UMIN-CTR Clinical Trial
| Unique ID issued by UMIN | UMIN000025872 |
|---|---|
| Receipt number | 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 10:28:09 |
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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
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/
yasukoga@med.kurume-u.ac.jp
Sponsor or person
Institute
Japan Agency of Medical Research and Development
Institute
Department
Personal name
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
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
Value
https://center6.umin.ac.jp/cgi-open-bin/ctr_e/ctr_view.cgi?recptno=R000029639
