drugset / Trial / NCT00935753
Trial of Kuvan in Lesch-Nyhan Disease
NaSingle-groupOpen-labelTreatment
Summary
To assess the possibility that treatment with Kuvan (a form of tetrahydrobiopterin) will lessen the abnormal behavior and/or neurology commonly found in Lesch-Nyhan disease (LND); to assess biochemical changes as measured in blood and urine.
Timeline
- Start
- 2009-04
- Primary completion
- 2010-02
- Completion
- 2010-02
Drugs
| Evaluation | Drug | Modality | Dose | Route |
|---|---|---|---|---|
| Subject | sapropterin | Unknown | 20 mg/kg | Oral |