DMD Genotypes and Motor Function in Duchenne Muscular Dystrophy: A Multi-institution Meta-analysis With Implications for Clinical Trials.
Francesco MuntoniJames SignorovitchGautam SajeevHenry LaneMadeline JenkinsIbrahima DieyeSusan J WardCraig McDonaldNathalie M GoemansErik H NiksBrenda WongLaurent ServaisVolker StraubMichela GuglieriImelda J M de GrootMary ChesshyreCuixia TianAdnan Y ManzurEugenio MercuriAnnemieke Aartsma-RusPublished in: Neurology (2023)
These findings suggest viability of trial designs incorporating genotypically mixed or unmatched controls for up to 12 months in duration for motor function outcomes, which would ease recruitment challenges and reduce numbers of patients assigned to placebos. Such trial designs, including multi-genotype platform trials and hybrid designs, should ensure baseline balance between treatment and control groups for the most important prognostic factors, while accounting for small remaining genotype effects quantified in the present study.
Keyphrases
- prognostic factors
- duchenne muscular dystrophy
- clinical trial
- systematic review
- phase ii
- phase iii
- end stage renal disease
- newly diagnosed
- ejection fraction
- chronic kidney disease
- muscular dystrophy
- peritoneal dialysis
- high throughput
- finite element analysis
- patient reported outcomes
- combination therapy
- insulin resistance
- replacement therapy
- smoking cessation