COVID-19 Safety Measures Announced for Biomarker Study

Mitsubishi Tanabe Pharma America (MTPA) is taking proactive measures to address patient safety in its REFINE-ALS biomarker study during the COVID-19 pandemic.

The company announced revisions to the study’s protocol that give participants the option to participate virtually, through the use of telemedicine (medical care delivered virtually) and home health agencies instead of hospital or in-office visits.

“In an effort to address the evolving concerns of the ALS community this year, we made it a priority to do what we can to support them during these unprecedented times,” Gustavo A. Suarez Zambrano, MD, MTPA’s vice president of medical affairs, said in a press release.

“With that in mind, we are thankful to be able to offer a potential alternative for patients to be able to participate in the REFINE-ALS study in a virtual setting when in-person assessments are not possible, enabling us to continue fostering scientific knowledge,” Zambrano added.

Sponsored by MTPA and led by Massachusetts General Hospital’s Neurological Clinical Research Institute (NCRI), the observational trial (NCT04259255) is designed to measure the levels of specific biomarkers in up to 300 adults with amyotrophic lateral sclerosis (ALS) beginning treatment with MTPA’s Radicava (edaravone).

Radicava works by removing free radicals, potentially harmful molecules that can cause oxidative stress — an imbalance between the natural production of oxidant molecules and the ability of cells to detoxify them. Of note, oxidative stress is thought to contribute to nerve cell death in ALS.

Participants, being recruited at 39 sites across the U.S., will receive six cycles of Radicava, given directly into the bloodstream, over 24 weeks (nearly six months). Biomarker levels and ALS progression will be assessed before treatment initiation, at the start of the therapy, and at pre-determined time points throughout the treatment period.

Select biomarkers examined in the study include those involved in oxidative stress, inflammation, nerve cell damage or death, and muscle injury. The goal is to identify markers in urine or blood that could be used to predict a patient’s response to treatment.

Specialty laboratories, including Oxford BioDynamics and SomaLogic, are also assisting with the study by providing access to cutting-edge tools that will allow researchers to evaluate a new class of epigenetic biomarkers and to measure changes in up to 5,000 proteins throughout the body. Epigenetic biomarkers represent mechanisms that influence a gene’s activity without altering its underlying DNA sequence.

In addition, participants will be given the choice to receive their genetic results for five genes — SOD1, TARDBP, C9ORF72, FUS, and VCP — whose mutations are associated with ALS. Advanced tele-genetic counseling will be made available for those who choose to receive this information.

“We are grateful to MTPA for their support with these important changes to the REFINE-ALS study, and excited to be able to share with participants more about their genetic makeup in relation to ALS,” said James Berry, MD, the trial’s principal investigator at the NCRI.

“There is much about ALS that we still don’t know, but we’re working toward learning more every day,” he added, noting that “for many patients, participating in a study that evaluates biomarkers is a way of giving back to the ALS community at large.”

Notably, REFINE-ALS represents one of the first clinical studies in ALS where ALS Research Ambassadors — a group of ALS patients and their caregivers — have been proactively involved in the design of a trial.