UMIN-CTR Clinical Trial
| Recruitment status | Terminated |
| Unique ID issued by UMIN | UMIN000008341 |
| Receipt No. | R000009761 |
| Scientific Title | Therapeutic application of sodium pyruvate to patients of severe mitochondrial disorders |
| Date of disclosure of the study information | 2012/07/05 |
| Last modified on | 2019/04/24 (Ver. 10) |
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| Basic information | ||
| Public title | Therapeutic application of sodium pyruvate to patients of severe mitochondrial disorders | |
| Acronym | Sodium pyruvate therapy for mitochondrial disorders | |
| Scientific Title | Therapeutic application of sodium pyruvate to patients of severe mitochondrial disorders | |
| Scientific Title:Acronym | Sodium pyruvate therapy for mitochondrial disorders | |
| Region |
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| Condition | ||
| Condition | mitochondrial disorders | |
| Classification by specialty |
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| Classification by malignancy | Others | |
| Genomic information | YES | |
| Objectives | |
| Narrative objectives1 | To evaluate and establish the efficacy of sodium pyruvate therapy to severe mitochondrial disorders for which no effective therapy with scientific evidences exists. |
| Basic objectives2 | Efficacy |
| Basic objectives -Others | |
| Trial characteristics_1 | Exploratory |
| Trial characteristics_2 | Pragmatic |
| Developmental phase | Phase II |
| Assessment | |
| Primary outcomes | Changes in scores of Mitochondrial Disease Adult Scale (NMDAS; for adults), or Newcastle Paediatric Mitochondrial Disease Scale (NPMDS; for children) before and after 6-month administration. |
| Key secondary outcomes | Reduction rate of lactate/pyruvate ratio in blood as effectiveness, and rate of adverse events as safety. |
| Base | |
| Study type | Interventional |
| Study design | |
| Basic design | Single arm |
| Randomization | Non-randomized |
| Randomization unit | |
| Blinding | Open -no one is blinded |
| Control | Uncontrolled |
| Stratification | |
| Dynamic allocation | |
| Institution consideration | |
| Blocking | |
| Concealment | |
| Intervention | ||
| No. of arms | 1 | |
| Purpose of intervention | Treatment | |
| Type of intervention |
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| Interventions/Control_1 | Oral administration of sodium pyruvate supplied as a reagent. | |
| Interventions/Control_2 | ||
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| Eligibility | ||||
| Age-lower limit |
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| Gender | Male and Female | |||
| Key inclusion criteria | 1) Patients who meet the criteria of mitochondrial disorders defined by Ministry of Health, Labour and Welfare of Japan. 2) Any inpatient, or outpatients who can visit at least once in two weeks, can participate. 3) Written informed consents are required in advance. | |||
| Key exclusion criteria | 1) Women who are pregnant, breastfeeding, or of possible pregnancy. 2) Patients who are judged to be inappropriate for any other reason by their doctor in charge. | |||
| Target sample size | 20 | |||
| Research contact person | |||||||
| Last name of lead principal investigator |
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| Organization | Hiroshima University Hospital | ||||||
| Division name | Pediatrics | ||||||
| Zip code | |||||||
| Address | 1-2-3 Kasumi, Minami-ku, Hiroshima | ||||||
| TEL | 082-257-5212 | ||||||
| ishikan@hiroshima-u.ac.jp | |||||||
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| 1st name of contact person |
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| Organization | Hiroshima University Hospital | ||||||
| Division name | Pediatrics | ||||||
| Zip code | |||||||
| Address | 1-2-3 Kasumi, Minami-ku, Hiroshima | ||||||
| TEL | 082-257-5212 | ||||||
| Homepage URL | http://home.hiroshima-u.ac.jp/syoni/hiroped/doctors/research.html | ||||||
| hiropedpyruvate@gmail.com | |||||||
| Sponsor | |
| Institute | Department of Pediatrics, Hiroshima University Hospital |
| Institute | |
| Department | |
| Funding Source | |
| Organization | Research grant from Ministry of Health, Labour and Welfare of Japan |
| Organization | |
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| Category of Funding Organization | |
| Nationality of Funding Organization | Japan |
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| Secondary IDs | |
| Secondary IDs | NO |
| Study ID_1 | |
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| IND to MHLW | |
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| Date of disclosure of the study information |
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| Related information | |
| URL releasing protocol | http://home.hiroshima-u.ac.jp/syoni/hiroped/doctors/research.html |
| Publication of results | Partially published |
| Result | |
| URL related to results and publications | |
| Number of participants that the trial has enrolled | |
| Results | Five patients were enrolled in the study. All had withdrawn by the end of 2014. |
| Results date posted | |
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| Date of the first journal publication of results | |
| Baseline Characteristics | |
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| Plan to share IPD | |
| IPD sharing Plan description | |
| Progress | |||||||
| Recruitment status | Terminated | ||||||
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| Other | |
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| Management information | |||||||
| Registered date |
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| Link to view the page | |
| URL(English) | https://upload.umin.ac.jp/cgi-open-bin/ctr_e/ctr_view.cgi?recptno=R000009761 |
