Trial of Kuvan in Lesch-Nyhan Disease

Withdrawn before enrolling · Phase 2/Phase 3

Conditions studied: Behavioral Manifestations of Lesch-Nyhan Disease

In brief

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.

Key facts

Study ID

NCT00935753

Run by

University of California, San Diego

People needed

0

Starts

2009-04-01

Expected to finish

2010-02-01

Last updated by the study team

2019-08-02

Who can join

Age: 4 and older. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• Ages 4 years and older
• Must have documented evidence of HPRT deficiency.
• Be on a stable treatment regimen for 30 days or more
• Willing and able to travel to San Diego for the study visits
• Have a local neurologist or physician familiar with the patient or experienced in managing behavioral/neuromuscular disorders and willing to assist with study procedures and adverse events if necessary

You may not qualify if…

• Concurrent enrollment in an investigational drug study
• Currently taking levodopa
• Elevated liver enzymes
• Renal or liver impairment or disease
• Inability to comply with required study procedures

Where it is running

• UCSD Mitochondrial and Metabolic Disease Center — San Diego, California, United States

Full record on ClinicalTrials.gov

Trial information comes from ClinicalTrials.gov and is refreshed daily. TrialsForMe does not provide medical care and does not run the studies it lists.