Efficacy and Safety of DHEA for Myotonic Dystrophy

Phase 2/3 Completed 75 enrolled University of Versailles Assistance Publique - Hôpitaux de Paris · collab Association Française contre les Myopathies (AFM), Paris · collab

RandomizedParallel-groupDouble-blindTreatment

Summary

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

Timeline

Start: 2004-11
Primary completion: —
Completion: 2006-12

Outcome

Missed primary endpoint

paper There were no differences between placebo and combined DHEA groups (p = 0.34) PMID 18678823 ↗

paper There is no evidence that a 12-week treatment with replacement or pharmacologic doses of dehydroepiandrosterone improves muscle strength PMID 18678823 ↗

Publications

Results 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.

Drugs

Evaluation	Drug	Modality	Dose	Route
Subject	Dehydroepiandrosterone	Other / unclassified	100 mg	Oral
Subject	Dehydroepiandrosterone	Other / unclassified	400 mg	Oral