The REFINE-ALS study, an effort to identify and measure the levels of specific biomarkers in amyotrophic lateral sclerosis (ALS) patients being treated for a first time with Radicava (edaravone), has enrolled its first participant, Mitsubishi Tanabe Pharma America (MTPA) announced.
This observational trial, sponsored by MTPA — which developed and markets Radicava — is being led by the Neurological Clinical Research Institute (NCRI) at Massachusetts General Hospital (MGH) under a collaborative agreement with the company.
“We are honored to be working with so many esteemed collaborators on this novel study and excited to have reached this milestone of enrolling the first patient,” Stephen Apple, MD, senior medical director of medical affairs at MTPA, said in a press release. “This research has great potential to provide a deeper understanding of the biological effects of Radicava.”
REFINE-ALS will enroll about 300 ALS patients across 40 sites in the U.S., and measure the levels of distinct biomarkers involved in oxidative stress — the cellular damage that occurs as a consequence of high levels of oxidant molecules — and in inflammatory response, neuronal injury or death, and muscle injury.
Specialty laboratories, including Oxford BioDynamics and SomaLogic, are also assisting with the study by giving its researchers access to cutting-edge technology. These tools will allow them to evaluate a new class of epigenetic biomarkers — molecules that determine which genes are ‘on’ and ‘off’, without affecting the genetic sequence of DNA — and to measure changes in up to 5,000 proteins throughout the body.
The study is now enrolling adults, age 18 or older, with sporadic or familial ALS. All must be able to be prescribed Radicava, but to not have used it or only to have taken it for one month before being screened to enter the study. Patients will be given six cycles of Radicava as an intravenous (IV) infusion over 24 weeks, with blood and urine samples taken for analyses.
Biomarker levels and ALS progression will be assessed before initiating treatment, at the start of treatment, and at specific times throughout the study. Biomarkers will be compared with information from biorepositories, while evidence of disease progression will be measured against models for ALS.
Interim results of REFINE-ALS are expected to be announced next year.
“ALS is a multifaceted and complex disease with few treatment options,” said James Berry, MD, MPH, primary investigator of the study at MGH NCRI. “We’re proud to be investigating the biological impact of Radicava on this disease using a combination of genomic analysis and cutting edge biomarker platforms, potentially enhancing our understanding of the disease and its treatments and improving clinical treatment plans for people with ALS.”