UMIN-CTR Clinical Trial
| Unique ID issued by UMIN | UMIN000017152 |
|---|---|
| Receipt number | 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 17:26:11 |
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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
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
sakuraba@my-pharm.ac.jp
Public contact
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
tadayasu@my-pharm.ac.jp
Sponsor or person
Institute
Meiji Pharmaceutical University
Institute
Department
Personal name
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
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
Value
https://center6.umin.ac.jp/cgi-open-bin/ctr_e/ctr_view.cgi?recptno=R000019896
