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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

Tracking Information
First Submitted Date  ICMJE September 10, 2005
First Posted Date  ICMJE September 14, 2005
Last Update Posted Date April 6, 2010
Study Start Date  ICMJE November 2004
Primary Completion Date Not Provided
Current Primary Outcome Measures  ICMJE
 (submitted: May 8, 2007)
Variation in a Muscle Strength Score between randomization and study week 12 [ Time Frame: 3 months ]
Original Primary Outcome Measures  ICMJE
 (submitted: September 10, 2005)
Variation in a Muscle Strength Score between randomization and study week 12
Change History
Current Secondary Outcome Measures  ICMJE
 (submitted: May 8, 2007)
  • 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 Measures  ICMJE
 (submitted: September 10, 2005)
  • - evaluation of myotonia
  • - Appel score
  • - Epworth score
  • - Forced vital capacity
  • - arterial blood gas
  • - changes in EKG and echocardiography
  • - tolerance
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Efficacy and Safety of DHEA for Myotonic Dystrophy
Official Title  ICMJE 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 Type  ICMJE Interventional
Study Phase  ICMJE Phase 2
Phase 3
Study Design  ICMJE Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Double
Primary Purpose: Treatment
Condition  ICMJE Myotonic Dystrophy
Intervention  ICMJE Drug: dehydroepiandrosterone 100 and 400 mg
Study Arms  ICMJE Not Provided
Publications * Pénisson-Besnier I, Devillers M, Porcher R, Orlikowski D, Doppler V, Desnuelle C, Ferrer X, Bes MC, Bouhour F, Tranchant C, Lagrange E, Vershueren A, Uzenot D, Cintas P, Solé G, Hogrel JY, Laforêt P, Vial C, Vila AL, Sacconi S, Pouget J, Eymard B, Chevret S, Annane D. Dehydroepiandrosterone for myotonic dystrophy type 1. Neurology. 2008 Aug 5;71(6):407-12. doi: 10.1212/01.wnl.0000324257.35759.40.

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Completed
Actual Enrollment  ICMJE
 (submitted: September 10, 2005)
75
Original Enrollment  ICMJE Same as current
Actual Study Completion Date  ICMJE December 2006
Primary Completion Date Not Provided
Eligibility Criteria  ICMJE

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
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 18 Years to 70 Years   (Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE France
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT00167609
Other Study ID Numbers  ICMJE P001108
PS001108
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement  ICMJE Not Provided
Responsible Party Not Provided
Study Sponsor  ICMJE University of Versailles
Collaborators  ICMJE
  • Association Française contre les Myopathies (AFM), Paris
  • Assistance Publique - Hôpitaux de Paris
Investigators  ICMJE
Study Chair: Djillali annane, MD, PhD Assistance Publique Hôpitaux de Paris - University of Versailles
Study Director: martine devillers, MD AFM
PRS Account University of Versailles
Verification Date April 2010

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP