Status:
COMPLETED
Tetrahydrobiopterin Treatment in Children With Idiopathic Cognitive Developmental Disorders
Lead Sponsor:
The University of Texas Health Science Center, Houston
Collaborating Sponsors:
BioMarin Pharmaceutical
Conditions:
Autism Spectrum Disorder
Eligibility:
All Genders
2-6 years
Phase:
NA
Brief Summary
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 supplem...
Detailed Description
SUMMARY PURPOSE: 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 benefi...
Eligibility Criteria
Inclusion
- 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.
Exclusion
- 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.
Key Trial Info
Start Date :
July 1 2010
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
September 1 2011
Estimated Enrollment :
10 Patients enrolled
Trial Details
Trial ID
NCT01141595
Start Date
July 1 2010
End Date
September 1 2011
Last Update
April 1 2014
Active Locations (1)
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1
Arkansas Children's Hospital Research Institute
Little Rock, Arkansas, United States, 72202