Safety and efficacy of fordadistrogene movaparvovec in ambulatory participants with Duchenne muscular dystrophy (CIFFREO): a phase 3, double-blind, randomised, placebo-controlled study - PubMed
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- #Clinical trial
- #Duchenne muscular dystrophy
- #Gene therapy
- Study evaluates fordadistrogene movaparvovec, a gene therapy for Duchenne muscular dystrophy (DMD), in a phase 3 trial.
- Randomized, double-blind, placebo-controlled study with 122 ambulatory male participants aged 4 to <8 years.
- Primary endpoint: Change in North Star Ambulatory Assessment (NSAA) score at week 52 showed no significant difference between treatment and placebo groups.
- Adverse events were more common in the fordadistrogene movaparvovec group (99%) vs. placebo (77%), with vomiting, pyrexia, and decreased appetite being most frequent.
- Serious adverse events occurred in 32% of the treatment group vs. 14% in the placebo group, with no deaths reported.
- The study did not meet its primary efficacy endpoint, leading to discontinuation of further clinical development of fordadistrogene movaparvovec by the sponsor.