Overview

Trial of Kuvan in Lesch-Nyhan Disease

Status:
Withdrawn

Trial end date:
2010-02-01

Target enrollment:

Participant gender:

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.

Phase:

Phase 2/Phase 3

Details

Lead Sponsor:

University of California, San Diego

Collaborator:

BioMarin Pharmaceutical