Intranasal Oxytocin in the Treatment of Autism

NCT00490802 | Completed Phase 2 Results DMC

• 1 other identifier: 06-0230 0001 02 PS
• Study Type: interventional
• Target: 19
• Locations: 1 country
• Sites: 1

Brief Summary

The purpose of this study is to learn whether or not the drug called oxytocin is helpful in improving mood and social functioning in adults with autism.

Conditions

Autism

Keywords

Autism; Treatment; Oxytocin; Adults; Autism Spectrum Disorder

Outcome Measures

Primary Outcomes (3)

• Clinical Global Impressions Scale - Improvement - Social

  The Clinical Global Impressions Scale - Improvement - Social is a well validated measure employing a 7-point scale of clinical global impression of improvement ( 1- very much improved, 2 - much improved, 3 - minimally improved, 4 - no change, 5 - minimally worse, 6 - much worse, 7 - very much worse) that the clinician fills out after considering all the available information on the participant including the parent history, the examination in clinic, reports from the school and other sources. Therefore the score is filtered through the judgment of the clinician evaluator. The Week 6 Improvement Ratings were used to categorize patients as clinically improved (≤2) or not (\>2). Sixteen of the 19 patients (84%) had data at Week 6. For the remaining three subjects, Week 6 ratings were imputed using expectation-maximization methods and the earlier Clinical Global Impression ratings. In all three cases the imputed ratings were \>2 and the patients were classified as not improved.

  6 Weeks

• Repetitive Behavior Scale - Revised

  The Repetitive Behavior Scale - Revised was developed to capture the breadth of repetitive behaviors that are specific to autism and is a parent report measure. In particular, it consists of 43-items that tap six repetitive behavior subtypes: Stereotyped, Self-injurious, Compulsive, Ritualistic, Sameness, and Restricted Interests. Two scores were calculated (higher-order vs. lower-order repetitive behaviors) in an effort to decrease the number of variables analyzed. This is based on previous factor analysis that produced these two factors: higher order (ritualistic, sameness, compulsive and restricted subscales) and lower order (stereotypy and self-injury). 1. The higher order behaviors have 29 items that can be endorsed with a maximum score of 87 and a minimum score of 0 2. The lower order behaviors have 14 items that can be endorsed, with a maximum score of 42 and a minimum score of 0 In both cases, a lower score represents a positive response.

  6 Weeks

• Diagnostic Analysis of Nonverbal Accuracy, Paralanguage Test

  The Diagnostic Analysis of Nonverbal Accuracy is a measure of emotion recognition across multiple modalities. It consists of five subtests: the Adult Facial Expression Test, the Child Facial Expression Test, the Adult Paralanguage Test, the Child Paralanguage Test, and the Adult Posture Test. The Diagnostic Analysis of Nonverbal Accuracy has established reliability and validity for children as young as 3 and adults as old as 100. The subtests of the test vary on four basic core emotions: happiness, sadness, anger, and fear, and the test provides measures of both high intensity and low intensity emotional reactions. We utilized both the Child Paralanguage and Adult Paralanguage Tests, therefore the minimum score that can be obtained is 0 and the maximum is 48. A higher score represents a positive response.

  6 Weeks

Secondary Outcomes (2)

• Yale-Brown Obsessive-Compulsive Scale

  6 Weeks

• Social Responsiveness Scale

  6 Weeks

Study Arms (2)

Intranasal Oxytocin

EXPERIMENTAL

Subjects were given 24 IU intranasal oxytocin twice daily, in the morning and afternoon for 6 weeks.

Drug: Oxytocin

Placebo

PLACEBO COMPARATOR

Subjects were given placebo twice daily, in the morning and afternoon for 6 weeks.

Drug: Placebo

Interventions

Oxytocin DRUG

Intranasal Oxytocin

Also known as: Syntocinon

Intranasal Oxytocin

Placebo DRUG

Placebo Comparator

Placebo

Eligibility Criteria

• Age: 18 Years - 60 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64)

You may qualify if:

• Male or female outpatients 18 to 60 years of age.
• Meet Diagnostic and Statistical Manual of Mental Disorders, 4th Edition, Text Revision. The diagnosis will be confirmed with Autism Diagnostic Interview-Revised and Autism Diagnostic Observation Schedule .
• Have a Clinician's Global Impression-Severity score ≥ 4 (moderately ill) at Screening and Baseline.
• If already receiving stable nonpharmacologic educational, behavioral, and/or dietary interventions, have continuous participation during the preceding 3 months prior to Screening and will not electively initiate new or modify ongoing interventions for the duration of the study.
• Have normal physical examination and laboratory test results at Screening. If abnormal, the finding(s) must be deemed clinically insignificant by the Investigators.
• The patient must be able to speak and understand English sufficiently to understand the nature of the study and to allow for the completion of all study assessments.
• Have a normal Intelligence Quotient (\>70) supported by the Wechsler Abbreviated Scales of Intelligence.

You may not qualify if:

• Patients born prior to 35 weeks gestational age.
• Patients with any primary psychiatric diagnosis other than autism at Screening: a history of attention deficit hyperactivity disorder, bipolar disorder, psychosis, posttraumatic stress disorder, schizophrenia, or major depressive disorder.
• Patients with a medical history of neurological disease, including, but not limited to, epilepsy/seizure disorder (except simple febrile seizures), movement disorder, tuberous sclerosis, fragile X, and any other known genetic syndromes, or known abnormal magnetic resonance imaging/structural lesion of the brain.
• Pregnant female patients.
• Patients with a medical condition that might interfere with the conduct of the study, confound interpretation of the study results, or endanger their own well-being. Patients with evidence or history of malignancy or any significant hematological, endocrine, cardiovascular (including any rhythm disorder), respiratory, renal, hepatic, or gastrointestinal disease.
• Patients taking psychoactive medication(s) (e.g., stimulants, antidepressants, antipsychotics, antiepileptics, anxiolytics, clonidine).
• Patients who plan to initiate or change nonpharmacologic interventions during the course of the study.
• Patients unable to tolerate venipuncture procedures for blood sampling.
• Patients who, in the Investigator's opinion, might not be suitable for the study.

Sponsors & Collaborators

• Evdokia Anagnostou: lead
• Icahn School of Medicine at Mount Sinai: collaborator

Study Sites (1)

Mount Sinai School of Medicine

New York, New York, 10029-6574, United States

Location

Related Publications (2)

• 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.

  PMID: 37811711 DERIVED

• Anagnostou E, Soorya L, Chaplin W, Bartz J, Halpern D, Wasserman S, Wang AT, Pepa L, Tanel N, Kushki A, Hollander E. Intranasal oxytocin versus placebo in the treatment of adults with autism spectrum disorders: a randomized controlled trial. Mol Autism. 2012 Dec 5;3(1):16. doi: 10.1186/2040-2392-3-16.

  PMID: 23216716 DERIVED

MeSH Terms

Conditions

Autistic Disorder; Autism Spectrum Disorder

Interventions

Oxytocin

Condition Hierarchy (Ancestors)

Child Development Disorders, Pervasive; Neurodevelopmental Disorders; Mental Disorders

Intervention Hierarchy (Ancestors)

Pituitary Hormones, Posterior; Pituitary Hormones; Peptide Hormones; Hormones; Hormones, Hormone Substitutes, and Hormone Antagonists; Peptides; Amino Acids, Peptides, and Proteins

Limitations and Caveats

Small sample size resulted in baseline differences; short duration of study; didn't examine impact of other characteristics; short-half life in the blood; inactive ingredients not identical in placebo; no participant follow-up after study

Results Point of Contact

• Title: Dr. Evdokia Anagnostou
• Organization: Holland Bloorview Kids Rehabilitation Hospital

eanagnostou@hollandbloorview.ca

416-753-6005

Study Officials

• Evdokia Anagnostou, MD

  Icahn School of Medicine at Mount Sinai

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: No

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDIV
• Responsible Party: SPONSOR INVESTIGATOR
• PI Title: Clinician Scientist

Study Record Dates

• First Submitted: June 22, 2007
• First Posted: June 25, 2007
• Study Start: June 1, 2006
• Primary Completion: September 1, 2011
• Study Completion: April 1, 2012
• Last Updated: October 23, 2015
• Results First Posted: November 5, 2014

Record last verified: 2015-10

Locations

US (1)