Efficacy and Safety of DHEA for Myotonic Dystrophy - Full Text View

Sponsors and Collaborators:	University of Versailles Association Française de Lutte contre la Myopathie AP-HP
Information provided by:	University of Versailles
ClinicalTrials.gov Identifier:	NCT00167609

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 II Phase III

Genetics Home Reference related topics: myotonic dystrophy potassium-aggravated myotonia

Drug Information available for: Prasterone Dehydroepiandrosterone sulfate

U.S. FDA Resources

Study Type:	Interventional
Study Design:	Treatment, Randomized, Double-Blind, Placebo Control, Parallel Assignment, Safety/Efficacy Study
Official Title:	Phase 3 Study of Oral Dehydroepiandrosterone (DHEA) in Adults With Myotonic Dystrophy

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
Raymond Poincaré Hospital
Garches, France, 92380
CHU
Angers, France
CHU
Toulouse, France
CHU
Nice, France
Institut de myologie
Paris, France
CHU
Strasbourg, France
CHU Pouget
Marseilles, France
CHU
Lyon, France
CHU
Grenoble, France
CHU
Bordeaux, France

Sponsors and Collaborators

University of Versailles

Association Française de Lutte contre la Myopathie

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

No publications provided

Study ID Numbers:	P001108, PS001108
Study First Received:	September 10, 2005
Last Updated:	April 28, 2008
ClinicalTrials.gov Identifier:	NCT00167609 History of Changes
Health Authority:	France: Afssaps - French Health Products Safety Agency

Keywords provided by University of Versailles:

myotonic dystrophy
hypersomnia
dehydroepiandrosterone

Study placed in the following topic categories:

Immunologic Factors
Adjuvants, Immunologic
Dehydroepiandrosterone
Myotonic Disorders
Neurodegenerative Diseases
Muscular Dystrophies
Muscular Diseases
Heredodegenerative Disorders, Nervous System
Muscular Disorders, Atrophic

Musculoskeletal Diseases
Neuromuscular Diseases
Genetic Diseases, Inborn
Myotonic Dystrophy
Atrophy
Myotonic Dystrophy 1
Myotonia Atrophica
Muscular Dystrophy

Additional relevant MeSH terms:

Immunologic Factors
Physiological Effects of Drugs
Nervous System Diseases
Adjuvants, Immunologic
Dehydroepiandrosterone
Myotonic Disorders
Neurodegenerative Diseases
Pharmacologic Actions

Muscular Dystrophies
Muscular Diseases
Heredodegenerative Disorders, Nervous System
Muscular Disorders, Atrophic
Musculoskeletal Diseases
Neuromuscular Diseases
Genetic Diseases, Inborn
Myotonic Dystrophy

ClinicalTrials.gov processed this record on May 07, 2009