To Evaluate the Safety of Long-term Use of HPN-100 in the Management of Urea Cycle Disorders (UCDs)

Completed · Phase 4

Conditions studied: Urea Cycle Disorders

In brief

This was an open-label, long-term safety study of HPN-100 (RAVICTI; glycerol phenylbutyrate) in participants with a urea cycle disorder (UCD) who completed the safety extensions of HPN-100-005 (NCT00947544; HPN-100-005SE), HPN-100-006 (NCT00947297; HPN-100-007), or HPN-100-012 (NCT01347073; HPN-100-012SE). The initial studies were 1- to 2-week crossover studies, and their associated safety extensions were 12-month, open-label studies. All participants who completed the initial studies were eligible to enroll in the associated safety extension studies, and new participants were also permitted to enroll directly into the safety extension studies.

Key facts

Study ID

NCT01257737

Run by

Amgen

People needed

88

Starts

2010-10-04

Expected to finish

2017-02-16

Last updated by the study team

2024-08-22

Who can join

Age: 1 and older. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• Male and female subjects who completed Studies HPN-100-005SE, HPN-100-007, or HPN-100-012SE
• Signed informed consent by participant and/or participant's legally authorized representative
• Negative pregnancy test for all females of childbearing potential

You may not qualify if…

• Any clinical or laboratory abnormality or medical condition that, at the discretion of the investigator, may have put the participant at increased risk when participating
• Known hypersensitivity to PAA (phenylacetate) or PBA (phenylbutyrate).
• Liver transplant, including hepatocellular transplant
• Pregnant, breastfeeding or lactating females

Where it is running

• UCLA Pediatrics/Genetics — Los Angeles, California, United States
• Stanford University School of Medicine — Palo Alto, California, United States
• Denver Children's Hospital — Aurora, Colorado, United States
• Children's National Medical Center — Washington D.C., District of Columbia, United States
• Maine Medical Center — Portland, Maine, United States
• University of Minnesota Medical Center — Minneapolis, Minnesota, United States
• Mount Sinai School of Medicine — New York, New York, United States
• University Hospitals Case Medical Center — Cleveland, Ohio, United States
• Nationwide Children's Hospital — Columbus, Ohio, United States
• Oregon Health & Science University — Portland, Oregon, United States
• Children's Hospital of Pittsburg of UPMC — Pittsburgh, Pennsylvania, United States
• Baylor College of Medicine — Houston, Texas, United States
• University of Utah — Salt Lake City, Utah, United States
• Seattle Children's Hospital — Seattle, Washington, United States
• Children's Hospital of Wisconsin — Milwaukee, Wisconsin, United States
• The Hospital for Sick Children — Toronto, Ontario, Canada

Full record on ClinicalTrials.gov

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