Efficacy and Safety of DHEA for Myotonic Dystrophy

• Register: ClinicalTrials.gov
• Last refreshed on: 19 February 2015
• Main ID: NCT00167609
• Date of registration: 10/09/2005
• Prospective Registration: No
• Primary sponsor: University of Versailles
• Public title: Efficacy and Safety of DHEA for Myotonic Dystrophy
• Scientific title: Phase 3 Study of Oral Dehydroepiandrosterone (DHEA) in Adults With Myotonic Dystrophy
• Date of first enrolment: November 2004
• Target sample size: 75
• Recruitment status: Completed
• URL: http://clinicaltrials.gov/show/NCT00167609
• Study type: Interventional
• Study design: Allocation: Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Parallel Assignment, Masking: Double-Blind, Primary Purpose: Treatment
• Phase: Phase 2/Phase 3

Countries of recruitment

France

Contacts

• Name: Djillali annane, MD, PhD
• Affiliation: Assistance Publique Hôpitaux de Paris - University of Versailles

• Name: martine devillers, MD
• Affiliation: AFM

Key inclusion & exclusion 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



Age minimum: 18 Years
Age maximum: 70 Years
Gender: Both

Health Condition(s) or Problem(s) studied

Myotonic Dystrophy

Intervention(s)

Drug: dehydroepiandrosterone 100 and 400 mg

Primary Outcome(s)

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

Secondary Outcome(s)

Appeal score [Time Frame: 3 months]

arterial blood gas [Time Frame: 3 months]

evaluation of myotonia [Time Frame: 3 months]

Forced vital capacity [Time Frame: 3 months]

tolerance [Time Frame: 3 months]

changes in EKG and echocardiography [Time Frame: 3 months]

Epworth score [Time Frame: 3 months]

Secondary ID(s)

P001108

PS001108

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Secondary Sponsor(s)

Association Française contre les Myopathies (AFM), Paris

AP-HP