UMIN-CTR Clinical Trial

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

Fabry disease

Classification by specialty

Medicine in general	Gastroenterology	Cardiology
Endocrinology and Metabolism	Nephrology	Neurology
Ophthalmology	Dermatology

Classification by malignancy

Others

Genomic information

YES

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

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

Study type

Observational

Basic design

Randomization

Randomization unit

Blinding

Control

Stratification

Dynamic allocation

Institution consideration

Blocking

Concealment

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

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

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

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

Institute

Meiji Pharmaceutical University

Institute

Department

Personal name

Organization

Sumitomo Dainippon Pharma Co., Ltd.

Organization

Division

Category of Funding Organization

Profit organization

Nationality of Funding Organization

Japan

Co-sponsor

Name of secondary funder(s)

Organization

Address

Tel

Email

Secondary IDs

NO

Study ID_1

Org. issuing International ID_1

Study ID_2

Org. issuing International ID_2

IND to MHLW

Institutions

Date of disclosure of the study information

2015

Year

04

Month

17

Day

URL releasing protocol

Publication of results

Published

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

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 related information

Retrospective observational study

Registered date

2015

Year

04

Month

16

Day

Last modified on

2018

Year

02

Month

22

Day

Link to view the page

Value
https://center6.umin.ac.jp/cgi-open-bin/ctr_e/ctr_view.cgi?recptno=R000019896