The Role of Vasopressin in the Social Deficits of Autism
Condition: Autism Spectrum Disorders
MeSH terms: Autistic Disorder; Autism Spectrum Disorder
Intervention: Vasopressin; Placebo
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 MeSH terms: Vasopressins; Arginine Vasopressin
Phase: Phase 2
Study type: Interventional
Primary purpose: Treatment
Allocation: Randomized
Intervention model: Parallel Assignment
Masking: Triple (Participant, Care Provider, Investigator)
Enrollment: 68.0
Sex: All
Minimum age: 6 Years
Maximum age: 12 Years
Healthy volunteers: No

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.
    

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