Tetrahydrobiopterin Treatment in Children With Idiopathic Cognitive Developmental Disorders

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Trial Status Completed

Trial Identifier

NCT01141595

Condition

Autism Spectrum Disorder

The below information is taken directly from public registry websites such as ClinicalTrials.gov, EuClinicalTrials.eu, ISRCTN.com, etc. and has not been edited.

Trial 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 supplementation. Kuvan is a synthetic form of BH4. BH4 is an enzyme cofactor that is essential for several critical metabolic functions.

Eligibility Criteria

Birth Sex

All

Age

2 Years to 6 Years

Healthy Volunteers

No

Drug/Treatment:

sapropterin dihydrochloride

Phase:

Na

Study Type:

Interventional

Number of Participants:

10

Study Started:

2010-07

Study Updated:

2014-04-01

Trial Locations

  • Arkansas Children's Hospital Research Institute

    Little Rock, Arkansas, United States

Inclusion Criteria

1. Children 2 years 0 months to 6 years 11 months of age will be recruited.
2. Delay in language and/or social development with or without delays in gross and/or fine motor development.
3. CSF BH4 level less than or equal to 30 nM/L as determined by lumbar puncture.
4. Adaptive Behavior greater than 50 as determined by the Vineland Adaptive Behavior Scale.

Exclusion Criteria

1. Epilepsy / Seizure disorder as determined by medical history.
2. Epileptiform discharges without clinical seizures.
3. Metabolic disorder that would be a contraindication to tetrahydrobiopterin treatment.
4. Genetic disorder
5. Therapies that cannot be maintained at a constant level or have not been ongoing for more than 3 months as determined by medical history
6. Prematurity
7. Developmental delay isolated to motor delay.
8. Current gastroesophageal reflux
9. Current or history of liver or kidney disease
10. Severe irritability (as determined by the aberrant behavior checklist)
11. Drugs known to affect folate metabolism (e.g., methotrexate) and their derivatives
12. Patients who are receiving drugs that affect nitric oxide-mediated vasorelaxation (e.g., PDE-5 inhibitors such as sildenafil, vardenafil, or tadalafil)
13. Patients who are receiving levodopa

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