Efficacy and Safety of DHEA for Myotonic Dystrophy

• ClinicalTrials.gov Identifier: NCT00167609
• Recruitment Status: Completed
• First Posted: September 14, 2005
• Last Update Posted: April 6, 2010
• Sponsor: University of Versailles
• Collaborators: Association Française contre les Myopathies (AFM), Paris; Assistance Publique - Hôpitaux de Paris
• Information provided by: University of Versailles

Tracking Information

• First Submitted Date: September 10, 2005
• First Posted Date: September 14, 2005
• Last Update Posted Date: April 6, 2010
• Study Start Date: November 2004
• Primary Completion Date: Not Provided
• Current Primary Outcome Measures (submitted: May 8, 2007): Variation in a Muscle Strength Score between randomization and study week 12 [ Time Frame: 3 months ]
• Original Primary Outcome Measures (submitted: September 10, 2005): Variation in a Muscle Strength Score between randomization and study week 12

Current Secondary Outcome Measures (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 (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: 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 Type: Interventional
• Study Phase: Phase 2; Phase 3
• Study Design: Allocation: Randomized; Intervention Model: Parallel Assignment; Masking: Double; Primary Purpose: Treatment
• Condition: Myotonic Dystrophy
• Intervention: Drug: dehydroepiandrosterone 100 and 400 mg
• Study Arms: Not Provided
• Publications *: Penisson-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, Sole G, Hogrel JY, Laforet 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.

Recruitment Information

• Recruitment Status: Completed
• Actual Enrollment (submitted: September 10, 2005): 75
• Original Enrollment: Same as current
• Actual Study Completion Date: December 2006
• Primary Completion Date: Not Provided

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

Sex/Gender

• Sexes Eligible for Study: All

• Ages: 18 Years to 70 Years (Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Contacts: Contact information is only displayed when the study is recruiting subjects
• Listed Location Countries: France

Administrative Information

• NCT Number: NCT00167609
• Other Study ID Numbers: P001108; PS001108
• Has Data Monitoring Committee: Yes
• U.S. FDA-regulated Product: Not Provided
• IPD Sharing Statement: Not Provided
• Current Responsible Party: Not Provided
• Original Responsible Party: Same as current
• Current Study Sponsor: University of Versailles
• Original Study Sponsor: Same as current

Collaborators

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

Investigators

• 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