Unique ID issued by UMIN | UMIN000025880 |
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Receipt number | R000029753 |
Scientific Title | Long term Phase 3 Open clinical trial of sodium pyruvate on lactic acidosis associated with mitochondrial disorders (Leigh syndrome) |
Date of disclosure of the study information | 2017/01/30 |
Last modified on | 2019/01/28 10:32:05 |
Long term Phase 3 Open clinical trial of sodium pyruvate on lactic acidosis associated with mitochondrial disorders (Leigh syndrome)
Long term Phase 3 Open clinical trial of sodium pyruvate on lactic acidosis associated with mitochondrial disorders (Leigh syndrome)
Long term Phase 3 Open clinical trial of sodium pyruvate on lactic acidosis associated with mitochondrial disorders (Leigh syndrome)
Long term Phase 3 Open clinical trial of sodium pyruvate on lactic acidosis associated with mitochondrial disorders (Leigh syndrome)
Japan |
Leigh disorders
Medicine in general | Neurology | Pediatrics |
Others
NO
To evaluate the long term (48W) efficacy and safety of sodium pyruvate on Leigh disorders
Safety,Efficacy
Confirmatory
Explanatory
Phase III
Safety
Total score of section 1 -3, 4 in NMDAS at 48 weeks after oral administration
Change of section 1-3,4 at 48 weeks after oral administration
Change of the score of section 1, 2, 3, 4 in NMDPS.
GDF15
emergency visit
lactate
lactate in proton MRS
mortality
period of in-patient during clinical trial
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 |
sodium pyruvate 0.5g/kg/day TID 48 weeks. We can use sodium pyruvate up to 2.0g/kg/day TID.
2 | years-old | < |
30 | years-old | > |
Male and Female
Leigh disease who fulfilled the following criteria
1)neurological findings (all matched)
progressive central neurological sings
symptoms suggesting bilateral brain stem abnormality
bilateral necrosis of brain stem by MRI
2)defined mitochondrial dysfunction (one of the following )
by repeated abnormality of serum lactate
by respiratory chain enzyme activity
by muscle pathology
by genetic abnormality
1) patients who is not evaluated by this protocol of the clinical trial
2)severe unconsciousness
3)sepsis
4)renal dysfunction by eGFR lower than 60 mL/min/1.73m2
5)heart failure (EF<40%)
6)status epileptics
7)pregnant
8)allergy against the sodium pyruvate
9)enrolled to the other clinical trial 24 weeks before this trial
10)patient who cannot evaluate the muscle activity by device
11)patients who are not recommended by the doctor
10
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)、千葉大学病院(千葉県)、千葉県立こども病院(千葉県)、名古屋市立大学病院(愛知県)、大阪府立母子保健総合医療センター(大阪府)、信州大学病院(長野県)
2017 | Year | 01 | Month | 30 | Day |
https://www.c-ctd.co.jp/project_PLA003/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
2017 | Year | 01 | Month | 16 | Day |
2018 | Year | 02 | Month | 01 | Day |
2019 | Year | 02 | Month | 28 | 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=R000029753
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