The Role of Vasopressin in the Social Deficits of Autism

NCT ID: NCT01962870

Last Updated: 2019-05-24

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE2
• Total Enrollment: 68 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2013-12-31
• Study Completion Date: 2017-05-30

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.

Conditions

Autism Spectrum Disorders

Study Design

• Allocation Method: RANDOMIZED
• Intervention Model: PARALLEL
• Primary Study Purpose: TREATMENT
• Blinding Strategy: TRIPLE

Study Groups

Placebo

Placebo Nasal Spray

Group Type: PLACEBO_COMPARATOR

Placebo

Intervention Type: DRUG

Vasopressin

Vasopressin Nasal Spray

Group Type: ACTIVE_COMPARATOR

Vasopressin

Intervention Type: 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).

Interventions

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).

Intervention Type: DRUG

Placebo

Intervention Type: DRUG

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

• Minimum Eligible Age: 6 Years
• Maximum Eligible Age: 12 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

National Institute of Mental Health (NIMH)

NIH

Sponsor Role: collaborator

Stanford University

OTHER

Sponsor Role: lead

Responsible Party

Antonio Hardan

MD

Responsibility Role: PRINCIPAL_INVESTIGATOR

Principal Investigators

Antonio Y Hardan, MD

Role: PRINCIPAL_INVESTIGATOR

Stanford University

Karen J Parker, PhD

Role: PRINCIPAL_INVESTIGATOR

Stanford University

Locations

Stanford University School of Medicine; Psychiatry and Behavioral Sciences

Stanford, California, United States

Countries

United States

References

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.

Reference Type: DERIVED

PMID: 37811711 (View on PubMed)

Provided Documents

Document Type: Statistical Analysis Plan

View Document

Document Type: Study Protocol

View Document

Other Identifiers

R21MH100387-01

Identifier Type: NIH

Identifier Source: secondary_id

View Link

IRB-26034

Identifier Type: -

Identifier Source: org_study_id