UMIN-CTR Clinical Trial
| Unique ID issued by UMIN | UMIN000001535 |
|---|---|
| Receipt number | R000001855 |
| Scientific Title | A multicenter, randomized open trial of ascorbic acid treatment in Charcot-Marie-Tooth disease type 1A |
| Date of disclosure of the study information | 2009/01/01 |
| Last modified on | 2010/07/02 08:32:52 |
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Basic information
Public title
A multicenter, randomized open trial of ascorbic acid treatment in Charcot-Marie-Tooth disease type 1A
Acronym
CMT-AA
Scientific Title
A multicenter, randomized open trial of ascorbic acid treatment in Charcot-Marie-Tooth disease type 1A
Scientific Title:Acronym
CMT-AA
Region
| Japan |
Condition
Condition
Charcot-Marie-Tooth disease 1A
Classification by specialty
| Neurology |
Classification by malignancy
Others
Genomic information
YES
Objectives
Narrative objectives1
Charcot-Marie-Tooth disease 1A (CMT1A) is an autosomal dominant hereditary neuropathy caused by duplication of PMP22 gene. There has been no treatment for CMT1A, but recent studies showed that ascorbic acid (AA) was efficacious in the transgenic mouse model. The purpose of this multicenter, randomized open trial is to evaluate the safety and efficacy of AA in CMT1A.
Basic objectives2
Safety,Efficacy
Basic objectives -Others
Trial characteristics_1
Exploratory
Trial characteristics_2
Explanatory
Developmental phase
Phase I,II
Assessment
Primary outcomes
An improvement in CMT neuropathy score at baseline, 4, 8 and 12 weeks thereafter.
Key secondary outcomes
An improvement in muscle strength and ulnar nerve conduction study at baseline, 4, 8 and 12 weeks thereafter.
Base
Study type
Interventional
Study design
Basic design
Parallel
Randomization
Randomized
Randomization unit
Individual
Blinding
Open -no one is blinded
Control
No treatment
Stratification
YES
Dynamic allocation
NO
Institution consideration
Institution is not considered as adjustment factor.
Blocking
YES
Concealment
Central registration
Intervention
No. of arms
2
Purpose of intervention
Treatment
Type of intervention
| Medicine |
Interventions/Control_1
Ascorbic acid (AA) treatment group
(12 weeks oral AA (20mg/kg/day)
Interventions/Control_2
no AA treatment group
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
| 15 | years-old | <= |
Age-upper limit
| 75 | years-old | > |
Gender
Male and Female
Key inclusion criteria
1) CMT1A patients with PMP duplications
(triple gene doses)
2) Patients with normal function of main
organs (heart, lung, liver and kidney)
3) Patients with no severe complications
Key exclusion criteria
1) Patients with dementia
2) Patients with severe complications
3) Patients who are not appropriate for the trial by the decision of the physician in charge
Target sample size
60
Research contact person
Name of lead principal investigator
| 1st name | |
| Middle name | |
| Last name | Masanori Nakagawa |
Organization
Kyoto Prefectural University of Medicine, Graduate School of Medical Science
Division name
Department of Neurology
Zip code
Address
Kawaramachi Hirokoji, Kajii-chou 465, Kamigyo-ku, Kyoto 602-0841, Japan
TEL
075-251-5793
Public contact
Name of contact person
| 1st name | |
| Middle name | |
| Last name | Masanori Nakagawa |
Organization
Kyoto Prefectural University of Medicine, Graduate School of Medical Science
Division name
Department of Neurology
Zip code
Address
Kawaramachi Hirokoji, Kajii-chou 465, Kamigyo-ku, Kyoto 602-0841, Japan
TEL
075-251-5793
Homepage URL
mnakagaw@koto.kpu-m.ac.jp
Sponsor or person
Institute
New treatment strategies for intractable neuropathies based on its pathomechanism.
The Research Grant 19A-5 for Nervous and Mental Disorders from the Ministry of Health, Labour and Welfare.
Institute
Department
Personal name
Funding Source
Organization
New treatment strategies for intractable neuropathies based on its pathomechanism.
The Research Grant 19A-5 for Nervous and Mental Disorders from the Ministry of Health, Labour and Welfare.
Organization
Division
Category of Funding 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
京都府立医科大学附属病院（京都府）
Other administrative information
Date of disclosure of the study information
| 2009 | Year | 01 | Month | 01 | 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
Completed
Date of protocol fixation
| 2005 | Year | 03 | Month | 04 | Day |
Date of IRB
Anticipated trial start date
| 2005 | Year | 04 | Month | 01 | Day |
Last follow-up date
| 2009 | Year | 09 | Month | 01 | Day |
Date of closure to data entry
| 2009 | Year | 12 | Month | 01 | Day |
Date trial data considered complete
| 2010 | Year | 03 | Month | 01 | Day |
Date analysis concluded
| 2010 | Year | 07 | Month | 01 | Day |
Other
Other related information
Management information
Registered date
| 2008 | Year | 11 | Month | 30 | Day |
Last modified on
| 2010 | Year | 07 | Month | 02 | Day |
Link to view the page
Value
https://center6.umin.ac.jp/cgi-open-bin/ctr_e/ctr_view.cgi?recptno=R000001855
| Research Plan | |
|---|---|
| Registered date | File name |
| Research case data specifications | |
|---|---|
| Registered date | File name |
| Research case data | |
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| Registered date | File name |
