Trial Review

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Trial registered on ANZCTR


Registration number
ACTRN12606000481572
Ethics application status
Approved
Date submitted
15/11/2006
Date registered
23/11/2006
Date last updated
15/11/2010
Type of registration
Prospectively registered

Titles & IDs
Public title
Efficacy of Vitamin C treatment for children with Charcot-Marie-Tooth disease type 1A
Scientific title
Efficacy of ascorbic acid supplementation in children with Charcot-Marie-Tooth disease type 1A to improve muscle strength and peripheral nerve function
Secondary ID [1] 253088 0
Nil
Universal Trial Number (UTN)
Trial acronym
vitCmt
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Children with Charcot-Marie-Tooth disease type 1A 1458 0
Condition category
Condition code
Neurological 1554 1554 0 0
Other human genetics and inherited disorders

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Experimental group: Daily oral ascorbic acid supplementation for 12 months as per the established age-specific tolerable upper intake levels of ascorbic acid (US Food and Nutrition Board Institute of Medicine, 2000).
Intervention code [1] 1450 0
Treatment: Drugs
Comparator / control treatment
Control group: Daily oral placebo for 12 months.
Control group
Placebo

Outcomes
Primary outcome [1] 2146 0
a) Isometric muscle strength of the foot and ankle
Timepoint [1] 2146 0
Measured at baseline and 12 months
Primary outcome [2] 2147 0
b) Disease progression via nerve conduction studies of the median nerve
Timepoint [2] 2147 0
Measured at baseline and 12 months
Secondary outcome [1] 3728 0
Neurophysiological studies of the median nerve, isometric hand strength, functional ability, health-related quality of life
Timepoint [1] 3728 0
Measured at baseline and at 12-months follow-up

Eligibility
Key inclusion criteria
Diagnosis of Charcot-Marie-Tooth disease type 1A.
Minimum age
2 Years
Maximum age
16 Years
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
Pregnancy; glucose-6-phosphate dehydrogenase deficiency; hereditary hemochromatosis; thalassemia; polycythemia; leukaemia or sideroblastic anaemia; sickle cell anaemia; acute foot injuries and recent foot surgery; inflammatory arthritis; diabetes.

Study design
Purpose of the study
Treatment
Allocation to intervention
Randomised controlled trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Central randomisation by telephone
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Permuted block randomisation stratified for centre and age
Masking / blinding
Blinded (masking used)
Who is / are masked / blinded?



Intervention assignment
Parallel
Other design features
Blinded subjects, therapists and assessors
Phase
Type of endpoint/s
Safety/efficacy
Statistical methods / analysis

Recruitment
Recruitment status
Completed
Date of first participant enrolment
Anticipated
15/01/2007
Actual
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
Sample size
Target
80
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
Recruitment outside Australia
Country [1] 418 0
New Zealand
State/province [1] 418 0

Funding & Sponsors
Funding source category [1] 1694 0
University
Name [1] 1694 0
The University of Sydney
Country [1] 1694 0
Australia
Funding source category [2] 1695 0
Other
Name [2] 1695 0
Institute for Neuromuscular Research
Country [2] 1695 0
Australia
Primary sponsor type
Hospital
Name
The Children's Hospital at Westmead
Address
Locked Bag 4001, Westmead NSW 2145 Australia
Country
Australia
Secondary sponsor category [1] 1495 0
Government body
Name [1] 1495 0
The Children's Hospital at Westmead
Address [1] 1495 0
Locked Bag 4001, Westmead NSW 2145 Australia
Country [1] 1495 0
Australia

Ethics approval
Ethics application status
Approved
Ethics committee name [1] 3145 0
The Children's Hospital at Westmead
Ethics committee address [1] 3145 0
Ethics committee country [1] 3145 0
Australia
Date submitted for ethics approval [1] 3145 0
Approval date [1] 3145 0
Ethics approval number [1] 3145 0
2006/056
Ethics committee name [2] 3146 0
Royal Children's Hospital, Melbourne
Ethics committee address [2] 3146 0
Ethics committee country [2] 3146 0
Australia
Date submitted for ethics approval [2] 3146 0
Approval date [2] 3146 0
Ethics approval number [2] 3146 0
26144

Summary
Brief summary
The purpose of this trial is to determine the effect of high-dose vitamin C on muscle strength, nerve function, gross motor coordination and health-related quality of life in children with Charcot-Marie-Tooth disease type 1A.

We hypothesise that muscle strength, nerve function, gross motor coordination and health-related quality of life will be greater in children with Charcot-Marie-Tooth disease type 1A who receive dietary vitamin C supplementation compared to children receiving the placebo.
Trial website
Trial related presentations / publications
Burns J. Ouvrier RA. Yiu EM. Joseph PD. Kornberg AJ. Fahey MC. Ryan MM. Ascorbic acid for Charcot–Marie–Tooth disease type 1A in children: a randomised, double-blind, placebo-controlled, safety and efficacy trial. Lancet Neurology. 8 (6) 537-544, 2009.
Public notes

Contacts
Principal investigator
Name 27384 0
Address 27384 0
Country 27384 0
Phone 27384 0
Fax 27384 0
Email 27384 0
Contact person for public queries
Name 10639 0
Dr. Joshua Burns
Address 10639 0
Institute for Neuromuscular Research
The Children's Hospital at Westmead
Locked Bag 4001
Westmead NSW 2145
Country 10639 0
Australia
Phone 10639 0
+61 2 98451228
Fax 10639 0
+61 2 98451317
Email 10639 0
[email protected]
Contact person for scientific queries
Name 1567 0
Dr. Joshua Burns
Address 1567 0
Institute for Neuromuscular Research
The Children's Hospital at Westmead
Locked Bag 4001
Westmead NSW 2145
Country 1567 0
Australia
Phone 1567 0
+61 2 98451228
Fax 1567 0
+61 2 98451317
Email 1567 0
[email protected]

No information has been provided regarding IPD availability


What supporting documents are/will be available?

No Supporting Document Provided



Results publications and other study-related documents

Documents added manually
No documents have been uploaded by study researchers.

Documents added automatically
No additional documents have been identified.