Deflazacort in Dysferlinopathies

• Status: Completed
• Trial end date: 2008-09-01
• Target enrollment: 0
• Participant gender: All

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.

• Phase: Phase 2/Phase 3
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Ludwig-Maximilians - University of Munich
• Treatments: Deflazacort

Criteria

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.