Efficacy and Safety of DHEA for Myotonic Dystrophy

Myotonic Dystrophy Clinical Trial

Official title:

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT00167609
• Other study ID #: P001108
• Secondary ID: PS001108
• Status: Completed
• Phase: Phase 2/Phase 3
• First received: September 10, 2005
• Last updated: April 5, 2010
• Start date: November 2004
• Est. completion date: December 2006

Study information

• Verified date: April 2010
• Source: University of Versailles
• Contact: n/a
• Is FDA regulated: No
• Health authority: France: Afssaps - Agence française de sécurité sanitaire des produits de santé (Saint-Denis)
• Study type: Interventional

Clinical Trial Summary

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

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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 75
• Est. completion date: December 2006
• Accepts healthy volunteers: No
• Gender: Both
• Age group: 18 Years to 70 Years

Eligibility

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

Allocation: Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Parallel Assignment, Masking: Double-Blind, Primary Purpose: Treatment

Related Conditions & MeSH terms

• Myotonic Dystrophy

Intervention

Drug:
• dehydroepiandrosterone 100 and 400 mg

Locations

Country	Name	City	State
France	CHU	Angers
France	CHU	Bordeaux
France	Raymond Poincaré Hospital	Garches
France	CHU	Grenoble
France	CHU	Lyon
France	CHU Pouget	Marseilles
France	CHU	Nice
France	Institut de myologie	Paris
France	CHU	Strasbourg
France	CHU	Toulouse

Sponsors (3)

Lead Sponsor	Collaborator
University of Versailles	Assistance Publique - Hôpitaux de Paris, Association Française contre les Myopathies (AFM), Paris

Country where clinical trial is conducted

France, 

References & Publications (1)

Pénisson-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, Solé G, Hogrel JY, Laforêt P, Vial C, Vila AL, Sacconi S, Pouget J, Eymard B, Chevret — View Citation

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Variation in a Muscle Strength Score between randomization and study week 12		3 months
Secondary	evaluation of myotonia		3 months
Secondary	Appeal score		3 months
Secondary	Epworth score		3 months
Secondary	Forced vital capacity		3 months
Secondary	arterial blood gas		3 months
Secondary	changes in EKG and echocardiography		3 months
Secondary	tolerance		3 months