CVBF collaborated with IRCSS Istituto Clinico Humanitas and Bruschettini Srl on an article published on the Frontiers in Neurology Journal, entitled “Cox regression and survival analysis from the tauro-urso-deoxycholic trial in amyotrophic lateral sclerosis”.

This research report article regards a post-hoc analysis of a complete dataset of a phase II clinical trial related to the treatment with the tauro-urso-desoxycholic acid (TUDCA) of amyotrophic lateral sclerosis (ALS), a neurodegenerative condition with limited treatment options.

The enrolled participants were adults with defined ALS and were randomized 1:1 to receive 1g of TUDCA daily or placebo by mouth. The study had a total duration of 66 weeks, including an initial period of 12 weeks and a treatment period of 54 weeks. The phase II study (NCT00877604) was conducted in three Italian centers and showed that TUDCA could improve survival and slow functional decline in ALS patients.

The post-hoc analysis was performed to better define the treatment effect and allow comparability with other studies.

It included an assessment of change from baseline and multivariate Cox regression, which is a special technique used in cohort studies to evaluate the relationship between a risk factor and the incidence of a given clinical outcome by correcting for one or more confounding factors. The post-hoc analysis shows that participants in the active arm (participants in a clinical trial receiving a treatment considered effective and most often used) had a 95% lower risk of mortality compared to participants in the placebo arm.

The results of the TUDCA study were also compared with a recent post-hoc analysis of the CENTAUR study (NCT03127514), which tested the combination of tauro-urso-desoxycholic acid (1g twice daily) and NaPB (3g twice daily), founding considerable similarities. However, further phase III studies are necessary to draw significant conclusions.

With a team of skilled biostatisticians and statistical programmers, CVBF provides excellent expertise in study design, data analysis, and reporting to produce accurate and reproducible clinical study results.

The full text manuscript is available here.