drugset / Trial / NCT01141595
Tetrahydrobiopterin Treatment in Children With Idiopathic Cognitive Developmental Disorders
Completed
10 enrolled
The University of Texas Health Science Center, Houston
BioMarin Pharmaceutical · collab
NaSingle-groupOpen-labelTreatment
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.
Timeline
- Start
- 2010-07
- Primary completion
- 2011-09
- Completion
- 2011-09
Publications
- Background Frye RE, Huffman LC, Elliott GR. Tetrahydrobiopterin as a novel therapeutic intervention for autism. Neurotherapeutics. 2010 Jul;7(3):241-9. doi: 10.1016/j.nurt.2010.05.004.
- Background Frye RE. Central tetrahydrobiopterin concentration in neurodevelopmental disorders. Front Neurosci. 2010 Jul 8;4:52. doi: 10.3389/fnins.2010.00052. eCollection 2010.
- Results Frye RE, DeLatorre R, Taylor HB, Slattery J, Melnyk S, Chowdhury N, James SJ. Metabolic effects of sapropterin treatment in autism spectrum disorder: a preliminary study. Transl Psychiatry. 2013 Mar 5;3(3):e237. doi: 10.1038/tp.2013.14.
Drugs
| Evaluation | Drug | Modality | Dose | Route |
|---|---|---|---|---|
| Subject | sapropterin | Unknown | 20 mg/kg | Oral |