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| Sponsor | University College London |
| Funder | Muscular Dystrophy Campaign (MDC) |
| CI | Professor Mary M Reilly |
| UK sites | London Queen Square |
| Contact details | m.laura@ucl.ac.uk |
| More information |
Background
CMT1A is associated with a duplication of the gene for the peripheral
myelin protein 22 (PMP22). To date there is no pharmacological treatment for
CMT1A patients.
Treatment for CMT is limited to symptomatic therapy such as physiotherapy and surgery for skeletal deformities.
Recently, treatment with ascorbic acid (AA) has been shown to be effective in a mouse-model of the human disease. Treated animals had much less severe neuropathy compared to untreated controls. Some clinical parameters even improved during treatment.
Trial information
This is a phase III prospective, multi-centre, randomised, double-blind, placebo-controlled study.
Primary objective
To evaluate the efficacy of AA in treatment of CMT1A.
Recruitment
The study is now complete. 50 patients were enrolled at the MRC Centre for Neuromuscular Diseases.