Intranasal Oxytocin in the Treatment of Autism

NCT ID: NCT00490802

Last Updated: 2015-10-23

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE2
• Total Enrollment: 19 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2006-06-30
• Study Completion Date: 2012-04-30

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.

Detailed Description

Autism is a developmental disorder characterized by abnormalities in speech and communication, impaired social functioning, and repetitive behaviors and restricted interests. A number of researchers have suggested that the neuropeptide oxytocin may be implicated in the etiology of autism.

Given the likely possibility of dysregulated oxytocin in autism, the goal of this pilot study is to investigate the long-term therapeutic effects of oxytocin in the treatment of autism. One practical issue with oxytocin is that it does not exist in a pill form. Only the intravenous form is available in the United States and this form may or may not pass the blood-brain barrier. In addition, intravenous oxytocin is not practical for treatment studies. One alternative is intranasal oxytocin; this form of administration is known to pass the blood-brain barrier, and it is easy for participants to self-administer. Although not available in the United States, we are in the process of receiving an Investigational New Drug exemption for its use and can import it from Europe.

Thus, this pilot investigation will explore daily intranasal oxytocin in the treatment of autism. Also, there are very few, if any, outcome measures to assess social functioning in the "real world" in the context of clinical trials; yet, this is a major target for intervention, especially in autism. Thus, a final goal of this study will be to explore the use of Event Contingent Recording to index changes in social functioning and affect. Event Contingent Recording is a methodology developed by personality/social psychologists, which allows participants to report on symptoms, affect, and behavior close in time to experience. In addition, to enabling more sensitive assessments, this methodology allows for the assessment of more diverse (e.g., at home versus work) and more detailed measurements of mood and behavior.

Finally, a portion of this study aims to perform gene expression profiling using fresh whole blood to explore the molecular mechanisms underlying oxytocin therapy and oxytocin efficacy in adults with high functioning autism or Asperger's syndrome. The systemic effects of oxytocin therapy and the molecular basis for a positive treatment response to oxytocin are not well understood. An understanding of the former may help predict those persons who may suffer side-effects from treatment and the latter may help provide easily accessible peripheral biomarkers that could predict treatment response.

Conditions

Autism

Study Design

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

Study Groups

Intranasal Oxytocin

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

Group Type: EXPERIMENTAL

Oxytocin

Intervention Type: DRUG

Intranasal Oxytocin

Placebo

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

Group Type: PLACEBO_COMPARATOR

Placebo

Intervention Type: DRUG

Placebo Comparator

Interventions

Oxytocin

Intranasal Oxytocin

Intervention Type: DRUG

Placebo

Placebo Comparator

Intervention Type: DRUG

Other Intervention Names

Syntocinon

Eligibility Criteria

Inclusion Criteria

1. Male or female outpatients 18 to 60 years of age.

2. 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 .

3. Have a Clinician's Global Impression-Severity score ≥ 4 (moderately ill) at Screening and Baseline.

4. 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.

5. Have normal physical examination and laboratory test results at Screening. If abnormal, the finding(s) must be deemed clinically insignificant by the Investigators.

6. 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.

7. Have a normal Intelligence Quotient (>70) supported by the Wechsler Abbreviated Scales of Intelligence.

Exclusion Criteria

1. Patients born prior to 35 weeks gestational age.

2. 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.

3. 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.

4. Pregnant female patients.

5. 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.

6. Patients taking psychoactive medication(s) (e.g., stimulants, antidepressants, antipsychotics, antiepileptics, anxiolytics, clonidine).

7. Patients who plan to initiate or change nonpharmacologic interventions during the course of the study.

8. Patients unable to tolerate venipuncture procedures for blood sampling.

9. Patients who, in the Investigator's opinion, might not be suitable for the study.

• Minimum Eligible Age: 18 Years
• Maximum Eligible Age: 60 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Icahn School of Medicine at Mount Sinai

OTHER

Sponsor Role: collaborator

Evdokia Anagnostou

INDIV

Sponsor Role: lead

Responsible Party

Evdokia Anagnostou

Clinician Scientist

Responsibility Role: SPONSOR_INVESTIGATOR

Principal Investigators

Evdokia Anagnostou, MD

Role: PRINCIPAL_INVESTIGATOR

Icahn School of Medicine at Mount Sinai

Locations

Mount Sinai School of Medicine

New York, New York, 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)

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.

Reference Type: DERIVED

PMID: 23216716 (View on PubMed)

Other Identifiers

06-0230 0001 02 PS

Identifier Type: -

Identifier Source: org_study_id