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Name:
UMIN ID:

Recruitment status Completed
Unique ID issued by UMIN UMIN000001535
Receipt No. 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

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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
Email

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
Email mnakagaw@koto.kpu-m.ac.jp

Sponsor
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

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
Email

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
URL(English) https://upload.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
Registered date File name


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