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CompletedNCT00167609Updated Apr 6, 2010

Efficacy and Safety of DHEA for Myotonic Dystrophy

A Phase 2/3 interventional study of dehydroepiandrosterone 100 and 400 mg in Myotonic Dystrophy, sponsored by University of Versailles. Completed at 10 sites in France. Open to participants aged 18 Years to 70 Years. Per ClinicalTrials.gov, last updated 2010-04-06.

Sponsored by University of Versailles · Phase 2/3, Interventional, and Treatment

Phase
Phase 2/3
Study type
Interventional
Enrollment
75
Allocation
Randomized
Ages
18 Years to 70 Years
Sex
All
01

Study summary

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

Read the 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.

02

Conditions studied

  • Myotonic Dystrophy

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Keywords

  • myotonic dystrophy
  • hypersomnia
  • dehydroepiandrosterone
03

In context

Myotonic Dystrophy

125 studies on the registry are indexed under Myotonic Dystrophy; 51 are open to participants now.

This study's enrollment of 75 is above the median of 41 across 66 interventional studies indexed under Myotonic Dystrophy.

Browse Myotonic Dystrophy studies →

Lead sponsor

University of Versailles is the lead sponsor of 14 studies on the registry; none are open to participants now.

Counted across the registry records on this site, refreshed daily.

04

Who can participate

Ages eligible
18 Years to 70 Years
Sexes eligible
All
Accepts healthy volunteers
No

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
05

Study design

Phase
Phase 2 / Phase 3
Primary purpose
Treatment
Allocation
Randomized
Intervention model
Parallel assignment
Masking
Double
Enrollment
75 participants (actual)

Interventions

  • Drugdehydroepiandrosterone 100 and 400 mg
06

What researchers measure

Primary outcomes

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

    Time frame: 3 months

Secondary outcomes

  1. evaluation of myotonia

    Time frame: 3 months

  2. Appeal score

    Time frame: 3 months

  3. Epworth score

    Time frame: 3 months

  4. Forced vital capacity

    Time frame: 3 months

  5. arterial blood gas

    Time frame: 3 months

  6. changes in EKG and echocardiography

    Time frame: 3 months

  7. tolerance

    Time frame: 3 months

07

Study locations

10 sites
  • CHU
    Angers, France
  • CHU
    Bordeaux, France
  • Raymond Poincaré Hospital
    Garches, 92380, France
  • CHU
    Grenoble, France
  • CHU
    Lyon, France
  • CHU Pouget
    Marseilles, France
  • CHU
    Nice, France
  • Institut de myologie
    Paris, France
  • CHU
    Strasbourg, France
  • CHU
    Toulouse, France
08

References and documents

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. PubMed 18678823 ↗
09

Updates

Tracking since Sep 25, 2026
No changes since tracking began. The registry record was last updated on Apr 6, 2010, before this site started recording changes on Sep 25, 2026. Its history is on ClinicalTrials.gov ↗
10

Registry details

Key details

Study ID
NCT00167609
Lead sponsor
University of Versailles
Collaborators
Association Française contre les Myopathies (AFM), Paris, Assistance Publique - Hôpitaux de Paris
First posted
Sep 14, 2005
Start date
Nov 2004
Completion
Dec 2006
Last update
Apr 6, 2010

Study contacts

Djillali annane, MD, PhD
study chair · Assistance Publique Hôpitaux de Paris - University of Versailles
martine devillers, MD
study director · AFM

Oversight

Data monitoring committee
Yes
View the source record on ClinicalTrials.gov ↗

Not currently enrolling

This study is completed, as verified in Apr 2010. You cannot join it, but the record below documents what was studied.

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