Tetrahydrobiopterin Treatment in Children With Idiopathic Cognitive Developmental Disorders
Completed · Not applicable
Conditions studied: Autism Spectrum Disorder
In brief
The purpose of this study is to develop biomarkers for central nervous system BH4 concentration in children with autism so they can be easily screened to determine if they may benefit from BH4 supplementation. Kuvan is a synthetic form of BH4. BH4 is an enzyme cofactor that is essential for several critical metabolic functions.
Key facts
- Study ID
- NCT01141595
- Run by
- The University of Texas Health Science Center, Houston
- People needed
- 10
- Starts
- 2010-07-01
- Expected to finish
- 2011-09-01
- Last updated by the study team
- 2014-04-01
Who can join
Age: 2 and older, up to 6. Sex: any. Healthy volunteers: not accepted.
You may qualify if…
- Children 2 years 0 months to 6 years 11 months of age will be recruited.
- Delay in language and/or social development with or without delays in gross and/or fine motor development.
- CSF BH4 level less than or equal to 30 nM/L as determined by lumbar puncture.
- Adaptive Behavior greater than 50 as determined by the Vineland Adaptive Behavior Scale.
You may not qualify if…
- Epilepsy / Seizure disorder as determined by medical history.
- Epileptiform discharges without clinical seizures.
- Metabolic disorder that would be a contraindication to tetrahydrobiopterin treatment.
- Genetic disorder
- Therapies that cannot be maintained at a constant level or have not been ongoing for more than 3 months as determined by medical history
- Prematurity
- Developmental delay isolated to motor delay.
- Current gastroesophageal reflux
- Current or history of liver or kidney disease
- Severe irritability (as determined by the aberrant behavior checklist)
- Drugs known to affect folate metabolism (e.g., methotrexate) and their derivatives
- Patients who are receiving drugs that affect nitric oxide-mediated vasorelaxation (e.g., PDE-5 inhibitors such as sildenafil, vardenafil, or tadalafil)
- Patients who are receiving levodopa.
Where it is running
- Arkansas Children's Hospital Research Institute — Little Rock, Arkansas, United States
Full record on ClinicalTrials.gov
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