Givinostat in Duchenne Muscular Dystrophy: Natural history comparison applying propensity score matching

Efficacy and safety of Givinostat have been assessed in the Epidys phase 3 study (NCT02851797). DSC/14/2357/51 (NCT03373968) is an ongoing open label, long-term study in 194 DMD subjects enrolled in givinostat studies. 142 subjects from DSC/14/2357/51 were matched to 142 subjects from ImagingDMD (NCT01484678) and CINRG (NCT00468832) natural history studies (control group) using propensity score matching based on baseline results of functional tests and type of steroid.

Twenty-one (14.8%) patients showed Lo4SC in the Givinostat group vs 52 (36.6%) in the control group (HR=0.39 [0.24, 0.65], p<0.001). Median age at Lo4SC was 17.9 years (95% CI 15.65-NE) in the Givinostat group vs 13.9 (95% CI 13.50 14.88) in the control group. Fourty-five (31.7%) patients show LoR in the Givinostat vs 61 (43.0%) in the control group (HR=0.66 (0.45, 0.96), p=0.028) at a median age of 14.9 years (95% CI 13.60-15.97) in the Givinostat group vs 12.9 (95% CI 12.20 14.33) in the control group. Fourteen (9.9%) subjects show LoA in the Givinostat group vs 39 (27.5%) in the ctrl group (HR=0.42 (0.23, 0.76), p=0.004) at a median age of 18.1 years (95%CI 18.09, NE) in the givinostat group vs 15.2 (95%CI 14.70 18.31) in the control group.

These results show that patients treated with givinostat, when compared with matched, steroid-treated controls, delays the occurrence of disease milestones. These results confirm givinostat efficacy demonstrated in Epidys Study with longer-term treatment.

Publication History

Article published online:
11 March 2025