UMIN-CTR Clinical Trial
| Unique ID issued by UMIN | UMIN000037839 |
|---|---|
| Receipt number | R000043042 |
| Scientific Title | A study of usefulness of plasma lyso-GL-3 as a biomarker in patients with Fabry disease |
| Date of disclosure of the study information | 2019/09/01 |
| Last modified on | 2019/12/30 07:25:52 |
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Basic information
Public title
A study of usefulness of plasma lyso-GL-3 as a biomarker in patients with Fabry disease
Acronym
Plasma lyso-GL-3 in patients with Fabry disease
Scientific Title
A study of usefulness of plasma lyso-GL-3 as a biomarker in patients with Fabry disease
Scientific Title:Acronym
Plasma lyso-GL-3 in patients with Fabry disease
Region
| Japan |
Condition
Condition
Fabry disease
Classification by specialty
| Cardiology | Nephrology | Pediatrics |
| Laboratory medicine |
Classification by malignancy
Others
Genomic information
NO
Objectives
Narrative objectives1
The aim of the study is to confirm the usefulness of plasma lyso-GL-3 as an effective bio-marker for Fabry disease patients not only for diagnosis but also to confirm their clinical conditions by comparing collected data of the value and its change in Japanese Fabry disease patients between plasma lyso-GL-3, plasma GL-3 which is already used for diagnosis of male Fabry disease patients, and tests values and change of renal function and heart function by which clinical symptoms are observed in Fabry disease patients.
Basic objectives2
Others
Basic objectives -Others
The usefulness of plasma lyso-GL-3 as a bio-marker for condition management in patients with Fabry disease.
Trial characteristics_1
Trial characteristics_2
Developmental phase
Assessment
Primary outcomes
To investigate correlation between plasma lyso-GL-3 and clinical features as follows; Plasma GL-3, eGFR, Urinary protein-to-creatinine ratio, BNP, LV mass index, EF, Presence of cardiac hypertrophy, Urinary mulberry body
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
1) Patients who were diagnosed as Fabry disease and meet the following criteria.
Male
-Male patients who can be diagnosed as having Fabry disease by their enzyme activities.
Female
-Female patients who can be diagnosed by genetic test or genetically confirmed (family history) as having Fabry disease. And, can be diagnosed as Fabry disease by decrease of enzyme activities or clinical presentation.
2) Patients who are receiving, or scheduled to receive enzyme replacement therapy with agalsidase beta BS.
3) Patients who are fully informed of and understand about the study and provide the written consent to participate in the study by themselves or their representatives. (For minor patients aged 16 years or older at informed consent, consent generally should be obtained from themselves.)
Key exclusion criteria
1) Patients who received before, are receiving now or are scheduled to receive Hematopoietic Stem Cell Transplantation / Chaperone therapy.
2) Patients who are considered as ineligible for the study by the investigator.
Target sample size
100
Research contact person
Name of lead principal investigator
| 1st name | Norio |
| Middle name | |
| Last name | Sakai |
Organization
Osaka University Graduate School of Medicine, Division of Health Science
Division name
Child Healthcare and Genetic Science Laboratory
Zip code
565-0871
Address
1-7 Yamadaoka, Suita, Osaka
TEL
06-6879-2531
norio@sahs.med.osaka-u.ac.jp
Public contact
Name of contact person
| 1st name | Norio |
| Middle name | |
| Last name | Sakai |
Organization
Osaka University Graduate School of Medicine, Division of Health Science
Division name
Child Healthcare and Genetic Science Laboratory
Zip code
565-0871
Address
1-7 Yamadaoka, Suita, Osaka
TEL
06-6879-2531
Homepage URL
norio@sahs.med.osaka-u.ac.jp
Sponsor or person
Institute
Osaka University Graduate School of Medicine, Division of Health Science, Child Healthcare and Genetic Science Laboratory
Institute
Department
Personal name
Funding Source
Organization
JCR Pharmaceuticals Co., Ltd.
Organization
Division
Category of Funding Organization
Profit organization
Nationality of Funding Organization
Other related organizations
Co-sponsor
Translational Research Center for Medical Innovation, Foundation for Biomedical Research and Innovation at Kobe
Name of secondary funder(s)
IRB Contact (For public release)
Organization
Osaka University Clinical Research Review Committee
Address
2-2 Yamadaoka, Suita, Osaka, Japan
Tel
06-6210-8296
rinri@hp-crc.med.osaka-u.ac.jp
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
国立大学法人大阪大学医学部附属病院
国立大学法人岐阜大学医学部附属病院
大阪市立大学医学部附属病院
独立行政法人国立病院機構関門医療センター
埼玉医科大学病院
Other administrative information
Date of disclosure of the study information
| 2019 | Year | 09 | Month | 01 | Day |
Related information
URL releasing protocol
Publication of results
Unpublished
Result
URL related to results and publications
Number of participants that the trial has enrolled
Results
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
Open public recruiting
Date of protocol fixation
| 2019 | Year | 05 | Month | 21 | Day |
Date of IRB
| 2019 | Year | 07 | Month | 31 | Day |
Anticipated trial start date
| 2019 | Year | 09 | Month | 01 | Day |
Last follow-up date
| 2024 | Year | 03 | Month | 31 | Day |
Date of closure to data entry
Date trial data considered complete
Date analysis concluded
Other
Other related information
Publication of results:
Unpublished, Partially published, Published
Management information
Registered date
| 2019 | Year | 08 | Month | 29 | Day |
Last modified on
| 2019 | Year | 12 | Month | 30 | Day |
Link to view the page
Value
https://center6.umin.ac.jp/cgi-open-bin/ctr_e/ctr_view.cgi?recptno=R000043042
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