A Folinic Acid Intervention for Autism Spectrum Disorders
Researchers at Arkansas Children's Hospital Research Institute, in conjunction with the Rossignol Medical Center and Driscoll Children's Hospital, are conducting a study looking at the effects of Folinic Acid on language in Autism Spectrum Disorder and language impairment. The study has 3 phases. Phase 1 confirms that your child has language impairment (there is no compensation for this visit). If language impairment is verified in the phase 1 screening, then your child will be eligible for phase 2. Phase 2 consists of receiving 12 weeks of folinic acid or an inactive placebo, in addition to several evaluations of your child's abilities and a single blood test. Children that complete phase 2 will be eligible for a 12 week open-label trial of folinic acid which is phase 3.
Autism Spectrum Disorder
Pervasive Development Disorders
Drug: Folinic Acid and placebo
Drug: Folinic Acid
|Study Design:||Allocation: Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Parallel Assignment
Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
|Official Title:||A Folinic Acid Intervention for ASD: Links to Folate Receptor-alpha Autoimmunity & Redox Metabolism|
- Language Improvement [ Time Frame: (baseline and 12 weeks ) ] [ Designated as safety issue: No ]Language (measured by the receptive and expressive CELF language index, and preschool language scale (PLS), as needed) will be the primary outcome for the study. Both preliminary studies have suggested that the folinic acid intervention will be associated with receptive and expressive language improvements.
- Improved Stereotyped Behavior and Improved Social Skills [ Time Frame: (baseline, 6, and 12 weeks) ] [ Designated as safety issue: No ]Stereotyped behavior (as measured by the OACIS (not at 6 weeks), ASQ, RBS-R, and ABC) and social skills (as measured by the Vineland (not at 6 weeks), ASQ, and SRS) will be the secondary outcomes.
|Study Start Date:||May 2012|
|Estimated Study Completion Date:||September 2017|
|Estimated Primary Completion Date:||September 2017 (Final data collection date for primary outcome measure)|
No Intervention: Phase I
Baseline Visit Phase 1: The screening portion of the CELF will be administered to the child to screen for language impairment. If a child is determined to be pre-verbal they will automatically qualify,If there is no language impairment, the subject will not be eligible. If language impairment is confirmed, the participant will immediately go on to the baseline visit of Phase 2.
Phase II: 12 week Folinic Acid or Placebo intervention
The child will be consented for Phase 2 (the RCT) and undergo a blood draw (up to 20mL) for metabolic and autoantibody testing. the child will undergo language and behavioral assessment while the parent will be interviewed for the Vineland and other questionnaires (ASQ, RBS-R, SRS, and ABC). Demographic information including; age, race, gender, and ethnicity will be collected. The research pharmacist will randomize the participant to either Intervention A or B (only the research pharmacist will know which intervention has the folinic acid). The research pharmacist will distribute the drug or placebo to the parent and instruct the parent of the proper administration of the intervention. This will be considered the 12 week randomly controlled clinical trial that is investigating the safety and efficacy of folinic acis interventions in ASD and will last for approximately 12 weeks. At the end of 12 weeks, the same assessments that were conducted at baseline will be readministered
Drug: Folinic Acid and placebo
Capsules of folinic acid and placebo will be administered in 1mg/kg/day in two divided doses (0.5mg/kg/dose; 25mg/day maximum) for two weeks followed by 2 mg/kg/day with a maximum dose of 50mg/day provided the lower dose is well tolerated for 10 weeks.
Experimental: Phase III: Open Label Extension of Folinic Acid
If consent for Phase 3 is signed by parents with children who qualify for Phase 3, the research pharmacist will provide a 12 week supply of folinic acid to the parent. This arm will be offered to all clients that completed phase 2 of the trial. After consenting and 12 weeks of folinic acid dosing, the client will come back and complete the same protocol and be tested on the same measures used in phase II of the study. This will be a rolling stopping point so that new therapies can be started, if the parent/caregiver is so inclined
Drug: Folinic Acid
capsules of folinic acis will be provided. The target dose will be 1mg/kg/day in two divided doses (0.5mg/kg/dose; 25mg/day maximum) for two weeks followed by 2 mg/kg/day with a maximum dose of 50mg/day provided the lower dose is well tolerated, for 10 weeks.
Show Detailed Description
Please refer to this study by its ClinicalTrials.gov identifier: NCT01602016
|United States, Arkansas|
|Arkansas Children's Hospital Research Institute|
|Little Rock, Arkansas, United States, 72205|
|United States, California|
|Rossignol Medical Center|
|Irvine, California, United States, 92618-8623|
|United States, Texas|
|Driscoll Children's Hospital|
|Corpus Christi, Texas, United States, 78411|
|Principal Investigator:||Richard E Frye, M.D./Ph.D.||Director of Autism Research|
|Principal Investigator:||Daniel Rossignol, MD||Rossignol Medical Center|
|Principal Investigator:||William Reed, MD||Driscoll Children's Hospital|