Investigators:
We would like to invite you to consider involving your children with CMT type 1A to participate in an important clinical trial being conducted at The Children's Hospital at Westmead (Sydney) and Royal Children's Hospital (Melbourne).
This study will establish if Vitamin C helps slow the progression of CMT type 1A in 80 children aged 2-16 years. There has been some very promising research showing improved endurance, speed and muscle strength in mice with CMT type 1A treated with high doses of oral Vitamin C (Passage et al, Nat Med 10: 396-401, 2004). We want to know if children treated with vitamin C will show similar benefits.
Participants in this study will be given chewable vitamin C or a placebo daily for the 12 months. We will assess each child at the start and at the end of the study, looking at measurements that we would expect to change over time in CMT type 1A (muscle strength, functional ability, balance, quality of life and nerve conduction speed). From this, we will be able to see if there is a difference between groups.
Participants will be required to attend The Children's Hospital at Westmead (Sydney) or Royal Children's Hospital (Melbourne) on 2 occasions for approximately 2 hours. Transport and accommodation costs to attend either Sydney or Melbourne will be reimbursed from anywhere in Australia or New Zealand.
The trial has Ethics Committee approval from The Children's Hospital at Westmead (Sydney), The University of Sydney and Royal Children's Hospital (Melbourne).
If you have would like to be involved please contact:
Dr Joshua Burns
NHMRC Australian Clinical Research Fellow
Institute for Neuromuscular Research
The Children's Hospital at Westmead
Telephone: 02 9845 1228
Email: joshuab2@chw.edu.au