UMIN-CTR Clinical Trial
| Unique ID issued by UMIN | UMIN000008341 |
|---|---|
| Receipt number | R000009761 |
| Scientific Title | Therapeutic application of sodium pyruvate to patients of severe mitochondrial disorders |
| Date of disclosure of the study information | 2012/07/05 |
| Last modified on | 2019/04/24 15:28:48 |
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Basic information
Public title
Therapeutic application of sodium pyruvate to patients of severe mitochondrial disorders
Acronym
Sodium pyruvate therapy for mitochondrial disorders
Scientific Title
Therapeutic application of sodium pyruvate to patients of severe mitochondrial disorders
Scientific Title:Acronym
Sodium pyruvate therapy for mitochondrial disorders
Region
| Japan |
Condition
Condition
mitochondrial disorders
Classification by specialty
| Endocrinology and Metabolism |
Classification by malignancy
Others
Genomic information
YES
Objectives
Narrative objectives1
To evaluate and establish the efficacy of sodium pyruvate therapy to severe mitochondrial disorders for which no effective therapy with scientific evidences exists.
Basic objectives2
Efficacy
Basic objectives -Others
Trial characteristics_1
Exploratory
Trial characteristics_2
Pragmatic
Developmental phase
Phase II
Assessment
Primary outcomes
Changes in scores of Mitochondrial Disease Adult Scale (NMDAS; for adults), or Newcastle Paediatric Mitochondrial Disease Scale (NPMDS; for children) before and after 6-month administration.
Key secondary outcomes
Reduction rate of lactate/pyruvate ratio in blood as effectiveness, and rate of adverse events as safety.
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
| Other |
Interventions/Control_1
Oral administration of sodium pyruvate supplied as a reagent.
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) Patients who meet the criteria of mitochondrial disorders defined by Ministry of Health, Labour and Welfare of Japan. 2) Any inpatient, or outpatients who can visit at least once in two weeks, can participate. 3) Written informed consents are required in advance.
Key exclusion criteria
1) Women who are pregnant, breastfeeding, or of possible pregnancy. 2) Patients who are judged to be inappropriate for any other reason by their doctor in charge.
Target sample size
20
Research contact person
Name of lead principal investigator
| 1st name | |
| Middle name | |
| Last name | Nobutsune Ishikawa |
Organization
Hiroshima University Hospital
Division name
Pediatrics
Zip code
Address
1-2-3 Kasumi, Minami-ku, Hiroshima
TEL
082-257-5212
ishikan@hiroshima-u.ac.jp
Public contact
Name of contact person
| 1st name | |
| Middle name | |
| Last name | Nobutsune Ishikawa |
Organization
Hiroshima University Hospital
Division name
Pediatrics
Zip code
Address
1-2-3 Kasumi, Minami-ku, Hiroshima
TEL
082-257-5212
Homepage URL
http://home.hiroshima-u.ac.jp/syoni/hiroped/doctors/research.html
hiropedpyruvate@gmail.com
Sponsor or person
Institute
Department of Pediatrics, Hiroshima University Hospital
Institute
Department
Personal name
Funding Source
Organization
Research grant from Ministry of Health, Labour and Welfare of Japan
Organization
Division
Category of Funding Organization
Nationality of Funding Organization
Japan
Other related organizations
Co-sponsor
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
Other administrative information
Date of disclosure of the study information
| 2012 | Year | 07 | Month | 05 | Day |
Related information
URL releasing protocol
http://home.hiroshima-u.ac.jp/syoni/hiroped/doctors/research.html
Publication of results
Partially published
Result
URL related to results and publications
Number of participants that the trial has enrolled
Results
Five patients were enrolled in the study.
All had withdrawn by the end of 2014.
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
Terminated
Date of protocol fixation
| 2012 | Year | 06 | Month | 27 | Day |
Date of IRB
| 2012 | Year | 07 | Month | 02 | Day |
Anticipated trial start date
| 2012 | Year | 07 | Month | 02 | Day |
Last follow-up date
| 2022 | Year | 03 | Month | 31 | Day |
Date of closure to data entry
Date trial data considered complete
Date analysis concluded
Other
Other related information
Management information
Registered date
| 2012 | Year | 07 | Month | 04 | Day |
Last modified on
| 2019 | Year | 04 | Month | 24 | Day |
Link to view the page
Value
https://center6.umin.ac.jp/cgi-open-bin/ctr_e/ctr_view.cgi?recptno=R000009761
| Research Plan | |
|---|---|
| Registered date | File name |
| Research case data specifications | |
|---|---|
| Registered date | File name |
| Research case data | |
|---|---|
| Registered date | File name |
