Mitsubishi Tanabe Pharma America (MTPA), in collaboration with the Massachusetts General Hospital (MGH), is conducting a study to identify biomarkers related to amyotrophic lateral sclerosis (ALS), which may be helpful to measure patient response to Radicava (edaravone).
They plan to enroll 200 patients at multiple centers across the U.S., who must be new to Radicava and complete a total of six cycles (24 weeks) of treatment. Clinical assessments and several potential ALS biomarkers will be obtained prior to and at the start of the treatment, as well as at specified points over the course of the study.
Patients will be tested for biomarkers addressing a range of ALS-associated parameters: oxidative stress (4-hydroxynonenal, 8-isoprostanes, 3-nitrotyrosine, 8-hydroxy-2′-deoxyguanosine, and urate), inflammation (matrix metalloproteinase-9), nerve cell injury and death (neurofilament heavy and light chain proteins, urinary neurotrophin receptor p75), and muscle injury (creatinine).
Biomarker and disease progression results will be compared with samples stored at biorepositories and progression models, respectively.
They anticipate enrolling the first patient late this spring, and early interim analyses are planned for the end of the year.
“ALS is a complex disorder with diverse pathophysiology, and we do not currently have validated biomarkers for diagnosing or following ALS progression,” James Berry, MD, a neurologist at MGH NCRI and the study’s primary investigator, said in a press release. “This study will broaden our understanding of numerous biomarkers that may be associated with ALS. It will increase our understanding of ALS and of the biological effects of [Radicava] in people with ALS undergoing therapy.”
Radicava is an ALS-specific treatment administered into the vein (intravenously). It was developed and is marketed by MTPA and, in 2017, became the first ALS therapy approved by the U.S. Food and Drug Administration in more than 20 years.
The medicine’s approval was primarily based on favorable efficacy and safety results from a pivotal Phase 3 trial (NCT01492686), which showed that a six-month treatment significantly slowed disability progression in ALS patients, compared with placebo.
“This biomarker trial has sparked exponential interest in the ALS community, and with the help of MGH, we hope to further our understanding of the potential role these measures may have in evaluating a treatment response,” said Stephen Apple, MD, MTPA’s director of medical affairs. “We are honored to be working with the team at MGH on our first clinical trial in the U.S.”