Enrollment Resumes in Masitinib Trials at Some European Sites
A French national agency has cleared AB Science to resume patient enrollment in the Phase 3 clinical trial investigating masitinib in people with amyotrophic lateral sclerosis (ALS), the company announced in a press release.
AB had voluntarily suspended recruitment and treatment in all its masitinib clinical studies in early June, after data suggested that the medication conferred a potential risk of ischemic heart disease, a condition characterized by recurring chest pain or discomfort.
The go-ahead to resume enrollment in the AB19001 trial (NCT03127267), given by the French National Agency for the Safety of Medicines and Health Products, follows the agency’s approval of certain proposed measures to ensure patient safety. These included the exclusion of patients with a history of severe cardiovascular disease and more monitoring of heart function.
A similar decision has been reached in Norway for the ALS trial. Other affected trials — including a Phase 3 trial in mastocytosis, a rare immune disorder, and a Phase 2 study in moderate-to-severe COVID-19 infection — also are resuming patient enrollment across several European countries.
Masitinib, an oral therapy, is designed to block the activity of several immune cells involved in inflammatory and neurodegenerative processes. It works by inhibiting certain enzymes called tyrosine kinases.
A prior Phase 2/3 trial, AB10015 (NCT02588677), involving 394 adult patients with ALS, showed that using masitinib as an add-on to Rilutek (riluzole) significantly slowed patients’ functional decline by 27%.
While no survival differences were observed in the overall population, researchers found that the therapy significantly extended survival in patients with mild or moderate disability — about 25 months, corresponding to a 44% reduction in the risk of death.
The AB19001 Phase 3 trial now seeks to confirm the findings in a larger population of ALS patients with mild-to-moderate functional impairment.
It will recruit 495 adults, ages 18 to 81, who were diagnosed in the past two years and score at least two points on each item of the ALS Functional Rating Scale-revised (ALSFRS-R).
Participants will be assigned randomly to receive one of two doses of masitinib (4.5 or 6 mg/kg per day), or a placebo, given twice daily for 48 weeks (nearly one year). All will continue to receive a stable dose of Rilutek.
The trial’s main goal is to determine if masitinib lowers the rate of disease progression — defined as change from baseline in ALSFRS-R scores. Measures of quality of life, muscle strength, lung function, and survival will be assessed as secondary goals.