Efficacy and Safety of DHEA for Myotonic Dystrophy

University of Versailles

Status and phase: Completed; Phase 3; Phase 2

Conditions: Myotonic Dystrophy

Treatments: Drug: dehydroepiandrosterone 100 and 400 mg

Study type: Interventional

Funder types: Other

Identifiers

NCT00167609

PS001108

P001108

Details and patient eligibility

About

To test the efficacy and safety of two doses of dehydroepiandrosterone (DHEA) in adults with myotonic dystrophy

Full 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.

Enrollment: 75 patients

Sex: All

Ages: 18 to 70 years old

Volunteers: No Healthy Volunteers

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

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Double Blind