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| ID | Type | Description | Link |
|---|---|---|---|
| R21MH100387-01 | U.S. NIH Grant/Contract | View source |
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| Name | Class |
|---|---|
| National Institute of Mental Health (NIMH) | NIH |
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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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Placebo | Placebo Comparator | Placebo Nasal Spray |
|
| Vasopressin | Active Comparator | Vasopressin Nasal Spray |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Vasopressin | Drug | 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). |
| Measure | Description | Time Frame |
|---|---|---|
| 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 ) | Baseline; Week 4 |
| Measure | Description | Time Frame |
|---|---|---|
| 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) | Baseline; Week 4 |
| Change From Baseline in Reading the Mind in the Eyes Test, Child Version (RMET-child) Scores During Treatment. |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Antonio Y Hardan, MD | Stanford University | Principal Investigator |
| Karen J Parker, PhD | Stanford University | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Stanford University School of Medicine; Psychiatry and Behavioral Sciences | Stanford | California | 94305 | United States |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 37811711 | Derived | Iffland M, Livingstone N, Jorgensen M, Hazell P, Gillies D. Pharmacological intervention for irritability, aggression, and self-injury in autism spectrum disorder (ASD). Cochrane Database Syst Rev. 2023 Oct 9;10(10):CD011769. doi: 10.1002/14651858.CD011769.pub2. |
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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.
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.
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| ID | Title | Description |
|---|---|---|
| FG000 | Placebo | Placebo Nasal Spray Placebo |
| FG001 | 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). |
| Title | Milestones | Reasons Not Completed | ||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Overall Study |
|
Participants who were randomized in the trial.
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| ID | Title | Description |
|---|---|---|
| BG000 | Placebo | Placebo Nasal Spray Placebo |
| BG001 | 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). |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes |
|---|---|---|---|---|---|---|---|---|---|
| Age, Categorical | Count of Participants |
| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | ||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| 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 ) | Participants who completed the protocol are included in the analysis. | Posted | Least Squares Mean | Standard Error | units on a scale | Baseline; Week 4 |
|
4 weeks
Dosage Record Treatment Emergent Symptom Scale (DOTES) and Overt Aggression Scale (OAS) were used to assess side effects.
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| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | Placebo | Placebo Nasal Spray Placebo | 0 |
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| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Fever | General disorders | Systematic Assessment |
The final sample was 83% male and was not statistically powered to detect sex differences in treatment response. Participants were permitted to take other medications during the intervention. Many of our outcome measures relied on parent report.
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Antonio Hardan, MD | Stanford University | (650) 736-1235 | hardanay@stanford.edu |
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| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| SAP | No | Yes | No | Statistical Analysis Plan | Jan 11, 2019 | Mar 21, 2019 | SAP_000.pdf |
| Prot | Yes | No | No | Study Protocol | Aug 2, 2016 | Mar 26, 2019 | Prot_001.pdf |
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| ID | Term |
|---|---|
| D000067877 | Autism Spectrum Disorder |
| ID | Term |
|---|---|
| D002659 | Child Development Disorders, Pervasive |
| D065886 | Neurodevelopmental Disorders |
| D001523 | Mental Disorders |
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| ID | Term |
|---|---|
| D014667 | Vasopressins |
| ID | Term |
|---|---|
| D010909 | Pituitary Hormones, Posterior |
| D010907 | Pituitary Hormones |
| D036361 | Peptide Hormones |
| D006728 | Hormones |
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| Placebo | Drug |
|
Higher scores mean better ability to read emotions and lower scores mean worse ability to read emotions. Range 0-28. |
| Baseline; Week 4 |
| 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). | Baseline; Week 4 |
| 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) | Baseline; Week 4 |
| 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) | Baseline; Week 4 |
| 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. | Baseline through Week 4 |
| 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). | Baseline through Week 4 |
| Change From Baseline in Heart Rate After Treatment. | Sitting heart rate (beats per minute). | Baseline; Week 4 |
| 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. | Baseline; Week 4 |
| 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). | Baseline; Week 4 |
| 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. | Baseline; Week 4 |
| Change From Baseline in Clinical Chemistry Labs (NA+, K+, Cl-) During Treatment. | Clinical chemistry labs(sodium, potassium, chloride) | Baseline; Week 4 |
| Change From Baseline in Laboratory Based Eye-gaze to Social Cues During Treatment. | Baseline; Week 4 |
| Change From Baseline in Laboratory Based Social Mimicry Abilities During Treatment. | Baseline; Week 4 |
| Change From Baseline in Blood Pressure After Treatment | Sitting Systolic and Diastolic blood pressure. | Baseline; Week 4 |
| 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. | 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. | Baseline; Week 4 |
| 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. | Baseline |
| BG002 | Total | Total of all reporting groups |
| Participants |
|
| Sex: Female, Male | Count of Participants | Participants |
|
| Race/Ethnicity, Customized | Count of Participants | Participants |
|
| Region of Enrollment | Number | participants |
|
|
|
| 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) | Participants with available data are included. | Posted | Least Squares Mean | Standard Error | units on a scale | Baseline; Week 4 |
|
|
|
| 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. | Participants with available data are included in the analysis. | Posted | Mean | Standard Error | units on a scale | Baseline; Week 4 |
|
|
|
| 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). | Participants with available data are included in the analysis. | Posted | Mean | Standard Error | units on a scale | Baseline; Week 4 |
|
|
|
| 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) | Participants with available data were analyzed. | Posted | Mean | Standard Error | units on a scale | Baseline; Week 4 |
|
|
|
| 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) | Participants with available data were analyzed. | Posted | Least Squares Mean | Standard Error | units on a scale | Baseline; Week 4 |
|
|
|
| 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. | Participants who completed the protocol are included in the analysis. | Posted | Number | participants | Baseline through Week 4 |
|
|
|
| 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). | Participants with available data are included in the analysis. | Posted | Count of Participants | Participants | Baseline through Week 4 |
|
|
|
| Secondary | Change From Baseline in Heart Rate After Treatment. | Sitting heart rate (beats per minute). | Participants who completed the protocol are included in the analysis. | Posted | Least Squares Mean | Standard Error | Beats per minute | Baseline; Week 4 |
|
|
|
| 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. | Participants with available data were included in analysis. | Posted | Mean | Standard Deviation | units on a scale | Baseline; Week 4 |
|
|
|
| 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). | Participants with available data were analyzed. | Posted | Mean | Standard Deviation | units on a scale | Baseline; Week 4 |
|
|
|
| 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. | Not all participants were able to fully complete the measure so not all of the numbers analyzed are the same. | Posted | Mean | Standard Deviation | units on a scale | Baseline; Week 4 |
|
|
|
| Secondary | Change From Baseline in Clinical Chemistry Labs (NA+, K+, Cl-) During Treatment. | Clinical chemistry labs(sodium, potassium, chloride) | Participants with available data are included in the analysis. | Posted | Least Squares Mean | Standard Error | mmol/L | Baseline; Week 4 |
|
|
|
| Secondary | Change From Baseline in Laboratory Based Eye-gaze to Social Cues During Treatment. | Data were not not collected for this outcome. | Posted | Baseline; Week 4 |
|
|
| Secondary | Change From Baseline in Laboratory Based Social Mimicry Abilities During Treatment. | Data were not not collected for this outcome. | Posted | Baseline; Week 4 |
|
|
| Secondary | Change From Baseline in Blood Pressure After Treatment | Sitting Systolic and Diastolic blood pressure. | Participants with available data are included in the analysis. | Posted | Least Squares Mean | Standard Error | mmHG | Baseline; Week 4 |
|
|
|
| Secondary | Change From Baseline in Body Weight After Treatment. | Participants with available data are included in the analysis. | Posted | Least Squares Mean | Standard Error | kilograms | Baseline; Week 4 |
|
|
|
| Secondary | Change From Baseline in Body Temperature After Treatment | Participants with available data are included in the analysis. | Posted | Least Squares Mean | Standard Error | Degrees (Farenheit) | Baseline; Week 4 |
|
|
|
| Secondary | Change From Baseline in the Awareness of Social Inference Test Revised (TASIT-R) Scores During Treatment. | Data were not not collected for this outcome. | Posted | Baseline, Week 4 |
|
|
| 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. | Participants with available data were included. | Posted | Least Squares Mean | Standard Error | units on a scale | Baseline; Week 4 |
|
|
|
| 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. | Participants with available data were included in the analysis. | Posted | Mean | Standard Error | pg/ml | Baseline |
|
|
|
| 13 |
| 0 |
| 13 |
| 12 |
| 13 |
| EG001 | 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). | 0 | 17 | 0 | 17 | 13 | 17 |
| Cough | General disorders | Systematic Assessment |
|
| Body Ache | General disorders | Systematic Assessment |
|
| Excitement/Agitation | Psychiatric disorders | Systematic Assessment |
|
| Insomnia | Nervous system disorders | Systematic Assessment |
|
| Increased Motor Activity | Nervous system disorders | Systematic Assessment |
|
| Depressive Affect | Psychiatric disorders | Systematic Assessment |
|
| Headache | Nervous system disorders | Systematic Assessment |
|
| Drowsiness | Nervous system disorders | Systematic Assessment |
|
| Decreased Motor Activity | Nervous system disorders | Systematic Assessment |
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| Aggression | Psychiatric disorders | Systematic Assessment |
|
| Akathisia | Nervous system disorders | Systematic Assessment |
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| Head Banging | Psychiatric disorders | Systematic Assessment |
|
| Dizziness | Nervous system disorders | Systematic Assessment |
|
| Lethargy/Tiredness | Nervous system disorders | Systematic Assessment |
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| Nasal Congestion | General disorders | Systematic Assessment |
|
| Dry Mouth | General disorders | Systematic Assessment |
|
| Blurred Vision | Eye disorders | Systematic Assessment |
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| Ear Infection | Ear and labyrinth disorders | Systematic Assessment |
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| Runny Nose | General disorders | Systematic Assessment |
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| Sore Throat | General disorders | Systematic Assessment |
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| Cold Sore | Skin and subcutaneous tissue disorders | Systematic Assessment |
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| Decreased Appetite | Gastrointestinal disorders | Systematic Assessment |
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| Nausea/Vomiting | Gastrointestinal disorders | Systematic Assessment |
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| Constipation | Gastrointestinal disorders | Systematic Assessment |
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| Diarrhea | Gastrointestinal disorders | Systematic Assessment |
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| Increased Urination | General disorders | Systematic Assessment |
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| Bed Wetting | General disorders | Systematic Assessment |
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| Skin Rash | Skin and subcutaneous tissue disorders | Systematic Assessment |
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| Bug Bite | Skin and subcutaneous tissue disorders | Systematic Assessment |
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| Skin Burn | Skin and subcutaneous tissue disorders | Systematic Assessment |
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| D006730 |
| Hormones, Hormone Substitutes, and Hormone Antagonists |
| D009479 | Neuropeptides |
| D010455 | Peptides |
| D000602 | Amino Acids, Peptides, and Proteins |
| D009842 | Oligopeptides |
| D009419 | Nerve Tissue Proteins |
| D011506 | Proteins |
| Body Ache |
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| Excitement/Agitation |
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| Insomnia |
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| Increased Motor Activity |
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| Depressive Affect |
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| Headache |
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| Drowsiness |
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| Decreased Motor Activity |
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| Aggression |
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| Akathisia |
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| Head Banging |
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| Dizziness |
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| Lethargy/Tiredness |
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| Nasal Congestion |
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| Dry Mouth |
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| Blurred Vision |
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| Ear Infection |
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| Runny Nose |
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| Sore Throat |
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| Cold Sore |
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| Decreased Appetite |
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| Nausea/Vomiting |
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| Constipation |
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| Diarrhea |
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| Increased Urination |
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| Bed Wetting |
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| Skin Rash |
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| Bug Bite |
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| Skin Burn |
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| Lethargy Baseline |
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| Lethargy Week 4 |
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| Stereotypy Baseline |
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| Stereotypy Week 4 |
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| Hyperactivity Baseline |
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| Hyperactivity Week 4 |
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| Inappropriate Speech Baseline |
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| Inappropriate Speech Week 4 |
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| Week 4 Social Standard Score |
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| Baseline Communication Standard Score |
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| Week 4 Communication Standard Score |
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| Change from Baseline on Chloride |
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