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

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

Study Overview

• Status: Completed
• Conditions: Autistic Disorder
• Intervention / Treatment: Other: Placebo; Other: Medical Food : Vayarin_005

• Study Type: Interventional
• Enrollment (Actual): 56
• Phase: Not Applicable

Contacts and Locations

Study Locations

• United States
  • New Jersey
    • Livingston, New Jersey, United States, 07039
      • Institute of Neurology and Neurosurgery at St. Barnabas
  • New York
    • New York, New York, United States, 10021
      • Spectrum Neuroscience and Treatment Institute

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 6 years to 17 years (CHILD)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

Inclusion Criteria:

1. Males or females, ages 6-17 inclusive
2. Must have a valid diagnosis of autism spectrum disorder via a clinical review of the DSM-IV, confirmed by Autism Diagnostic Observation Schedule (ADOS)
3. Clinical Global Impression Scale of Severity of Illness for Pervasive Developmental Disorders (CGI-S-PDD) rating of 4 or higher (moderately ill or worse)
4. IQ >50 evaluated by KBIT-2 or Stanford Binet Fifth Edition
5. Able, and likely to fully comply with the study procedures and instructions
6. Subjects with mood, anxiety, or disruptive behavior disorders will be allowed to participate in the study provided they do not meet any exclusionary criteria.
7. Have normal physical examination and laboratory test results at screening. If abnormal, the finding(s) must be deemed clinically insignificant by the study Clinician.
8. Parents or legal guardian must be able to read, write and speak English
9. Parents or legal guardian have given written informed consent to participate in the study

Exclusion Criteria:

1. The subject is significantly underweight under the 5th percentile or obese above the 95th percentile
2. 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.
3. Patients with any primary psychiatric diagnosis other than autism at screening or a known genetic syndrome(s) that cause autism.
4. Suspected or established CNS injury
5. 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
6. Use of alpha-agonists, or ADHD medications 4 weeks prior to study initiation and throughout the study
7. Use of dietary supplements, 60 days before study initiation and throughout the study
8. Change in educational/behavioral interventions within one month prior to participation or during the study
9. A known comorbid psychiatric diagnoses of bipolar I disorder, suicidality, or substantial psychotic disorder.
10. Subject who has participated in another clinical trial within 30 days of screening for this trial and/or any experimental treatment for this population
11. Current history of physical, sexual, or emotional abuse
12. History of alcohol or substance abuse as defined by DSM-IV criteria
13. Consumption of >250 mg/day of caffeine
14. History of allergic reactions or sensitivity to marine products and soy
15. Has any illness which may jeopardize the participants' health or limit their successful trial completion.
16. Any other reason that, in the opinion of the investigator, prevents the subject from participating in the study or compromise the patient safety

Study Plan

How is the study designed?

Design Details

• Primary Purpose: OTHER
• Allocation: RANDOMIZED
• Interventional Model: CROSSOVER
• Masking: QUADRUPLE

Number of Arms

3

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
PLACEBO_COMPARATOR: Sequence 3: placebo/placebo; Placebo/placebo consists of placebo for 7 weeks followed by 7 weeks of placebo treatment	Other: Placebo; Other Names: Cellulose
ACTIVE_COMPARATOR: Sequence 2: placebo/ Treatment; Sequence 2: placebo/ Treatment , consists of placebo for 7 weeks followed by 7 weeks of treatment with Vayarin_005	Other: Placebo; Other Names: Cellulose; Other: Medical Food : Vayarin_005
ACTIVE_COMPARATOR: Sequence one: Treatment/Treatment; Treatment/Treatment- consists of PS_005 for 7 weeks followed by 7 weeks of additional treatment with Vayarin_005	Other: Medical Food : Vayarin_005

What is the study measuring?

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	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

Collaborators and Investigators

• Sponsor: Enzymotec
• Investigators: Principal Investigator: Eric Hollander, M.D., Spectrum Neuroscience and Treatment Institute

Study record dates

Study Major Dates

• Study Start (ACTUAL): August 1, 2014
• Primary Completion (ACTUAL): May 1, 2017
• Study Completion (ACTUAL): October 1, 2017

Study Registration Dates

• First Submitted: August 12, 2014
• First Submitted That Met QC Criteria: August 20, 2014
• First Posted (ESTIMATE): August 21, 2014

Study Record Updates

• Last Update Posted (ACTUAL): April 11, 2018
• Last Update Submitted That Met QC Criteria: April 10, 2018
• Last Verified: January 1, 2017

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Mental Disorders; Neurodevelopmental Disorders; Child Development Disorders, Pervasive; Autistic Disorder; Autism Spectrum Disorder
• Other Study ID Numbers: Vayarin_005