Efficacy and Safety of DHEA for Myotonic Dystrophy

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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