We're building a better ClinicalTrials.gov. Check it out and tell us what you think!
Try the New Site
We're building a modernized ClinicalTrials.gov! Visit Beta.ClinicalTrials.gov to try the new functionality.
ClinicalTrials.gov Menu

Efficacy and Safety of DHEA for Myotonic Dystrophy

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
ClinicalTrials.gov Identifier: NCT00167609
Recruitment Status : Completed
First Posted : September 14, 2005
Last Update Posted : April 6, 2010
Association Française contre les Myopathies (AFM), Paris
Assistance Publique - Hôpitaux de Paris
Information provided by:
University of Versailles

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

Condition or disease Intervention/treatment Phase
Myotonic Dystrophy Drug: dehydroepiandrosterone 100 and 400 mg Phase 2 Phase 3

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.

Layout table for study information
Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 75 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Double
Primary Purpose: Treatment
Official Title: Phase 3 Study of Oral Dehydroepiandrosterone (DHEA) in Adults With Myotonic Dystrophy
Study Start Date : November 2004
Actual Study Completion Date : December 2006

Resource links provided by the National Library of Medicine

Drug Information available for: Prasterone

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

Secondary Outcome Measures :
  1. evaluation of myotonia [ Time Frame: 3 months ]
  2. Appeal score [ Time Frame: 3 months ]
  3. Epworth score [ Time Frame: 3 months ]
  4. Forced vital capacity [ Time Frame: 3 months ]
  5. arterial blood gas [ Time Frame: 3 months ]
  6. changes in EKG and echocardiography [ Time Frame: 3 months ]
  7. tolerance [ Time Frame: 3 months ]

Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.

Layout table for eligibility information
Ages Eligible for Study:   18 Years to 70 Years   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT00167609

Layout table for location information
Angers, France
Bordeaux, France
Raymond Poincaré Hospital
Garches, France, 92380
Grenoble, France
Lyon, France
CHU Pouget
Marseilles, France
Nice, France
Institut de myologie
Paris, France
Strasbourg, France
Toulouse, France
Sponsors and Collaborators
University of Versailles
Association Française contre les Myopathies (AFM), Paris
Assistance Publique - Hôpitaux de Paris
Layout table for investigator information
Study Chair: Djillali annane, MD, PhD Assistance Publique Hôpitaux de Paris - University of Versailles
Study Director: martine devillers, MD AFM
Publications of Results:
Layout table for additonal information
ClinicalTrials.gov Identifier: NCT00167609    
Other Study ID Numbers: P001108
First Posted: September 14, 2005    Key Record Dates
Last Update Posted: April 6, 2010
Last Verified: April 2010
Keywords provided by University of Versailles:
myotonic dystrophy
Additional relevant MeSH terms:
Layout table for MeSH terms
Myotonic Dystrophy
Muscular Dystrophies
Muscular Disorders, Atrophic
Muscular Diseases
Musculoskeletal Diseases
Myotonic Disorders
Heredodegenerative Disorders, Nervous System
Neurodegenerative Diseases
Nervous System Diseases
Neuromuscular Diseases
Genetic Diseases, Inborn
Adjuvants, Immunologic
Immunologic Factors
Physiological Effects of Drugs