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UMIN ID:

Recruitment status Terminated
Unique ID issued by UMIN UMIN000025874
Receipt No. R000029752
Scientific Title Phase 3 Open Clinical Trial of sodium pyruvate on lactic acidosis associated with mitochondrial disorders (MELAS/MELA)
Date of disclosure of the study information 2017/01/30
Last modified on 2019/01/28

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Basic information
Public title Phase 3 Open Clinical Trial of sodium pyruvate on lactic acidosis associated with mitochondrial disorders (MELAS/MELA)
Acronym Phase 3 Open Clinical Trial of sodium pyruvate on lactic acidosis associated with mitochondrial disorders (MELAS/MELA)
Scientific Title Phase 3 Open Clinical Trial of sodium pyruvate on lactic acidosis associated with mitochondrial disorders (MELAS/MELA)
Scientific Title:Acronym Phase 3 Open 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 Phase II Open Clinical Trial for 48 weeks.
Basic objectives2 Safety,Efficacy
Basic objectives -Others
Trial characteristics_1
Trial characteristics_2
Developmental phase

Assessment
Primary outcomes Safet
JMDRS section 1 & 2 change at 48 weeks after the administration of sodium pyruvate
JMDRS section 1-3 at 48 weeks after the administration of sodium pyruvate
NMDAS/NMDPS section 1-3, change.
lactate, pyruvate,
Lactate in the CNS by proton MRS
GDF15
6 min walk,

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 NO
Dynamic allocation NO
Institution consideration Institution is not considered as adjustment factor.
Blocking NO
Concealment No need to know

Intervention
No. of arms 1
Purpose of intervention Treatment
Type of intervention
Medicine
Interventions/Control_1 48 weeks of sodium pyruvate 0.5g/kg/day TID. We can administered sodium pyruvate for up to 2.0g/kg/day TID.
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
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 18

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
Email 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/
Email yasukoga@med.kurume-u.ac.jp

Sponsor
Institute Japan Agency of Medical Research and Development
Institute
Department

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
Email

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 30 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 12 Month 12 Day
Date of IRB
Anticipated trial start date
2017 Year 04 Month 01 Day
Last follow-up date
2018 Year 05 Month 31 Day
Date of closure to data entry
2018 Year 06 Month 15 Day
Date trial data considered complete
2018 Year 06 Month 30 Day
Date analysis concluded
2018 Year 09 Month 30 Day

Other
Other related information

Management information
Registered date
2017 Year 01 Month 27 Day
Last modified on
2019 Year 01 Month 28 Day


Link to view the page
URL(English) https://upload.umin.ac.jp/cgi-open-bin/ctr_e/ctr_view.cgi?recptno=R000029752

Research Plan
Registered date File name

Research case data specifications
Registered date File name

Research case data
Registered date File name


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