Study of the Safety and Tolerability of HPN-100 Compared to Sodium Phenylbutyrate in Children With Urea Cycle Disorders

PHASE2CompletedINTERVENTIONAL
Enrollment

17

Participants

Timeline

Start Date

March 31, 2010

Primary Completion Date

August 31, 2011

Study Completion Date

August 31, 2011

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 that 40 tablets of NapBA do.

DRUG

NaPBA

NaPBA tablets for oral administration and NaPBA powder for oral, nasogastric, or gastrostomy tube administration contain the active ingredient sodium phenylbutyrate. NaPBA is a prodrug and is rapidly metabolized to PAA, the metabolically active compound that conjugates with glutamine via acetylation to form PAGN, which is excreted by the kidneys.

Trial Locations (8)

10029

Mount Sinai School of Medicine, New York

15201

University of Pittsburgh, Pittsburgh

20010

The George Washington DC Children's National Medical Center, Washington D.C.

53226

Medical College of Wisconsin, Milwaukee

77030

Baylor College of Medicine, Houston

90095

UCLA, Los Angeles

98105

Seattle Children's Hospital, Seattle

M5G1X8

The Hospital for Sick Children, Toronto

Sponsors

Lead Sponsor

All Listed Sponsors
lead

Amgen

INDUSTRY

NCT00947544 - Study of the Safety and Tolerability of HPN-100 Compared to Sodium Phenylbutyrate in Children With Urea Cycle Disorders | Biotech Hunter | Biotech Hunter