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Efficacy and Safety of DHEA for Myotonic Dystrophy

Primary Purpose

Myotonic Dystrophy

Status
Completed
Phase
Phase 2
Locations
France
Study Type
Interventional
Intervention
dehydroepiandrosterone 100 and 400 mg
Sponsored by
University of Versailles
About
Eligibility
Locations
Outcomes
Full info

About this trial

This is an interventional treatment trial for Myotonic Dystrophy focused on measuring myotonic dystrophy, hypersomnia, dehydroepiandrosterone

Eligibility Criteria

18 Years - 70 Years (Adult, Older Adult)All SexesDoes not accept healthy volunteers

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

Sites / Locations

  • CHU
  • CHU
  • Raymond Poincaré Hospital
  • CHU
  • CHU
  • CHU Pouget
  • CHU
  • Institut de myologie
  • CHU
  • CHU

Outcomes

Primary Outcome Measures

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

Secondary Outcome Measures

evaluation of myotonia
Appeal score
Epworth score
Forced vital capacity
arterial blood gas
changes in EKG and echocardiography
tolerance

Full Information

First Posted
September 10, 2005
Last Updated
April 5, 2010
Sponsor
University of Versailles
Collaborators
Association Française contre les Myopathies (AFM), Paris, Assistance Publique - Hôpitaux de Paris
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1. Study Identification

Unique Protocol Identification Number
NCT00167609
Brief Title
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

2. Study Status

Record Verification Date
April 2010
Overall Recruitment Status
Completed
Study Start Date
November 2004 (undefined)
Primary Completion Date
undefined (undefined)
Study Completion Date
December 2006 (Actual)

3. Sponsor/Collaborators

Name of the Sponsor
University of Versailles
Collaborators
Association Française contre les Myopathies (AFM), Paris, Assistance Publique - Hôpitaux de Paris

4. Oversight

Data Monitoring Committee
Yes

5. Study Description

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Myotonic Dystrophy
Keywords
myotonic dystrophy, hypersomnia, dehydroepiandrosterone

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 2, Phase 3
Interventional Study Model
Parallel Assignment
Masking
Double
Allocation
Randomized
Enrollment
75 (Actual)

8. Arms, Groups, and Interventions

Intervention Type
Drug
Intervention Name(s)
dehydroepiandrosterone 100 and 400 mg
Primary Outcome Measure Information:
Title
Variation in a Muscle Strength Score between randomization and study week 12
Time Frame
3 months
Secondary Outcome Measure Information:
Title
evaluation of myotonia
Time Frame
3 months
Title
Appeal score
Time Frame
3 months
Title
Epworth score
Time Frame
3 months
Title
Forced vital capacity
Time Frame
3 months
Title
arterial blood gas
Time Frame
3 months
Title
changes in EKG and echocardiography
Time Frame
3 months
Title
tolerance
Time Frame
3 months

10. Eligibility

Sex
All
Minimum Age & Unit of Time
18 Years
Maximum Age & Unit of Time
70 Years
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
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Djillali annane, MD, PhD
Organizational Affiliation
Assistance Publique Hôpitaux de Paris - University of Versailles
Official's Role
Study Chair
First Name & Middle Initial & Last Name & Degree
martine devillers, MD
Organizational Affiliation
AFM
Official's Role
Study Director
Facility Information:
Facility Name
CHU
City
Angers
Country
France
Facility Name
CHU
City
Bordeaux
Country
France
Facility Name
Raymond Poincaré Hospital
City
Garches
ZIP/Postal Code
92380
Country
France
Facility Name
CHU
City
Grenoble
Country
France
Facility Name
CHU
City
Lyon
Country
France
Facility Name
CHU Pouget
City
Marseilles
Country
France
Facility Name
CHU
City
Nice
Country
France
Facility Name
Institut de myologie
City
Paris
Country
France
Facility Name
CHU
City
Strasbourg
Country
France
Facility Name
CHU
City
Toulouse
Country
France

12. IPD Sharing Statement

Citations:
PubMed Identifier
18678823
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.
Results Reference
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Efficacy and Safety of DHEA for Myotonic Dystrophy

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