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Last update:2022-06-14
 
|Essential information|
Clinical study of HAES transplantation in patients with neonatal onset urea cycle disorder 
Trial status Active 
JMACCT ID JMA-IIA00412  Date JMACCT ID assigned 2019-02-27 
Recruitment status Recruiting  Trial phas 1-2 
Trial Type INTERVENTIONAL / SAFETY, EFFICACY, EXPLORATORY  Trial Design Single arm, non-control, open label 
Condition or Problem  Neonatal onset urea cycle disorder  Countries of Recruitment Japan 
 

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●Trial Title
Public title Clinical study of HAES transplantation in patients with neonatal onset urea cycle disorder 
Scientific title Clinical study of HAES transplantation in patients with neonatal onset urea cycle disorder 
Title acronym HAES transplantation study in patients with neonatal onset urea cycle disorder 
 

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●Brief Description of Study
Brief summary HAES is derived from human ES cells and directed to differentiate to cells with ammonia metabolism capacity. HAES transplantation is expected as an effective bridging therapy to liver transplantation for patients with urea cycle disorder. This study is conducted to assess the safety and efficacy of HAES transplantation for the neonatal onset patients who have difficulty in instant liver transplantation surgery due to low body weight (6 kg or less). 
 

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●Purpose
Primary objective Assess the safety of HAES transplantation in patients with neonatal onset urea cycle disorder 
Secondary objective(s) Assess the efficacy exploratively 
 

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●Trial Design
Description of trial design Single arm, non-control, open label 
Number of treatment arms 1  Type of control NONE 
Trial blinding schema OPEN LABEL  Randomized NO 
Randomization unit NOT APPLICABLE  Stratified randomization NO 
Dynamic randomization NO  Blocked randomization  NO 
Method of allocation concealment NOT APPLICABLE  
 

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●Trial Phase
Trial phase 1-2 
 

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●Trial Type
Nature of investigation INTERVENTIONAL 
Type of trial SAFETY, EFFICACY, EXPLORATORY 
Clinical trial for drug device development 26001 
 

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●Health Condition(s) or Problem(s) Studied
Trial indication(s) Neonatal onset urea cycle disorder 
Background information Although liver transplantation should be done soon for patients with neonatal onset urea cycle disorder if possible, patients with low body weight (6 kg or less) are high-risk for the surgery. Hepatocyte transplantation is expected therapy as bridging to liver transplantation, but it's difficult to secure enough and qualified hepatocytes constantly. So, HASE has been developed as a promising alternative to qualified hepatocytes. 
Does this study involve oncology? NO  Therapeutic area under study Pediatrics, Endocrinology and Metabolism, Hepato-biliary-pancreatic surgery 
Does this study involve collection of genetic information YES     
 

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●Interventions
Trial indication type TREATMENT  Intervention type  
      Study Intervention(s):
Name of intervention Cell transplantation  Dose form / Japanese Medical Device Nomenclature OTHER 
Route of administration / Site of application INTRAHEPATIC 
Dose per administration 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  
       Control Intervention(s):
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  
 

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●Study Timeline
Date of protocol finalization 2017-12-21  Date of first ethics committee meeting 2018-01-18 
Date of first enrollment 2019-03-02  Study start date 2018-04-30 
Date of last follow-up 2022-09-30  Date of data entry closure  
Data lock date   Completion of data analysis  
Study end date      
 

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●Target sample size
Planned number of subjects  5 
Performed number of subjects  5 
 

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●Primary Outcome(s)
Primary outcome(s) Incidence and severity of adverse events/failures 
Timepoint(s) From HAES transplantation to liver transplantation 
Primary outcome measure Incidence and severity of adverse events/failures 
 

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●Key Secondary Outcome(s)
Secondary outcome(s) Transition of blood ammonia level, protein intake, treatments  
Timepoint(s) From HAES transplantation to liver transplantation  
Secondary outcome measure Transition of blood ammonia level, protein intake, treatments 
 

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●Key Inclusion and Exclusion Criteria
Minimum age No Limit   Maximum age No Limit  
Sex of participants BOTH  Accepts healthy volunteers NO 
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 (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
 
 

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●Trial Status
Recruitment status Recruiting 
Trial status Active 
Reason for study stop  
 

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●Countries of Recruitment
Countrie(s) of recruitment Japan 
 

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●Sponsor
Primary sponsor National center for child health and development 
Secondary sponsor(s)  
 

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●Sources of Funding
Sources of funding Japan agency for medical research and development 
Type of funding organization GOVERNMENT OR PUBLIC CORPORATION 
Country of funding sourc Japan 
 

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●Trial Institutions
Number of study sites 1 
Type of medical institution SPECIALTY CARE HOSPITAL or CLINIC 
Countrie(s) of Study Site(s) Japan 
Prefecture(s) of Study Site(s) Tokyo 
 

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●Contact for Public Queries
Contact name Yasuyuki Fukuhara 
Contact organization National center for child health and development 
Department Division of medical genetics 
Address 2-10-1 Okura Setagaya Tokyo 157-8535 
TEL +81-3-3416-0181  FAX +81-3-3416-2222 
E-mail fukuhara-ys@ncchd.go.jp 
Study website  
 

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●Contact for Scientific Queries
Investigator name Akinari Fukuda 
Investigator organization National center for child health and development 
Department Division of transplant surgery, Organ transplantation center  
Address 2-10-1 Okura Setagaya Tokyo 157-8535 
TEL +81-3-3416-0181  FAX +81-3-3416-2222 
E-mail fukuda-a@ncchd.go.jp 
 

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●Secondary IDs and Issuers
Secondary study ID Nill Known  Issuer name  
 

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●Clinical Trial Notification (CTN) Reference Number
First CTN reference number   Date of first CTN 2018-03-28 
 

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●Ethics Review
Approved by Ethics Committee YES 
Name of ethics committee Institutional review board of National Center for Child Health and Development 
Date of approved 2018-03-15 
Address  
TEL +81-3-3416-0181 
FAX +81-3-3416-2222 
E-mail  
Website  
Committee approval number  
Reason for unapproval  
 

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●Publication of Study Results
Are the results published? NO 
Posted date  
Date of first publication  
Baseline characteristics  
Participant flow  
Protocol links  
Overall study design - description  
Efficacy conclusions  
Safety conclusions  
Discussion and overall conclusions  
Results links  
 

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●Sharing of Individual clinical trial participant-level data(IPD)
Results IPD Plan  
Results IPD Description  
 

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●Other Information
Other Information  

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