The European phase III multicentre randomised placebo-controlled TAMDMD trial (NCT03354039) evaluated, from May 2018 to October 2020, 20 mg of tamoxifen daily for 48 weeks in 78 boys with Duchenne muscular dystrophy (DMD), aged 6.5 to 12 years, on corticosteroids.
- Changes in the D1 dimension of the measure of motor function (MFM) were not significantly different between the treated group and the placebo group.
- There was also no significant difference between the two groups in changes in the secondary efficacy endpoints studied (total MFM, dimensions D2 and D3 of the MFM, North Star scale, PUL, timed tests, or fat fraction and T2 on quantitative muscle MRI).
- The trial confirms the good tolerability of tamoxifen, already observed in a phase I trial in Israel.
