Dehydroepiandrosterone and DHEA (Dehydroepiandrosterone) and dehydroepiandrosterone 100 and 400 mg in Myotonic Dystrophy - Clinical Trials Registry

Study Overview

Status

Completed

Conditions

Myotonic Dystrophy

Intervention / Treatment

Drug: dehydroepiandrosterone 100 and 400 mg

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 Type

Interventional

Enrollment (Actual)

75

Phase

Phase 2
Phase 3

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

France
- - Angers, France
    - CHU
  - Bordeaux, France
    - CHU
  - Garches, France, 92380
    - Raymond Poincaré Hospital
  - Grenoble, France
    - CHU
  - Lyon, France
    - CHU
  - Marseilles, France
    - CHU Pouget
  - Nice, France
    - CHU
  - Paris, France
    - Institut de Myologie
  - Strasbourg, France
    - CHU
  - Toulouse, France
    - CHU

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

18 years to 70 years (Adult, Older Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

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 Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

Primary Purpose: Treatment
Allocation: Randomized
Interventional Model: Parallel Assignment
Masking: Double

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Time Frame
Variation in a Muscle Strength Score between randomization and study week 12 Time Frame: 3 months	3 months

Secondary Outcome Measures

Outcome Measure	Time Frame
evaluation of myotonia Time Frame: 3 months	3 months
Appeal score Time Frame: 3 months	3 months
Epworth score Time Frame: 3 months	3 months
Forced vital capacity Time Frame: 3 months	3 months
arterial blood gas Time Frame: 3 months	3 months
changes in EKG and echocardiography Time Frame: 3 months	3 months
tolerance Time Frame: 3 months	3 months

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

University of Versailles

Collaborators

Assistance Publique - Hôpitaux de Paris

Association Française contre les Myopathies (AFM), Paris

Investigators

Study Director: martine devillers, MD, AFM

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General 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.

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start

November 1, 2004

Study Completion (Actual)

December 1, 2006

Study Registration Dates

First Submitted

September 10, 2005

First Submitted That Met QC Criteria