Basic methodologies for clinical trials in myopathies

Location: Naarden, The Netherlands

The ENMC workshop "Basic methodologies for clinical trials in myopathies"was held in Naarden from 11th - 13th May 2001. It was attended by 17 active participants from Belgium, Denmark, France, Germany, The Netherlands, United Kingdom and Switzerland.

The meeting opened with a review of studies performed in athletes to understand mechanisms to improve muscle function and in animal models of muscular dystrophy. Reviews of the present status in surgical and physiotherapeutic studies followed.

In a second session, the practical organisation of multicenter clinical trials was examined, taking into consideration the fact that many myopathies are rare disorders.
Co-ordination between the different groups is important, in order to avoid duplication and to be able to compare results in systematic reviews with metaanalyses where possible.

In a third session assessment instruments were discussed, in the light of experience in other neuromuscular disorders, in particular amyotrophic lateral sclerosis.
Different methods to quantify force, using manual testing and apparatus, to assess functional capacity and impairment, and to measure quality of life were reviewed.
No consensus has yet been achieved in the use of these instruments, and a lack of uniformity hampers generalised use even of common methods.
Laboratory measurements, such as blood sampling, muscle biopsy, imaging and magnetic resonance spectroscopy are important within specific trials, but not for broader therapeutic trials.

In a last session, statistical methods were examined and the importance of obtaining professional advice from the onset of trial planning was stressed.
The participants agreed upon continuing this evaluation process in order to provide a framework for the organisation of therapeutic trials within ENMC.

An extended report of the meeting and a critical evaluation of available assessment methods will be submitted for publication in Neuromuscular Disorders.

Prof. J.-M. Burgunder (Bern, Switzerland)
Chairman