Clinical trial NCT01308749: A Study of Oxytocin in Children and Adolescents With Autistic Disorder
Official title: A Pilot Study of Oxytocin in Children and Adolescents With Autistic Disorder
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Autism
Lead sponsor: University of North Carolina, Chapel Hill
Sponsor class: OTHER
Collaborators: name: Autism Speaks
First posted: 2011-03-04
Enrollment: 25
Sex: ALL
Minimum age (years): 3
Maximum age (years): 17
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief summary: The investigators propose to conduct this pilot study to evaluate oxytocin as a supplemental treatment for improving social difficulties in individuals with autism.

Detailed description: The proposed pilot study is an essential first step toward rigorously evaluating oxytocin treatment of individuals with autism. The biologic actions of oxytocin on social cognition and prosocial behaviors and the clinical, genetic and epigenetic evidence for involvement of the oxytocin system in the pathophysiology of some cases of autism strongly suggest that supplemental oxytocin therapy could significantly improve the social disabilities involved in autism. Many people feel that these social difficulties are the most characteristic and central feature of autism. Overall, this study aims to determine the tolerability, accessibility, and feasibility of an oxytocin pilot study.

This study will consent up to 30 subjects in order to randomize up to 25 subjects into a 2-month (8 weeks) randomized double-blind, placebo-controlled initial treatment period, a subsequent 2-month (8 weeks) period in which all participants receive oxytocin, and up to three post-treatment visits that occur at week 28 (±2 weeks),an interim visit anytime between week 16 and week 76 only for those patients who plan to start oxytocin on their own outside of study treatment and who will experience a lag time between week 16 (end of open label treatment) and when outside oxytocin treatment will begin, and some time before week 76. The investigators hope that this study will help to inform future study designs in determining whether a short term or long term treatment trial is necessary to observe significant effects. This will also help to develop systematic preliminary safety measures.

Eligibility criteria: Inclusion Criteria:

* Between 3 and 17 years old, inclusive.
* Have a clinical diagnosis of autistic disorder confirmed according to Diagnostic Statistical Manual of Mental Disorders-IV criteria by using the Autism Diagnostic Interview - Revised (ADI-R) and/or the Autism Diagnostic Observation Scale (ADOS, Lord et al., 1989).

Exclusion Criteria:

* Changes in allied health therapies, behavioral or educational interventions within the past 2 months of the baseline visit other than those associated with school holidays.
* Changes in psychotropic and alternative medication doses in the last 30 days of the baseline visit.
* Subjects 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. This includes, but is not limited to, Rett Syndrome, impairment of renal function, evidence or history of malignancy or any significant hematological, endocrine, cardiovascular (including any rhythm disorder and uncontrolled hypertension), respiratory, hepatic, or gastrointestinal disease.
* Marked sensory impairment such as deafness or blindness that would interfere with conduct of the study.
* Pregnancy/Nursing because of the unknown effects of oxytocin to unborn babies and/or nursing infants. All females of child-bearing potential will be administered a urine or serum pregnancy test at screening and at any point during the study at physician discretion. Refusal to undergo a pregnancy test will result in exclusion from the study. The investigators will share results of pregnancy test with the subject's legal guardian.
* Refusal to practice contraception if sexually active because the effects of exposure to high concentrations of oxytocin on sperm or newly conceived embryos are unknown. Sexually active men and women should not take part in this study if they and their partners are not both using an effective birth control method (for example, women use birth control pills, an intrauterine device (IUD) or a diaphragm and men use condoms).
* Inability of caretakers to speak English.
* Absence of a consistent caretaker to report on symptoms.
* Subjects who, in the Investigator's opinion, might not be suitable for the study.

Primary outcomes:
measure: Number of Participants Who Could Tolerate Twice Daily Oxytocin
timeFrame: Week 0 to week 8
measure: Number of Participants Who Could Tolerate Twice Daily Oxytocin
timeFrame: Week 0 to week 16
Secondary outcomes:
measure: Change in Mean Plasma Oxytocin Level During Period 1 - Double Blind Phase
timeFrame: Week 0 to week 8
measure: Change in Mean Weight
timeFrame: between weeks 0 and 8
measure: Change in Mean Total Social Social Responsiveness Scale (SRS) T-score
timeFrame: 0-8 weeks, blinded treatment, period 1
measure: Change in Mean Autism Diagnostic Observation Schedule (ADOS) Total Score
timeFrame: Baseline to 16 Weeks
measure: Change in Mean Aberrant Behavior Checklist (ABC)-Social Withdrawal Subscale Score Over Both Periods
timeFrame: Baseline to 16 Weeks
measure: Change in Mean Pervasive Developmental Disorder Behavior Inventory - Screening Version (PDDBI-SV) Total Score Over Both Periods
timeFrame: Baseline to 16 Weeks
measure: Change in Mean Systolic Blood Pressure During Period 1
timeFrame: Week 0 to 8
measure: Mean Change in Prolactin Levels Over Period 1
timeFrame: Week 0 to 8
measure: Mean Change in Temperature During Period 1
timeFrame: Week 0 to 8
Other outcomes:
Locations:
city: Chapel Hill
country: United States
facility: University of North Carolina Chapel Hill
state: North Carolina
Study officials:
affiliation: University of North Carolina, Chapel Hill
name: Linmarie Sikich, M.D.
role: PRINCIPAL_INVESTIGATOR
References: