Clinical trial NCT01908205: Intranasal Oxytocin for the Treatment of Children and Adolescents With Autism Spectrum Disorders (ASD)
Official title: Intranasal Oxytocin for the Treatment of Children and Adolescents With Autism
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Autism Spectrum Disorder
Keywords: Autism
Social
Functioning
Cognition
Repetitive Behaviors
Anxiety
Quality of Life
Lead sponsor: Anagnostou, Evdokia, M.D.
Sponsor class: INDIV
Collaborators: name: United States Department of Defense
First posted: 2013-07-25
Enrollment: 60
Sex: ALL
Minimum age (years): 10
Maximum age (years): 17
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief summary: We are studying an investigational drug called intranasal oxytocin (Syntocinon®). Syntocinon® has been approved by the U.S. Food and Drug Administration for use in helping women breastfeed, but it has not been approved for use in children with ASD. However, there is previous research conducted that has indicated that after administration of oxytocin, adults with ASD demonstrated improvements in social cognition, and reduced repetitive behaviours and anxiety. There is also early research to suggest that children may also benefit in these areas. The purpose of this study is to test if oxytocin works to help children and adolescents with ASD.

Detailed description: Extensive data has been accumulated to suggest that central release of oxytocin is important for social cognition and function, as well as likely involved in anxiety modulation and repetitive behaviors. The Principal Investigator and Co-Principal Investigator of this study have previously documented: 1) an association between ASD and a single nuclear polymorphism of the oxytocin receptor gene, 2) ability to measure oxytocin levels in the blood by enzyme immunoassay and 3) preliminary data to support safety and efficacy of intranasal oxytocin in the treatment of social deficits and repetitive behaviors in adults with autism. A medication treatment targeting the core deficits of ASD in childhood is highly valuable because it could influence the developmental trajectory and make further psychosocial interventions possible. In this context, we propose a randomized placebo controlled trial of intranasal oxytocin in children and adolescents with ASD.

Eligibility criteria: Inclusion Criteria

1. Male or female outpatients, 10-17 years of age inclusive.
2. Meet Diagnostic and Statistical Manual of Mental Disorders, 4th Edition. Diagnostic and Statistical Manual (DSM-IV) criteria will be established by a clinician with expertise with individuals with ASD. Best estimate Diagnosis will be reached using DSM-IV criteria, the Autism Diagnostic Observation Schedule (ADOS-2) and the Autism Diagnostic Interview (ADI-R).
3. Have a Clinician's Global Impression-Severity (CGI-S) score ≥ 4 (moderately ill) at Screening.
4. Verbal and performance scale Intelligence Quotient (IQ) ≥ 70 (both subtests of the Wechsler Abbreviated Scale of Intelligence (WASI-I or WASI-II ≥ 70).
5. If already receiving stable concomitant medications affecting behavior, have continuous participation for 1 month prior to Screening (with the exception of fluoxetine, where a period of 6 weeks is needed), and not electively initiate new or modify ongoing medications for the duration of the study.
6. If already receiving stable non-pharmacologic educational, behavioral, and/or dietary interventions, have continuous participation during the preceding 3 months prior to Screening, and not electively initiate new or modify ongoing interventions for the duration of the study.
7. Have normal physical examination and laboratory test results at Screening. If abnormal, the finding(s) must be deemed not clinically significant by the Treating Clinician.
8. Ability to speak and understand English sufficiently to allow for the completion of all study assessments.
9. Ability to obtain written informed consent from the participant, if developmentally appropriate. If a participant does not have the capacity to consent, ability to obtain assent (if developmentally appropriate), as well as written informed consent from their parent(s)/legal guardian.

Exclusion Criteria

1. Patients born prior to 35 weeks gestational age.
2. Patients with a primary psychiatric diagnosis other than ASD.
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 brain MRI/structural lesion.
4. Pregnant female patients, sexually active female patients on hormonal birth control and sexually active females who do not use at least two types of non-hormonal birth control.
5. 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 with one or more of the following: HIV, Hepatitis B virus, Hepatitis C virus, hemophilia (bleeding problems, recent nose and brain injuries), abnormal blood pressure (hypotension or hypertension), drug abuse, immunity disorder or severe depression.
7. Patients who are currently taking oxytocin or have taken intranasal oxytocin in the past with no response.
8. Patients with a sensitivity to oxytocin or any components of its formulation.
9. Patients unable to tolerate venipuncture procedures for blood sampling.
10. Patients in foster care for whom the province/state is defined as a legal guardian

Primary outcomes:
measure: Efficacy of Intranasal Oxytocin vs. Placebo on Measures of Social Function
timeFrame: 12 and 24 weeks
Secondary outcomes:
measure: Efficacy of Intranasal Oxytocin vs. Placebo on Measures of Social Cognition
timeFrame: 12 Weeks
measure: Efficacy of Intranasal Oxytocin vs. Placebo on Measures of Social Cognition
timeFrame: 12 Weeks
measure: Efficacy of Intranasal Oxytocin vs. Placebo on Measures of Social Function
timeFrame: 12 Weeks
measure: Number of Participant Considered Social Responders
timeFrame: 12 Weeks
measure: Efficacy of Intranasal Oxytocin vs. Placebo on Measures of Repetitive Behaviors
timeFrame: 12 Weeks
measure: Efficacy of Intranasal Oxytocin vs. Placebo on Measures of Repetitive Behaviors
timeFrame: 12 Weeks
measure: Efficacy of Intranasal Oxytocin vs. Placebo on Measures of Anxiety
timeFrame: 12 Weeks
measure: Efficacy of Intranasal Oxytocin vs. Placebo on Measures of Quality of Life
timeFrame: 12 Weeks
measure: Number of Participant Considered Overall Responders
timeFrame: 12 Weeks
measure: Safety and Tolerability of Intranasal Oxytocin in Children and Adolescents With ASD
timeFrame: 12 Weeks
Other outcomes:
Locations:
city: Minneapolis
country: United States
facility: University of Minnesota
state: Minnesota
city: Toronto
country: Canada
facility: Holland Bloorview Kids Rehabilitation Hospital
state: Ontario
Study officials:
affiliation: Holland Bloorview Kids Rehabilitation Hospital
name: Evdokia Anagnostou, M.D.
role: PRINCIPAL_INVESTIGATOR
affiliation: University of Minnesota
name: Suma Jacob, M.D., Ph.D.
role: PRINCIPAL_INVESTIGATOR
References: