How clinically meaningful are the differences in the primary outcome between treatment and placebo groups in the CENTAUR trial?
Defining the term "clinically meaningful" proves challenging. Patients involved in the three positive and approved drug trials are unable to detect a slowdown in the progression rate. Attempts were made to gauge ALS providers' opinions regarding a meaningful change in the ALSFRS-R scale, suggesting ...
In the CENTAUR trial, AMX0035 (now called Relyvrio) produced a 25% slowing in the rate of decline in a measure of disability called the Revised ALS Functional Rating Scale (ALSFRS-R). Most patients I have cared for in the past 22 years would find this meaningful (indeed, many patients testified to t...
The differences seen in the CENTAUR trial include a 25% reduction in disease progression rates and prolongation of survival (by about 5 months). These results are clinically meaningful in the context of a rapidly progressive and uniformly fatal disease like ALS.
My concern with the current "3 Rs" is that if each slows the disease's progression by 25-35%, then shouldn't the combined effect result in a 75% slowdown if one takes all three? I don't think so. Realistically, some might respond (whatever that means) to each treatment but not the others. However, o...