TUDCA trial did not meet its endpoints

April 4, 2024 – An announcement of the top-line results from a European phase 3 clinical trial of TUDCA revealed that the primary endpoint of the study was not met as TUDCA failed to demonstrate an ability to significantly alter the progression of ALS as measured by the ALS Functional Rating Scale – Revised (ALSFRS-R). Additional measurements (secondary endpoints) were also unable to demonstrate a benefit of TUDCA. These included an evaluation of effect on survival and key biomarkers including neurofilament light chain (NfL), and several other analyses.

The TUDCA-ALS phase 3 clinical trial was a randomized, double-blind, placebo-controlled study, testing a regimen of four oral capsules (250 mg), twice daily in 336 participants over 18 months. It was an academic (also referred to as investigator-initiated) trial, run by the TUDCA-ALS consortium of UK and European ALS clinicians, which means it was not sponsored by a pharmaceutical company. The consortium has stated that they will be exploring further statistical analyses to better understand if there was any potential benefit of TUDCA in a subgroup of individuals, and it is anticipated that some of this data will be presented in June at the European Network for the Cure of ALS (ENCALS) meeting in Sweden. The ALS Society of Canada will keep the community updated as more information becomes available to share.

TUDCA or tauroursodeoxycholic acid (also referred to as TURSO) is also one of the two drugs compounded in Albrioza (also referred to as Relyvrio and AMX0035), which was recently announced to have failed an effect on primary and secondary outcome measures in a phase 3 clinical trial.

For anyone interested in discussing TUDCA use, it is recommended to speak to your clinician.