Urea cycle disorder
Conditions
Interventions
- The switch-over part of the study will last 2 weeks during which subjects will be switched from sodium phenylbutyrate (NaPBA) to HPN100 (NaPBA for the first 7 days and HPN-100 for the next 7 days).
Sponsors
Hiroiku Furukawa
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: - Patients diagnosed with urea cycle disorder (UCD) by genetic testing, enzymatic testing, and/or biochemical testing. - If participating in the switch-over part, patients being treated for UCD with NaPBA and who has not changed dosage and administration of NaPBA for at least one week prior to the start of study drug administration (Day 1). - If newly participating in the extension part, NaPBA has not been administered for at least 30 days prior to the start of study drug administration (Month 0). - Patients who are judged to be sufficiently capable of undergoing the tests and observations conducted in this study.
Exclusion criteria
Exclusion criteria: - Patients who have a blood ammonia level 100 micro mol/L (170 micro g/dL) or more at screening or has symptoms and/or signs of hyperammonemia within at least 2 weeks prior to the screening visit. If ammonia level is controlled and stable for at least 14 days, re-screening may be allowed at the discretion of the Investigator etc. - Patients with active infections (viral and bacterial) or other diseases that may affect blood ammonia levels. - Patients who have received sodium benzoate within 1 week prior to the start of study drug administration. - Patients with history of hypersensitivity to phenylbutyrate and/or phenylacetic acid. - Patients who have undergone liver transplantation (including hepatocellular transplantation)
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| The blood ammonia levels 24-hour area under the curve at the last day of NaPBA administration (Day 7) and the last day of HPN-100 administration (Day 14) | — |
Secondary
| Measure | Time frame |
|---|---|
| - Maximum blood ammonia levels on the last day of NaPBA and HPN-100 administration - Mean blood ammonia levels on the last day of NaPBA and HPN-100 administration - During NaPBA and HPN-100 administration period, the number and percentage of patients whose ammonia levels exceeded the upper limit of reference (ULN) - NaPBA and PK parameters of the main metabolites of HPN-100 (PAA, PBA and PAGN) - Cumulative 24-hour urinary PAGN (U-PAGN0-24) excretion on the last day of NaPBA and HPN-100 administration - The correlation between U-PAGN0-24 excretion and AUCNH3, 0-24 - Glutamine values on the last day of NaPBA and HPN-100 administration - Adverse events | — |
Contacts
Public ContactFurukawa Hiroiku
OrphanPacific, Inc.
Outcome results
None listed