An innovative platform trial
Within an innovative platform design study, we will evaluate the efficacy of multiple promising compounds, all of which have been shown to be safe in patients with ALS and have a high potential for therapeutic efficacy. Patients will be randomized into the three treatment arms based on their genetic profile. The primary aim is to assess the efficacy of the drugs versus placebo on overall survival. A large advantage of this design is that we can decrease the chance of being randomized to placebo, namely in a ratio of 2:1 instead of 1:1. In the future, other compounds may be added to this platform study.
PreLude – Lithium carbonate treatment arm
In the last decade, it has become increasingly clear ALS is not a single disease, but rather a collection of disorders with varying genetic backgrounds but highly similar clinical symptoms. It seems, therefore, increasingly unlikely that we will be able treat all forms of ALS with a single drug. With this in mind, previous findings of lithium as a drug for ALS were analysed again, showing an increased survival probability for UNC13a homozygous patients treated with lithium. This treatment arm aims to confirm the efficacy of lithium in UNC13a homozygous patients.
TUDCA (tauroursodeoxycholic acid) is a small molecule that is being explored for its potential as a treatment for ALSRead more
Within an innovative platform design study, we will evaluate the efficacy of Triumeq, a therapy developed for treatment of HIV that has shown to slow progression in ALSRead more
iAdore (Oral edaravone)
Within an innovative platform design study, we will evaluate the efficacy of oral edaravone (TW001), a formulation that reduces oxidative stressRead more
TRICALS aims to speed up the development of new medicines for ALS. You can help by registering with TRICALS. The participation of patients in clinical trials is necessary to find better treatments.