Unique ID issued by UMIN | UMIN000025874 |
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Receipt number | 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 10:30:52 |
Phase 3 Open Clinical Trial of sodium pyruvate on lactic acidosis associated with mitochondrial disorders (MELAS/MELA)
Phase 3 Open Clinical Trial of sodium pyruvate on lactic acidosis associated with mitochondrial disorders (MELAS/MELA)
Phase 3 Open Clinical Trial of sodium pyruvate on lactic acidosis associated with mitochondrial disorders (MELAS/MELA)
Phase 3 Open Clinical Trial of sodium pyruvate on lactic acidosis associated with mitochondrial disorders (MELAS/MELA)
Japan |
MELAS/MELA
Medicine in general | Neurology | Pediatrics |
Others
YES
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.
Safety,Efficacy
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,
Interventional
Single arm
Non-randomized
Open -no one is blinded
Uncontrolled
NO
NO
Institution is not considered as adjustment factor.
NO
No need to know
1
Treatment
Medicine |
48 weeks of sodium pyruvate 0.5g/kg/day TID. We can administered sodium pyruvate for up to 2.0g/kg/day TID.
6 | years-old | < |
70 | years-old | > |
Male and Female
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.
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
18
1st name | |
Middle name | |
Last name | Yasutoshi Koga |
Kurume University
Department of Pediatrics and Child Health
67 Asahi-machi, Kurume, Fukuoka 830-0011, Japan
0942-31-7565
yasukoga@med.kurume-u.ac.jp
1st name | |
Middle name | |
Last name | Yasutoshi Koga |
Kurume University
Department of Pediatrics and Child Health
67 Asahi-machi, Kurume, Fukuoka 830-0011, Japan
0942-31-7565
http://www.ped-kurume.com/
yasukoga@med.kurume-u.ac.jp
Japan Agency of Medical Research and Development
Japan Agency of Medical Research and Development
Government offices of other countries
Japan
NO
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)
2017 | Year | 01 | Month | 30 | Day |
https://www.c-ctd.co.jp/project_PLA002/index.html
Unpublished
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.
Terminated
2016 | Year | 12 | Month | 12 | Day |
2017 | Year | 04 | Month | 01 | Day |
2018 | Year | 05 | Month | 31 | Day |
2018 | Year | 06 | Month | 15 | Day |
2018 | Year | 06 | Month | 30 | Day |
2018 | Year | 09 | Month | 30 | Day |
2017 | Year | 01 | Month | 27 | Day |
2019 | Year | 01 | Month | 28 | Day |
Value
https://center6.umin.ac.jp/cgi-open-bin/ctr_e/ctr_view.cgi?recptno=R000029752
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