UMIN-CTR Clinical Trial
| Unique ID issued by UMIN | UMIN000004290 |
|---|---|
| Receipt number | R000005141 |
| Scientific Title | sodium pyruvate therapy for the patients with citrin deficiency |
| Date of disclosure of the study information | 2010/10/01 |
| Last modified on | 2010/09/29 16:46:05 |
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Basic information
Public title
sodium pyruvate therapy for the patients with citrin deficiency
Acronym
pyruvate therapy for citrin deficiency
Scientific Title
sodium pyruvate therapy for the patients with citrin deficiency
Scientific Title:Acronym
pyruvate therapy for citrin deficiency
Region
| Japan |
Condition
Condition
citrin deficiency
Classification by specialty
| Hepato-biliary-pancreatic medicine | Neurology | Pediatrics |
Classification by malignancy
Others
Genomic information
YES
Objectives
Narrative objectives1
We examine effects of sodium pyruvate on neonatal intrahepatic cholestasis caused by citrin deficiency (NICCD), easy fatigability, growth disturbance, and hypoglycemia in the compensation period, and adult onset type II citrullinemia (CTLN2) in citrin deficiency.
Basic objectives2
Efficacy
Basic objectives -Others
Trial characteristics_1
Trial characteristics_2
Developmental phase
Assessment
Primary outcomes
Adult onset type II citrullinemia (16 weeks later):
1) improvement of citrullinemia and hyperannmonemia
2) improvement of impaired consciousness.
3) improvement of QOL.
Compensation period (16 weeks later):
1) improvement of citrullinemia and oxidative stress
2) improvement of QOL,
3) improvement of appetite habit.
Neonatal intrahepatic cholestasis caused by citrin deficiency:
1) improvement of cholestasis and citrullinemia
2) catch up of body weight
Key secondary outcomes
Base
Study type
Interventional
Study design
Basic design
Single arm
Randomization
Non-randomized
Randomization unit
Blinding
Open -no one is blinded
Control
Uncontrolled
Stratification
Dynamic allocation
Institution consideration
Blocking
Concealment
Intervention
No. of arms
1
Purpose of intervention
Treatment
Type of intervention
| Medicine |
Interventions/Control_1
1) Medicine: sodium pyruvate
2) Duration: 16 weeks, If the patient hopes, it can continue.
3) Amount: 100 - 300 mg/kg/day
4) Number: 2-3 times/day after eating orally
Interventions/Control_2
Interventions/Control_3
Interventions/Control_4
Interventions/Control_5
Interventions/Control_6
Interventions/Control_7
Interventions/Control_8
Interventions/Control_9
Interventions/Control_10
Eligibility
Age-lower limit
| Not applicable |
Age-upper limit
| Not applicable |
Gender
Male and Female
Key inclusion criteria
1) the patients with adult onset type II citrullinemia.
2) the patients with the symptoms of easy fatigability, growth retardation, and hypoglycemia in the compensation period.
3)the patients with severe hepatic damage by NICCD.
4)the patient that informed consent by the document was provided.
Key exclusion criteria
1) pregnant woman
Target sample size
30
Research contact person
Name of lead principal investigator
| 1st name | |
| Middle name | |
| Last name | Yoshiyuki Okano |
Organization
Osaka City University Graduate School of Medicine
Division name
Department of Pediatrics
Zip code
Address
1-4-3 Asahimachi, Abenoku, Osaka 545-8585
TEL
06-6645-3816
Public contact
Name of contact person
| 1st name | |
| Middle name | |
| Last name |
Organization
Osaka City University Graduate School of Medicine
Division name
Department of Pediatrics
Zip code
Address
TEL
06-6645-3816
Homepage URL
Sponsor or person
Institute
Working group for citrin deficiency
Institute
Department
Personal name
Funding Source
Organization
The Ministry of Health, Labor and Welfare of Japan
Organization
Division
Category of Funding Organization
Nationality of Funding Organization
Japan
Other related organizations
Co-sponsor
Tokushima Bunri University
Kagoshima University
Shinshu University
Tohoku University
Musashino Kagaku Co.
Name of secondary funder(s)
IRB Contact (For public release)
Organization
Address
Tel
Secondary IDs
Secondary IDs
NO
Study ID_1
Org. issuing International ID_1
Study ID_2
Org. issuing International ID_2
IND to MHLW
Institutions
Institutions
大阪市立大学病院（大阪） Osaka City University Hospital
東北大学病院（宮城） Tohoku University Hospital
済生会横浜市東部病院（神奈川） Yokohama Eastern Hospital
京都第一赤十字病院（京都） Japanese Red Cross Kyoto Daiichi Hospital
信州大学病院（長野） Shinshu University Hospital
Other administrative information
Date of disclosure of the study information
| 2010 | Year | 10 | Month | 01 | Day |
Related information
URL releasing protocol
Publication of results
Unpublished
Result
URL related to results and publications
Number of participants that the trial has enrolled
Results
Results date posted
Results Delayed
Results Delay Reason
Date of the first journal publication of results
Baseline Characteristics
Participant flow
Adverse events
Outcome measures
Plan to share IPD
IPD sharing Plan description
Progress
Recruitment status
Enrolling by invitation
Date of protocol fixation
| 2010 | Year | 07 | Month | 01 | Day |
Date of IRB
Anticipated trial start date
| 2010 | Year | 10 | Month | 01 | Day |
Last follow-up date
Date of closure to data entry
Date trial data considered complete
Date analysis concluded
Other
Other related information
Management information
Registered date
| 2010 | Year | 09 | Month | 29 | Day |
Last modified on
| 2010 | Year | 09 | Month | 29 | Day |
Link to view the page
Value
https://center6.umin.ac.jp/cgi-open-bin/ctr_e/ctr_view.cgi?recptno=R000005141
| Research Plan | |
|---|---|
| Registered date | File name |
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| Registered date | File name |
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