Clinical trial NCT01039792: Trial of Methyl B12 on Behavioral and Metabolic Measures in Children With Autism
Official title: Double Blind Placebo Controlled Trial of Methyl B12 on Behavioral and Metabolic Measures in Children With Autism
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
PHASE3
Conditions: Autistic Disorder
Keywords: Autism
Pervasive Developmental Disorders
Lead sponsor: University of California, San Francisco
Sponsor class: OTHER
Collaborators: name: University of California, Davis
name: Arkansas Children's Hospital Research Institute
First posted: 2009-12-25
Enrollment: 57
Sex: ALL
Minimum age (years): 3
Maximum age (years): 7
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief summary: 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: 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.

Eligibility criteria: 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

Primary outcomes:
measure: Clinical Global Impression-Improvement (CGI-I)
timeFrame: 8 weeks
Secondary outcomes:
Other outcomes:
Locations:
city: San Francisco
country: United States
facility: UCSF
state: California
Study officials:
affiliation: University of California, San Francisco
name: Robert L. Hendren, DO
role: PRINCIPAL_INVESTIGATOR
References:
citation: Hendren RL, James SJ, Widjaja F, Lawton B, Rosenblatt A, Bent S. Randomized, Placebo-Controlled Trial of Methyl B12 for Children with Autism. J Child Adolesc Psychopharmacol. 2016 Nov;26(9):774-783. doi: 10.1089/cap.2015.0159. Epub 2016 Feb 18.