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

University of California San Francisco (UCSF)

Status and phase: Completed; Phase 3; Phase 2

Conditions: Autistic Disorder

Treatments: Dietary Supplement: Placebo; Drug: Methyl B12

Study type: Interventional

Funder types: Other

Identifiers

NCT01039792

Autism Speaks 3031

Details and patient eligibility

About

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

Full description

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.

Enrollment: 57 patients

Sex: All

Ages: 3 to 7 years old

Volunteers: No Healthy Volunteers

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

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Triple Blind

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57 participants in 2 patient groups, including a placebo group

Placebo

Placebo Comparator group

Description:

Placebo

Treatment:

Dietary Supplement: Placebo

Active

Experimental group

Description:

Active Methyl B12

Treatment:

Drug: Methyl B12