Overview

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.