Treatment of Duchenne Muscular Dystrophy with Cyclosporine A
- Conditions
- G71.0Muscular dystrophy
- Registration Number
- DRKS00000445
- Lead Sponsor
- niversitätsklinikum FreiburgZentrum für Kinder- und Jugendmedizin
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Complete
- Sex
- Male
- Target Recruitment
- 150
Male patients, minimum age 5
- Ability to walk independently (minimum 50 meters)
- Proven diagnosis of duchenne muscular dystrophy:
clinical symptoms, increase of CK (more than 10-fold), together with at least one of the following criteria:
dystrophine immunofluorescence and/or
immunoblot negative muscle biopsy;
positive gene deletion for duchenne;
positive family history (with proven duchenne dystrophy)
- Cooperation concerning visits
- Able to swallow study medication (capsules)
- Parents´ written informed consent
- Child´s written informed consent if able to understand meaning of the study
- Preceding therapy of duchenne with steroids
- Change of a medication or alimentary additive within the last 3 months
- Intake of clenbuterol or other sympathicomimetic drugs within 3 months of study entry
- Participation on another trial within the last 3 months
- Contraindications against intake of csA: functional renal impairment (creatinine above 0.7 mg/dl), uncontrolled hypertension (blood pressure above 97 percentile), known allergy against csA, uncontrolled infectious diseases, malignant tumors, severe liver disease, hyperuricemia, hypo magnesemia, necessity of therapy with drugs interfering pharmacokinetically with csA
- Contraindications against intake of prednisone: known anamnestic gastrointestinal bleeding, cataract, diabetes, uncontrolled hypertension, uncontrolled infectious diseases (tuberculosis, infections with herpes, systemic mycosis), pronounced osteoporosis, 8 weeks before or 2 weeks after vaccination, glaucoma
Study & Design
- Study Type
- interventional
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method Primary outcome will be manual muscle testing using an extended 11-point version of the Medical Research Council (MRC) score. MRC is a validated instrument to assess muscle strength in DMD patients in 28 different muscle groups including neck, shoulder, elbow, wrist, hip, knee, and ankle in defined positions using an ordinal 11-point scale. It has been used as primary outcome measure in various controlled trials for DMD. The MRC score is calculated as a percentage of a possible total score (%MRC) and the change of %MRC between baseline (M0) and month 3 (M3) was used to assess CSA monotherapy. Respectively, change between month 3 (M3) and month 15 (M15) was used to assess the combination of CSA with intermittent prednisone.
- Secondary Outcome Measures
Name Time Method