Brief Title
Efficacy and Safety of DHEA for Myotonic Dystrophy
Official Title
Phase 3 Study of Oral Dehydroepiandrosterone (DHEA) in Adults With Myotonic Dystrophy
Brief Summary
To test the efficacy and safety of two doses of dehydroepiandrosterone (DHEA) in adults with myotonic dystrophy
Detailed Description
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 Phase
Phase 2/Phase 3
Study Type
Interventional
Primary Outcome
Variation in a Muscle Strength Score between randomization and study week 12
Secondary Outcome
evaluation of myotonia
Condition
Myotonic Dystrophy
Intervention
dehydroepiandrosterone 100 and 400 mg
Publications
* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.
Recruitment Information
Recruitment Status
Drug
Estimated Enrollment
75
Start Date
November 2004
Completion Date
December 2006
Eligibility Criteria
Inclusion Criteria: - Myotonic dystrophy (genetically proven) - MDRS score of 3 or 4 Exclusion Criteria: - Age <18 years or >70 years - Pregnancy or breastfeeding - Poor compliance to treatment and follow up - Inclusion in any other clinical trial - Severe cardiac disease: acute myocardial infarction in the preceding 6 months, unstable heart failure, uncontrolled hypertension (systolic blood pressure >180 mmHg or diastolic blood pressure >100 mmHg after 10 minutes of rest in the lying position), severe arteritis, any past history of thrombose or embolic event, any past history of symptomatic arrhythmia) - Chronic renal failure - Chronic liver disease - Long term mechanical ventilation - Any ongoing cancer - Any underlying endocrine disorders - Impaired swallowing - Previous treatment with DHEA
Gender
All
Ages
18 Years - 70 Years
Accepts Healthy Volunteers
No
Contacts
Djillali annane, MD, PhD, ,
Location Countries
France
Location Countries
France
Administrative Informations
NCT ID
NCT00167609
Organization ID
P001108
Secondary IDs
PS001108
Study Sponsor
University of Versailles
Collaborators
Association Française contre les Myopathies (AFM), Paris
Study Sponsor
Djillali annane, MD, PhD, Study Chair, Assistance Publique Hôpitaux de Paris - University of Versailles
Verification Date
April 2010