Trial of Kuvan in Lesch-Nyhan Disease
Public ClinicalTrials.gov record NCT00935753. Field values are reproduced from the official study page; the official ClinicalTrials.gov record remains the source of truth for eligibility, enrollment, and contact information.
Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.
Official title
Trial of Kuvan™ (Sapropterin) Treatment in Patients With Lesch Nyhan Disease
Brief summary
Reproduced verbatim from the official ClinicalTrials.gov record. Not medical advice.
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.
Study identification
- NCT ID
- NCT00935753
- Recruitment status
- Withdrawn
- Study type
- Interventional
- Phase
- Phase 2, Phase 3
- Enrollment
- Not listed
Conditions and interventions
Eligibility (public fields only)
- Age range
- 4 Years and older
- Sex
- All
- Healthy volunteers
- Healthy volunteers not accepted
This page does not interpret eligibility. Detailed inclusion and exclusion criteria are on the official ClinicalTrials.gov record.
Study timeline
- Start date
- Mar 31, 2009
- Primary completion
- Jan 31, 2010
- Completion
- Jan 31, 2010
- Last update posted
- Aug 1, 2019
2009 – 2010
United States locations
- U.S. sites
- 1
- U.S. states
- 1
- U.S. cities
- 1
| Facility | City | State | ZIP | Site status |
|---|---|---|---|---|
| UCSD Mitochondrial and Metabolic Disease Center | San Diego | California | 92103 | — |
Site contact phone numbers, emails, and investigator names are intentionally not displayed here. Open the official ClinicalTrials.gov record for site contact information.
About this trial record page
- What this page shows
- Public field values for ClinicalTrials.gov record NCT00935753, including study identification, conditions, interventions, eligibility (age, sex, healthy volunteer), timeline, and U.S. site list.
- What this page does not do
- No medical advice, eligibility judgments, treatment recommendations, study quality scoring, or AI-generated medical summaries. No site contact phone numbers, emails, or investigator names.
- Where the data comes from
- Sourced from the official ClinicalTrials.gov public API. The official record is the source of truth.
- Last refresh
- Last update posted Aug 1, 2019 · Synced Sep 5, 2026
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Open the official record
The complete protocol, eligibility criteria, and contact information for NCT00935753 live on ClinicalTrials.gov.