Autism Spectrum DisordersPhase 2NCT01962870
The Role of Vasopressin in the Social Deficits of Autism
A Phase 2 study testing Vasopressin in people with Autism Spectrum Disorders, run by Stanford University at 1 site in United States.
Trial 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. Read more about this study
- Who is it for?
- Ages 6 to 12 years
- Conditions the study is for
- Autism Spectrum Disorders
- What is being tested?
- Vasopressin
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 68 participants
- Where
- 1 siteUnited States
- Study dates
- Dec 2013to 30 May 2017
- Phase
- Phase 2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 6 to 12 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- 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
Show all 11Show fewer
- 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
Who cannot join
- 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
Show all 12Show fewer
- 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
Trial locations and contacts
1 site in 1 country.
United States1 site
Stanford University School of Medicine; Psychiatry and Behavioral Sciences
Stanford, California, 94305
About this study
Official title: Randomized Placebo-controlled Trial of Vasopressin Treatment for Social Deficits in Children With Autism
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.
- Conditions
- Autism Spectrum Disorders
- Being tested
- Vasopressin, Placebo
- Medical subject headings
- Autism Spectrum Disorder
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
VasopressinDrug
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).
PlaceboDrug
| Study group | Role | What they receive |
|---|---|---|
| Placebo | Placebo comparator | Placebo Nasal Spray Receives: Drug: Placebo |
| Vasopressin | Active comparator | Vasopressin Nasal Spray Receives: Drug: Vasopressin |
What is being measured
The primary outcome is the main question the study is designed to answer.
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 )
Measured: Baseline; Week 4
Secondary outcomes (20)
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)
Measured: Baseline; Week 4
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.
Measured: 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).
Measured: 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)
Measured: 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)
Measured: 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.
Measured: 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).
Measured: Baseline through Week 4
Change From Baseline in Heart Rate After Treatment.
Sitting heart rate (beats per minute).
Measured: 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.
Measured: 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).
Measured: 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.
Measured: Baseline; Week 4
Change From Baseline in Clinical Chemistry Labs (NA+, K+, Cl-) During Treatment.
Clinical chemistry labs(sodium, potassium, chloride)
Measured: Baseline; Week 4
Change From Baseline in Laboratory Based Eye-gaze to Social Cues During Treatment.
Measured: Baseline; Week 4
Change From Baseline in Laboratory Based Social Mimicry Abilities During Treatment.
Measured: Baseline; Week 4
Change From Baseline in Blood Pressure After Treatment
Sitting Systolic and Diastolic blood pressure.
Measured: Baseline; Week 4
Change From Baseline in Body Weight After Treatment.
Measured: Baseline; Week 4
Change From Baseline in Body Temperature After Treatment
Measured: Baseline; Week 4
Change From Baseline in the Awareness of Social Inference Test Revised (TASIT-R) Scores During Treatment.
Measured: 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.
Measured: 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.
Measured: Baseline
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted14 Oct 2013 (estimated)
- Study startDec 2013
- Primary completion30 May 2017
- Study completion30 May 2017
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Autism Spectrum DisordersStudies of the same condition, open and under way.
- More trials run by Stanford UniversityThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Triple blind
- Who is masked
- Participant, Care provider, Investigator
- Enrolment
- 68 participants (actual)
Sponsor and investigators
- Lead sponsor
- Stanford University
- Sponsor type
- University, hospital or other
- Collaborators
- National Institute of Mental Health (NIMH)
- Responsible party
- Principal investigator, Antonio Hardan, Stanford University
- Investigators
- Antonio Y Hardan, MD, Principal investigator, Stanford UniversityKaren J Parker, PhD, Principal investigator, Stanford University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01962870
- Sponsor's protocol ID
- IRB-26034
- Other IDs
- R21MH100387-01
- Status last verified
- May 2019
- First submitted
- 8 Oct 2013
- Last updated
- 24 May 2019
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- Individual data sharing
- No
Publications
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.
PubMed 37811711
Study at a glance
From the registry record, last updated by the sponsor on 24 May 2019. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Randomized Placebo-controlled Trial of Vasopressin Treatment for Social Deficits in Children With Autism
- ClinicalTrials.gov ID
- NCT01962870
- Acronym / sponsor ID
- IRB-26034
- Conditions
- Autism Spectrum Disorders
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 68 participants (actual)
- Lead sponsor
- Stanford UniversityUniversity, hospital or other
- Collaborators
- National Institute of Mental Health (NIMH)
- Study start
- Dec 2013 (actual)
- Primary completion
- 30 May 2017 (actual)
- Study completion
- 30 May 2017 (actual)
- First posted
- 14 Oct 2013 (estimated)
- Results
- Posted 24 May 2019
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Triple blind (participant, care provider, investigator)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Placebo | Placebo comparator | Drug: PlaceboPlacebo Nasal Spray |
| Vasopressin | Active comparator | Drug: VasopressinVasopressin Nasal Spray |
Who may qualify
- Ages
- 6 to 12 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (11 listed)
- 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)
Key exclusion criteria (12 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| VasopressinParticipants 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). | Drug | Vasopressin |
| Placebo | Drug | Placebo |
Primary outcome
Change From Baseline in Parent Rated Social Responsiveness Scale, 2nd Edition (SRS-2) T-Score After Treatment.
Time frame: 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 )
20 secondary outcomes
Change From Baseline in Clinical Global Impression (CGI) Severity, Social and Communication Scores During Treatment.
Time frame: Baseline; Week 4
Change From Baseline in Reading the Mind in the Eyes Test, Child Version (RMET-child) Scores During Treatment.
Time frame: Baseline; Week 4
Change From Baseline in Laboratory Based Facial Emotion Recognition Abilities During Treatment.
Time frame: Baseline; Week 4
Change From Baseline in Parent Rated Repetitive Behavior Scale Revised (RBS-R) Scores During Treatment.
Time frame: Baseline; Week 4
Change From Baseline in Parent Rated Spence Children's Anxiety Scale (SCAS) During Treatment.
Time frame: Baseline; Week 4
Number of Participants With Side Effects Assessed Using Parent Rated Dosage Record Treatment Emergent Symptom Scale (DOTES) Scores During Treatment
Time frame: Baseline through Week 4
Change From Baseline on the Overt Aggression Scale (OAS) During Treatment.
Time frame: Baseline through Week 4
Change From Baseline in Heart Rate After Treatment.
Time frame: Baseline; Week 4
Change From Baseline in Parent Rated Aberrant Behavior Checklist (ABC) Scores During Treatment.
Time frame: Baseline; Week 4
Change From Baseline in Parent Rated Pediatric Quality of Life (PedQL) Inventory Scores During Treatment.
Time frame: Baseline; Week 4
Change From Baseline in Parent Rated Vineland Adaptive Behavior Scales Second Edition (VABS-II) - Social and Communication Subscales During Treatment.
Time frame: Baseline; Week 4
Change From Baseline in Clinical Chemistry Labs (NA+, K+, Cl-) During Treatment.
Time frame: Baseline; Week 4
Change From Baseline in Laboratory Based Eye-gaze to Social Cues During Treatment.
Time frame: Baseline; Week 4
Change From Baseline in Laboratory Based Social Mimicry Abilities During Treatment.
Time frame: Baseline; Week 4
Change From Baseline in Blood Pressure After Treatment
Time frame: Baseline; Week 4
Change From Baseline in Body Weight After Treatment.
Time frame: Baseline; Week 4
Change From Baseline in Body Temperature After Treatment
Time frame: Baseline; Week 4
Change From Baseline in the Awareness of Social Inference Test Revised (TASIT-R) Scores During Treatment.
Time frame: Baseline, Week 4
Change From Baseline in a Developmental Neuropsychological Assessment, Second Edition. (NEPSY-II) Affect Recognition Scores During Treatment.
Time frame: Baseline; Week 4
Change From Baseline in Plasma Vasopressin Levels During Treatment.
Time frame: Baseline
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 24 May 2019.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Derived from this study: PubMed 37811711Iffland 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.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Antonio Y Hardan, MD principal investigator
Stanford University
Karen J Parker, PhD principal investigator
Stanford University
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Vasopressin in people with Autism Spectrum Disorders, run by Stanford University at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 11 inclusion and 12 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 24 May 2019. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 24 May 2019). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Change From Baseline in Parent Rated Social Responsiveness Scale, 2nd Edition (SRS-2) T-Score After Treatment. (time frame: Baseline; Week 4). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT01962870, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 24 May 2019.