Autistic DisorderPhase 2/3B12NCT01039792
Trial of Methyl B12 on Behavioral and Metabolic Measures in Children With Autism
A Phase 2/3 study testing Methyl B12 in people with Autistic Disorder, run by University of California, San Francisco at 1 site in United States.
Trial summary
The purpose of this study is to determine whether the supplement Methyl B12 is effective in treating some of the symptoms of Autism. Read more about this study
- Who is it for?
- Ages 3 to 7 years
- Conditions the study is for
- Autistic Disorder
- What is being tested?
- Methyl B12
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 57 participants
- Where
- 1 siteUnited States
- Study dates
- Jan 2010to Nov 2013
- Phase
- Phase 2/3
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 3 to 7 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- 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
Who cannot join
- 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 locations and contacts
1 site in 1 country.
About this study
Official title: Double Blind Placebo Controlled Trial of Methyl B12 on Behavioral and Metabolic Measures in Children With Autism
The purpose of this study is to determine whether the supplement Methyl B12 is effective in treating some of the symptoms of Autism.
Read the 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.
- Conditions
- Autistic Disorder
- Being tested
- Methyl B12, Placebo
- Keywords
- Autism, Pervasive Developmental Disorders
- Medical subject headings
- Autistic Disorder, Child Development Disorders, Pervasive
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Methyl B12Drug
Also called Vitamin B12, methylcobalamin
75 µg/Kg subcutaneously injected once every 3 days
PlaceboDietary supplement
placebo
| Study group | Role | What they receive |
|---|---|---|
| Placebo | Placebo comparator | Placebo Receives: Dietary Supplement: Placebo |
| Active | Experimental | Active Methyl B12 Receives: Drug: Methyl B12 |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: 8 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted25 Dec 2009 (estimated)
- Study startJan 2010
- Primary completionOct 2013
- Study completionNov 2013
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Autistic DisorderStudies of the same condition, open and under way.
- More trials run by University of California, San FranciscoThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2/3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Triple blind
- Who is masked
- Participant, Care provider, Investigator
- Enrolment
- 57 participants (actual)
Sponsor and investigators
- Lead sponsor
- University of California, San Francisco
- Sponsor type
- University, hospital or other
- Collaborators
- University of California, Davis, Arkansas Children's Hospital Research Institute
- Responsible party
- Principal investigator, Robert Hendren, University of California, San Francisco
- Investigators
- Robert L. Hendren, DO, Principal investigator, University of California, San Francisco
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01039792
- Sponsor's protocol ID
- Autism Speaks 3031
- Acronym
- B12
- Status last verified
- Jan 2017
- First submitted
- 23 Dec 2009
- Last updated
- 24 Feb 2017
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
Publications
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.
PubMed 26889605
Study at a glance
From the registry record, last updated by the sponsor on 24 Feb 2017. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Double Blind Placebo Controlled Trial of Methyl B12 on Behavioral and Metabolic Measures in Children With Autism
- ClinicalTrials.gov ID
- NCT01039792
- Acronym / sponsor ID
- B12 / Autism Speaks 3031
- Conditions
- Autistic Disorder
- Keywords
- Autism, Pervasive Developmental Disorders
- Phase
- Phase 2/3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 57 participants (actual)
- Lead sponsor
- University of California, San FranciscoUniversity, hospital or other
- Collaborators
- University of California, Davis, Arkansas Children's Hospital Research Institute
- Study start
- Jan 2010
- Primary completion
- Oct 2013 (actual)
- Study completion
- Nov 2013 (actual)
- First posted
- 25 Dec 2009 (estimated)
- Results
- Posted 24 Feb 2017
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Triple blind (participant, care provider, investigator)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Placebo | Placebo comparator | Dietary Supplement: PlaceboPlacebo |
| Active | Experimental | Drug: Methyl B12Active Methyl B12 |
Who may qualify
- Ages
- 3 to 7 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (5 listed)
- 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
Key exclusion criteria (7 listed)
- Bleeding disorder
- Cancer
- Seizure disorder
- Fragile X or other known genetic cause of autism
- Perinatal brain injury (i.e.: cerebral palsy)
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Methyl B12Also: Vitamin B12, methylcobalamin75 µg/Kg subcutaneously injected once every 3 days | Drug | Active |
| Placeboplacebo | Dietary supplement | Placebo |
Primary outcome
Clinical Global Impression-Improvement (CGI-I)
Time frame: 8 weeks
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.
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 24 Feb 2017.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Derived from this study: PubMed 26889605Hendren 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.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Robert L. Hendren, DO principal investigator
University of California, San Francisco
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2/3 study testing Methyl B12 in people with Autistic Disorder, run by University of California, San Francisco at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 5 inclusion and 7 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 24 Feb 2017. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 24 Feb 2017). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Clinical Global Impression-Improvement (CGI-I) (time frame: 8 weeks). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT01039792, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 24 Feb 2017.