Clinical trial NCT00167609: Efficacy and Safety of DHEA for Myotonic Dystrophy
Official title: Phase 3 Study of Oral Dehydroepiandrosterone (DHEA) in Adults With Myotonic Dystrophy
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
PHASE3
Conditions: Myotonic Dystrophy
Keywords: myotonic dystrophy
hypersomnia
dehydroepiandrosterone
Lead sponsor: University of Versailles
Sponsor class: OTHER
Collaborators: name: Association Française contre les Myopathies (AFM), Paris
name: Assistance Publique - Hôpitaux de Paris
First posted: 2005-09-14
Enrollment: 75
Sex: ALL
Minimum age (years): 18
Maximum age (years): 70
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

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.

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

Primary outcomes:
measure: Variation in a Muscle Strength Score between randomization and study week 12
timeFrame: 3 months
Secondary outcomes:
measure: evaluation of myotonia
timeFrame: 3 months
measure: Appeal score
timeFrame: 3 months
measure: Epworth score
timeFrame: 3 months
measure: Forced vital capacity
timeFrame: 3 months
measure: arterial blood gas
timeFrame: 3 months
measure: changes in EKG and echocardiography
timeFrame: 3 months
measure: tolerance
timeFrame: 3 months
Other outcomes:
Locations:
city: Angers
country: France
facility: CHU
city: Bordeaux
country: France
facility: CHU
city: Garches
country: France
facility: Raymond Poincaré Hospital
city: Grenoble
country: France
facility: CHU
city: Lyon
country: France
facility: CHU
city: Marseilles
country: France
facility: CHU Pouget
city: Nice
country: France
facility: CHU
city: Paris
country: France
facility: Institut de myologie
city: Strasbourg
country: France
facility: CHU
city: Toulouse
country: France
facility: CHU
Study officials:
affiliation: Assistance Publique Hôpitaux de Paris - University of Versailles
name: Djillali annane, MD, PhD
role: STUDY_CHAIR
affiliation: AFM
name: martine devillers, MD
role: STUDY_DIRECTOR
References:
citation: 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.