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The Role of Vasopressin in the Social Deficits of Autism

Primary Purpose

Autism Spectrum Disorders

Status
Completed
Phase
Phase 2
Locations
United States
Study Type
Interventional
Intervention
Vasopressin
Placebo
Sponsored by
Stanford University
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Autism Spectrum Disorders

Eligibility Criteria

6 Years - 12 Years (Child)All SexesDoes not accept healthy volunteers

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

Sites / Locations

  • Stanford University School of Medicine; Psychiatry and Behavioral Sciences

Arms of the Study

Arm 1

Arm 2

Arm Type

Placebo Comparator

Active Comparator

Arm Label

Placebo

Vasopressin

Arm Description

Placebo Nasal Spray

Vasopressin Nasal Spray

Outcomes

Primary Outcome Measures

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 )

Secondary Outcome Measures

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)
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.
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).
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)
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)
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.
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).
Change From Baseline in Heart Rate After Treatment.
Sitting heart rate (beats per minute).
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.
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).
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.
Change From Baseline in Clinical Chemistry Labs (NA+, K+, Cl-) During Treatment.
Clinical chemistry labs(sodium, potassium, chloride)
Change From Baseline in Laboratory Based Eye-gaze to Social Cues During Treatment.
Change From Baseline in Laboratory Based Social Mimicry Abilities During Treatment.
Change From Baseline in Blood Pressure After Treatment
Sitting Systolic and Diastolic blood pressure.
Change From Baseline in Body Weight After Treatment.
Change From Baseline in Body Temperature After Treatment
Change From Baseline in the Awareness of Social Inference Test Revised (TASIT-R) Scores During Treatment.
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.
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.

Full Information

First Posted
October 8, 2013
Last Updated
May 2, 2019
Sponsor
Stanford University
Collaborators
National Institute of Mental Health (NIMH)
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1. Study Identification

Unique Protocol Identification Number
NCT01962870
Brief Title
The Role of Vasopressin in the Social Deficits of Autism
Official Title
Randomized Placebo-controlled Trial of Vasopressin Treatment for Social Deficits in Children With Autism
Study Type
Interventional

2. Study Status

Record Verification Date
May 2019
Overall Recruitment Status
Completed
Study Start Date
December 2013 (Actual)
Primary Completion Date
May 30, 2017 (Actual)
Study Completion Date
May 30, 2017 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Principal Investigator
Name of the Sponsor
Stanford University
Collaborators
National Institute of Mental Health (NIMH)

4. Oversight

Data Monitoring Committee
Yes

5. Study Description

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Autism Spectrum Disorders

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Parallel Assignment
Masking
ParticipantCare ProviderInvestigator
Allocation
Randomized
Enrollment
68 (Actual)

8. Arms, Groups, and Interventions

Arm Title
Placebo
Arm Type
Placebo Comparator
Arm Description
Placebo Nasal Spray
Arm Title
Vasopressin
Arm Type
Active Comparator
Arm Description
Vasopressin Nasal Spray
Intervention Type
Drug
Intervention Name(s)
Vasopressin
Intervention Description
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).
Intervention Type
Drug
Intervention Name(s)
Placebo
Primary Outcome Measure Information:
Title
Change From Baseline in Parent Rated Social Responsiveness Scale, 2nd Edition (SRS-2) T-Score After Treatment.
Description
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
Secondary Outcome Measure Information:
Title
Change From Baseline in Clinical Global Impression (CGI) Severity, Social and Communication Scores During Treatment.
Description
Higher Scores on the CGI severity scale mean more greater social and communication deficits (Range 1-7)
Time Frame
Baseline; Week 4
Title
Change From Baseline in Reading the Mind in the Eyes Test, Child Version (RMET-child) Scores During Treatment.
Description
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
Title
Change From Baseline in Laboratory Based Facial Emotion Recognition Abilities During Treatment.
Description
Higher scores mean better facial emotion recognition abilities. Lower scores mean worse facial emotion recognition abilities (Range: 0-42).
Time Frame
Baseline; Week 4
Title
Change From Baseline in Parent Rated Repetitive Behavior Scale Revised (RBS-R) Scores During Treatment.
Description
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
Title
Change From Baseline in Parent Rated Spence Children's Anxiety Scale (SCAS) During Treatment.
Description
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
Title
Number of Participants With Side Effects Assessed Using Parent Rated Dosage Record Treatment Emergent Symptom Scale (DOTES) Scores During Treatment
Description
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
Title
Change From Baseline on the Overt Aggression Scale (OAS) During Treatment.
Description
Count of participants reporting an increase of aggression during treatment compared to baseline (pretreatment).
Time Frame
Baseline through Week 4
Title
Change From Baseline in Heart Rate After Treatment.
Description
Sitting heart rate (beats per minute).
Time Frame
Baseline; Week 4
Title
Change From Baseline in Parent Rated Aberrant Behavior Checklist (ABC) Scores During Treatment.
Description
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
Title
Change From Baseline in Parent Rated Pediatric Quality of Life (PedQL) Inventory Scores During Treatment.
Description
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
Title
Change From Baseline in Parent Rated Vineland Adaptive Behavior Scales Second Edition (VABS-II) - Social and Communication Subscales During Treatment.
Description
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
Title
Change From Baseline in Clinical Chemistry Labs (NA+, K+, Cl-) During Treatment.
Description
Clinical chemistry labs(sodium, potassium, chloride)
Time Frame
Baseline; Week 4
Title
Change From Baseline in Laboratory Based Eye-gaze to Social Cues During Treatment.
Time Frame
Baseline; Week 4
Title
Change From Baseline in Laboratory Based Social Mimicry Abilities During Treatment.
Time Frame
Baseline; Week 4
Title
Change From Baseline in Blood Pressure After Treatment
Description
Sitting Systolic and Diastolic blood pressure.
Time Frame
Baseline; Week 4
Title
Change From Baseline in Body Weight After Treatment.
Time Frame
Baseline; Week 4
Title
Change From Baseline in Body Temperature After Treatment
Time Frame
Baseline; Week 4
Title
Change From Baseline in the Awareness of Social Inference Test Revised (TASIT-R) Scores During Treatment.
Time Frame
Baseline, Week 4
Title
Change From Baseline in a Developmental Neuropsychological Assessment, Second Edition. (NEPSY-II) Affect Recognition Scores During Treatment.
Description
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
Title
Change From Baseline in Plasma Vasopressin Levels During Treatment.
Description
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

10. Eligibility

Sex
All
Minimum Age & Unit of Time
6 Years
Maximum Age & Unit of Time
12 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
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
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Antonio Y Hardan, MD
Organizational Affiliation
Stanford University
Official's Role
Principal Investigator
First Name & Middle Initial & Last Name & Degree
Karen J Parker, PhD
Organizational Affiliation
Stanford University
Official's Role
Principal Investigator
Facility Information:
Facility Name
Stanford University School of Medicine; Psychiatry and Behavioral Sciences
City
Stanford
State/Province
California
ZIP/Postal Code
94305
Country
United States

12. IPD Sharing Statement

Plan to Share IPD
No

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The Role of Vasopressin in the Social Deficits of Autism

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