Dose-Escalation Safety Study of HPN-100 to Treat Urea Cycle Disorders

Status Completed

Clinical Trial Summary

The purpose of this study is to determine whether HPN-100 is safe and tolerable in subjects with Urea Cycle Disorders.

Clinical Trial Description

When protein is broken down in the body, nitrogen is formed. In healthy individuals, the body combines this nitrogen with other molecules to create a harmless substance called urea, which is excreted in the urine. Patients with Urea Cycle Disorders (UCD) are unable to create as much urea from nitrogen, and therefore, toxic levels of nitrogen can accumulate in the body, causing harm. To treat these patients, doctors usually have the patient consume less protein and supplement certain amino acids that may be lacking. A drug called Buphenyl® is sometimes prescribed as an adjunctive treatment for the chronic maintenance of UCD patients in order to keep ammonia levels down. Some issues with Buphenyl® include a high pill burden (up to 40 pills per day), bad taste and odor, and high sodium content. Like Buphenyl®, HPN-100 provides an alternate way for the body to dispose of nitrogen, other than through the urea cycle. Unlike Buphenyl®, HPN-100 is an odorless, tasteless, concentrated oil that does not contain large amounts of sodium. ;

Study Design

Related Conditions & MeSH terms

Clinical Trial Details

NCT number	NCT00551200
Study type	Interventional
Source	Horizon Pharma Ireland, Ltd., Dublin Ireland
Contact
Status	Completed
Phase	Phase 2
Start date	October 2007
Completion date	December 2008

View Details

See also
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