ALS Research Ambassador Collaboration Enabled More Patient-centered Design of REFINE-ALS Trial

ALS Research Ambassador Collaboration Enabled More Patient-centered Design of REFINE-ALS Trial
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Including ALS Research Ambassadors — a group of amyotrophic lateral sclerosis (ALS) patients and their caregivers — in discussions about future clinical trial planning and design facilitates changes in study protocol that make the studies more patient-centered.

That result is shown in the case of the REFINE-ALS trial, which is a collaboration between ALS Ambassadors and Mitsubishi Tanabe Pharma America (MTPA), the company sponsoring REFINE-ALS. The outcome was described in the poster, “Engaging ALS research ambassadors to help design the REFINE-ALS biomarker study,” (see page 264), which was presented at the 30th International Symposium on ALS/MND.

REFINE-ALS is a prospective, observational, longitudinal, multi-center study designed to detect and measure the levels of specific biomarkers among people with ALS being treated for the first time with Radicava (edaravone).

Still enrolling participants, it is expected to include 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.

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All participants will receive six cycles of the therapy for a period of 24 weeks. Biomarker levels and ALS progression will be assessed at three major intervals: before starting the treatment; at the start of therapy; and at specific time points over the course of the 24 weeks.

The goal is to find markers in urine or blood that could be used to predict a patient’s prognosis and their response to treatment.

The ALS Research Ambassadors are a group of ALS patients and caregivers that help encourage participation in clinical trials for people with ALS, and help researchers design clinical trials that incorporate the patient perspective.

The researchers designing REFINE-ALS — including both the doctors spearheading the investigation and the medical team at MTPA — had two meetings with 12 ambassadors.

The ambassadors voiced specific concerns about the trial design, including the frequency of sample collections, participants’ ability to travel, and exclusion criteria that limited participants’ use of other experimental ALS therapies.

“Based on Ambassador feedback, the study protocol was amended to reduce the number of sample collections,” the researchers wrote. “Additional measures were put in place to help address issues of travel logistics, entry criteria, and the use of experimental treatments for ALS.”

According to the researchers, this represents one of the first clinical studies in ALS where ambassadors have been proactively involved in the design of a trial. This collaboration “has been instrumental in making the study design more patient-centered while still maintaining the required scientific rigor,” they said.

“We are pleased to present this additional data, which we hope will provide important insights as we work to increase understanding of ALS and the patient journey,” Atsushi Fujimoto, president of MTPA, said in a press release.

Marisa holds an MS in Cellular and Molecular Pathology from the University of Pittsburgh, where she studied novel genetic drivers of ovarian cancer. She specializes in cancer biology, immunology, and genetics. Marisa began working with BioNews in 2018, and has written about science and health for SelfHacked and the Genetics Society of America. She also writes/composes musicals and coaches the University of Pittsburgh fencing club.
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Inês holds a PhD in Biomedical Sciences from the University of Lisbon, Portugal, where she specialized in blood vessel biology, blood stem cells, and cancer. Before that, she studied Cell and Molecular Biology at Universidade Nova de Lisboa and worked as a research fellow at Faculdade de Ciências e Tecnologias and Instituto Gulbenkian de Ciência. Inês currently works as a Managing Science Editor, striving to deliver the latest scientific advances to patient communities in a clear and accurate manner.
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Marisa holds an MS in Cellular and Molecular Pathology from the University of Pittsburgh, where she studied novel genetic drivers of ovarian cancer. She specializes in cancer biology, immunology, and genetics. Marisa began working with BioNews in 2018, and has written about science and health for SelfHacked and the Genetics Society of America. She also writes/composes musicals and coaches the University of Pittsburgh fencing club.
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