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Folate Rechallenge
This study is currently recruiting participants.
Verified by Baylor College of Medicine, May 2008
Sponsored by: Baylor College of Medicine
Information provided by: Baylor College of Medicine
ClinicalTrials.gov Identifier: NCT00672360
  Purpose

New evidence suggests that autistic disorder (AD) may be associated with abnormalities in folate metabolism, which is a process that affects genetic expression by facilitating the formation of methyl donors for DNA methylation. Limited data show that some children with AD show behavioral improvements with folic acid (FA) therapy, while others show a worsening effect. If behavioral worsening is linked with abnormalities in folate metabolism, then nutritional modifications could normalize these processes and result in clinical improvements. To address this premise, we propose a randomized, placebo-controlled crossover pilot study with two phases. The first phase will focus on the behavioral and biochemical responses of children with AD to high-dose folic acid supplementation. Because FA is an inactive folate that requires biochemical conversion to become active, and select genotypes impede this conversion, our general hypothesis is that FA will yield behavioral improvements in some children but exacerbate problem behaviors in others. During the second phase, children who had a worsened behavioral response to FA during phase 1 will participate in an open-label trial of high-dose Metafolin® supplementation. The focus here would similarly be on the behavioral and biochemical outcomes of participating children following treatment with the study supplement. Because Metafolin® is an active folate metabolite that should not be affected by genotypes in the folate pathway, our general hypothesis for phase 2 is that Metafolin® would yield behavioral improvements without the risk for behavioral worsening. Results from this project may provide support for continued study of the potential relationship between folate metabolism and problem behaviors among children with AD, potentially justifying the need to examine effects of folate supplementation among a larger sample of affected children.


Condition Intervention Phase
Autism
Dietary Supplement: Folic acid
Dietary Supplement: Placebo
Phase II

MedlinePlus related topics: Autism Dietary Supplements
Drug Information available for: Folic acid
U.S. FDA Resources
Study Type: Interventional
Study Design: Treatment, Randomized, Double Blind (Subject, Caregiver, Investigator), Placebo Control, Crossover Assignment, Efficacy Study
Official Title: Folate Rechallenge: A Pilot Study

Further study details as provided by Baylor College of Medicine:

Primary Outcome Measures:
  • Behavior as measured with the Aberrant Behavior Checklist (ABC) and the Pervasive Developmental Disorder Behavior Inventory (PDDBI). [ Time Frame: Pre-, mid-, and post-treatment during each phase (folic acid and placebo). ] [ Designated as safety issue: No ]

Secondary Outcome Measures:
  • Level of folate metabolites in plasma and site-specific DNA methylation. [ Time Frame: Pre- and post-treatment during each phase (folic acid and placebo). ] [ Designated as safety issue: No ]

Estimated Enrollment: 16
Study Start Date: May 2007
Estimated Study Completion Date: May 2009
Estimated Primary Completion Date: December 2008 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
1: Experimental Dietary Supplement: Folic acid
Compounded capsule, 7.6mg, taken orally, twice daily for 4 weeks.
2: Placebo Comparator Dietary Supplement: Placebo
Compounded capsule of NaCl, taken orally, twice daily for 4 weeks.

  Show Detailed Description

  Eligibility

Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  1. Child participant has a confirmed diagnosis of AD by Autism Diagnostic Observation Schedule and Autism Diagnostic Interview--Revised criteria
  2. Child participant is in stable condition with relatively good control of seizures and no other significant medical problems, including liver, kidney, or heart problems, at the time of entrance to the study. If the child participant is taking medication for a seizure disorder, the investigators will assess his/her eligibility with particular regard to type of seizure medication and other health-related information gleaned during the medical examination
  3. Child participant and parents are willing to comply with the proposed treatments
  4. Child participant is able to take oral medication
  5. Family is fluent in the English language
  6. Parent/caregiver agrees to provide behavioral data on participating children at the requested time points
  7. Family agrees to be contacted weekly by study personnel during the treatment phases

Exclusion Criteria:

  1. Child participant has co-morbid medical and/or genetic disorders, including celiac disease
  2. Child participant has a history of liver or renal disease
  3. Child participant is currently being treated for a serious acute illness
  4. Child participant has a known allergy to any of the proposed supplements
  5. Child participant has uncontrolled seizures
  6. Child participant meets criteria for Asperger's syndrome, PDD-NOS, or does not meet strict criteria for AD
  7. Family is not proficient in the English language
  Contacts and Locations
Please refer to this study by its ClinicalTrials.gov identifier: NCT00672360

Contacts
Contact: Robin P Kochel, Ph.D. 832-822-4299 kochel@bcm.tmc.edu

Locations
United States, Texas
Baylor College of Medicine Recruiting
Houston, Texas, United States, 77030
Contact: Helen Shepherd     713-798-1297     spo@bcm.edu    
Principal Investigator: Arthur L. Beaudet, M.D.            
Sub-Investigator: Robin P. Kochel, Ph.D.            
Sponsors and Collaborators
Baylor College of Medicine
  More Information

Responsible Party: Baylor College of Medicine ( Arthur L. Beaudet, M.D., Chair, Department of Molecular & Human Genetics )
Study ID Numbers: H-20959
Study First Received: May 5, 2008
Last Updated: May 5, 2008
ClinicalTrials.gov Identifier: NCT00672360  
Health Authority: United States: Institutional Review Board

Keywords provided by Baylor College of Medicine:
Autism Spectrum Disorder

Study placed in the following topic categories:
Folic Acid
Developmental Disabilities
Child Development Disorders, Pervasive
Mental Disorders
Autistic Disorder
Mental Disorders Diagnosed in Childhood

Additional relevant MeSH terms:
Vitamin B Complex
Hematinics
Therapeutic Uses
Growth Substances
Vitamins
Hematologic Agents
Physiological Effects of Drugs
Micronutrients
Pharmacologic Actions

ClinicalTrials.gov processed this record on January 16, 2009