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Completed

NCT Number: NCT00167609

Efficacy and Safety of DHEA for Myotonic Dystrophy

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

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Key information

Conditions

Myotonic Dystrophy Congenital, Hereditary, and Neonatal Diseases and Abnormalities Disorders of Excessive Somnolence Dyssomnias Genetic Diseases, Inborn Heredodegenerative Disorders, Nervous System Mental Disorders Muscular Diseases Muscular Disorders, Atrophic Muscular Dystrophies Musculoskeletal Diseases Myotonic Disorders Nervous System Diseases Neurodegenerative Diseases Neuromuscular Diseases Sleep Disorders, Intrinsic Sleep Wake Disorders

Age range

18 year–70 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 2 / Phase 3

Primary location

CHU, Angers, France

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About this study

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.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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

Treatment and study plan

dehydroepiandrosterone 100 and 400 mg

Drug

Primary outcomes

  1. Variation in a Muscle Strength Score between randomization and study week 12

    Time frame: 3 months

Secondary outcomes

  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

Sponsors and collaborators

Lead sponsor

University of Versailles

Other

Collaborators

  • Assistance Publique - Hôpitaux de Paris
  • Association Française contre les Myopathies (AFM), Paris

Registry information

Official study title

Phase 3 Study of Oral Dehydroepiandrosterone (DHEA) in Adults With Myotonic Dystrophy

Important dates

Study start
2004
Study completion
2006
First posted
Sep 14, 2005
Registry last updated
Apr 6, 2010

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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