UMIN-CTR Clinical Trial

Basic information
Official scientific title of the study	Retrospective study of lyso-Gb3 plasma concentration in Fabry patients receiving ERT/Replagal
Title of the study (Brief title)	Retrospective study of lyso-Gb3 plasma concentration in Fabry patients receiving ERT/Replagal
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	1) To investigate the lyso-Gb3 plasma concentration in Japanese patients with Fabry disease and to investigate the relation between the concentration and GLA activity in plasma 2) To evaluate the usefulness of lyso-Gb3 as a therapeutic bio-marker by measuring change in lyso-Gb3 plasma concentration in patients receiving Replagal
Basic objectives2	Others
Basic objectives -Others	To evaluate the usefulness of lyso-Gb3 as a bio-marker
Trial characteristics_1
Trial characteristics_2
Developmental phase

Assessment
Primary outcomes	As a diagnostic bio-marker; 1) Plasma concentration of lyso-Gb3 2) Plasma concentration of lyso-Gb3 analogs (lyso-Gb3 [-2], lyso-Gb3 [+34] and other analogs) 3) GLA activity in plasma As a therapeutic bio-marker;; 1) Plasma concentration of lyso-Gb3 and the change from baseline in the concentration after initiation of the treatment with Replagal 2) Plasma concentration of lyso-Gb3 analogs (lyso-Gb3 [-2], lyso-Gb3 [+34]) and other analogs) after initiation of the treatment with Replagal, and the change from baseline in the analog concentration after initiation of the treatment with Replagal
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	Patients should meet all the following criteria; 1) Patients with Fabry disease who participated in the Replagal Post Marketing Surveillance and have no history of any enzyme replacement therapies before the treatment with Replagal 2) Patients whose stored blood sample taken before the treatment with Replagal was at least 0.3 mL 3) Patients whose any stored blood samples taken after initiation of the treatment with Replagal were at least 0.2 mL 4) Patients 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 patients aged 15 years or older and younger than 20 years at informed consent, consent should be obtained from the patients'legal acceptable representatives in addition to the consent from the patients themselves. For patients younger than 15 years at informed consent, consent should be obtained from the patients'legal acceptable representatives in addition to the assent from the patients themselves.
Key exclusion criteria	The following patients should not be enrolled to the study; Patients who are 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, 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, 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

Other administrative information
Date of disclosure of the study information	2015 Year 04 Month 17 Day

Progress
Recruitment status	Completed
Date of protocol fixation	2015 Year 02 Month 25 Day
Anticipated trial start date	2015 Year 04 Month 17 Day
Last follow-up date	2016 Year 06 Month 30 Day
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	Retrospective observational study

Management information
Registered date	2015 Year 04 Month 16 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=R000019896