Number of Participants With Side Effects Assessed Using Parent Rated Dosage Record Treatment Emergent Symptom Scale (DOTES) Scores During Treatment | Sore Throat | 1 participants |
| Placebo | Number of Participants With Side Effects Assessed Using Parent Rated Dosage Record Treatment Emergent Symptom Scale (DOTES) Scores During Treatment | Aggression | 1 participants |
| Placebo | Number of Participants With Side Effects Assessed Using Parent Rated Dosage Record Treatment Emergent Symptom Scale (DOTES) Scores During Treatment | Cold Sore | 1 participants |
| Placebo | Number of Participants With Side Effects Assessed Using Parent Rated Dosage Record Treatment Emergent Symptom Scale (DOTES) Scores During Treatment | Increased Motor Activity | 0 participants |
| Placebo | Number of Participants With Side Effects Assessed Using Parent Rated Dosage Record Treatment Emergent Symptom Scale (DOTES) Scores During Treatment | Decreased Appetite | 5 participants |
| Placebo | Number of Participants With Side Effects Assessed Using Parent Rated Dosage Record Treatment Emergent Symptom Scale (DOTES) Scores During Treatment | Akathisia | 0 participants |
| Placebo | Number of Participants With Side Effects Assessed Using Parent Rated Dosage Record Treatment Emergent Symptom Scale (DOTES) Scores During Treatment | Nausea/Vomiting | 2 participants |
| Placebo | Number of Participants With Side Effects Assessed Using Parent Rated Dosage Record Treatment Emergent Symptom Scale (DOTES) Scores During Treatment | Cough | 0 participants |
| Placebo | Number of Participants With Side Effects Assessed Using Parent Rated Dosage Record Treatment Emergent Symptom Scale (DOTES) Scores During Treatment | Constipation | 0 participants |
| Placebo | Number of Participants With Side Effects Assessed Using Parent Rated Dosage Record Treatment Emergent Symptom Scale (DOTES) Scores During Treatment | Head Banging | 0 participants |
| Placebo | Number of Participants With Side Effects Assessed Using Parent Rated Dosage Record Treatment Emergent Symptom Scale (DOTES) Scores During Treatment | Diarrhea | 1 participants |
| Placebo | Number of Participants With Side Effects Assessed Using Parent Rated Dosage Record Treatment Emergent Symptom Scale (DOTES) Scores During Treatment | Depressive Affect | 1 participants |
| Placebo | Number of Participants With Side Effects Assessed Using Parent Rated Dosage Record Treatment Emergent Symptom Scale (DOTES) Scores During Treatment | Increased Urination | 1 participants |
| Placebo | Number of Participants With Side Effects Assessed Using Parent Rated Dosage Record Treatment Emergent Symptom Scale (DOTES) Scores During Treatment | Dizziness | 1 participants |
| Placebo | Number of Participants With Side Effects Assessed Using Parent Rated Dosage Record Treatment Emergent Symptom Scale (DOTES) Scores During Treatment | Bed Wetting | 0 participants |
| Placebo | Number of Participants With Side Effects Assessed Using Parent Rated Dosage Record Treatment Emergent Symptom Scale (DOTES) Scores During Treatment | Excitement/Agitation | 1 participants |
| Placebo | Number of Participants With Side Effects Assessed Using Parent Rated Dosage Record Treatment Emergent Symptom Scale (DOTES) Scores During Treatment | Skin Rash | 1 participants |
| Placebo | Number of Participants With Side Effects Assessed Using Parent Rated Dosage Record Treatment Emergent Symptom Scale (DOTES) Scores During Treatment | Lethargy/Tiredness | 1 participants |
| Placebo | Number of Participants With Side Effects Assessed Using Parent Rated Dosage Record Treatment Emergent Symptom Scale (DOTES) Scores During Treatment | Bug Bite | 0 participants |
| Placebo | Number of Participants With Side Effects Assessed Using Parent Rated Dosage Record Treatment Emergent Symptom Scale (DOTES) Scores During Treatment | Skin Burn | 1 participants |
| Placebo | Number of Participants With Side Effects Assessed Using Parent Rated Dosage Record Treatment Emergent Symptom Scale (DOTES) Scores During Treatment | Headache | 0 participants |
| Vasopressin | Number of Participants With Side Effects Assessed Using Parent Rated Dosage Record Treatment Emergent Symptom Scale (DOTES) Scores During Treatment | Skin Burn | 0 participants |
| Vasopressin | Number of Participants With Side Effects Assessed Using Parent Rated Dosage Record Treatment Emergent Symptom Scale (DOTES) Scores During Treatment | Fever | 2 participants |
| Vasopressin | Number of Participants With Side Effects Assessed Using Parent Rated Dosage Record Treatment Emergent Symptom Scale (DOTES) Scores During Treatment | Cough | 1 participants |
| Vasopressin | Number of Participants With Side Effects Assessed Using Parent Rated Dosage Record Treatment Emergent Symptom Scale (DOTES) Scores During Treatment | Body Ache | 1 participants |
| Vasopressin | Number of Participants With Side Effects Assessed Using Parent Rated Dosage Record Treatment Emergent Symptom Scale (DOTES) Scores During Treatment | Excitement/Agitation | 0 participants |
| Vasopressin | Number of Participants With Side Effects Assessed Using Parent Rated Dosage Record Treatment Emergent Symptom Scale (DOTES) Scores During Treatment | Insomnia | 4 participants |
| Vasopressin | Number of Participants With Side Effects Assessed Using Parent Rated Dosage Record Treatment Emergent Symptom Scale (DOTES) Scores During Treatment | Increased Motor Activity | 4 participants |
| Vasopressin | Number of Participants With Side Effects Assessed Using Parent Rated Dosage Record Treatment Emergent Symptom Scale (DOTES) Scores During Treatment | Depressive Affect | 2 participants |
| Vasopressin | Number of Participants With Side Effects Assessed Using Parent Rated Dosage Record Treatment Emergent Symptom Scale (DOTES) Scores During Treatment | Headache | 2 participants |
| Vasopressin | Number of Participants With Side Effects Assessed Using Parent Rated Dosage Record Treatment Emergent Symptom Scale (DOTES) Scores During Treatment | Drowsiness | 1 participants |
| Vasopressin | Number of Participants With Side Effects Assessed Using Parent Rated Dosage Record Treatment Emergent Symptom Scale (DOTES) Scores During Treatment | Decreased Motor Activity | 1 participants |
| Vasopressin | Number of Participants With Side Effects Assessed Using Parent Rated Dosage Record Treatment Emergent Symptom Scale (DOTES) Scores During Treatment | Aggression | 1 participants |
| Vasopressin | Number of Participants With Side Effects Assessed Using Parent Rated Dosage Record Treatment Emergent Symptom Scale (DOTES) Scores During Treatment | Akathisia | 1 participants |
| Vasopressin | Number of Participants With Side Effects Assessed Using Parent Rated Dosage Record Treatment Emergent Symptom Scale (DOTES) Scores During Treatment | Head Banging | 1 participants |
| Vasopressin | Number of Participants With Side Effects Assessed Using Parent Rated Dosage Record Treatment Emergent Symptom Scale (DOTES) Scores During Treatment | Dizziness | 0 participants |
| Vasopressin | Number of Participants With Side Effects Assessed Using Parent Rated Dosage Record Treatment Emergent Symptom Scale (DOTES) Scores During Treatment | Lethargy/Tiredness | 0 participants |
| Vasopressin | Number of Participants With Side Effects Assessed Using Parent Rated Dosage Record Treatment Emergent Symptom Scale (DOTES) Scores During Treatment | Nasal Congestion | 3 participants |
| Vasopressin | Number of Participants With Side Effects Assessed Using Parent Rated Dosage Record Treatment Emergent Symptom Scale (DOTES) Scores During Treatment | Dry Mouth | 1 participants |
| Vasopressin | Number of Participants With Side Effects Assessed Using Parent Rated Dosage Record Treatment Emergent Symptom Scale (DOTES) Scores During Treatment | Blurred Vision | 1 participants |
| Vasopressin | Number of Participants With Side Effects Assessed Using Parent Rated Dosage Record Treatment Emergent Symptom Scale (DOTES) Scores During Treatment | Ear Infection | 0 participants |
| Vasopressin | Number of Participants With Side Effects Assessed Using Parent Rated Dosage Record Treatment Emergent Symptom Scale (DOTES) Scores During Treatment | Runny Nose | 0 participants |
| Vasopressin | Number of Participants With Side Effects Assessed Using Parent Rated Dosage Record Treatment Emergent Symptom Scale (DOTES) Scores During Treatment | Sore Throat | 0 participants |
| Vasopressin | Number of Participants With Side Effects Assessed Using Parent Rated Dosage Record Treatment Emergent Symptom Scale (DOTES) Scores During Treatment | Cold Sore | 0 participants |
| Vasopressin | Number of Participants With Side Effects Assessed Using Parent Rated Dosage Record Treatment Emergent Symptom Scale (DOTES) Scores During Treatment | Decreased Appetite | 4 participants |
| Vasopressin | Number of Participants With Side Effects Assessed Using Parent Rated Dosage Record Treatment Emergent Symptom Scale (DOTES) Scores During Treatment | Nausea/Vomiting | 2 participants |
| Vasopressin | Number of Participants With Side Effects Assessed Using Parent Rated Dosage Record Treatment Emergent Symptom Scale (DOTES) Scores During Treatment | Constipation | 1 participants |
| Vasopressin | Number of Participants With Side Effects Assessed Using Parent Rated Dosage Record Treatment Emergent Symptom Scale (DOTES) Scores During Treatment | Diarrhea | 0 participants |
| Vasopressin | Number of Participants With Side Effects Assessed Using Parent Rated Dosage Record Treatment Emergent Symptom Scale (DOTES) Scores During Treatment | Increased Urination | 1 participants |
| Vasopressin | Number of Participants With Side Effects Assessed Using Parent Rated Dosage Record Treatment Emergent Symptom Scale (DOTES) Scores During Treatment | Bed Wetting | 1 participants |
| Vasopressin | Number of Participants With Side Effects Assessed Using Parent Rated Dosage Record Treatment Emergent Symptom Scale (DOTES) Scores During Treatment | Skin Rash | 1 participants |
| Vasopressin | Number of Participants With Side Effects Assessed Using Parent Rated Dosage Record Treatment Emergent Symptom Scale (DOTES) Scores During Treatment | Bug Bite | 1 participants |