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| Sponsored by: |
Ludwig-Maximilians - University of Munich |
| Information provided by: | Ludwig-Maximilians - University of Munich |
| ClinicalTrials.gov Identifier: | NCT00527228 |
Purpose
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.
| Condition | Intervention | Phase |
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LGMD2B Miyoshi Myopathy Dysferlinopathy |
Drug: deflazacort Drug: placebo |
Phase II Phase III |
| MedlinePlus related topics: | Muscle Disorders |
| ChemIDplus related topics: | Corticosteroids Deflazacort |
| Study Type: | Interventional |
| Study Design: | Treatment, Randomized, Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor), Placebo Control, Crossover Assignment, Safety/Efficacy Study |
| Official Title: | Deflazacort in Dysferlinopathies (LGMD2B/MM) - a Double Blind, Placebo-Controlled Clinical Study |
| Estimated Enrollment: | 35 |
| Study Start Date: | September 2003 |
| Estimated Study Completion Date: | September 2008 |
| Primary Completion Date: | April 2008 (Final data collection date for primary outcome measure) |
| Arms | Assigned Interventions |
|
A: Active Comparator
After 6 months of treatment, and a 3-months wash-out, there is cross-over to Arm B
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Drug: deflazacort
In the first 12 months, patients will receive no treatment to assess the natural history of the disease. Afterwards, patients will be treated with deflazacort 1mg/kg/day or placebo for the first month on treatment, from the second month on deflazacort or placebo will be administered on an alternate day regimen). Patients will be randomized to six months verum or placebo each, after a 3-months wash-out patients cross over to the alternate treatment for six months. In a 2-years follow-up phase after the double-blind treatment phase, long-term development of the disorder will be documented.
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B: Placebo Comparator
After 6 months of treatment, and a 3-months wash-out, there is cross-over to Arm A
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Drug: placebo |
Limb girdle muscular dystrophies (LGMDs) are a genetically heterogeneous group of disorders encompassing various genetically defined subtypes (LGMD 2A-2H). Therapeutic trials should address each disease entity separately to assess effectiveness of medical treatments. A placebo-controlled trial in patients with dysferlinopathy may reveal insights in the natural course of the disease and show therapeutic options in a homogeneous group of patients. So far, steroids are the only drugs showing efficacy in muscular dystrophies, mainly in Duchenne muscular dystrophy (DMD). Both dystrophin and dysferlin are attached to the sarcolemma and deficiency of both proteins cause sarcolemmal defects; therefore, any membrane-stabilizing steroid effect may be beneficial in both DMD and LGMD2B/ Myoshi myopathy (MM). Furthermore, there is marked inflammation in muscle biopsies of many LGMD2B patients. Therefore, the anti-inflammatory effect of steroids may improve muscle function in LGMD2B/MM. In our trial, effects of deflazacort in patients with dysferlinopathy (LGMD2B/MM) on strength and daily-life activities are addressed. The present study is designed to assess the natural history and evaluate therapeutic efficacy and side effects of deflazacort / placebo in LGMD2B/MM patients.
Although no major therapeutic breakthrough has been achieved and curative treatment modalities are not yet applicable, life expectancy and quality of life of dysferlinopathy patients could be remarkably improved by establishing a drug therapy, capable of delaying the dystrophic process and improving muscle strength and function. Therefore, the results of this study are warranted and may influence further guidelines for steroid treatment in dysferlinopathies. Furthermore, the assessment of the natural history of the disease will provide new insights in the clinical understanding of dysferlinopathies.
Eligibility
| Ages Eligible for Study: | 18 Years and older |
| Genders Eligible for Study: | Both |
| Accepts Healthy Volunteers: | No |
Inclusion Criteria:
Exclusion Criteria:
Contacts and Locations| Contact: Maggie C. Walter, MD | +49-89-5160- ext 7400 | maggie.walter@lrz.uni-muenchen.de |
| Germany | |||||
| Friedrich-Baur-Institute, Dept. of Neurology, Ludwig-Maximilians-University of Munich | Recruiting | ||||
| Munich, Germany, 80801 | |||||
| Contact: Maggie C. Walter, MD +49-89-5160- ext 7400 maggie.walter@lrz.uni-muenchen.de | |||||
| Principal Investigator: Maggie C. Walter, MD | |||||
| Ludwig-Maximilians - University of Munich |
| Principal Investigator: | Maggie C. Walter, MD | Friedrich-Baur-Institute, Dept. of Neurology, Ludwig-Maximilians-University of Munich, Germany |
More Information
Homepage of the German Muscular Dystrophy Network (MD-NET)
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| Responsible Party: | Friedrich-Baur-Institute ( PD Dr. Maggie Walter ) |
| Study ID Numbers: | 274/02 |
| First Received: | September 6, 2007 |
| Last Updated: | May 6, 2008 |
| ClinicalTrials.gov Identifier: | NCT00527228 |
| Health Authority: | Germany: Federal Institute for Drugs and Medical Devices |
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