MedTrace Logo

Trial of Kuvan in Lesch-Nyhan Disease

NCT00935753 · Status: WITHDRAWN · Phase: PHASE2/PHASE3 · Type: INTERVENTIONAL

Last updated 2019-08-02

No results posted yet for this study

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.

Conditions

  • Behavioral Manifestations of Lesch-Nyhan Disease

Interventions

DRUG

sapropterin

oral 100mg tablets taken intact or dissolved in water or apple juice with morning meal for up to 60 days

Sponsors & Collaborators

Principal Investigators

  • William L Nyhan, MD, PhD · University of California, San Diego

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
4 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2009-04-30
Primary Completion
2010-02-28
Completion
2010-02-28

Countries

  • United States

Study Locations

More Related Trials

Entities

Companies

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT00935753 on ClinicalTrials.gov