Status:
COMPLETED
To Evaluate the Safety of Long-term Use of HPN-100 in the Management of Urea Cycle Disorders (UCDs)
Lead Sponsor:
Amgen
Conditions:
Urea Cycle Disorders
Eligibility:
All Genders
1+ years
Phase:
PHASE4
Brief Summary
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 (NCT009...
Detailed Description
The duration of treatment in this study was open-ended. Participants were to return for clinic visits as prescribed by the investigator, and were to be seen at a minimum of every 6 months. At each cli...
Eligibility Criteria
Inclusion
- 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
Exclusion
- 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
Key Trial Info
Start Date :
October 4 2010
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
February 16 2017
Estimated Enrollment :
88 Patients enrolled
Trial Details
Trial ID
NCT01257737
Start Date
October 4 2010
End Date
February 16 2017
Last Update
August 22 2024
Active Locations (16)
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1
UCLA Pediatrics/Genetics
Los Angeles, California, United States, 90095
2
Stanford University School of Medicine
Palo Alto, California, United States, 94305
3
Denver Children's Hospital
Aurora, Colorado, United States, 80045
4
Children's National Medical Center
Washington D.C., District of Columbia, United States, 20010