The Activate study, conducted by the pharmaceutical company Bial R&D Investments, is a recently-started clinical trial to test a new medication for the treatment of Parkinson’s disease in patients with a GBA1 gene mutation. While participation in this clinical study is very important, it will advance the knowledge of research on Parkinson’s disease, there may be no direct benefit to participants, however this research may benefit others in the future.
Parkinson's disease is the second most common neurodegenerative disorder and the GBA1 gene mutations are identified as a common genetic risk factor for the disease.
Activate is specifically a Phase 2, multi-centre, randomised (computer generated allocation to placebo, drug at 10mg or drug at 60mg), double-blind (the participants and the researchers do not know if you are taking the drug or placebo), placebo-controlled study to evaluate the efficacy, safety, and tolerability of two fixed dose levels of the test drug (10mg and 60mg/day) The study will take place in about 90 study clinics in 11 countries in Europe and North America, and involve more than 200 volunteer subjects, all of whom must have been diagnosed with Parkinson’s disease within the last seven years and have a confirmed GBA1 gene mutation.
Because the symptoms of Parkinson’s disease usually get worse over time, effects will be measured on whether and how the drug can slow down the worsening of the disease.
The investigational medication must be taken daily for 78 weeks (about one year and six months). A smartwatch must worn for six hours per day and while sleeping for the duration of the study. The study team will do regular tests and procedures to check health, symptoms, and how the disease is affecting day-to-day living. You can learn more about the practicalities of taking part in Brendan Hawdon's article in the My Parkinson's tab.
The results of the drug trial are expected during 2025.