Trial of Methyl B12 on Behavioral and Metabolic Measures in Children With Autism

Study Overview
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Brief Summary
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The purpose of this study is to determine whether the supplement Methyl B12 is effective in treating some of the symptoms of Autism.

Detailed Description
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Autism is a complex neurodevelopmental disorder with early childhood onset characterized by impairments in communication, social interaction, and repetitive behavior. Due to the lack of known treatments for autism, many parents seek complementary and alternative medical (CAM) therapies hoping to help their affected child. Methylcobalamin (methyl B12) is a commonly used CAM treatment that has anecdotal reports of remarkable clinical improvements with few side effects. Prior studies have found that children with autism have deficiencies in key metabolites and antioxidants which can be caused by methyl B12 deficiency; additional studies have shown that methyl B12 normalizes deficiencies in these metabolites and antioxidants. Based on these reports, a pilot study was conducted at UC Davis on the effect of methyl B12 on the behavioral and metabolic measures in children with autism. The preliminary results of 29 subjects revealed a subgroup of 9 responders to clinical behavior assessments. These responders also demonstrated significant improvement on the plasma measures of antioxidant capacity, suggesting methyl B12 improves symptoms in a subgroup of children with autism by increasing key antioxidants. The current study will have an 8 week double blind design with 50 subjects, designed to evaluate improvements from methyl B12 by using behavioral assessments and analysis of specific metabolites in the subjects' blood. This study will determine whether methyl B12 will lead to benefits in any of the core features of autism, and will examine metabolic changes with the hope of potentially identifying a biomarker for treatment response in a subgroup of subjects.

Official Title
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Double Blind Placebo Controlled Trial of Methyl B12 on Behavioral and Metabolic Measures in Children With Autism

Conditions
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Autistic Disorder

Intervention / Treatment
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* Drug: Methyl B12
* Dietary Supplement: Placebo


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Diagnosis of DSM IV defined autism and meets cut off on Autism Diagnostic Inventory-Revised (ADI-R) and/or the Autism Diagnostic Observation Scale (ADOS) Age 3 through 7 years IQ of 50 or above Parental agreement to continue present dietary, behavioral or psychotropic drug treatment but not change treatment during 8 week intervention Willingness to have blood drawn, without the use of a sedative prescription from the study doctor Exclusion Criteria: Bleeding disorder Cancer Seizure disorder Fragile X or other known genetic cause of autism Perinatal brain injury (i.e.: cerebral palsy) Other serious medical illnesses Current use of any B12 supplement

Ages Eligible for Study
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Minimum Age: 3 Years
Maximum Age: 7 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: Treatment
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Triple


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Placebo Comparator: Placebo<br>Placebo | Dietary Supplement: Placebo<br>* placebo<br>|
| Experimental: Active<br>Active Methyl B12 | Drug: Methyl B12<br>* 75 µg/Kg subcutaneously injected once every 3 days<br>* Other names: methylcobalamin;|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Clinical Global Impression-Improvement (CGI-I) | PI assesses subject's change using the CGI-I measure. This is a 7-point Likert scale that assesses improvement of the patient's condition. Scores range from the worst score of 7 (Very much worse) to the best score of 1 (Very much improved). Lower scores are better on this scale, and indicate greater improvement. | 8 weeks | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Pervasive Developmental Disorders, Autism