Contact information
Type
Scientific
Primary contact
Dr C. Verhamme
ORCID ID
Contact details
Academic Medical Center
Department of Neurology
P.O. Box 22660
Amsterdam
-
Netherlands
+31 (0)20 5663856
c.verhamme@amc.uva.nl
Additional identifiers
EudraCT number
ClinicalTrials.gov number
Protocol/serial number
N/A
Study information
Scientific title
Acronym
AATIC
Study hypothesis
Ascorbic acid has been shown to have a favorable influence on myelination in in vitro studies and in a mouse model for CMT1A. We will study the efficacy and safety of ascorbic acid treatment in young patients with CMT1A.
Ethics approval
Received from local medical ethics committee
Study design
Randomised double blind placebo controlled parallel group trial
Primary study design
Interventional
Secondary study design
Randomised controlled trial
Trial setting
Not specified
Trial type
Not Specified
Patient information sheet
Condition
Charcot-Marie-Tooth Disease Type 1A (CMT1A), Hereditary Motor and Sensory Neuropathies (HMSN Ia)
Intervention
Ascorbic acid 1000 mg (4 capsules of 250 mg) twice daily for one year or placebo in 4 capsules twice daily for one year
Intervention type
Drug
Phase
Not Specified
Drug names
Ascorbic acid
Primary outcome measures
Change in motor nerve conduction velocity of the median nerve after 1 year
Secondary outcome measures
1. Change in minimal F response latency of the median nerve after 1 year
2. Changes in compound muscle action potential amplitude and area after 1 year
3. Change in motor unit number estimation of the abductor pollicis brevis muscle after 1 year
4. Changes in handgrip strength, strength of armflexors, foot dorsiflexors, knee extensors and hip flexors after 1 year
5. Change in overall disability sum score after 1 year
6. Change in AMC Linear Disability Scale score after 1 year
7. Evaluation of serum ascorbic acid concentrations during 1 year
8. Evaluation of side effects during 1 year
Overall trial start date
01/11/2005
Overall trial end date
01/11/2007
Reason abandoned
Eligibility
Participant inclusion criteria
1. DNA-proven CMT1A patients
2. Age 12-25 years
3. CMT1A patients with symptomatology defined as muscle weakness in at least foot dorsiflexion
Participant type
Patient
Age group
Other
Gender
Both
Target number of participants
12
Participant exclusion criteria
1. Due to possible influence on severity of the neuropathy:
1.1. Known other disease that may cause a neuropathy, that may decrease mobility, or that may lead to severe disability or death in a short time
1.2. Medication that may cause a neuropathy
1.3. Chronic alcohol abuse
2. Due to study medication (ascorbic acid):
2.1. Regular use of vitamin C
2.2. Clinical or echographic signs of nephrolithiasis
2.3. Reduced glomerular filtration rate
2.4. Iron overload
2.5. No regular dental control at the dentist
2.6. Pregnancy or active pregnancy wish for women
3. Due to study design and primary outcome:
3.1. Not signing the informed consent
3.2. Psychiatric co-morbidity which may influence compliance
3.3. Not being comfortable during nerve conduction studies of the median nerve
3.4. A too small Compound Muscle Action Potential (CMAP) amplitude of the abductor pollicis brevis muscle for a proper determination of the nerve conduction velocity of the median nerve
Recruitment start date
01/11/2005
Recruitment end date
01/11/2007
Locations
Countries of recruitment
Netherlands
Trial participating centre
Academic Medical Center
Amsterdam
-
Netherlands
Sponsor information
Organisation
Academic Medical Centre, Department of Neurology (Netherlands)
Sponsor details
P.O. Box 22660
Amsterdam
1100 DD
Netherlands
Sponsor type
University/education
Website
Funders
Funder type
Hospital/treatment centre
Funder name
Academic Medical Centre, Department of Neurology (Netherlands)
Alternative name(s)
Funding Body Type
Funding Body Subtype
Location
Results and Publications
Publication and dissemination plan
Not provided at time of registration
Intention to publish date
Participant level data
Not provided at time of registration
Results - basic reporting
Publication summary
2009 results in http://www.ncbi.nlm.nih.gov/pubmed/19909499
Publication citations
-
Results
Verhamme C, de Haan RJ, Vermeulen M, Baas F, de Visser M, van Schaik IN, Oral high dose ascorbic acid treatment for one year in young CMT1A patients: a randomised, double-blind, placebo-controlled phase II trial., BMC Med, 2009, 7, 70, doi: 10.1186/1741-7015-7-70.