UMIN-CTR Clinical Trial
| Unique ID issued by UMIN | UMIN000030341 |
|---|---|
| Receipt number | R000034630 |
| Scientific Title | Clinical study of tolcapone for transthyretin (TTR)-type cerebral amyloid angiopathy in patients with familial amyloid polyneuropahty (FAP) |
| Date of disclosure of the study information | 2018/01/01 |
| Last modified on | 2022/12/15 10:10:11 |
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Basic information
Public title
Clinical study of tolcapone for transthyretin (TTR)-type cerebral amyloid angiopathy in patients with familial amyloid polyneuropahty (FAP)
Acronym
Clinical study of tolcapone for ATTR-type CAA
Scientific Title
Clinical study of tolcapone for transthyretin (TTR)-type cerebral amyloid angiopathy in patients with familial amyloid polyneuropahty (FAP)
Scientific Title:Acronym
Clinical study of tolcapone for ATTR-type CAA
Region
| Japan |
Condition
Condition
Familial amyloid polyneuropahty
Classification by specialty
| Neurology |
Classification by malignancy
Others
Genomic information
NO
Objectives
Narrative objectives1
pharmacokinetics
Basic objectives2
Safety
Basic objectives -Others
Trial characteristics_1
Trial characteristics_2
Developmental phase
Assessment
Primary outcomes
stability ratio of CSF TTR tetramer
Key secondary outcomes
Base
Study type
Interventional
Study design
Basic design
Parallel
Randomization
Non-randomized
Randomization unit
Blinding
Open -no one is blinded
Control
Dose comparison
Stratification
Dynamic allocation
Institution consideration
Blocking
Concealment
Intervention
No. of arms
2
Purpose of intervention
Treatment
Type of intervention
| Medicine |
Interventions/Control_1
Administer tolcapone at 300 mg/day orally for 7 days.
Interventions/Control_2
Administer tolcapone at 600 mg/day orally for 7 days.
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
| 20 | years-old | <= |
Age-upper limit
| Not applicable |
Gender
Male and Female
Key inclusion criteria
Biopsy proven amyloid deposition
Genotyping of variant TTR
Age > 20
Key exclusion criteria
Asymptomatic mutation carriers
Contraindication for tolcapone
An ALT or AST measurement > 2 times the ULN (Upper Limit of Normal)
Treatment with NSAID or a known TTR binder within the last 4 weeks
Stage 3 disease
Target sample size
10
Research contact person
Name of lead principal investigator
| 1st name | |
| Middle name | |
| Last name | Yoshiki Sekijima |
Organization
Shinshu University School of Medicine
Division name
Department of Medicine (Neurology & Rheumatology)
Zip code
Address
3-1-1 Asahi, Matsumoto 390-8621, Japan
TEL
0263372673
sekijima@shinshu-u.ac.jp
Public contact
Name of contact person
| 1st name | |
| Middle name | |
| Last name | Yoshiki Sekijima |
Organization
Shinshu University School of Medicine
Division name
Department of Medicine (Neurology & Rheumatology)
Zip code
Address
3-1-1 Asahi, Matsumoto 390-8621, Japan
TEL
0263372673
Homepage URL
sekijima@shinshu-u.ac.jp
Sponsor or person
Institute
Shinshu University School of Medicine
Institute
Department
Personal name
Funding Source
Organization
Corino Therapeutics, Inc.
Organization
Division
Category of Funding Organization
Outside Japan
Nationality of Funding Organization
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
| 2018 | Year | 01 | 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
9
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
| 2017 | Year | 12 | Month | 06 | Day |
Date of IRB
| 2017 | Year | 12 | Month | 07 | Day |
Anticipated trial start date
| 2018 | Year | 01 | Month | 01 | Day |
Last follow-up date
| 2019 | Year | 03 | Month | 19 | Day |
Date of closure to data entry
| 2019 | Year | 03 | Month | 19 | Day |
Date trial data considered complete
| 2019 | Year | 03 | Month | 19 | Day |
Date analysis concluded
| 2019 | Year | 03 | Month | 19 | Day |
Other
Other related information
Management information
Registered date
| 2017 | Year | 12 | Month | 11 | Day |
Last modified on
| 2022 | Year | 12 | Month | 15 | Day |
Link to view the page
Value
https://center6.umin.ac.jp/cgi-open-bin/ctr_e/ctr_view.cgi?recptno=R000034630
| Research Plan | |
|---|---|
| Registered date | File name |
| Research case data specifications | |
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| Registered date | File name |
| Research case data | |
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| Registered date | File name |
