Efficacy and Safety of HPN-100 for the Treatment of Adults With Urea Cycle Disorders

PHASE3CompletedINTERVENTIONAL
Enrollment

46

Participants

Timeline

Start Date

October 31, 2009

Primary Completion Date

September 30, 2010

Study Completion Date

September 30, 2010

Conditions
Urea Cycle Disorders
Interventions
DRUG

HPN-100

HPN-100 is a triglyceride that has a similar mechanism of action as NaPBA. It is a liquid with minimal taste and odor. Three teaspoons of HPN-100 (\~17.4mL) delivers equivalent amount of PBA in 40 tablets of NaPBA.

DRUG

Buphenyl (NaPBA)

Buphenyl (NaPBA) will be the comparator drug to HPN-100 in this study.

Trial Locations (22)

10029

Mount Sinai School of Medicine, New York

10595

Westchester Medical Center, Valhalla

15224

Children's Hospital of Pittsburgh of UPMC, Pittsburgh

20010

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

21201

SNBL-Clinical Pharmacology Center, Baltimore

32611

University of Florida, Gainesville

43205

Nationwide Children's Hospital, Columbus

44106

University Hospitals Case Medical Center, Cleveland

52242

University of Iowa, Iowa City

53226

Medical College of Wisconsin, Milwaukee

55454

University of Minnesota Medical Center, Minneapolis

77030

Baylor College of Medicine, Houston

80045

Denver Children's Hospital, Aurora

84132

University of Utah, Salt Lake City

90095

UCLA, Los Angeles

90806

Long Beach Memorial, Long Beach

94304

Stanford University, Stanford

97239

Oregon Health & Science University, Portland

06510

Yale School of Medicine, New Haven

04102

Maine Medical Center, Portland

02111

Tufts-New England Medical Center, Boston

M5G 1X8

The Hospital for Sick Children, Toronto

Sponsors

Lead Sponsor

All Listed Sponsors
lead

Amgen

INDUSTRY