Clinical Trial: Efficacy and Safety of DHEA for Myotonic Dystrophy

Study Status: Completed
Recruit Status: Completed
Study Type: Interventional

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 Summary: 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.
Sponsor: University of Versailles

Current Primary Outcome: Variation in a Muscle Strength Score between randomization and study week 12 [ Time Frame: 3 months ]

Original Primary Outcome: Variation in a Muscle Strength Score between randomization and study week 12

Current Secondary Outcome:

  • 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 ]


Original Secondary Outcome:

  • - evaluation of myotonia
  • - Appel score
  • - Epworth score
  • - Forced vital capacity
  • - arterial blood gas
  • - changes in EKG and echocardiography
  • - tolerance


Information By: University of Versailles

Dates:
Date Received: September 10, 2005
Date Started: November 2004
Date Completion:
Last Updated: April 5, 2010
Last Verified: April 2010