Trial of Kuvan in Lesch-Nyhan Disease

Interested in this Trial?

Contact Us

Trial Status Withdrawn

Trial Identifier

NCT00935753

Condition

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.

Trial 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.

Eligibility Criteria

Birth Sex

All

Age

From 4 Years

Healthy Volunteers

No

Drug/Treatment:

sapropterin

Phase:

Phase 2/Phase 3

Study Type:

Interventional

Study Started:

2009-04

Study Updated:

2019-08-02

Trial Locations

  • UCSD Mitochondrial and Metabolic Disease Center

    San Diego, California, United States

Inclusion Criteria

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

Exclusion Criteria

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

Contact BioMarin Clinical Trials

Please complete and submit the following form to express interest in participating and request more information about one of our active clinical trials

*required fields

"*" indicates required fields

Please select the trial that you are interested in from the dropdown below. Note that this list only includes currently active trials. If you cannot find the trial you are seeking, please enter the study identifier and title in the comments box below, along with any other details you would like us to know to help address your inquiry.
For more Information about BioMarin's privacy obligations or to exercise your privacy rights, please visit BioMarin's Data Privacy Center.
This field is hidden when viewing the form
This field is hidden when viewing the form
This field is hidden when viewing the form
This field is hidden when viewing the form