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UMIN ID:

Recruitment status Terminated
Unique ID issued by UMIN UMIN000025872
Receipt No. R000029639
Scientific Title Placebo controlled double blind randomized Phase 2 clinical trial of sodium pyruvate on lactic acidosis associated with mitochondrial disorders (Leigh syndrome)
Date of disclosure of the study information 2017/02/01
Last modified on 2019/01/28

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Basic information
Public title Placebo controlled double blind randomized Phase 2 clinical trial of sodium pyruvate on lactic acidosis associated with mitochondrial disorders (Leigh syndrome)
Acronym Placebo controlled double blind randomized Phase 2 clinical trial of sodium pyruvate on lactic acidosis associated with mitochondrial disorders (Leigh syndrome)
Scientific Title Placebo controlled double blind randomized Phase 2 clinical trial of sodium pyruvate on lactic acidosis associated with mitochondrial disorders (Leigh syndrome)
Scientific Title:Acronym 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
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 Japanese Governmental office
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)、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

Related information
URL releasing protocol https://www.c-ctd.co.jp/project_PLA003/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 11 Month 22 Day
Date of IRB
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

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=R000029639

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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