CompletedPhase 2/3

Deflazacort in Dysferlinopathies

Germany25 participantsStarted 2003-09

Plain-language summary

The present study is designed to assess the natural history in a one year pre-phase of the trial and evaluate therapeutic efficacy and side effects of deflazacort in LGMD2B/MM patients in a placebo-controlled trial. Furthermore, long-term development of the disease under naturalistic conditions will be documented in a 2-year follow-up after the end of the double-blind treatment phase.

Who can participate

Age range18 Years

SexALL

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AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

Inclusion Criteria: * Clinically, histologically, immunohistochemically and genetically defined muscular dystrophy with dysferlin-deficiency (LGMD2B/MM). * Patients should fulfill clinical, morphological, immunohistochemical and immunoblot criteria of LGMD 2B and definite mutation in dysferlin gene. * There is no limitation on age for study inclusion. Exclusion Criteria: * Patients confined to bed or wheelchair. * Patients with other neurologic or internistic diseases and patients with former or current steroid treatment will not be included. * Exclusion criteria during the trial are withdrawal of informed consent or lack of compliance.

What they're measuring

Muscle strength according to Medical Research Council Scales (MRC) and quantitative strength measurement evaluated by hand-held dynamometry (Citec, Groningen, The Netherlands)in the same muscle groups.

Timeframe: each 6 months

Trial details

NCT IDNCT00527228

SponsorLudwig-Maximilians - University of Munich

Sponsor typeOTHER

Study typeINTERVENTIONAL

Primary completion2008-04

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