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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
Sponsor:
Collaborators:
Association Française contre les Myopathies (AFM), Paris
Assistance Publique - Hôpitaux de Paris
Information provided by:
University of Versailles

Study Description
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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.
Study Design
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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

Arms and Interventions
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Outcome Measures
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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 ]

Eligibility Criteria
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Ages Eligible for Study:   18 Years to 70 Years   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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
Contacts and Locations
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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


Locations
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France
CHU
Angers, France
CHU
Bordeaux, France
Raymond Poincaré Hospital
Garches, France, 92380
CHU
Grenoble, France
CHU
Lyon, France
CHU Pouget
Marseilles, France
CHU
Nice, France
Institut de myologie
Paris, France
CHU
Strasbourg, France
CHU
Toulouse, France
Sponsors and Collaborators
University of Versailles
Association Française contre les Myopathies (AFM), Paris
Assistance Publique - Hôpitaux de Paris
Investigators
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Study Chair: Djillali annane, MD, PhD Assistance Publique Hôpitaux de Paris - University of Versailles
Study Director: martine devillers, MD AFM
More Information
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Publications of Results:

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ClinicalTrials.gov Identifier: NCT00167609    
Other Study ID Numbers: P001108
PS001108
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
hypersomnia
dehydroepiandrosterone
Additional relevant MeSH terms:
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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
Dehydroepiandrosterone
Adjuvants, Immunologic
Immunologic Factors
Physiological Effects of Drugs