Outcome Measures and Clinical trials in CHARCOT-MARIE-TOOTH DISEASE

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Outcome Measures and Clinical trials in CHARCOT-MARIE-TOOTH DISEASE

The 168th ENMC workshop organized from 18 - 20 September 2009 in The Netherlands was the second ENMC workshop on Charcot-Marie-Tooth. The first one was the 136th ENMC workshop held in April 2005.

Twenty-three participants from 9 countries (Australia; United Kingdom; France; Germany; Italy; Spain,Belgium, U.S.A. and The Netherlands) attended this ENMC workshop on Outcome Measures and Clinical Trials in Charcot-Marie-Tooth disease. Participants included adult and paediatric neurologists, biologists, molecular geneticists, two allied health professionals and representatives from TREAT NMD and two CMT patient organisations (Italian and US).

The ENMC workshop on outcome measures and clinical trials in CMT was very successful.

The main aims of the workshop were:

1) to discuss and agree how to modify the CMTNS for adults to increase sensitivity following the experience with the Vitamin C trials.

2) to prepare a draft paediatric impairment and disability scale for children with CMT.

3) to discuss an international CMT patient registry done in collaboration with Treat-NMD and to agree a minimal dataset

4) to discuss trial design for CMT.

Achievements.

1) During the workshop we worked on a modified version of the CMTNS for adults and plan to finalise and validate this version.

2a) A draft paediatric score was prepared and this will be further modified and validated over the next year.

2b) Furthermore, a joint ENMC working group was established to develop and test CMT-specific QoL measures for children and adults.

3) The workshop was enthusiastic that an international CMT patient registry be developed in collaboration with Treat-NMD and a minimal dataset is being developed. Furthermore, the facility is available to host the registry in the University of South Florida, USA.

4) A very useful discussion on trial design for CMT took place based on our experience with Vitamin C trials and the importance of powering trials sufficiently, choosing appropriate primary outcome measures, running trials for an adequate length of time, proper preclinical and early clinical trials, and biological markers evaluation was emphasized. A full report is published in Neuromuscular Disorders(pdf)

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