Neonatal onset urea cycle disorder
Conditions
Interventions
Intervention type:
Name of intervention:Cell transplantation
Dose form / Japanese Medical Device Nomenclature:OTHER
Route of administration / Site of application:INTRAHEPATIC
Dose per administration:2
Sponsors
National center for child health and development
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: (1)Patient presents with neonatal onset urea cycle disorder (2)Patient's body weight is 6 kg or less; that is why patient has to wait the timing of liver transplantation (3)Patient's LVEF is 50% or more (4)Patient's SPO2 is 95% or more at rest
Exclusion criteria
Exclusion criteria: (1)Patient is suspected of HBV, HCV or HIV infection (2)Patient is suspected of hypersensitivity to bovine-, pig- or murine-derived ingredients (3)Patient has a severe complication irrelevant to urea cycle disorder
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Incidence and severity of adverse events/failures | — |
Secondary
| Measure | Time frame |
|---|---|
| Transition of blood ammonia level, protein intake, treatments | — |
Countries
Japan
Contacts
Public ContactYasuyuki Fukuhara
National center for child health and development
Outcome results
None listed