Overview

Ascorbic Acid Treatment in CMT1A Trial (AATIC)

Status:
Completed
Trial end date:
2007-07-01
Target enrollment:
0
Participant gender:
All
Summary
Charcot-Marie-Tooth type IA (CMT1A) is the most prevalent hereditary peripheral neuropathy. Demyelination of peripheral nerves is the hallmark of CMT1A. 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.
Phase:
Phase 2
Accepts Healthy Volunteers?
No
Details
Lead Sponsor:
Academisch Medisch Centrum - Universiteit van Amsterdam (AMC-UvA)
Treatments:
Ascorbic Acid
Vitamins
Criteria
Inclusion Criteria:

- DNA-proven CMT1A patients

- Age 12-25 years

- CMT 1A patients with symptomatology defined as muscle weakness in at least foot
dorsiflexion

Exclusion Criteria:

Due to possible influence on severity of the neuropathy:

- 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

- Medication that may cause a neuropathy

- Chronic alcohol abuse

Due to study medication (ascorbic acid):

- Regular use of vitamin C

- Clinical or echographic signs of nephrolithiasis

- Reduced glomerular filtration rate

- Iron overload

- No regular dental control at the dentist

- Pregnancy or active pregnancy wish for women

Due to study design and primary outcome:

- Not signing the informed consent

- Psychiatric co-morbidity which may influence compliance

- Not being comfortable during nerve conduction studies of the median nerve

- A too small CMAP amplitude of the abductor pollicis brevis muscle for a proper
determination of the nerve conduction velocity of the median nerve