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Clinical study of HAES transplantation in patients with neonatal onset urea cycle disorder (HAES transplantation study in patients with neonatal onset urea cycle disorder)

Clinical study of HAES transplantation in patients with neonatal onset urea cycle disorder

Akinari Fukuda

National center for child health and development

2-10-1 Okura Setagaya Tokyo 157-8535

fukuda-a@ncchd.go.jp

Yasuyuki Fukuhara

National center for child health and development

2-10-1 Okura Setagaya Tokyo 157-8535

+81-3-3416-0181

fukuhara-ys@ncchd.go.jp

RECRUITING

Mar. 02, 2019

Interventional

Single arm, non-control, open label

open(masking not used)

No

1-2

(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

(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

Both

Neonatal onset urea cycle disorder

Intervention type: Name of intervention:Cell transplantation Dose form / Japanese Medical Device Nomenclature:OTHER Route of administration / Site of application:INTRAHEPATIC Dose per administration:2.5x10^7 5x10^7 cells/kg Dosing frequency / Frequency of use:QD Twice Planned duration of intervention: Intended dose regimen:2.5 x 10^7 cells/kg/day, two consecutive days, portal injection detailes of teratment arms: Comparative intervention name: Dose form / Japanese Medical Device Nomenclature: Route of administration / Site of application: Dose per administration: Dosing frequency / Frequency of use: Planned duration of intervention: Intended dose regimen:

Incidence and severity of adverse events/failures

Transition of blood ammonia level, protein intake, treatments

+81-3-3416-0181

+81-3-3416-2222

Mar. 15, 2018