UMIN-CTR Clinical Trial
| Unique ID issued by UMIN | UMIN000025772 |
|---|---|
| Receipt number | R000029645 |
| Scientific Title | Placebo controlled double blind randomized Phase II Clinical Trial of sodium pyruvate on lactic acidosis associated with mitochondrial disorders (MELAS/MELA) |
| Date of disclosure of the study information | 2017/01/23 |
| Last modified on | 2019/01/28 10:29:50 |
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Basic information
Public title
Placebo controlled double blind randomized Phase II Clinical Trial of sodium pyruvate on lactic acidosis associated with mitochondrial disorders (MELAS/MELA)
Acronym
Placebo controlled double blind randomized Phase II Clinical Trial of sodium pyruvate on lactic acidosis associated with mitochondrial disorders (MELAS/MELA)
Scientific Title
Placebo controlled double blind randomized Phase II Clinical Trial of sodium pyruvate on lactic acidosis associated with mitochondrial disorders (MELAS/MELA)
Scientific Title:Acronym
Placebo controlled double blind randomized Phase II Clinical Trial of sodium pyruvate on lactic acidosis associated with mitochondrial disorders (MELAS/MELA)
Region
| Japan |
Condition
Condition
MELAS/MELA
Classification by specialty
| Medicine in general | Neurology | Pediatrics |
Classification by malignancy
Others
Genomic information
YES
Objectives
Narrative objectives1
To evaluate efficacy and safety of sodium pyruvate in patient with lactic acidosis associated with mitochondrial disorders(MELAS/MELA) by Placebo controlled double blind randomized Phase II Clinical Trial.
Basic objectives2
Safety,Efficacy
Basic objectives -Others
Trial characteristics_1
Trial characteristics_2
Developmental phase
Assessment
Primary outcomes
JMDRS section 1 & 2 change at 24 weeks after the administration of sodium pyruvate
Key secondary outcomes
NMDAS section 1-3, brain lactate by MRS, GDF15 changes at 24 weeks after the administration of sodium pyruvate
Base
Study type
Interventional
Study design
Basic design
Parallel
Randomization
Randomized
Randomization unit
Individual
Blinding
Double blind -all involved are blinded
Control
Placebo
Stratification
YES
Dynamic allocation
YES
Institution consideration
Institution is not considered as adjustment factor.
Blocking
NO
Concealment
Central registration
Intervention
No. of arms
2
Purpose of intervention
Treatment
Type of intervention
| Medicine |
Interventions/Control_1
24 weeks of sodium pyruvate 0.5g/kg/day TID
Interventions/Control_2
24 weeks of Placebo (Lactose + NaCl) 0.17g/kg/day TID
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
| 6 | years-old | < |
Age-upper limit
| 70 | years-old | > |
Gender
Male and Female
Key inclusion criteria
Mitochondrial disease who fulfilled the following criteria
1) patient whoes age is over than 6 years-old
2) patient who are fulfilled the diagnostic criteria of MELAS/MELA
3) patientwho has an A3243G mutation.
4) no past history of sodium pyruvate administration for the therapeutic purpose
5) patient whose score of section 1+2 by JMDRS is more than 10, and one of the specific score is more than 2.
6) patient who have a written informed conscent or ascent by his/her own opinion.
Key exclusion criteria
1) patient who has bed ridden status
2) patient who has dementic status and cannot be evaluated the JMDRS/NMDAS by medical doctor
3) patient who has uncontrolled status epileptics
4) patient who has septic status
5) patient who has renal failre, and less than 60mL/min/1.73m by eGFR
6) patient who has hypertrophic cardiomyopathy or cardiac failre
7) patient who has allergic state against sodium pyruvate
8) patient who enrolled to the other clinical trial within 6 months
9) patient who has pregnant
10) patient who is not recommended to the entry by medical doctor
Target sample size
15
Research contact person
Name of lead principal investigator
| 1st name | |
| Middle name | |
| Last name | Yasutoshi Koga |
Organization
Kurume University
Division name
Department of Pediatrics and Child Health
Zip code
Address
67 Asahi-machi, Kurume, Fukuoka 830-0011, Japan
TEL
0942-31-7565
yasukoga@med.kurume-u.ac.jp
Public contact
Name of contact person
| 1st name | |
| Middle name | |
| Last name | Yasutoshi Koga |
Organization
Kurume University
Division name
Department of Pediatrics and Child Health
Zip code
Address
67 Asahi-machi, Kurume, Fukuoka 830-0011, Japan
TEL
0942-31-7565
Homepage URL
http://www.ped-kurume.com/
yasukoga@med.kurume-u.ac.jp
Sponsor or person
Institute
Japan Agency of Medical Research and Development
Institute
Department
Personal name
Funding Source
Organization
Japan Agency of Medical Research and Development
Organization
Division
Category of Funding Organization
Government offices of other countries
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
久留米大学病院（福岡県）、筑波大学病院（茨城県）、福井大学病院（福井県）、信州大学病院（長野県）
Other administrative information
Date of disclosure of the study information
| 2017 | Year | 01 | Month | 23 | Day |
Related information
URL releasing protocol
https://www.c-ctd.co.jp/project_PLA002/index.html
Publication of results
Unpublished
Result
URL related to results and publications
Number of participants that the trial has enrolled
Results
Unfortunately, data obtained by the clinical trials could not prove the efficacy overall, though there are several patients with perfect improvement by clinical rating scales. After the strategy consultation to PMDA on October 18, 2018, we decided to terminate the SP project.
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
| 2016 | Year | 03 | Month | 19 | Day |
Date of IRB
Anticipated trial start date
| 2016 | Year | 06 | Month | 01 | Day |
Last follow-up date
| 2017 | Year | 06 | Month | 12 | Day |
Date of closure to data entry
| 2017 | Year | 06 | Month | 18 | Day |
Date trial data considered complete
| 2017 | Year | 06 | Month | 20 | Day |
Date analysis concluded
| 2017 | Year | 11 | Month | 30 | Day |
Other
Other related information
Management information
Registered date
| 2017 | Year | 01 | Month | 20 | Day |
Last modified on
| 2019 | Year | 01 | Month | 28 | Day |
Link to view the page
Value
https://center6.umin.ac.jp/cgi-open-bin/ctr_e/ctr_view.cgi?recptno=R000029645
