UMIN-CTR Clinical Trial
| Unique ID issued by UMIN | UMIN000036465 |
|---|---|
| Receipt number | R000041554 |
| Scientific Title | Evaluation of effectiveness of plasma lyso-Gb3 analogues as biomarker for diagnosing of Fabry disease |
| Date of disclosure of the study information | 2019/04/11 |
| Last modified on | 2021/12/07 14:42:49 |
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Basic information
Public title
Evaluation of effectiveness of plasma lyso-Gb3 analogues as biomarker for diagnosing of Fabry disease
Acronym
Plasma lyso-Gb3 analogues measurement for diagnosis of Fabry disease
Scientific Title
Evaluation of effectiveness of plasma lyso-Gb3 analogues as biomarker for diagnosing of Fabry disease
Scientific Title:Acronym
Evaluation of effectiveness of plasma lyso-Gb3 analogues as biomarker for diagnosing of Fabry disease
Region
| Japan |
Condition
Condition
Fabry disease
Classification by specialty
| Medicine in general | Gastroenterology | Cardiology |
| Endocrinology and Metabolism | Nephrology | Neurology |
| Pediatrics | Ophthalmology | Dermatology |
Classification by malignancy
Others
Genomic information
YES
Objectives
Narrative objectives1
The plasma lyso-Gb3 is effective as a biomarker for screening high-risk patients with Fabry disease. However, heterozygote and late-onset Fabry disease remain difficult to be diagnosed due to its lower plasma lyso-Gb3. The plasma lyso-Gb3 and clinical symptoms are not always correlated. Plasma lyso-Gb3 analogues were discovered in Fabry disease patients, but it is still not clear whether there is a correlation among the type of analogues, genotype, phenotype and classification (classic or late-onset). The hypothesis is that plasma lyso-Gb3 analogues [total concentrations and pattern (ratio of each analogue to total concentrations)] rather than lyso-Gb3 alone may be effective biomarker for diagnosing of Fabry disease. We investigate plasma lyso-Gb3 analogues concentration, plasma GLA activity, GLA genotype, and clinical features in patients suspected of having Fabry disease and investigate the relation among these parameters.
Basic objectives2
Others
Basic objectives -Others
Evaluation of the usefulness of plasma lyso-Gb3 analogues as diagnosing biomarker for Fabry disease
Trial characteristics_1
Trial characteristics_2
Developmental phase
Assessment
Primary outcomes
Plasma lyso-Gb3 concentration
Plasma lyso-Gb3 analogues [lyso-Gb3(-28), lyso-Gb3(-2), lyso-Gb3(+16), lyso-Gb3(+18), lyso-Gb3(+34), lyso-Gb3(+50)] concentration
Plasma GLA activity
GLA analysis
Clinical features
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 suspected of having classic Fabry disease: Patients with classic early manifestations, such as acroparesthesia, clustered angiokeratoma, and cornea verticillata
2 Patients suspected of having late-onset Fabry disease: Patients suspected of having Fabry disease by possessing pathologic findings consistent with Fabry disease in kidney biopsy, endomyocardial biopsy or biopsy of other organs
3 Relatives of Fabry disease patients
4 The patients by whom a mulberry body or cell is detected in the urine
Key exclusion criteria
Patients have pathogenic GLA mutation and already diagnosed as Fabry disease should be excluded.
Target sample size
100
Research contact person
Name of lead principal investigator
| 1st name | Hiroki |
| Middle name | |
| Last name | Maruyama |
Organization
Niigata University Graduate School of Medical and Dental Sciences
Division name
Department of Clinical Nephroscience
Zip code
951-8120
Address
1-757 Asahimachi-dori, Chuo-ku, Niigata, Niigata
TEL
025-227-0436
hirokim@med.niigata-u.ac.jp
Public contact
Name of contact person
| 1st name | Hiroki |
| Middle name | |
| Last name | Maruyama |
Organization
Niigata University Graduate School of Medical and Dental Sciences
Division name
Department of Clinical Nephroscience
Zip code
951-8120
Address
1-757 Asahimachi-dori, Chuo-ku, Niigata, Niigata
TEL
025-227-0436
Homepage URL
hirokim@med.niigata-u.ac.jp
Sponsor or person
Institute
Department of Clinical Nephroscience, Niigata University Graduate School of Medical and Dental Sciences
Institute
Department
Personal name
Funding Source
Organization
Amicus Therapeutics
Organization
Division
Category of Funding Organization
Profit organization
Nationality of Funding Organization
Japan
Other related organizations
Co-sponsor
Name of secondary funder(s)
Japan Medical Supply Co.,Ltd.
Terumo Corp.
IRB Contact (For public release)
Organization
Ethics Committee on Genetic Analysis of Niigata University
Address
1-757 Asahimachi-dori, Chuo-ku, Niigata, Niigata 951-8120
Tel
025-227-2625
ethics@adm.niigata-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
Other administrative information
Date of disclosure of the study information
| 2019 | Year | 04 | Month | 11 | 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
Completed
Date of protocol fixation
| 2019 | Year | 03 | Month | 27 | Day |
Date of IRB
| 2019 | Year | 03 | Month | 27 | Day |
Anticipated trial start date
| 2019 | Year | 04 | Month | 15 | Day |
Last follow-up date
| 2021 | Year | 12 | Month | 31 | Day |
Date of closure to data entry
| 2021 | Year | 12 | Month | 31 | Day |
Date trial data considered complete
| 2021 | Year | 12 | Month | 31 | Day |
Date analysis concluded
| 2021 | Year | 12 | Month | 31 | Day |
Other
Other related information
Prospective multicenter study
Management information
Registered date
| 2019 | Year | 04 | Month | 10 | Day |
Last modified on
| 2021 | Year | 12 | Month | 07 | Day |
Link to view the page
Value
https://center6.umin.ac.jp/cgi-open-bin/ctr_e/ctr_view.cgi?recptno=R000041554
