Ascorbic Acid Treatment in Charcot-Marie-Tooth Disease Type 1A (CMT1A) Trial
| ISRCTN | ISRCTN56968278 |
|---|---|
| DOI | https://doi.org/10.1186/ISRCTN56968278 |
| Protocol serial number | N/A |
| Sponsor | Academic Medical Centre, Department of Neurology (Netherlands) |
| Funder | Academic Medical Centre, Department of Neurology (Netherlands) |
- 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
Plain English summary of protocol
Not provided at time of registration
Contact information
Scientific
Academic Medical Center
Department of Neurology
P.O. Box 22660
Amsterdam
-
Netherlands
| Phone | +31 (0)20 5663856 |
|---|---|
| c.verhamme@amc.uva.nl |
Study information
| Primary study design | Interventional |
|---|---|
| Study design | Randomised double blind placebo controlled parallel group trial |
| Secondary study design | Randomised controlled trial |
| 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 |
| Phase | Not Specified |
| Drug / device / biological / vaccine name(s) | Ascorbic acid |
| Primary outcome measure(s) |
Change in motor nerve conduction velocity of the median nerve after 1 year |
| Key secondary outcome measure(s) |
1. Change in minimal F response latency of the median nerve after 1 year |
| Completion date | 01/11/2007 |
Eligibility
| Participant type(s) | Patient |
|---|---|
| Age group | Other |
| Sex | All |
| Target sample size at registration | 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
-
Netherlands
Results and Publications
| Individual participant data (IPD) Intention to share | No |
|---|---|
| IPD sharing plan summary | 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 |
