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Efficacy and Safety of DHEA for Myotonic Dystrophy

This study has been completed.
Sponsor:
Collaborators:
Association Française contre les Myopathies (AFM), Paris
AP-HP
Information provided by:
University of Versailles
ClinicalTrials.gov Identifier:
NCT00167609
First received: September 10, 2005
Last updated: April 5, 2010
Last verified: April 2010
History of Changes
  Purpose

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


Condition Intervention Phase
Myotonic Dystrophy
 Drug: dehydroepiandrosterone 100 and 400 mg
 Phase 2
Phase 3

Study Type: Interventional
Study Design: Allocation: Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Parallel Assignment
Masking: Double-Blind
Primary Purpose: Treatment
Official Title: Phase 3 Study of Oral Dehydroepiandrosterone (DHEA) in Adults With Myotonic Dystrophy

Resource links provided by NLM:

Drug Information available for: Prasterone
U.S. FDA Resources

Further study details as provided by University of Versailles:

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

Secondary Outcome Measures:
  • 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 ]

Enrollment: 75
Study Start Date: November 2004
Study Completion Date: December 2006
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.

  Eligibility

Ages Eligible for Study:   18 Years to 70 Years
Genders Eligible for Study:   Both
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
Please refer to this study by its ClinicalTrials.gov identifier: NCT00167609

Locations
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
AP-HP
Investigators
Study Chair: Djillali annane, MD, PhD Assistance Publique Hôpitaux de Paris - University of Versailles
Study Director: martine devillers, MD AFM
  More Information

Publications:

ClinicalTrials.gov Identifier: NCT00167609     History of Changes
Other Study ID Numbers: P001108, PS001108
Study First Received: September 10, 2005
Last Updated: April 5, 2010
Health Authority: France: Afssaps - Agence française de sécurité sanitaire des produits de santé (Saint-Denis)

Keywords provided by University of Versailles:
myotonic dystrophy
hypersomnia
dehydroepiandrosterone

Additional relevant 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
Dehydroepiandrosterone
Adjuvants, Immunologic
Immunologic Factors
Physiological Effects of Drugs
Pharmacologic Actions

ClinicalTrials.gov processed this record on June 18, 2013