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Name:
UMIN ID:

Recruitment status Completed
Unique ID issued by UMIN UMIN000017152
Receipt No. R000019896
Scientific Title Retrospective study of lyso-Gb3 plasma concentration in Fabry patients receiving ERT/Replagal
Date of disclosure of the study information 2015/04/17
Last modified on 2018/02/22

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Basic information
Public title Retrospective study of lyso-Gb3 plasma concentration in Fabry patients receiving ERT/Replagal
Acronym Retrospective study of lyso-Gb3 plasma concentration in Fabry patients receiving ERT/Replagal
Scientific Title Retrospective study of lyso-Gb3 plasma concentration in Fabry patients receiving ERT/Replagal
Scientific Title:Acronym 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
Last name of lead principal investigator
1st name
Middle name
Last name Hitoshi Sakuraba
Organization Meiji Pharmaceutical University
Division name Department of Clinical Genetics
Zip code
Address 2-522-1 Noshio,Kiyose, Tokyo 204-8588,Japan
TEL 042-495-8923
Email sakuraba@my-pharm.ac.jp

Public contact
1st name of contact person
1st name
Middle name
Last name Tadayasu Togawa
Organization Meiji Pharmaceutical University
Division name Department of Functional Bioanalysis
Zip code
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)

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

Other administrative information
Date of disclosure of the study information
2015 Year 04 Month 17 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
Clin Exp Nephrol.  doi: 10.1007/s10157-017-1525-3.(open access)
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
2015 Year 02 Month 25 Day
Date of IRB
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

Other
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

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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