Lack of benefit of tamoxifen confirmed in non-marching DMD patients

The results of the second part of the tamoxifen trial focused on non-marching Duchenne muscular dystrophy (DMD) patients, aged between 10 and 16 years, who had not received corticosteroid treatment for at least six months.

• In this group, 8 participants received tamoxifen (20 mg/day) and 6 a placebo for one year.
• The international trial included two French centers (Garches and Strasbourg).
• The primary endpoint was not met: there was no significant difference in motor function (MFM D2 score) between the treatment and placebo groups.

Although well tolerated, tamoxifen is not recommended for the treatment of DMD.

 

Safety and efficacy of tamoxifen in non-ambulant patients with Duchenne muscular dystrophy: a multicentre, randomised, double-blind, placebo-controlled, phase 3 trial (TAMDMD Group B). Henzi BC, Putananickal N, Schmidt S et al. Neuromuscul Disord. 2025 Jan.