Efficacy and Safety of DHEA for Myotonic Dystrophy

Phase 2

Completed

• Conditions: Myotonic Dystrophy

• Registration Number: NCT00167609
• Lead Sponsor: University of Versailles

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 Timeline

• Study Start: 2004-11
• Study First Submitted: 2005-09-10
• Study First Submitted QC: 2005-09-10
• Study First Posted: 2005-09-14
• Study Completion: 2006-12
• Status Verified: 2010-04
• Last Update Submitted: 2010-04-05
• Last Update Posted: 2010-04-06

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 75

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

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Primary Outcome Measures

Name	Time	Method
Variation in a Muscle Strength Score between randomization and study week 12	3 months

Secondary Outcome Measures

Name	Time	Method
evaluation of myotonia	3 months
Appeal score	3 months
Epworth score	3 months
Forced vital capacity	3 months
arterial blood gas	3 months
changes in EKG and echocardiography	3 months
tolerance	3 months

Trial Locations

Locations (4)

Institut de myologie; 🇫🇷 Paris, France

CHU; 🇫🇷 Toulouse, France

Raymond Poincaré Hospital; 🇫🇷 Garches, France

CHU Pouget; 🇫🇷 Marseilles, France