UMIN-CTR Clinical Trial

Basic information
Official scientific title of the study	Placebo controlled double blind randomized Phase 2 clinical trial of sodium pyruvate on lactic acidosis associated with mitochondrial disorders (Leigh syndrome)
Title of the study (Brief title)	Placebo controlled double blind randomized Phase 2 clinical trial of sodium pyruvate on lactic acidosis associated with mitochondrial disorders (Leigh syndrome)
Region	Japan

Condition
Condition	Leigh disorders
Classification by specialty	Medicine in general Pediatrics
Classification by malignancy	Others
Genomic information	NO

Objectives
Narrative objectives1	To evaluate the efficacy and safety of sodium pyruvate on Leigh disorders
Basic objectives2	Safety,Efficacy
Basic objectives -Others
Trial characteristics_1	Exploratory
Trial characteristics_2
Developmental phase	Phase II

Assessment
Primary outcomes	Total score of section 1-3 in NMDPS at 24 weeks after oral administration
Key secondary outcomes	Total score of NMDAS section 1-3, Score of section 1, 2, 3, 4. brain lactate by MRS, GDF15 at 24 weeks after oral administration. Duration of in-patient, Emergency visit, mortality

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	YES
Concealment	Central registration

Intervention
No. of arms	2
Purpose of intervention	Treatment
Type of intervention	Medicine
Interventions/Control_1	sodium pyruvate 0.5g/kg/day TID 24 weeks
Interventions/Control_2	Placebo (lactate+NaCl) 0.17/kg/day TID 24 weeks
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	2 years-old <=
Age-upper limit	30 years-old >
Gender	Male and Female
Key inclusion criteria	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
Key exclusion criteria	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
Target sample size	10

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	Japanese Governmental office
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）、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）

Other administrative information
Date of disclosure of the study information	2017 Year 02 Month 01 Day

Progress
Recruitment status	Terminated
Date of protocol fixation	2016 Year 11 Month 22 Day
Anticipated trial start date	2017 Year 02 Month 01 Day
Last follow-up date	2017 Year 11 Month 30 Day
Date of closure to data entry	2017 Year 12 Month 15 Day
Date trial data considered complete	2017 Year 12 Month 27 Day
Date analysis concluded	2018 Year 03 Month 31 Day

Related information
URL releasing protocol	https://www.c-ctd.co.jp/project_PLA003/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 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=R000029639