Study of the Safety, Pharmacokinetics and Efficacy of HPN-100, in Pediatric Subjects With Urea Cycle Disorders (UCDs)

PHASE3CompletedINTERVENTIONAL
Enrollment

23

Participants

Timeline

Start Date

July 31, 2011

Primary Completion Date

February 28, 2013

Study Completion Date

March 31, 2013

Conditions
Urea Cycle Disorders
Interventions
DRUG

HPN-100

HPN-100 is a pro-drug of PAA that combines with glutamine to provide an alternative vehicle for waste nitrogen elimination. It is a liquid with minimal taste and odor. Approximately three teaspoons of HPN-100 (\~17.4 mL) delivers an equivalent amount as PBA that 40 tablets of NaPBA.

Trial Locations (8)

10029

Mount Sinai School of Medicine, New York

20010

Children's National Medical Center, Washington D.C.

44106

University Hospitals Case Medical Center, Cleveland

55455

University of Minnesota, Minneapolis

77030

Baylor College of Medicine, Houston

90404

UCLA Pediatrics/Genetics, Los Angeles

97239

Oregon Health & Science University, Portland

04101

Maine Medical Center, Portland

Sponsors

Lead Sponsor

All Listed Sponsors
lead

Amgen

INDUSTRY