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