UMIN-CTR Clinical Trial

Basic information
Official scientific title of the study	Validation study for lyso-Gb3 plasma concentration in healthy adult or child Japanese volunteers
Title of the study (Brief title)	Validation study of lyso-Gb3 plasma concentration in healthy Japanese volunteers
Region	Japan

Condition
Condition	Fabry disease
Classification by specialty	Medicine in general Gastroenterology Cardiology Endocrinology and Metabolism Nephrology Neurology Ophthalmology Dermatology
Classification by malignancy	Others
Genomic information	YES

Objectives
Narrative objectives1	To investigate the reference range of plasma Lyso-Gb3 in healthy adult or child Japanese volunteers
Basic objectives2	Others
Basic objectives -Others	To investigate the reference range of plasma Lyso-Gb3 for the diagnosis of Fabry disease
Trial characteristics_1	Others
Trial characteristics_2	Others
Developmental phase	Not applicable

Assessment
Primary outcomes	Lyso-Gb3 concentration in plasma
Key secondary outcomes	GLA activity in plasma

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	15 years-old <=
Age-upper limit	70 years-old >
Gender	Male and Female
Key inclusion criteria	*Subjects who fulfill the following 1)-3) criteria 1) Japanese men or women aged 15 through 70 year at informed consent 2) Volunteers who are fully informed of and understand the objectives, procedures, and possible risks of the study and provide the written voluntary consent to participate in the study. For volunteers younger than 20 years at informed consent, Consent should be obtained from the volunteers' legal acceptable representatives in addition to the consent from the volunteers. 3) Volunteers who can claim their symptoms
Key exclusion criteria	1)Volunteers who were diagnosed or suspected as Fabry disease 2)Volunteers with a history of receiving GLA enzyme replacement therapy (Replagal&reg; or Fabrazyme&reg;) 3)Volunteers who have previous or current disease or symptoms shown in Table 3 4)Volunteers who any their relatives (parents, brothers, sisters, sons, daughters, grandparents, or cousins) have Fabry disease 5)Volunteers who received amiodarone, chloroquine or hydroxychloroquine within 6 months before informed consent or plan to receive either of the drugs during the study 6)Volunteers who are considered unhealthy (having clinical issues for which any treatments or clinical observation are necessary) by the investigator at screening 7)Volunteers who have any histories of cardiovascular, liver, kidney, lung, endocrine, digestive, blood, respiratory, neuropsychiatric, or central nervous system diseases and are considered ineligible for the study by the investigator. 8)Volunteers who are otherwise considered ineligible for the study by the investigator
Target sample size	100

Research contact person
Name of lead principal investigator	Hitoshi Sakuraba
Organization	Meiji Pharmaceutical University
Division name	Department of Clinical Genetics
Address	2-522-1 Noshio,Kiyose City Tokyo 204-8588,Japan
TEL	042-495-8923
Email	sakuraba@my-pharm.ac.jp

Public contact
Name of contact person	Tadayasu Togawa
Organization	Meiji Pharmaceutical University
Division name	Department of Functional Bioanalysis
Address	2-522-1 Noshio,Kiyose City Tokyo 204-8588,Japan
TEL	042-495-8992
Homepage URL
Email	tadayasu@my-pharm.ac.jp

Sponsor
Institute	Meiji Pharmaceutical University
Institute
Department

Funding Source
Organization	Sumitomo Dainippon Pharma Co., Ltd.
Organization
Division
Category of Funding Organization	Profit organization
Nationality of Funding Organization	Japan

Other related organizations
Co-sponsor
Name of secondary funｄer(s)

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	シーピーシー治験病院（鹿児島県）/　CPC Clinical Trial Hospital (KAGOSHIMA)

Other administrative information
Date of disclosure of the study information	2015 Year 03 Month 20 Day

Progress
Recruitment status	Completed
Date of protocol fixation	2015 Year 03 Month 17 Day
Anticipated trial start date	2015 Year 03 Month 25 Day
Last follow-up date
Date of closure to data entry
Date trial data considered complete
Date analysis concluded

Related information
URL releasing protocol
Publication of results	Published
URL releasing results
Results	Clin Exp Nephrol. doi: 10.1007/s10157-017-1525-3.(open access)
Other related information	Prospective observational study

Management information
Registered date	2015 Year 03 Month 20 Day
Last modified on	2018 Year 02 Month 22 Day

Link to view the page
URL(English)	https://upload.umin.ac.jp/cgi-open-bin/ctr_e/ctr_view.cgi?recptno=R000019564