UMIN-CTR Clinical Trial

Unique ID issued by UMIN UMIN000025772
Receipt number R000029645
Scientific Title Placebo controlled double blind randomized Phase II Clinical Trial of sodium pyruvate on lactic acidosis associated with mitochondrial disorders (MELAS/MELA)
Date of disclosure of the study information 2017/01/23
Last modified on 2019/01/28 10:29:50

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Basic information

Public title

Placebo controlled double blind randomized Phase II Clinical Trial of sodium pyruvate on lactic acidosis associated with mitochondrial disorders (MELAS/MELA)

Acronym

Placebo controlled double blind randomized Phase II Clinical Trial of sodium pyruvate on lactic acidosis associated with mitochondrial disorders (MELAS/MELA)

Scientific Title

Placebo controlled double blind randomized Phase II Clinical Trial of sodium pyruvate on lactic acidosis associated with mitochondrial disorders (MELAS/MELA)

Scientific Title:Acronym

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

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 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

Government offices of other countries

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)、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


Related information

URL releasing protocol

https://www.c-ctd.co.jp/project_PLA002/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 03 Month 19 Day

Date of IRB


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


Other

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

Value
https://center6.umin.ac.jp/cgi-open-bin/ctr_e/ctr_view.cgi?recptno=R000029645


Research Plan
Registered date File name

Research case data specifications
Registered date File name

Research case data
Registered date File name