Efficacy and Safety of DHEA for Myotonic Dystrophy

Phase 3 Study of Oral Dehydroepiandrosterone (DHEA) in Adults With Myotonic Dystrophy

Status

Completed

Phases

Phase 2Phase 3

Study type

Interventional

Source

ClinicalTrials.gov

Registry ID

NCT00167609

Enrollment

Registered

2005-09-14

Start date

2004-11-30

Completion date

2006-12-31

Last updated

2010-04-06

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Myotonic Dystrophy

Keywords

myotonic dystrophy, hypersomnia, dehydroepiandrosterone

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.

Interventions

DRUGdehydroepiandrosterone 100 and 400 mg

Study design

Allocation

RANDOMIZED

Intervention model

PARALLEL

Primary purpose

TREATMENT

Masking

DOUBLE

Eligibility

Sex/Gender

ALL

Age

18 Years to 70 Years

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

Design outcomes

Primary

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

Secondary

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

Countries

France

Outcome results

None listed

Source: ClinicalTrials.gov · Data processed: Apr 5, 2026