NCT00935753
Behavioral Manifestations of Lesch-Nyhan Disease
The below information is taken directly from public registry websites such as ClinicalTrials.gov, EuClinicalTrials.eu, ISRCTN.com, etc. and has not been edited.
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.
All
From 4 Years
No
sapropterin
Phase 2/Phase 3
Interventional
2009-04
2019-08-02
San Diego, California, United States
1. Ages 4 years and older
2. Must have documented evidence of HPRT deficiency.
3. Be on a stable treatment regimen for 30 days or more
4. Willing and able to travel to San Diego for the study visits
5. 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
1. Concurrent enrollment in an investigational drug study
2. Currently taking levodopa
3. Elevated liver enzymes
4. Renal or liver impairment or disease
5. Inability to comply with required study procedure
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