UMIN-CTR Clinical Trial

Basic information
Public title	Epidemiological studies for clinical manifestations and case numbers of inborn errors of ketone body metabolism (HMG-CoA syntase deficiency, HMG-CoA lyase deficiency, beta-ketothiolase deficiency, SCOT deficiency)
Acronym	Epidemiological studies of inborn errors of ketone body metabolism
Scientific Title	Epidemiological studies for clinical manifestations and case numbers of inborn errors of ketone body metabolism (HMG-CoA syntase deficiency, HMG-CoA lyase deficiency, beta-ketothiolase deficiency, SCOT deficiency)
Scientific Title:Acronym	Epidemiological studies of inborn errors of ketone body metabolism
Region	Japan

Condition
Condition	HMG-CoA synthase deficiency, HMG-CoA lyase deficiency, Beta-ketothiolase deficiency, Succinyl-CoA:3-ketoacid CoA transferase deficiency
Classification by specialty	Pediatrics
Classification by malignancy	Others
Genomic information	NO

Objectives
Narrative objectives1	We will collect clinical information for definite and possible cases for these 4 disorders by questionnaire, will make guidelines for diagnosis and treatments, and will use these information for further study.
Basic objectives2	Others
Basic objectives -Others	Epidemiological purpose
Trial characteristics_1	Confirmatory
Trial characteristics_2	Explanatory
Developmental phase	Not applicable

Assessment
Primary outcomes	clinical data
Key secondary outcomes

Base
Study type	Observational

Study design
Basic design
Randomization
Randomization unit
Blinding
Control
Stratification
Dynamic allocation
Institution consideration
Blocking
Concealment

Intervention
No. of arms
Purpose of intervention
Type of intervention
Interventions/Control_1
Interventions/Control_2
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	Not applicable
Age-upper limit	Not applicable
Gender	Male and Female
Key inclusion criteria	definite diagnosed possibly diagnosed
Key exclusion criteria	obviously excluded
Target sample size	100

Research contact person
Name of lead principal investigator	1st name Middle name Last name Toshiyuki Fukao
Organization	Gifu University
Division name	Graduate School of Medicine
Zip code
Address	Yanagido 1-1, Gifu
TEL	058-230-6386
Email	toshi-gif@umin.net

Public contact
Name of contact person	1st name Middle name Last name Toshiyuki Fukao
Organization	Gifu University
Division name	Graduate School of Medicine
Zip code
Address	1-1 yanagido, Gifu
TEL	056-230-6380
Homepage URL
Email	toshi-gif@umin.net

Sponsor
Institute	department of Pediatrics, Graduate School of Medicine, Gifu University
Institute
Department

Funding Source
Organization	Health and Labor Science Research Grants for Research on Intractable Diseases from The Ministry of Health, Labor and Welfare of Japan
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	岐阜大学（岐阜県）

Other administrative information
Date of disclosure of the study information	2010 Year 06 Month 10 Day

Related information
URL releasing protocol
Publication of results	Published

Result
URL related to results and publications
Number of participants that the trial has enrolled
Results	We could not identified new patients with inborn errors of ketone body metabolism.
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	Completed
Date of protocol fixation	2010 Year 06 Month 04 Day
Date of IRB
Anticipated trial start date	2010 Year 06 Month 01 Day
Last follow-up date
Date of closure to data entry
Date trial data considered complete
Date analysis concluded

Other
Other related information	epidemiologic study

Management information
Registered date	2010 Year 06 Month 04 Day
Last modified on	2016 Year 08 Month 28 Day

Link to view the page
URL(English)	https://upload.umin.ac.jp/cgi-open-bin/ctr_e/ctr_view.cgi?recptno=R000004495

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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