UMIN-CTR Clinical Trial

Basic information
Official scientific title of the study	Japan Fabry Disease Registry and Cohort Study
Title of the study (Brief title)	J-FDR-CS
Region	Japan

Condition
Condition	Fabry disease
Classification by specialty	Medicine in general Nephrology Pediatrics
Classification by malignancy	Others
Genomic information	NO

Objectives
Narrative objectives1	To elucidate the effect of enzyme replacement therapy on renal prognosis and patient survival in Japanese patients with Fabry disease
Basic objectives2	Others
Basic objectives -Others	To establish the clinical parameters affecting therapy and prognosis in patients with Fabry disease
Trial characteristics_1
Trial characteristics_2
Developmental phase

Assessment
Primary outcomes	renal prognosis, end-stage renal disese, death (CVD, total)
Key secondary outcomes	proteinuria, enzyme replacement therapy

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 with Fabry disease (newly-identified or already-diagnosed)
Key exclusion criteria	not particular
Target sample size	200

Research contact person
Name of lead principal investigator	Ichiei Narita
Organization	Niigata University Graduate School of Medical and Dental Sciences
Division name	Biological Functions and Medical Control, Homeostatic Regulation and Development
Address	754, Ichibancho, Asahimachidori, Chuo-ku, Niigata-shi, NIIGATA, 951-8510,
TEL	03-3814-4455
Email	fab@n-place.co.jp

Public contact
Name of contact person	Hiroshi Morinaga
Organization	Japan Fabry Nephropathy Study Group
Division name	Working Group for cohort study
Address	2-5-1 Shikata-cho, Kita-ku, Okayama, Japan
TEL	086-223-7151
Homepage URL
Email	fab@n-place.co.jp

Sponsor
Institute	Japan Fabry Nephropathy Study Group
Institute
Department

Funding Source
Organization	The Kidney Foundation, Japan
Organization
Division
Category of Funding Organization	Non profit foundation
Nationality of Funding Organization

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	2013 Year 01 Month 15 Day

Progress
Recruitment status	Completed
Date of protocol fixation	2012 Year 11 Month 24 Day
Anticipated trial start date	2013 Year 01 Month 15 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	Unpublished
URL releasing results
Results
Other related information	The data on patients with Fabry disease were recorded at each institution and registered on the web page of the J-FDR-CS utilizing the UMIN-INDICE system. Multicenter prospective observational study will be performed in annual survey for five years.

Management information
Registered date	2013 Year 01 Month 15 Day
Last modified on	2018 Year 06 Month 19 Day

Link to view the page
URL(English)	https://upload.umin.ac.jp/cgi-open-bin/icdr_e/ctr_view.cgi?recptno=R000011459