UMIN-CTR Clinical Trial
| Unique ID issued by UMIN | UMIN000025321 |
|---|---|
| Receipt number | R000029125 |
| Scientific Title | The prospective cohort study of the enzyme replacement therapy in lysosomal storage disease. |
| Date of disclosure of the study information | 2016/12/19 |
| Last modified on | 2016/12/19 10:04:24 |
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Basic information
Public title
The prospective cohort study of the enzyme replacement therapy in lysosomal storage disease.
Acronym
Kanagawa Fabry Cohort Study
Scientific Title
The prospective cohort study of the enzyme replacement therapy in lysosomal storage disease.
Scientific Title:Acronym
Kanagawa Fabry Cohort Study
Region
| Japan |
Condition
Condition
Lysosomal storage disease
Classification by specialty
| Medicine in general |
Classification by malignancy
Others
Genomic information
NO
Objectives
Narrative objectives1
To clarify the effects of the enzyme replacement therapy on the lysosomal storage diseases.
Basic objectives2
Safety,Efficacy
Basic objectives -Others
Trial characteristics_1
Confirmatory
Trial characteristics_2
Explanatory
Developmental phase
Phase IV
Assessment
Primary outcomes
QOL( including SF-36)
Key secondary outcomes
renal function
state of cerebral lesion
heart function
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
The lysosomal storage disease patients who need to treat with enzyme replacement therapy.
Key exclusion criteria
none
Target sample size
30
Research contact person
Name of lead principal investigator
| 1st name | |
| Middle name | |
| Last name | Nobuhito Hirawa |
Organization
Yokohama City University Medical Center
Division name
Department of Hemodialysis and Aphaeresis/ Nephrology and Hypertension
Zip code
Address
4-57, Urafune-cho, Minami-ku, Yokohama
TEL
045-261-5656
hirawa@yokokohama-cu.ac.jp
Public contact
Name of contact person
| 1st name | |
| Middle name | |
| Last name | Nobuhito Hirawa |
Organization
Yokohama City University Medical Center
Division name
Department of Hemodialysis and Aphaeresis/ Nephrology and Hypertension
Zip code
Address
4-57, Urafune-cho, Minami-ku, Yokohama
TEL
045-261-5656
Homepage URL
hirawa@yokokohama-cu.ac.jp
Sponsor or person
Institute
Department of Hemodialysis and Aphaeresis/ Nephrology and Hypertension
Yokohama City University Medical Center
Institute
Department
Personal name
Funding Source
Organization
Yokohama City University Medical Center
Organization
Division
Category of Funding Organization
Other
Nationality of Funding Organization
Other related organizations
Co-sponsor
Kanazawa Society of Enzyme Replacement Therapy
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
| 2016 | Year | 12 | Month | 19 | Day |
Related information
URL releasing protocol
Publication of results
Partially published
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
Enrolling by invitation
Date of protocol fixation
| 2012 | Year | 01 | Month | 05 | Day |
Date of IRB
Anticipated trial start date
| 2012 | Year | 01 | Month | 26 | Day |
Last follow-up date
Date of closure to data entry
Date trial data considered complete
Date analysis concluded
Other
Other related information
QOL; SF-36, KD-QOL
DS-3
renal function
heart function
MRI of cerebrum
Management information
Registered date
| 2016 | Year | 12 | Month | 19 | Day |
Last modified on
| 2016 | Year | 12 | Month | 19 | Day |
Link to view the page
Value
https://center6.umin.ac.jp/cgi-open-bin/ctr_e/ctr_view.cgi?recptno=R000029125
