Efficacy and Safety of DHEA for Myotonic Dystrophy

NCT00167609 | Completed Phase 2 DMC

• 2 other identifiers: P001108PS001108
• Study Type: interventional
• Target: 75
• Locations: 1 country
• Sites: 10

Brief Summary

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

Conditions

Myotonic Dystrophy

Keywords

myotonic dystrophy; hypersomnia; dehydroepiandrosterone

Outcome Measures

Primary Outcomes (1)

• Variation in a Muscle Strength Score between randomization and study week 12

  3 months

Secondary Outcomes (7)

• evaluation of myotonia

  3 months

• Appeal score

  3 months

• Epworth score

  3 months

• Forced vital capacity

  3 months

• arterial blood gas

  3 months

Interventions

dehydroepiandrosterone 100 and 400 mg DRUG

Eligibility Criteria

• Age: 18 Years - 70 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Myotonic dystrophy (genetically proven)
• MDRS score of 3 or 4

You may not qualify if:

• Age \<18 years or \>70 years
• Pregnancy or breastfeeding
• Poor compliance to treatment and follow up
• 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

Sponsors & Collaborators

• University of Versailles: lead
• Association Française contre les Myopathies (AFM), Paris: collaborator
• Assistance Publique - Hôpitaux de Paris: collaborator

Study Sites (10)

CHU

Angers, France

Location

CHU

Bordeaux, France

Location

Raymond Poincaré Hospital

Garches, 92380, France

Location

CHU

Grenoble, France

Location

CHU

Lyon, France

Location

CHU Pouget

Marseille, France

Location

CHU

Nice, France

Location

Institut de myologie

Paris, France

Location

CHU

Strasbourg, France

Location

CHU

Toulouse, France

Location

Related Publications (1)

• 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.

  PMID: 18678823 RESULT

MeSH Terms

Conditions

Myotonic Dystrophy; Disorders of Excessive Somnolence

Condition Hierarchy (Ancestors)

Muscular Dystrophies; Muscular Disorders, Atrophic; Muscular Diseases; Musculoskeletal Diseases; Myotonic Disorders; Heredodegenerative Disorders, Nervous System; Neurodegenerative Diseases; Nervous System Diseases; Neuromuscular Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Sleep Disorders, Intrinsic; Dyssomnias; Sleep Wake Disorders; Mental Disorders

Study Officials

• Djillali annane, MD, PhD

  Assistance Publique Hôpitaux de Paris - University of Versailles

  STUDY CHAIR

• martine devillers, MD

  AFM

  STUDY DIRECTOR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: DOUBLE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER

Study Record Dates

• First Submitted: September 10, 2005
• First Posted: September 14, 2005
• Study Start: November 1, 2004
• Study Completion: December 1, 2006
• Last Updated: April 6, 2010

Record last verified: 2010-04

Locations

FR (10)