Ascorbic Acid Treatment in Charcot-Marie-Tooth Disease Type 1A (CMT1A) Trial

ISRCTN	ISRCTN56968278
DOI	https://doi.org/10.1186/ISRCTN56968278
Secondary identifying numbers	N/A

Submission date: 22/11/2005
Registration date: 22/11/2005
Last edited: 18/12/2009

Recruitment status: No longer recruiting
Overall study status: Completed
Condition category: Nervous System Diseases

Prospectively registered

Protocol

Statistical analysis plan

Results

Individual participant data

Plain English summary of protocol

Not provided at time of registration

Contact information

Dr C. Verhamme
Scientific

Academic Medical Center
Department of Neurology
P.O. Box 22660
Amsterdam
-
Netherlands

Phone	+31 (0)20 5663856
Email	c.verhamme@amc.uva.nl

Study information

Study design	Randomised double blind placebo controlled parallel group trial
Primary study design	Interventional
Secondary study design	Randomised controlled trial
Study setting(s)	Not specified
Study type	Not Specified
Scientific title
Study acronym	AATIC
Study objectives	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(s)	Received from local medical ethics committee
Health condition(s) or problem(s) studied	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
Pharmaceutical study type(s)
Phase	Not Specified
Drug / device / biological / vaccine name(s)	Ascorbic acid
Primary outcome measure	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 study start date	01/11/2005
Completion date	01/11/2007

Eligibility

Participant type(s)	Patient
Age group	Other
Sex	Both
Target number of participants	12
Key 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
Key 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
Date of first enrolment	01/11/2005
Date of final enrolment	01/11/2007

Locations

Countries of recruitment

Netherlands

Study participating centre

Academic Medical Center

Amsterdam
-
Netherlands

Sponsor information

Academic Medical Centre, Department of Neurology (Netherlands)
University/education

P.O. Box 22660
Amsterdam
1100 DD
Netherlands

Website	http://www.amc.nl/
"ROR"	https://ror.org/03t4gr691

Funders

Funder type

Hospital/treatment centre

Academic Medical Centre, Department of Neurology (Netherlands)

No information available

Results and Publications

Intention to publish date
Individual participant data (IPD) Intention to share	No
IPD sharing plan summary	Not provided at time of registration
Publication and dissemination plan	Not provided at time of registration
IPD sharing plan

Study outputs

Output type	Details	Date created	Date added	Peer reviewed?	Patient-facing?
Results article	results	12/11/2009		Yes	No