UMIN-CTR Clinical Trial

Basic information
Official scientific title of the study	Placebo controlled double blind randomized Phase II Clinical Trial of sodium pyruvate on lactic acidosis associated with mitochondrial disorders (MELAS/MELA)
Title of the study (Brief title)	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	Yasutoshi Koga
Organization	Kurume University
Division name	Department of Pediatrics and Child Health
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	Yasutoshi Koga
Organization	Kurume University
Division name	Department of Pediatrics and Child Health
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)

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

Progress
Recruitment status	Terminated
Date of protocol fixation	2016 Year 03 Month 19 Day
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

Related information
URL releasing protocol	https://www.c-ctd.co.jp/project_PLA002/index.html
Publication of results	Unpublished
URL releasing results
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.
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
URL(English)	https://upload.umin.ac.jp/cgi-open-bin/ctr_e/ctr_view.cgi?recptno=R000029645