This trial is active, not recruiting.

Condition urea cycle disorders
Treatment hpn-100
Phase phase 4
Sponsor Horizon Therapeutics, LLC
Start date October 2010
End date December 2016
Trial size 88 participants
Trial identifier NCT01257737, HPN-100-011


The present protocol is a treatment protocol to allow continued use of HPN-100 for the treatment of UCD subjects who complete 12 months of treatment in Study HPN-100-005, (NCT00947544), HPN-100-007, (NCT00947297), or HPN-100-012, (NCT01347073). Under this protocol, long-term safety will continue to be assessed.

United States No locations recruiting
Other Countries No locations recruiting

Study Design

Endpoint classification safety study
Intervention model single group assignment
Masking open label
Primary purpose treatment
Subjects will continue treatment with HPN-100 after having completed HPN-100 treatment in HPN-100-005, HPN-100-007, or HPN-100-012.
hpn-100 GT4P, Glyceryl tri-(4-phenylbutyrate)
HPN-100 will be administered orally three times daily (TID) with meals. The maximum recommended dose of HPN-100 in subjects weighing less than 20 kg is 0.53 mL/kg/day (equivalent to 600 mg/kg/day of NaPBA), and is 11.48 mL/m2/day in heavier subjects (equivalent to 13g/m2/day of NaPBA). The maximum HPN-100 dose should be 17.4 mL/day, which is equivalent to 20 g/day of NaPBA.

Primary Outcomes

Rate of Adverse Events
time frame: Every six months for 2 years

Secondary Outcomes

Venous ammonia levels
time frame: Every six months for two years
Number of hyperammonemic crises
time frame: Every six months for two years
Neuropsychological testing
time frame: Every twelve months for two years
Causes of hyperammonemic crises
time frame: Every six month for two years

Eligibility Criteria

Male or female participants at least 1 year old.

Inclusion Criteria: - Male and female subjects who completed HPN-100-005, HPN-100-007, or HPN-100-012 - Signed informed consent by subject and/or subject's legally authorized representative - Negative pregnancy test for all females of childbearing potential Exclusion Criteria: - Any clinical or laboratory abnormality or medical condition that, at the discretion of the investigator, may put the subject at increased risk when participating - Known hypersensitivity to PAA or PBA - Liver transplant, including hepatocellular transplant - Pregnant, breastfeeding or lactating females

Additional Information

Official title Long Term Use of HPN-100 in Urea Cycle Disorders
Description The present protocol will allow subjects continued use of HPN-100 following their treatment and study completion in Study HPN-100-005, HPN-100-007, or HPN-100-012. Subjects will be able to continue in this study until approval and commercial availability of HPN-100 in each country participating in this study. Subjects will be required to return to the clinic every 6 months in which physical exam, safety labs, dietary assessment and neuropsychological testing will occur. Subjects will be dispensed sufficient HPN-100 to last until the next clinic visit.
Trial information was received from ClinicalTrials.gov and was last updated in September 2016.
Information provided to ClinicalTrials.gov by Horizon Pharma Ireland, Ltd., Dublin Ireland.