N/A
N=10
Tetrahydrobiopterin Treatment in Children With Idiopathic Cognitive Developmental Disorders
Autism Spectrum Disorder
Bottom Line
View on ClinicalTrials.gov: NCT01141595 ↗Enrolled (actual)
10
Serious AEs
0.0%
Results posted
Feb 2014
Primary outcome: Primary: Preschool Language Scales — 7.6 Raw score units / 16 weeks
Study Design & Population
- Study type
- Interventional
- Phase
- N/A
- Interventions
- sapropterin dihydrochloride (Drug)
- Age
- Pediatric · 2+ yrs
- Sex
- All
- Sponsor
- The University of Texas Health Science Center, Houston
- Primary completion
- Sep 2011
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Preschool Language Scales |
7.6 | — |
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
Inclusion Criteria
- 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 Criteria
- 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.
Data sourced from ClinicalTrials.gov (NCT01141595). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.