To test the efficacy and safety of two doses of dehydroepiandrosterone (DHEA) in adults with myotonic dystrophy
Myotonic dystrophy is an inherited disorder that affects 1 per 8000 adults. The disease is characterize by muscular dystrophy, myotonia, cardiac disorders, cognitive function impairment, hypersomnia, hair loss, endocrine disorders. Recent small studies suggested that DHEA treatment may improve muscle strength in adults with myotonic dystrophy. Thus, the current study aims at investigating the safety and efficacy of a prolonged treatment with DHEA in adults with myotonic dystrophy.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
DOUBLE
Enrollment
75
CHU
Angers, France
CHU
Bordeaux, France
Raymond Poincaré Hospital
Garches, France
CHU
Grenoble, France
Variation in a Muscle Strength Score between randomization and study week 12
Time frame: 3 months
evaluation of myotonia
Time frame: 3 months
Appeal score
Time frame: 3 months
Epworth score
Time frame: 3 months
Forced vital capacity
Time frame: 3 months
arterial blood gas
Time frame: 3 months
changes in EKG and echocardiography
Time frame: 3 months
tolerance
Time frame: 3 months
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CHU
Lyon, France
CHU Pouget
Marseille, France
CHU
Nice, France
Institut de myologie
Paris, France
CHU
Strasbourg, France
CHU
Toulouse, France