An Exploratory, Double-Blind, Placebo-Controlled Study of the Medical Food Vayarin in Children With Autism Spectrum Disorder (ASD)

Study Overview
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Brief Summary
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The primary study objective is to evaluate the efficacy of Vayarin_005 on ASD related symptoms in children.

Official Title
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An Exploratory, Double-Blind, Placebo-Controlled Study of the Medical Food Vayarin in Children With Autism Spectrum Disorder (ASD)

Conditions
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Autistic Disorder

Intervention / Treatment
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* Other: Medical Food : Vayarin_005
* Other: Placebo


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Males or females, ages 6-17 inclusive Must have a valid diagnosis of autism spectrum disorder via a clinical review of the DSM-IV, confirmed by Autism Diagnostic Observation Schedule (ADOS) Clinical Global Impression Scale of Severity of Illness for Pervasive Developmental Disorders (CGI-S-PDD) rating of 4 or higher (moderately ill or worse) IQ >50 evaluated by KBIT-2 or Stanford Binet Fifth Edition Able, and likely to fully comply with the study procedures and instructions Subjects with mood, anxiety, or disruptive behavior disorders will be allowed to participate in the study provided they do not meet any exclusionary criteria. Have normal physical examination and laboratory test results at screening. If abnormal, the finding(s) must be deemed clinically insignificant by the study Clinician. Parents or legal guardian must be able to read, write and speak English Parents or legal guardian have given written informed consent to participate in the study Exclusion Criteria: The subject is significantly underweight under the 5th percentile or obese above the 95th percentile Clinically significant systemic illness including hepatic, renal, gastroenterological, metabolic, respiratory, cardiovascular (including ischemic heart disease), endocrinologic, neurologic, immunologic, or hematologic disease, as determined by the study clinician. Patients with any primary psychiatric diagnosis other than autism at screening or a known genetic syndrome(s) that cause autism. Suspected or established CNS injury Change in dosage of psychiatric pharmacotherapy or other medications that have central nervous system effects or that affect performance 4 weeks before study initiation and throughout the study phase Use of alpha-agonists, or ADHD medications 4 weeks prior to study initiation and throughout the study Use of dietary supplements, 60 days before study initiation and throughout the study Change in educational/behavioral interventions within one month prior to participation or during the study A known comorbid psychiatric diagnoses of bipolar I disorder, suicidality, or substantial psychotic disorder. Subject who has participated in another clinical trial within 30 days of screening for this trial and/or any experimental treatment for this population Current history of physical, sexual, or emotional abuse History of alcohol or substance abuse as defined by DSM-IV criteria Consumption of >250 mg/day of caffeine History of allergic reactions or sensitivity to marine products and soy Has any illness which may jeopardize the participants' health or limit their successful trial completion. Any other reason that, in the opinion of the investigator, prevents the subject from participating in the study or compromise the patient safety

Ages Eligible for Study
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Minimum Age: 6 Years
Maximum Age: 17 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Other
Allocation: Randomized
Intervention Model: Crossover Assignment
Masking: Quadruple


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Placebo Comparator: Sequence 3: placebo/placebo<br>Placebo/placebo consists of placebo for 7 weeks followed by 7 weeks of placebo treatment | Other: Placebo<br> <br> * Other names: Cellulose;|
| Active Comparator: Sequence 2: placebo/ Treatment<br>Sequence 2: placebo/ Treatment , consists of placebo for 7 weeks followed by 7 weeks of treatment with Vayarin_005 | Other: Medical Food : Vayarin_005<br> <br> Other: Placebo<br> <br> * Other names: Cellulose;|
| Active Comparator: Sequence one: Treatment/Treatment<br>Treatment/Treatment- consists of PS_005 for 7 weeks followed by 7 weeks of additional treatment with Vayarin_005 | Other: Medical Food : Vayarin_005<br> <br> |

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Aberrant Behavior Checklist will be used to asses Autism spectrum symptoms | A significant reduction from baseline to endpoint on the Aberrant Behavior Checklist (ABC) compared to patients administered placebo | over 14 weeks | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Clinical Global Impression of Severity assesment |  | over 14 weeks | 
| Clinical Global Impression of Improvement assesment |  | over 14 weeks | 
| Conners Rating Scale questionnaire |  | over 14 weeks | 
| Behavior Rating Inventory of Executive function |  | over 14 weeks | 
| Child Health Questionnaire |  | over 14 weeks | 
| Caregiver Strain Questionnaire |  | over 14 weeks | 
| Adverse events monitoring |  | over 14 weeks |